% 0期刊文章% Mamatha Pasnoor % Laura Herbelin Jianghua他% % Ted伯恩斯%沙龙国家%维拉成Bril %一个安娜贝利k王% Bakri h Elsheikh %约翰·T·Kissel %大卫·萨珀斯坦% j·阿齐兹Shaibani %安德里亚·斯文森Carlayne杰克逊% %詹姆斯·f·霍华德,小% Namita Goyal %威廉·马修·威克伦大卫% %一个滑轮迈克尔%马拉贝克尔% Tahseen Mozaffar %迈克尔·贝纳塔尔%艾丽卡•辛普森Robert Pazcuzzi % %杰弗里·罗森菲尔德%阿巴斯杰弗里·m·Statland m . Dimachkie % %甲氨蝶呤的理查德·j . Barohn % MG肌肉的调查研究小组% T甲氨蝶呤的随机对照试验广义重症肌无力患者% D R 10.1212 / WNL 2016%。0000000000002795 % J首页神经病学% P 57 - 64 X % V 87% N 1%目的:确定steroid-sparing影响患者的甲氨蝶呤(MTX)的症状广义重症肌无力(MG)。方法:我们进行了一个12个月的多中心、随机、双盲、安慰剂对照试验的MTX 20毫克口服每周和安慰剂在50乙酰胆碱受体抗体阳性患者毫克2009年4月至2014年8月。主要结果测量是强的松dose-time曲线下面积(AUDTC)从4个月到12个。次要结果定量重症肌无力的措施包括12个月的变化分数,重症肌无力的综合得分,人工肌肉测试,重症肌无力的生活质量,重症肌无力日常生活活动。结果:58例筛选和50了。MTX不会降低4 - 12月强的松AUDTC相比安慰剂(区别MTX−安慰剂:−488.0毫克,95%置信区间2443−到1467。3,p = 0.26);然而,两组的平均每日强的松的剂量减少。MTX没有改善二次措施MG相比安慰剂超过12个月。八个参与者退出过程中研究(1 MTX, 7安慰剂)。没有严重的MTX-related不良事件。最常见的不良事件是特异性的疼痛(19%)。Conclusions: We found no steroid-sparing benefit of MTX in MG over 12 months of treatment, despite being well-tolerated. This study demonstrates the challenges of conducting clinical trials in MG, including difficulties with recruitment, participants improving on prednisone alone, and the need for a better understanding of outcome measure variability for future clinical trials.Classification of evidence: This study provides Class I evidence that for patients with generalized MG MTX does not significantly reduce the prednisone AUDTC over 12 months of therapy.AUC=area under the curve; AUDTC=area under the dose-time curve; CI=confidence interval; LOCF=last observation carried forward; MG=myasthenia gravis; MG-ADL=Myasthenia Gravis Activities of Daily Living scale; MGC=Myasthenia Gravis Composite Score; MGFA=Myasthenia Gravis Foundation of America; MMT=manual muscle testing; MTX=methotrexate; QMG=Quantitative Myasthenia Gravis Score %U //www.ez-admanager.com/content/neurology/87/1/57.full.pdf