TM National Center on Birth Defects and Developmental Disabilities Kernicterus Surveillance Presented at the FDA Pediatric Advisory Committee Meeting on.

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National Center on Birth Defects and Developmental DisabilitiesNational Center on Birth Defects and Developmental DisabilitiesNational Center on Birth Defects and Developmental DisabilitiesNational Center on Birth Defects and Developmental Disabilities

Kernicterus Surveillance Kernicterus Surveillance

Presented at the FDA Pediatric Advisory Committee Meeting onCurrent Epidemiology and Therapeutic Interventions Relevant to

Hyperbilirubinemia in the Term & Near-term NewbornWashington DC: June 11, 2003

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Overview: Framework for DD surveillanceOverview: Framework for DD surveillance

• Health education & prevention strategies

• Public policy

• Monitor prevalence rates, trends, & prevention programs

• Provide registry of cases

Surveillance

Systems

Epidemiological Studies

Prevention Programs

• Identify risk & protective factors

• Address public concerns

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Definitions– Attributable to an impairment in physical,

cognitive, speech or language, psychological, or self-care areas

Measurement – Not always straight forward, especially for

conditions that rely on a behavioral patterns Population-based– Limited surveillance and not always well defined

Above issues can make generalizing results difficult or impossible to interpret

Overview: Complexities of DD surveillanceOverview: Complexities of DD surveillance

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Issues: Complexities of KI surveillanceIssues: Complexities of KI surveillance

Range of impairment and associated conditions– Brain damage that is associated with athetoid

cerebral palsy, hearing loss, vision impairment, dental dysplasia, and sometimes mental retardation

Changes in level of awareness and use of diagnosis over time

Variability in how cases are diagnosed– Physical markers and behavioral pattern utilized

for identification Early onset but often delayed diagnosis

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No systematic population-based surveillance in place to monitor kernicterus or hyperbilirubinemia

Case reports from convenience samples or select populations (e.g., medical insurance records)

No accepted standard for surveillance definition

Historical perspectives: KI surveillanceHistorical perspectives: KI surveillance

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True population estimates unknown to date Case reports from a convenience sample– Johnson, Bhutani, Brown (2002). 90 cases

reported from 1984-Jan 2001 Rates versus numbers

Prevalence of kernicterusPrevalence of kernicterus

– Rate = # of people with condition

# of people in the population Is kernicterus on the rise?

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Summary of issues for KI surveillanceSummary of issues for KI surveillance

Case definition– Debate over cut-offs for inclusion

Low prevalence condition– Requires a substantial population to detect cases

Lack of recognition– Acute event with specific features– Permanent damage, long-term clinical features

Litigation– Possible deterrent for case identification

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Evaluation of extant databases– National Hospital Discharge Data

Preponderance of hyperbilirubinemia codes KI codes not readily used

– MADDSP surveillance Examined athetoid CP cases

Kernicterus is not a reportable condition– CSTE approached regarding national reporting

Managed Care System– Proposed cooperative agreement– Limited interest

Preliminary actions: CDCPreliminary actions: CDC

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PERT announcement 2002 Objectives– Applicant should seek to review cases of extreme

jaundice in otherwise healthy full-term infants – Provide a body of evidence to inform why cases of

extreme jaundice may lead to kernicterus and why kernicterus may be re-emerging

– A forum of concerned scientists and health-care professionals will be convened to develop a strategic plan for national kernicterus prevention

Current activities: CDC surveillance effortsCurrent activities: CDC surveillance efforts

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UMDNJ Robert Wood Johnson Medical SchoolPrincipal Investigators: T. Hegyi, B. Ostfeld, A. Petrova

Infant mortality and morbidity of kernicterus Surveillance Identification of risk factors (clinical & systemic) Support network

Objectives of award recipientObjectives of award recipient

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UMDNJ Robert Wood Johnson Medical SchoolPrincipal Investigators: T. Hegyi, B. Ostfeld, A. Petrova

IRB submissions and approvals Initial discussion & process for population-based

surveillance with the NJ Dept of Health Analyses of extant data on infant morbidity and

mortality due to kernicterus

Progress to dateProgress to date

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Preliminary results: MorbidityPreliminary results: Morbidity

UMDNJ Robert Wood Johnson Medical SchoolPrincipal Investigators: T. Hegyi, B. Ostfeld, A. Petrova

1992 – 2001(NJ Hospital Discharge Data)– 82 cases of kernicterus– 7.5/100,000 live births (cumulative incidence)– Significant variation by race and ethnicity with the

lowest rate among Hispanics and the highest rate among Asians

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Pennsylvania Hospital, University of PennsylvaniaPrincipal Investigators: V. Bhutani & L. Johnson

Surveillance Identification of risk factors (clinical & systemic) Prevention Taskforce/Steering Committee National Prevention Campaign

Objectives of award recipientObjectives of award recipient

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Pennsylvania Hospital, University of Pennsylvania Principal Investigators: V. Bhutani & L. Johnson

Initial Advisory Board teleconference Peer Review Group meeting for consensus on

kernicterus definition (planned) Database development Collaboration with PICK on kernicterus

educational video

Progress to dateProgress to date

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Future directionsFuture directions

Partner for prevention– American Academy of Pediatrics, AWHONN,

HRSA, JCAHO, March of Dimes, NICHD, PICK, University researchers, others…

Forum for developing consensus on surveillance case definition

Identify a mechanism for population-based surveillance– State– National

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Working to prevent kernicterusWorking to prevent kernicterus

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