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dc.contributor.authorZeng, Leilei
dc.contributor.authorCook, Richard J.
dc.contributor.authorLee, Ker-Ai 17:23:03 (GMT) 17:23:03 (GMT)
dc.descriptionThis is the peer reviewed version of the following article: Leilei Zeng, Richard J. Cook and Ker-Ai Lee, Design of cancer trials based on progression-free survival with intermittent assessment. Statistics in Medicine (2018), 37(12): 1947–2066 which has been published in final form at
dc.description.abstractTherapeutic advances in cancer mean that it is now impractical to performed phase III randomized trials evaluating experimental treatments on the basis of overall survival. As a result, the composite endpoint of progression-free survival has been routinely adopted in recent years as it is viewed as enabling a more timely and cost-effective approach to assessing the clinical benefit of novel interventions. This article considers design of cancer trials directed at the evaluation of treatment effects on progression-free survival. In particular, we derive sample size criteria based on an illness-death model that considers cancer progression and death jointly while accounting for the fact that progression is assessed only intermittently. An alternative approach to design is also considered in which the sample size is derived based on a misspecified Cox model, which uses the documented time of progression as the progression time rather than dealing with the interval censoring. Simulation studies show the validity of the proposed methods.en
dc.description.sponsorshipThis research was supported by Discovery Grant from the Natural Sciences and Engineering Research Council of Canada to L. Zeng (RGPIN 115928) and R. J. Cook (RGPIN 155849) and the Canadian Institutes for Health Research to R. J. Cook (FRN 13887). R. J. Cook is a Canada Research Chair in Statistical Methods for Health Research.en
dc.relation.ispartofseriesStatistics in Medicine;37(12)
dc.subjectcancer trialsen
dc.subjectintermittent assessment of progressionen
dc.subjectmultistate modelen
dc.subjectprogression free survivalen
dc.subjectsample size estimationen
dc.titleDesign of cancer trials based on progression-free survival with intermittent assessmenten
uws.contributor.affiliation1Faculty of Mathematicsen
uws.contributor.affiliation2Statistics and Actuarial Scienceen

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