Title: Power and sample size calculations for discrete bounded outcome scores
Authors: Tsonaka, Spyridoula ×
Rizopoulos, Dimitrios
Lesaffre, Emmanuel #
Issue Date: Dec-2006
Publisher: John Wiley & Sons
Series Title: Statistics in Medicine vol:25 issue:24 pages:4241-4252
Abstract: We consider power and sample size calculations for randomized trials with a bounded outcome score (BOS) as primary response adjusted for a priori chosen covariates. We define BOS to be a random variable restricted to a finite interval. Typically, a BOS has a J- or U-shaped distribution hindering traditional parametric methods of analysis. When no adjustment for covariates is needed, a non-parametric test could be chosen. However, there is still a problem with calculating the power since the common location-shift alternative does not hold in general for a BOS. In this paper, we consider a parametric approach and assume that the observed BOS is a coarsened version of a true BOS, which has a logit-normal distribution in each treatment group allowing correction for covariates. A two-step procedure is used to calculate the power. Firstly, the power function is defined conditionally on the covariate values. Secondly, the marginal power is obtained by averaging the conditional power with respect to an assumed distribution for the covariates using Monte Carlo integration. A simulation study evaluates the performance of our method which is also applied to the ECASS-1 stroke study.
ISSN: 0277-6715
Publication status: published
KU Leuven publication type: IT
Appears in Collections:Leuven Biostatistics and Statistical Bioinformatics Centre (L-BioStat)
× corresponding author
# (joint) last author

Files in This Item:

There are no files associated with this item.

Request a copy


All items in Lirias are protected by copyright, with all rights reserved.

© Web of science