Incorporating a Patient Dichotomous Characteristic in Cancer Phase I Clinical Trials Using Escalation with Overdose Control

We describe a design for cancer phase I clinical trials that takes into account patients heterogeneity thought to be related to treatment susceptibility. The goal is to estimate the maximum tolerated dose (MTD) given patient’s specific dichotomous covariate value. The design is Bayesian adaptive and...

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Main Authors: Mourad Tighiouart, Galen Cook-Wiens, André Rogatko
Format: Article
Language:English
Published: Wiley 2012-01-01
Series:Journal of Probability and Statistics
Online Access:http://dx.doi.org/10.1155/2012/567819
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author Mourad Tighiouart
Galen Cook-Wiens
André Rogatko
author_facet Mourad Tighiouart
Galen Cook-Wiens
André Rogatko
author_sort Mourad Tighiouart
collection DOAJ
description We describe a design for cancer phase I clinical trials that takes into account patients heterogeneity thought to be related to treatment susceptibility. The goal is to estimate the maximum tolerated dose (MTD) given patient’s specific dichotomous covariate value. The design is Bayesian adaptive and is an extension of escalation with overdose control (EWOC). We will assess the performance of this method by comparing the following designs via extensive simulations: (1) design using a covariate; patients are accrued to the trial sequentially and the dose given to a patient depends on his/her baseline covariate value, (2) design ignoring the covariate; patients are accrued to the trial sequentially and the dose given to a patient does not depend on his/her baseline covariate value, and (3) design using separate trials; in each group, patients are accrued to the trial sequentially and EWOC is implemented in each group. These designs are compared with respect to safety of the trial and efficiency of the estimates of the MTDs via extensive simulations. We found that ignoring a significant baseline binary covariate in the model results in a substantial number of patients being overdosed. On the other hand, accounting for a nonsignificant covariate in the model has practically no effect on the safety of the trial and efficiency of the estimates of the MTDs.
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spelling doaj-art-e2e20428e56d489c9d7974bec74febd02025-08-20T02:21:14ZengWileyJournal of Probability and Statistics1687-952X1687-95382012-01-01201210.1155/2012/567819567819Incorporating a Patient Dichotomous Characteristic in Cancer Phase I Clinical Trials Using Escalation with Overdose ControlMourad Tighiouart0Galen Cook-Wiens1André Rogatko2Biostatistics and Bioinformatics Research Center, Samuel Oschin Comprehensive Cancer Institute, 8700 Beverly Boulevard, PACT, Suite 900C, Los Angeles, CA 90048, USABiostatistics and Bioinformatics Research Center, Samuel Oschin Comprehensive Cancer Institute, 8700 Beverly Boulevard, PACT, Suite 900C, Los Angeles, CA 90048, USABiostatistics and Bioinformatics Research Center, Samuel Oschin Comprehensive Cancer Institute, 8700 Beverly Boulevard, PACT, Suite 900C, Los Angeles, CA 90048, USAWe describe a design for cancer phase I clinical trials that takes into account patients heterogeneity thought to be related to treatment susceptibility. The goal is to estimate the maximum tolerated dose (MTD) given patient’s specific dichotomous covariate value. The design is Bayesian adaptive and is an extension of escalation with overdose control (EWOC). We will assess the performance of this method by comparing the following designs via extensive simulations: (1) design using a covariate; patients are accrued to the trial sequentially and the dose given to a patient depends on his/her baseline covariate value, (2) design ignoring the covariate; patients are accrued to the trial sequentially and the dose given to a patient does not depend on his/her baseline covariate value, and (3) design using separate trials; in each group, patients are accrued to the trial sequentially and EWOC is implemented in each group. These designs are compared with respect to safety of the trial and efficiency of the estimates of the MTDs via extensive simulations. We found that ignoring a significant baseline binary covariate in the model results in a substantial number of patients being overdosed. On the other hand, accounting for a nonsignificant covariate in the model has practically no effect on the safety of the trial and efficiency of the estimates of the MTDs.http://dx.doi.org/10.1155/2012/567819
spellingShingle Mourad Tighiouart
Galen Cook-Wiens
André Rogatko
Incorporating a Patient Dichotomous Characteristic in Cancer Phase I Clinical Trials Using Escalation with Overdose Control
Journal of Probability and Statistics
title Incorporating a Patient Dichotomous Characteristic in Cancer Phase I Clinical Trials Using Escalation with Overdose Control
title_full Incorporating a Patient Dichotomous Characteristic in Cancer Phase I Clinical Trials Using Escalation with Overdose Control
title_fullStr Incorporating a Patient Dichotomous Characteristic in Cancer Phase I Clinical Trials Using Escalation with Overdose Control
title_full_unstemmed Incorporating a Patient Dichotomous Characteristic in Cancer Phase I Clinical Trials Using Escalation with Overdose Control
title_short Incorporating a Patient Dichotomous Characteristic in Cancer Phase I Clinical Trials Using Escalation with Overdose Control
title_sort incorporating a patient dichotomous characteristic in cancer phase i clinical trials using escalation with overdose control
url http://dx.doi.org/10.1155/2012/567819
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