incorporating a patient dichotomous characteristic in cancer phase i clinical trials using escalation with overdose control

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ID: 194432
2012
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Abstract
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.
Reference Key
tighiouart2012journalincorporating Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors ;Mourad Tighiouart;Galen Cook-Wiens;André Rogatko
Journal nature protocols
Year 2012
DOI
10.1155/2012/567819
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