Rodeghiero F, Castaman G, Meyer D, Mannucci P M
Department of Hematology, San Bortolo Hospital, Vicenza, Italy.
Vox Sang. 1992;62(4):193-9. doi: 10.1111/j.1423-0410.1992.tb01198.x.
In von Willebrand disease, the goal of treatment is to correct the two laboratory hallmarks of abnormal hemostasis, i.e. the deficiency of factor VIII (FVIII) and the prolonged bleeding time (BT). Since desmopressin (DDAVP) is able to achieve both these goals in the majority of patients, it is the treatment of choice. Some patients, however, are not responsive or become refractory to DDAVP. In these, blood products maintain an important therapeutic role, and there is a need to assess the efficacy of the recently available virus-inactivated plasma concentrates, which contain both FVIII and von Willebrand factor and carry a low risk of transmitting blood-borne viruses. Our survey of the data reported in the literature indicates that all available concentrates are similarly effective in attaining high and sustained levels of FVIII after infusion. Although they often shorten or normalize the prolonged BT, that effect is less uniform. Since concentrates appear efficacious in the majority of clinical situations that require the use of blood products, they should be preferred, because of their greater safety, to cryoprecipitate produced by blood banks, which cannot be virus inactivated.
在血管性血友病中，治疗的目标是纠正异常止血的两个实验室特征，即因子VIII（FVIII）缺乏和出血时间（BT）延长。由于去氨加压素（DDAVP）能够在大多数患者中实现这两个目标，因此它是首选治疗方法。然而，一些患者对DDAVP无反应或变得难治。在这些患者中，血液制品仍起着重要的治疗作用，因此有必要评估最近可用的病毒灭活血浆浓缩物的疗效，这些浓缩物同时含有FVIII和血管性血友病因子，且传播血源病毒的风险较低。我们对文献报道数据的调查表明，所有可用的浓缩物在输注后达到高且持续的FVIII水平方面同样有效。尽管它们通常会缩短延长的BT或使其正常化，但这种效果不太一致。由于浓缩物在大多数需要使用血液制品的临床情况下似乎有效，而且因其更高的安全性，应优先于血库生产的无法进行病毒灭活的冷沉淀。
