Tacrolimus Shows Promise in Refractory Paediatric AIH : Frontline Gastroenterol | May 2026

Introduction

Autoimmune hepatitis (AIH) in children is typically managed with corticosteroids and azathioprine, achieving remission in most patients. However, a subset of paediatric patients remain refractory to standard therapy or develop significant treatment intolerance, creating a major therapeutic challenge with risk of progressive fibrosis and liver failure.

Problem Statement

Evidence supporting second-line immunosuppressive therapy in difficult-to-treat paediatric AIH remains limited, particularly regarding long-term efficacy and safety of tacrolimus. Optimal management strategies for children with incomplete response or intolerance to conventional therapy are not well established.

Summary

This single-centre retrospective study evaluated tacrolimus as rescue immunosuppression in children with refractory or treatment-intolerant autoimmune hepatitis. Most patients received tacrolimus because of inadequate biochemical response to standard therapy, while a smaller proportion required escalation because of medication intolerance. Notably, many children already demonstrated progression of hepatic fibrosis at the time tacrolimus was initiated, underscoring the aggressive nature of difficult-to-control paediatric AIH. Tacrolimus induced complete biochemical remission in approximately two-thirds of patients, with remission often achieved rapidly after treatment initiation. Importantly, even patients who failed to achieve full remission demonstrated significant biochemical improvement, suggesting clinically meaningful disease control despite persistent low-grade activity. The treatment was well tolerated overall, with no major adverse effects or treatment discontinuations observed during follow-up. These findings support tacrolimus as a viable second-line or third-line immunosuppressive strategy in carefully selected paediatric AIH patients managed within specialized hepatology centres. The study also reinforces the importance of early recognition of treatment-refractory disease, as ongoing inflammatory activity may contribute to progressive fibrosis despite conventional therapy. Although limited by retrospective design and small cohort size, the findings add important real-world evidence supporting calcineurin inhibitor–based rescue therapy in paediatric AIH and highlight the need for prospective multicentre studies to better define long-term outcomes, optimal therapeutic timing and predictors of response.