Coppes M J, Zandvoort S W, Sparling C R, Poon A O, Weitzman S, Blanchette V S
Department of Paediatrics, Hospital for Sick Children, Toronto, Canada.
J Clin Oncol. 1992 Mar;10(3):422-7. doi: 10.1200/JCO.1992.10.3.422.
A prospective study was performed to determine the incidence of acquired von Willebrand disease (vWD) in children with newly diagnosed Wilms' tumor.
Fifty consecutive children with newly diagnosed Wilms' tumor were evaluated. Detailed family and bleeding histories were obtained in all cases. Laboratory evaluation included measurement of the circulating platelet count, bleeding time (BT), factor VIII (FVIII) and von Willebrand factor (vWF) levels, and ristocetin cofactor (RCoF) activity. A vWF multimer analysis was obtained in all cases in which vWD was suspected.
Four of 50 (8%) consecutive children with a diagnosis of Wilms' tumor were found to have acquired vWD. Laboratory findings indicated type III vWD in two patients and type I vWD in the other two.
The incidence of acquired vWD in association with Wilms' tumor merits further study through a large prospective trial. Such a trial should include careful family and clinical bleeding histories plus measurement of a platelet count, BT, coagulant FVIII and vWF levels, RCoF activity, and vWF multimer analysis. The response to 1-desamino-8-D-arginine vasopressin (DDAVP) should be tested in all patients with Wilms' tumor and acquired vWD, including patients with a type III profile, before an invasive procedure is performed. Successful use of DDAVP may avoid exposure of affected patients to blood products.
进行一项前瞻性研究以确定新诊断的肾母细胞瘤患儿获得性血管性血友病（vWD）的发病率。
对50例新诊断的肾母细胞瘤患儿进行评估。所有病例均详细询问家族史和出血史。实验室评估包括循环血小板计数、出血时间（BT）、凝血因子VIII（FVIII）和血管性血友病因子（vWF）水平以及瑞斯托霉素辅因子（RCoF）活性的测定。所有怀疑患有vWD的病例均进行vWF多聚体分析。
50例诊断为肾母细胞瘤的连续患儿中有4例（8%）被发现患有获得性vWD。实验室检查结果显示，2例患者为III型vWD，另外2例为I型vWD。
与肾母细胞瘤相关的获得性vWD的发病率值得通过大型前瞻性试验进一步研究。这样的试验应包括仔细询问家族和临床出血史，以及血小板计数、BT、凝血因子FVIII和vWF水平、RCoF活性的测定和vWF多聚体分析。在进行侵入性操作之前，应对所有患有肾母细胞瘤和获得性vWD的患者，包括III型患者，进行1-去氨基-8-D-精氨酸血管加压素（DDAVP）反应测试。成功使用DDAVP可能避免受影响患者接触血液制品。
