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Front Immunol. 2018 Sep 11;9:2066. doi: 10.3389/fimmu.2018.02066. eCollection 2018.

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

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Department of Neurology, The Third Affiliated Hospital of Sun Yat-sen University, Guangzhou, China.
Department of Neurology, Zhaoqing No. 2 People's Hospital, Zhaoqing, China.
Department of Neuro-Ophthalmology, Zhongshan Ophthalmic Center, Sun Yat-sen University, Guangzhou, China.
Department of Neurology, Guangdong 999 Brain Hospital, Guangzhou, China.
Department of Neurology, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, China.
Department of Neurology, Second Affiliated Hospital of Guangzhou Medical University, Guangzhou, China.
Department of Neurology, Nanfang Hospital, Guangzhou, China.
Department of Neurology, First Affiliated Hospital of Sun Yat-sen University, Guangzhou, China.
School of Mathematics, Sun Yat-sen University, Guangzhou, China.


Objective: To evaluate the efficacy and safety of low-dose mycophenolate mofetil (MMF, 1,000 mg/day) treatment of neuromyelitis optica spectrum disorders (NMOSDs). Methods: 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. Results: 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 (P < 0.0001) after treatment, and the Expanded Disability Status Scale (EDSS) score decreased from 4 to 3 (P < 0.0001). The EDSS score was significantly lower (P = 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 (P < 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 (P < 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. Conclusion: 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.



South China; a prospective study; mycophenolate mofetil; neuromyelitis optica spectrum disorders; therapy

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