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Comprehensive care and Sickle Cell Disease and

Hemoglobinopathies

Roshni Kulkarni MD.Distinguished Hematology Consultant

Division of Blood DisordersNational Center on Birth Defects and Developmental

DisabilitiesCenters for Disease Control and Prevention

The findings and conclusions in this presentation are those of the author and do not necessarily represent the views of the Centers for Disease Control and Prevention

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0-6moEarly

childhoodPediatric

Adolescent

Young AdultAdult

Older Adults

No symptoms

Mild anemia (thal variants)

Jaundice

Pain

Anemia

Aplastic crises

Hand foot syndrome

Organo-megaly

Pain Anemia

Stroke

Priapism

Lung (ACS, PHT etc.)

Delayed puberty

Pain, Anemia

Stroke

Lung

Bone (AVN)

Priapism

Leg ulcers

Pregnancy

Pain, Anemia

Lung

Bone

Kidney

Heart, HTN

Eye

Pregnancy

Leg ulcers

Disabilities

Chronic

Lung

Kidney

Heart

Eye

NBS TCD, HU HU,, Transplant

Infections

Transfusions

Co- Morbid conditions

Quality of Life? Nutrition? Mental health? Vocation? Academic achievement?

SCD issues across life span

Roshni Kulkarni M.D

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Impact of SCD on Public Health

• Insufficient data on prevalence may hamper effective management

(immigrant hematology) and resource allocation

• Impact of SCD on Mortality and Morbidity

• Disability from complications

• Impact on QOL

• Preventable organ damage

• Economic impact/ cost of care

• Loss of workforce

• Hospitalizations, emergency room care, blood transfusions/blood safety

• Effectiveness of programs - must be sensitive to cultural practices

and appropriate for given social context (World Health Assembly 2006)

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Challenges in SCD

• Lack access to specialty care - expensive emergency department

and inpatient services

• Only a small fraction take Hydroxyurea

• Many patients on Medicaid

• Not enough funding compared to other genetic disorders

• Federal agencies need to pool resources to coordinate services

by supporting network of centers (large and small) across USA

• Comprehensive care not available nationwide

• Few funded sickle cell centers that serve fraction of the population

• Current research advances need to be widely disseminated

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What About Sickle Cell trait?

• 2 million SCT (1in 12 AA, 1 in 100 Hispanic Americans). Emerging

Hispanic/migrant population

• Thrombosis1 (VTE) 2-fold risk PE 4-fold risk

• Renal disease: renal medullary carcinoma, hematuria, papillary necrosis

• Exercise related sudden death

• Pregnancy related complications

• ↓odds of early preterm delivery and odds of multiple gestations.

fetal losses and placental abnormalities.

Austin Blood. 2007, Tsaras. Am J Med, 2009110:908, 2007. 2. Bryant AS. Obstetrics & Gynecology, 2007

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Public Health & SCD:Reduce Death, Disability, Promote

Health • Determine Incidence and Prevalence

• Surveillance for :

• Complications

• Blood safety monitoring: pathogens, alloimmunization

• Long term follow up of NBS & Screening of

older/migrant population

• Adult vaccinations

• Gynecologic and reproductive issues

• Rural/Urban outcomes

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Public Health & SCD (cont’d)• SCD specific QOL instrument :

• Pain & Disability • Psychosocial and behavioral issues

• Transition of care/ effectiveness• Health Education

• Patient/provider/school education• Prevention message through Community based organizations

• Public health laboratory• Transfusion transmitted disease• Genotyping on a mass scale: high throughput genotyping• Role of gender and race

Health Promotion through Comprehensive care

Comprehensive Care Centers

Registry Surveillance

Diagnosis:Disease burdenTrends in healthEmerging/existing risksHemovigilanceSurveillance maps

Education and prevention messages

Comprehensive care

Comprehensive Care Centers

Public Health Research

Clinical and basic research

Quality indicators

Training program support

Education and prevention messages

Registry and Prospective Surveillance

New Drug DevelopmentBlood safety

Access to specialty care

and laboratory

Health Systems Research

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Comprehensive Care Model• Organized specialized care delivery• Integrated multi-disciplinary teams• Emphasis on

• Early diagnosis• Prevention of complications

• Reduction in disability

• Improves survival and QOL

• Education and training of patients/providers• Coordinated network of centers to improve care

through public health and clinical / translational research (bench to trench)

• Liaison with community organization (s)

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How do we develop comprehensive hemophilia

care in a country?

“BUT”--We must first understand the Economics of Care!

Evatt WFH

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In countries with emerging economies,economic issues will be the force determining how far we can pushour goals…

In developed countries, only constant pressureprevents reduction in services.

We must understand these issues and use them to our advantage…

Economics: The Primary Force in Available Care

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Economic Reality

• Care is expensive.

• Cost of care beyond reach of individual.

• Will rely on others to pay bill.

• Will have to convince others to do so.

• Government must be involved.

• Government focuses on bottom line, economics and the most served for the least cost.

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Effect of Economic Capacityon Hemophilia Adulthood

0

0.5

1

1.5

2

2.5

3

3.5

4

Ra

tio

(P

/Pc)

<13 yrs >19 yrs

Evatt & Robillard., Haemophilia (2000)

<$2,000 $2,000 – $10,000 >$10,000Per Capita Gross National Product

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Effect of Care Programson Hemophilia Adulthood

0

0.2

0.4

0.6

0.8

1

1.2

1.4

1.6

Ra

tio

(P

/Pc)

<13 yrs >19 yrs

<$2,000 <$2,000 + HTC

Per Capita Gross National Product

Evatt & Robillard., Haemophilia (2000)

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Effect of Comprehensive Care

• Life Expectancy 13-40 65

• Days in hospital 12-15 1

• Percent with Joint disease 70-80% <5%

• Annual Visits to Clinic 12-30 0-6

• Unemployment 35-90% 15%

Without Comprehensive Care

With Comprehensive Care

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Components of Comprehensive care team

• Core team:• Medical Director- a hematologist• Coordinator- Nurse/program• Psychosocial professional• Secretary

• Extended team• Pediatrician/internist/Family practitioner• Genetic counselor /Ob-Gyn• Nutritionist /Dental/ other subspecialist – Pulmonologist etc

• Must have field or outreach services

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Registry and SurveillanceWorld Federation of Hemophilia 2005.

• Prevalence

• Determine current and future needs of the community

• Empowering advocacy groups, physicians

• Resource allocation: set priorities of health outcomes

• ↓ Hospitalizations, Improved QOL

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Registry and SurveillanceEvatt B. World Federation of Hemophilia 2005

Maintaining and Using a registry• Develop policy “ we will do….will help 300 people

and cost $24,000”

• Develop and manage resources: identify areas of need

• Define changing/expanding populations needs: vaccination

• Disease management, Disaster preparedness*

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Surveillance Aim: reduce incidence, morbidity and mortality

• Definition: Systematic collection, analysis, interpretation, and

dissemination of health data

• Assist: planning, implementation, and evaluation of public health

interventions and programs.

• SCD surveillance (web based)• Complications across life span

• Impact of risk factors, co-morbid conditions

• Impact of newborn screening

• Blood safety. Lab studies.

• Quality of Life: cultural and social context/transition issues

• Preconceptual care/Reproductive issues

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Components of a SCD Surveillance System

• Comprehensive • Linking multiple data sources

• Population-based• Longitudinal Design• Modular Design• Quality Assurance Measures• Web-based /Electronic Reporting• Unified and Standardized Laboratory Testing• Specimen Collection and Blood Safety Monitoring

Identify data sources for indicators Grant A. DBD/NCBDDD/CDC

Data Source Example Indicator

Vital Records (birth records, death records)

Denominator for incidence, mortality

Newborn screening data Incidence

Hospital Discharge Records Costs, complications

Program Services Records (Medicaid , WIC, school records)

SES, access to care, school performance, quality of life

Private or public health laboratory reports

Incidence, validate behaviors (smoking, drugs), risk factors

Data abstracted from hospital charts Incidence, co-morbidities, outcomes, trends in clinical practice

Pharmacy records Use and adherence to therapy

News media (newspapers) Mortality

Disease registries Prevalence, incidence, trends

Surveys Lifestyle/Behavior, Prevention program data

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Mapping• Geographic Information Systems (GIS) can be

used to analyze data and create maps• Over 90% public health data have a geospatial

component• Maps can be useful for:

• Describing the distribution of patients• Planning of care• Resource planning• Outreach activities• Emergency /disaster preparedness

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Geographical Information SystemMcClellan A. Division of Blood Disorders. NCBDDD.CDC

Hospitals with ED and patients

Hospitals with Emergency

Departments

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McClellan Ann. CDC DBD GIS expert

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Role of Patient Advocacy

• Do not become complacent!

• Must understand health care needs and set

clear realistic goals to achieve them

• Must work hand in hand with physicians to

speak as one voice

• Must understand the political system

• Must work closely with government officials

SCD and Hemophilia

Hemophilia SCDExpensive disease/poor QOL Expensive disease/poor QOL

Govt funded (CDC/HRSA/State) Funding for basic/clinical research

National network+ outreach clinics Centers of Excellence

Industry supported, costly drugs None

Advocacy groups: State, National, Global

Advocacy group not well organized

Patients motivated, higher socioeconomic status

Mostly low socioeconomic, difficulty in accessing care

No Newborn Screening/ poor lab support

NBS nationwide, good lab support

Dwindling adult providers Scarcity of adult providers, poor reimbursement

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So What Can One Do?

• In Hemophilia- population expanded to include thrombosis, Women and other groups

• In SCD can partner with hemoglobinopathies

• Ultimate solution for viability………

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Hematology Specialty Care: Future Blood Disorders Center Model

Advocacy groups

Lifespan issuesComplicationsHemovigilanceManagement

ResearchDisparities

Red cell/Hbpathies

Iron

Ble

edin

g

Clotting

BM failureW

hite

cel

l

S Shurin & R Kulkarni

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Blood Disorders Center Model

Guam

Puerto RicoCoordinating centers

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International Blood Disorder Centers

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Acknowledgements

• Hani Atrash • Mike Soucie• Althea Grant• Bruce Evatt• Melissa Creary• Ann McClellan• Christopher Parker• Susan Shurin NHLBI