Haemophilia Centre, Medical Clinic II, Institute of Transfusion Medicine, Goethe University Hospital, Frankfurt am Main, Germany.
Department of Coagulation Disorders, Lund University, University Hospital, Malmo, Sweden.
Thromb Res. 2021 Mar;199:67-74. doi: 10.1016/j.thromres.2020.12.030. Epub 2021 Jan 6.
There is limited awareness of von Willebrand disease (VWD), leading to challenges in both diagnosis and defining the optimal treatment approach for these patients. Patients with VWD are typically treated on-demand, with short-term prophylaxis used during surgery. In contrast, early initiation, and long-term use of prophylaxis is the standard of care in patients with severe haemophilia and can be successfully used to prevent joint bleeding and reduce chronic arthropathy.
To provide an understanding of the current evidence for the prophylactic treatment of patients with VWD and compare this to the management of patients with haemophilia.
Review of published literature using a non-systematic search of PubMed and reference lists of sourced articles.
The successes seen with prophylaxis in haemophilia provide the rationale for long-term prophylaxis in patients with severe forms of VWD; preventing spontaneous, excessive and sometimes life-threatening bleeding, and reducing chronic joint disease. Currently, there are a few clinical trials assessing the long-term benefits of prophylaxis in VWD, and guidelines for the optimal prophylaxis treatment approach are lacking. Greater attempts to provide comprehensive, long-term care for patients with VWD are needed but still lacking within the community. This review highlights the success of prophylaxis in haemophilia and how this knowledge might be applied and translated to patients with VWD.
Lessons can be learned from the use of prophylaxis in haemophilia and prophylaxis should be considered the standard of care for a subgroup of patients with severe VWD.
由于对血管性血友病（VWD）的认识有限，导致在诊断和确定这些患者的最佳治疗方法方面存在挑战。VWD 患者通常按需治疗，在手术期间短期使用预防措施。相比之下，在严重血友病患者中，早期开始和长期使用预防措施是标准的治疗方法，可以成功预防关节出血和减少慢性关节病。
了解目前关于 VWD 患者预防性治疗的证据，并将其与血友病患者的管理进行比较。
使用非系统性搜索 PubMed 和来源文章的参考文献列表，对已发表的文献进行回顾。
血友病患者预防治疗取得的成功为严重 VWD 患者长期预防治疗提供了依据；预防自发性、过度和有时危及生命的出血，并减少慢性关节疾病。目前，有一些临床试验评估了 VWD 患者长期预防治疗的益处，缺乏最佳预防治疗方法的指南。需要但尚未在社区中为 VWD 患者提供更全面、长期的护理。这篇综述强调了预防治疗在血友病中的成功，以及如何将这些知识应用和转化为 VWD 患者。
可以从血友病中预防治疗的应用中吸取经验教训，预防治疗应该被视为严重 VWD 患者亚组的标准治疗方法。
