Mycophenolate for the treatment of autoimmune hepatitis: Prospective assessment of its efficacy and safety for induction and maintenance of remission in a large cohort of treatment-naive patients

Background & Aims: Standard therapy for autoimmune hepatitis (AIH) is corticosteroids with or without azathioprine. However, 20% of patients do not respond or are intolerant to conventional treatment. Therefore, we evaluated prospectively the efficacy and safety of mycophenolate mofetil (MMF) in inducing and/or maintaining remission in treatment-naive AIH patients. Methods: Fifty-nine treatment-naive patients with well defined AIH were treated with prednisolone plus 1.5-2 g/d of MMF. Patients were candidates for MMF withdrawal after at least 4 years. Treatment outcomes were defined according to the International Autoimmune Hepatitis Group report. Results: Treatment duration with MMF was 26 months (range 3 92). Eighty-eight percent (52/59) of patients responded initially clinically and biochemically (normalization of transaminases and gamma-globulins) most of them within 3 months. The remaining 7 patients (12%) had partial response. In total, 59.3% (35/59) of patients had complete response (CR) with 37% (22/59) of them having achieved CR off prednisolone, while 28.8% (17/59) had initial CR with relapses. No patient was non-responder. Prednisolone withdrew in 57.6% (34/59) of patients in 8 months. The only independent predictor of treatment outcome, was gamma-GT (baseline gamma-GT, p = 0.008 and gamma-GT on month 24, p < 0.05). Severe side effects leading to MMF discontinuation occurred in only 3.4% (2/59) of patients. Six patients (2 according to protocol and 4 for personal reasons), stopped treatment with MMF, but 3 relapsed. Conclusions: MMF seems safe and effective as first-line therapy in inducing and maintaining remission in treatment-naive patients with AIH, having a significant and rapid steroid sparing effect as attested by the fact that so far, 37% (22/59) of AIH patients achieved CR off prednisolone. (C) 2011 European Association for the Study of the Liver. Published by Elsevier B.V. All rights reserved.