BEST-MS:一项比较Natalizumab与Fingolimod治疗活动性复发性多发性硬化症疗效的标准化前瞻性研究(769)

摘要目的:比较natalizumab (NTZ)和fingolimod (FTY)治疗活动性复发-缓解型多发性硬化症(RRMS)的疗效。

背景:活动期RRMS患者的治疗选择越来越多，但很少有前瞻性研究可以比较它们的疗效。MS的最佳升级策略(Best MS)于2013年在法国开始，当时NTZ和FTY是活跃RRMS中最常用的两种治疗方法。

设计/方法:BEST-MS是一项法国前瞻性多中心研究。活动期RRMS患者(定义为在过去12个月内至少有1次复发^圣在MRI上至少有9次T2高信号，或在过去12个月内至少有2次复发并有MRI活动性疾病证据(naïve患者)的一线治疗被纳入NTZ或FTY治疗。治疗的选择由医生自行决定。在基线和12个月时收集复发、EDSS和脑MRI。

结果:230例患者(平均年龄:38.2岁，F/M: 3.1, FTY: 117, NTZ: 113)。两组间基线临床和放射学特征无统计学差异。

在M12时，NTZ对新发T2病变(0.7 vs 1.4, p=0.01)和钆增强病变(0.03 vs 0.5, p<0.0001)的疗效更好。NTZ组的复发率(ARR)较低(0.20 vs 0.27, p = 0.04)，即使两组的大多数复发发生在治疗的前4个月。50%的NTZ患者无疾病活动，而FTY组为25% (p=0.05)。FTY组的退出率更高(22% vs 12%， p<0.0001)。

结论:在活动期RRMS患者的MRI上，NTZ的疗效高于FTY。ARR较低，有利于NTZ，且大多数复发发生早。由于不良事件，FTY的辍学率较高。

披露:Cohen博士曾因在Biogen、Roche、Merck、Ad scientiam担任顾问、科学顾问委员会成员、演讲或其他活动而获得个人报酬。蒙多没有什么可透露的。布恰雷利博士没有什么可透露的。皮格诺莱医生没什么可透露的。拉普劳德医生没什么可透露的。他曾因咨询、在科学顾问委员会任职、演讲或其他活动获得个人报酬，并从Novartis、Biogen-Idec、Merck KGaA、Roche和Cellgene获得咨询费、演讲费或酬金。Brochet博士获得了机构的研究支持，获得了诺华、Biogen-Idec、默克、罗氏、Medday、Actelion和Genzyme Sanofi的研究资助(非个人)和/或临床试验合同。Defer博士获得了来自Biogen、Merck-Serono、Novartis、Sanofi-Genzyme、Roche和Teva的个人咨询、演讲或其他活动的个人报酬，以及来自Biogen、Merck-Serono、Novartis、Sanofi-Genzyme的旅行和/或演讲酬金。Defer博士获得了Biogen、Merck-Serono、Novartis、Sanofi Genzyme等机构的研究资助。他曾在默克雪兰诺(Merck Serono)、百健(Biogen)、诺华(Novartis)、Teva、赛诺菲-健赞(Sanofi-Genzyme)担任顾问、科学顾问委员会成员、演讲或其他活动，获得个人报酬。 Dr. Vermersch has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Almirall, Biogen, Celgene, Merck Serono, Novartis, Roche, Sanofi, Servier, and Teva. Dr. Vermersch has received research support from Almirall, Biogen, Celgene, Merck Serono, Novartis, Roche, Sanofi, Servier, and Teva.Dr. Zephir has nothing to disclose. Dr. Debouverie has nothing to disclose. Dr. Berger has nothing to disclose. Dr. Labauge has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biogen,Sanofi-Genzyme, Bayer, Novartis, Teva, Merck-Serono, Roche and Medday.. Dr. Labauge has received research support from Biogen, Novartis, Sanofi-Genzyme, Roche and Medday.. Dr. Ayrignac has nothing to disclose. Dr. De Seze has nothing to disclose. Dr. Brassat has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Bayer, Biogen, Medday, Merck, Novartis, Roche, Sanofi-Genzyme, Teva. Dr. Brassat has received research support from Novartis, Medday Pharmaceutical. Dr. Lebrun-Frenay has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Consulting fees, honoraria or scientific committee support: Bayer Schering, Biogen, Genzyme, Merck Serono, Novartis, Teva.