Federici A B, Budde U, Rand J H
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Department of Internal Medicine and Dermatology, IRCCS Maggiore Hospital and University of Milan, Via Pace 9, 20122 Milano, Italy.
Hamostaseologie. 2004 Feb;24(1):50-5. doi: 10.1267/hamo04010050.
The acquired von Willebrand syndrome (AVWS) is a rare bleeding disorder with laboratory findings similar to those for congenital von Willebrand disease. Unlike the congenital form, AVWS usually occurs in individuals with no personal or family history of bleeding. Large studies on AVWS are not available, diagnosis remains difficult and treatment empirical. Acquired von Willebrand syndrome is especially frequent in lympho- or myeloproliferative disorders. It is associated with solid tumours, immunological and cardiovascular disorders as well as other miscellaneous conditions. Diagnosis of AVWS is based on assays measuring ristocetin cofactor activity or collagen binding, which are usually abnormally low, while factor VIII coagulant activity is some-times within the reference range. FVIII/VWF inhibiting activities are found in only a minority of cases. Bleeding episodes in patients with AVWS are mostly of the mucocutaneous type and can be managed with desmopressin, plasma-derived factor VIII/von Willebrand factor (FVIII/VWF) concentrates and intravenous immunoglobulin. Recombinant activated factor VII can be useful in cases unresponsive to standard therapy. In conclusion, the AVWS, although rare, is certainly underestimated in clinical practice: The actual clinical impact of AVWS should be evaluated by prospective studies. The authors are co-ordinating an updated version of the International Registry on AVWS that will allow data to be entered directly online.
获得性血管性血友病综合征（AVWS）是一种罕见的出血性疾病，其实验室检查结果与先天性血管性血友病相似。与先天性形式不同，AVWS通常发生在没有个人或家族出血史的个体中。目前尚无关于AVWS的大型研究，诊断仍然困难，治疗多为经验性。获得性血管性血友病综合征在淋巴或骨髓增殖性疾病中尤为常见。它与实体瘤、免疫和心血管疾病以及其他杂症有关。AVWS的诊断基于测量瑞斯托霉素辅因子活性或胶原结合的检测，这些检测通常异常低，而因子VIII凝血活性有时在参考范围内。仅在少数病例中发现FVIII/VWF抑制活性。AVWS患者的出血发作大多为黏膜皮肤型，可用去氨加压素、血浆源性因子VIII/血管性血友病因子（FVIII/VWF）浓缩物和静脉注射免疫球蛋白治疗。重组活化因子VII对标准治疗无反应的病例可能有用。总之，AVWS虽然罕见，但在临床实践中肯定被低估了：AVWS的实际临床影响应由前瞻性研究评估。作者正在协调AVWS国际登记册的更新版本，该版本将允许直接在线输入数据。
