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Operational Definitions for Chronic Liver Disease Manifestations and Recurring Clinical Events in Biliary Atresia
Henry Shiau1, Jeremy M Schraw2, Laurel Cavallo1
1Division of Gastroenterology, Hepatology, and Nutrition.
Insights
Standardized definitions for chronic liver disease (CLD) events in biliary atresia (BA) were developed. These definitions help consistently track disease progression and identify patient outcomes in pediatric liver disease research.
Area of Science:
- Pediatric Hepatology
- Clinical Research Methodology
- Gastroenterology
Background:
- Biliary atresia (BA) disease progression is often complicated by chronic liver disease (CLD) manifestations and events like ascites and cholangitis.
- Lack of standardized operational definitions for these CLD manifestations/events hinders consistent identification in clinical research.
- This study aimed to develop research definitions for CLD manifestations/events in BA for retrospective analysis.
Purpose of the Study:
- To develop and validate operational research definitions for CLD manifestations and events in infants with biliary atresia (BA).
- To improve consistency in identifying and analyzing disease progression in pediatric CLD research.
- To establish definitions that can serve as endpoints in therapeutic trials and variables for multicenter studies.
Main Methods:
- Operational definitions for CLD manifestations/events were created through literature review and expert consensus from pediatric hepatologists.
- Definitions were applied to a single-center cohort of infants with BA post-Kasai procedure.
- Data on manifestations/events were collected until last clinical visit, liver transplantation (LT), or death; survival analyses and cluster analysis were performed.
Main Results:
- Of 65 infants with BA, 76% of CLD manifestations/events occurred within the first year.
- The presence of portal hypertension, thrombocytopenia, and ascites was linked to poorer transplant-free survival (P < 0.01).
- Cluster analysis identified three distinct outcome groups based on manifestation/event patterns: poor outcomes, stable native liver with events, and stable native liver with few/no events.
Conclusions:
- Developed operational definitions enable consistent tracking of disease progression in infants with BA.
- These research definitions can enhance consistency in future pediatric CLD studies.
- The definitions can be utilized as endpoints in clinical trials and variables for multicenter disease pattern identification.
Background:
The disease course of biliary atresia (BA) may be complicated by development of chronic liver disease (CLD) manifestations (eg, ascites) and recurring clinical events (eg, cholangitis). Most pediatric CLD-manifestations/events lack standardized operational definitions, leading to inconsistent identification in clinical research. This study aimed to develop operational research definitions of CLD-manifestations/events in BA for application in retrospective analysis.
Methods:
Operational definitions of CLD-manifestations/events were developed by literature review and revised by a panel of experienced pediatric hepatologists. Definitions were applied to a single-center review of infants with BA post-Kasai. Manifestations/events were captured until last clinical visit with native liver (SNL), liver transplantation (LT), or death. Native liver survival and event-free survival were estimated by Kaplan-Meier method. Cluster analysis was performed to identify similar patterns of manifestation/event development.
Results:
Of 65 infants with BA post-Kasai (2006-18; median Kasai 56.8 days; 65% girls), 29 underwent LT (median 12.9 months) and 4 died without LT (median 6.9 months). Seventy-six percent of CLD-manifestations/events presented within the first year. Presence of definite clinically evident portal hypertension, thrombocytopenia, and dCE ascites were associated with poor transplant-free survival (P < 0.01). Similar pattern developments of CLD-manifestations/events identified 3 outcome groups: poor outcomes (87.8% LT/death), SNL with manifestations/events, and SNL with few/no events.
Conclusions:
Operational definitions can provide a timely description of the disease course progression in infants with BA. Research definitions may provide better consistency in future pediatric CLD studies. Furthermore, definitions may serve as endpoints in therapeutic trials or used as variables for disease pattern identification in potential multicenter studies.
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