心脏粉症注册研究 (CARS):理由,设计和方法论
Lily K Stern1, Justin L Grodin2, Mathew S Maurer3
1Department of Cardiology, Cedars-Sinai Smidt Heart Institute, Los Angeles, CA, USA.
Journal of cardiac failure
|October 31, 2023
概括
心脏粉症注册研究 (CARS) 追踪心脏粉症 (ATTR) 和轻链 (AL) 患者,以了解不同人群中的疾病进展和治疗有效性.
科学领域:
- 心脏病学 心脏病学
- 血液学 血液学 血液学
- 罕见疾病 罕见疾病
背景情况:
- 心脏粉症注册表研究 (CARS) 是一项于2019年建立的多中心注册表.
- 它包括患有过静素 (ATTR) 和轻链 (AL) 心脏粉症 (CA) 的患者.
- 在1997年至2025年期间,CARS评估了来自主要粉样性粉症中心的患者.
研究的目的:
- 为了描述心脏粉症的自然史.
- 在诊断时分析临床和诊断变量.
- 评估现实世界治疗模式和结果在一个多元化的队列.
主要方法:
- 描述了CARS的数据收集程序和方法.
- 截至2023年2月,20个美国中心注册了1415名患者.
- 该注册表捕获了多样化的患者人口统计数据,包括种族和性别分布.
主要成果:
- 1155名患者 (82%) 患有ATTR心肌粉症,260名 (18%) 患有AL心肌粉症.
- 野生型ATTR是最常见的 (71%),p.V142I突变在68%的ATTR病例中发生.
- 总人口的四分之一是黑人;AL患者比ATTR患者更常见的是女性.
结论:
- 汽车将解决有关CA自然历史的关键临床问题.
- 它将使不同治疗方法的比较能够超越目前临床试验的限制.
- 建议开展国际合作,以提高这种已知的疾病发现的有效性.
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