Development and Validation of a Novel Fibrosis Marker in Biliary Atresia during Infancy

OBJECTIVES: Most biliary atresia (BA) patients suffer from liver fibrosis and often require liver transplantation. The aim of this study was to develop and validate a novel fibrosis marker for BA patients aged <1 year—the infant BA liver fibrosis (iBALF) score—subsequent to the previously reported fibrosis marker for BA patients aged ≥1 year. METHODS: From three institutions for pediatric surgery, BA patients and their native liver histology examinations performed at the age of <1 year were retrospectively identified and assigned to a development cohort (58 patients and 73 examinations) or validation cohort (92 patients and 117 examinations) according to their institutions. Histological fibrosis stages (F0–F4), blood test results, and clinical information at the time of liver histology examination were reviewed. The iBALF score was determined using multivariate ordered logistic regression analysis and was assessed for its associations with histological fibrosis stages. RESULTS: The iBALF score equation was composed of natural logarithms, including serum total bilirubin level, blood platelet counts, and days of age. The score revealed a strong correlation with fibrosis stage (r=0.80 and 0.73 in the development and validation cohorts, respectively; P<0.001). The areas under the receiver-operating characteristic curves for diagnosing each fibrosis stage were 0.86–0.94 in the development cohort and 0.86–0.90 in the validation cohort (P<0.001), indicating good diagnostic power. In addition, no patient with an iBALF score >6 (equivalent to F4) at the initial surgery survived with their native liver at 1 year of age (n=9). CONCLUSIONS: The iBALF score that was developed was a good noninvasive marker of native liver fibrosis for BA patients aged <1 year.