Mannucci P M, Giangrande P L
Department of Internal Medicine, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Maggiore Hospital and University of Milan, Italy.
Hematol J. 2000;1(2):72-6. doi: 10.1038/sj.thj.6200006.
In the early 1980s it was recognized that coagulation factor concentrates prepared from plasma pooled from thousands of donors transmitted the hepatitis and the human immunodeficiency viruses. The last 15 years have witnessed the development of methods that, applied during concentrate manufacturing, inactivate viruses escaping the screening procedures. The adoption of these measures has dramatically reduced the risk of transmission of bloodborne infections by plasma-derived coagulation factor concentrates. The production of recombinant factor VIII and IX and their availability for patient treatment epitomize progress in hemophilia care through DNA technology. No transmission of infectious agents has been documented and the small amount of residual animal proteins, particularly in the early products, has not been associated with any clinically significant adverse reaction. There is little doubt that recombinant factor VIII and IX are the products of choice for the treatment of patients with hemophilia, particularly the second-generation products containing no plasma-derived human protein and fewer and fewer animal proteins. However, availability and cost still oblige most physicians to ration recombinant products and prioritize certain patient groups (previously untreated, previously uninfected).
在20世纪80年代早期，人们认识到，由数千名献血者的血浆混合制备的凝血因子浓缩物会传播肝炎病毒和人类免疫缺陷病毒。在过去的15年里，出现了一些方法，这些方法应用于浓缩物制造过程中，可灭活那些逃过筛查程序的病毒。采取这些措施已显著降低了血浆源性凝血因子浓缩物传播血源性感染的风险。重组因子VIII和IX的生产及其用于患者治疗体现了通过DNA技术在血友病治疗方面取得的进展。目前尚无感染因子传播的记录，并且少量残留的动物蛋白，尤其是早期产品中的动物蛋白，并未与任何具有临床意义的不良反应相关联。毫无疑问，重组因子VIII和IX是治疗血友病患者的首选产品，尤其是第二代产品，其不含血浆源性人类蛋白且动物蛋白越来越少。然而，可及性和成本问题仍迫使大多数医生限制使用重组产品，并优先考虑某些患者群体（以前未接受治疗、以前未感染）。
