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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.

Author information

1
Department of Neurology, The Third Affiliated Hospital of Sun Yat-sen University, Guangzhou, China.
2
Department of Neurology, Zhaoqing No. 2 People's Hospital, Zhaoqing, China.
3
Department of Neuro-Ophthalmology, Zhongshan Ophthalmic Center, Sun Yat-sen University, Guangzhou, China.
4
Department of Neurology, Guangdong 999 Brain Hospital, Guangzhou, China.
5
Department of Neurology, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, China.
6
Department of Neurology, Second Affiliated Hospital of Guangzhou Medical University, Guangzhou, China.
7
Department of Neurology, Nanfang Hospital, Guangzhou, China.
8
Department of Neurology, First Affiliated Hospital of Sun Yat-sen University, Guangzhou, China.
9
School of Mathematics, Sun Yat-sen University, Guangzhou, China.

Abstract

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.

CLINICAL TRIAL REGISTRATION:

www.ClinicalTrials.gov, identifier : NCT02809079.

KEYWORDS:

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

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