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Estimation of Survival Distributions of Treatment Policies in Two-Stage Randomization Designs in Clinical Trials

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Some clinical trials follow a design where patients are randomized to a primary therapy at entry followed by another randomization to maintenance therapy contingent upon disease remission. Ideally, analysis would allow different treatment policies, i.e., combinations of primary and maintenance therapy if specified up-front, to be compared. Standard practice is to conduct separate analyses for the primary and follow-up treatments, which does not address this issue directly. We propose consistent estimators for the survival distribution and mean restricted survival time for each treatment policy in such two-stage studies and derive large-sample properties. The methods are demonstrated on a leukemia clinical trial data set and through simulation.

Keywords: Induction therapy; Intent to treat; Inverse weighting; Maintenance therapy; Potential outcomes; Survival analysis

Document Type: Research Article


Affiliations: 1: Clinical Biostatistics, Merck Research Laboratories, P.O. Box 2000, Rahway, New Jersey 07065-0900, U.S.A. 2: Department of Statistics, North Carolina State University, Raleigh, North Carolina 27695-8203, U.S.A.

Publication date: March 1, 2002


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