没有抑制剂的血友病A/B中的Marstacimab预防:来自第3期BASIS试验的结果
Davide Matino1, Andrew Palladino2, Carrie Turich Taylor3
1McMaster University, Hamilton, Ontario, Canada.
Blood
|July 3, 2025
概括
与标准疗法相比,马斯塔西马布显著减少了血友病A和B患者的出血. 这种新型抗体治疗在第三阶段试验中证明了安全性和有效性.
科学领域:
- 血液学 血液学 血液学
- 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:
- 血友病A和B是遗传性出血障碍,需要因子替代疗法.
- 目前的治疗包括按需 (OD) 或常规预防 (RP) 的因子替代.
- 马斯塔西马布是一种单克隆抗体向组织因子通路抑制剂,用于重新平衡血静.
研究的目的:
- 评估马斯塔西马布在患有血友病A或B的患者中预防的疗效和安全性.
- 为了比较接受马斯塔西马布治疗的患者的出血率与之前的OD或RP治疗.
主要方法:
- 第3期,开放标签BASIS试验涉及12-74岁的男性,患有严重的血友病A或B,没有抑制剂.
- 参与者接受了6个月的OD或RP观察阶段 (OP),随后是12个月的活性治疗阶段 (ATP),每周进行皮下马斯塔西马布.
- 主要终点:年均出血率 (ABR) 和安全性.
主要成果:
- 马斯塔西马布在OD组显著降低了ABR (39.86至3.20,P<.0001),并在RP组中显示出非劣势性和优势性 (7.90至5.09,P=.0349).
- 没有报告死亡或血栓塞栓事件.
- 马斯塔西马布是安全的,耐受性很好,没有意外的副作用.
结论:
- 在没有抑制剂的血友病A和B患者中,每周皮下马斯塔西马布有效减少出血.
- 马斯塔西马布为传统的因子替代疗法提供了一种安全且耐受良好的替代方案.
- 这项研究支持马斯塔西马布作为血友病管理的新预防方案.
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