Prospective European registry data indicates that Paediatric Autoimmune Liver Disease (AILD) exhibits excellent short-term survival but struggles with complete biochemical control after one year of standard therapy. AILD encompasses conditions like autoimmune hepatitis (AIH) and autoimmune sclerosing cholangitis (ASC).
The findings from the European Reference Network R-LIVER Registry tracked 116 patients under 18 diagnosed between 2017 and 2023. At diagnosis, a significant portion presented with cirrhosis (27%) or inflammatory bowel disease (14%).
Most patients (94%) began treatment with prednisolone, often combined with thiopurines. While 78% achieved normal alanine aminotransferase (ALT) levels by 12 months, only 45% normalized both ALT and immunoglobulin G (IgG) levels. Complete biochemical control was achieved in just 42% of patients at both six and 12 months. All patients survived, though two required liver transplants for advanced disease.
Autoimmune sclerosing cholangitis (ASC) and cirrhosis at diagnosis were linked to a failure to achieve complete biochemical control. This suggests these factors may impact early treatment response.
The study highlights a critical gap in managing paediatric autoimmune liver disease, underscoring a need for improved, tailored therapeutic strategies, especially for those diagnosed with more advanced conditions.