What to measure in biliary atresia research: study protocol for developing a core outcome set

Area of Science:

• Pediatric Gastroenterology
• Hepatology
• Clinical Trial Methodology

Background:

• Extrahepatic biliary atresia is a rare condition presenting research challenges.
• Heterogeneity in reported outcomes complicates research synthesis and evidence evaluation.
• Standardized reporting is essential for advancing the understanding and management of biliary atresia.

Purpose of the Study:

• To establish a consensus core outcome set for research in biliary atresia management.
• To standardize outcome reporting in clinical studies and systematic reviews.
• To improve the quality and comparability of evidence on biliary atresia treatments.

Main Methods:

• A comprehensive literature review will identify potential outcomes.
• A three-stage Delphi process involving diverse stakeholders will refine the outcome list.
• Consensus will be reached on a prioritized set of core outcomes for biliary atresia.

Main Results:

• (Study results not yet available, pending completion of the Delphi process.)
• The study aims to produce a finalized core outcome set.
• The developed set will reflect the priorities of patients, parents, and healthcare professionals.

Conclusions:

• A core outcome set is essential for guiding future biliary atresia research.
• Standardized outcomes will facilitate robust systematic reviews and evidence appraisal.
• This initiative will enhance the quality of research and clinical decision-making in biliary atresia.