Cutter G R, Baier M L, Rudick R A, Cookfair D L, Fischer J S, Petkau J, Syndulko K, Weinshenker B G, Antel J P, Confavreux C, Ellison G W, Lublin F, Miller A E, Rao S M, Reingold S, Thompson A, Willoughby E
AMC Cancer Research Center, Center for Research Methodology and Biometrics, Lakewood, Colarado 80214, USA.
Brain. 1999 May;122 ( Pt 5):871-82. doi: 10.1093/brain/122.5.871.
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 Clinical 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.
在临床试验中，用于评估多发性硬化症的主要临床结局指标一直是库尔特克扩展残疾状态量表（EDSS）。新疗法似乎对多发性硬化症的病程产生了积极影响，使得继续使用安慰剂对照组变得更加困难。因此，未来的试验可能会比较活性治疗组，这很可能需要增加样本量才能检测出治疗效果。由于活性治疗组对比研究需要更敏感的结局指标，美国国家多发性硬化症协会新药临床试验咨询委员会任命了一个特别工作组，负责制定改进的临床结局指标。该特别工作组收集了当代临床试验数据和多发性硬化症历史数据，用于对主要和次要结局评估进行荟萃分析，以便为推荐新的结局指标提供依据。一种涵盖上肢、下肢和认知功能主要临床维度的综合指标被确定，并命名为多发性硬化症功能综合指标（MSFC）。MSFC由三项易于实施且客观的神经功能定量测试组成。在观察的第一年中，MSFC的变化预测了EDSS随后的变化，这表明MSFC比EDSS对变化更敏感。本文详细介绍了MSFC的制定和测试情况。
