Key result
Only ~40% of real-world HFpEF patients meet eligibility criteria for PARAGON-HF.
Why the study?
The proportion of real-world HFpEF patients superimposable with the PARAGON-HF population, along with their clinical characteristics and prognosis compared to non-eligible patients, was unknown.
What proportion of real-world HFpEF patients meet the eligibility criteria for the PARAGON-HF trial?
Cohort (n=427)
No
What proportion of real-world HFpEF patients meet the eligibility criteria for the PARAGON-HF trial?
Absolute Event Rate: 39.8% vs 60.2%
Less than 40% of real-world HFpEF patients meet PARAGON-HF eligibility criteria, highlighting that trial results may not apply to the majority of clinical patients, particularly those with the most advanced disease.
May limit PARAGON-HF generalizability to most real-world HFpEF patients; leaves open efficacy in ineligible subgroups.
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); p < 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; p < 0.001) and better outcomes (log-rank: p < 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); p < 0.001), highest pulmonary pressures (mPAP: 38.2 mmHg, SD: ±12.4; p < 0.001) and worst outcome (log-rank: p = 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.
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Rettl et al. (2020) conducted a cohort in Heart failure with preserved ejection fraction (HFpEF) (n=427). Eligibility for the PARAGON-HF trial vs. Ineligibility for the PARAGON-HF trial was evaluated on Theoretical eligibility for the PARAGON-HF trial. Only 39.8% of real-world HFpEF patients in a prospective registry met the theoretical eligibility criteria for the PARAGON-HF trial, suggesting a large proportion of patients may not qualify for this treatment.
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