Bayesian borrowing for basket trials with longitudinal outcomes.
Stat Med
; 42(16): 2819-2840, 2023 07 20.
Article
en En
| MEDLINE
| ID: mdl-37120858
ABSTRACT
Basket trials are a novel clinical trial design in which a single intervention is investigated in multiple patient subgroups, or "baskets." They offer the opportunity to share information between subgroups, potentially increasing power to detect treatment effects. Basket trials offer several advantages over running a series of separate trials, including reduced sample sizes, increased efficiency, and reduced costs. Primarily, basket trials have been undertaken in Phase II oncology settings, but could be a promising design in other areas where a shared underlying biological mechanism drives different diseases. One such area is chronic aging-related diseases. However, trials in this area frequently have longitudinal outcomes, and therefore suitable methods are needed to share information in this setting. In this paper, we extend three Bayesian borrowing methods for a basket design with continuous longitudinal endpoints. We demonstrate our methods on a real-world dataset and in a simulation study where the aim is to detect positive basketwise treatment effects. Methods are compared with standalone analysis of each basket without borrowing. Our results confirm that methods that share information can improve power to detect positive treatment effects and increase precision over independent analysis in many scenarios. In highly heterogeneous scenarios, there is a trade-off between increased power and increased risk of type I errors. Our proposed methods for basket trials with continuous longitudinal outcomes aim to facilitate their applicability in the area of aging related diseases. Choice of method should be made based on trial priorities and the expected basketwise distribution of treatment effects.
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1
Base de datos:
MEDLINE
Asunto principal:
Proyectos de Investigación
/
Oncología Médica
Tipo de estudio:
Prognostic_studies
/
Risk_factors_studies
Idioma:
En
Revista:
Stat Med
Año:
2023
Tipo del documento:
Article