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Multicenter Study
. 2022 Sep;76(3):712-726.
doi: 10.1002/hep.32451. Epub 2022 Apr 18.

Risk of variceal hemorrhage and pretransplant mortality in children with biliary atresia

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

Risk of variceal hemorrhage and pretransplant mortality in children with biliary atresia

Lee M Bass et al. Hepatology. 2022 Sep.

Abstract

Background and 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 multicenter study.

Approach and 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 on 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, 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, whereas 10 (12.5%) underwent transplant within 6 weeks of VH.

Conclusions: 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.

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Conflict of interest statement

Dr. Miethke consults for Mirum and Metacrine. Dr. Ng consults for Albireo. Dr. Murray consults for Gilead and Albireo. Dr. Loomes consults for and received grants from Mirum and Albireo. Dr. Molleston received grants from Mirum, AbbVie, Gilead, and Albireo. Dr. Rosenthal consults for and received grants from Takeda/Vertex, Gilead, AbbVie, Retrophin, Albireo, Mirum, and Travere. He consults for Audentes and Dicerna. He received grants from Merck and Arrowhead. Dr. Karpen consults for Albireo, Intercept, and Mirum. Dr. Sokol consults for Albireo and Mirum.

Figures

FIGURE 1
FIGURE 1
Flow chart for participants in both BASIC and PROBE. Abbreviation: LFU, long‐term follow‐up
FIGURE 2
FIGURE 2
Kaplan‐Meier curve for transplant‐free survival in each cohort based on number of features of PHT
FIGURE 3
FIGURE 3
Kaplan‐Meier curve demonstrating transplant‐free survival in PROBE by functioning versus nonfunctioning HPE, based on number of features of PHT
FIGURE 4
FIGURE 4
(A) Cumulative incidence curve for first observed VH in BASIC and PROBE based on number of features of PHT. (B) Cumulative incidence curve for first observed VH in PROBE by functioning versus nonfunctioning HPE based on number of features of PHT
FIGURE 5
FIGURE 5
Risk factors at baseline significantly associated with VH in both BASIC and PROBE
See this image and copyright information in PMC

References

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