在第3期ATTR-ACT和正在进行的长期扩展研究中,塔法米迪斯在八分生患者中的有效性
Pablo Garcia-Pavia1, Marla B Sultan2, Balarama Gundapaneni3
1Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV, Madrid, Spain; Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid, Spain; Universidad Francisco de Vitoria (UFV), Pozuelo de Alarcón, Madrid, Spain.
JACC. Heart failure
|November 9, 2023
概括
塔法米迪斯在治疗所有年龄组的甲基氨酸转化素心肌病症 (ATTR-CM) 中表现出有效性. 这包括80岁及以上的老年人,他们显示生活质量改善和生存益处.
科学领域:
- 心脏病学 心脏病学
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
背景情况:
- 塔法米迪斯已获批准用于治疗跨甲基氨基基酸心肌病变 (ATTR-CM).
- 它的有效性最初是在Tafamidis in Transthyretin Cardiomyopathy临床试验 (ATTR-ACT) 中确立的.
研究的目的:
- 在老年患者 (年龄≥80岁) 中对塔法米迪斯疗效进行后期分析.
- 为了比较八十岁患者与ATTR-CM的年轻患者 (<80岁) 的结局.
主要方法:
- 从ATTR-ACT试验及其开放式长期延伸 (LTE) 研究中对80岁以下和80岁以上的患者进行分析.
- 评估有效性终点,包括6分钟步行测试 (6MWT),NT-proBNP水平,堪萨斯城心肌病问卷总结 (KCCQ-OS) 成绩和生存率.
主要成果:
- 在80岁以上的患者中,与安慰剂相比,tafamidis治疗在6MWT,NT-proBNP和KCCQ-OS中显示出显著的益处.
- 在LTE研究中,持续的tafamidis治疗与KCCQ-OS的较小下降以及八十岁的中位生存时间更长的趋势相关.
- 在80岁以下的患者中观察到类似的疗效,功能状况改善,NT-proBNP,KCCQ-OS和住院减少.
结论:
- 塔法米迪斯在治疗年轻 (<80岁) 和老年 (≥80岁) 患者群体中有效治疗ATTR-CM.
- 这项研究支持在广泛的年龄范围内使用tafamidis用于ATTR-CM管理.
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