Rettl René, Dachs Theresa-Marie, Duca Franz, Binder Christina, Dusik Fabian, Seirer Benjamin, Schönauer Johannes, Kronberger Christina, Camuz Ligios Luciana, Hengstenberg Christian, Derkits Nina, Kastner Johannes, Badr Eslam Roza, Bonderman Diana
Division of Cardiology, Department of Internal Medicine II, Medical University of Vienna, Waehringer Guertel 18-20, 1090 Vienna, Austria.
Novartis Pharma GmbH, Stella-Klein-Loew-Weg 17, 1020 Vienna, Austria.
J Clin Med. 2020 Nov 15;9(11):3669. doi: 10.3390/jcm9113669.
The PARAGON-HF clinical trial suggested that sacubitril/valsartan may become a treatment option for particular subgroups of patients with heart failure and preserved ejection fraction (HFpEF). However, the proportion of real-world HFpEF patients who are theoretically superimposable with the PARAGON-HF population is yet unknown. The present study was performed to define the proportion of real-world PARAGON-HF-like patients and to describe their clinical characteristics and long-term prognosis in comparison with those who would not meet PARAGON-HF criteria. We systematically applied PARAGON-HF inclusion and exclusion criteria to a total of 427 HFpEF patients who have been participating in a prospective national registry between December 2010 and December 2019. In total, only 170 (39.8%) registry patients were theoretically eligible for PARAGON-HF. Patients not meeting inclusion criteria (41.0%) were less impaired with respect to exercise capacity (median 6-min walk distance: 385 m (IQR: 300-450) versus 323 m (IQR: 240-383); < 0.001) had lower pulmonary pressures (mean pulmonary artery pressure (mPAP): 31.2 mmHg, standard deviation (SD): ±10.2 versus 32.8 mmHg, SD: ±9.7; < 0.001) and better outcomes (log-rank: < 0.001) as compared to the PARAGON-like cohort. However, patients theoretically excluded from the trial (19.2%) were those with most advanced heart failure symptoms (median 6-min walk test: 252 m (IQR: 165-387); < 0.001), highest pulmonary pressures (mPAP: 38.2 mmHg, SD: ±12.4; < 0.001) and worst outcome (log-rank: = 0.037). We demonstrate here that < 40% of real-world HFpEF patients meet eligibility criteria for PARAGON-HF. We conclude that despite reasons for optimism after PARAGON-HF, a large proportion of HFpEF patients will remain without meaningful treatment options.
PARAGON-HF临床试验表明，沙库巴曲缬沙坦可能成为射血分数保留的心力衰竭（HFpEF）特定亚组患者的一种治疗选择。然而，理论上与PARAGON-HF研究人群重叠的真实世界HFpEF患者的比例尚不清楚。本研究旨在确定真实世界中类PARAGON-HF患者的比例，并描述他们的临床特征以及与不符合PARAGON-HF标准的患者相比的长期预后。我们将PARAGON-HF纳入和排除标准系统地应用于2010年12月至2019年12月期间参与一项全国前瞻性登记研究的427例HFpEF患者。总体而言，理论上只有170例（39.8%）登记患者符合PARAGON-HF的入选标准。不符合纳入标准的患者（41.0%）在运动能力方面受损较轻（6分钟步行距离中位数：385米（四分位间距：300-450）对323米（四分位间距：240-383）；P<0.001），肺压力较低（平均肺动脉压（mPAP）：31.2mmHg，标准差（SD）：±10.2对32.8mmHg，SD：±9.7；P<0.001），与类PARAGON队列相比结局更好（对数秩检验：P<0.001）。然而，理论上被排除在试验之外的患者（19.2%）是那些心力衰竭症状最严重的患者（6分钟步行试验中位数：252米（四分位间距：165-387）；P<0.001），肺压力最高（mPAP：38.2mmHg，SD：±12.4；P<0.001），结局最差（对数秩检验：P=0.037）。我们在此证明，真实世界中<40%的HFpEF患者符合PARAGON-HF的入选标准。我们得出结论，尽管PARAGON-HF研究结果带来了乐观的理由，但很大一部分HFpEF患者仍将没有有效的治疗选择。
