Comprehensive care and Sickle Cell Disease and Hemoglobinopathies

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Comprehensive care and Sickle Cell Disease and
Hemoglobinopathies

• Roshni Kulkarni MD.
• Distinguished Hematology Consultant
• Division of Blood Disorders
• National Center on Birth Defects and
  Developmental Disabilities
• Centers 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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SCD issues across life span
0-6mo Early childhood Early childhood Pediatric Adolescent Young Adult Adolescent Young Adult Adult Older Adults
No symptoms Mild anemia (thal variants) Jaundice Pain Anemia Aplastic crises Hand foot syndrome Organo-megaly 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 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 HU,, Transplant HU,, Transplant HU,, Transplant
Infections Infections Infections Infections Infections Infections Infections
Transfusions Transfusions Transfusions Transfusions Transfusions Transfusions
Co- Morbid conditions Co- Morbid conditions Co- Morbid conditions
Quality of Life? Nutrition? Mental health? Vocation? Academic achievement? Quality of Life? Nutrition? Mental health? Vocation? Academic achievement? Quality of Life? Nutrition? Mental health? Vocation? Academic achievement? Quality of Life? Nutrition? Mental health? Vocation? Academic achievement? Quality of Life? Nutrition? Mental health? Vocation? Academic achievement? Quality of Life? Nutrition? Mental health? Vocation? Academic achievement? Quality of Life? Nutrition? Mental health? Vocation? Academic achievement? Quality of Life? Nutrition? Mental health? Vocation? Academic achievement?
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,
2009110908, 2007. 2. Bryant AS. Obstetrics
Gynecology, 2007
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Public Health SCDReduce 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 (contd)

• 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

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Health Promotion through Comprehensive care
Comprehensive Care Centers
Registry
Surveillance
DiagnosisDisease burden Trends in
health Emerging/existing risks Hemovigilance Surve
illance maps
Education and prevention messages
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Comprehensive care
Health Systems Research
Clinical and basic research Quality
indicators Training program support
Registry and Prospective Surveillance
Comprehensive Care Centers
Public Health Research
New Drug Development Blood safety
Education and prevention messages
Access to specialty care and laboratory
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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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Economics The Primary Force in Available Care
In countries with emerging economies, economic
issues will be the force determining how far we
can push our goals In developed countries, only
constant pressure prevents reduction in
services. We must understand these issues and
use them to our advantage
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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
lt2,000
2,000 10,000
gt10,000
Per Capita Gross National Product
Evatt Robillard., Haemophilia (2000)
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Effect of Care Programson Hemophilia Adulthood
lt2,000
lt2,000 HTC
Per Capita Gross National Product
Evatt Robillard., Haemophilia (2000)
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Effect of Comprehensive Care
Without Comprehensive Care
With Comprehensive Care

• Life Expectancy 13-40 65
• Days in hospital 12-15 1
• Percent with Joint disease 70-80 lt5
• Annual Visits to Clinic 12-30 0-6
• Unemployment 35-90 15

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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

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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
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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

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SCD and Hemophilia
Hemophilia SCD
Expensive 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
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Blood Disorders Center Model
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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