A marginalized two-part joint model for a longitudinal biomarker and a terminal event with application to advanced head and neck cancers
dc.rights.license | open | en_US |
hal.structure.identifier | Bordeaux population health [BPH] | |
dc.contributor.author | RUSTAND, Denis | |
dc.contributor.author | BRIOLLAIS, Laurent | |
hal.structure.identifier | Bordeaux population health [BPH] | |
dc.contributor.author | RONDEAU, Virginie
ORCID: 0000-0001-7109-4831 IDREF: 16662988X | |
dc.date.accessioned | 2023-11-06T16:29:58Z | |
dc.date.available | 2023-11-06T16:29:58Z | |
dc.date.issued | 2023-09-17 | |
dc.identifier.issn | 1539-1612 | en_US |
dc.identifier.uri | https://oskar-bordeaux.fr/handle/20.500.12278/184636 | |
dc.description.abstractEn | The 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.iso | EN | en_US |
dc.rights | Attribution 3.0 United States | * |
dc.rights.uri | http://creativecommons.org/licenses/by/3.0/us/ | * |
dc.subject.en | Conditional two-part | |
dc.subject.en | Joint model | |
dc.subject.en | Left-censoring | |
dc.subject.en | Marginalized two-part | |
dc.subject.en | Randomized clinical trial | |
dc.subject.en | Semicontinuous | |
dc.subject.en | Solid tumors | |
dc.title.en | A marginalized two-part joint model for a longitudinal biomarker and a terminal event with application to advanced head and neck cancers | |
dc.title.alternative | Pharm Stat | en_US |
dc.type | Article de revue | en_US |
dc.identifier.doi | 10.1002/pst.2338 | en_US |
dc.subject.hal | Sciences du Vivant [q-bio]/Santé publique et épidémiologie | en_US |
dc.identifier.pubmed | 37717945 | en_US |
bordeaux.journal | Pharmaceutical Statistics | en_US |
bordeaux.hal.laboratories | Bordeaux Population Health Research Center (BPH) - UMR 1219 | en_US |
bordeaux.institution | Université de Bordeaux | en_US |
bordeaux.institution | INSERM | en_US |
bordeaux.team | BIOSTAT | en_US |
bordeaux.peerReviewed | oui | en_US |
bordeaux.inpress | non | en_US |
bordeaux.identifier.funderID | Canadian Institutes of Health Research | en_US |
bordeaux.identifier.funderID | Canadian Statistical Sciences Institute | en_US |
bordeaux.identifier.funderID | École des Hautes Études en Santé Publique | en_US |
bordeaux.identifier.funderID | Institut National Du Cancer | en_US |
bordeaux.identifier.funderID | Natural Sciences and Engineering Research Council of Canada | en_US |
bordeaux.import.source | pubmed | |
hal.identifier | hal-04272638 | |
hal.version | 1 | |
hal.date.transferred | 2023-11-06T16:30:00Z | |
hal.popular | non | en_US |
hal.audience | Internationale | en_US |
hal.export | true | |
workflow.import.source | pubmed | |
dc.rights.cc | Pas de Licence CC | en_US |
bordeaux.COinS | ctx_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 |