Center for Vascular Anomalies (a VASCERN VASCA European Reference Centre), Cliniques universitaires St Luc, University of Louvain, Brussels, Belgium. Electronic address: https://twitter.com/emmanuelseront.
Center for Vascular Anomalies (a VASCERN VASCA European Reference Centre), Cliniques universitaires St Luc, University of Louvain, Brussels, Belgium; Institut Roi Albert II, Division of Hematology, Cliniques universitaires Saint-Luc, University of Louvain, Brussels, Belgium. Electronic address: https://twitter.com/HermansCedric.
J Thromb Haemost. 2024 Nov;22(11):2961-2975. doi: 10.1016/j.jtha.2024.07.013. Epub 2024 Aug 2.
Vascular malformations, which arise from anomalies in angiogenesis, encompass capillary, lymphatic, venous, arteriovenous, and mixed malformations, each affecting specific vessel types. Historically, therapeutic options such as sclerotherapy and surgery have shown limited efficacy in complicated malformations. Most vascular malformations stem from hereditary or somatic mutations akin to oncogenic alterations, activating the PI3K-AKT-mTOR, RAS-MAPK-ERK, and G-protein coupled receptor pathways. Recognizing the parallels with oncogenic mutations, we emphasize the potential of targeted molecular inhibitors in the treatment of vascular malformations by repurposing anticancer drugs. This review delves into the recent development and future use of such agents for the management of slow- and fast-flow vascular malformations, including in more specific situations, such as prenatal treatment and the management of associated coagulopathies.
血管畸形源于血管生成异常，包括毛细血管畸形、淋巴管畸形、静脉畸形、动静脉畸形和混合畸形，每种类型都影响特定的血管。历史上，硬化疗法和手术等治疗选择在复杂畸形中显示出有限的疗效。大多数血管畸形源于遗传性或体细胞突变，类似于致癌改变，激活 PI3K-AKT-mTOR、RAS-MAPK-ERK 和 G 蛋白偶联受体途径。鉴于与致癌突变的相似性，我们强调通过重新利用抗癌药物，靶向分子抑制剂在治疗血管畸形方面的潜力。本综述深入探讨了这些药物在管理慢血流和快血流血管畸形中的最新发展和未来用途，包括在更具体的情况下，如产前治疗和相关凝血障碍的管理。
