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Updated: Jul 18, 2025

A Patient-Derived Xenograft Model for Venous Malformation
Published on: June 15, 2020
Repurposing of antiangiogenic agents for treatment of vascular anomalies
Julie Blatt1, Jennifer E Brondon1, Elizabeth L Nieman2
1Division of Hematology Oncology, Department of Pediatrics, and the Lineberger Clinical Cancer Center, University of North Carolina School of Medicine, Chapel Hill, NC, USA.
Abstract:
Vascular anomalies (VA) are developmental anomalies of veins, arteries, lymphatics or capillaries thought to be caused by mutations in genes that drive angiogenesis. Treatments targeting these genes are limited. We review the literature for conventional medications and products from traditional medicine cultures that have been found to have antiangiogenic activity. Fewer than 50 drugs with credible human activity in VA were identified and include β blockers, monoclonal antibodies, microtubule inhibitors, multi-kinase inhibitors, PIK3CA- and RAS-MAPK pathway inhibitors, and thalidomides. Other drug categories of potential interest are ACE-inhibitors, antifungals, antimalarials, MMP9-inhibitors, and over-the-counter compounds used in Eastern traditional medicine. Low toxicity for some offers the possibility of combined use with known effective agents. In addition to already familiar drugs, others with antiangiogenic capabilities already in use in children or adults may deserve further attention for repurposing for VA.
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