血友病B替代治疗药物研究现状及进展  

Hemophilia B replacement therapy drugs

作　　者：严红[1,2] 曾凡一[1,2] 

机构地区：[1]上海市儿童医院上海交通大学附属儿童医院上海医学遗传研究所,上海200040 [2]卫生部医学胚胎分子生物学重点实验室暨上海市胚胎与生殖工程重点实验室,上海200040

出　　处：《生物工程学报》2016年第2期164-171,共8页Chinese Journal of Biotechnology

基　　金：国家自然科学基金(No.81500108)资助~~

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。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.

关 键 词：血友病B 凝血因子IX替代治疗药物 长效凝血因子IX 

分 类 号：R554.1[医药卫生—血液循环系统疾病]