Simultaneous inference of a binary composite endpoint and its components

Publikation: Beitrag in FachzeitschriftArtikelForschungPeer-Review

Autoren

  • M. Große Ruse
  • C. Ritz
  • L. A. Hothorn

Organisationseinheiten

Externe Organisationen

  • Lund University
  • University of Copenhagen
Forschungs-netzwerk anzeigen

Details

OriginalspracheEnglisch
Seiten (von - bis)56-69
Seitenumfang14
FachzeitschriftJournal of Biopharmaceutical Statistics
Jahrgang27
Ausgabenummer1
Frühes Online-Datum3 Juni 2016
PublikationsstatusElektronisch veröffentlicht (E-Pub) - 3 Juni 2016

Abstract

Binary composite endpoints offer some advantages as a way to succinctly combine evidence from a number of related binary endpoints recorded in the same clinical trial into a single outcome. However, as some concerns about the clinical relevance as well as the interpretation of such composite endpoints have been raised, it is recommended to evaluate the composite endpoint jointly with the involved components. We propose an approach for carrying out simultaneous inference based on separate model fits for each endpoint, yet controlling the familywise type I error rate asymptotically. The key idea is to stack parameter estimates from the different fits and derive their joint asymptotic distribution. Simulations show that the proposed approach comes closer to nominal levels and has comparable or higher power as compared to existing approaches, even for moderate sample sizes (around 100-200 observations). The method is compared to the gatekeeping approach and results are provided in the Supplementary Material. In two data examples we show how the procedure may be adapted to handle local significance levels specified through a priori given weights.

ASJC Scopus Sachgebiete

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Simultaneous inference of a binary composite endpoint and its components. / Große Ruse, M.; Ritz, C.; Hothorn, L. A.
in: Journal of Biopharmaceutical Statistics, Jahrgang 27, Nr. 1, 03.06.2016, S. 56-69.

Publikation: Beitrag in FachzeitschriftArtikelForschungPeer-Review

Große Ruse M, Ritz C, Hothorn LA. Simultaneous inference of a binary composite endpoint and its components. Journal of Biopharmaceutical Statistics. 2016 Jun 3;27(1):56-69. Epub 2016 Jun 3. doi: https://doi.org/10.6084/m9.figshare.3409921.v1, 10.1080/10543406.2016.1148704
Große Ruse, M. ; Ritz, C. ; Hothorn, L. A. / Simultaneous inference of a binary composite endpoint and its components. in: Journal of Biopharmaceutical Statistics. 2016 ; Jahrgang 27, Nr. 1. S. 56-69.
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