The Efficacy and Tolerability of Mycophenolate Mofetil in Treating Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder in Western China

Abstract

Objectives: The aim of this study was to assess the efficacy and tolerability of mycophenolate mofetil (MMF) in neuromyelitis optica (NMO) or NMO spectrum disorder (NMOSD) in western China.

Methods: We enrolled 90 patients with NMO or NMOSD who had received MMF between January 1, 2010, and June 15, 2015.

Results: Of 90 patients, 62 (4 men and 58 women; aged 44.6 [11.5] years) were included in the study. After being treated for a median of 1.5 years (range, 0.5-4.1 years), the median annualized relapse rate for these 62 patients decreased from 1.2 (range, 0.2-7.0) pre-MMF to 0 (range, 0-1.7) post-MMF (P = 0.000), and the median Expanded Disability Status Scale score decreased from 4 (range, 0.5-8.0) pre-MMF to 2 (range, 0.5-7.5) post-MMF (P = 0.000). Thirty-six of the 62 patients were relapse free during MMF treatment. In the Cox regression, none of the following were identified as risk factors: disease duration, pre-MMF annualized relapse rate and Expanded Disability Status Scale, sex, concurrent use of prednisolone during MMF treatment, previous use of other immunosuppressive therapies (other than chronic prednisolone), and abnormal autoantibodies (other than NMO-IgG). However, serum NMO-IgG positivity (hazard ratio [HR], 11.408; 95% confidence interval [CI], 1.330-97.833; P = 0.026) and older age at onset (HR, 0.957; 95% CI, 0.917-0.999; P = 0.043) were significant risk factors. Kaplan-Meier survival analysis indicated a lower risk of relapse during MMF treatment relative to the pre-MMF period (HR, 0.439; 95% CI, 0.272-0.707; P = 0.001). None of the 62 patients discontinued MMF because of adverse effects.

Conclusions: Mycophenolate mofetil is an effective and tolerable agent for reducing relapse and improving or stabilizing disabilities resulting from NMO or NMOSD.