Department of Medicine, Queen’s University, Kingston, ON, Canada.
Blood. 2013 Aug 1;122(5):636-40. doi: 10.1182/blood-2012-10-462085. Epub 2013 Jan 7.
The development of alloantibodies against von Willebrand factor (VWF) represents a rare but serious complication of treatment of von Willebrand disease (VWD), occurring in ~5% to 10% of type 3 VWD patients. Affected patients can present with a range of symptoms, including lack or loss of hemostatic response to infused VWF concentrates up to anaphylactic reactions in rare cases. It is classically reported in multitransfused patients and occurs most frequently in patients with partial or complete VWF gene deletions. A positive family history of anti-VWF antibodies also appears to be a risk factor. There is a lack of standardization of laboratory methods for antibody identification and characterization. Issues of variability in laboratory approaches as well as the rarity of the complication act as a barrier to future studies. Recombinant factor VIII as well as bypassing agents and immune tolerance have been reported as effective treatments; however, aside from case reports, little exists in the literature to guide management. The imminent clinical availability of recombinant VWF has prompted a resurgence of interest in this area. Additional study is warranted to address the deficiencies in our understanding of this treatment complication.
针对血管性血友病因子 (VWF) 的同种抗体的产生，代表了血管性血友病 (VWD) 治疗中一种罕见但严重的并发症，在 3 型 VWD 患者中发生率约为 5%至 10%。受影响的患者可能会出现一系列症状，包括缺乏或丧失对输注的 VWF 浓缩物的止血反应，在极少数情况下甚至会发生过敏反应。这种情况在多次输血的患者中经典报道，并且最常发生在 VWF 基因部分或完全缺失的患者中。抗 VWF 抗体的阳性家族史似乎也是一个危险因素。针对抗体鉴定和特征描述的实验室方法缺乏标准化。实验室方法的可变性问题以及并发症的罕见性，成为未来研究的障碍。重组因子 VIII 以及旁路制剂和免疫耐受已被报道为有效治疗方法；然而，除了病例报告外，文献中几乎没有什么内容可以指导管理。重组 VWF 即将上市，这促使人们对该领域重新产生了兴趣。需要进一步的研究来解决我们对这种治疗并发症理解的不足。
