A型血友病替代治疗药物凝血因子Ⅷ的临床研究进展  

Advances of clinical research on alternative therapy for hemophilia A,coagulation factor Ⅷ

作　　者：兰志龙 陈春宇 赵丹丹 张梦 王俊龙 倪娜 LAN Zhilong;CHEN Chunyu;ZHAO Dandan;ZHANG Meng;WANG Junlong;NI Na(Clinical Research Center,China State Institute of Pharmaceutical Industry,Shanghai 200437,China;Shanghai InnoClinic Pharmaceutical Developmeng Co.,Ltd.,Shanghai 200437,China)

机构地区：[1]中国医药工业研究总院药物临床研究中心,上海200437 [2]上海益临思医药开发有限公司,上海200437

出　　处：《世界临床药物》2024年第4期368-371,共4页World Clinical Drug

摘　　要：A型血友病(hemophilia A,HA)是一种罕见的X染色体连锁隐性遗传性出血性疾病,因血液中凝血因子Ⅷ(factor Ⅷ,FⅧ)缺乏或者功能障碍所致。其发病率低,治疗费用高,且目前暂无有效的根治方法,按需替代治疗仍然是我国治疗HA最有效的方式。但目前HA患者需要终身用药,现有的药物半衰期短、注射频次高,易产生FⅧ抑制物,且患者的生存质量差。针对这种未被满足的临床需求,该文主要综述血源性人FⅧ、重组人FⅧ及长效重组人FⅧ-Fc融合蛋白3种替代治疗药物的临床研究进展,为后期药物研发及临床治疗提供新的思路和依据。Hemophilia A(HA)is a rare X-chromosome linked recessive hemorrhagic disorder caused by the deficiency or dysfunction of coagulation factor Ⅷ(FⅧ)in the blood.The incidence of HA is low,the cost of treatment is high,and there is no effective radical treatment at present.On-demand alternative treatment is still the most effective way to treat HA in our country.However,HA patients need lifelong medication,the existing drugs have short half-life,high injection frequency,easy to produce coagulation FⅧ inhibitors,and poor quality of life of patients.In view of such unmet clinical needs,this paper mainly reviewed the clinical research progress of three alternative therapeutic drugs:plasmaderived human coagulation FⅧ,recombinant human coagulation FⅧ and long-acting recombinant human coagulation FⅧ Fc fusion protein,to provide new ideas and basis for later drug development and clinical treatment.

关 键 词：A型血友病 替代治疗 血源性人凝血因子Ⅷ 重组人凝血因子Ⅷ 长效重组人凝血因子Ⅷ-Fc融合蛋白 

分 类 号：R979.1[医药卫生—药品]