RT期刊文章SR电子T1 NFL模式在MD1003治疗进展型多发性硬化症队列(p4.2 - 008)摩根富林明神经病学神经学乔FD Lippincott Williams &威尔金斯SP p4.2补充92 - 008签证官是15 A1贝雅特丽齐Pign首页olet A1弗朗西斯卡Ammoscato A1乔纳森Ciron A1佛罗伦萨Bucciarelli A1丽丝Scandella A1 Damien Biotti A1弗勒Lerebours A1 Guillaume Dorcet A1席琳Rabadeux A1 Noellie Freitas A1法布里斯博纳维尔A1 Sharmilee Gnanapavan A1大卫Brassat年2019 UL //www.ez-admanager.com/content/92/15_supplement/p4.2 - 008. -文摘AB目的:确定血清神经丝轻链(sNFL)浓度与MD1003(制药级生物素高剂量300毫克/天)治疗进步MS.Background: MS-SPI研究表明MD1003实现持续逆转的医学相关残疾患者的一个子集进步女士(Tourbah et al ., 2016)。各种神经疾病伴随神经损伤已被证明与NFL脑脊液中浓度升高有关。在女士,最近的一项研究表明血清NFL水平作为一个敏感的和临床有意义的生物标志物监测组织损伤和生物疗法效果(Disanto et al ., 2017)。设计/方法:Monocentric阶段4,2年前瞻性单臂MD1003研究进步的MS患者遵循临床MRI数据和生物采样(包含:2016年1月- 2017年10月)。主要终点:sNFL浓度(SIMOA™技术)在基线,6个月,1和2年。次要终点:临床/ MRI疗效和不良反应。类似的军团相比,数据将从提升,扩大,通知和清唱剧试验。结果:到目前为止,203名患者已经被包括在内。我们提出一个中间分析65 1年随访患者的亚群体。我们发现sNFL从基线水平(平均11.8 + /−5.6 pg / ml) MD1003 6个月(平均16.1 + /−14.1 pg / ml;p = 0.0043, Wilcoxon matched-paired),达到高原1年(平均16.9 + /−12.9 pg / ml)。分析40例的sNFL水平将在2年。23%的病人有经验的临床改善。活动性疾病,定义为一个clinically-defined复发和/或Gd-enhancing T1病变,观察到11%的患者。然而,sNFL水平并不相关的临床和MRI加重。Conclusions: This intermediate analysis suggests that MD1003 has an impact on sNFL and clinical evolution. However, sNFL levels are not predictive of disease worsening.Disclosure: Dr. Pignolet has nothing to disclose. Dr. Ammoscato has nothing to disclose. Dr. Ciron has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Merck Serono, Roche. Dr. Bucciarelli has nothing to disclose. Dr. Scandella has nothing to disclose. Dr. Biotti has nothing to disclose. Dr. Lerebours has nothing to disclose. Dr. Dorcet has nothing to disclose. Dr. Rabadeux has nothing to disclose. Dr. Freitas has nothing to disclose. Dr. Bonneville has nothing to disclose. Dr. Gnananpavan has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Genzyme and Takeda Pharmaceutical Company Limited. 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 and Medday Pharmaceutical.