Desai Nihar, Olewinska Elzbieta, Famulska Agata, Remuzat Cécile, Francois Clément, Folkerts Kerstin
Section of Cardiovascular Medicine, Department of Internal Medicine, Yale School of Medicine, New Haven, CT, USA.
Putnam, Cracow, Poland.
Heart Fail Rev. 2024 May;29(3):631-662. doi: 10.1007/s10741-024-10385-y. Epub 2024 Feb 27.
This review provides a comprehensive overview of heart failure with mildly reduced and preserved ejection fraction (HFmrEF/HFpEF), including its definition, diagnosis, and epidemiology; clinical, humanistic, and economic burdens; current pharmacologic landscape in key pharmaceutical markets; and unmet needs to identify key knowledge gaps. We conducted a targeted literature review in electronic databases and prioritized articles with valuable insights into HFmrEF/HFpEF. Overall, 27 randomized controlled trials (RCTs), 66 real-world evidence studies, 18 clinical practice guidelines, and 25 additional publications were included. Although recent heart failure (HF) guidelines set left ventricular ejection fraction thresholds to differentiate categories, characterization and diagnosis criteria vary because of the incomplete disease understanding. Recent epidemiological data are limited and diverse. Approximately 50% of symptomatic HF patients have HFpEF, more common than HFmrEF. Prevalence varies with country because of differing definitions and study characteristics, making prevalence interpretation challenging. HFmrEF/HFpEF has considerable mortality risk, and the mortality rate varies with study and patient characteristics and treatments. HFmrEF/HFpEF is associated with considerable morbidity, poor patient outcomes, and common comorbidities. Patients require frequent hospitalizations; therefore, early intervention is crucial to prevent disease burden. Recent RCTs show promising results like risk reduction of composite cardiovascular death or HF hospitalization. Costs data are scarce, but the economic burden is increasing. Despite new drugs, unmet medical needs requiring new treatments remain. Thus, HFmrEF/HFpEF is a growing global healthcare concern. With improving yet incomplete understanding of this disease and its promising treatments, further research is required for better patient outcomes.
本综述全面概述了射血分数轻度降低和保留的心力衰竭(HFmrEF/HFpEF),包括其定义、诊断和流行病学;临床、人文和经济负担;主要制药市场的当前药物格局;以及未满足的需求以识别关键知识差距。我们在电子数据库中进行了有针对性的文献综述,并对具有HFmrEF/HFpEF宝贵见解的文章进行了优先排序。总体而言,纳入了27项随机对照试验(RCT)、66项真实世界证据研究、18项临床实践指南和25篇其他出版物。尽管最近的心力衰竭(HF)指南设定了左心室射血分数阈值以区分类别,但由于对该疾病的理解不完整,其特征和诊断标准各不相同。最近的流行病学数据有限且多样。大约50%的有症状HF患者患有HFpEF,比HFmrEF更常见。由于定义和研究特征不同,患病率因国家而异,这使得患病率的解释具有挑战性。HFmrEF/HFpEF具有相当大的死亡风险,死亡率因研究、患者特征和治疗方法而异。HFmrEF/HFpEF与相当大的发病率、不良患者预后和常见合并症相关。患者需要频繁住院;因此,早期干预对于预防疾病负担至关重要。最近的RCT显示出有希望的结果,如复合心血管死亡或HF住院风险降低。成本数据稀缺,但经济负担正在增加。尽管有新药,但仍存在需要新治疗方法的未满足医疗需求。因此,HFmrEF/HFpEF是一个日益受到全球医疗关注的问题。随着对这种疾病及其有前景的治疗方法的理解有所改善但仍不完整,需要进一步研究以实现更好的患者预后。