Effectiveness And Tolerability Of Mycophenolate Mofetil In The Treatment Of Neuromyelitis Optica Spectrum Disorders: A Systematic Review

Neuromyelitis optica spectrum disorders (NMOSD) are a group of autoimmune inflammatory process associated with the presence of aquaporin-4 autoantibody (AQP4-IgG), preferentially affecting the optic nerves and spinal cord. Mycophenolate mofetil (MMF) is an immunosuppressant proven to be effective in a number of immune-mediated disorders. The main objective of this study was to conduct a systematic review of case series and cohort studies to evaluate the effectiveness in the reduction of relapse rate and disability progression, and tolerability of MMF in patients with NMOSD Articles were searched from Cochrane Central Register for Controlled Trials by The Cochrane Library, MEDLINE by PubMed, LILACS, and ClinicalTrials.gov websites from June to December 2018. Four retrospective case series, two retrospective cohorts, and three prospective cohort, which utilized MMF for patients with NMOSD, were included. Data collected from the included studies were study design, population characteristics, treatment regimen, annualized relapse rate (ARR), expanded disability status scale (EDSS) scores, and adverse events. Mycophenolate mofetil may be effective in reducing ARR to up to 92% when used as initial, second-line, or adjunct therapy. It may also be effective in reducing disability progression as noted in the improved or stabilized median EDSS scores in 83-91% of patients. It is well tolerated in patients with NMOSD, with hair loss (4-6.78%), elevated liver enzymes (0.95-6.67%), and low white blood cell and neutrophil counts (0.92-4%) as the most frequent adverse events reported. There is a need for randomized controlled trials to determine the true treatment effect and risks of MMF therapy.