Group sequential tests for treatment effect on survival and cumulative incidence at a fixed time point.

TitleGroup sequential tests for treatment effect on survival and cumulative incidence at a fixed time point.
Publication TypeJournal Article
Year of Publication2020
AuthorsMartens, MJ, Logan, BR
JournalLifetime Data Anal
Volume26
Issue3
Pagination603-623
Date Published2020 07
ISSN1572-9249
KeywordsBias, Clinical Trials as Topic, Computer Simulation, Humans, Incidence, Regression Analysis, Survival Analysis, Time, Treatment Outcome
Abstract

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.

DOI10.1007/s10985-019-09491-z
Alternate JournalLifetime Data Anal
PubMed ID31729633
PubMed Central IDPMC7365590
Grant ListU10 HL069294 / HL / NHLBI NIH HHS / United States
F31 HL134317 / HL / NHLBI NIH HHS / United States
U10HL069294 / CA / NCI NIH HHS / United States
U24 CA076518 / CA / NCI NIH HHS / United States
U24 HL138660 / HL / NHLBI NIH HHS / United States