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dc.rights.licenseopenen_US
hal.structure.identifierBordeaux population health [BPH]
dc.contributor.authorRUSTAND, Denis
dc.contributor.authorBRIOLLAIS, Laurent
hal.structure.identifierBordeaux population health [BPH]
dc.contributor.authorRONDEAU, Virginie
ORCID: 0000-0001-7109-4831
IDREF: 16662988X
dc.date.accessioned2023-11-06T16:29:58Z
dc.date.available2023-11-06T16:29:58Z
dc.date.issued2023-09-17
dc.identifier.issn1539-1612en_US
dc.identifier.urihttps://oskar-bordeaux.fr/handle/20.500.12278/184636
dc.description.abstractEnThe sum of the longest diameter (SLD) of the target lesions is a longitudinal biomarker used to assess tumor response in cancer clinical trials, which can inform about early treatment effect. This biomarker is semicontinuous, often characterized by an excess of zeros and right skewness. Conditional two-part joint models were introduced to account for the excess of zeros in the longitudinal biomarker distribution and link it to a time-to-event outcome. A limitation of the conditional two-part model is that it only provides an effect of covariates, such as treatment, on the conditional mean of positive biomarker values, and not an overall effect on the biomarker, which is often of clinical relevance. As an alternative, we propose in this article, a marginalized two-part joint model (M-TPJM) for the repeated measurements of the SLD and a terminal event, where the covariates affect the overall mean of the biomarker. Our simulation studies assessed the good performance of the marginalized model in terms of estimation and coverage rates. Our application of the M-TPJM to a randomized clinical trial of advanced head and neck cancer shows that the combination of panitumumab in addition with chemotherapy increases the odds of observing a disappearance of all target lesions compared to chemotherapy alone, leading to a possible indirect effect of the combined treatment on time to death.
dc.language.isoENen_US
dc.rightsAttribution 3.0 United States*
dc.rights.urihttp://creativecommons.org/licenses/by/3.0/us/*
dc.subject.enConditional two-part
dc.subject.enJoint model
dc.subject.enLeft-censoring
dc.subject.enMarginalized two-part
dc.subject.enRandomized clinical trial
dc.subject.enSemicontinuous
dc.subject.enSolid tumors
dc.title.enA marginalized two-part joint model for a longitudinal biomarker and a terminal event with application to advanced head and neck cancers
dc.title.alternativePharm Staten_US
dc.typeArticle de revueen_US
dc.identifier.doi10.1002/pst.2338en_US
dc.subject.halSciences du Vivant [q-bio]/Santé publique et épidémiologieen_US
dc.identifier.pubmed37717945en_US
bordeaux.journalPharmaceutical Statisticsen_US
bordeaux.hal.laboratoriesBordeaux Population Health Research Center (BPH) - UMR 1219en_US
bordeaux.institutionUniversité de Bordeauxen_US
bordeaux.institutionINSERMen_US
bordeaux.teamBIOSTATen_US
bordeaux.peerReviewedouien_US
bordeaux.inpressnonen_US
bordeaux.identifier.funderIDCanadian Institutes of Health Researchen_US
bordeaux.identifier.funderIDCanadian Statistical Sciences Instituteen_US
bordeaux.identifier.funderIDÉcole des Hautes Études en Santé Publiqueen_US
bordeaux.identifier.funderIDInstitut National Du Canceren_US
bordeaux.identifier.funderIDNatural Sciences and Engineering Research Council of Canadaen_US
bordeaux.import.sourcepubmed
hal.identifierhal-04272638
hal.version1
hal.date.transferred2023-11-06T16:30:00Z
hal.popularnonen_US
hal.audienceInternationaleen_US
hal.exporttrue
workflow.import.sourcepubmed
dc.rights.ccPas de Licence CCen_US
bordeaux.COinSctx_ver=Z39.88-2004&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.jtitle=Pharmaceutical%20Statistics&rft.date=2023-09-17&rft.eissn=1539-1612&rft.issn=1539-1612&rft.au=RUSTAND,%20Denis&BRIOLLAIS,%20Laurent&RONDEAU,%20Virginie&rft.genre=article


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