Caranfa JT, Kohn CG, Baker WL and Waitzman DM
Background: Neuromyelitis optica (NMO) and NMO spectrum disorder (NMOSD) is a rare autoimmune disorder that often leaves patients severely disabled, despite aggressive treatment with traditional immunosuppressive medication. While monoclonal antibody therapy has shown efficacy in treating NMO/NMOSD, no large randomized control trials exist. In lieu of such trials, we performed a systematic review and meta-analysis to assess the efficacy and safety of rituximab, eculizumab and tocilizumab in NMO/NMOSD patients.
Methods: We searched MEDLINE, the Cochrane Central Register of Controlled Trials (CENTRAL) and Embase from January 1, 2006 through December 31, 2018 for prospective studies using rituximab, eculizumab or tocilizumab in NMO/NMOSD patients and reporting annual relapse rate (ARR) and Expanded Disability Status Scale (EDSS) score before and after monoclonal therapy.
Results: Sixteen studies involving 324 patients were included in the systematic review and meta-analysis. Monoclonal therapy resulted in a statistically significant mean reduction in ARR of 1.77 (95% CI, 1.37 to 2.17) and a statistically significant mean reduction in EDSS of 1.14 (95% CI, 0.87 to 1.41).
Conclusion: Treatment with monoclonal antibodies, specifically rituximab, eculizumab and tocilizumab, significantly reduced the frequency of disease relapses and improved neurological disability in NMO/NMOSD patients.
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