iStats Inc., Long Island City, New York, USA.
Pfizer Inc., Groton, Connecticut, USA.
Pharm Stat. 2021 May;20(3):440-450. doi: 10.1002/pst.2086. Epub 2020 Nov 28.
For composite outcomes whose components can be prioritized on clinical importance, the win ratio, the net benefit and the win odds apply that order in comparing patients pairwise to produce wins and subsequently win proportions. Because these three statistics are derived using the same win proportions and they test the same hypothesis of equal win probabilities in the two treatment groups, we refer to them as win statistics. These methods, particularly the win ratio and the net benefit, have received increasing attention in methodological research and in design and analysis of clinical trials. For time-to-event outcomes, however, censoring may introduce bias. Previous work has shown that inverse-probability-of-censoring weighting (IPCW) can correct the win ratio for bias from independent censoring. The present article uses the IPCW approach to adjust win statistics for dependent censoring that can be predicted by baseline covariates and/or time-dependent covariates (producing the CovIPCW-adjusted win statistics). Theoretically and with examples and simulations, we show that the CovIPCW-adjusted win statistics are unbiased estimators of treatment effect in the presence of dependent censoring.
对于可以根据临床重要性对其组成部分进行优先排序的复合结局,赢率、净效益和赢odds 适用于按序比较患者对,以产生赢和随后的赢比例。由于这三个统计量是使用相同的赢比例得出的,并且它们检验了两组治疗中相等赢概率的相同假设,因此我们将它们称为赢统计量。这些方法,特别是赢率和净效益,在方法学研究以及临床试验的设计和分析中受到越来越多的关注。然而,对于生存时间结局,删失可能会引入偏倚。先前的工作表明,逆概率删失加权(Inverse-Probability-of-Censoring Weighting,IPCW)可以纠正由独立删失引起的赢率偏倚。本文使用 IPCW 方法调整依赖于可由基线协变量和/或时变协变量预测的删失的赢统计量(产生 CovIPCW 调整后的赢统计量)。通过理论、实例和模拟,我们表明,在存在依赖删失的情况下,CovIPCW 调整后的赢统计量是治疗效果的无偏估计量。