从血衍生物转换为重组因子VIII在以前未经治疗的严重血友病A患者中:PUP-SWITCH研究:在从血衍生物转换为重组因子VIII后,抑制剂的发展
Syna Miri1, Frits R Rosendaal2, Kaan Kavakli3
1Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
Research and practice in thrombosis and haemostasis
|December 9, 2024
概括
在50个暴露日 (EDs) 后,从血衍生因子VIII (pdFVIII) 切换到重组因子VIII (rFVIII) 的严重血友病A患者 (PUP) 似乎是安全的. 在PUP-SWITCH研究中发现,在这种治疗转换后,新抑制剂的发病率较低.
科学领域:
- 血液学 血液学 血液学
- 临床试验 临床试验
- 免疫学 免疫学 免疫学
背景情况:
- SIPPET试验表明,在以前未接受治疗的严重血友病A患者 (PUP) 中,在50个暴露日内使用血衍生因子VIII (pdFVIII) 与重组因子VIII (rFVIII) 相比,抑制剂较少.
- 许多中心在50次ED后将PUP从pdFVIII切换到rFVIII,这引发了关于切换后潜在的抑制剂开发风险的问题.
研究的目的:
- 调查50个ED后从pdFVIII切换到rFVIII的严重血友病A的PUP中新抑制剂发展的发生率.
- 在这个患者群体中评估转换因子VIII产品的安全性.
主要方法:
- 在PUP-SWITCH观察后期研究中,评估了从pdFVIII转换为rFVIII的PUP在50到150个ED之间.
- 参与者在转换后被跟踪至少50个EDs.
- 从经常执行此类切换的血友病中心收集数据.
主要成果:
- 97名患者被评估,其中87人符合资格标准.
- 只有一个患者在切换后20个ED开发了抑制剂.
- 新抑制剂的累计发病率为1.15% (95% CI,0.03%-6.24%).
结论:
- PUP-SWITCH研究表明,在患有严重血友病A的PUP患者中,在50次ED后从pdFVIII切换到rFVIII是安全的,这与新的抑制剂的发展有关.
- 研究结果表明,这种产品类别转换策略似乎不会增加抑制剂风险.
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