Novel end point analytic techniques and interpreting shifts across the entire range of outcome scales in acute stroke trials
Novel end point analytic techniques and interpreting shifts across the entire range of outcome scales in acute stroke trials
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DOI：
10.1161/strokeaha.107.488536
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发表时间：
2007-11-01
期刊：
影响因子：
8.3
通讯作者：
Saver, Jeffrey L.
中科院分区：
文献类型：
--
作者：
Saver, Jeffrey L.
背景和目的--卒中治疗通常不能治愈，而是在整个功能测量范围内改变患者的结果。结果量表的二分法降低了计算复杂性，但丢弃了大量的结果信息，人为地仅将单个健康状态转变视为具有临床意义，并且通常降低了研究能力。终点分析的新方法有几个优点。综述总结-全球统计同时评估多个结局指标的治疗效果。然而，将群体水平的全局统计多维向量效应转化为个体患者预期的获益或损害是有问题的。应答者分析根据研究入组时患者卒中严重程度调整结局阈值，确定每位患者可实现的目标。然而，应答者分析仍然丢弃了大量结果信息。变化分析测量在确定的结果的全部范围内的结果分布的变化，在患者和临床医生重视的所有健康状态转变中纳入益处和危害，并且通常增加研究力量。使用最近开发的联合结果表规范技术，可以将变化分析的结果翻译成临床可访问的术语，该技术产生了以下值，即1例患者以临床重要方式改善所需治疗的数量：蛛网膜下腔出血的尼莫地平，6.8;弹簧圈夹闭术，5.9;急性脑缺血的动脉内尿激酶原，4.8;静脉注射组织纤溶酶原激活剂，3.3.结论--二分法、整体统计法、应答者分析法和变化分析法各有其独特的优点和缺点。主要终点分析技术的选择应根据研究人群、预期治疗反应和研究目的进行调整。变化分析通常提供治疗的临床影响的最全面的指标。
Background and Purpose - Stroke treatments are generally not curative, but rather alter patient outcome over the entire range of functional measures. Dichotomizing outcome scales reduces computational complexity, but discards substantial outcome information, artificially privileges only a single health state transition as clinically meaningful, and often reduces study power. Newer approaches to endpoint analysis have several advantageous properties.Summary of Review - The global statistic assesses treatment effects on multiple outcome measures simultaneously. However, translating the global statistic multidimensional vector effect at the population level into benefit or harm expected in the individual patient is problematic. Responder analysis adjusts outcome thresholds to patient stroke severity at study entry, identifying achievable goals for each patient. However, responder analysis still discards substantial outcome information. Shift analysis gauges change in outcome distributions over the full range of ascertained outcomes, incorporating benefit and harm at all health state transitions valued by patients and clinicians, and often increasing study power. Translation of findings of shift analyses into clinically accessible terms may be accomplished using the recently developed joint outcome table specification technique, which yields the following values for the number needed to treat for 1 patient to improve in a clinically important manner: nimodipine in subarachnoid hemorrhage, 6.8; coiling over clipping, 5.9; intra- arterial pro- urokinase in acute cerebral ischemia, 4.8; intravenous tissue plasminogen activator, 3.3.Conclusions - Dichotomized, global statistic, responder, and shift analyses each offer distinctive benefits and drawbacks. Choice of primary end point analytic technique should be tailored to the study population, expected treatment response, and study purpose. Shift analysis generally provides the most comprehensive index of a treatment's clinical impact.