Targeted medical therapies for vascular anomalies

Hematology Am Soc Hematol Educ Program. 2024 Dec 6;2024(1):709-717. doi: 10.1182/hematology.2024000599.

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.

Publication types

  • Review

MeSH terms

  • Angiogenesis Inhibitors / therapeutic use
  • Humans
  • Molecular Targeted Therapy
  • Signal Transduction / drug effects
  • TOR Serine-Threonine Kinases / antagonists & inhibitors
  • TOR Serine-Threonine Kinases / metabolism
  • Vascular Malformations* / drug therapy
  • Vascular Malformations* / genetics
  • Vascular Malformations* / therapy

Substances

  • Angiogenesis Inhibitors
  • TOR Serine-Threonine Kinases
  • MTOR protein, human