https://pubmed.ncbi.nlm.nih.gov/35271743/ Biliary atresia

Hepatology. 2022 Mar 10.
doi: 10.1002/hep.32451. Online ahead of print.

The Risk of Variceal Hemorrhage and Pre-Transplant Mortality in Children with Biliary Atresia

Lee M Bass 1, Wen Ye 2, Kieran Hawthorne 3, Daniel H Leung 4, Karen F Murray 5 6, Jean P Molleston 7, Rene Romero 8, Saul Karpen 8, Philip Rosenthal 9, Kathleen M Loomes 10, Kasper S Wang 11, Robert H Squires 12, Alexander Miethke 13, Vicky L Ng 14, Simon Horslen 15, M Kyle Jensen 16, Ronald J Sokol 17, John C Magee 18, Benjamin L Shneider 19, ChiLDReN

Abstract

Background & aims: The natural history of gastroesophageal variceal hemorrhage (VH) in biliary atresia (BA) is not well characterized. We analyzed risk factors, incidence and outcomes of VH in a longitudinal multi-center study.

Approach & results: Participants enrolled in either an incident [Prospective Database of Infants with Cholestasis (PROBE)] or prevalent [Biliary Atresia Study of Infants and Children (BASIC)] cohort of BA were included. Variceal hemorrhage (VH) was defined based upon gastrointestinal bleeding in the presence of varices accompanied by endoscopic or nontransplant surgical intervention. Cumulative incidence of VH and transplant-free survival was compared based on features of portal hypertension (e.g. splenomegaly and thrombocytopenia) and clinical parameters at baseline in each cohort [PROBE - 1.5 to 4.5 months after hepatoportoenterostomy (HPE), BASIC - at enrollment >3 years of age]. Analyses were conducted on 869 children with BA enrolled between June 2004 and December 2020 [521 in PROBE (262 [51%] with a functioning HPE) and 348 in BASIC]. The overall incidence of first observed VH at 5 years was 9.4% (95% CI: 7.0-12.4%) in PROBE and 8.0% (5.2-11.5%) in BASIC. Features of portal hypertension, platelet count, total bilirubin, aspartate aminotransferase (AST), albumin, and AST to platelet ratio index at baseline were associated with an increased risk of subsequent VH in both cohorts. Transplant-free survival at 5 years was 45.1% (40.5-49.6%) in PROBE and 79.2% (74.1-83.4%) in BASIC. Two (2.5%) of 80 participants who had VH died, while 10 (12.5%) underwent transplant within six weeks of VH.

Conclusion: The low risk of VH and associated mortality in children with BA needs to be considered in decisions related to screening for varices and primary prophylaxis of VH.