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

Autor: Anker SD; Innovative Clinical Trials, Depar tment of Cardiology & Pneumology, University Medical Center Göttingen (UMG), Germany., Schroeder S; Bayer Pharma AG, Berlin, Germany., Atar D; Department of Cardiology B, Oslo University Hospital Ulleval and University of Oslo, Norway., Bax JJ; Department of Cardiology, Leiden University Medical Center, Leiden, The Netherlands., Ceconi C; Unit of Cardiology, Hospital of Desenzano del Garda, Desenzano del Garda, Italy., Cowie MR; National Heart and Lung Institute, Imperial College London, London, UK., Crisp A; GlaxoSmithKline, Middlesex, UK., Dominjon F; Servier International, Paris, France., Ford I; Robertson Centre for Biostatistics, University of Glasgow, Glasgow, UK., Ghofrani HA; University of Giessen and Marburg Lung Center, UGMLC [member of the German Center for Lung Research (DZL)], Giessen, Germany.; Kerckhoff-Klinik Bad Nauheim, Germany.; Imperial College London, UK., Gropper S; Boehringer-Ingelheim GmBH & Co. KG, Ingelheim, Germany., Hindricks G; Department of Electrophysiology, Heart Center, University of Leipzig, Leipzig, Germany., Hlatky MA; Stanford University School of Medicine, Stanford, CA, USA., Holcomb R; Independent Biostatistician, Minneapolis, MN, USA., Honarpour N; Amgen, Inc., Thousand Oaks, CA, USA., Jukema JW; Department of Cardiology, Leiden University Medical Center, Leiden, The Netherlands., Kim AM; Pfizer, Inc., Cambridge, MA, USA., Kunz M; Bayer Pharma AG, Berlin, Germany., Lefkowitz M; Novartis Pharmaceuticals, East Hanover, NJ, USA., Le Floch C; Servier International, Paris, France., Landmesser U; Department of Cardiology, Charité Universitätsmedizin Berlin, Berlin, Germany., McDonagh TA; King's College Hospital, London, UK., McMurray JJ; BHF Cardiovascular Research Centre, University of Glasgow, Glasgow, UK., Merkely B; Semmelweis University Heart and Vascular Center, Budapest, Hungary., Packer M; Department of Clinical Sciences, University of Texas Southwestern Medical Center, Dallas, TX, USA., Prasad K; United Kingdom Medicines and Healthcare Products Regulatory Agency, London, UK., Revkin J; Pfizer, Inc., Cambridge, MA, USA., Rosano GM; IRCCS San Raffaele Hospital Roma, Rome, Italy.; Cardiovascular and Cell Sciences Institute, St. George's University of London, London, UK., Somaratne R; Amgen, Inc., Thousand Oaks, CA, USA., Stough WG; Campbell University College of Pharmacy and Health Sciences, NC, USA., Voors AA; University of Groningen, Groningen, The Netherlands., Ruschitzka F; Department of Cardiology, Heart Failure Clinic and Transplantation, University Heart Center Zurich, Zurich, Switzerland.
Jazyk: angličtina
Zdroj: European journal of heart failure [Eur J Heart Fail] 2016 May; Vol. 18 (5), pp. 482-9. Date of Electronic Publication: 2016 Apr 12.
DOI: 10.1002/ejhf.516
Abstrakt: 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.
(© 2016 The Authors. European Journal of Heart Failure © 2016 European Society of Cardiology.)
Databáze: MEDLINE
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