PAEDIATRIC autoimmune liver disease (AILD) shows excellent short-term survival but limited complete biochemical control after one year of standard therapy, according to prospective European registry data. AILD includes conditions such as autoimmune hepatitis (AIH) and autoimmune sclerosing cholangitis (ASC).
The condition involves chronic immune-driven inflammation of the liver that can progress to fibrosis and cirrhosis if not adequately controlled.
The findings highlight a key clinical challenge: while most children respond biochemically to treatment, a substantial proportion do not achieve full normalisation of key disease markers within the first year of care.
Registry Data Reveals Real-World Paediatric AILD Outcomes
The study used data from the European Reference Network R-LIVER Registry, including 116 patients under 18 years diagnosed with AIH or ASC between 2017 and 2023, all with more than 12 months of follow-up. At diagnosis, 27% already had cirrhosis and 14% had inflammatory bowel disease. Large duct disease was present in 45% of ASC cases.
Most patients (94%) started standard therapy with the corticosteroid prednisolone, with or without thiopurines, and 80% remained on this regimen throughout the first year.
alanine aminotransferase normalisation was achieved in 72% of cases at six months and 78% at 12 months. However, only 45% achieved both normal ALT and immunoglobulin G levels after one year.
Complete biochemical control was reached in 42% of patients at both six and 12 months. All patients were alive at the end of follow-up, although two required liver transplantation due to advanced disease.
Disease Type and Severity Linked to Outcomes
ASC and cirrhosis at diagnosis were both associated with failure to achieve complete biochemical control. This suggests that ASC as a disease phenotype, alongside established liver damage in cirrhosis, may influence early response to standard therapy.
No differences in presentation or outcomes were observed between AIH types 1 and 2, (71 and 8 patients respectively).
Diagnosis followed European Society for Paediatric Gastroenterology, Hepatology and Nutrition criteria, incorporating autoantibody testing, elevated immunoglobulin G, compatible histology, exclusion of other liver diseases, and cholangiography using magnetic resonance cholangiopancreatography to assess biliary involvement.
Implications for Paediatric Liver Disease Management
While survival outcomes in paediatric AILD are reassuring in the short term, the study highlights a persistent gap in achieving complete biochemical control within the first year of therapy. Children presenting with ASC or cirrhosis appear particularly less likely to achieve optimal disease control under standard regimens.
These findings point towards a need for improved and potentially more tailored therapeutic strategies in paediatric autoimmune liver disease, particularly for those presenting with more advanced or complex disease at diagnosis.
Reference
Malham M et al. Paediatric autoimmune liver disease in Europe, the prospective ERN R-Liver registry. JHEP Rep. 2026;DOI:10.1016/j.jhepr.2026.101855.
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