与抑制剂 (ATLAS-INH) 配合的血友病A或血友病B患者的菲图西兰预防的有效性和安全性:多中心,开放,随机的第3期试验
在PubMed上查看摘要
概括
此摘要是机器生成的。在使用抑制剂的血友病A或B患者中,菲图西兰预防显著减少出血,三分之二的患者没有出血. 这种正在研究的疗法有望改善血友病的治疗.
科学领域
- 血液静止和血栓形成
- 有关RNA疗法
- 药理学
背景情况
- 血友症A和B是遗传性出血疾病.
- 抑制剂会使治疗复杂化.
- 目前的治疗方法往往涉及到具有不同功效的绕过药物.
研究的目的
- 在使用抑制剂的血友病A或B患者中评估菲图西兰预防的疗效和安全性.
- 为了比较fitusiran的预防与按需的绕道剂.
主要方法
- 第三阶段,多中心,随机,开放的研究.
- 57名患有严重血友病A或B的参与者被分为2:1的Fitusiran或绕道药物.
- 主要终点:9个月的年均出血率.
主要成果
- 与绕行药物相比,菲图西兰预防降低了每年出血率90. 8% (1. 7与18. 1相比,p< 0. 0001).
- 66% 的受试者没有经过治疗的出血,而 5% 的受试者使用了绕道剂.
- 氨酸转移酶增加是最常见的不良反应;与菲图西兰一起发生了两次血栓塞栓事件.
结论
- 使用抑制剂的皮下菲图西兰预防可显著降低血友症A或B的出血.
- 菲图西兰显示出血液静止的潜力,并可能改善血友病的治疗模式.
- 进一步的研究可能会证实fitusiran是一种有价值的治疗选择.
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