Comprehensive analyses of neurodevelopmental outcomes and quality of life of children with biliary atresia

ObjectivesTo holistically evaluate neurodevelopmental outcomes and quality of life (QOL) of Japanese patients with biliary atresia (BA) and to investigate the factors associated with the outcomes.MethodsThis study enrolled patients with BA aged 5-18 years who visited Osaka University Hospital in 2021. Neurodevelopmental assessments were performed to evaluate intellectual ability, cognitive functions and adaptive skill levels. Furthermore, emotional and behavioral issues, characteristics of attention deficit hyperactivity disorder, and QOL were concomitantly assessed in the same cohort. Biochemical and social factors associated with the results were examined.ResultsFifty-three patients, with a median age of 11.2 years were included in the analyses. Patients with BA had a significantly lower Full-Scale Intelligence Quotient or developmental quotient (FSIQ/DQ) score and Vineland Adaptive Behavior Scale (VABS) composite score than the general Japanese population. Household education level and short stature were associated with low and borderline FSIQ/DQ and VABS composite scores, respectively. Among patients with low and borderline FSIQ/DQ scores, those with average or high VABS composite scores received significantly less neuroeducational care than those with low and borderline VABS composite scores. Despite the low FSIQ/DQ and VABS composite scores, the total QOL scores were higher than those of the general population.ConclusionPatients with BA had intellectual and behavioral impairments. Notably, patients with intellectual impairments are overlooked and not followed up, especially if adaptive skills are maintained. With the improvement in prognosis through Kasai surgery and liver transplantation, the focus has shifted to neurodevelopmental outcomes and quality of life of patients with biliary atresia. Japanese patients with biliary atresia exhibited intellectual and behavioral impairments that were positively correlated, though some patients showed discrepancies between the two aspects. Patients with low and borderline intellectual impairments, particularly those with unimpaired adaptive skills, were overlooked and did not receive neuroeducational care at school and medical institutions. image What is Known The prognosis of biliary atresia (BA) has improved due to Kasai portoenterostomy and liver transplantation. The neurodevelopmental outcome in patients with BA varies according to studies. No significant difference in neurodevelopmental outcomes were observed between patients with native liver and those who had undergone liver transplantation.What is New Japanese patients with BA exhibited intellectual and behavioral impairments that were positively correlated, though some patients showed discrepancies between the two aspects. The total quality of life child self-report score and the attention deficit hyperactivity disorder rating scale were correlated with adaptive skills score but not with intellectual functioning score. Patients with BA with borderline intellectual impairments, particularly those with unimpaired adaptive skills, were overlooked and did not receive neuroeducational care at school and medical institutions.