BOIN-ETC: A Bayesian optimal interval design considering efficacy and toxicity to identify the optimal dose combinations.
Stat Methods Med Res
; 33(4): 716-727, 2024 Apr.
Article
en En
| MEDLINE
| ID: mdl-38444354
ABSTRACT
One of the primary objectives of a dose-finding trial for novel anti-cancer agent combination therapies, such as molecular targeted agents and immune-oncology therapies, is to identify optimal dose combinations that are tolerable and therapeutically beneficial for subjects in subsequent clinical trials. The goal differs from that of a dose-finding trial for traditional cytotoxic agents, in which the goal is to determine the maximum tolerated dose combinations. This paper proposes the new design, named 'BOIN-ETC' design, to identify optimal dose combinations based on both efficacy and toxicity outcomes using the waterfall approach. The BOIN-ETC design is model-assisted, so it is expected to be robust, and straightforward to implement in actual oncology dose-finding trials. These characteristics are quite valuable from a practical perspective. Simulation studies show that the BOIN-ETC design has advantages compared with the other approaches in the percentage of correct optimal dose combination selection and the average number of patients allocated to the optimal dose combinations across various realistic settings.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Neoplasias
/
Antineoplásicos
Límite:
Humans
Idioma:
En
Revista:
Stat Methods Med Res
Año:
2024
Tipo del documento:
Article
País de afiliación:
Japón
Pais de publicación:
Reino Unido