Transcript Adult SCD as a public health issue

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
No
symptoms
Mild
anemia
(thal
variants)
Jaundice
NBS
Early
childhood
Pain
Anemia
Aplastic
crises
Hand foot
syndrome
Organomegaly
Pediatric
Adolescent
Young Adult
Adult
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
TCD, HU
HU,, Transplant
Older
Adults
Disabilities
Chronic
Lung
Kidney
Heart
Eye
Infections
Transfusions
Co- Morbid conditions
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, 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
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Health Promotion through
Comprehensive care
Comprehensive Care
Centers
Registry
Education
and
prevention
messages
Surveillance
Diagnosis:Disease burden
Trends in health
Emerging/existing risks
Hemovigilance
Surveillance maps
Comprehensive care
Registry and
Prospective
Surveillance
Health
Systems
Research
Comprehensive
Care Centers
Public Health
Research
Education and
prevention
messages
Clinical and
basic research
Quality
indicators
Training program
support
New Drug
Development
Blood safety
Access to
specialty care
and laboratory
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 Capacity
on Hemophilia Adulthood
4
3.5
<13 yrs
>19 yrs
Ratio (P/Pc)
3
2.5
2
1.5
1
0.5
0
<$2,000
$2,000 – $10,000
>$10,000
Per Capita Gross National Product
Evatt & Robillard., Haemophilia (2000)
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Effect of Care Programs
on Hemophilia Adulthood
1.6
Ratio (P/Pc)
1.4
<13 yrs
>19 yrs
1.2
1
0.8
0.6
0.4
0.2
0
<$2,000
<$2,000 + HTC
Per Capita Gross National Product
Evatt & Robillard., Haemophilia (2000)
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Effect of Comprehensive Care
Without
Comprehensive Care
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•
•
•
•
Life Expectancy
Days in hospital
Percent with Joint disease
Annual Visits to Clinic
Unemployment
13-40
12-15
70-80%
12-30
35-90%
With
Comprehensive Care
65
1
<5%
0-6
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 Surveillance
World 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 Surveillance
Evatt 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
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•
•
•
•
•
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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
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 System
McClellan A. Division of Blood Disorders. NCBDDD.CDC
Hospitals with
Emergency
Departments
Hospitals with
ED and patients
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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
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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Bleeding
Hematology Specialty Care:
Future Blood Disorders Center Model
Lifespan issues
Complications
Hemovigilance
Management
Research
Disparities
Advocacy groups
S Shurin & R Kulkarni
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Blood Disorders Center Model
Guam
Coordinating centers
Puerto Rico
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International Blood Disorder
Centers
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Acknowledgements
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Hani Atrash
Mike Soucie
Althea Grant
Bruce Evatt
Melissa Creary
Ann McClellan
Christopher Parker
Susan Shurin NHLBI
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