血友病B替代治疗药物研究现状及进展

Hemophilia B replacement therapy drugs

摘要:

血友病B是一种X染色体连锁的隐性遗传性出血性疾病，患者因体内缺乏凝血因子IX (FIX) 而易发生出血事件，病情严重程度与FIX的缺乏程度相关，严重影响患者的寿命和生存质量。文中通过相关文献资料的查阅和整理，对血友病B替代治疗药物进行归纳和综述，重点阐述上市重组凝血因子IX制品和相关在研药物尤其是长效凝血因子IX的研究进展，为血友病B治疗药物的研发提供基础。

Abstract:

Hemophilia B is an X chromosome linked hereditary hemorrhagic disease, which is caused by the lose function mutation of factor IX (FIX), and significantly affects the patients’ lifespan and life quality. The severity of hemophilia B depends on the FIX level in the plasma. By referring to the relevant literatures, we reviewed and summarized hemophilia B replacement therapies. Specifically, we focus on recombinant factor IX products on the market and those in the pipeline, especially on the long-acting factor IX drugs, to provide the basis for researches of new hemophilia B drugs.