Targeted treatments for vascular malformations: current state of the art.

J Thromb Haemost

Center for Vascular Anomalies (a VASCERN VASCA European Reference Centre), Cliniques universitaires St Luc, University of Louvain, Brussels, Belgium; Deprtment of Human Molecular Genetics, de Duve Institute, University of Louvain, Brussels, Belgium; Walloon ExceLlence in Life Sciences and Biotechnology (WELBIO) and Walloon ExceLlence Research Institute (WEL Research Institute), Wavre, Belgium. Electronic address:

Published: November 2024

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.

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Source
http://dx.doi.org/10.1016/j.jtha.2024.07.013DOI Listing

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