Biological Approaches to the Aggressive CVM Lesion (Antiangiogenic Therapy)

While drug treatment has been used to treat infantile hemangiomas for decades, targeted treatment for vascular malformations is relatively new, based largely on better understanding of the molecular regulatory pathways that control angiogenesis (Blatt et al., Pharmacol Ther 139(3):327–333, 2013; Margolin et al., Semin Plast Surg 28(2):79–86, 2014). In a small number of cases, drugs that inhibit various aspects of angiogenesis have been used for arteriovenous malformation (AVM). Mammalian target of rapamycin (MTOR) inhibitors are used in patients with severely symptomatic venous and lymphatic malformations as well as patients with symptomatic hamartomas in phosphatase and tensin homolog (PTEN) tumor hamartoma syndrome (Iacobas et al., Pediatr Blood Cancer 57(2):321–323, 2011).