血友病
医学
抗凝血酶
遗传增强
血友病B
抗体
血友病A
因子IX
重症监护医学
双特异性抗体
外科
免疫学
内科学
基因
肝素
单克隆抗体
生物
遗传学
作者
Gavin Ling,Amit C. Nathwani,Edward G. D. Tuddenham
摘要
Summary Haemophilia therapy has undergone very rapid evolution in the last 10 years. The major limitation of current replacement therapy is the short half‐life of factors VIII and IX . These half‐lives have been extended by the addition of various moieties, allowing less frequent infusion regimens. Entirely novel approaches have also entered the clinic, including a bispecific antibody that mimics factor VIII and strategies that rebalance the haemostatic mechanism by reducing antithrombin through inhibition of synthesis. These two treatments are available by subcutaneous injection at infrequent intervals and both can be used in patients with neutralising antibodies (inhibitors). Finally, a cure may be on the horizon with preliminary evidence of success for gene therapy in haemophilia B and A.
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