First Results From a Propensity Matching Trial of Mycophenolate Mofetil vs. Azathioprine in Treatment-Naive AIH Patients

Abstract

Background/AimsAs previous real-world studies and meta-analyses have shown that mycophenolate mofetil (MMF) might have better efficacy than azathioprine (AZA) in autoimmune hepatitis (AIH), we conducted a propensity matching study to assess the efficacy and safety of MMF vs. AZA.MethodsAll 126 consecutive treatment-naive adult AIH patients, diagnosed and followed in our department since 2016, were included. Patients received prednisolone 0.5–1 mg/kg/day plus either AZA 1–2 mg/kg/day or 1.5–2 g/day MMF. The tapering of prednisolone was identical between groups.ResultsAfter propensity matching score and adjustment for known factors affecting response to treatment and outcome, 64 patients were included in the study (MMF = 32 and AZA = 32). Rates of non-response, complete biochemical response (CBR) at 6 and 12 months, and prednisolone withdrawal (6 months, 12 months, and end of follow-up) were identical between groups. However, MMF treatment was significantly associated with CBR at the end of follow-up [odds ratio (OR) 11.259; 95% CI: 1.3–97.4, p = 0.028]. AZA patients were more prone to stop treatment due to AZA intolerance/insufficient response (p = 0.0001). At the end of follow-up, the overall efficacy of each schedule was also significantly higher in the MMF group compared to the AZA group (p = 0.0001).ConclusionWe showed for the first time in a propensity matching study that MMF can be used as first-line therapy in AIH as attested by the significantly higher CBR at end of follow-up compared to AZA. Whether this better efficacy is also associated with higher histological remission rates and sustained CBR off immunosuppression needs further evaluation.

Recent advances in the diagnosis and management of autoimmune hepatitis

“…To validate these previous findings, we reported for the first-time recently, a face-to-face comparison regarding the efficacy and safety of MMF vs. azathioprine by using a very strict propensity matching trial in treatment-naive AIH patients who received induction and maintenance therapy for 3-5 years [80]. Our findings revealed that MMF was significantly associated with higher CBR rates at the end of the study, whereas patients from the AZA group were more likely to discontinue treatment because of intolerance and/or insufficient response.…”

“…Our findings revealed that MMF was significantly associated with higher CBR rates at the end of the study, whereas patients from the AZA group were more likely to discontinue treatment because of intolerance and/or insufficient response. Furthermore, at the end of the study, the MMF group had significantly higher overall efficacy than the AZA group [80].…”

Assessing the efficacy and safety of mycophenolate mofetil versus azathioprine in patients with autoimmune hepatitis (CAMARO trial): study protocol for a randomised controlled trial

“…A select number of prospective studies evaluated MMF as first-line therapy in AIH. (29)(30)(31) Prospective randomised data on first-line therapy with MMF are lacking. Previous studies, mostly retrospective observational case series, have indicated that MMF is able to rescue azathioprine intolerant AIH patients, but not patients in whom azathioprine was unable to achieve remission.…”

Inflammatory bowel disease and immune-mediated inflammatory diseases: looking at the less frequent associations

“…In addition to that, recently, azathioprine has been proposed as an effective first-line therapy for AIH, 106 and this should be kept in mind by clinicians when managing patients with both IBD and AIH.…”