An update on cystic fibrosis Hepato-biliary involvement
A Jay Freeman1, Frank A J A Bodewes2
1Division of Pediatric Gastroenterology, Hepatology and Nutrition, Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus, OH, United States.
Summary
Cystic Fibrosis Hepato-Biliary Involvement (CFHBI) is now classified by phenotype, improving understanding and management. Research is advancing screening, evaluation, and treatment, with ongoing studies on modulator therapies
Area of Science:
- Hepatology
- Gastroenterology
- Pulmonology
Background:
- Liver involvement in cystic fibrosis (CF) is significant but historically lacked consistent terminology and understanding.
- Previous challenges included poor natural history data and inadequate clinical trial endpoints for CF-related liver disease.
- Recent initiatives have established standardized classification and terminology for CF liver conditions.
Purpose of the Study:
- To review recent advancements in understanding and managing Cystic Fibrosis Hepato-Biliary Involvement (CFHBI).
- To summarize current knowledge on the impact of modulator therapies on CFHBI.
- To identify outstanding research questions for CFHBI.
Main Methods:
- Review of longitudinal studies (e.g., PUSH) and national patient registry data.
- Assessment of non-invasive markers (serological, fibrosis indices, elastography) for CFHBI screening and progression monitoring.
- Analysis of emerging data on modulator therapies' effects on CFHBI.
Main Results:
- A new phenotype-based classification, Cystic Fibrosis Hepato-Biliary Involvement (CFHBI), and advanced CF liver disease (aCFLD) have been defined.
- Evidence-based consensus recommendations for CFHBI screening, evaluation, and management in children have been developed.
- Modulator therapies may be reducing aCFLD rates, but their full impact on CFHBI requires further investigation.
Conclusions:
- Standardized classification and consensus recommendations are improving CFHBI care.
- Further research is needed to fully elucidate the impact of modulator therapies on CFHBI.
- Addressing outstanding questions is crucial for advancing CFHBI research and clinical practice.
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