Kelly Jacob P, Mentz Robert J, Mebazaa Alexandre, Voors Adriaan A, Butler Javed, Roessig Lothar, Fiuzat Mona, Zannad Faiez, Pitt Bertram, O'Connor Christopher M, Lam Carolyn S P
Division of Cardiology, Department of Medicine, Duke University Medical Center, Durham, North Carolina.
Division of Cardiology, Department of Medicine, Duke University Medical Center, Durham, North Carolina.
J Am Coll Cardiol. 2015 Apr 28;65(16):1668-1682. doi: 10.1016/j.jacc.2015.03.043.
Recent clinical trials in patients with heart failure with preserved ejection fraction (HFpEF) have provided important insights into participant selection strategies. Historically, HFpEF trials have included patients with relatively preserved left ventricular ejection fraction ranging from 40% to 55% and a clinical history of heart failure. Contemporary HFpEF trials have also incorporated inclusion criteria such as hospitalization for HFpEF, altered functional capacity, cardiac structural and functional abnormalities, and abnormalities in neurohormonal status (e.g., elevated natriuretic peptide levels). Careful analyses of the effect of these patient selection criteria on outcomes in prior trials provide valuable lessons for future trial design. We review recent and ongoing HFpEF clinical trials from a patient selection perspective and appraise trial patient selection methodologies in relation to outcomes. This review reflects discussions between clinicians, scientists, trialists, regulators, and regulatory representatives at the 10th Global CardioVascular Clinical Trialists Forum in Paris, France, on December 6, 2013.
近期针对射血分数保留的心力衰竭（HFpEF）患者开展的临床试验，为受试者选择策略提供了重要见解。从历史上看，HFpEF试验纳入的患者左心室射血分数相对保留，范围在40%至55%之间，且有心力衰竭临床病史。当代HFpEF试验还纳入了诸如因HFpEF住院、功能能力改变、心脏结构和功能异常以及神经激素状态异常（如利钠肽水平升高）等纳入标准。对这些患者选择标准在既往试验中对结局的影响进行仔细分析，可为未来试验设计提供宝贵经验。我们从患者选择角度回顾近期及正在进行的HFpEF临床试验，并根据结局评估试验患者选择方法。本综述反映了2013年12月6日在法国巴黎举行的第10届全球心血管临床试验专家论坛上临床医生、科学家、试验人员、监管机构及监管代表之间的讨论。
