Biliary atresia is a neonatal cholangiopathy of unknown etiology that affects 1 in 8,000 to 18,000 newborns.1, 2 It is the most frequent indication for liver transplantation in children accounting for almost 50% of pediatric liver transplants in the United States.1, 2 Early surgery with hepatoportoenterostomy (HPE) to relieve extrahepatic biliary obstruction and restore bile flow is critical. Despite surgery, however, progressive hepatic fibrosis often ensues and liver transplantation is eventually necessary in 70%-80% of patients. At least half of infants with biliary atresia are likely to undergo liver transplantation within the first 2 years of life.3–5
The evaluation of outcomes in infants with biliary atresia after HPE has been one focus of the Biliary Atresia Research Consortium (BARC), which was formed in 2002 as a National Institutes of Health (NIH)-supported network of 9 pediatric clinical centers and a data coordinating center. BARC has previously reported that survival to 24 months of age with native liver in children with biliary atresia was predicted by lower serum total bilirubin concentrations (<2 mg/dL compared to >6 mg/dL) at 3 months after HPE.6 However, less than half of the entire cohort who required transplantation or died had a total serum bilirubin >6 mg/dL at 3 months after HPE. Clearly, other factors that predict rapid progression to end-stage liver disease need to be identified.
Malnutrition is a significant clinical problem in children with biliary atresia7–16 particularly during the first year of life and indeed has been shown to increase mortality risk once a patient has been listed for liver transplantation. Recognition of this risk is best illustrated by the inclusion of growth failure as a parameter in the Pediatric End-Stage Liver Disease (PELD) scoring system used to rank candidates awaiting liver transplantation in the United States.7 It has been proposed that maintaining optimal nutritional status in young biliary atresia patients might improve outcomes before and after liver transplantation.13–15
The aims of this study were to evaluate growth in infants with biliary atresia and to determine growth patterns over the first 2 years of life that were associated with an increased risk for early liver transplantation or death. This study is unique because it is the first multicenter assessment of growth after HPE in a large cohort of infants with biliary atresia in the United States.