射血分数保留的心力衰竭患者的健康相关生活质量：PARAGON-HF 试验。

Health-Related Quality of Life in Heart Failure With Preserved Ejection Fraction: The PARAGON-HF Trial.

机构信息

Cardiovascular Division, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts.

Novartis Pharmaceutical Corporation, East Hanover, New Jersey.

出版信息

JACC Heart Fail. 2019 Oct;7(10):862-874. doi: 10.1016/j.jchf.2019.05.015. Epub 2019 Jul 10.

DOI:10.1016/j.jchf.2019.05.015

PMID:31302043

Abstract

OBJECTIVES

This study sought to describe baseline health-related quality of life (HRQL) in the PARAGON-HF (Prospective Comparison of ARNI with ARB Global Outcomes in HFpEF) trial, the largest heart failure with preserved ejection fraction (HFpEF) trial to date.

BACKGROUND

There are limited data characterizing HRQL in patients with HFpEF using validated metrics.

METHODS

The PARAGON-HF trial randomized symptomatic patients with HFpEF (≥45%) ≥50 years of age to either sacubitril/valsartan or valsartan. The study reports comprehensive baseline HRQL using Kansas City Cardiomyopathy Questionnaire (KCCQ) administered at randomization after active run-in period. The study then compares baseline HRQL with patients with heart failure with reduced ejection fraction (HFrEF) (≤40%) enrolled in the PARADIGM-HF (Prospective Comparison of ARNI with an ACE-Inhibitor to Determine Impact on Global Mortality and Morbidity in Heart Failure) trial. Forward multivariable stepwise regression modeling was performed separately in both trials to identify independent clinical correlates of KCCQ-Overall Summary (KCCQ-OS) score. PARADIGM-HF trial patients <50 years of age were excluded to enable comparison.

RESULTS

In the PARAGON-HF trial, 4,735 of 4,822 patients (mean age 73 ± 8 years; 48% men) completed baseline KCCQ at randomization. Mean KCCQ-OS score was 71. Women had worse mean KCCQ-OS score than men did. Patients in the PARAGON-HF trial reported lower KCCQ scores in nearly all domains when compared with the PARADIGM-HF trial (KCCQ-OS score 71 ± 19 vs. 73 ± 19; p < 0.001). The strongest independent clinical correlates of adverse HRQL in both the PARAGON-HF and PARADIGM-HF trials were New York Heart Association functional class, female gender, lower extremity edema, body mass index, angina, dyspnea, and paroxysmal nocturnal dyspnea. After accounting for these clinical correlates of adverse HRQL that were common to both HFpEF and HFrEF patients, KCCQ-OS score did not differ significantly.

CONCLUSIONS

HRQL was largely worse in women and was similar in HFpEF and HFrEF after accounting for variation in demographics, functional status, and symptom burden. (Prospective Comparison of ARNI with ARB Global Outcomes in HFpEF [PARAGON-HF] NCT01920711; Prospective Comparison of ARNI with an ACE-Inhibitor to Determine Impact on Global Mortality and Morbidity in Heart Failure [PARADIGM-HF]; NCT01035255).

摘要

目的

本研究旨在描述 PARAGON-HF（HFpEF 中 ARNI 与 ARB 全球结局的前瞻性比较）试验中基线健康相关生活质量（HRQL），这是迄今为止最大的射血分数保留心力衰竭（HFpEF）试验。

背景

使用经过验证的指标描述 HFpEF 患者 HRQL 的数据有限。

方法

PARAGON-HF 试验将有症状的 HFpEF（≥45%）≥50 岁的患者随机分为 sacubitril/valsartan 或 valsartan 组。该研究使用 Kansas City 心肌病问卷（KCCQ）在主动洗脱期后随机分组时报告全面的基线 HRQL。然后，将基线 HRQL 与 PARADIGM-HF（ARNI 与 ACE 抑制剂对心力衰竭全球死亡率和发病率影响的前瞻性比较）试验中射血分数降低的心力衰竭（HFrEF）（≤40%）患者进行比较。在两项试验中分别进行向前逐步多元回归建模，以确定 KCCQ 总体综合评分（KCCQ-OS）的独立临床相关因素。排除 PARADIGM-HF 试验中年龄<50 岁的患者，以进行比较。

结果

在 PARAGON-HF 试验中，4822 例患者中有 4735 例（平均年龄 73±8 岁；48%为男性）在随机分组时完成了基线 KCCQ。平均 KCCQ-OS 评分为 71。女性的平均 KCCQ-OS 评分比男性差。与 PARADIGM-HF 试验相比，PARAGON-HF 试验的患者在几乎所有领域的 KCCQ 评分均较低（KCCQ-OS 评分 71±19 与 73±19；p<0.001）。在 PARAGON-HF 和 PARADIGM-HF 两项试验中，对不良 HRQL 具有最强独立临床相关性的因素是纽约心脏协会功能分级、女性、下肢水肿、体重指数、心绞痛、呼吸困难和阵发性夜间呼吸困难。在考虑到 HFpEF 和 HFrEF 患者共有的这些不良 HRQL 的临床相关因素后，KCCQ-OS 评分无显著差异。

结论

在考虑人口统计学、功能状态和症状负担的差异后，女性的 HRQL 总体较差，HFpEF 和 HFrEF 之间的 HRQL 相似。（HFpEF 中 ARNI 与 ARB 全球结局的前瞻性比较[PARAGON-HF]NCT01920711；ARNI 与 ACE 抑制剂对心力衰竭全球死亡率和发病率影响的前瞻性比较[PARADIGM-HF]NCT01035255）。