Generalized pairwise comparisons of prioritized outcomes in the two-sample problem

被引:169
作者
Buyse, Marc [1 ,2 ]
机构
[1] Int Inst Drug Dev, B-1340 Louvain, Belgium
[2] Hasselt Univ, Interuniv Inst Biostat & Stat Bioinformat I BioSt, Hasselt, Belgium
关键词
generalized pairwise comparisons; prioritized outcomes; measure of treatment effect; randomization test; INTUITIVE NONPARAMETRIC APPROACH; ENHANCE COMMUNICATION; PROBABILISTIC INDEX; CLINICAL-TRIALS; HAZARD RATIO; PROPORTION; MORTALITY; SIZE;
D O I
10.1002/sim.3923
中图分类号
Q [生物科学];
学科分类号
07 ; 0710 ; 09 ;
摘要
This paper extends the idea behind the U-statistic of the Wilcoxon-Mann-Whitney test to perform generalized pairwise comparisons between two groups of observations. The observations are outcomes captured by a single variable, possibly repeatedly measured, or by several variables of any type (e.g. discrete, continuous, time to event). When several outcomes are considered, they must be prioritized. We show that generalized pairwise comparisons extend well-known non-parametric tests, and illustrate their interest using data from two randomized clinical trials. We also show that they lead to a general measure of the difference between the groups called the 'proportion in favor of treatment', denoted Delta, which is related to traditional measures of treatment effect for a single variable. Copyright (C) 2010 John Wiley & Sons, Ltd.
引用
收藏
页码:3245 / 3257
页数:13
相关论文
共 43 条
[1]   Probabilistic index: an intuitive non-parametric approach to measuring the size of treatment effects [J].
Acion, L ;
Peterson, JJ ;
Temple, S ;
Arndt, S .
STATISTICS IN MEDICINE, 2006, 25 (04) :591-602
[2]  
[Anonymous], 2007, Randomization tests
[3]  
[Anonymous], 1976, Educational Researcher, DOI [DOI 10.3102/0013189X005010003, DOI 10.2307/1174772, 10.3102/0013189x005010003, 10.2307/1174772ISSN0536-1036]
[4]  
[Anonymous], 2000, GROUP SEQUENTIAL MET
[5]   Visual acuity as an outcome measure in clinical trials of retinal diseases [J].
Beck, Roy W. ;
Maguire, Maureen G. ;
Bressler, Neil M. ;
Glassman, Adam R. ;
Lindblad, Anne S. ;
Ferris, Frederick L. .
OPHTHALMOLOGY, 2007, 114 (10) :1804-1809
[6]   Reformulating the hazard ratio to enhance communication with clinical investigators [J].
Buyse, Marc .
CLINICAL TRIALS, 2008, 5 (06) :641-642
[7]  
COX DR, 1972, J R STAT SOC B, V34, P187
[8]   Leucovorin and fluorouracil with or without oxaliplatin as first-line treatment in advanced colorectal cancer [J].
de Gramont, A ;
Figer, A ;
Seymour, M ;
Homerin, M ;
Hmissi, A ;
Cassidy, J ;
Boni, C ;
Cortes-Funes, H ;
Cervantes, A ;
Freyer, G ;
Papamichael, D ;
Le Bail, N ;
Louvet, C ;
Hendler, D ;
de Braud, F ;
Wilson, C ;
Morvan, F ;
Bonetti, A .
JOURNAL OF CLINICAL ONCOLOGY, 2000, 18 (16) :2938-2947
[9]  
Desu M.M., 2004, NONPARAMETRIC STAT M, DOI DOI 10.1201/9781482285895
[10]  
Efron B., 1967, Proceedings of the Fifth Berkeley Symposium on Mathematical Statistics and Probability, Volume 4: Biology and Problems of Health, P831