Mannucci P M
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.
Blood. 2001 Apr 1;97(7):1915-9. doi: 10.1182/blood.v97.7.1915.
Von Willebrand disease (vWD) is a frequent inherited disorder of hemostasis that affects both sexes. Two abnormalities are characteristic of the disease, which is caused by a deficiency or a defect in the multimeric glycoprotein called von Willebrand factor: low platelet adhesion to injured blood vessels and defective intrinsic coagulation owing to low plasma levels of factor VIII. There are 2 main options available for the treatment of spontaneous bleeding episodes and for bleeding prophylaxis: desmopressin and transfusional therapy with plasma products. Desmopressin is the treatment of choice for most patients with type 1 vWD, who account for approximately 70% to 80% of cases. This nontransfusional hemostatic agent raises endogenous factor VIII and von Willebrand factor 3 to 5 times and thereby corrects both the intrinsic coagulation and the primary hemostasis defects. In patients with the more severe type 3 and in most patients with type 2 disease, desmopressin is ineffective or is contraindicated and it is usually necessary to resort to plasma concentrates containing both factor VIII and von Willebrand factor. Concentrates treated with virucidal methods should be preferred to cryoprecipitate because they are equally effective and are perceived as safer. (Blood. 2001;97:1915-1919)
血管性血友病（vWD）是一种常见的遗传性止血障碍疾病，影响男女两性。该疾病有两个特征性异常，由称为血管性血友病因子的多聚体糖蛋白缺乏或缺陷引起：血小板对受损血管的粘附力降低，以及由于血浆中因子VIII水平低导致内源性凝血缺陷。治疗自发性出血发作和预防出血有两种主要选择：去氨加压素和使用血浆制品的输血治疗。去氨加压素是大多数1型vWD患者的首选治疗方法，1型vWD患者约占病例的70%至80%。这种非输血性止血剂可使内源性因子VIII和血管性血友病因子升高3至5倍，从而纠正内源性凝血和原发性止血缺陷。对于更严重的3型患者和大多数2型疾病患者，去氨加压素无效或禁忌，通常需要使用含有因子VIII和血管性血友病因子的血浆浓缩物。采用灭病毒方法处理的浓缩物应优于冷沉淀，因为它们同样有效且被认为更安全。（《血液》。2001年；97：1915 - 1919）
