重组猪序列因子VIII在获得的血友病A中的真实世界的安全性和有效性:一个非干预性,授权后的安全性研究
Michael D Tarantino1, Brandon Hardesty2, Ara Metjian3
1Bleeding and Clotting Disorders Institute, Peoria, Illinois, USA.
概括
重组猪因子VIII (rpFVIII) 在现实环境中有效治疗了成年人获得性血友病A (AHA) 80%的出血发作. 没有发现新的安全问题,与先前的临床试验数据保持一致.
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.
- 临床研究 临床研究
背景情况:
- 重组猪因子VIII (rpFVIII,susoctocog alfa) 是一种已批准的治疗方法,用于治疗获得性血友病A (AHA) 的成年人的出血发作.
- 现实世界的数据对于了解rpFVIII在管理AHA中的长期安全性和有效性至关重要.
研究的目的:
- 评估rpFVIII在获得血友病A的成年患者的长期安全性和有效性.
- 在现实环境中评估与治疗相关的严重不良事件 (SAE) 和rpFVIII的静血有效性.
主要方法:
- 美国PASS研究 (NCT02610127) 是一项多中心的,不受控制的,开放的,营销后安全监测研究.
- 接受rpFVIII治疗的AHA成年患者被纳入,数据被追溯或前性收集了180天.
- 主要结局:与治疗相关的SAE发生率;次要结局:静血效率和rpFVIII利用率.
主要成果:
- 招募了53名患者;12.0%的患者经历了与治疗相关的SAE,最常见的是FVIII抑制 (8.0%).
- 七个血栓栓塞事件中的一个是可能与rpFVIII相关的SAE.
- rpFVIII解决了80.3%的治疗出血发作,每次输注的中位数剂量为50单位/公斤.
结论:
- 这项现实研究在AHA患者中没有发现rpFVIII的新安全信号,这与之前的临床试验结果一致.
- rpFVIII显示出显著的静血效果,解决了80%的出血发作.
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