Sample size calculation for studies with grouped survival data.

TitleSample size calculation for studies with grouped survival data.
Publication TypeJournal Article
Year of Publication2018
AuthorsLi, Zhiguo, Xiaofei Wang, Yuan Wu, and Kouros Owzar
JournalStat Med
Volume37
Issue27
Pagination3904-3917
Date Published2018 11 30
ISSN1097-0258
KeywordsData Interpretation, Statistical, Humans, Likelihood Functions, Models, Statistical, Proportional Hazards Models, Sample Size, Statistics as Topic, Survival Analysis
Abstract

Grouped survival data arise often in studies where the disease status is assessed at regular visits to clinic. The time to the event of interest can only be determined to be between two adjacent visits or is right censored at one visit. In data analysis, replacing the survival time with the endpoint or midpoint of the grouping interval leads to biased estimators of the effect size in group comparisons. Prentice and Gloeckler developed a maximum likelihood estimator for the proportional hazards model with grouped survival data and the method has been widely applied. Previous work on sample size calculation for designing studies with grouped data is based on either the exponential distribution assumption or the approximation of variance under the alternative with variance under the null. Motivated by studies in HIV trials, cancer trials and in vitro experiments to study drug toxicity, we develop a sample size formula for studies with grouped survival endpoints that use the method of Prentice and Gloeckler for comparing two arms under the proportional hazards assumption. We do not impose any distributional assumptions, nor do we use any approximation of variance of the test statistic. The sample size formula only requires estimates of the hazard ratio and survival probabilities of the event time of interest and the censoring time at the endpoints of the grouping intervals for one of the two arms. The formula is shown to perform well in a simulation study and its application is illustrated in the three motivating examples.

DOI10.1002/sim.7847
Alternate JournalStat Med
Original PublicationSample size calculation for studies with grouped survival data.
PubMed ID29888484
PubMed Central IDPMC6262878
Grant ListP01 CA142538 / CA / NCI NIH HHS / United States
S10 OD018164 / OD / NIH HHS / United States
P01CA142538 / CA / NCI NIH HHS / United States
Project: