Generalised pairwise comparisons for trend: An extension to the win ratio and win odds for dose-response and prognostic variable analysis with arbitrary statements of outcome preference

被引:0
作者
Johns, Hannah [1 ]
Campbell, Bruce [2 ,3 ]
Bernhardt, Julie [4 ]
Churilov, Leonid [1 ]
机构
[1] Univ Melbourne, Melbourne Med Sch, Melbourne, Australia
[2] Univ Melbourne, Melbourne Brain Ctr, Dept Med & Neurol, Melbourne, Australia
[3] Univ Melbourne, Royal Melbourne Hosp, Melbourne, Australia
[4] Univ Melbourne, Florey Inst Neurosci & Mental Hlth, Melbourne, Australia
关键词
Pairwise comparisons; win ratio; generalised odds ratio; dose-response analysis; prognostic variable analysis; COMPOSITE END-POINTS; CLINICAL-TRIALS; STROKE;
D O I
10.1177/09622802221146306
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
The win ratio is a novel approach for handling complex patient outcomes that have seen considerable interest in the medical statistics literature, and operates by considering all-to-all pairwise statements of preference on outcomes. Recent extensions to the method have focused on the two-group case, with few developments made for considering the impact of a well-ordered explanatory variable, which would allow for dose-response analysis or the analysis of links between complex patient outcomes and prognostic variables. Where such methods have been developed, they are semiparametric methods that can only be applied to survival outcomes. In this article, we introduce the generalised pairwise comparison for trend, a modified form of Agresti's generalised odds ratio. This approach is capable of considering arbitrary statements of preference, thus enabling its use across all types of outcome data. We provide a simulation study validating the approach and illustrate it with three clinical applications in stroke research.
引用
收藏
页码:609 / 625
页数:17
相关论文
共 50 条
[1]   GENERALIZED ODDS RATIOS FOR ORDINAL DATA [J].
AGRESTI, A .
BIOMETRICS, 1980, 36 (01) :59-67
[2]  
[Anonymous], 2011, Oxford Handbook of Medical Statistics
[3]   Outcomes validity and reliability of the modified Rankin scale: Implications for stroke clinical trials - A literature review and synthesis [J].
Banks, Jamie L. ;
Marotta, Charles A. .
STROKE, 2007, 38 (03) :1091-1096
[4]   Large sample inference for a win ratio analysis of a composite outcome based on prioritized components [J].
Bebu, Ionut ;
Lachin, John M. .
BIOSTATISTICS, 2016, 17 (01) :178-187
[5]   Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS [J].
Benatar, Michael ;
Wuu, Joanne ;
Andersen, Peter M. ;
Atassi, Nazem ;
David, William ;
Cudkowicz, Merit ;
Schoenfeld, David .
NEUROLOGY, 2018, 90 (07) :E565-E574
[6]   Efficacy and safety of very early mobilisation within 24 h of stroke onset (AVERT): a randomised controlled trial [J].
Bernhardt, Julie ;
Langhorne, Peter ;
Lindley, Richard I. ;
Thrift, Amanda G. ;
Ellery, Fiona ;
Collier, Janice ;
Churilov, Leonid ;
Moodie, Marjory ;
Dewey, Helen ;
Donnan, Geoffrey .
LANCET, 2015, 386 (9988) :46-55
[7]   Systemic steroids in patients with COVID-19: pros and contras, an endocrinological point of view [J].
Berton, A. M. ;
Prencipe, N. ;
Giordano, R. ;
Ghigo, E. ;
Grottoli, S. .
JOURNAL OF ENDOCRINOLOGICAL INVESTIGATION, 2021, 44 (04) :873-875
[8]   Latent variables in psychology and the social sciences [J].
Bollen, KA .
ANNUAL REVIEW OF PSYCHOLOGY, 2002, 53 :605-634
[9]   Simulating Survival Data Using the simsurv R Package [J].
Brilleman, Samuel L. ;
Wolfe, Rory ;
Moreno-Betancur, Margarita ;
Crowther, Michael J. .
JOURNAL OF STATISTICAL SOFTWARE, 2021, 97 (03) :1-27
[10]   MEASUREMENTS OF ACUTE CEREBRAL INFARCTION - A CLINICAL EXAMINATION SCALE [J].
BROTT, T ;
ADAMS, HP ;
OLINGER, CP ;
MARLER, JR ;
BARSAN, WG ;
BILLER, J ;
SPILKER, J ;
HOLLERAN, R ;
EBERLE, R ;
HERTZBERG, V ;
RORICK, M ;
MOOMAW, CJ ;
WALKER, M .
STROKE, 1989, 20 (07) :864-870