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Testing for the presence of cured patients: a simulation study

Peng, Paul Yingwei; Dear, Keith; Carriere, K

Description

An important but difficult problem in clinical trials is to determine the presence of cured patients when long-term survivors are observed. The likelihood ratio test has been studied for this purpose in the gamma mixture model. However, its asymptotic null distribution is not readily available due to a violation of boundary conditions in the standard asymptotic theory. In this paper, a simulation study is employed to examine a proposed asymptotic null distribution of the likelihood ratio test....[Show more]

dc.contributor.authorPeng, Paul Yingwei
dc.contributor.authorDear, Keith
dc.contributor.authorCarriere, K
dc.date.accessioned2015-12-13T23:26:37Z
dc.date.available2015-12-13T23:26:37Z
dc.identifier.issn0277-6715
dc.identifier.urihttp://hdl.handle.net/1885/92917
dc.description.abstractAn important but difficult problem in clinical trials is to determine the presence of cured patients when long-term survivors are observed. The likelihood ratio test has been studied for this purpose in the gamma mixture model. However, its asymptotic null distribution is not readily available due to a violation of boundary conditions in the standard asymptotic theory. In this paper, a simulation study is employed to examine a proposed asymptotic null distribution of the likelihood ratio test. We find that the distribution can also be used to approximate the asymptotic null distribution of the likelihood ratio test in the Weibull and log-normal mixture models when the censoring rate is not too light. However, the simulation study also shows that null distribution of the likelihood ratio test deviates significantly from the suggested distribution under moderate sample sizes when the censoring rate is small or the hazard rate is large. Consequently caution is needed in this case to determine the presence of cured patients. Finally, the results are used to confirm the presence of cured patients in a leukaemia study.
dc.publisherJohn Wiley & Sons Inc
dc.sourceStatistics in Medicine
dc.subjectKeywords: acute lymphoblastic leukemia; allogenic bone marrow transplantation; article; autologous bone marrow transplantation; clinical article; clinical observation; clinical trial; controlled clinical trial; controlled study; human; long term care; simulation; s
dc.titleTesting for the presence of cured patients: a simulation study
dc.typeJournal article
local.description.notesImported from ARIES
local.description.refereedYes
local.identifier.citationvolume20
dc.date.issued2001
local.identifier.absfor111706 - Epidemiology
local.identifier.ariespublicationMigratedxPub26170
local.type.statusPublished Version
local.contributor.affiliationPeng, Paul Yingwei, Queen's University
local.contributor.affiliationDear, Keith, College of Medicine, Biology and Environment, ANU
local.contributor.affiliationCarriere, K, University of Alberta
local.bibliographicCitation.startpage1783
local.bibliographicCitation.lastpage1796
local.identifier.doi10.1002/sim.781
dc.date.updated2015-12-12T09:47:17Z
local.identifier.scopusID2-s2.0-0035973253
CollectionsANU Research Publications

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