Design and analysis considerations for comparing dynamic treatment regimens with binary outcomes from sequential multiple assignment randomized trials

2018
In behavioral, educational and medical practice, interventions are often personalized over time using strategies that are based on individual behaviors and characteristics and changes in symptoms, severity, or adherence that are a result of one's treatment. Such strategies that more closely mimic real practice, are known as dynamic treatment regimens (DTRs). A sequential multiple assignment randomized trial (SMART) is a multi-stage trial design that can be used to construct effective DTRs. This article reviews a simple to use weighted and replicated' estimation technique for comparing DTRs embedded in a SMART design using logistic regression for a binary, end-of-study outcome variable. Based on a Wald test that compares two embedded DTRs of interest from the weighted and replicated' regression model, a sample size calculation is presented with a corresponding user-friendly applet to aid in the process of designing a SMART. The analytic models and sample size calculations are presented for three of the more commonly used two-stage SMART designs. Simulations for the sample size calculation show the empirical power reaches expected levels. A data analysis example with corresponding code is presented in the appendix using data from a SMART developing an effective DTR in autism.
JOURNAL OF APPLIED STATISTICS
页码:1628-1651|卷号:45|期号:9
ISSN:0266-4763
收录类型
SSCI
发表日期
2018
学科领域
循证社会科学-方法
国家
美国
语种
英语
DOI
10.1080/02664763.2017.1386773
其他关键词
HIGH-RISK NEUROBLASTOMA; SAMPLE-SIZE; LOGISTIC-REGRESSION; OLDER PATIENTS; MODELS; SURVIVAL; THERAPY; INTERVENTIONS; CHEMOTHERAPY; STRATEGIES
EISSN
1360-0532
资助机构
[ME-1507-31108]; [P50DA039838]; [R01DA039901]; [R01HD073975]; [R03M H09795401]; [5T32CA083654-12]; EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENTUnited States Department of Health & Human ServicesNational Institutes of Health (NIH) - USANIH Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD) [R01HD073975] Funding Source: NIH RePORTER; NATIONAL CANCER INSTITUTEUnited States Department of Health & Human ServicesNational Institutes of Health (NIH) - USANIH National Cancer Institute (NCI) [T32CA083654] Funding Source: NIH RePORTER; NATIONAL INSTITUTE OF MENTAL HEALTHUnited States Department of Health & Human ServicesNational Institutes of Health (NIH) - USANIH National Institute of Mental Health (NIMH) [R03MH097954] Funding Source: NIH RePORTER; NATIONAL INSTITUTE ON DRUG ABUSEUnited States Department of Health & Human ServicesNational Institutes of Health (NIH) - USANIH National Institute on Drug Abuse (NIDA)European Commission [P50DA039838, R01DA039901] Funding Source: NIH RePORTER
资助信息
The development of this article was funded by the following grants: ME-1507-31108, P50DA039838, R01DA039901, R01HD073975, R03M H09795401, and 5T32CA083654-12.
被引频次(WOS)
9
被引更新日期
2022-01
来源机构
University of Michigan System University of Michigan University of Michigan System University of Michigan University of California System University of California Los Angeles University of Michigan System University of Michigan
关键词
Adaptive interventions dynamic treatment regimes sequential multiple assignment randomized trial inverse-probability-of-treatment weighting sample size