To estimate the efficacy of mycophenolate mofetil (MMF) in myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD)

MOGAD is an emerging disease entity related to a wide range of inflammatory demyelination of the central nervous system. MMF is one of the commonly used immunotherapies in MOGAD. Previous observational studies showed mixed results. Herein, we performed a meta-analysis to evaluate the effect of MMF in MOGAD. 

A comprehensive search of the databases, including MEDLINE, EMBASE, and Cochrane, was performed from their inception dates to August 2020. We included observational studies that evaluate MMF's efficacy outcomes, including the change in annualized relapse rate (ARR), ARR during treatment, relapse probability, the difference in expanded disability status scale (EDSS), and the prevalence of freedom from EDSS progression. The studies with less than two patients with MMF and unpublished studies were excluded. Meta-analysis was performed using Comprehensive Meta-analysis Version 3.3 from Biostat, Inc (Eaglewood, NJ). Study results were statistically combined using a random-effect model.

The initial search yield 192 studies. Thirty-eight articles underwent full-text review. Ten studies (120 MOGAD patients) were included in the meta-analysis. The standard mean difference (SMD) of pre- and post-treatment ARR was -0.493 (95%CI -0.770 to -0.216, p<0.001). The pooled mean (SD) of ARR during MMF therapy was 0.836 (0.176). The overall relapse probability with MMF was 38.5% (95%CI 19.4% to 62.0%). The EDSS change before and after treatment was not statistically significant, with the SMD of -0.159 (95%CI -1.051 to 0.734, p 0.728). Approximately 84.3% (95%CI 60.6% to 94.9%) of patients with MOGAD had no EDSS progression with MMF.

The meta-analysis estimated the pooled efficacy outcome of MMF in MOGAD. Although MMF reduced ARR significantly, more than one-third of patients continued to relapse. Prospective controlled studies are needed to validate these results and investigate safety outcomes.