Low-Dose Mycophenolate Mofetil for Treatment of Neuromyelitis Optica Spectrum Disorders: A Prospective Multicenter Study in South China

• Published in: Frontiers in immunology Vol. 9; p. 2066
• Main Authors: Huang, Qiao, Wang, Jingqi, Zhou, Yifan, Yang, Hui, Wang, Zhanhang, Yan, Zhenwen, Long, Youming, Yin, Jia, Feng, Huiyu, Li, Caixia, Lu, Zhengqi, Hu, Xueqiang, Qiu, Wei
• Format: Journal Article
• Language: English
• Published: Switzerland Frontiers Media S.A 11.09.2018
• Subjects: a prospective study; Immunology; mycophenolate mofetil; neuromyelitis optica spectrum disorders; South China; therapy; a prospective study; mycophenolate mofetil; South China; therapy; neuromyelitis optica spectrum disorders
• ISSN: 1664-3224; 1664-3224
• DOI: 10.3389/fimmu.2018.02066

To evaluate the efficacy and safety of low-dose mycophenolate mofetil (MMF, 1,000 mg/day) treatment of neuromyelitis optica spectrum disorders (NMOSDs). This study was a multicenter, open, prospective, follow-up clinical trial. The data include retrospective clinical data from the pretreatment phase and prospective data from the post-treatment phase. From September 2014 to February 2017, NMOSD patients seropositive for aquaporin 4-IgG (AQP4-IgG) were treated with low-dose MMF. Ninety NMOSD patients were treated with MMF for a median duration of 18 months (range 6-40 months). The median annual recurrence rate (ARR) decreased from 1.02 before treatment to 0 ( < 0.0001) after treatment, and the Expanded Disability Status Scale (EDSS) score decreased from 4 to 3 ( < 0.0001). The EDSS score was significantly lower ( = 0.038) after the first 90 days of treatment. The serum AQP4-IgG titer decreased in 50 cases (63%). The median Simple McGill pain score (SF-MPQ) was reduced in 65 patients (88%) with myelitis from 17 (range 0-35) to 11 (range 0-34) after treatment ( < 0.0001). The median Hauser walking index (Hauser Walk Rating Scale) was reduced from 2 (range 1-9) before treatment to 1 (range 0-7) after treatment ( < 0.0001). Adverse events were documented in 43% of the patients, and eight patients discontinued MMF due to intolerable adverse events. Fourteen (16%) of the total patients discontinued MMF after our last follow-up for various reasons and switched to azathioprine or rituximab. Low-dose MMF reduced clinical relapse and disability in NMOSD patients in South China. However, some patients still suffered from adverse events at this dosage. www.ClinicalTrials.gov, identifier : NCT02809079.