AUTHOR=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 

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

JOURNAL=Frontiers in Immunology

VOLUME=9

YEAR=2018

URL=https://www.frontiersin.org/journals/immunology/articles/10.3389/fimmu.2018.02066

DOI=10.3389/fimmu.2018.02066

ISSN=1664-3224

ABSTRACT=<p><bold>Objective:</bold> To evaluate the efficacy and safety of low-dose mycophenolate mofetil (MMF, 1,000 mg/day) treatment of neuromyelitis optica spectrum disorders (NMOSDs).</p><p><bold>Methods:</bold> 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.</p><p><bold>Results:</bold> 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 (<italic>P</italic> &lt; 0.0001) after treatment, and the Expanded Disability Status Scale (EDSS) score decreased from 4 to 3 (<italic>P</italic> &lt; 0.0001). The EDSS score was significantly lower (<italic>P</italic> = 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 (<italic>P</italic> &lt; 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 (<italic>P</italic> &lt; 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.</p><p><bold>Conclusion:</bold> 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.</p><p>Clinical Trial Registration: <ext-link ext-link-type="uri" xlink:href="http://www.ClinicalTrials.gov" xmlns:xlink="http://www.w3.org/1999/xlink">www.ClinicalTrials.gov</ext-link>, identifier : NCT02809079.</p>