Estimating the Lifetime Benefits of Treatments for Heart Failure

Autor: Pardeep S. Jhund, Stuart J. Pocock, Brian Claggett, Kieran F. Docherty, Faiez Zannad, John J.V. McMurray, Scott D. Solomon, John Gregson, João Pedro Ferreira, Susan Stienen, Mark C. Petrie
Přispěvatelé: Cardiology, ACS - Heart failure & arrhythmias, BHF Glasgow Cardiovascular Research Centre, University of Glasgow, Centre d'investigation clinique plurithématique Pierre Drouin [Nancy] (CIC-P), Centre d'investigation clinique [Nancy] (CIC), Centre Hospitalier Régional Universitaire de Nancy (CHRU Nancy)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Lorraine (UL)-Centre Hospitalier Régional Universitaire de Nancy (CHRU Nancy)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Lorraine (UL), Défaillance Cardiovasculaire Aiguë et Chronique (DCAC), Université de Lorraine (UL)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre Hospitalier Régional Universitaire de Nancy (CHRU Nancy), Cardiovascular and Renal Clinical Trialists [Vandoeuvre-les-Nancy] (INI-CRCT), Institut Lorrain du Coeur et des Vaisseaux Louis Mathieu [Nancy], French-Clinical Research Infrastructure Network - F-CRIN [Paris] (Cardiovascular & Renal Clinical Trialists - CRCT ), Heart Center, Department of Clinical and Experimental Cardiology, Amsterdam Cardiovascular Sciences. Amsterdam University Medical Center, University of Amsterdam, Cardiovascular Division Brigham and Women's Hospital 75 Francis Street Boston, Brigham and Women's Hospital [Boston], Department of Biostatistics, London School of Hygiene and Tropical Medicine, JPF & FZ are supported by ANR-15-RHU-0004 and ANR-15-IDEX-04-LUE. JJM, PSJ, and MCP are supported by BHF Grant RE/18/6/34217., ANR-15-RHUS-0004,FIGHT-HF,Combattre l'insuffisance cardiaque(2015), ANR-15-IDEX-0004,LUE (ISITE),Lorraine Université d'Excellence(2016), Centre Hospitalier Régional Universitaire de Nancy (CHRU Nancy)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Lorraine (UL)
Jazyk: angličtina
Rok vydání: 2020
Předmět:
medicine.medical_specialty
Spironolactone
030204 cardiovascular system & hematology
Lower risk
Placebo
HF
heart failure

Sacubitril
RMST
restricted mean survival time

03 medical and health sciences
0302 clinical medicine
[SDV.MHEP.CSC]Life Sciences [q-bio]/Human health and pathology/Cardiology and cardiovascular system
Mini-Focus: Clinical Trial Considerations
Risk Factors
Internal medicine
Patient-Centered Care
medicine
Humans
030212 general & internal medicine
NYHA
New York Heart Association

HFrEF
heart failure with reduced ejection fraction

Survival analysis
Mineralocorticoid Receptor Antagonists
Original Research
Heart Failure
survival models
business.industry
Hazard ratio
Absolute risk reduction
trials
Infant
Stroke Volume
treatment effects
HR
hazard ratio

Confidence interval
Eplerenone
3. Good health
CI
confidence interval

Survival Rate
restricted mean survival time
Research Design
Relative risk
NNT
number needed to treat

NT-proBNP
N-terminal pro–B-type natriuretic peptide

Cardiology and Cardiovascular Medicine
business
medicine.drug
Zdroj: JACC. Heart failure, 8(12), 984-995. Elsevier BV
Jacc. Heart Failure
JACC: Heart Failure
JACC: Heart Failure, Elsevier/American College of Cardiology, 2020, 8 (12), pp.984-995. ⟨10.1016/j.jchf.2020.08.004⟩
ISSN: 2213-1779
DOI: 10.1016/j.jchf.2020.08.004⟩
Popis: Objectives This study compared ways of describing treatment effects. The objective was to better explain to clinicians and patients what they might expect from a given treatment, not only in terms of relative and absolute risk reduction, but also in projections of long-term survival. Background The restricted mean survival time (RMST) can be used to estimate of long-term survival, providing a complementary approach to more conventional metrics (e.g., absolute and relative risk), which may suggest greater benefits of therapy in high-risk patients compared with low-risk patients. Methods Relative and absolute risk, as well as the RMST, were calculated in heart failure with reduced ejection fraction (HFrEF) trials. Results As examples, in the RALES trial (more severe HFrEF), the treatment effect metrics for spironolactone versus placebo on heart failure hospitalization and/or cardiovascular death were a hazard ratio (HR) of 0.67 (95% confidence interval [CI]: 0.5 to 0.77), number needed to treat = 9 (7 to 14), and age extension of event-free survival +1.1 years (−0.1 to + 2.3 years). The corresponding metrics for EMPHASIS-HF (eplerenone vs. placebo in less severe HFrEF) were 0.64 (0.54 to 0.75), 14 (1 to 22), and +2.9 (1.2 to 4.5). In patients in PARADIGM-HF aged younger than 65 years, the metrics for sacubitril/valsartan versus enalapril were 0.77 (95% CI: 0.68 to 0.88), 23 (15 to 44), and +1.7 (0.6 to 2.8) years; for those aged 65 years or older, the metrics were 0.83 (95% CI: 0.73 to 0.94), 29 (17 to 83), and +0.9 (0.2 to 1.6) years, which provided evidence of a greater potential life extension in younger patients. Similar observations were found for lower risk patients. Conclusions RMST event-free (and overall) survival estimates provided a complementary means of evaluating the effect of therapy in relation to age and risk. They also provided a clinically useful metric that should be routinely reported and used to explain the potential long-term benefits of a given treatment, especially to younger and less symptomatic patients.
Central Illustration
Databáze: OpenAIRE