Djambas Khayat Claudia, Pavlova Anna, Werner Sylvia, Knaub Sigurd, Sidonio Robert F
Hotel Dieu de France Hospital, Saint Joseph University, Beirut, Lebanon.
Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany.
TH Open. 2025 Jun 6;9:a26069625. doi: 10.1055/a-2606-9625. eCollection 2025.
The development of inhibitors to von Willebrand factor (VWF) is a rare but potentially serious complication of VWF replacement therapy in patients with von Willebrand disease (VWD). Patients who develop VWF inhibitors may become unresponsive and/or may develop severe anaphylactic reactions to VWF concentrates. Data on inhibitor development and management in VWD remain limited, and better understanding of inhibitor development is an important goal in VWD management. The WIL-31 study demonstrated the efficacy and safety of prophylaxis with wilate, a plasma-derived VWF/factor VIII (pdVWF/FVIII) concentrate, in children and adults with VWD of all types. The annualized bleeding rate (ABR) was reduced by 84% with wilate prophylaxis compared with on-demand treatment, and prophylaxis was well tolerated. No inhibitors developed during the WIL-31 study. Here, we report two brothers with type 3 VWD who at the 6-month visit were found to have VWF inhibitors, which on further investigation were found to have already been present before the study. Despite the presence of inhibitors, neither patient showed any clinical symptoms, and prophylaxis with wilate led to a ≥85% reduction in ABR in both boys compared with on-demand treatment.
血管性血友病因子（VWF）抑制剂的产生是血管性血友病（VWD）患者接受VWF替代治疗时罕见但可能严重的并发症。出现VWF抑制剂的患者可能对VWF浓缩物无反应和/或出现严重过敏反应。关于VWD中抑制剂产生及管理的数据仍然有限，更好地了解抑制剂的产生是VWD管理中的一个重要目标。WIL-31研究证明了威尔特（一种血浆源性VWF/凝血因子VIII（pdVWF/FVIII）浓缩物）对各类型VWD儿童和成人进行预防的有效性和安全性。与按需治疗相比，威尔特预防使年化出血率（ABR）降低了84%，且预防耐受性良好。在WIL-31研究期间未产生抑制剂。在此，我们报告两名3型VWD兄弟，在6个月随访时发现他们有VWF抑制剂，进一步调查发现这些抑制剂在研究之前就已存在。尽管存在抑制剂，但两名患者均未表现出任何临床症状与按需治疗相比，威尔特预防使两个男孩的ABR均降低了≥85%。
