Van Thillo Quentin, Segers Finn, Brijs Jan, Douven Ulrike, Ramanan Radha, Delforge Michel, Janssens Ann, Hermans Cédric, De Bent Johan, Jacquemin Marc, Vanassche Thomas, Verhamme Peter
Department of Cardiovascular Sciences, and Vascular Medicine and Hemostasis, University Hospitals Leuven, Herestraat 49, Leuven 3000, Belgium.
Center for Molecular and Vascular Biology, KU Leuven, Leuven, Belgium.
Ther Adv Hematol. 2025 Jun 24;16:20406207251347235. doi: 10.1177/20406207251347235. eCollection 2025.
Acquired von Willebrand syndrome (AVWS) is a well-known complication of a monoclonal gammopathy with a potentially severe bleeding tendency. Treatment with von Willebrand factor (VWF)/factor VIII (FVIII) concentrate yields mixed results in controlling the bleeding diathesis, while the use of intravenous immunoglobulins may be effective. However, clear guidelines for the optimal management of AVWS are lacking. Therefore, we retrospectively analyzed the cases of AVWS secondary to monoclonal gammopathy at the University Hospitals of Leuven. We confirm the beneficial effect of intravenous immunoglobulins in IgG-associated AVWS. For IgM-associated AVWS, we observed better results with the administration of VWF/FVIII concentrate or a combination of therapies. Of note, one patient with IgG-associated AVWS did not respond to immunoglobulins and had mutations in the and fibrinogen gamma chain () genes. This report adds additional cases to the literature of this rare cause of acquired bleeding.
获得性血管性血友病综合征（AVWS）是单克隆丙种球蛋白病的一种众所周知的并发症，具有潜在的严重出血倾向。使用血管性血友病因子（VWF）/凝血因子VIII（FVIII）浓缩物治疗在控制出血素质方面效果不一，而静脉注射免疫球蛋白可能有效。然而，目前缺乏关于AVWS最佳管理的明确指南。因此，我们对鲁汶大学医院单克隆丙种球蛋白病继发的AVWS病例进行了回顾性分析。我们证实了静脉注射免疫球蛋白在IgG相关AVWS中的有益作用。对于IgM相关的AVWS，我们观察到给予VWF/FVIII浓缩物或联合治疗效果更好。值得注意的是，一名IgG相关AVWS患者对免疫球蛋白无反应，且其α2和纤维蛋白原γ链（γ）基因存在突变。本报告为这种罕见的获得性出血原因的文献增加了更多病例。
