Current and Emerging Targeted Therapies for Vascular Malformations

Invasive treatments for vascular malformations (surgery, embolization, laser, and sclerotherapy) are not always feasible or efficacious; they also carry risks of morbidity and recurrence. Recently, specific hereditary or somatic mutations found in vascular malformations linked to cellular pathways have opened doors to targeted therapies: pathway inhibitors for mTOR (e.g. sirolimus), AKT (e.g. miransertib), PIK3CA (e.g. alpelisib), MEK (e.g. trametinib), and VEGFR (e.g. bevacizumab, pomalidomide). Clinical, functional, and radiographic responses may however be disparate. Understanding pathways and data on efficacy and safety of these therapies are important for appropriate selection of agents for those with vascular malformations.