BILACO Trial: Biliary Atresia - a Severe Complex Congenital Liver Disease With High Mortality, Compromised Neurological Development, Severe Malnutrition and Unknown Etiology

What this Trial Is About

Biliary atresia is a severe liver disease affecting children, leading to high illness and death rates, with symptoms like intense itching, fatigue, poor growth, liver failure, and impaired brain development. The cause is mostly unknown, and over half of affected children need a liver transplant during childhood. Determining the best time for transplantation is difficult due to lack of consensus based on clinical tools. This research aims to find risk factors for brain-related problems in these children to help decide the optimal transplant timing from a neurological perspective. The study will observe 75 children aged 0 to 18 years with biliary atresia in Denmark, along with 30 healthy children and 20 with tetralogy of Fallot as comparison groups. Participants will undergo detailed evaluations including liver function tests, genetic profiling, nutrition and immune status assessments, brain imaging (MRI), and neurocognitive tests at diagnosis, ages 2, preschool, pre-teen, and teenage. Additional neurocognitive tests will be done if a liver transplant occurs. The study involves advanced monitoring of brain, heart, gut, and immune functions. Children in the study will have regular assessments of their neurocognitive abilities using various tests and brain MRIs at multiple ages. Liver and heart function will be closely evaluated through imaging and lab tests. Researchers will also analyze genetics and microbiome samples. The main outcomes include brain MRI and multiple neurocognitive tests to evaluate development and function. The study is observational and will follow participants over many years to track changes and outcomes related to biliary atresia and transplantation timing.

BILACO Trial: Biliary Atresia - a Severe Complex Congenital Liver Disease