Continuous toxicity monitoring in phase II trials in oncology

Biometrics. 2005 Jun;61(2):540-5. doi: 10.1111/j.1541-0420.2005.00311.x.

Abstract

The goal of a phase II trial in oncology is to evaluate the efficacy of a new therapy. The dose investigated in a phase II trial is usually an estimate of a maximum-tolerated dose obtained in a preceding phase I trial. Because this estimate is imprecise, stopping rules for toxicity are used in many phase II trials. We give recommendations on how to construct stopping rules to monitor toxicity continuously. A table is provided from which Pocock stopping boundaries can be easily obtained for a range of toxicity rates and sample sizes. Estimation of the probability of toxicity and response is also discussed.

MeSH terms

  • Bayes Theorem
  • Bias
  • Biometry / methods*
  • Clinical Trials as Topic
  • Clinical Trials, Phase I as Topic / methods
  • Clinical Trials, Phase II as Topic / methods*
  • Data Interpretation, Statistical*
  • Decision Theory
  • Dose-Response Relationship, Drug
  • Humans
  • Likelihood Functions
  • Maximum Tolerated Dose
  • Medical Oncology / methods*
  • Models, Statistical
  • Probability
  • Sample Size
  • Time Factors
  • Treatment Outcome