针对A型血友病患者的有前景的凝血因子VIII替代策略。

Promising coagulation factor VIII bypassing strategies for patients with haemophilia A.

作者信息

Duan Xunwei, Tang Mingqing, Zhang Junping, Yu Hao, Xu Ruian

机构信息

aSchool of Biomedical Sciences and Institute of Molecular Medicine, Huaqiao University bEngineering Research Center of Molecular Medicine, Ministry of Education, Fujian, PR China.

出版信息

Blood Coagul Fibrinolysis. 2014 Sep;25(6):539-52. doi: 10.1097/MBC.0000000000000098.

DOI:10.1097/MBC.0000000000000098

PMID:24614429

Abstract

Haemophilia A is an X-linked recessive monogenic hereditary bleeding disorder caused by a deficiency or functional defect in coagulation factor VIII (FVIII). Typically, only 30% haemophilia A patients are treated with FVIII-specific products successfully. Therefore, other promising clotting factors and FVIII-bypassing factors exhibiting sufficient FVIII-independent activity, low immunogenicity and prolonged half-life are needed to conquer this malady. Here, we will systematically review the current status of the diverse FVIII-bypassing factors for the treatment of FVIII-insensitive haemophilia A patients.

摘要

甲型血友病是一种X连锁隐性单基因遗传性出血性疾病，由凝血因子VIII（FVIII）缺乏或功能缺陷引起。通常，只有30%的甲型血友病患者成功接受了FVIII特异性产品治疗。因此，需要其他有前景的凝血因子和FVIII旁路因子，它们具有足够的不依赖FVIII的活性、低免疫原性和延长的半衰期，以攻克这种疾病。在此，我们将系统回顾用于治疗对FVIII不敏感的甲型血友病患者的各种FVIII旁路因子的现状。