Clinical outcome endpoints in heart failure trials

A European Society of Cardiology Heart Failure Association consensus document

Faiez Zannad, Kenneth Stein, Angeles Alonso Garcia, Stefan D. Anker, Paul W. Armstrong, Gonzalo Calvo, John G F Cleland, Jay N. Cohn, Kenneth Dickstein, Michael J. Domanski, Inger Ekman, Gerasimos S. Filippatos, Mihai Gheorghiade, Adrian F. Hernandez, Tiny Jaarsma, Joerg Koglin, Marvin Konstam, Stuart Kupfer, Aldo P. Maggioni, Alexandre Mebazaa & 20 others Marco Metra, Christina Nowack, Burkert Pieske, Ileana L. Pina, Stuart J. Pocock, Piotr Ponikowski, Giuseppe Rosano, Luis M. Ruilope, Frank Ruschitzka, Thomas Severin, Scott Solomon, Norman L. Stockbridge, Wendy Gattis Stough, Karl Swedberg, Luigi Tavazzi, Adriaan A. Voors, Scott M. Wasserman, Holger Woehrle, Andrew Zalewski, John J V McMurray

Research output: Contribution to journalArticle

106 Citations (Scopus)

Abstract

Endpoint selection is a critically important step in clinical trial design. It poses major challenges for investigators, regulators, and study sponsors, and it also has important clinical and practical implications for physicians and patients. Clinical outcomes of interest in heart failure trials include all-cause mortality, cause-specific mortality, relevant non-fatal morbidity (e.g. all-cause and cause-specific hospitalization), composites capturing both morbidity and mortality, safety, symptoms, functional capacity, and patient-reported outcomes. Each of these endpoints has strengths and weaknesses that create controversies regarding which is most appropriate in terms of clinical importance, sensitivity, reliability, and consistency. Not surprisingly, a lack of consensus exists within the scientific community regarding the optimal endpoint(s) for both acute and chronic heart failure trials. In an effort to address these issues, the Heart Failure Association of the European Society of Cardiology (HFA-ESC) convened a group of expert heart failure clinical investigators, biostatisticians, regulators, and pharmaceutical industry scientists (Nice, France, 12-13 February 2012) to evaluate the challenges of defining heart failure endpoints in clinical trials and to develop a consensus framework. This report summarizes the group's recommendations for achieving common views on heart failure endpoints in clinical trials.

Original languageEnglish (US)
Pages (from-to)1082-1094
Number of pages13
JournalEuropean Journal of Heart Failure
Volume15
Issue number10
DOIs
StatePublished - Oct 2013

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Heart Failure
Clinical Trials
Mortality
Research Personnel
Morbidity
Drug Industry
France
Hospitalization
Physicians
Safety

Keywords

  • Clinical trials
  • Heart failure
  • Morbidity
  • Mortality

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

Cite this

Zannad, F., Stein, K., Garcia, A. A., Anker, S. D., Armstrong, P. W., Calvo, G., ... McMurray, J. J. V. (2013). Clinical outcome endpoints in heart failure trials: A European Society of Cardiology Heart Failure Association consensus document. European Journal of Heart Failure, 15(10), 1082-1094. https://doi.org/10.1093/eurjhf/hft095

Clinical outcome endpoints in heart failure trials : A European Society of Cardiology Heart Failure Association consensus document. / Zannad, Faiez; Stein, Kenneth; Garcia, Angeles Alonso; Anker, Stefan D.; Armstrong, Paul W.; Calvo, Gonzalo; Cleland, John G F; Cohn, Jay N.; Dickstein, Kenneth; Domanski, Michael J.; Ekman, Inger; Filippatos, Gerasimos S.; Gheorghiade, Mihai; Hernandez, Adrian F.; Jaarsma, Tiny; Koglin, Joerg; Konstam, Marvin; Kupfer, Stuart; Maggioni, Aldo P.; Mebazaa, Alexandre; Metra, Marco; Nowack, Christina; Pieske, Burkert; Pina, Ileana L.; Pocock, Stuart J.; Ponikowski, Piotr; Rosano, Giuseppe; Ruilope, Luis M.; Ruschitzka, Frank; Severin, Thomas; Solomon, Scott; Stockbridge, Norman L.; Stough, Wendy Gattis; Swedberg, Karl; Tavazzi, Luigi; Voors, Adriaan A.; Wasserman, Scott M.; Woehrle, Holger; Zalewski, Andrew; McMurray, John J V.

In: European Journal of Heart Failure, Vol. 15, No. 10, 10.2013, p. 1082-1094.

Research output: Contribution to journalArticle

Zannad, F, Stein, K, Garcia, AA, Anker, SD, Armstrong, PW, Calvo, G, Cleland, JGF, Cohn, JN, Dickstein, K, Domanski, MJ, Ekman, I, Filippatos, GS, Gheorghiade, M, Hernandez, AF, Jaarsma, T, Koglin, J, Konstam, M, Kupfer, S, Maggioni, AP, Mebazaa, A, Metra, M, Nowack, C, Pieske, B, Pina, IL, Pocock, SJ, Ponikowski, P, Rosano, G, Ruilope, LM, Ruschitzka, F, Severin, T, Solomon, S, Stockbridge, NL, Stough, WG, Swedberg, K, Tavazzi, L, Voors, AA, Wasserman, SM, Woehrle, H, Zalewski, A & McMurray, JJV 2013, 'Clinical outcome endpoints in heart failure trials: A European Society of Cardiology Heart Failure Association consensus document', European Journal of Heart Failure, vol. 15, no. 10, pp. 1082-1094. https://doi.org/10.1093/eurjhf/hft095
Zannad, Faiez ; Stein, Kenneth ; Garcia, Angeles Alonso ; Anker, Stefan D. ; Armstrong, Paul W. ; Calvo, Gonzalo ; Cleland, John G F ; Cohn, Jay N. ; Dickstein, Kenneth ; Domanski, Michael J. ; Ekman, Inger ; Filippatos, Gerasimos S. ; Gheorghiade, Mihai ; Hernandez, Adrian F. ; Jaarsma, Tiny ; Koglin, Joerg ; Konstam, Marvin ; Kupfer, Stuart ; Maggioni, Aldo P. ; Mebazaa, Alexandre ; Metra, Marco ; Nowack, Christina ; Pieske, Burkert ; Pina, Ileana L. ; Pocock, Stuart J. ; Ponikowski, Piotr ; Rosano, Giuseppe ; Ruilope, Luis M. ; Ruschitzka, Frank ; Severin, Thomas ; Solomon, Scott ; Stockbridge, Norman L. ; Stough, Wendy Gattis ; Swedberg, Karl ; Tavazzi, Luigi ; Voors, Adriaan A. ; Wasserman, Scott M. ; Woehrle, Holger ; Zalewski, Andrew ; McMurray, John J V. / Clinical outcome endpoints in heart failure trials : A European Society of Cardiology Heart Failure Association consensus document. In: European Journal of Heart Failure. 2013 ; Vol. 15, No. 10. pp. 1082-1094.
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