Monitoring futility and efficacy in phase II trials with Bayesian posterior distributions-A calibration approach.


Journal

Biometrical journal. Biometrische Zeitschrift
ISSN: 1521-4036
Titre abrégé: Biom J
Pays: Germany
ID NLM: 7708048

Informations de publication

Date de publication:
05 2019
Historique:
received: 25 10 2017
revised: 06 07 2018
accepted: 07 07 2018
pubmed: 4 9 2018
medline: 6 2 2020
entrez: 4 9 2018
Statut: ppublish

Résumé

A multistage single arm phase II trial with binary endpoint is considered. Bayesian posterior probabilities are used to monitor futility in interim analyses and efficacy in the final analysis. For a beta-binomial model, decision rules based on Bayesian posterior probabilities are converted to "traditional" decision rules in terms of number of responders among patients observed so far. Analytical derivations are given for the probability of stopping for futility and for the probability to declare efficacy. A workflow is presented on how to select the parameters specifying the Bayesian design, and the operating characteristics of the design are investigated. It is outlined how the presented approach can be transferred to statistical models other than the beta-binomial model.

Identifiants

pubmed: 30175405
doi: 10.1002/bimj.201700209
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

488-502

Informations de copyright

© 2018 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim.

Auteurs

Annette Kopp-Schneider (A)

Division of Biostatistics, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Manuel Wiesenfarth (M)

Division of Biostatistics, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Ruth Witt (R)

Clinical Trial Center, National Center for Tumor Diseases, Heidelberg, Germany.
Hopp Children's Cancer Center at NCT Heidelberg (KiTZ), Department of Pediatric Oncology and Hematology and Clinical Cooperation Unit Pediatric Oncology, University Hospital and German Cancer Research Center (DKFZ), Heidelberg, Germany.

Dominic Edelmann (D)

Division of Biostatistics, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Olaf Witt (O)

Hopp Children's Cancer Center at NCT Heidelberg (KiTZ), Department of Pediatric Oncology and Hematology and Clinical Cooperation Unit Pediatric Oncology, University Hospital and German Cancer Research Center (DKFZ), Heidelberg, Germany.

Ulrich Abel (U)

Clinical Trial Center, National Center for Tumor Diseases, Heidelberg, Germany.

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Classifications MeSH