Patient-specific dose finding based on bivariate outcomes and covariates

Peter F. Thall, Hoang Q. Nguyen, Elihu H. Estey

Research output: Contribution to journalArticlepeer-review

54 Scopus citations

Abstract

A Bayesian sequential dose-finding procedure based on bivariate (efficacy, toxicity) outcomes that accounts for patient covariates and dose-covariate interactions is presented. Historical data are used to obtain an informative prior on covariate main effects, with uninformative priors assumed for all dose effect parameters. Elicited limits on the probabilities of efficacy and toxicity for each of a representative set of covariate vectors are used to construct bounding functions that determine the acceptability of each dose for each patient. Elicited outcome probability pairs that are equally desirable for a reference patient are used to define two different posterior criteria, either of which may be used to select an optimal covariate-specific dose for each patient. Because the dose selection criteria are covariate specific, different patients may receive different doses at the same point in the trial, and the set of eligible patients may change adaptively during the trial. The method is illustrated by a dose-finding trial in acute leukemia, including a simulation study.

Original languageEnglish (US)
Pages (from-to)1126-1136
Number of pages11
JournalBiometrics
Volume64
Issue number4
DOIs
StatePublished - Dec 2008

Keywords

  • Adaptive design
  • Bayesian design
  • Biologic agents
  • Individualized treatment
  • Phase I clinical trial
  • Phase I/II clinical trial

ASJC Scopus subject areas

  • Statistics and Probability
  • General Biochemistry, Genetics and Molecular Biology
  • General Immunology and Microbiology
  • General Agricultural and Biological Sciences
  • Applied Mathematics

MD Anderson CCSG core facilities

  • Biostatistics Resource Group

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