重组凝血因子VIII研究进展
Advances in Recombinant Coagulation Factor VIII
摘要: 凝血因子VIII (FVIII)替代疗法极大地改善了血友病A患者的护理。80年代初，由于使用非病毒灭活血浆生产的凝血因子浓缩物引起的血源性病毒感染，使得更安全治疗的需求变得至关重要。重组凝血因子VIII (rFVIII)的开发最初是由于需要用浓缩的FVIII治疗血友病A患者而无传播感染风险。最近20年来，rFVIII产品在生产、安全性和止血效果上得到了进一步的提高，完全从生产过程中去除了动物或人类蛋白，从而使患者不会暴露于已知的或正在出现的病原体。病毒灭活的血浆衍生凝血因子VIII (PdFVIII)以及重组产品的产生，彻底改变了患者的治疗方式，即定期进行FVIII替代治疗，以防止出血和由此引起的关节损伤，从而使血友病A患者几乎达到了正常的生活方式和预期寿命。目前血友病A治疗最具有挑战的是FVIII抑制剂并发症的出现以及预防性治疗所需的频繁静脉输注的负担。本文回顾了血友病A治疗的rFVIII产品的主要特征，尤其是标准半衰期和延长半衰期rFVIII产品的结构特点以及上市前临床研究中的疗效和安全性。
Abstract: The coagulation factor replacement therapy has contributed to the improved care of people with hemophilia (HA). Following the blood-borne viral infections in early 1980 caused by using non-virally inactivated pooled plasma, the need for safer treatment became crucial to the hemophilia. The development of recombinant factor VIII (rFVIII) was initially driven by the necessity to treat HA patients with FVIII concentrates without the risk of transmitting infectious agents. Over the last two decades, the safety and efficacy of rFVIII have been further improved by completely removing animal or human proteins from the manufacturing process, so that patients would not be exposed to known or emerging pathogens. The availability for replacement therapy of plasma derived factor VIII (PdFVIII) and rFVIII with virus inactivated, has changed the treatment regimen, that is factor VIII replacement therapy, in order to prevent bleeding and resultant joint damage, ultimately allowing patients to maintain a near normal lifestyle. The major challenges of current treatment of HA are the occurrence of FVIII inhibitors and the burden of frequent intravenous infusions required for prophylactic therapy. We reviewed the main characteristics of rFVIII products for treatment of hemophilia A, especially the characteristics of the standard and extended half-life rFVIII products, as well as the efficacy and safety in premarket clinical studies.
文章引用：马传荣. 重组凝血因子VIII研究进展[J]. 药物资讯, 2021, 10(5): 268-273. https://doi.org/10.12677/PI.2021.105034

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