患者报告的结果作为心力衰竭试验的终点
Javed Butler1,2, Muhammad Shariq Usman3,4, Charu Gandotra5
1Baylor Scott and White Research Institute, Baylor Scott and White Health, Dallas, TX (J.B.).
Circulation
|April 14, 2025
概括
患者报告的结果对于心力衰竭治疗至关重要,但解释试验结果具有挑战性. 本文讨论了PRO评估和分析的问题,以改善其在研究和临床实践中的使用.
科学领域:
- 心脏病学
- 临床研究
- 健康问题
背景情况:
- 心力衰竭影响全球数以百万计的患者,患者报告的结果 (PROs) 与住院和死亡率等传统指标的认识越来越多.
- 监管机构和专业协会承认PRO,但由于方法不一致,它们在心力衰竭试验中的解释仍然困难.
- 挑战包括缺乏关于PRO评估时间/频率的共识,以及定义临床重要差异的最小值.
研究的目的:
- 为了解决心力衰竭临床试验中解读PRO数据的困难.
- 讨论与PRO评估,临床重要差异和分析方法相关的挑战.
- 促进PRO在心力衰竭研究,监管评估和临床实践中的有效使用.
主要方法:
- 深入讨论围绕心力衰竭的PRO评估和分析的问题.
- 使用堪萨斯市心肌病问卷 (KCCQ) 作为一个具体的例子.
- 批评传统的分析方法,如平均差异和响应者分析的统计局限性.
主要成果:
- 目前在心力衰竭试验中评估和分析PRO的方法往往难以解释.
- 缺乏对PRO评估时间和频率的标准化协议阻碍了数据收集的一致性.
- 对于PROs来说,确定最小的临床重要差异仍然是一个重大挑战.
结论:
- 标准化PRO评估和分析对于心力衰竭研究的可靠解释至关重要.
- 解决方法上的挑战将增强PRO对评估干预和指导临床决策的有用性.
- 在心力衰竭管理中,提高PRO利用率可以带来更好的以患者为中心的治疗.
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Natriuretic Peptides (BNP)
Cardiac myocytes produce these hormones in response to ventricular stretching...
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