Copyright ©The Author(s) 2017. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastroenterol. Sep 7, 2017; 23(33): 6030-6048
Published online Sep 7, 2017. doi: 10.3748/wjg.v23.i33.6030
Autoimmune hepatitis: Standard treatment and systematic review of alternative treatments
Benedetta Terziroli Beretta-Piccoli, Giorgina Mieli-Vergani, Diego Vergani
Benedetta Terziroli Beretta-Piccoli, Epatocentro Ticino, 6900 Lugano, Switzerland
Giorgina Mieli-Vergani, Paediatric Liver, GI and Nutrition Centre, MowatLabs, King’s College Hospital, Denmark Hill, London SE5 9RS, United Kingdom
Diego Vergani, Institute of Liver Studies, MowatLabs, King’s College Hospital, Denmark Hill, London SE5 9RS, United Kingdom
Author contributions: Terziroli Beretta-Piccoli B contributed to writing up the manuscript; Mieli-Vergani G and Vergani D contributed to critical revision and final approval of the report.
Conflict-of-interest statement: No potential conflicts of interest.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See:
Correspondence to: Diego Vergani, Professor, Institute of Liver Studies, MowatLabs, King’s College Hospital, Denmark Hill, London SE5 9RS, United Kingdom.
Telephone: +44-20-32994643 Fax: +44-20-32994224
Received: June 6, 2017
Peer-review started: June 7, 2017
First decision: June 22, 2017
Revised: July 18, 2017
Accepted: August 2, 2017
Article in press: August 2, 2017
Published online: September 7, 2017

Autoimmune hepatitis is a rare chronic inflammatory liver disease, affecting all ages, characterised by elevated transaminase and immunoglobulin G levels, positive autoantibodies, interface hepatitis at liver histology and good response to immunosuppressive treatment. If untreated, it has a poor prognosis. The aim of this review is to summarize the evidence for standard treatment and to provide a systematic review on alternative treatments for adults and children. Standard treatment is based on steroids and azathioprine, and leads to disease remission in 80%-90% of patients. Alternative first line treatment has been attempted with budesonide or cyclosporine, but their superiority compared to standard treatment remains to be demonstrated. Second-line treatments are needed for patients not responding or intolerant to standard treatment. No randomized controlled trials have been performed for second-line options. Mycophenolate mofetil is the most widely used second-line drug, and has good efficacy particularly for patients intolerant to azathioprine, but has the major disadvantage of being teratogenic. Only few and heterogeneous data on cyclosporine, tacrolimus, everolimus and sirolimus are available. More recently, experience with the anti-tumour necrosis factor-alpha infliximab and the anti-CD20 rituximab has been published, with ambivalent results; these agents may have severe side-effects and their use should be restricted to specialized centres. Clinical trials with new therapeutic options are ongoing.

Keywords: Autoimmune hepatitis, Standard treatment, Second-line treatment, Adults, Children

Core tip: The first part of this review summarizes the standard therapeutic approach for autoimmune hepatitis (steroids and azathioprine) and the evidence on which it is based. The second part reviews systematically published data on first and second line alternative treatments. This information is summarized in two comprehensive tables, one for adult and one for paediatric patients.