The Multiple Sclerosis Functional Composite: a new clinical outcome measure for multiple sclerosis trials
The Multiple Sclerosis Functional Composite: a new clinical outcome measure for multiple sclerosis trials
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多发性硬化症功能复合物：多发性硬化症试验的新临床结果测量
DOI：
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发表时间：
2002
期刊：
影响因子：
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通讯作者：
S. Reingold
中科院分区：
文献类型：
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作者：
R. Rudick;G. Cutter;S. Reingold
随着多发性硬化症(MS)部分有效的疾病修饰药物疗法的出现和广泛使用，未来的临床试验无疑将测试实验性干预和标准治疗，或者将测试药物组合和标准治疗。在任何一种情况下，在未来的多发性硬化症临床试验中减缓残疾进展的渐进进展将需要更大的样本量、更敏感的结果衡量标准，或者两者的组合。由于改进的临床结果方法可能会加速多发性硬化症治疗的进展，美国国家多发性硬化症学会(NMSS)在1994年召集了一个国际工作组来建议改进的临床结果测量方法。经过两年的讨论和数据分析，工作组推荐多发性硬化症功能性复合体(MSFC)作为未来MS试验的一种新的临床结果衡量标准。MSFC由步行、手臂功能和认知功能的计时测试组成，表示为连续尺度上的单一分数。工作组建议将MSFC纳入未来的多发性硬化症试验，并建议进行一系列验证研究。随后的研究证明，MSFC与扩展的残疾状态量表(EDSS)有适度的相关性，这种相关性是由与行走功能部分的强烈相关性驱动的；手臂功能和认知功能与EDSS在较低水平上相关。MSFC与包括脑萎缩在内的磁共振成像(MRI)变量的相关性比EDSS更好，并与患者报告的疾病相关生活质量(QOL)显示出显著的相关性。MSFC和MSFC的短期变化与未来的临床和MRI状态相关，而且这种相关性的强度与众所周知的心血管危险因素相比更有利。总体而言，研究表明MSFC和MSFC的变化具有临床意义，并且MSFC在MS临床试验中比其他临床结果指标具有实质性的优势。
With the advent and widespread use of partially effective disease modifying drug therapies for multiple sclerosis (MS), future clinical trials will undoubtedly test experimental interventions against standard therapy, or will test combinations of drugs against standard therapy. In either case, incremental progress in slowing disability progression in future MS clinical trials will require much larger sample sizes, more sensitive outcome measures, or a combination of the two. Because improved clinical outcome methods would likely accelerate progress in MS therapeutics, the National Multiple Sclerosis Society (NMSS) convened an international task force in 1994 to recommend improved clinical outcome measures. As the result of a two-year process of discussion and data analysis, the task force recommended the Multiple Sclerosis Functional Composite (MSFC) as a new clinical outcome measure for future MS trials. MSFC consists of timed tests of walking, arm function, and cognitive function, expressed as a single score along a continuous scale. The task force recommended that MSFC be included in future MS trials, and recommended a series of validation studies. Subsequent studies have provided evidence that MSFC correlates moderately with Expanded Disability Status Scale (EDSS), and that correlation is driven by strong correlations with the ambulatory function component; arm function and cognitive function correlate at lower levels with EDSS. The MSFC correlates better than EDSS with magnetic resonance imaging (MRI) variables, including brain atrophy, and shows significant correlation with patient-reported disease-related quality of life (QOL). MSFC and short-term change in MSFC correlate with future clinical and MRI status, and the strength of the correlations compares favorably with well-known cardiovascular risk factors. The studies in aggregate indicate that MSFC and MSFC change are clinically meaningful, and that MSFC has substantial advantages over alternative clinical outcome measures for MS clinical trials.