RRC Thrombosis & Haemophilia Centre, AOU "Città della Salute e della Scienza" - Molinette Hospital, Turin, Italy.
Centre for Bleeding Disorders, Careggi University Hospital, Florence, Italy.
Blood Transfus. 2018 Jul;16(4):371-381. doi: 10.2450/2017.0354-16. Epub 2017 May 26.
Recent advances in the care of von Willebrand's disease (vWD) have allowed the majority of patients to be managed adequately. Even in the more severe forms, it is now possible to control recurrent bleeding through secondary long-term prophylaxis with von Willebrand factor-containing concentrates. Moreover, in the setting of surgical prophylaxis, the combination of interdisciplinary management and close patient monitoring yields a positive outcome in nearly all cases, although safety concerns remain. In clinical practice, the effectiveness of therapy is hindered by the difficulties in making a rapid, yet accurate diagnosis, in identifying the subgroup of bleeders who may benefit most from a specific strategy, and in selecting the optimal product and regimen.Since specific guidelines for heavy bleeders requiring short- and long-term prophylaxis are still lacking, sharing the experience of experts dealing with vWD patients on a daily basis is crucial to fill gaps in information relating to patient management. To address this important issue, 13 Italian haematologists met in Milan on April, 2, 2016 and in Florence on July, 9, 2016. A 30-question survey constituted the input to discuss (i) optimisation of the diagnostic workflow for vWD, (ii) the characteristics of patients who may benefit from secondary long-term prophylaxis (in particular with the purified von Willebrand factor concentrate with a low content of factor VIII), (iii) the key elements to consider when selecting a concentrate and (iv) the pre-operative and post-operative management of vWD patients. A summary of the main points covered is provided in this report.
近年来,对血管性血友病(vWD)的治疗进展使得大多数患者能够得到充分的治疗。即使在更严重的形式中,通过使用富含血管性血友病因子的浓缩物进行二级长期预防治疗,也可以控制反复出血。此外,在手术预防的情况下,通过多学科管理和密切的患者监测相结合,几乎可以在所有情况下取得积极的结果,尽管仍存在安全性问题。在临床实践中,治疗的有效性受到以下因素的阻碍:快速做出准确诊断的困难、确定最可能从特定策略中受益的出血者亚组的困难,以及选择最佳产品和方案的困难。由于仍然缺乏需要短期和长期预防治疗的重度出血患者的具体指南,因此分享每天处理 vWD 患者的专家经验对于填补与患者管理相关的信息空白至关重要。为了解决这个重要问题,13 位意大利血液学家于 2016 年 4 月 2 日在米兰和 2016 年 7 月 9 日在佛罗伦萨举行了会议。一个 30 个问题的调查问卷构成了讨论的基础:(i)优化 vWD 的诊断工作流程,(ii)可能从二级长期预防治疗(特别是使用低含量因子 VIII 的纯化血管性血友病因子浓缩物)中获益的患者的特征,(iii)选择浓缩物时需要考虑的关键要素,以及(iv)vWD 患者的术前和术后管理。本报告提供了涵盖的主要要点的摘要。