Interim monitoring of sequential multiple assignment randomized trials using partial information.
augmented inverse probability weighting
clinical trials
double robustness
dynamic treatment regimes
early stopping
group sequential analysis
Journal
Biometrics
ISSN: 1541-0420
Titre abrégé: Biometrics
Pays: United States
ID NLM: 0370625
Informations de publication
Date de publication:
10 Mar 2023
10 Mar 2023
Historique:
received:
13
09
2022
accepted:
02
03
2023
pubmed:
11
3
2023
medline:
11
3
2023
entrez:
10
3
2023
Statut:
aheadofprint
Résumé
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.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2023 The Authors. Biometrics published by Wiley Periodicals LLC on behalf of International Biometric Society.
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