Interim monitoring of sequential multiple assignment randomized trials using partial information.
Biometrics
; 79(4): 2881-2894, 2023 12.
Article
en En
| MEDLINE
| ID: mdl-36896962
ABSTRACT
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.
Palabras clave
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Proyectos de Investigación
/
Neoplasias de la Mama
Tipo de estudio:
Clinical_trials
Límite:
Female
/
Humans
Idioma:
En
Revista:
Biometrics
Año:
2023
Tipo del documento:
Article
País de afiliación:
Estados Unidos