Department of Clinical Chemistry, Thrombosis and Hemostasis Unit, University Hospital of Kiel and Lübeck, Kiel, Germany.
Medical Clinic II, Institute of Transfusion Medicine, Goethe University, Frankfurt, Germany.
Hamostaseologie. 2019 Nov;39(4):326-338. doi: 10.1055/s-0039-1692688. Epub 2019 Jul 30.
In patients with von Willebrand disease (VWD), replacement therapy may be indicated in the case of spontaneous bleeding, surgical interventions and injuries/trauma or as a prophylaxis of spontaneous bleeding episodes. The deficient von Willebrand factor (VWF) is replaced with or without factor VIII (FVIII). Dual VWF/FVIII concentrates can be beneficial in the case of low FVIII level, while repeated dosing may lead to very high FVIII levels, with a potential thrombogenic effect in individual VWD patients. An excessive FVIII:C increase can be limited by using a VWF product with a low level of FVIII, achieving a haemostatic adequate FVIII:C increase after 6 to 12 hours. Replacement therapy in patients with VWD shall be individualised considering VWD type, history and risk of bleeding and risk of thrombosis, as well as indication and the individually variable VWF and FVIII increase. Deviations from the dosages and minimum trough levels mentioned in guidelines or recommendations can be considered in justified cases. The objective of this review is to provide recommendations for specific constellations of replacement therapy based on the VWD-specific guidelines available in Europe, the available evidence, own experiences and the consensus of the interdisciplinary German author group.
在血管性血友病（VWD）患者中，在自发性出血、手术干预和损伤/创伤的情况下，或者作为自发性出血发作的预防措施，可能需要进行替代治疗。缺乏的血管性血友病因子（VWF）可以与或不与因子 VIII（FVIII）一起替代。双重 VWF/FVIII 浓缩物在 FVIII 水平低的情况下可能有益，而重复给药可能导致 FVIII 水平非常高，在个别 VWD 患者中具有潜在的血栓形成作用。通过使用 FVIII 水平低的 VWF 产品，可以限制 FVIII:C 的过度增加，在 6 至 12 小时后达到止血所需的足够 FVIII:C 增加。应根据 VWD 类型、出血和血栓形成风险、适应症以及个体差异的 VWF 和 FVIII 增加，考虑个体化的替代治疗。在有理由的情况下，可以考虑偏离指南或建议中提到的剂量和最低谷浓度。本综述的目的是根据欧洲现有的 VWD 特异性指南、现有证据、自身经验以及跨学科德国作者组的共识，为特定的替代治疗情况提供建议。
