From the, Division of Cardiology, Department of Medicine, Karolinska Institutet, Stockholm, Sweden.
Department of Cardiology, University Heart and Vascular Center Hamburg, Hamburg, Germany.
J Intern Med. 2021 Mar;289(3):369-384. doi: 10.1111/joim.13165. Epub 2020 Sep 1.
Randomized controlled trials (RCT) generalizability may be limited due to strict patient selection.
In a real-world heart failure (HF) population, we assessed eligibility for sacubitril/valsartan based on PARADIGM-HF (sacubitril/valsartan effective)/PARAGON-HF [sacubitril/valsartan effective in mildly reduced ejection fraction (EF)].
Outpatients from the Swedish HF Registry (SwedeHF) were analysed. In SwedeHF, EF is recorded as <30, 30-39, 40-49 and ≥50%. In PARAGON-HF, sacubitril/valsartan was effective with EF ≤ 57% (i.e. median). We defined reduced EF/PARADIGM-HF as EF < 40%, mildly reduced EF/PARAGON-HF ≤ median as EF 40-49%, and normal EF/PARAGON-HF > median as EF ≥ 50%. We assessed 2 scenarios: (i) criteria likely to influence treatment decisions (pragmatic scenario); (ii) all criteria (literal scenario).
Of 37 790 outpatients, 57% had EF < 40%, 24% EF 40-49% and 19% EF ≥ 50%. In the pragmatic scenario, 63% were eligible in EF < 50% (67% for EF < 40% and 52% for 40-49%) and 52% in EF ≥ 40% (52% for EF ≥ 50%). For the literal scenario, 32% were eligible in EF < 50% (38% of EF < 40%, 20% of EF 40-49%) and 22% in EF ≥ 40% (25% for EF ≥ 50%). Eligible vs. noneligible patients had more severe HF, more comorbidities and overall worse outcomes.
In a real-world HF outpatient cohort, 81% of patients had EF < 50%, with 63% eligible for sacubitril/valsartan based on pragmatic criteria and 32% eligible based on literal trial criteria. Similar eligibility was observed for EF 40-49% and ≥50%, suggesting that our estimates for EF < 50% may be reproduced whether or not a higher cut-off for EF is considered.
随机对照试验（RCT）的普遍性可能由于严格的患者选择而受到限制。
在真实世界的心衰（HF）人群中，我们根据 PARADIGM-HF（沙库巴曲缬沙坦有效）/PARAGON-HF[沙库巴曲缬沙坦在轻度射血分数降低（EF）中的有效性]评估基于 sacubitril/valsartan 的适用性。
分析来自瑞典心衰注册（SwedeHF）的门诊患者。在 SwedeHF 中，EF 记录为 <30、30-39、40-49 和 ≥50%。在 PARAGON-HF 中，沙库巴曲缬沙坦在 EF≤57%（即中位数）时有效。我们将降低的 EF/PARADIGM-HF 定义为 EF<40%，轻度降低的 EF/PARAGON-HF≤中位数为 EF 40-49%，正常的 EF/PARAGON-HF>中位数为 EF≥50%。我们评估了 2 种情况：（i）可能影响治疗决策的标准（实用方案）；（ii）所有标准（字面方案）。
在 37790 名门诊患者中，57%的 EF<40%，24%的 EF 40-49%，19%的 EF≥50%。在实用方案中，EF<50%的患者中 63%符合条件（EF<40%的患者中为 67%，EF 40-49%的患者中为 52%），EF≥40%的患者中 52%符合条件（EF≥50%的患者中为 52%）。对于字面方案，EF<50%的患者中有 32%符合条件（EF<40%的患者中为 38%，EF 40-49%的患者中为 20%），EF≥40%的患者中有 22%符合条件（EF≥50%的患者中为 25%）。符合条件与不符合条件的患者具有更严重的心衰、更多的合并症和整体更差的结局。
在真实世界的心衰门诊患者队列中，81%的患者 EF<50%，根据实用标准，63%的患者符合 sacubitril/valsartan 的条件，32%的患者符合字面试验标准。EF 为 40-49%和≥50%的患者也有类似的适用性，这表明，无论是否考虑更高的 EF 截止值，我们对 EF<50%的估计都可能重现。
