Outcome Measures in Relapsing-Remitting Multiple Sclerosis: Capturing Disability and Disease Progression in Clinical Trials

被引:60
|
作者
Lavery, Amy M. [1 ]
Verhey, Leonard H. [2 ]
Waldman, Amy T. [1 ,3 ,4 ]
机构
[1] Childrens Hosp Philadelphia, Div Neurol, Philadelphia, PA 19104 USA
[2] Univ Toronto, Hosp Sick Children, Program Neurosci & Mental Hlth, Pediat Demyelinating Dis Program, Toronto, ON M5G 1X8, Canada
[3] Univ Penn, Perelman Sch Med, Dept Neurol, Philadelphia, PA 19104 USA
[4] Univ Penn, Perelman Sch Med, Dept Pediat, Philadelphia, PA 19104 USA
关键词
D O I
10.1155/2014/262350
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Multiple sclerosis (MS) is a chronic inflammatory and neurodegenerative disease that manifests as acute relapses and progressive disability. As a primary endpoint for clinical trials in MS, disability is difficult to both characterize and measure. Furthermore, the recovery from relapses and the rate of disability vary considerably among patients. Given these challenges, investigators have developed and studied the performance of various outcome measures and surrogate endpoints in MS clinical trials. This review defines the outcome measures and surrogate endpoints used to date in MS clinical trials and presents challenges in the design of both adult and pediatric trials.
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页数:13
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