Tacrolimus in difficult-to-treat paediatric autoimmune hepatitis: a viable alternative

Objectives The objective of the study was to assess the safety and outcomes of tacrolimus in children unresponsive or intolerant to standard therapy with azathioprine and corticosteroids. Method Single-centre retrospective study identified paediatric patients with autoimmune hepatitis (AIH) treated with tacrolimus between 2010 and 2024. The study evaluated clinical and biochemical responses, outcomes at 6 months, 12 months and final follow-up. Results Tacrolimus was initiated as a second-line agent in 8, third line in 3 and fourth line in 1 of 12 children. Indications included non-response (n=10) and intolerance (n=2) to prior therapy. The median age at initiation was 14.5 years (IQR 13–16), and the median interval from diagnosis to tacrolimus initiation was 14 months (IQR 9–60). Progression of fibrosis compared with baseline was observed in 72% (n=8) at initiation. Complete biochemical remission was achieved in 66% (n=8) within a median of 47 days (IQR 34–117), and 58% (n=7) maintained remission at 12 months. Those not in remission (42%, n=5) demonstrated partial biochemical response with statistically significant reductions in liver enzymes at 12 months. Tacrolimus was well tolerated, with no significant adverse effects or discontinuation. Conclusion Tacrolimus can be a valuable alternative as a second-line or third-line option, particularly in specialised centres, for a specific subset of children with AIH. Prospective multicentre studies are needed to confirm long-term efficacy and define predictors of response.