Evaluation of tacrolimus and mycophenolate mofetil as second-line therapy in autoimmune hepatitis – real-world data from the Charité Berlin cohort

Introduction: Azathioprine with steroids is considered the standard first-line treatment for autoimmune hepatitis (AIH). The CAMARO study suggests mycophenolate mofetil (MMF) with steroids being equally effective in treatment-naive AIH. The optimal second line therapy for AIH, e.g. efficacy and tolerability of tacrolimus, is less well defined.

Methods: We performed a retrospective single-centre study on second-line therapies for AIH. Criteria for exclusion were additional autoimmune liver diseases or liver transplantation. Biochemical parameters were collected at change of therapy, after 12 months and at last follow-up. Biochemical remission was defined as normalization of transaminases and immunoglobulin G.

Results: At Charité Berlin, 59 out of 490 AIH patients received MMF or tacrolimus as second-line therapy. Of 59 patients, 12 received tacrolimus (50% with cirrhosis) and 47 received MMF (19.1% with cirrhosis) (cirrhosis: p=0.036). Both groups showed significant ALT reductions after 12 months. Despite significantly higher levels of alanine aminotransferase (ALT) at change of therapy in the tacrolimus group (ALT/ULN 4.1 vs. 1.6 in the MMF group, p=0.031), biochemical remission was achieved in more patients on tacrolimus compared to MMF (66.6% vs. 50%, p=0.187). The median duration of second-line therapy was significantly longer with MMF (46 months) than with tacrolimus (25 months) (p=0.007). Drug discontinuation due to side effects occurred in 2 tacrolimus patients and 5 MMF patients.

Conclusion: Our results indicate that both MMF and tacrolimus are effective second-line therapies for AIH, with tacrolimus showing numerically higher remission rates and being used more frequently in patients with advanced and more active liver disease.

Publication History

Article published online:
20 January 2025