Adopting a Patient-Centered Approach to Primary Outcome Analysis of Acute Stroke Trials Using a Utility-Weighted Modified Rankin Scale.
Adopting a Patient-Centered Approach to Primary Outcome Analysis of Acute Stroke Trials Using a Utility-Weighted Modified Rankin Scale.
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DOI：
10.1161/strokeaha.114.008547
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发表时间：
2015-08
期刊：
影响因子：
8.3
通讯作者：
Additional contributors from DAWN Trial Steering Committee
中科院分区：
文献类型：
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作者：
Chaisinanunkul N;Adeoye O;Lewis RJ;Grotta JC;Broderick J;Jovin TG;Nogueira RG;Elm JJ;Graves T;Berry S;Lees KR;Barreto AD;Saver JL;DAWN Trial and MOST Trial Steering Committees;Additional contributors from DAWN Trial Steering Committee
尽管改良兰金量表（mRS）是急性卒中试验中最常用的主要终点，但以二分方式分析时其把握度有限，并且在进行常规分析时其效应量指示难以解释。通过效用对七个兰金水平进行加权可以在保持统计功效的同时提高规模可解释性。通过对时间权衡（以患者为中心）和人员权衡（以临床医生为中心）研究的平均值推导出效用加权mRS（UW-mRS）。将UW-mRS、标准顺序mRS和二分mRS应用于11项急性卒中治疗试验或荟萃分析，包括溶解性、血管内再灌注、血压调节和半颅骨切除术干预。效用值为：mRS 0-1.0; mRS 1 - 0.91; mRS 2 - 0.76; mRS 3 - 0.65; mRS 4 - 0.33; mRS 5和6 - 0。对于具有单向治疗效应的试验，UW-mRS优于有序mRS，优于二分mRS分析。UW-mRS和顺序mRS在8项单向效应试验中的6项中具有统计学显著性，而二分分析在8项试验中的2 - 4项中具有统计学显著性。在双向效应试验中，UW-mRS和顺序检验均通过显示中性结果捕获了不同的治疗效应，而一些二分分析显示了阳性结果。具有统计学显著性阳性结果的试验的平均效用差异范围为0.026至0.249。效用加权mRS在检测实际卒中试验中的治疗效果方面与标准顺序mRS相似，并确保定量结局有效反映以患者为中心的获益。
Although the modified Rankin Scale (mRS) is the most commonly employed primary endpoint in acute stroke trials, its power is limited when analyzed in dichotomized fashion and its indication of effect size challenging to interpret when analyzed ordinally. Weighting the seven Rankin levels by utilities may improve scale interpretability while preserving statistical power. A utility weighted mRS (UW-mRS) was derived by averaging values from time-tradeoff (patient centered) and person-tradeoff (clinician centered) studies. The UW-mRS, standard ordinal mRS, and dichotomized mRS were applied to 11 trials or meta-analyses of acute stroke treatments, including lytic, endovascular reperfusion, blood pressure moderation, and hemicraniectomy interventions. Utility values were: mRS 0–1.0; mRS 1 - 0.91; mRS 2 - 0.76; mRS 3 - 0.65; mRS 4 - 0.33; mRS 5 & 6 - 0. For trials with unidirectional treatment effects, the UW-mRS paralleled the ordinal mRS and outperformed dichotomous mRS analyses. Both the UW-mRS and the ordinal mRS were statistically significant in six of eight unidirectional effect trials, while dichotomous analyses were statistically significant in two to four of eight. In bidirectional effect trials, both the UW-mRS and ordinal tests captured the divergent treatment effects by showing neutral results whereas some dichotomized analyses showed positive results. Mean utility differences in trials with statistically significant positive results ranged from 0.026 to 0.249. A utility-weighted mRS performs similarly to the standard ordinal mRS in detecting treatment effects in actual stroke trials and ensures the quantitative outcome is a valid reflection of patient-centered benefits.