Mannucci P M
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Maggiore Hospital, University of Milano, Italy.
J Intern Med Suppl. 1997;740:129-32.
von Willebrand's disease is the most frequent of inherited bleeding disorders (1:100 affected individuals in the general population). The aim of therapy is to correct the dual defects of haemostasis, i.e. abnormal coagulation expressed by low levels of factor VIII and abnormal platelet adhesion expressed by a prolonged bleeding time. There are two main options available for the management of von Willebrand's disease: desmopressin and transfusion therapy with blood products. Desmopressin is the treatment of choice in patients with Type 1 von Willebrand's disease, who account for approximately 80% of cases. The pharmacological compound raises endogenous factor VIII and von Willebrand factor and corrects the prolonged bleeding time in most patients. In Type 3 and in the majority of Type 2 patients, desmopressin is not effective and it is necessary to resort to plasma concentrates containing factor VIII and von Willebrand factor. Treated with virucidal methods, these concentrates are currently effective and quite safe, even though the bleeding time defect is not always corrected by them. Platelet concentrates or desmopressin can be used as adjunctive treatments when poor correction of the bleeding time is associated with continued bleeding.
血管性血友病是最常见的遗传性出血性疾病(普通人群中每100人中有1人患病)。治疗的目的是纠正止血的双重缺陷,即由低水平的凝血因子VIII所表现出的异常凝血,以及由延长的出血时间所表现出的异常血小板黏附。治疗血管性血友病主要有两种选择:去氨加压素和血液制品输血治疗。去氨加压素是1型血管性血友病患者的首选治疗方法,这类患者约占病例总数的80%。这种药物化合物可提高内源性凝血因子VIII和血管性血友病因子,并纠正大多数患者延长的出血时间。在3型以及大多数2型患者中,去氨加压素无效,必须使用含有凝血因子VIII和血管性血友病因子的血浆浓缩物。经过灭病毒处理后,这些浓缩物目前有效且相当安全,尽管它们并不总能纠正出血时间缺陷。当出血时间纠正不佳且伴有持续出血时,血小板浓缩物或去氨加压素可作为辅助治疗手段。