Design of sequentially randomized trials for testing adaptive treatment strategies
Abstract
An adaptive treatment strategy (ATS) is an outcome‐guided algorithm that allows personalized treatment of complex diseases based on patients' disease status and treatment history. Conditions such as AIDS, depression, and cancer usually require several stages of treatment because of the chronic, multifactorial nature of illness progression and management. Sequential multiple assignment randomized (SMAR) designs permit simultaneous inference about multiple ATSs, where patients are sequentially randomized to treatments at different stages depending upon response status. The purpose of the article is to develop a sample size formula to ensure adequate power for comparing two or more ATSs. Based on a Wald‐type statistic for comparing multiple ATSs with a continuous endpoint, we develop a sample size formula and test it through simulation studies. We show via simulation that the proposed sample size formula maintains the nominal power. The proposed sample size formula is not applicable to designs with time‐to‐event endpoints but the formula will be useful for practitioners while designing SMAR trials to compare adaptive treatment strategies. Copyright © 2015 John Wiley & Sons, Ltd.
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SMART designs make no a priori assumptions about the existence or form of delayed treatment effects.
“SMART designs make no a priori assumptions about the existence or form of delayed treatment effects [51, 52]”
From A Sequential Multiple Assignment Randomized Trial (SMART) study of medication and CBT sequencing in the treatment of pediatric anxiety disorders (2021) · cites this paper as [52]
SMART designs are ideal for developing an adaptive intervention that optimizes outcomes based on patient treatment history.
“making them ideal for developing an adaptive intervention that optimizes outcomes based on patient treatment history [51–54].”
From A Sequential Multiple Assignment Randomized Trial (SMART) study of medication and CBT sequencing in the treatment of pediatric anxiety disorders (2021) · cites this paper as [52]
This will be a single-blind SMART design of 24-week treatment duration that will employ two sequential levels of randomization, one in each of two 12-week stages.
“This will be a single-blind [126, 127] SMART design [51, 52] of 24-week treatment duration that will employ two sequential levels of randomization, one in each of two 12-week stages of the study (Fig. 2).”
From A Sequential Multiple Assignment Randomized Trial (SMART) study of medication and CBT sequencing in the treatment of pediatric anxiety disorders (2021) · cites this paper as [52]
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Peterson, B. S., West, A. E., Weisz, J. R., Mack, W. J., Kipke, M. D., Findling, R. L., Mittman, B. S., Bansal, R., Piantadosi, S., Takata, G., et al. (2021) · BMC Psychiatry · doi:10.1186/s12888-021-03314-y · Cited by 13 · see citing works
- Authors
- Ogbagaber, S. B., Karp, J., & Wahed, A. S.
- Venue
- Statistics in Medicine
- Publisher
- Wiley
- Published
- 2015-09-27
- Volume
- 35
- Issue
- 6
- Pages
- 840-858
- ISSN
- 0277-6715
- 1097-0258
- PMC
- PMC5150988
- Cited by
- 26
- References
- 2
- Language
- en
- Licence
- Funders
- National Institute of Mental Health (P30 MH090333)
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