Win statistics (win ratio, win odds, and net benefit) can complement one another to show the strength of the treatment effect on time-to-event outcomes

被引:24
|
作者
Dong, Gaohong [1 ]
Huang, Bo [2 ]
Verbeeck, Johan [3 ]
Cui, Ying [4 ]
Song, James [1 ]
Gamalo-Siebers, Margaret [5 ]
Wang, Duolao [6 ]
Hoaglin, David C. [7 ]
Seifu, Yodit [8 ]
Mutze, Tobias [9 ]
Kolassa, John [10 ]
机构
[1] BeiGene, 55 Challenger Rd, Ridgefield Pk, NJ 07660 USA
[2] Pfizer Inc, Groton, CT 06340 USA
[3] Univ Hasselt, I Biostat, DSI, Hasselt, Belgium
[4] Emory Univ, Dept Biostat & Bioinformat, Atlanta, GA 30322 USA
[5] Pfizer Inc, Collegeville, PA USA
[6] Univ Liverpool Liverpool Sch Trop Med, Liverpool, Merseyside, England
[7] UMass Chan Med Sch, Dept Populat & Quantitat Hlth Sci, Worcester, MA USA
[8] Bristol Myers Squibb, Berkeley Hts, NJ USA
[9] Novartis Pharma AG, Stat Methodol, Basel, Switzerland
[10] Rutgers State Univ, Dept Stat, Piscataway, NJ USA
关键词
IPCW; IPCW-adjusted win statistics; inverse-probability-of-censoring weighting; generalized pairwise comparisons; Mann-Whitney U statistic; GENERALIZED PAIRWISE COMPARISONS; PRIORITIZED OUTCOMES; CLINICAL-TRIALS;
D O I
10.1002/pst.2251
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Conventional analyses of a composite of multiple time-to-event outcomes use the time to the first event. However, the first event may not be the most important outcome. To address this limitation, generalized pairwise comparisons and win statistics (win ratio, win odds, and net benefit) have become popular and have been applied to clinical trial practice. However, win ratio, win odds, and net benefit have typically been used separately. In this article, we examine the use of these three win statistics jointly for time-to-event outcomes. First, we explain the relation of point estimates and variances among the three win statistics, and the relation between the net benefit and the Mann-Whitney U statistic. Then we explain that the three win statistics are based on the same win proportions, and they test the same null hypothesis of equal win probabilities in two groups. We show theoretically that the Z-values of the corresponding statistical tests are approximately equal; therefore, the three win statistics provide very similar p-values and statistical powers. Finally, using simulation studies and data from a clinical trial, we demonstrate that, when there is no (or little) censoring, the three win statistics can complement one another to show the strength of the treatment effect. However, when the amount of censoring is not small, and without adjustment for censoring, the win odds and the net benefit may have an advantage for interpreting the treatment effect; with adjustment (e.g., IPCW adjustment) for censoring, the three win statistics can complement one another to show the strength of the treatment effect. For calculations we use the R package WINS, available on the CRAN (Comprehensive R Archive Network).
引用
收藏
页码:20 / 33
页数:14
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