长效重组人凝血因子Ⅷ研究现状及进展

Status and advances of long-acting factor Ⅷ

摘要:

重组凝血因子Ⅷ (rFⅧ) 替代疗法是目前临床上治疗A型血友病最为有效的方法。过去十几年的临床试验已经验证了rFⅧ替代疗法的安全性和有效性，但由于FⅧ的半衰期较短，患者需要隔天或者每周3次进行静脉注射治疗。长期频繁静脉注射治疗不仅给患者带来肉体上的痛苦，还容易产生FⅧ抗体，严重影响治疗效果。以下就目前长效型重组凝血因子Ⅷ的研究进展及存在的主要问题进行了综述。

关键词:长效重组人凝血因子Ⅷ，血友病A，重组蛋白，血管性血友病因子，FⅧ与vWF相互作用

Abstract:

Current treatment for hemophilia A is based on replacement therapy that is the most effective method by using recombinant clotting factor FⅧ (rFⅧ). Although the safety and effectiveness of replacement therapy has been proved by clinical practice for the last decades, FⅧ products are temporally limited because of a short half-life and requiring prophylactic injections frequently for most patients, usually three times per week or every other day. Frequent intravenous injection not only brings physical pain to the patient, but also produces FⅧ antibodies that seriously affect the treatment effect. In this paper, we review the present status, research progress and main problems of the long-acting recombinant factor Ⅷ.

Key words:long-acting clotting factor Ⅷ, hemophilia A, recombinant protein, von Willebrand factor, interaction between FⅧ and vWF