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Design and analysis considerations for comparing dynamic treatment regimens with binary outcomes from sequential multiple assignment randomized trials
| Content Provider | Scilit |
|---|---|
| Author | Kidwell, Kelley M. Seewald, Nicholas Tran, Qui Kasari, Connie Almirall, Daniel |
| Copyright Year | 2017 |
| Description | 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. |
| Related Links | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6290910/pdf |
| Ending Page | 1651 |
| Page Count | 24 |
| Starting Page | 1628 |
| ISSN | 02664763 |
| e-ISSN | 13600532 |
| DOI | 10.1080/02664763.2017.1386773 |
| Journal | Journal of Applied Statistics |
| Issue Number | 9 |
| Volume Number | 45 |
| Language | English |
| Publisher | Informa UK Limited |
| Publisher Date | 2018-07-04 |
| Access Restriction | Open |
| Subject Keyword | Journal: Journal of Applied Statistics Mathematical Psychology Adaptive Interventions Dynamic Treatment Regimes Sequential Multiple Assignment Randomized Trial Inverse-probability-of-treatment Weighting Sample Size |
| Content Type | Text |
| Resource Type | Article |
| Subject | Statistics and Probability Statistics, Probability and Uncertainty |
