Butler Javed, Hamo Carine E, Udelson James E, Pitt Bertram, Yancy Clyde, Shah Sanjiv J, Desvigne-Nickens Patrice, Bernstein Harold S, Clark Richard L, Depre Christophe, Dinh Wilfried, Hamer Andrew, Kay-Mugford Patricia, Kramer Frank, Lefkowitz Martin, Lewis Kelly, Maya Juan, Maybaum Simon, Patel Mahesh J, Pollack Pia S, Roessig Lothar, Rotman Sarit, Salsali Afshin, Sims J Jason, Senni Michele, Rosano Giuseppe, Dunnmon Preston, Stockbridge Norman, Anker Stefan D, Zile Michael R, Gheorghiade Mihai
From the Department of Medicine, Stony Brook University, NY (J.B., C.E.H.); Division of Cardiology, Tufts Medical Center, Tufts University School of Medicine, Boston, MA (J.E.U.); Division of Cardiology, University of Michigan School of Medicine, Ann Arbor (B.P.); Division of Cardiology (C.Y., S.J.S.) and Center for Cardiovascular Innovation (M.G.), Feinberg School of Medicine, Northwestern University, Chicago, IL; Division of Cardiovascular Sciences, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD (P.D.-N); Merck & Co, Kenilworth, NJ (H.S.B.); Medtronic, Mounds View, MN (R.L.C., J.J.S.); Amgen Inc, Thousand Oaks, CA (C.D., A.H., J.M.); Bayer Pharma AG, Wuppertal, Germany (W.D., F.K., K.L., L.R., S.R.); Department of Cardiology, HELIOS Clinic Wuppertal, University Hospital Witten/Herdecke, Germany (W.D.); Novartis Pharmaceuticals Inc, East Hanover, NJ (P.K.-M., M.L., S.M.); Clinical Research in Cardiovascular Disease Division, Merck Research Laboratories, Rahway, NJ (M.J.P.); AstraZeneca Global Medicines Development, Gaithersburg, MD (P.S.P.); Boehringer Ingelheim Pharmaceuticals Inc, Ridgefield, CT (A.S.); Dipartimento Cardiovascolare, Azienda Ospedaliera Papa Giovannni XXIII, Bergamo, Italy (M.S.); Department of Medical Sciences, IRCCS San Raffaele Pisana, Roma, Italy (G.R.); Cardiovascular and Cell Science Institute, St George's University of London, United Kingdom (G.R.); Division of Cardiovascular and Renal Products, the United States Food and Drug Administration, Silver Spring, MD (P.D., N.S.); Innovative Clinical Trials, Department of Cardiology and Pneumology, University Medical Center Göttingen (UMG), Germany (S.D.A.); and Medical University of South Carolina and RHJ Department of Veterans Administration Medical Center, Charleston (M.R.Z.).
Circ Heart Fail. 2016 Nov;9(11). doi: 10.1161/CIRCHEARTFAILURE.116.003358.
The epidemiological, clinical, and societal implications of the heart failure (HF) epidemic cannot be overemphasized. Approximately half of all HF patients have HF with preserved ejection fraction (HFpEF). HFpEF is largely a syndrome of the elderly, and with aging of the population, the proportion of patients with HFpEF is expected to grow. Currently, there is no drug known to improve mortality or hospitalization risk for these patients. Besides mortality and hospitalization, it is imperative to realize that patients with HFpEF have significant impairment in their functional capacity and their quality of life on a daily basis, underscoring the need for these parameters to ideally be incorporated within a regulatory pathway for drug approval. Although attempts should continue to explore therapies to reduce the risk of mortality or hospitalization for these patients, efforts should also be directed to improve other patient-centric concerns, such as functional capacity and quality of life. To initiate a dialogue about the compelling need for and the challenges in developing such alternative endpoints for patients with HFpEF, the US Food and Drug Administration on November 12, 2015, facilitated a meeting represented by clinicians, academia, industry, and regulatory agencies. This document summarizes the discussion from this meeting.
心力衰竭（HF）流行所带来的流行病学、临床及社会影响再怎么强调都不为过。所有HF患者中约有一半患有射血分数保留的心力衰竭（HFpEF）。HFpEF在很大程度上是一种老年综合征，随着人口老龄化，HFpEF患者的比例预计将会增加。目前，尚无已知药物可改善这些患者的死亡率或住院风险。除了死亡率和住院率，必须认识到HFpEF患者的日常功能能力和生活质量有显著损害，这突出表明理想情况下需要将这些参数纳入药物批准的监管途径。尽管应继续尝试探索降低这些患者死亡率或住院风险的疗法，但也应致力于改善其他以患者为中心的问题，如功能能力和生活质量。为了就开发针对HFpEF患者的此类替代终点的迫切需求和挑战展开对话，美国食品药品监督管理局于2015年11月12日促成了一次由临床医生、学术界、产业界和监管机构代表参加的会议。本文件总结了此次会议的讨论内容。
