A group sequential test for treatment effect based on the Fine-Gray model.

TitleA group sequential test for treatment effect based on the Fine-Gray model.
Publication TypeJournal Article
Year of Publication2018
AuthorsMartens, MJ, Logan, BR
JournalBiometrics
Volume74
Issue3
Pagination1006-1013
Date Published2018 09
ISSN1541-0420
KeywordsBone Marrow Transplantation, Clinical Trials as Topic, Computer Simulation, Endpoint Determination, Humans, Long Term Adverse Effects, Peripheral Blood Stem Cell Transplantation, Risk, Treatment Failure, Treatment Outcome
Abstract

Competing risks endpoints arise when patients can fail therapy from several causes. Analyzing these outcomes allows one to assess directly the benefit of treatment on a primary cause of failure in a clinical trial setting. Regression models can be used in clinical trials to adjust for residual imbalances in patient characteristics, improving the power to detect treatment differences. But, none of the competing risks methods currently available for use in group sequential trials adjust for covariates. We propose a group sequential test for treatment effect that, because it is based on the Fine-Gray model, permits adjustment for covariates. Our derivations show that its sequence of test statistics has an asymptotic distribution with an independent increments structure, which allows standard techniques such as O'Brien-Fleming designs and error spending functions to be employed to meet type I error rate and power specifications. We demonstrate the test in a reanalysis of BMT CTN 0402, a phase III clinical trial that evaluated an experimental treatment for the prevention of adverse outcomes following blood and marrow transplant. Moreover, using a simulation study of randomized group sequential trials, we demonstrate that the proposed method preserves the type I error rate and power at their nominal levels in the presence of influential covariates.

DOI10.1111/biom.12871
Alternate JournalBiometrics
PubMed ID29534294
PubMed Central IDPMC6146968
Grant ListU10 HL069294 / HL / NHLBI NIH HHS / United States
U24 CA076518 / CA / NCI NIH HHS / United States
U24 HL138660 / HL / NHLBI NIH HHS / United States
UG1 HL069278 / HL / NHLBI NIH HHS / United States
F31 HL134317 / HL / NHLBI NIH HHS / United States