Group sequential tests for treatment effect on survival and cumulative incidence at a fixed time point. Lifetime Data Anal 2020 Jul;26(3):603-623
Date
11/16/2019Pubmed ID
31729633Pubmed Central ID
PMC7365590DOI
10.1007/s10985-019-09491-zScopus ID
2-s2.0-85075483839 (requires institutional sign-in at Scopus site) 1 CitationAbstract
Medical research frequently involves comparing an event time of interest between treatment groups. Rather than comparing the entire survival or cumulative incidence curves, it is sometimes preferable to evaluate these probabilities at a fixed point in time. Performing a covariate adjusted analysis can improve efficiency, even in randomized clinical trials, but no currently available group sequential test for fixed point analysis provides this adjustment. This paper introduces covariate adjusted group sequential pointwise comparisons of survival and cumulative incidence probabilities. Their test statistics have an asymptotic distribution with independent increments, permitting use of common stopping boundary specification methods. These tests are demonstrated through a redesign of BMT CTN 0402, a clinical trial that evaluated a prophylactic treatment for adverse outcomes following blood and marrow transplantation. A simulation study demonstrates that these tests maintain the type I error rate and power at nominal levels under a variety of settings involving influential covariates.
Author List
Martens MJ, Logan BRAuthors
Brent R. Logan PhD Director, Professor in the Institute for Health and Equity department at Medical College of WisconsinMichael Martens PhD Assistant Professor in the Institute for Health and Equity department at Medical College of Wisconsin
MESH terms used to index this publication - Major topics in bold
BiasClinical Trials as Topic
Computer Simulation
Humans
Incidence
Regression Analysis
Survival Analysis
Time
Treatment Outcome
