Traditional and new composite endpoints in heart failure clinical trials: Facilitating comprehensive efficacy assessments and improving trial efficiency

Stefan D. Anker, Stefan Schroeder, Dan Atar, Jeroen J. Bax, Claudio Ceconi, Martin R. Cowie, Adam Crisp, Fabienne Dominjon, Ian Ford, Hossein Ardeschir Ghofrani, Savion Gropper, Gerhard Hindricks, Mark A. Hlatky, Richard Holcomb, Narimon Honarpour, J. Wouter Jukema, Albert M. Kim, Michael Kunz, Martin Lefkowitz, Chantal Le FlochUlf Landmesser, Theresa A. McDonagh, John J. McMurray, Bela Merkely, Milton Packer, Krishna Prasad, James Revkin, Giuseppe M C Rosano, Ransi Somaratne, Wendy Gattis Stough, Adriaan A. Voors, Frank Ruschitzka

Research output: Contribution to journalReview article

42 Scopus citations

Abstract

Composite endpoints are commonly used as the primary measure of efficacy in heart failure clinical trials to assess the overall treatment effect and to increase the efficiency of trials. Clinical trials still must enrol large numbers of patients to accrue a sufficient number of outcome events and have adequate power to draw conclusions about the efficacy and safety of new treatments for heart failure. Additionally, the societal and health system perspectives on heart failure have raised interest in ascertaining the effects of therapy on outcomes such as repeat hospitalization and the patient's burden of disease. Thus, novel methods for using composite endpoints in clinical trials (e.g. clinical status composite endpoints, recurrent event analyses) are being applied in current and planned trials. Endpoints that measure functional status or reflect the patient experience are important but used cautiously because heart failure treatments may improve function yet have adverse effects on mortality. This paper discusses the use of traditional and new composite endpoints, identifies qualities of robust composites, and outlines opportunities for future research.

Original languageEnglish (US)
Pages (from-to)482-489
Number of pages8
JournalEuropean Journal of Heart Failure
Volume18
Issue number5
DOIs
StatePublished - May 1 2016

Keywords

  • Clinical trial
  • Endpoint determination
  • Heart failure
  • Surrogate endpoints

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

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    Anker, S. D., Schroeder, S., Atar, D., Bax, J. J., Ceconi, C., Cowie, M. R., Crisp, A., Dominjon, F., Ford, I., Ghofrani, H. A., Gropper, S., Hindricks, G., Hlatky, M. A., Holcomb, R., Honarpour, N., Jukema, J. W., Kim, A. M., Kunz, M., Lefkowitz, M., ... Ruschitzka, F. (2016). Traditional and new composite endpoints in heart failure clinical trials: Facilitating comprehensive efficacy assessments and improving trial efficiency. European Journal of Heart Failure, 18(5), 482-489. https://doi.org/10.1002/ejhf.516