Adaptive group sequential design for phase II clinical trials: a Bayesian decision theoretic approach

Yiyi Chen; Brian J Smith

doi:10.1002/sim.3711

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Adaptive group sequential design for phase II clinical trials: a Bayesian decision theoretic approach

Journal article

Peer reviewed

Adaptive group sequential design for phase II clinical trials: a Bayesian decision theoretic approach

Yiyi Chen and Brian J Smith

Statistics in medicine, Vol.28(27), pp.3347-3362

11/30/2009

DOI: 10.1002/sim.3711

PMID: 19725024

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Abstract

Bayesian decision theoretic approaches (BDTAs) have been widely studied in the literature as tools for designing and conducting phase II clinical trials. However, full Bayesian approaches that consider multiple endpoints are lacking. Since the monitoring of toxicity is a major goal of phase II trials, we propose an adaptive group sequential design using a BDTA, which characterizes efficacy and toxicity as correlated bivariate binary endpoints. We allow trade-off between the two endpoints. Interim evaluations are conducted group sequentially, but the number of interim looks and the size of each group are chosen adaptively based on current observations.We utilize a loss function consisting of two components: the loss associated with accruing, treating, and monitoring patients, and the loss associated with making incorrect decisions. The performance of our Bayesian modeling, and the operating characteristics of decision rules under a wide range of loss function parameters are evaluated using seven scenarios in a simulation study.Our method is illustrated in the context of a single-arm phase II trial of bevacizumab, gemcitabine, and oxaliplatin in patients with metastatic pancreatic adenocarcinoma.

Humans

Antibodies, Monoclonal - adverse effects

Antibodies, Monoclonal - therapeutic use

Models, Statistical

Adenocarcinoma - drug therapy

Bevacizumab

Pancreatic Neoplasms - drug therapy

Antibodies, Monoclonal, Humanized

Deoxycytidine - therapeutic use

Adenocarcinoma - therapy

Models, Biological

Bayes Theorem

Deoxycytidine - adverse effects

Clinical Trials, Phase II as Topic - methods

Organoplatinum Compounds - therapeutic use

Research Design

Deoxycytidine - analogs & derivatives

Organoplatinum Compounds - adverse effects

Details

Title: Subtitle: Adaptive group sequential design for phase II clinical trials: a Bayesian decision theoretic approach
Creators: Yiyi Chen - Division of Biostatistics, Department of Public Health and Preventive Medicine, Oregon Health and Science University, Portland, OR 97239, USA. chenyiy@ohsu.edu
Brian J Smith
Resource Type: Journal article
Publication Details: Statistics in medicine, Vol.28(27), pp.3347-3362
Publisher: England
DOI: 10.1002/sim.3711
PMID: 19725024
ISSN: 0277-6715
eISSN: 1097-0258
Language: English
Date published: 11/30/2009
Academic Unit: Biostatistics; Holden Comprehensive Cancer Center
Record Identifier: 9983997356702771

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