practical approach to a bleeding child peri kamalakar,md director the valerie fund children’s...
TRANSCRIPT
Practical approach to a Practical approach to a bleeding childbleeding child
Peri KamalakarMDPeri KamalakarMDDirectorDirector
The Valerie Fund Childrenrsquos Centers The Valerie Fund Childrenrsquos Centers For Cancer ampBlood DisordersFor Cancer ampBlood Disorders
AtAtSaint Barnabas Health Care SystemSaint Barnabas Health Care System
Associate Director Hemophilia Center Associate Director Hemophilia Center Newark Beth israel Medical CenterNewark Beth israel Medical Center
Practical approach to a bleeding Practical approach to a bleeding childchild
OBJECTIVESOBJECTIVESbull Overview of hemostasisOverview of hemostasis
bull Clinical approach in making a diagnosisClinical approach in making a diagnosis
bull Review the most common bleeding conditionsReview the most common bleeding conditions
bull Discuss the current treatment strategiesDiscuss the current treatment strategies
Overview of HaemostasisOverview of Haemostasis
INJURY
Collagen Exposure
Platelet Adhesion and release reaction
Platelet aggregation
VASOCONSTRICTION
Serotonin Platelet Phospolipid
Thromboxane A2 ADP
Primary haemostatic plug
Stable haemostatic plug
Tissue Factor
Coagulation
Thrombin
Fibrin
Fibrinolysis
Overview of HaemostasisOverview of Haemostasis
INJURY
Collagen Exposure
Platelet Adhesion and release reaction
Platelet aggregation
VASOCONSTRICTION
Serotonin Platelet Phospolipid
Thromboxane A2 ADP
Primary haemostatic plug
Stable haemostatic plug
Tissue Factor
Coagulation
Thrombin
Fibrin
Fibrinolysis
Kinins HMW Kininogen
Kallikrein Contact Activation
XII Prekallikrein
XIIa
XIa XI
IXa Ca++ IX
VIIIa VIII
Ca++
Phospholipid
Intrinsic Pathway
Xa X
Va V
Ca++
Phospholipid XIII
II IIa
XIIa
Fibrinogen Fibrin XIII
Common Pathway
VII VIIa
Ca++
Tissue Factor
Coagulation cascadeCoagulation cascade
Extrinsic Pathway
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull HISTORY ndash HISTORY ndash HISTORY ndashHISTORY ndash HISTORY HISTORY
gtgtAGE OF ONSETAGE OF ONSET
gt SEXgt SEX
gtFREQUENCY gtFREQUENCY
gtLOCATION TYPE OF BLEEDINGgtLOCATION TYPE OF BLEEDING
gtDURATION OF BLEEDINGgtDURATION OF BLEEDING
gt MEDICATIONSgt MEDICATIONS
gt ASSOCIATED SYMPTOMS gt ASSOCIATED SYMPTOMS
gt REVIEW OF SYSTEMSgt REVIEW OF SYSTEMS
Approach to a bleeding Approach to a bleeding patientpatient
ndash What is the type of bleeding disorderWhat is the type of bleeding disorder
Primary hemostasis ndash Vascular causesPrimary hemostasis ndash Vascular causes
Platelets-Number vsPlatelets-Number vs
Function Function
Fibrin formation ndash clotting factorsFibrin formation ndash clotting factors
Premature clot dissolution- post clot Premature clot dissolution- post clot formationformation
Approach to a bleeding Approach to a bleeding patientpatient
ndash Is a bleeding tendency presentIs a bleeding tendency present
Easy BruisingEasy Bruising
Mucosal bleedingMucosal bleeding
MenorrhagiaMenorrhagia
Surgical Hemorrhage ndash Procedure Surgical Hemorrhage ndash Procedure vsDiathesisvsDiathesis
Postpartum HemorrhagePostpartum Hemorrhage
Joint and Muscle bleed ndashSeverity of Joint and Muscle bleed ndashSeverity of trauamatrauama
Approach to a bleeding Approach to a bleeding patientpatient
ndashIs the disorder Familial or Is the disorder Familial or AcquiredAcquired
Family history ndash MOTHER amp OTHER Family history ndash MOTHER amp OTHER FEMALE MEMBERS IN THE FEMALE MEMBERS IN THE IMMEDIATE FAMILY ndash IMMEDIATE FAMILY ndash Detailed Detailed Menstrual historyMenstrual history
bull Vascular causes ndashVascular causes ndash
First and foremost rule out First and foremost rule out infectious causes ndash ldquoinfectious causes ndash ldquoMeningococcemiardquoMeningococcemiardquo
Vasculitis ndash Henoch-Schonlein PurpuraVasculitis ndash Henoch-Schonlein Purpura
Hemangiomas- Kassalback-Merritt Hemangiomas- Kassalback-Merritt syndromesyndrome
bull Petechiae and PurpuraPetechiae and Purpurabull 1048708 1048708 InfectiousInfectiousbull ndash ndash MeningococcemiaMeningococcemiabull ndash ndash Rocky mountain spottedRocky mountain spottedbull feverfeverbull ndash ndash Group A strepGroup A strepbull ndash ndash Atypical measlesAtypical measlesbull ndash ndash Echovirus 9 4 7Echovirus 9 4 7bull ndash ndash Epstein-Barr virusEpstein-Barr virusbull ndash ndash Coxsackie virus A9Coxsackie virus A9
bull 1048708 1048708 Non-infectiousNon-infectious
bull ndash ndash Normal plateletsNormal platelets
bull 1048708 1048708 HSPHSP
bull 1048708 1048708 Coagulation disordersCoagulation disorders
bull 1048708 1048708 TraumaTrauma
bull ndash ndash Low plateletsLow platelets
bull 1048708 1048708 ITPITP
bull 1048708 1048708 LeukemiaLeukemia
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Practical approach to a bleeding Practical approach to a bleeding childchild
OBJECTIVESOBJECTIVESbull Overview of hemostasisOverview of hemostasis
bull Clinical approach in making a diagnosisClinical approach in making a diagnosis
bull Review the most common bleeding conditionsReview the most common bleeding conditions
bull Discuss the current treatment strategiesDiscuss the current treatment strategies
Overview of HaemostasisOverview of Haemostasis
INJURY
Collagen Exposure
Platelet Adhesion and release reaction
Platelet aggregation
VASOCONSTRICTION
Serotonin Platelet Phospolipid
Thromboxane A2 ADP
Primary haemostatic plug
Stable haemostatic plug
Tissue Factor
Coagulation
Thrombin
Fibrin
Fibrinolysis
Overview of HaemostasisOverview of Haemostasis
INJURY
Collagen Exposure
Platelet Adhesion and release reaction
Platelet aggregation
VASOCONSTRICTION
Serotonin Platelet Phospolipid
Thromboxane A2 ADP
Primary haemostatic plug
Stable haemostatic plug
Tissue Factor
Coagulation
Thrombin
Fibrin
Fibrinolysis
Kinins HMW Kininogen
Kallikrein Contact Activation
XII Prekallikrein
XIIa
XIa XI
IXa Ca++ IX
VIIIa VIII
Ca++
Phospholipid
Intrinsic Pathway
Xa X
Va V
Ca++
Phospholipid XIII
II IIa
XIIa
Fibrinogen Fibrin XIII
Common Pathway
VII VIIa
Ca++
Tissue Factor
Coagulation cascadeCoagulation cascade
Extrinsic Pathway
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull HISTORY ndash HISTORY ndash HISTORY ndashHISTORY ndash HISTORY HISTORY
gtgtAGE OF ONSETAGE OF ONSET
gt SEXgt SEX
gtFREQUENCY gtFREQUENCY
gtLOCATION TYPE OF BLEEDINGgtLOCATION TYPE OF BLEEDING
gtDURATION OF BLEEDINGgtDURATION OF BLEEDING
gt MEDICATIONSgt MEDICATIONS
gt ASSOCIATED SYMPTOMS gt ASSOCIATED SYMPTOMS
gt REVIEW OF SYSTEMSgt REVIEW OF SYSTEMS
Approach to a bleeding Approach to a bleeding patientpatient
ndash What is the type of bleeding disorderWhat is the type of bleeding disorder
Primary hemostasis ndash Vascular causesPrimary hemostasis ndash Vascular causes
Platelets-Number vsPlatelets-Number vs
Function Function
Fibrin formation ndash clotting factorsFibrin formation ndash clotting factors
Premature clot dissolution- post clot Premature clot dissolution- post clot formationformation
Approach to a bleeding Approach to a bleeding patientpatient
ndash Is a bleeding tendency presentIs a bleeding tendency present
Easy BruisingEasy Bruising
Mucosal bleedingMucosal bleeding
MenorrhagiaMenorrhagia
Surgical Hemorrhage ndash Procedure Surgical Hemorrhage ndash Procedure vsDiathesisvsDiathesis
Postpartum HemorrhagePostpartum Hemorrhage
Joint and Muscle bleed ndashSeverity of Joint and Muscle bleed ndashSeverity of trauamatrauama
Approach to a bleeding Approach to a bleeding patientpatient
ndashIs the disorder Familial or Is the disorder Familial or AcquiredAcquired
Family history ndash MOTHER amp OTHER Family history ndash MOTHER amp OTHER FEMALE MEMBERS IN THE FEMALE MEMBERS IN THE IMMEDIATE FAMILY ndash IMMEDIATE FAMILY ndash Detailed Detailed Menstrual historyMenstrual history
bull Vascular causes ndashVascular causes ndash
First and foremost rule out First and foremost rule out infectious causes ndash ldquoinfectious causes ndash ldquoMeningococcemiardquoMeningococcemiardquo
Vasculitis ndash Henoch-Schonlein PurpuraVasculitis ndash Henoch-Schonlein Purpura
Hemangiomas- Kassalback-Merritt Hemangiomas- Kassalback-Merritt syndromesyndrome
bull Petechiae and PurpuraPetechiae and Purpurabull 1048708 1048708 InfectiousInfectiousbull ndash ndash MeningococcemiaMeningococcemiabull ndash ndash Rocky mountain spottedRocky mountain spottedbull feverfeverbull ndash ndash Group A strepGroup A strepbull ndash ndash Atypical measlesAtypical measlesbull ndash ndash Echovirus 9 4 7Echovirus 9 4 7bull ndash ndash Epstein-Barr virusEpstein-Barr virusbull ndash ndash Coxsackie virus A9Coxsackie virus A9
bull 1048708 1048708 Non-infectiousNon-infectious
bull ndash ndash Normal plateletsNormal platelets
bull 1048708 1048708 HSPHSP
bull 1048708 1048708 Coagulation disordersCoagulation disorders
bull 1048708 1048708 TraumaTrauma
bull ndash ndash Low plateletsLow platelets
bull 1048708 1048708 ITPITP
bull 1048708 1048708 LeukemiaLeukemia
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Overview of HaemostasisOverview of Haemostasis
INJURY
Collagen Exposure
Platelet Adhesion and release reaction
Platelet aggregation
VASOCONSTRICTION
Serotonin Platelet Phospolipid
Thromboxane A2 ADP
Primary haemostatic plug
Stable haemostatic plug
Tissue Factor
Coagulation
Thrombin
Fibrin
Fibrinolysis
Overview of HaemostasisOverview of Haemostasis
INJURY
Collagen Exposure
Platelet Adhesion and release reaction
Platelet aggregation
VASOCONSTRICTION
Serotonin Platelet Phospolipid
Thromboxane A2 ADP
Primary haemostatic plug
Stable haemostatic plug
Tissue Factor
Coagulation
Thrombin
Fibrin
Fibrinolysis
Kinins HMW Kininogen
Kallikrein Contact Activation
XII Prekallikrein
XIIa
XIa XI
IXa Ca++ IX
VIIIa VIII
Ca++
Phospholipid
Intrinsic Pathway
Xa X
Va V
Ca++
Phospholipid XIII
II IIa
XIIa
Fibrinogen Fibrin XIII
Common Pathway
VII VIIa
Ca++
Tissue Factor
Coagulation cascadeCoagulation cascade
Extrinsic Pathway
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull HISTORY ndash HISTORY ndash HISTORY ndashHISTORY ndash HISTORY HISTORY
gtgtAGE OF ONSETAGE OF ONSET
gt SEXgt SEX
gtFREQUENCY gtFREQUENCY
gtLOCATION TYPE OF BLEEDINGgtLOCATION TYPE OF BLEEDING
gtDURATION OF BLEEDINGgtDURATION OF BLEEDING
gt MEDICATIONSgt MEDICATIONS
gt ASSOCIATED SYMPTOMS gt ASSOCIATED SYMPTOMS
gt REVIEW OF SYSTEMSgt REVIEW OF SYSTEMS
Approach to a bleeding Approach to a bleeding patientpatient
ndash What is the type of bleeding disorderWhat is the type of bleeding disorder
Primary hemostasis ndash Vascular causesPrimary hemostasis ndash Vascular causes
Platelets-Number vsPlatelets-Number vs
Function Function
Fibrin formation ndash clotting factorsFibrin formation ndash clotting factors
Premature clot dissolution- post clot Premature clot dissolution- post clot formationformation
Approach to a bleeding Approach to a bleeding patientpatient
ndash Is a bleeding tendency presentIs a bleeding tendency present
Easy BruisingEasy Bruising
Mucosal bleedingMucosal bleeding
MenorrhagiaMenorrhagia
Surgical Hemorrhage ndash Procedure Surgical Hemorrhage ndash Procedure vsDiathesisvsDiathesis
Postpartum HemorrhagePostpartum Hemorrhage
Joint and Muscle bleed ndashSeverity of Joint and Muscle bleed ndashSeverity of trauamatrauama
Approach to a bleeding Approach to a bleeding patientpatient
ndashIs the disorder Familial or Is the disorder Familial or AcquiredAcquired
Family history ndash MOTHER amp OTHER Family history ndash MOTHER amp OTHER FEMALE MEMBERS IN THE FEMALE MEMBERS IN THE IMMEDIATE FAMILY ndash IMMEDIATE FAMILY ndash Detailed Detailed Menstrual historyMenstrual history
bull Vascular causes ndashVascular causes ndash
First and foremost rule out First and foremost rule out infectious causes ndash ldquoinfectious causes ndash ldquoMeningococcemiardquoMeningococcemiardquo
Vasculitis ndash Henoch-Schonlein PurpuraVasculitis ndash Henoch-Schonlein Purpura
Hemangiomas- Kassalback-Merritt Hemangiomas- Kassalback-Merritt syndromesyndrome
bull Petechiae and PurpuraPetechiae and Purpurabull 1048708 1048708 InfectiousInfectiousbull ndash ndash MeningococcemiaMeningococcemiabull ndash ndash Rocky mountain spottedRocky mountain spottedbull feverfeverbull ndash ndash Group A strepGroup A strepbull ndash ndash Atypical measlesAtypical measlesbull ndash ndash Echovirus 9 4 7Echovirus 9 4 7bull ndash ndash Epstein-Barr virusEpstein-Barr virusbull ndash ndash Coxsackie virus A9Coxsackie virus A9
bull 1048708 1048708 Non-infectiousNon-infectious
bull ndash ndash Normal plateletsNormal platelets
bull 1048708 1048708 HSPHSP
bull 1048708 1048708 Coagulation disordersCoagulation disorders
bull 1048708 1048708 TraumaTrauma
bull ndash ndash Low plateletsLow platelets
bull 1048708 1048708 ITPITP
bull 1048708 1048708 LeukemiaLeukemia
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Overview of HaemostasisOverview of Haemostasis
INJURY
Collagen Exposure
Platelet Adhesion and release reaction
Platelet aggregation
VASOCONSTRICTION
Serotonin Platelet Phospolipid
Thromboxane A2 ADP
Primary haemostatic plug
Stable haemostatic plug
Tissue Factor
Coagulation
Thrombin
Fibrin
Fibrinolysis
Kinins HMW Kininogen
Kallikrein Contact Activation
XII Prekallikrein
XIIa
XIa XI
IXa Ca++ IX
VIIIa VIII
Ca++
Phospholipid
Intrinsic Pathway
Xa X
Va V
Ca++
Phospholipid XIII
II IIa
XIIa
Fibrinogen Fibrin XIII
Common Pathway
VII VIIa
Ca++
Tissue Factor
Coagulation cascadeCoagulation cascade
Extrinsic Pathway
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull HISTORY ndash HISTORY ndash HISTORY ndashHISTORY ndash HISTORY HISTORY
gtgtAGE OF ONSETAGE OF ONSET
gt SEXgt SEX
gtFREQUENCY gtFREQUENCY
gtLOCATION TYPE OF BLEEDINGgtLOCATION TYPE OF BLEEDING
gtDURATION OF BLEEDINGgtDURATION OF BLEEDING
gt MEDICATIONSgt MEDICATIONS
gt ASSOCIATED SYMPTOMS gt ASSOCIATED SYMPTOMS
gt REVIEW OF SYSTEMSgt REVIEW OF SYSTEMS
Approach to a bleeding Approach to a bleeding patientpatient
ndash What is the type of bleeding disorderWhat is the type of bleeding disorder
Primary hemostasis ndash Vascular causesPrimary hemostasis ndash Vascular causes
Platelets-Number vsPlatelets-Number vs
Function Function
Fibrin formation ndash clotting factorsFibrin formation ndash clotting factors
Premature clot dissolution- post clot Premature clot dissolution- post clot formationformation
Approach to a bleeding Approach to a bleeding patientpatient
ndash Is a bleeding tendency presentIs a bleeding tendency present
Easy BruisingEasy Bruising
Mucosal bleedingMucosal bleeding
MenorrhagiaMenorrhagia
Surgical Hemorrhage ndash Procedure Surgical Hemorrhage ndash Procedure vsDiathesisvsDiathesis
Postpartum HemorrhagePostpartum Hemorrhage
Joint and Muscle bleed ndashSeverity of Joint and Muscle bleed ndashSeverity of trauamatrauama
Approach to a bleeding Approach to a bleeding patientpatient
ndashIs the disorder Familial or Is the disorder Familial or AcquiredAcquired
Family history ndash MOTHER amp OTHER Family history ndash MOTHER amp OTHER FEMALE MEMBERS IN THE FEMALE MEMBERS IN THE IMMEDIATE FAMILY ndash IMMEDIATE FAMILY ndash Detailed Detailed Menstrual historyMenstrual history
bull Vascular causes ndashVascular causes ndash
First and foremost rule out First and foremost rule out infectious causes ndash ldquoinfectious causes ndash ldquoMeningococcemiardquoMeningococcemiardquo
Vasculitis ndash Henoch-Schonlein PurpuraVasculitis ndash Henoch-Schonlein Purpura
Hemangiomas- Kassalback-Merritt Hemangiomas- Kassalback-Merritt syndromesyndrome
bull Petechiae and PurpuraPetechiae and Purpurabull 1048708 1048708 InfectiousInfectiousbull ndash ndash MeningococcemiaMeningococcemiabull ndash ndash Rocky mountain spottedRocky mountain spottedbull feverfeverbull ndash ndash Group A strepGroup A strepbull ndash ndash Atypical measlesAtypical measlesbull ndash ndash Echovirus 9 4 7Echovirus 9 4 7bull ndash ndash Epstein-Barr virusEpstein-Barr virusbull ndash ndash Coxsackie virus A9Coxsackie virus A9
bull 1048708 1048708 Non-infectiousNon-infectious
bull ndash ndash Normal plateletsNormal platelets
bull 1048708 1048708 HSPHSP
bull 1048708 1048708 Coagulation disordersCoagulation disorders
bull 1048708 1048708 TraumaTrauma
bull ndash ndash Low plateletsLow platelets
bull 1048708 1048708 ITPITP
bull 1048708 1048708 LeukemiaLeukemia
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Kinins HMW Kininogen
Kallikrein Contact Activation
XII Prekallikrein
XIIa
XIa XI
IXa Ca++ IX
VIIIa VIII
Ca++
Phospholipid
Intrinsic Pathway
Xa X
Va V
Ca++
Phospholipid XIII
II IIa
XIIa
Fibrinogen Fibrin XIII
Common Pathway
VII VIIa
Ca++
Tissue Factor
Coagulation cascadeCoagulation cascade
Extrinsic Pathway
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull HISTORY ndash HISTORY ndash HISTORY ndashHISTORY ndash HISTORY HISTORY
gtgtAGE OF ONSETAGE OF ONSET
gt SEXgt SEX
gtFREQUENCY gtFREQUENCY
gtLOCATION TYPE OF BLEEDINGgtLOCATION TYPE OF BLEEDING
gtDURATION OF BLEEDINGgtDURATION OF BLEEDING
gt MEDICATIONSgt MEDICATIONS
gt ASSOCIATED SYMPTOMS gt ASSOCIATED SYMPTOMS
gt REVIEW OF SYSTEMSgt REVIEW OF SYSTEMS
Approach to a bleeding Approach to a bleeding patientpatient
ndash What is the type of bleeding disorderWhat is the type of bleeding disorder
Primary hemostasis ndash Vascular causesPrimary hemostasis ndash Vascular causes
Platelets-Number vsPlatelets-Number vs
Function Function
Fibrin formation ndash clotting factorsFibrin formation ndash clotting factors
Premature clot dissolution- post clot Premature clot dissolution- post clot formationformation
Approach to a bleeding Approach to a bleeding patientpatient
ndash Is a bleeding tendency presentIs a bleeding tendency present
Easy BruisingEasy Bruising
Mucosal bleedingMucosal bleeding
MenorrhagiaMenorrhagia
Surgical Hemorrhage ndash Procedure Surgical Hemorrhage ndash Procedure vsDiathesisvsDiathesis
Postpartum HemorrhagePostpartum Hemorrhage
Joint and Muscle bleed ndashSeverity of Joint and Muscle bleed ndashSeverity of trauamatrauama
Approach to a bleeding Approach to a bleeding patientpatient
ndashIs the disorder Familial or Is the disorder Familial or AcquiredAcquired
Family history ndash MOTHER amp OTHER Family history ndash MOTHER amp OTHER FEMALE MEMBERS IN THE FEMALE MEMBERS IN THE IMMEDIATE FAMILY ndash IMMEDIATE FAMILY ndash Detailed Detailed Menstrual historyMenstrual history
bull Vascular causes ndashVascular causes ndash
First and foremost rule out First and foremost rule out infectious causes ndash ldquoinfectious causes ndash ldquoMeningococcemiardquoMeningococcemiardquo
Vasculitis ndash Henoch-Schonlein PurpuraVasculitis ndash Henoch-Schonlein Purpura
Hemangiomas- Kassalback-Merritt Hemangiomas- Kassalback-Merritt syndromesyndrome
bull Petechiae and PurpuraPetechiae and Purpurabull 1048708 1048708 InfectiousInfectiousbull ndash ndash MeningococcemiaMeningococcemiabull ndash ndash Rocky mountain spottedRocky mountain spottedbull feverfeverbull ndash ndash Group A strepGroup A strepbull ndash ndash Atypical measlesAtypical measlesbull ndash ndash Echovirus 9 4 7Echovirus 9 4 7bull ndash ndash Epstein-Barr virusEpstein-Barr virusbull ndash ndash Coxsackie virus A9Coxsackie virus A9
bull 1048708 1048708 Non-infectiousNon-infectious
bull ndash ndash Normal plateletsNormal platelets
bull 1048708 1048708 HSPHSP
bull 1048708 1048708 Coagulation disordersCoagulation disorders
bull 1048708 1048708 TraumaTrauma
bull ndash ndash Low plateletsLow platelets
bull 1048708 1048708 ITPITP
bull 1048708 1048708 LeukemiaLeukemia
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull HISTORY ndash HISTORY ndash HISTORY ndashHISTORY ndash HISTORY HISTORY
gtgtAGE OF ONSETAGE OF ONSET
gt SEXgt SEX
gtFREQUENCY gtFREQUENCY
gtLOCATION TYPE OF BLEEDINGgtLOCATION TYPE OF BLEEDING
gtDURATION OF BLEEDINGgtDURATION OF BLEEDING
gt MEDICATIONSgt MEDICATIONS
gt ASSOCIATED SYMPTOMS gt ASSOCIATED SYMPTOMS
gt REVIEW OF SYSTEMSgt REVIEW OF SYSTEMS
Approach to a bleeding Approach to a bleeding patientpatient
ndash What is the type of bleeding disorderWhat is the type of bleeding disorder
Primary hemostasis ndash Vascular causesPrimary hemostasis ndash Vascular causes
Platelets-Number vsPlatelets-Number vs
Function Function
Fibrin formation ndash clotting factorsFibrin formation ndash clotting factors
Premature clot dissolution- post clot Premature clot dissolution- post clot formationformation
Approach to a bleeding Approach to a bleeding patientpatient
ndash Is a bleeding tendency presentIs a bleeding tendency present
Easy BruisingEasy Bruising
Mucosal bleedingMucosal bleeding
MenorrhagiaMenorrhagia
Surgical Hemorrhage ndash Procedure Surgical Hemorrhage ndash Procedure vsDiathesisvsDiathesis
Postpartum HemorrhagePostpartum Hemorrhage
Joint and Muscle bleed ndashSeverity of Joint and Muscle bleed ndashSeverity of trauamatrauama
Approach to a bleeding Approach to a bleeding patientpatient
ndashIs the disorder Familial or Is the disorder Familial or AcquiredAcquired
Family history ndash MOTHER amp OTHER Family history ndash MOTHER amp OTHER FEMALE MEMBERS IN THE FEMALE MEMBERS IN THE IMMEDIATE FAMILY ndash IMMEDIATE FAMILY ndash Detailed Detailed Menstrual historyMenstrual history
bull Vascular causes ndashVascular causes ndash
First and foremost rule out First and foremost rule out infectious causes ndash ldquoinfectious causes ndash ldquoMeningococcemiardquoMeningococcemiardquo
Vasculitis ndash Henoch-Schonlein PurpuraVasculitis ndash Henoch-Schonlein Purpura
Hemangiomas- Kassalback-Merritt Hemangiomas- Kassalback-Merritt syndromesyndrome
bull Petechiae and PurpuraPetechiae and Purpurabull 1048708 1048708 InfectiousInfectiousbull ndash ndash MeningococcemiaMeningococcemiabull ndash ndash Rocky mountain spottedRocky mountain spottedbull feverfeverbull ndash ndash Group A strepGroup A strepbull ndash ndash Atypical measlesAtypical measlesbull ndash ndash Echovirus 9 4 7Echovirus 9 4 7bull ndash ndash Epstein-Barr virusEpstein-Barr virusbull ndash ndash Coxsackie virus A9Coxsackie virus A9
bull 1048708 1048708 Non-infectiousNon-infectious
bull ndash ndash Normal plateletsNormal platelets
bull 1048708 1048708 HSPHSP
bull 1048708 1048708 Coagulation disordersCoagulation disorders
bull 1048708 1048708 TraumaTrauma
bull ndash ndash Low plateletsLow platelets
bull 1048708 1048708 ITPITP
bull 1048708 1048708 LeukemiaLeukemia
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Approach to a bleeding Approach to a bleeding patientpatient
ndash What is the type of bleeding disorderWhat is the type of bleeding disorder
Primary hemostasis ndash Vascular causesPrimary hemostasis ndash Vascular causes
Platelets-Number vsPlatelets-Number vs
Function Function
Fibrin formation ndash clotting factorsFibrin formation ndash clotting factors
Premature clot dissolution- post clot Premature clot dissolution- post clot formationformation
Approach to a bleeding Approach to a bleeding patientpatient
ndash Is a bleeding tendency presentIs a bleeding tendency present
Easy BruisingEasy Bruising
Mucosal bleedingMucosal bleeding
MenorrhagiaMenorrhagia
Surgical Hemorrhage ndash Procedure Surgical Hemorrhage ndash Procedure vsDiathesisvsDiathesis
Postpartum HemorrhagePostpartum Hemorrhage
Joint and Muscle bleed ndashSeverity of Joint and Muscle bleed ndashSeverity of trauamatrauama
Approach to a bleeding Approach to a bleeding patientpatient
ndashIs the disorder Familial or Is the disorder Familial or AcquiredAcquired
Family history ndash MOTHER amp OTHER Family history ndash MOTHER amp OTHER FEMALE MEMBERS IN THE FEMALE MEMBERS IN THE IMMEDIATE FAMILY ndash IMMEDIATE FAMILY ndash Detailed Detailed Menstrual historyMenstrual history
bull Vascular causes ndashVascular causes ndash
First and foremost rule out First and foremost rule out infectious causes ndash ldquoinfectious causes ndash ldquoMeningococcemiardquoMeningococcemiardquo
Vasculitis ndash Henoch-Schonlein PurpuraVasculitis ndash Henoch-Schonlein Purpura
Hemangiomas- Kassalback-Merritt Hemangiomas- Kassalback-Merritt syndromesyndrome
bull Petechiae and PurpuraPetechiae and Purpurabull 1048708 1048708 InfectiousInfectiousbull ndash ndash MeningococcemiaMeningococcemiabull ndash ndash Rocky mountain spottedRocky mountain spottedbull feverfeverbull ndash ndash Group A strepGroup A strepbull ndash ndash Atypical measlesAtypical measlesbull ndash ndash Echovirus 9 4 7Echovirus 9 4 7bull ndash ndash Epstein-Barr virusEpstein-Barr virusbull ndash ndash Coxsackie virus A9Coxsackie virus A9
bull 1048708 1048708 Non-infectiousNon-infectious
bull ndash ndash Normal plateletsNormal platelets
bull 1048708 1048708 HSPHSP
bull 1048708 1048708 Coagulation disordersCoagulation disorders
bull 1048708 1048708 TraumaTrauma
bull ndash ndash Low plateletsLow platelets
bull 1048708 1048708 ITPITP
bull 1048708 1048708 LeukemiaLeukemia
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Approach to a bleeding Approach to a bleeding patientpatient
ndash Is a bleeding tendency presentIs a bleeding tendency present
Easy BruisingEasy Bruising
Mucosal bleedingMucosal bleeding
MenorrhagiaMenorrhagia
Surgical Hemorrhage ndash Procedure Surgical Hemorrhage ndash Procedure vsDiathesisvsDiathesis
Postpartum HemorrhagePostpartum Hemorrhage
Joint and Muscle bleed ndashSeverity of Joint and Muscle bleed ndashSeverity of trauamatrauama
Approach to a bleeding Approach to a bleeding patientpatient
ndashIs the disorder Familial or Is the disorder Familial or AcquiredAcquired
Family history ndash MOTHER amp OTHER Family history ndash MOTHER amp OTHER FEMALE MEMBERS IN THE FEMALE MEMBERS IN THE IMMEDIATE FAMILY ndash IMMEDIATE FAMILY ndash Detailed Detailed Menstrual historyMenstrual history
bull Vascular causes ndashVascular causes ndash
First and foremost rule out First and foremost rule out infectious causes ndash ldquoinfectious causes ndash ldquoMeningococcemiardquoMeningococcemiardquo
Vasculitis ndash Henoch-Schonlein PurpuraVasculitis ndash Henoch-Schonlein Purpura
Hemangiomas- Kassalback-Merritt Hemangiomas- Kassalback-Merritt syndromesyndrome
bull Petechiae and PurpuraPetechiae and Purpurabull 1048708 1048708 InfectiousInfectiousbull ndash ndash MeningococcemiaMeningococcemiabull ndash ndash Rocky mountain spottedRocky mountain spottedbull feverfeverbull ndash ndash Group A strepGroup A strepbull ndash ndash Atypical measlesAtypical measlesbull ndash ndash Echovirus 9 4 7Echovirus 9 4 7bull ndash ndash Epstein-Barr virusEpstein-Barr virusbull ndash ndash Coxsackie virus A9Coxsackie virus A9
bull 1048708 1048708 Non-infectiousNon-infectious
bull ndash ndash Normal plateletsNormal platelets
bull 1048708 1048708 HSPHSP
bull 1048708 1048708 Coagulation disordersCoagulation disorders
bull 1048708 1048708 TraumaTrauma
bull ndash ndash Low plateletsLow platelets
bull 1048708 1048708 ITPITP
bull 1048708 1048708 LeukemiaLeukemia
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Approach to a bleeding Approach to a bleeding patientpatient
ndashIs the disorder Familial or Is the disorder Familial or AcquiredAcquired
Family history ndash MOTHER amp OTHER Family history ndash MOTHER amp OTHER FEMALE MEMBERS IN THE FEMALE MEMBERS IN THE IMMEDIATE FAMILY ndash IMMEDIATE FAMILY ndash Detailed Detailed Menstrual historyMenstrual history
bull Vascular causes ndashVascular causes ndash
First and foremost rule out First and foremost rule out infectious causes ndash ldquoinfectious causes ndash ldquoMeningococcemiardquoMeningococcemiardquo
Vasculitis ndash Henoch-Schonlein PurpuraVasculitis ndash Henoch-Schonlein Purpura
Hemangiomas- Kassalback-Merritt Hemangiomas- Kassalback-Merritt syndromesyndrome
bull Petechiae and PurpuraPetechiae and Purpurabull 1048708 1048708 InfectiousInfectiousbull ndash ndash MeningococcemiaMeningococcemiabull ndash ndash Rocky mountain spottedRocky mountain spottedbull feverfeverbull ndash ndash Group A strepGroup A strepbull ndash ndash Atypical measlesAtypical measlesbull ndash ndash Echovirus 9 4 7Echovirus 9 4 7bull ndash ndash Epstein-Barr virusEpstein-Barr virusbull ndash ndash Coxsackie virus A9Coxsackie virus A9
bull 1048708 1048708 Non-infectiousNon-infectious
bull ndash ndash Normal plateletsNormal platelets
bull 1048708 1048708 HSPHSP
bull 1048708 1048708 Coagulation disordersCoagulation disorders
bull 1048708 1048708 TraumaTrauma
bull ndash ndash Low plateletsLow platelets
bull 1048708 1048708 ITPITP
bull 1048708 1048708 LeukemiaLeukemia
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
bull Vascular causes ndashVascular causes ndash
First and foremost rule out First and foremost rule out infectious causes ndash ldquoinfectious causes ndash ldquoMeningococcemiardquoMeningococcemiardquo
Vasculitis ndash Henoch-Schonlein PurpuraVasculitis ndash Henoch-Schonlein Purpura
Hemangiomas- Kassalback-Merritt Hemangiomas- Kassalback-Merritt syndromesyndrome
bull Petechiae and PurpuraPetechiae and Purpurabull 1048708 1048708 InfectiousInfectiousbull ndash ndash MeningococcemiaMeningococcemiabull ndash ndash Rocky mountain spottedRocky mountain spottedbull feverfeverbull ndash ndash Group A strepGroup A strepbull ndash ndash Atypical measlesAtypical measlesbull ndash ndash Echovirus 9 4 7Echovirus 9 4 7bull ndash ndash Epstein-Barr virusEpstein-Barr virusbull ndash ndash Coxsackie virus A9Coxsackie virus A9
bull 1048708 1048708 Non-infectiousNon-infectious
bull ndash ndash Normal plateletsNormal platelets
bull 1048708 1048708 HSPHSP
bull 1048708 1048708 Coagulation disordersCoagulation disorders
bull 1048708 1048708 TraumaTrauma
bull ndash ndash Low plateletsLow platelets
bull 1048708 1048708 ITPITP
bull 1048708 1048708 LeukemiaLeukemia
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
bull Petechiae and PurpuraPetechiae and Purpurabull 1048708 1048708 InfectiousInfectiousbull ndash ndash MeningococcemiaMeningococcemiabull ndash ndash Rocky mountain spottedRocky mountain spottedbull feverfeverbull ndash ndash Group A strepGroup A strepbull ndash ndash Atypical measlesAtypical measlesbull ndash ndash Echovirus 9 4 7Echovirus 9 4 7bull ndash ndash Epstein-Barr virusEpstein-Barr virusbull ndash ndash Coxsackie virus A9Coxsackie virus A9
bull 1048708 1048708 Non-infectiousNon-infectious
bull ndash ndash Normal plateletsNormal platelets
bull 1048708 1048708 HSPHSP
bull 1048708 1048708 Coagulation disordersCoagulation disorders
bull 1048708 1048708 TraumaTrauma
bull ndash ndash Low plateletsLow platelets
bull 1048708 1048708 ITPITP
bull 1048708 1048708 LeukemiaLeukemia
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
bull 1048708 1048708 Non-infectiousNon-infectious
bull ndash ndash Normal plateletsNormal platelets
bull 1048708 1048708 HSPHSP
bull 1048708 1048708 Coagulation disordersCoagulation disorders
bull 1048708 1048708 TraumaTrauma
bull ndash ndash Low plateletsLow platelets
bull 1048708 1048708 ITPITP
bull 1048708 1048708 LeukemiaLeukemia
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORY
bull PHYSICAL EXAMINATION-PHYSICAL EXAMINATION-
gt PETICHEAEgt PETICHEAE
gtECHYMOSESgtECHYMOSES gtJOINT BLEED ampDEEPSEATED HEMATOMASgtJOINT BLEED ampDEEPSEATED HEMATOMAS gt HEPATOSPLENOMEGALYgt HEPATOSPLENOMEGALY gtSIGNIFICANT LYMPHADENOPATHYgtSIGNIFICANT LYMPHADENOPATHY gt ACTIVE AND PLAYFUL VS ILL LOOKINGgt ACTIVE AND PLAYFUL VS ILL LOOKING gt DYSMORPHIC FEATURESgt DYSMORPHIC FEATURES gt CAFEacute-AU-LAIT SPOTSgt CAFEacute-AU-LAIT SPOTS gtTELANGIECATIC VESSELSgtTELANGIECATIC VESSELS gtHEMANGIOMASgtHEMANGIOMAS
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
PRACTICAL APPROACH TO A CHILD WITH PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORYBLEEDING HISTORYbull LABORATORY WORK UP-LABORATORY WORK UP-
PMD -PMD -
gt CBCPLATELET COUNTgt CBCPLATELET COUNT
gtPERIPHERAL SMEAR- MORPHOLOGYgtPERIPHERAL SMEAR- MORPHOLOGY
gt PT [gt PT [Prothrombin time]Prothrombin time]
gt aPTT [ gt aPTT [ Activated partial thromboplastin Activated partial thromboplastin time]time]
----------------------------------------------------------------------------------------------------------------------------------
Hemophilia service --Hemophilia service --
gt TT [gt TT [Thrombin time]Thrombin time]
gt Bleeding timegt Bleeding time
gtPlatelet aggregation studiesgtPlatelet aggregation studies
gt Factor assaygt Factor assay
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Pandorarsquos box Pandorarsquos box coagulation testcoagulation test
The results are as good as The results are as good as the sample isthe sample is
Standards Standards TimeTime from sample to test PT 24 from sample to test PT 24 hours PTT 4 hourshours PTT 4 hours
Bloodcitrate ratio Bloodcitrate ratio 9 19 1
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Bleeding disordersBleeding disorders
bull Plateletsndash Plateletsndash Acquired causes much more commonAcquired causes much more common Thrombocytopenia more common than Thrombocytopenia more common than functional defectsfunctional defects Inherited disorders ndash both number ampfunctional Inherited disorders ndash both number ampfunctional
defects defects are extremely rare are extremely rare
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
PLATELETS ndash NUMBERPLATELETS ndash NUMBER
Acquired causes are most commonAcquired causes are most common
ITPITP
InfectionsInfections
CONGENITAL THROMBOCYTOPENIASCONGENITAL THROMBOCYTOPENIAS
TAR syndromeTAR syndrome
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
bull ITP-ITP- Most have benign amplimited courseMost have benign amplimited courseTreatment options-Treatment options- Conservative ndashwait ampwatchConservative ndashwait ampwatchAggressive-Aggressive- SteroidsSteroids IvIGGIvIGG RhogamRhogam RituximabRituximab
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
PLATELETSPLATELETS
bull Functional disorders-Functional disorders-
Acquired- Aspirin UremiaAcquired- Aspirin Uremia
Inherited ndashInherited ndash
GlanzmanrsquosGlanzmanrsquos
Bernard-SoulierBernard-Soulier
Gray platelet syndrome Gray platelet syndrome
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
von Willebrand Diseasevon Willebrand Disease
bull The most common inherited bleeding The most common inherited bleeding disorderdisorder
bull Occurs in 1 of the populationOccurs in 1 of the population
bull Less than 10 of patients have Less than 10 of patients have bleeding events due to vWDbleeding events due to vWD
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Inheritance of Type 1 vWDInheritance of Type 1 vWD
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Functions of vWFFunctions of vWF
bull Binds to platelet receptor GP Ib and to Binds to platelet receptor GP Ib and to subendothelial structures such as collagen subendothelial structures such as collagen serving as bridge between platelets and serving as bridge between platelets and subendothelium in damaged vesselssubendothelium in damaged vessels
bull Acts as bridge between adjacent platelets in Acts as bridge between adjacent platelets in vessels with high shear (arterioles) forming vessels with high shear (arterioles) forming small platelet aggregatessmall platelet aggregates
bull Binds to circulating factor VIII protecting it and Binds to circulating factor VIII protecting it and prolonging FVIII t12 in the circulation from 2 prolonging FVIII t12 in the circulation from 2 to 8-12 hoursto 8-12 hours
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Symptoms of vWDSymptoms of vWD
bull Easy bruisabilityEasy bruisability
bull Epistaxis or gingival bleedingEpistaxis or gingival bleeding
bull MenorrhagiaMenorrhagia
bull Post-partum hemorrhagePost-partum hemorrhage
bull Post-surgical bleedingPost-surgical bleeding
bull Bleeding post-dental extractionBleeding post-dental extraction
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Classification of vWDClassification of vWDSub types of VWSub types of VW
Type 1 Type 1 Partial quantitative deficiency of vWF Partial quantitative deficiency of vWF
Type 2Type 2 Qualitative variants of vWF Qualitative variants of vWF
bull AA Absence of HMW vWF Absence of HMW vWF multimers multimers
bull BB Same as 2A and increased Same as 2A and increased affinity affinity for platelet gp Ib for platelet gp Ib
bull MM Abnormal function not caused by Abnormal function not caused by absence of HMW multimers absence of HMW multimers
bull N Decreased affinity for factor VIIIN Decreased affinity for factor VIII
Type 3Type 3 Complete deficiencey of vWF amp Behave as Complete deficiencey of vWF amp Behave as Severe Severe
Hemophilia AHemophilia A
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Treatment Guidelines in Treatment Guidelines in VWDVWD TYPETYPE
11
2A2A
2B2B
2M2M
2N2N
33
TREATMENTTREATMENT
DDAVPDDAVP
DDAVPFVIII-VWFDDAVPFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
FVIII-VWFFVIII-VWF
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
DDAVP (1-desamino-8-D-DDAVP (1-desamino-8-D-arginine vasopressin)arginine vasopressin)
bullParenteral form DDAVP (for Parenteral form DDAVP (for IV or SC use 03 ugkg)IV or SC use 03 ugkg)
bullHighly concentrated Highly concentrated intranasal spray form intranasal spray form Stimate nasal spray Stimate nasal spray (150-300 ug )(150-300 ug )
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
HemophiliaHemophilia
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
HemophiliaHemophiliabullCaused by an absence or Caused by an absence or
decreased amount of a decreased amount of a procoagulant ndashprocoagulant ndash
bullVIII -Hemophilia A affects ~ VIII -Hemophilia A affects ~ 15000 males15000 males
bullXI -Hemophilia B affects ~ XI -Hemophilia B affects ~ 130000 males130000 males
bullXI ndashHemophilia C ndash Rare EthnicityXI ndashHemophilia C ndash Rare Ethnicity
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
EpidemiologyEpidemiology
Hemophilia A
Other
Hemophilia B
Incidence Hemophilia A - 15000Hempohilia B ndash 1 30 000
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
InheritanceInheritance
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
InheritanceInheritance
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Woman can have Woman can have hemophiliahemophilia
bullLyonization of the normal X Lyonization of the normal X chromosomechromosome
bullTurner syndrome ( XO)Turner syndrome ( XO)
bullFather with hemophilia mom as Father with hemophilia mom as a carriera carrier
bullvW type 2 N ( Normandy) vW type 2 N ( Normandy)
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
HEMOPHILIA SEVERITY LEVELSHEMOPHILIA SEVERITY LEVELSbull Severe lt1 activity level - Spontaneous bleeds
bull Moderate 1 to 5 activity --Traumasurgery bleeds Occasional joint
bleeds
bull Mild 5 to 30 activity - Major traumasurgery Rare joint bleeds
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Factor replacementFactor replacement
bull1 ukg raises FVIII levels 21 ukg raises FVIII levels 2
12 life 12 hrs12 life 12 hrs
bull1 ukg raises FIX levels 1 1 ukg raises FIX levels 1
12 life 20-24 hrs12 life 20-24 hrs
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Minor Bleeding EpisodesMinor Bleeding Episodesbull Early joint bleedsEarly joint bleeds
bull Soft tissue amp muscle bleedsSoft tissue amp muscle bleeds
bull Nose amp gum bleeding not Nose amp gum bleeding not responding to local measuresresponding to local measures
bull Treatment of minor bleeding Treatment of minor bleeding episodesepisodesndash 40 - 50 correction40 - 50 correctionndash FVIII 25 units kgFVIII 25 units kgndash FIX 50 units kgFIX 50 units kg
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Major Bleeding EpisodesMajor Bleeding Episodesbull Advanced soft tissue amp muscle bleedsAdvanced soft tissue amp muscle bleeds
bull Head amp neck injuriesHead amp neck injuries
bull Gastrointestinal bleedingGastrointestinal bleeding
bull Advanced joint bleedingAdvanced joint bleeding
bull Treatment of major bleeding Treatment of major bleeding episodesepisodesbull 80 ndash 100 correction80 ndash 100 correctionbull FVIII 50 units kgFVIII 50 units kgbull FIX 100 units kgFIX 100 units kg
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Current ProductsCurrent Productsbull Plasma Products plasma-derived factor VIII Plasma Products plasma-derived factor VIII
concentrateconcentrate
bull Porcine Factor Porcine Factor ndash Use was halted due to parvovirusretrovirus Use was halted due to parvovirusretrovirus
sequences discoveredsequences discovered
bull Recombinant products Recombinant products ndash First Generation Recombinate Kogenate HelixateFirst Generation Recombinate Kogenate Helixatendash Second Generation Kogenate FS Helixate FSSecond Generation Kogenate FS Helixate FSndash Third Generation AdvateThird Generation Advate
bull DDAVP DDAVP ndash Causes release of factor VIIIvWFCauses release of factor VIIIvWFndash Increased factor activity in 30-60rdquoIncreased factor activity in 30-60rdquondash For mild hemophiliacs and mild bleeding symptoms For mild hemophiliacs and mild bleeding symptoms
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Replacement therapy Joint Replacement therapy Joint diseasedisease
bullProphylaxisProphylaxisndash PrimaryPrimaryndash SecondarySecondary
bullIntensive infusion therapyIntensive infusion therapy
bullDose escalation modified prophylaxisDose escalation modified prophylaxis
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Clinical SeverityClinical Severity
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Chronic Joint Chronic Joint
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Hemophilia Treatment Center Hemophilia Treatment Center Team MembersTeam Members
bullPatient FamilyPatient Family
bullHematologistHematologist
bullNurseNurse
bullSocial WorkerSocial Worker
bullPhysical TherapistPhysical Therapist
bullOrthopedistOrthopedist
bullPrimary CarePrimary Care
bullInfectious DiseaseInfectious Disease
bullGeneticsGenetics
bullPharmacyPharmacy
bullDentalDental
bullHepatologistHepatologist
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Basis for Comprehensive Basis for Comprehensive CareCarebull HematologistHematologist
ndash Assumes overall careAssumes overall carebull MusculoskeletalMusculoskeletal
ndash Orthopedic Surgeon Physical therapistOrthopedic Surgeon Physical therapistbull NursingNursing
ndash Coordination of homeclinic care for rapid Coordination of homeclinic care for rapid treatment at the earliest symptoms suggestive treatment at the earliest symptoms suggestive of a bleedof a bleed
bull DentalDentalbull Genetic CounselingGenetic Counselingbull Infectious DiseaseInfectious Diseasebull PsychosocialPsychosocial
ndash social workersocial worker
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Role of Hemophilia Role of Hemophilia Treatment CentersTreatment CentersbullState-of-the-art medical treatment for State-of-the-art medical treatment for
persons with hemophilia through out persons with hemophilia through out the life spanthe life span
bullEducationEducation
bullResearchResearch
bullOutreachOutreach
bullModel of comprehensive care for Model of comprehensive care for chronic diseasechronic disease
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
The PasthellipThe Pasthellip
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
helliphellipthe promise of achieving your the promise of achieving your potentialpotential
PresentPresent
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Made possible by a STRONG amp Made possible by a STRONG amp Dedicated Hemophilia parent Dedicated Hemophilia parent association and dedicated NJHA staffassociation and dedicated NJHA staff
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-
Thank youThank you
- Slide 2
- Slide 3
- Slide 4
- Overview of Haemostasis
- Slide 6
- Slide 7
- Slide 8
- Slide 9
- Slide 10
- PRACTICAL APPROACH TO A CHILD WITH BLEEDING HISTORY
- Approach to a bleeding patient
- Approach to a bleeding patient
- Slide 14
- Slide 15
- Slide 16
- Slide 17
- Slide 18
- Slide 19
- Pandorarsquos box coagulation test
- Slide 21
- Slide 22
- Bleeding disorders
- Slide 24
- Slide 25
- Slide 26
- PLATELETS
- von Willebrand Disease
- Inheritance of Type 1 vWD
- Functions of vWF
- Symptoms of vWD
- Classification of vWD
- Slide 33
- DDAVP (1-desamino-8-D-arginine vasopressin)
- Slide 35
- Hemophilia
- Epidemiology
- Inheritance
- Inheritance
- Woman can have hemophilia
- HEMOPHILIA SEVERITY LEVELS
- Factor replacement
- Minor Bleeding Episodes
- Major Bleeding Episodes
- Current Products
- Replacement therapy Joint disease
- Clinical Severity
- Slide 48
- Slide 49
- Chronic Joint
- Hemophilia Treatment Center Team Members
- Basis for Comprehensive Care
- Role of Hemophilia Treatment Centers
- The Pasthellip
- Present
- Slide 56
- Thank you
-