Service de Neurologie A and Eugène Devic EDMUS Foundation against Multiple Sclerosis, Observatoire Français de la Sclérose en Plaques (OFSEP), Hôpital Neurologique Pierre Wertheimer-GHE, Hospices Civils de Lyon, Bron, France/Department of Neurology, Hôpital Dupuytren, Limoges, France.
Department of Neurology, and INSERM CIC-1434, CHU de Strasbourg, Strasbourg, France.
Mult Scler. 2017 Sep;23(10):1377-1384. doi: 10.1177/1352458516678474. Epub 2016 Nov 25.
To evaluate the effectiveness and tolerance of mycophenolate mofetil (MMF) as a first-line treatment in neuromyelitis optica spectrum disorder (NMOSD).
In all, 67 NMOSD patients treated by MMF as first-line therapy, from the NOMADMUS cohort were included. A total of 65 fulfilled 2015 NMOSD criteria, and 5 were myelin oligodendrocyte glycoprotein (MOG)-immunoglobulin G (IgG) positive. Effectiveness was evaluated on percentage of patients continuing MMF, percentage of patients free of relapse, pre- and post-treatment change in the annualized relapse rate (ARR), and Expanded Disability Status Scale (EDSS).
Among 67 patients, 40 (59.7%) continued treatment till last follow-up. A total of 33 (49.3%) were relapse-free. The median ARR decreased from one pre-treatment to zero post-treatment. Of 53 patients with complete EDSS data, the score improved or stabilized in 44 (83%; p < 0.05). Effectiveness was observed in aquaporin-4 (AQP4)-IgG (57.8% continued treatment, 46.7% relapse-free), MOG-IgG (3/5 continued treatment, 4/5 relapse-free), and seronegative NMOSD (64.7% continued treatment, 61.3% relapse-free). In 16 patients with associated steroids, 13 (81.2%) continued MMF till last follow-up versus 15 of 28 (53.6%) in the non-steroid group. Nine patients discontinued treatment for tolerability purpose.
MMF showed effectiveness and good tolerability as a first-line therapy in NMOSD, whatever the AQP4-IgG status. Concomitant use of oral steroids at start could limit the risk of treatment failure.
评估吗替麦考酚酯（MMF）作为视神经脊髓炎谱系疾病（NMOSD）一线治疗的有效性和耐受性。
共纳入 67 例接受 MMF 一线治疗的 NMOSD 患者，该队列来源于 NOMADMUS 研究。65 例患者符合 2015 年 NMOSD 诊断标准，5 例为髓鞘少突胶质细胞糖蛋白（MOG）-免疫球蛋白 G（IgG）阳性。通过继续使用 MMF 的患者比例、无复发患者比例、治疗前后年复发率（ARR）的变化和扩展残疾状况量表（EDSS）评分的变化评估疗效。
67 例患者中，40 例（59.7%）持续治疗至末次随访。33 例（49.3%）无复发。53 例患者完整 EDSS 数据显示，治疗后 ARR 中位数从 1 次/年降至 0 次/年。44 例（83%；p<0.05）患者 EDSS 评分改善或稳定。AQP4-IgG（57.8%继续治疗，46.7%无复发）、MOG-IgG（3/5 继续治疗，4/5 无复发）和血清阴性 NMOSD（64.7%继续治疗，61.3%无复发）患者均观察到疗效。16 例患者在治疗开始时联合使用了口服激素，其中 13 例（81.2%）继续使用 MMF 至末次随访，而在非激素组中，28 例中有 15 例（53.6%）继续使用 MMF。9 例患者因药物不耐受而停药。
无论 AQP4-IgG 状态如何，MMF 作为 NMOSD 的一线治疗均具有有效性和良好的耐受性。治疗开始时联合使用口服激素可能会降低治疗失败的风险。
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