A Molecular Signature Predicts 2-Year Outcome of Biliary Atresia and Identifies a Treatment Target to Suppress Fibrosis

Background & Aims: Age at diagnosis and response to surgery have been correlated with an improved survival with the native liver in children with biliary atresia. However, biological predictors of outcome are largely undefined. We investigated if gene expression profiles at diagnosis predict clinical outcome. Methods: Global gene expression was quantified by RNAseq of liver biopsies from 171 infants with BA at diagnosis and randomly assigned into discovery (N=121), validation (N=50) cohorts and 7 normal controls. Overall design: mRNA profiles of 171 livers from patients with biliary atresia and 7 livers from normal controls by deep sequencing using Hiseq 2500