按种族和性别划分的心力衰竭试验代表性：ASCEND-HF 和 GWTG-HF 的结果。
Representativeness of a Heart Failure Trial by Race and Sex: Results From ASCEND-HF and GWTG-HF.
机构信息
Duke Clinical Research Institute, Durham, North Carolina; Division of Cardiology, Duke University School of Medicine, Durham, North Carolina.
Duke Clinical Research Institute, Durham, North Carolina; Division of Cardiology, Duke University School of Medicine, Durham, North Carolina.
出版信息
JACC Heart Fail. 2019 Nov;7(11):980-992. doi: 10.1016/j.jchf.2019.07.011. Epub 2019 Oct 9.
OBJECTIVES
This study sought to determine the degree to which U.S. patients enrolled in a heart failure (HF) trial represent patients in routine U.S. clinical practice according to race and sex.
BACKGROUND
Black patients and women are frequently under-represented in HF clinical trials. However, the degree to which black patients and women enrolled in trials represent such patients in routine practice is unclear.
METHODS
The ASCEND-HF (Acute Study of Clinical Effectiveness of Nesiritide in Decompensated Heart Failure) trial randomized patients hospitalized for HF to receive nesiritide or placebo from May 2007 to August 2010 and was neutral for clinical endpoints. This analysis compared non-Hispanic white (n = 1,494) and black (n = 1,012) patients enrolled in ASCEND-HF from the U.S. versus non-Hispanic white and black patients included in a U.S. hospitalized HF registry (i.e., Get With The Guidelines-Heart Failure [GWTG-HF]) during the ASCEND-HF enrollment period and meeting trial eligibility criteria.
RESULTS
Among 79,291 white and black registry patients, 49,063 (62%) met trial eligibility criteria (white, n = 37,883 [77.2%]; black, n = 11,180 [22.8%]). Women represented 35% and 49% of the ASCEND-HF and trial-eligible GWTG-HF cohorts, respectively. Compared with trial-enrolled patients, trial-eligible GWTG-HF patients tended to be older with higher blood pressure and higher ejection fraction. Trial-eligible patients had higher in-hospital mortality (2.3% vs. 1.3%), 30-day readmission (20.2% vs. 16.8%), and 180-day mortality (21.2% vs. 18.6%) than those enrolled in the trial (all p < 0.02), with consistent mortality findings by race and sex. After propensity score matching, mortality rates were similar; however, trial-eligible patients continued to have higher rates of 30-day readmission (23.1% vs. 17.3%; p < 0.01), driven by differences among black patients and women (all p for interaction ≤0.02).
CONCLUSIONS
Patients with HF seen in U.S. practice and eligible for the ASCEND-HF trial had worse clinical outcomes than those enrolled in the trial. After accounting for clinical characteristics, trial-eligible real-world patients continued to have higher rates of 30-day readmission, driven by differences among black patients and women. Social, behavioral, and other unmeasured factors may impair representativeness of patients enrolled in HF trials, particularly among racial/ethnic minorities and women. (Acute Study of Clinical Effectiveness of Nesiritide in Decompensated Heart Failure [ASCEND-HF]; NCT00475852).
目的
本研究旨在根据种族和性别，确定纳入心力衰竭（HF）试验的美国患者在多大程度上代表了常规美国临床实践中的患者。
背景
黑人患者和女性在 HF 临床试验中经常代表性不足。然而，纳入试验的黑人患者和女性在多大程度上代表了常规实践中的此类患者尚不清楚。
方法
ASCEND-HF（急性心力衰竭中奈西立肽临床疗效的研究）试验于 2007 年 5 月至 2010 年 8 月期间随机分配因 HF 住院的患者接受奈西立肽或安慰剂治疗，该试验对临床终点无影响。本分析比较了 ASCEND-HF 试验中来自美国的非西班牙裔白人（n=1494）和黑人（n=1012）患者与 ASCEND-HF 招募期间符合试验入选标准的美国住院 HF 登记处（即 Get With The Guidelines-Heart Failure [GWTG-HF]）中登记的非西班牙裔白人（n=79291）和黑人患者。
结果
在 79291 名白人及黑人登记患者中，有 49063 名（62%）符合试验入选标准（白人，n=37883 [77.2%]；黑人，n=11180 [22.8%]）。女性分别占 ASCEND-HF 和符合试验入选标准的 GWTG-HF 队列的 35%和 49%。与试验入组患者相比，符合试验入选标准的 GWTG-HF 患者年龄更大，血压更高，射血分数更高。符合试验入选标准的患者院内死亡率（2.3% vs. 1.3%）、30 天再入院率（20.2% vs. 16.8%）和 180 天死亡率（21.2% vs. 18.6%）均高于试验入组患者（均 p<0.02），且种族和性别之间死亡率的结果一致。经倾向评分匹配后，死亡率相似；然而，符合试验入选标准的患者 30 天再入院率仍较高（23.1% vs. 17.3%；p<0.01），这主要是由于黑人患者和女性之间的差异（所有 p 交互作用值均≤0.02）。
结论
在美国临床实践中接受治疗且符合 ASCEND-HF 试验入选标准的 HF 患者的临床结局较试验入组患者更差。在考虑了临床特征后，符合试验入选标准的真实世界患者的 30 天再入院率仍较高，这主要是由于黑人患者和女性之间的差异所致。社会、行为和其他未测量的因素可能会影响 HF 试验中入组患者的代表性，尤其是在少数族裔和女性中。（急性心力衰竭中奈西立肽临床疗效的研究[ASCEND-HF]；NCT00475852）。
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