Disability Progression in a Clinical Trial of Relapsing-Remitting Multiple Sclerosis
Disability Progression in a Clinical Trial of Relapsing-Remitting Multiple Sclerosis
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DOI：
10.1001/archneurol.2010.150
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发表时间：
2010-11-01
影响因子：
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通讯作者：
You, Xiaojun
中科院分区：
文献类型：
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作者：
Rudick, Richard A.;Lee, Jar-Chi;You, Xiaojun
目的：调查持续至少 6 个月的扩展残疾状态量表 (EDSS) 恶化以及其他参数作为残疾状态预测因子的价值。设计：多发性硬化症协作研究组研究数据的回顾性分析。背景：肌内干扰素 β-1a 关键试验是一项双盲、安慰剂对照 3 期研究。参与者：接受至少 2 年治疗的复发缓解型多发性硬化症患者治疗并在随机化后 8 年完成 EDSS 评估。干预：在为期 1 年的临床试验期间，每周肌注 30 微克干扰素 β-1a 或安慰剂。主要结果指标：计算 2 年内 6 个月持续进展的阳性预测值，以确定预测 8 年残疾状态的能力。使用多变量逻辑回归模型来评估随访时预测因子与 EDSS 里程碑之间的关系。结果：45 名患者在临床试验期间持续了 6 个月的 EDSS 进展，115 名患者没有。试验期间的进展是随机分组 8 年后随访时达到 EDSS 里程碑的最强预测因素。其他独立预测因素是治疗组分配和基线 EDSS 评分。 结论：在这项肌注干扰素 β-1a 的 3 期临床试验中，与治疗效果、基线 EDSS 评分和研究期间的复发次数相比，持续 6 个月的 EDSS 较基线恶化 1 分或更多是随机进入临床试验 8 年后临床显着残疾的最强预测因素。
Objective: To investigate the value of Expanded Disability Status Scale (EDSS) worsening sustained for at least 6 months and other parameters as predictors for disability status.Design: Retrospective analysis of the Multiple Sclerosis Collaborative Research Group study data.Setting: The intramuscular interferon beta-1a pivotal trial was a double-blind, placebo-controlled phase 3 study.Participants: Patients with relapsing-remitting multiple sclerosis who received at least 2 years of treatment and completed an EDSS evaluation 8 years postrandomization.Intervention: Thirty micrograms of intramuscular interferon beta-1a or placebo once weekly during the -year clinical trial.Main Outcome Measures: Positive predictive values for 6-month sustained progression during 2 years were calculated to determine the ability to predict disability status at 8 years. A multivariate logistic regression model was used to assess the relationship between predictors and EDSS milestones at follow-up.Results: Forty-five patients had sustained 6-month EDSS progression during the clinical trial and 115 did not. Progression during the trial was the strongest predictor of reaching EDSS milestones at the follow-up visit, 8 years after randomization. Other independent predictors were treatment arm assignment and baseline EDSS score.Conclusion: In this phase 3 clinical trial of intramuscular interferon beta-1a, compared with effects of treatment, baseline EDSS score, and number of relapses during the study, worsening of 1 point or more on EDSS from baseline lasting 6 months was the strongest predictor of clinically significant disability 8 years after randomization into the clinical trial.