在血友病患者中发展因子VIII和因子IX抑制剂的发生率
S Ehrenforth1, W Kreuz, I Scharrer
1Department of Haematology and Oncology, J. W. Goethe University Hospital, Frankfurt am Main, Germany.
Lancet (London, England)
|March 7, 1992
概括
发育VIII因子抑制剂是血友病A患者的严重并发症. 这项前性研究发现,风险比以前报告的要高,特别是在严重的病例中.
科学领域:
- 血液学 血液学 血液学
- 免疫学 免疫学 免疫学
- 遗传学 是一个遗传学.
背景情况:
- 第八因子 (FVIII) 抑制剂是 A 血友病治疗中的重要并发症.
- 以前的风险评估依赖于追溯数据,可能低估了真正的发病率.
研究的目的:
- 为了前性地评估在血友病患者中发展FVIII抑制剂的发病率和风险因素.
- 与之前的回顾性研究相比,提供更准确的风险评估.
主要方法:
- 在1976年开始的一项前性研究中,纳入了1970年以后出生的血友病A和B患者,FVIII/FIX活性≤5%,接受了替代疗法.
- 包括63名患有血友病A型的儿童和17名患有血友病B型的儿童,符合招生标准.
- 随着时间的推移,监测抑制剂的发展.
主要成果:
- 在24%的血友病A患者 (15/63) 和52% (14/27) 患有严重血友病A患者中,抑制剂发生.
- 发病率为每1000个患者年39.1;所有抑制剂都在5.2岁时被检测出来.
- 在A型血友病患者中,到6岁时,累积风险达到33%.
结论:
- 以前对血友病A患者FVIII抑制剂风险的估计可能被低估.
- 展望性,标准化研究,特别是在儿科患者群体中,对于准确的风险评估至关重要.
- 鉴于6岁时累积风险很高,早期检测和管理策略至关重要.
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