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Interim monitoring of sequential multiple assignment randomized trials using partial information.

Publication ,  Journal Article
Manschot, C; Laber, E; Davidian, M
Published in: Biometrics
December 2023

The sequential multiple assignment randomized trial (SMART) is the gold standard trial design to generate data for the evaluation of multistage treatment regimes. As with conventional (single-stage) randomized clinical trials, interim monitoring allows early stopping; however, there are few methods for principled interim analysis in SMARTs. Because SMARTs involve multiple stages of treatment, a key challenge is that not all enrolled participants will have progressed through all treatment stages at the time of an interim analysis. Wu et al. (2021) propose basing interim analyses on an estimator for the mean outcome under a given regime that uses data only from participants who have completed all treatment stages. We propose an estimator for the mean outcome under a given regime that gains efficiency by using partial information from enrolled participants regardless of their progression through treatment stages. Using the asymptotic distribution of this estimator, we derive associated Pocock and O'Brien-Fleming testing procedures for early stopping. In simulation experiments, the estimator controls type I error and achieves nominal power while reducing expected sample size relative to the method of Wu et al. (2021). We present an illustrative application of the proposed estimator based on a recent SMART evaluating behavioral pain interventions for breast cancer patients.

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Published In

Biometrics

DOI

EISSN

1541-0420

Publication Date

December 2023

Volume

79

Issue

4

Start / End Page

2881 / 2894

Location

England

Related Subject Headings

  • Statistics & Probability
  • Sample Size
  • Research Design
  • Randomized Controlled Trials as Topic
  • Humans
  • Female
  • Computer Simulation
  • Breast Neoplasms
  • 4905 Statistics
  • 0199 Other Mathematical Sciences
 

Citation

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Manschot, C., Laber, E., & Davidian, M. (2023). Interim monitoring of sequential multiple assignment randomized trials using partial information. Biometrics, 79(4), 2881–2894. https://doi.org/10.1111/biom.13854
Manschot, Cole, Eric Laber, and Marie Davidian. “Interim monitoring of sequential multiple assignment randomized trials using partial information.Biometrics 79, no. 4 (December 2023): 2881–94. https://doi.org/10.1111/biom.13854.
Manschot C, Laber E, Davidian M. Interim monitoring of sequential multiple assignment randomized trials using partial information. Biometrics. 2023 Dec;79(4):2881–94.
Manschot, Cole, et al. “Interim monitoring of sequential multiple assignment randomized trials using partial information.Biometrics, vol. 79, no. 4, Dec. 2023, pp. 2881–94. Pubmed, doi:10.1111/biom.13854.
Manschot C, Laber E, Davidian M. Interim monitoring of sequential multiple assignment randomized trials using partial information. Biometrics. 2023 Dec;79(4):2881–2894.
Journal cover image

Published In

Biometrics

DOI

EISSN

1541-0420

Publication Date

December 2023

Volume

79

Issue

4

Start / End Page

2881 / 2894

Location

England

Related Subject Headings

  • Statistics & Probability
  • Sample Size
  • Research Design
  • Randomized Controlled Trials as Topic
  • Humans
  • Female
  • Computer Simulation
  • Breast Neoplasms
  • 4905 Statistics
  • 0199 Other Mathematical Sciences