title: Estimation of multiple response rates in phase II clinical trials with missing observations.
abstract: Responses are often correlated in clinical trials. A patient who is not evaluable for a response may still provide some information to the response through his or her status on other responses. Under the assumption of missing at random, we propose the utilization of a self-consistent estimator. We show that the proposed estimators are more efficient than the conventional estimators by asymptotic relative efficiency and simulation study. An example from a Phase II clinical trial on children with chronic myelogenous leukemia is provided.
authors.affiliation: Division of Biostatistics, Department of Epidemiology and Health Policy Research, College of Medicine, University of Florida, Gainesville, Florida 32608, USA. mchang@biostat.ufl.edu
authors.forename: Myron
authors.initials: M
authors.lastname: Chang
mesh_terms: D002648:Child; D017322:Clinical Trials, Phase II as Topic / Q000706:statistics & numerical data*; D003198:Computer Simulation; D003627:Data Interpretation, Statistical; D023381:Endpoint Determination; D006801:Humans; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000097:blood / Q000235:genetics / Q000628:therapy; D016013:Likelihood Functions; D015233:Models, Statistical; D011336:Probability; D016896:Treatment Outcome