Comparison of Treatment Effects Measured by the Hazard Ratio and by the Ratio of Restricted Mean Survival Times in Oncology Randomized Controlled Trials
Comparison of Treatment Effects Measured by the Hazard Ratio and by the Ratio of Restricted Mean Survival Times in Oncology Randomized Controlled Trials
复制标题
DOI：
10.1200/jco.2015.64.2488
复制
发表时间：
2016-05-20
影响因子：
45.3
通讯作者：
Porcher, Raphael
中科院分区：
文献类型：
--
作者：
Trinquart, Ludovic;Jacot, Justine;Porcher, Raphael
PurposeWe的目的是比较经验的治疗效果衡量的风险比（HR）和差异（和比率）的限制平均生存时间（RMST）在oncology randomized trials.MethodsWe选择肿瘤随机对照试验从五个领先的期刊在过去6个月的2014年。我们重建了每个试验的一个事件发生时间结局的个体患者数据，最好是主要结局。我们重新分析了每个试验，并比较了HR估计的治疗效果与RMST的差异（和比值）。我们估计的平均比率的HR的比率RMST;平均比率小于1表示更乐观的评估与HRs.ResultsWe分析了54项随机对照试验，共33,212例患者。21项（39%）试验的选定结局为总生存期。13项（24%）试验中存在风险不成比例的证据。HR和基于RRST的测量结果在统计学意义上一致，但有一种情况除外。中位HR为0.84（Q1至Q3范围，0.67至0.97），RMST的中位差异为1.12个月（范围，0.22至2.75个月）。HR与RMST比值的平均比值为1.11（95% CI，1.07至1.15），试验间变异性较大（I-2 = 86%）。结果是一致的结果类型（总生存率与其他结果），无论是比例风险假设举行或not.ConclusionOn平均，HR提供了显着更大的治疗效果估计比RMST的比率。当绝对效应很小时，HR可能看起来很大。在随机试验中应常规报告基于RCST的指标，并提供至事件发生时间结局。
PurposeWe aimed to compare empirically the treatment effects measured by the hazard ratio (HR) and by the difference (and ratio) of restricted mean survival times (RMST) in oncology randomized trials.MethodsWe selected oncology randomized controlled trials from five leading journals during the last 6 months of 2014. We reconstructed individual patient data for one time-to-event outcome from each trial, preferably the primary outcome. We reanalyzed each trial and compared the treatment effect estimated by the HR with that by the difference (and ratio) of RMST. We estimated an average ratio of the HR to the ratio of RMST; an average ratio less than one indicates more optimistic assessments with HRs.ResultsWe analyzed 54 randomized controlled trials totaling 33,212 patients. The selected outcome was overall survival in 21 (39%) trials. There was evidence of nonproportionality of hazards in 13 (24%) trials. The HR and RMST-based measures were in agreement regarding the statistical significance of the effect, except in one case. The median HR was 0.84 (Q1 to Q3 range, 0.67 to 0.97) and the median difference in RMST was 1.12 months (range, 0.22 to 2.75 months). The average ratio of the HR to the ratio of RMST was 1.11 (95% CI, 1.07 to 1.15), with substantial between-trial variability (I-2 = 86%). Results were consistent by outcome type (overall survival v other outcomes) and whether the proportional hazard assumption held or not.ConclusionOn average, the HR provided significantly larger treatment effect estimates than the ratio of RMST. The HR may seem large when the absolute effect is small. RMST-based measures should be routinely reported in randomized trials with time-to-event outcomes.