Abstract: Objective This study aims to assess therapeutic efficacy and safety of mycophenolate mofetil (MMF) in rescue treatment of patients with refractory autoimmune hepatitis (rAIH). Methods 23 patients with rAIH were encountered in our hospital between January 2015 and June 2021, all were treated with MMF and methylprednisolone combination, doses were gradually tapped according to the improvement, and the regimen lasted for up to 20 months and followed-up for two years. Liver biopsies were carried out before and after treatment. Univariate and multivariate Logistic regression analysis were applied to predict impacting factors of response. Results By end of treatment, serum ALT, AST and IgG levels in 23 patients with rAIH were (63.7±13.4)U/L,(54.1±14.5)U/L and (13.8±3.5)g/L, all significantly decreased as compared to [(167.3±43.1)U/L, (124.8±22.6)U/L and (17.4±4.4)g/L, respectively, P<0.05] at presentation; liver tissue fibrosis score was (2.0±1.1) and hepatic activity index (HAI) was (1.5±1.3), both much lower than [(2.7±1.6) and (2.4±1.2), P<0.05] before treatment; by end of follow-up, complete clinical remission (CCR) was obtained in 18 cases and incomplete response (IR) in 5 cases; at enrollment, female proportion, serum ALT and IgG levels, liver fibrosis and HAI scores in patients with CCR were significantly lower than in those with IR (P<0.05); multivariate Logistic regression analysis showed that female (OR=1.04, 95%CI=0.9-1.5), serum IgG level (OR =1.17, 95%CI=1.0-2.3) and hepatic fibrosis score (OR =4.4, 95%CI=1.8-9.3) were all the independent factors impacting response to rescue therapy. Conclusion Rescue therapy with MMF is safe, reliable and efficacious in the treatment of patients with rAIH, which is worth further clinical trial to verify.

Key words: Autoimmune hepatitis, Refractory, Mycophenolate mofetil, Prednisolone, Rescue therapy