Assessing disability progression with the Multiple Sclerosis Functional Composite
Assessing disability progression with the Multiple Sclerosis Functional Composite
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DOI：
10.1177/1352458509106212
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发表时间：
2009-08-01
影响因子：
5.8
通讯作者：
Sandrock, A. W.
中科院分区：
文献类型：
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作者：
Rudick, R. A.;Polman, C. H.;Sandrock, A. W.
背景 最初的多发性硬化症功能复合（MSFC）提案是由三部分组成的复合定量测量指标，包括步行、上肢功能和认知功能，以单个复合 Z 分数表示。然而，MSFC Z 分数变化的临床意义并不明显。本研究使用 MSFC 成分数据来定义患者特定的疾病进展事件。 目的 评估一种使用 MSFC 分析残疾进展的新方法。方法 MSFC 进展定义为至少一个 MSFC 成分的评分较基线恶化 20% (MSFC Progression-20) 或 15% (MSFC Progression-15)，持续时间 >= 3 个月。使用那他珠单抗临床研究（那他珠单抗治疗复发缓解型多发性硬化症的安全性和有效性 [AFFIRM] 和那他珠单抗联合干扰素 Beta-1a 治疗复发缓解型多发性硬化症患者的安全性和疗效 [SENTINEL]）的数据确定进展率。确定了 MSFC 进展与其他临床指标之间的相关性，以及对治疗效果的敏感性。结果 大量患者在 1 年和 2 年均符合 MSFC 进展标准，其中 MSFC Progression-15 比 MSFC Progression-20 更敏感。 MSFC Progression-20 和 MSFC Progression-15 与扩展残疾状态量表 (EDSS) 评分变化、复发率和 SF-36 身体成分摘要 (PCS) 评分变化显着相关。 1 年时的 MSFC Progression-20 和 MSFC Progression-15 可预测 2 年时的 EDSS 进展。两个 MSFC 进展终点均在 AFFIRM 中显示了治疗效果，并且结果在 SENTINEL 中得到了重复。 结论 MSFC Progression-20 和 MSFC Progression-15 是残疾进展的敏感指标；与 EDSS、复发率和 SF-36 PCS 相关；并能够在随机对照临床研究中证明治疗效果。多发性硬化症2009； 15：984-997。 http://msj.sagepub.com
Background The initial Multiple Sclerosis Functional Composite (MSFC) proposal was a three-part composite of quantitative measures of ambulation, upper extremity function, and cognitive function expressed as a single composite Z-score. However, the clinical meaning of an MSFC Z-score change is not obvious. This study instead used MSFC component data to define a patient-specific disease progression event.Objective Evaluate a new method for analyzing disability progression using the MSFC.Methods MSFC progression was defined as worsening from baseline on scores of at least one MSFC component by 20% (MSFC Progression-20) or 15% (MSFC Progression-15), sustained for >= 3 months. Progression rates were determined using data from natalizumab clinical studies (Natalizumab Safety and Efficacy in Relapsing Remitting Multiple Sclerosis [AFFIRM] and Safety and Efficacy of Natalizumab in Combination With Interferon Beta-1a in Patients With Relapsing Remitting Multiple Sclerosis [SENTINEL]). Correlations between MSFC progression and other clinical measures were determined, as was sensitivity to treatment effects.Results Substantial numbers of patients met MSFC progression criteria, with MSFC Progression-15 being more sensitive than MSFC Progression-20, at both 1 and 2 years. MSFC Progression-20 and MSFC Progression-15 were related significantly to Expanded Disability Status Scale (EDSS) score change, relapse rate, and the SF-36 Physical Component Summary (PCS) score change. MSFC Progression-20 and MSFC Progression-15 at 1 year were predictive of EDSS progression at 2 years. Both MSFC progression end points demonstrated treatment effects in AFFIRM, and results were replicated in SENTINEL.Conclusion MSFC Progression-20 and MSFC Progression-15 are sensitive measures of disability progression; correlate with EDSS, relapse rates, and SF-36 PCS; and are capable of demonstrating therapeutic effects in randomized, controlled clinical studies. Multiple Sclerosis 2009; 15: 984-997. http://msj.sagepub.com