The use of a binary composite endpoint and sample size requirement: influence of endpoints overlap

Although composite endpoints (CE) are common in clinical trials, the impact of the relationship between the components of a binary CE on the sample size requirement (SSR) has not been addressed. We performed a computational study considering 2 treatments and a CE with 2 components: the relevant endp...

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Autores: Ramon Marsal, Josep, Ferreira González, Ignacio, Bertrán, Sandra, Ribera, Aida, Permanyer-Miralda, Gaietà, Garcia Dorado, Antonio David, Gómez Melis, Guadalupe|||0000-0003-4252-4884
Formato: artículo
Fecha de publicación:2017
País:España
Recursos:Universitat Politècnica de Catalunya (UPC)
Repositorio:UPCommons. Portal del coneixement obert de la UPC
Idioma:inglés
OAI Identifier:oai:upcommons.upc.edu:2117/110928
Acesso em linha:https://hdl.handle.net/2117/110928
https://dx.doi.org/10.1093/aje/kww105
Access Level:acceso abierto
Palavra-chave:association measures
binary endpoints
composite endpoint
correlated endpoints
outcome assessment
sample size
Classificació AMS::90 Operations research, mathematical programming
Àrees temàtiques de la UPC::Matemàtiques i estadística::Investigació operativa
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spelling The use of a binary composite endpoint and sample size requirement: influence of endpoints overlapRamon Marsal, JosepFerreira González, IgnacioBertrán, SandraRibera, AidaPermanyer-Miralda, GaietàGarcia Dorado, Antonio DavidGómez Melis, Guadalupe|||0000-0003-4252-4884association measuresbinary endpointscomposite endpointcorrelated endpointsoutcome assessmentsample sizeClassificació AMS::90 Operations research, mathematical programmingÀrees temàtiques de la UPC::Matemàtiques i estadística::Investigació operativaAlthough composite endpoints (CE) are common in clinical trials, the impact of the relationship between the components of a binary CE on the sample size requirement (SSR) has not been addressed. We performed a computational study considering 2 treatments and a CE with 2 components: the relevant endpoint (RE) and the additional endpoint (AE). We assessed the strength of the components’ interrelation by the degree of relative overlap between them, which was stratified into 5 groups. Within each stratum, SSR was computed for multiple scenarios by varying the events proportion and the effect of the therapy. A lower SSR using CE was defined as the best scenario for using the CE. In 25 of 66 scenarios the degree of relative overlap determined the benefit of using CE instead of the RE. Adding an AE with greater effect than the RE leads to lower SSR using the CE regardless of the AE proportion and the relative overlap. The influence of overlapping decreases when the effect on RE increases. Adding an AE with lower effect than the RE constitutes the most uncertain situation. In summary, the interrelationship between CE components, assessed by the relative overlap, can help to define the SSR in specific situations and it should be considered for SSR computation.Peer Reviewed20172017-04-1020172017-11-20journal articlehttp://purl.org/coar/resource_type/c_6501VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttps://hdl.handle.net/2117/110928https://dx.doi.org/10.1093/aje/kww105reponame:UPCommons. Portal del coneixement obert de la UPCinstname:Universitat Politècnica de Catalunya (UPC)Inglésengopen accesshttp://purl.org/coar/access_right/c_abf2Attribution-NonCommercial-NoDerivs 3.0 Spainhttp://creativecommons.org/licenses/by-nc-nd/3.0/es/info:eu-repo/semantics/openAccessoai:upcommons.upc.edu:2117/1109282026-05-27T15:37:01Z
dc.title.none.fl_str_mv The use of a binary composite endpoint and sample size requirement: influence of endpoints overlap
title The use of a binary composite endpoint and sample size requirement: influence of endpoints overlap
spellingShingle The use of a binary composite endpoint and sample size requirement: influence of endpoints overlap
Ramon Marsal, Josep
association measures
binary endpoints
composite endpoint
correlated endpoints
outcome assessment
sample size
Classificació AMS::90 Operations research, mathematical programming
Àrees temàtiques de la UPC::Matemàtiques i estadística::Investigació operativa
title_short The use of a binary composite endpoint and sample size requirement: influence of endpoints overlap
title_full The use of a binary composite endpoint and sample size requirement: influence of endpoints overlap
title_fullStr The use of a binary composite endpoint and sample size requirement: influence of endpoints overlap
title_full_unstemmed The use of a binary composite endpoint and sample size requirement: influence of endpoints overlap
title_sort The use of a binary composite endpoint and sample size requirement: influence of endpoints overlap
dc.creator.none.fl_str_mv Ramon Marsal, Josep
Ferreira González, Ignacio
Bertrán, Sandra
Ribera, Aida
Permanyer-Miralda, Gaietà
Garcia Dorado, Antonio David
Gómez Melis, Guadalupe|||0000-0003-4252-4884
author Ramon Marsal, Josep
author_facet Ramon Marsal, Josep
Ferreira González, Ignacio
Bertrán, Sandra
Ribera, Aida
Permanyer-Miralda, Gaietà
Garcia Dorado, Antonio David
Gómez Melis, Guadalupe|||0000-0003-4252-4884
author_role author
author2 Ferreira González, Ignacio
Bertrán, Sandra
Ribera, Aida
Permanyer-Miralda, Gaietà
Garcia Dorado, Antonio David
Gómez Melis, Guadalupe|||0000-0003-4252-4884
author2_role author
author
author
author
author
author
dc.subject.none.fl_str_mv association measures
binary endpoints
composite endpoint
correlated endpoints
outcome assessment
sample size
Classificació AMS::90 Operations research, mathematical programming
Àrees temàtiques de la UPC::Matemàtiques i estadística::Investigació operativa
topic association measures
binary endpoints
composite endpoint
correlated endpoints
outcome assessment
sample size
Classificació AMS::90 Operations research, mathematical programming
Àrees temàtiques de la UPC::Matemàtiques i estadística::Investigació operativa
description Although composite endpoints (CE) are common in clinical trials, the impact of the relationship between the components of a binary CE on the sample size requirement (SSR) has not been addressed. We performed a computational study considering 2 treatments and a CE with 2 components: the relevant endpoint (RE) and the additional endpoint (AE). We assessed the strength of the components’ interrelation by the degree of relative overlap between them, which was stratified into 5 groups. Within each stratum, SSR was computed for multiple scenarios by varying the events proportion and the effect of the therapy. A lower SSR using CE was defined as the best scenario for using the CE. In 25 of 66 scenarios the degree of relative overlap determined the benefit of using CE instead of the RE. Adding an AE with greater effect than the RE leads to lower SSR using the CE regardless of the AE proportion and the relative overlap. The influence of overlapping decreases when the effect on RE increases. Adding an AE with lower effect than the RE constitutes the most uncertain situation. In summary, the interrelationship between CE components, assessed by the relative overlap, can help to define the SSR in specific situations and it should be considered for SSR computation.
publishDate 2017
dc.date.none.fl_str_mv 2017
2017-04-10
2017
2017-11-20
dc.type.none.fl_str_mv journal article
http://purl.org/coar/resource_type/c_6501
VoR
http://purl.org/coar/version/c_970fb48d4fbd8a85
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://hdl.handle.net/2117/110928
https://dx.doi.org/10.1093/aje/kww105
url https://hdl.handle.net/2117/110928
https://dx.doi.org/10.1093/aje/kww105
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
Attribution-NonCommercial-NoDerivs 3.0 Spain
http://creativecommons.org/licenses/by-nc-nd/3.0/es/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
Attribution-NonCommercial-NoDerivs 3.0 Spain
http://creativecommons.org/licenses/by-nc-nd/3.0/es/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:UPCommons. Portal del coneixement obert de la UPC
instname:Universitat Politècnica de Catalunya (UPC)
instname_str Universitat Politècnica de Catalunya (UPC)
reponame_str UPCommons. Portal del coneixement obert de la UPC
collection UPCommons. Portal del coneixement obert de la UPC
repository.name.fl_str_mv
repository.mail.fl_str_mv
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