Efficacy and Safety of Mycophenolate Mofetil Therapy in Neuromyelitis Optica Spectrum Disorders: A Systematic Review and Meta-Analysis
Abstract Background: Neuromyelitis optica spectrum disorders (NMOSD) is an autoimmune demyelinating disease of the central nervous system characterized by severe attacks of optic nerve and spinal cord. Mycophenolate mofetil (MMF) is an immunosuppressive agent (IS) which is widely prescribed for NMOSD patients. This systematic review and meta-analysis aims to assess the efficacy and safety of MMF in controlling relapse and disease severity.Methods: Studies were obtained from the EMBASE and Ovid MEDLINE databases. Eligible studies were the studies of NMOSD patients treated with MMF which reported treatment outcomes as Annualized Relapse Rate (ARR) or Expanded Disability Status Scale (EDSS) before and after treatment. Case reports, case series less than 3 patients, and reviews were excluded.Results: Fifteen studies included 1047 patients, of whom 915 (87.4%) were aquaporin-4 immunoglobulin seropositive. The total number of patients that received MMF was 799. Meta-analysis on ARR and EDSS were conducted in 4 studies with a total of 200 patients and 3 studies with a total of 158 patients, respectively. The result showed a significant improvement with a mean reduction of 1.13 (95% confidence interval (CI), 0.60 to 1.65) in ARR and a mean reduction of 0.85 (95% CI, 0.36 to 1.34) in EDSS after MMF therapy. Adverse drug reactions occurred in 106 (17.8%) of 594 patients that were documented having side effects during MMF therapy.Conclusion: This systematic review and meta-analysis showed that using MMF as a preventive therapy in NMOSD patients can significantly reduce relapse rate and improve disease severity with an acceptable tolerability.