Josephson Cassandra D, Abshire Thomas C
Division of Hematology/Oncology/Bone Marrow Transplant, Pathology, Laboratory Medicine, Pediatrics, Emory University School of Medicine, 2015 Uppergate Dr. NE, Atlanta, GA 30322, USA.
Best Pract Res Clin Haematol. 2006;19(1):35-49. doi: 10.1016/j.beha.2005.01.031.
The use of plasma-derived factor products to treat hemophilia A, hemophilia B, and von Willebrand disease (vWD) has changed since the start of the human immunodeficiency virus (HIV) epidemic. The use of plasma-derived factor concentrates for hemophilias A and B has decreased in developed countries because of the availability of recombinant products. However, in developing countries, which encompass most of the world's hemophilia community, plasma-product-based therapy remains the backbone of treatment because of economic constraints. Viral attenuation strategies have resulted in a much safer product profile. vWD product selection is less complicated than for hemophilas A and B because plasma-derived products are the only choice for patients who are unresponsive or who cannot receive pharmacologic therapy. As the majority of patients in the world with hemophilias A, B and vWD are treated with plasma-derived clotting factors, the need for these safe and efficacious therapies will continue in the future. This chapter discusses safety strategies for plasma-derived clotting factor, its availability, economics, efficacy, and inhibitor formation.
自人类免疫缺陷病毒（HIV）流行开始以来，用于治疗甲型血友病、乙型血友病和血管性血友病（vWD）的血浆源性因子产品的使用情况发生了变化。在发达国家，由于重组产品的出现，用于甲型和乙型血友病的血浆源性因子浓缩物的使用有所减少。然而，在占全球血友病群体大多数的发展中国家，基于血浆产品的治疗仍然是治疗的支柱，因为经济限制。病毒灭活策略已使产品安全性大大提高。与甲型和乙型血友病相比，血管性血友病的产品选择没那么复杂，因为血浆源性产品是无反应或无法接受药物治疗患者的唯一选择。由于世界上大多数甲型、乙型血友病和血管性血友病患者都接受血浆源性凝血因子治疗，未来对这些安全有效疗法的需求仍将持续。本章讨论血浆源性凝血因子的安全策略、其可得性、经济性、疗效和抑制剂形成。
