Table 11.

Current targeted therapies in-use or under investigation for vascular anomalies.

Drug	Mechanism	Uses	Status
Sirolimus, everolimus	mTOR inhibition	Complex vascular malformations and vascular tumors	Available, not FDA approved. Multiple ongoing phase I-III trials evaluating use in specific vascular anomalies and also investigating topical use
Trametinib, cobimetinib, selumetinib	MEK inhibition	AVM, possible use in complicated lymphatic anomalies (CCLA, GLA, KLA)	Has been used in some cases without additional therapeutic options (90–92). Upcoming Phase II trial in extracranial AVM
Alpelisib (BYL719)	PIK3CA inhibition	PIK3CA-related overgrowth spectrum disorders (PROS)	Agent is FDA approved for use in breast cancer. Currently under investigation for PROS. Some availability through compassionate use program
ARQ092	AKT inhibition	PROS and PROTEUS syndrome	Under investigation, Phase I/II studies
Propranolol, Atenolol, Timolol (topical)	Beta-blockade	Infantile hemangiomas	Approved as Hemangiol