Continuous toxicity monitoring in phase II trials in oncology.
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.
Ivanova
Anastasia
A
Department of Biostatistics, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599-7420, USA. aivanova@bios.unc.edu
Qaqish
Bahjat F
BF
Schell
Michael J
MJ
D001499:Bayes Theorem; D015982:Bias; D001699:Biometry / Q000379:methods*; D002986:Clinical Trials as Topic; D017321:Clinical Trials, Phase I as Topic / Q000379:methods; D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D003627:Data Interpretation, Statistical; D003662:Decision Theory; D004305:Dose-Response Relationship, Drug; D006801:Humans; D016013:Likelihood Functions; D020714:Maximum Tolerated Dose; D008495:Medical Oncology / Q000379:methods*; D015233:Models, Statistical; D011336:Probability; D018401:Sample Size; D013997:Time Factors; D016896:Treatment Outcome