A Generalized Logrank-type Test for Comparison of Treatment Regimes in Sequential Multiple Assignment Randomized Trials
arxiv(2024)
摘要
The sequential multiple assignment randomized trial (SMART) is the
ideal study design for the evaluation of multistage treatment
regimes, which comprise sequential decision rules that recommend
treatments for a patient at each of a series of decision points
based on their evolving characteristics. A common goal is to
compare the set of so-called embedded regimes represented in the
design on the basis of a primary outcome of interest. In the study
of chronic diseases and disorders, this outcome is often a time to
an event, and a goal is to compare the distributions of the
time-to-event outcome associated with each regime in the set. We
present a general statistical framework in which we develop a
logrank-type test for comparison of the survival distributions
associated with regimes within a specified set based on the data
from a SMART with an arbitrary number of stages that allows
incorporation of covariate information to enhance efficiency and can
also be used with data from an observational study. The framework
provides clarification of the assumptions required to yield a
principled test procedure, and the proposed test subsumes or offers
an improved alternative to existing methods. We demonstrate
performance of the methods in a suite of simulation
studies. The methods are applied to a SMART in patients with acute
promyelocytic leukemia.
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