Generalized pairwise comparison methods to analyze (non)prioritized composite endpoints.


Journal

Statistics in medicine
ISSN: 1097-0258
Titre abrégé: Stat Med
Pays: England
ID NLM: 8215016

Informations de publication

Date de publication:
30 12 2019
Historique:
received: 04 07 2018
revised: 02 09 2019
accepted: 13 09 2019
pubmed: 30 10 2019
medline: 1 12 2020
entrez: 30 10 2019
Statut: ppublish

Résumé

In the analysis of composite endpoints in a clinical trial, time to first event analysis techniques such as the logrank test and Cox proportional hazard test do not take into account the multiplicity, importance, and the severity of events in the composite endpoint. Several generalized pairwise comparison analysis methods have been described recently that do allow to take these aspects into account. These methods have the additional benefit that all types of outcomes can be included, such as longitudinal quantitative outcomes, to evaluate the full treatment effect. Four of the generalized pairwise comparison methods, ie, the Finkelstein-Schoenfeld, the Buyse, unmatched Pocock, and adapted O'Brien test, are summarized. They are compared to each other and to the logrank test by means of simulations while specifically evaluating the effect of correlation between components of the composite endpoint on the power to detect a treatment difference. These simulations show that prioritized generalized pairwise comparison methods perform very similarly, are sensitive to the priority rank of the components in the composite endpoint, and do not measure the true treatment effect from the second priority-ranked component onward. The nonprioritized pairwise comparison test does not suffer from these limitations and correlation affects only its variance.

Identifiants

pubmed: 31659790
doi: 10.1002/sim.8388
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

5641-5656

Informations de copyright

© 2019 John Wiley & Sons, Ltd.

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Auteurs

J Verbeeck (J)

I-BioStat, Universiteit Hasselt, Hasselt, Belgium.

E Spitzer (E)

Clinical Trial Management & Core Laboratories, Cardialysis, Rotterdam, The Netherlands.
Cardiology Thoraxcenter, Erasmus Medical Center, Rotterdam, The Netherlands.

T de Vries (T)

Clinical Trial Management & Core Laboratories, Cardialysis, Rotterdam, The Netherlands.

G A van Es (GA)

ECRI, Rotterdam, The Netherlands.

W N Anderson (WN)

Independent Consultant, Carpinteria, California.

N M Van Mieghem (NM)

Cardiology Thoraxcenter, Erasmus Medical Center, Rotterdam, The Netherlands.

M B Leon (MB)

Columbia University Medical Center, New York, New York.
Cardiovascular Research Foundation, New York, New York.

G Molenberghs (G)

I-BioStat, Universiteit Hasselt, Hasselt, Belgium.
I-BioStat, KU Leuven, Leuven, Belgium.

J Tijssen (J)

Clinical Trial Management & Core Laboratories, Cardialysis, Rotterdam, The Netherlands.
ECRI, Rotterdam, The Netherlands.

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