Use of the Multiple Sclerosis Functional Composite as an outcome measure in a phase 3 clinical trial
Use of the Multiple Sclerosis Functional Composite as an outcome measure in a phase 3 clinical trial
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DOI：
10.1001/archneur.58.6.961
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发表时间：
2001-06-01
影响因子：
--
通讯作者：
Whitaker, JN
中科院分区：
文献类型：
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作者：
Cohen, JA;Cutter, GR;Whitaker, JN
背景资料：多发性硬化症功能复合评分（MSFC）是一项多维临床结局指标，包括腿部功能/上肢功能（计时25英尺步行）、手臂功能（9孔桩测试）和认知功能（起搏听觉序列加法测试）的定量测试。MSFC是在进行中的多国干扰素β-1a（Avonex）在继发性进行性MS.Objective患者3期试验的主要结局指标：评估实践效果，可靠性和有效性的MSFC临床结局measure.Design：检查技术人员接受了正式的培训，使用标准化的材料。根据标准化方案进行MSFC。436例患者参加了国际多发性硬化症继发性进展Avonex对照试验进行3个基线前MSFC测试会议前randomization.Results：实践效果是明显的MSFC最初，但稳定的第四届政府。节奏听觉连续加法测验的练习效果最为显著。MSFC的可靠性极佳，第3阶段（最终基线前阶段）与第4阶段（基线）的组内相关系数为0.90。基线MSFC与Kurtzke扩展残疾状态量表中度强相关。在MSFC的组成部分中，定时25英尺步行的相关性最密切。3 MSFC组件之间的相关性较弱，表明他们评估MS患者的神经功能的不同方面。结论：在这项多国3期试验中，MSFC表现出良好的内部可靠性。三个基线前测试阶段足以补偿练习效果。MSFC，其组成部分，和Kurtzke扩展残疾状态量表之间的相关性模式支持MSFC的有效性。
Background: The Multiple Sclerosis Functional Composite (MSFC) is a multidimensional clinical outcome measure that includes quantitative tests of leg function/ambulation (Timed 25-Foot Walk), arm function (9-Hole Peg Test), and cognitive function (Paced Auditory Serial Addition Test). The MSFC is the primary outcome measure in the ongoing multinational phase 3 trial of interferon beta-1a (Avonex) in patients with secondary progressive MS.Objective: To assess the practice effects, reliability, and validity of the MSFC clinical outcome measure.Design: Examining technicians underwent formal training using standardized materials. The MSFC was performed according to a standardized protocol. The 436 patients enrolled in the international Multiple Sclerosis Secondary Progressive Avonex Controlled Trial underwent 3 prebaseline MSFC testing sessions before randomization.Results: Practice effects were evident initially for the MSFC but stabilized by the fourth administration. The Paced Auditory Serial Addition Test demonstrated the most prominent practice effects. The reliability of the MSFC was excellent, with an intraclass correlation coefficient for session 3 (final prebaseline session) vs session 4 (baseline) of 0.90. The MSFC at baseline correlated moderately strongly with the Kurtzke Expanded Disability Status Scale. Among the MSFC components, the Timed 25-Foot Walk correlated most closely. Correlations among the 3 MSFC components were weak, suggesting they assess distinct aspects of neurologic function in patients with MS.Conclusions: The MSFC demonstrated excellent intrarater reliability in this multinational phase 3 trial. Three prebaseline testing sessions were sufficient to compensate for practice effects. The pattern of correlations among the MSFC, its components, and the Kurtzke Expanded Disability Status Scale supported the validity of the MSFC.