Development of a multiple sclerosis functional composite as a clinical trial outcome measure
Development of a multiple sclerosis functional composite as a clinical trial outcome measure
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DOI：
10.1093/brain/122.5.871
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发表时间：
1999-05-01
期刊：
影响因子：
14.5
通讯作者：
Willoughby, E
中科院分区：
文献类型：
--
作者：
Cutter, GR;Baier, ML;Willoughby, E
在临床试验中评估多发性硬化症的主要临床结果指标是Kurtzke扩展残疾状态量表（EDSS）。新的治疗方法似乎对多发性硬化症的病程有积极的影响，并使安慰剂对照组的继续使用更加困难。因此，未来的试验可能会比较积极的治疗组，这很可能需要增加样本量，以检测治疗效果。由于主动对照研究需要更敏感的结果测量，国家多发性硬化症协会的多发性硬化症新药临床试验咨询委员会任命了一个工作组，负责开发改进的临床结果测量。该工作组获得了当代临床试验和多发性硬化症的历史数据，用于主要和次要结果评估的荟萃分析，为推荐新的结果指标提供基础。一个包含手臂、腿部和认知功能的主要临床维度的复合测量被确定并称为多发性硬化症功能复合（MSFC）。MSFC由三种客观的神经功能定量测试组成，易于管理。在第一年的观察中，MSFC的变化预测了随后EDSS的变化，这表明MSFC比EDSS对变化更敏感。本文提供了MSFC的开发和测试的细节。
The primary clinical outcome measure for evaluating multiple sclerosis in clinical trials has been Kurtzke's expanded disability status scale (EDSS). New therapies appear to favourably impact the course of multiple sclerosis and render continued use of placebo control groups more difficult. Consequently, future trials are likely to compare active treatment groups which will most probably require increased sample sizes in order to detect therapeutic efficacy. Because more responsive outcome measures will be needed for active arm comparison studies, the National Multiple Sclerosis Society's Advisory Committee on Cinical Trials of New Agents in Multiple Sclerosis appointed a Task Force that was charged with developing improved clinical outcome measures. This Task Force acquired contemporary clinical trial and historical multiple sclerosis data for meta-analyses of primary and secondary outcome assessments to provide a basis for recommending a new outcome measure. A composite measure encompassing the major clinical dimensions of arm, leg and cognitive function was identified and termed the multiple sclerosis functional composite (MSFC). The MSFC consists of three objective quantitative tests of neurological function which are easy to administer. Change in this MSFC over the first year of observation predicted subsequent change in the EDSS, suggesting that the MSFC is more sensitive to change than the EDSS, This paper provides details concerning the development and testing of the MSFC.