van Aken W G
Central Laboratory of The Netherlands Red Cross Blood Transfusion Service, Amsterdam, The Netherlands.
Transfus Sci. 1998 Mar;19(1):9-15. doi: 10.1016/s0955-3886(98)00004-6.
Recombinant clotting factor concentrates provide new opportunities for the treatment of hemostatic disorders such as haemophilia A and B and von Willebrand disease. This article briefly reviews the clinical results of currently available products, the development of concentrates of clotting factor IX and von Willebrand factor and the outlook of second generation products (e.g., deletion mutants) and gene therapy of haemophilia. Further elucidation of the structure/function relationship of plasma proteins will probably lead to a new category of medicinal products for the treatment of bleeding and thrombotic diseases.
重组凝血因子浓缩剂为治疗诸如甲型和乙型血友病以及血管性血友病等止血障碍提供了新的机会。本文简要回顾了现有产品的临床结果、凝血因子IX和血管性血友病因子浓缩剂的发展情况以及第二代产品（如缺失突变体）和血友病基因治疗的前景。对血浆蛋白结构/功能关系的进一步阐明可能会催生一类用于治疗出血性和血栓性疾病的新型药物产品。
