Efficacy and safety of chloroquine plus prednisone for the treatment of autoimmune hepatitis in a randomized trial

Lydia T de Moraes Falcão; Debora R B Terrabuio; Marcio A Diniz; Andreia da Silva Evangelista; Fabricio G Souza; Eduardo L R Cancado

doi:10.1002/jgh3.12258

Efficacy and safety of chloroquine plus prednisone for the treatment of autoimmune hepatitis in a randomized trial

JGH Open. 2019 Sep 10;4(3):371-377. doi: 10.1002/jgh3.12258. eCollection 2020 Jun.

Authors

Lydia T de Moraes Falcão¹, Debora R B Terrabuio¹, Marcio A Diniz², Andreia da Silva Evangelista¹, Fabricio G Souza¹, Eduardo L R Cancado^{1

3}

Affiliations

¹ Division of Gastroenterology and Hepatology Hospital das Clinicas, University of São Paulo School of Medicine São Paulo Brazil.
² Biostatistics and Bioinformatics Research Center Samuel Oschin Comprehensive Cancer Institute, Cedars Sinai Medical Center Los Angeles California USA.
³ Laboratory of Medical Investigation of Immunopathology of Schistosomiasis (LIM 06) Institute of Tropical Medicine of University of São Paulo São Paulo Brazil.

Abstract

Background and aim: Standard treatment for autoimmune hepatitis (AIH) consists of predniso(lo)ne and azathioprine. However, alternative therapy is required for non- or partial responders and in cases of side effects. The aim of this study was to evaluate the treatment outcomes associated with chloroquine plus prednisone in AIH patients.

Methods: Fifty-seven patients were recruited to receive either azathioprine or chloroquine, both with prednisone, in a randomized trial. The primary end-point was complete remission, based on normalization of aminotransferase levels in the first 6 months of treatment plus maintenance for at least 18 months, with minimal or no inflammatory activity in the liver biopsy. Secondary end-points were partial and nonresponse, severe side effects, and treatment withdrawal.

Results: There were no differences between groups regarding clinical, serological, histological, and treatment characteristics at baseline. There were no significant differences in the biochemical response rate (67.7 vs 53.8%, P = 0.41) or the complete remission rate (32.26 vs 15.38%, P = 0.217). However, despite the long study period, the sample size was smaller than that required for a noninferiority study. The mean prednisone dose was similar in both groups. There was a nonsignificantly higher rate of adverse effects and a tendency toward improvement in glycemic and cholesterol profiles in the chloroquine group (P = 0.09 and P = 0.07, respectively).

Conclusions: The combination of chloroquine and prednisone exhibited potentially beneficial effects in AIH patients (https://ClinicalTrials.gov: NCT02463331).

Keywords: antimalarial drug; autoimmune hepatitis; chloroquine; remission.

Associated data

ClinicalTrials.gov/NCT02463331