Background: Several immunosuppressants or monoclonal antibodies have been used as preventive treatment for neuromyelitis optica spectrum disorders (NMOSD); however, the optimal therapies have not been clarified. In this study, we aimed to compare and rank the effectiveness and tolerability of all preventive therapies for NMOSD.
Methods: Qualified studies were identified in a search of MEDLINE, Embase, Cochrane Central Register of Controlled Trials (CENTRAL), and ClinicalTrials.gov databases. We combined direct and indirect evidence via meta-analyses. The annualized relapse rate (ARR) was defined as the primary outcome. Secondary outcomes included the Expanded Disability Status Scale (EDSS) score and hazard ratios (HR) for the counts of adverse events (AEs).
Results: We identified one randomized controlled trial (RCT) and five observational studies including a total 631 patients with NMOSD. Among these, the follow-up time ranged from 12 to 40 months. For the primary outcome, rituximab (RTX) was hierarchically superior, with the significant standardized mean difference versus azathioprine (-0.86; 95% confidence interval: -1.60, -0.11). Mycophenolate mofetil (MMF) was ranked the most tolerable therapy, whereas cyclophosphamide was the least tolerable.
Conclusion: RTX and MMF may be recommended as optimal treatments to prevent relapse in NMOSD. Low-dose cyclosporine A could be a promising alternative therapy.
Keywords: Immunosuppressant; Immunotherapy; Monoclonal antibody; Neuromyelitis optica spectrum disorders.
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