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.
Front Immunol. 2018 Sep 11;9:2066. doi: 10.3389/fimmu.2018.02066. eCollection 2018.
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.
评估低剂量吗替麦考酚酯（MMF，1000mg/天）治疗视神经脊髓炎谱系疾病（NMOSD）的疗效和安全性。本研究为多中心、开放、前瞻性、随访临床研究。数据包括治疗前回顾性临床数据和治疗后前瞻性数据。2014 年 9 月至 2017 年 2 月，对水通道蛋白 4 免疫球蛋白 G（AQP4-IgG）阳性的 NMOSD 患者给予低剂量 MMF 治疗。90 例 NMOSD 患者接受 MMF 治疗，中位治疗时间为 18 个月（6-40 个月）。中位年复发率（ARR）从治疗前的 1.02 降至 0（<0.0001），扩展残疾状况量表（EDSS）评分从 4 降至 3（<0.0001）。治疗后第 90 天 EDSS 评分显著降低（=0.038）。50 例（63%）患者血清 AQP4-IgG 滴度下降。65 例（88%）脊髓炎患者的简易 McGill 疼痛评分（SF-MPQ）中位数从治疗前的 17（0-35）降至 11（0-34）（<0.0001）。治疗前 Hauser 行走指数（Hauser Walk Rating Scale）中位数为 2（1-9），治疗后降至 1（0-7）（<0.0001）。43%的患者出现不良事件，8 例患者因无法耐受不良事件而停用 MMF。14 例（16%）患者因各种原因停用 MMF，换用硫唑嘌呤或利妥昔单抗。在中国南方，低剂量 MMF 可降低 NMOSD 患者的临床复发率和残疾程度。但部分患者在该剂量下仍有不良反应。
