An extension of generalized pairwise comparisons for prioritized outcomes in the presence of censoring

被引:43
|
作者
Peron, Julien [1 ,2 ]
Buyse, Marc [3 ,4 ]
Ozenne, Brice [1 ,2 ]
Roche, Laurent [1 ,2 ]
Roy, Pascal [1 ,2 ]
机构
[1] Hosp Civils Lyon, Ctr Hosp Lyon Sud, Serv Biostat, Inst Canc, Lyon, France
[2] Univ Lyon 1, Lab Biometrie & Biol Evolut, Equipe Biostat Sante, CNRS,UMR 5558, Villeurbanne, France
[3] IDDI, Raleigh, NC USA
[4] Hasselt Univ, Interuniv Inst Biostat & Stat Bioinformat I BioSt, Hasselt, Belgium
关键词
Pairwise comparisons; multivariate analysis; randomized trial; survival outcome; COMPOSITE END-POINTS; CLINICAL-TRIALS; PANCREATIC-CANCER; ERLOTINIB; RATIO;
D O I
10.1177/0962280216658320
中图分类号
R19 [保健组织与事业(卫生事业管理)];
学科分类号
摘要
Generalized pairwise comparisons have been proposed to permit a comprehensive assessment of several prioritized outcomes between two groups of observations. This procedure estimates , the net chance of a better outcome with treatment than with control by comparing the patients outcomes among all possible pairs taking one patient from the treatment group and one patient from the control group. For time to event outcomes, the standard procedure of generalized pairwise comparisons is analogous to the Gehan's modification of the Mann-Whitney test which is biased in presence of censored observation and less powerful than Efron's modification of this test. We adapt Efron's modification to generalized pairwise comparisons. We show how a pairwise contribution to can be calculated from the estimates of the survival function in the presence of right-censored data. We performed a simulation study to assess the bias, the type I error and the power of the new procedure. The estimate of with the new procedure is only slightly biased even in presence of heavy censoring. We also show how this bias can be corrected when only one time-to-event outcome is analyzed. The new procedure has higher power in most cases compared to the standard procedure.
引用
收藏
页码:1230 / 1239
页数:10
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