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Updated: Jun 5, 2025

A Patient-Derived Xenograft Model for Venous Malformation
Published on: June 15, 2020
Targeted medical therapies for vascular anomalies
1Comprehensive Vascular Anomalies Program, The Children's Hospital of Philadelphia, Philadelphia, PA.
Abstract:
The last 2 decades of genetic discovery in the field of vascular anomalies have brought targeted medical therapies to the forefront of care patients with vascular anomalies and have broadened the role of hematologists/oncologists in this field. Many vascular anomalies have now been identified to be driven by somatic gain-of-function variants in the PI3K/AKT/ mTOR and Ras/MAPK intracellular signaling pathways. This has led to the introduction of various antiangiogenic agents that inhibit these pathways. Knowledge of the indications for and the safe administration of these agents in patients with vascular anomalies is now a crucial part of training for hematologists/oncologists.
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