A small sample study of the STEPP approach to assessing treatment-covariate interactions in survival data.
A new, intuitive method has recently been proposed to explore treatment-covariate interactions in survival data arising from two treatment arms of a clinical trial. The method is based on constructing overlapping subpopulations of patients with respect to one (or more) covariates of interest and in observing the pattern of the treatment effects estimated across the subpopulations. A plot of these treatment effects is called a subpopulation treatment effect pattern plot. Here, we explore the small sample characteristics of the asymptotic results associated with the method and develop an alternative permutation distribution-based approach to inference that should be preferred for smaller sample sizes. We then describe an extension of the method to the case in which the pattern of estimated quantiles of survivor functions is of interest.
Bonetti
Marco
M
Department of Decision Sciences, Bocconi University, Via Röntgen 1, Milan 20136, Italy. marco.bonetti@unibocconi.it
Zahrieh
David
D
Cole
Bernard F
BF
Gelber
Richard D
RD
D002986:Clinical Trials as Topic / Q000379:methods*; D006801:Humans; D015233:Models, Statistical; D016019:Survival Analysis; D016896:Treatment Outcome