Berntorp Erik
Malmö Centre for Thrombosis and Hemostasis, Malmö University Hospital, Malmö, Sweden.
Semin Thromb Hemost. 2006 Sep;32(6):621-5. doi: 10.1055/s-2006-949667.
Treatment of type 3 von Willebrand disease (vWD) relies on infusion with plasma-derived factor concentrates containing von Willebrand factor (vWF). Patients with types 1 and 2 vWD who do not respond satisfactorily after receiving desmopressin need treatment with concentrates. The rationale for long-term prophylaxis in vWD is obvious: prophylaxis has been successfully used in hemophilia, and joint hemorrhages with development of hemophilic arthropathy can occur, especially in type 3 vWD. In Sweden, prophylaxis for vWD began during the 1960s, and we now have experience from a cohort of 37 patients treated for a median of 11 years (range, 2 to 45 years). The majority of subjects (n = 28) have type 3 vWD. The mean dose used for treatment is 24 units factor VIII/kg body weight given one to three times weekly. Indications for prophylaxis have included joint bleeds, bleeds from nose and mouth, menorrhagia and gastrointestinal bleeds. The annual number of bleeds has decreased dramatically following onset of prophylaxis. We conclude that long-term prophylactic treatment of vWD is warranted in the majority of cases with type 3 and in some cases, depending on the clinical phenotype, for patients with other subtypes. Additional studies are ongoing in an international effort, the von Willebrand Disease Prophylaxis Network.
3型血管性血友病（vWD）的治疗依赖于输注含血管性血友病因子（vWF）的血浆源性凝血因子浓缩物。1型和2型vWD患者在接受去氨加压素治疗后若效果不佳，则需要使用凝血因子浓缩物进行治疗。对vWD进行长期预防的理由很明显：预防措施已在血友病治疗中成功应用，而且vWD患者尤其是3型患者可能会出现关节出血并发展为血友病性关节病。在瑞典，vWD的预防始于20世纪60年代，我们现在有一组37例患者的治疗经验，这些患者的中位治疗时间为11年（范围为2至45年）。大多数受试者（n = 28）为3型vWD。治疗所用的平均剂量为每千克体重24单位凝血因子VIII，每周给药1至3次。预防的指征包括关节出血、鼻口出血、月经过多和胃肠道出血。预防开始后，每年的出血次数大幅减少。我们得出结论，对于大多数3型患者以及某些取决于临床表型的其他亚型患者，vWD的长期预防性治疗是必要的。一项国际合作项目——血管性血友病预防网络正在进行更多研究。
