Molecular Diagnostics Centre (Haematology), Manchester Royal Infirmary, Manchester, United Kingdom.
Semin Thromb Hemost. 2011 Jul;37(5):488-94. doi: 10.1055/s-0031-1281033. Epub 2011 Nov 18.
The UK treatment strategy for von Willebrand disease (VWD) is based on consensus guidelines produced by the United Kingdom Haemophilia Centre Doctors' Organization (UKHCDO) relating to the diagnosis and management of VWD. Selection of therapeutic products suitable for treatment of this complex inherited bleeding disorder is based on the observed response. Desmopressin (DDAVP), an analog of vasopressin, is the recommended treatment in individuals who respond to this drug on trial infusion. DDAVP clearly has no effect in type 3 VWD but may have variable clinical effect in individuals with other subtypes or may be contraindicated in some cases. In patients where DDAVP treatment is unsuitable, replacement factor concentrate containing von Willebrand factor (VWF) is the recommended alternative. Relevant concentrates are available for all patients in the United Kingdom, and treatment is administered by a network of 67 hemophilia treatment centers that also provide specialist care for individuals diagnosed with VWD. Patients diagnosed with the condition are registered on a national inherited bleeding disorder database administered by the UKHCDO on behalf of the Department of Health to aid in service planning and commissioning. Genetic testing is employed in the United Kingdom in certain situations, which is also performed in accordance with current UKHCDO guidelines.
英国的血管性血友病（VWD）治疗策略基于英国血友病中心医生组织（UKHCDO）制定的共识指南，涉及 VWD 的诊断和管理。选择适合治疗这种复杂遗传性出血性疾病的治疗产品是基于观察到的反应。去氨加压素（DDAVP）是一种血管加压素类似物，是在试验输注中对该药有反应的个体的推荐治疗药物。DDAVP 在 3 型 VWD 中显然没有效果，但在其他亚型的个体中可能具有不同的临床效果，或者在某些情况下可能被禁忌。对于不适合 DDAVP 治疗的患者，含有血管性血友病因子（VWF）的替代因子浓缩物是推荐的替代物。相关的浓缩物在英国所有患者中均可获得，并且由 67 个血友病治疗中心网络提供治疗，这些中心还为诊断为 VWD 的个体提供专业护理。在 UKHCDO 代表卫生部管理的国家遗传性出血性疾病数据库中，登记有条件诊断的患者，以帮助进行服务规划和委托。英国在某些情况下使用基因检测，这也是根据 UKHCDO 的现行指南进行的。
