RoulaFARAH,J.Horkos,Y.Bustros,H.Farhat,O.AblaSaintGeorgeHospitalUniversityMedicalCenter-Beirut,LEBANON

A MULTICENTER EXPERIENCE FROM LEBANON IN CHILDHOOD AND ADOLESCENTS ACUTE MYELOID LEUKEMIA:

HIGH RATE OF EARLY DEATH IN CHILDHOOD APL

24-27,SEPTEMBER2017,Rome,ITALY

7thInterna1onalSymposiumonACUTEPROMYELOCYTICLEUKEMIA

•  PopulaQon:–  4millionLebanese–  2millionnon-Lebaneserefugees

•  10,452km2•  HighlevelofeducaQon•  Healthcaresystemmostly

private-cancerpaQentsparQallycoveredbyMOH

Background:LebaneseSocial&HealthFacts

•  NoregularCancerstaQsQcs.IncidenceofAMLandthereforeAPLunknown

•  AdvancedLabTests&TransfusionServicesavailableinterQarycarecentersbutnotidealinsmallcentersorruralareas

•  ATRAavailableuponrequest:notalwayspresentinHospitalstocks–coverageofATRAvaries

LebanonSocial&HealthFacts

•  AMLisadiseasewithmarkedheterogeneityinclinicaland

biologicfeatures,responsetotherapyandsurvival.

•  DespitemajorachievementsinthetreatmentofAML,longtermsurvivalremainspoor.HalfofpediatricAMLpaQentsrelapseanddieinternaQonally.

•  APLisauniquesubtypewithdisQnctbiologicandmolecularcharacterisQcs,fromhighlyfatal50yearsagotohighlycurablenowadaysbutsQllassociatedwithhighratesofearlydeathanddelaysindiagnosisinthe“realworld”.

•  ThereisnopublisheddataonpediatricAMLorAPLinLebanon

Background:AML&APL

•  IdenQfy:Clinical,CytogeneQc,MolecularFindingsandOutcomedataofpediatricAMLintheLebanesepopulaQonincomparisontotheInternaQonalandRegionaldataavailable.

•  FocusthestudyonChildhoodandAdolescentAPLinLebanon.

Objec1ves

•  RetrospecQvechartreviewofchildrenwithAMLandAPLdiagnosedat3terQarycarecentersinBeirutoverthepasttenyears.

•  DatacollecQonsheetswerefilledandanalyzed.

Methods

•  DemographicInformaQon

•  DateofDiagnosisandPresenQngSymptoms

•  BloodAnalysis,BoneMarrowAspirate,CytogeneQcFeatures,MolecularBiologyAnalysis,HLAtyping

•  TreatmentandOutcome

•  DataonHSCTandLong-TermFollow-up

DATACollec1onSheets

•  TwentyfourpaQentswerediagnosedwithAMLfrom2002-2010,12girlsand12boys.

•  Twohadpre-exisQngFanconianemia,1Downsyndrome,1MDSwithmonosomy7,1secondaryAMLaaertreatmentforBurkib’slymphoma.

•  6(25%)haddenovoAPLwitht(15;17)

•  Meanagewas8.6years(range,1-24)

•  MedianWBCatdx=31x109/L(range,2.1-376);PLT:46x109/L(range,10-164)

Results:TotalAML&APLPa1ents

NormalKaryotype;37.5%

t(15;17); 25% t(8;9); 4.1%

t(7;11); 4.1%

t(8;21); 8.3%

t(9;11); 4.1%

inv 16; 8.3%

ComplexAbnormaliQes;

4.1%Other;4.5%

Cytogene1cFindings

NormalKaryotype

t(15;17)

t(8;9)

t(7;11)

t(8;21)

t(9;11)

inv16

ComplexAbnormaliQes

Other

Results

•  FLT-3(InternalTandemDuplicaQons)weredetectedin3AMLpaQentsandwasassociatedwithhighWBCatpresentaQonandapooroutcome

•  NPM1wasscreenedinonepaQent,mutaQonswerenotfound.

Results:MolecularStudiesnotPerformedonallChildren

•  71%ofpaQentsdevelopedAMLatanage<10yearswiththeyoungestis1yearold.

•  Thisagegroupcarrieda50%survivalratecomparedto0%survivalinpaQents>10yrs.

Results

•  DeathininducQonwasobservedin3/6paQentswithAPL(50%).All3paQentshadaniniQalWBC>10x109/L

•  TwodiedduringinducQonduetoDICandCNSbleeddespitea

quickdiagnosisandearlystartofATRA;1diedfromCNSbleed2daysaaerdiagnosisandbeforestarQngtreatment.

• 

Results:EarlyDeathinAPL

•  Indicatedin15paQentswithAMLandwasperformedon9childreninEuropeortheUnitedStates:5MUDand4matchedrelatedsibling.

•  Survivalaaertransplantwas37.5%.

•  TwoAMLchildrendied:1withM6AMLand1AMLpost-Fanconianemia.

HSCTinAML

•  MediansurvivalforpaQentswhodiedfromdiseaseprogressionwas25.8months.

•  Overalldisease-freesurvivalwas30.4%.

Results:AllAMLPa1ents

•  APLrepresented25%ofallAMLinourseries

•  EarlydeathinAPLwas50%.•  OverallsurvivalintheAMLcohortwas30.4%.

•  EarlydeathinallAMLpaQentswas20%.

Conclusions

– MorerapidavailabilityofATRAinhospitals

–  StartATRAatfirstmorphologicsuspicion

– AggressiveearlytransfusionsofPLTsandFFPorCryoprecipitates

–  IncreaseawarenessamonggeneralpracQQonersforearlyreferraltospecializedcenters

–  FurtherdatacollecQontoincludetheenQrecountry

AreasofImprovementforAPL

EMAIL:roula.fs@dm.net.lb