Research News: Review Article of Targeted Therapies for Vascular Malformations

A new review article entitled New and Emerging Targeted Therapies for Vascular Malformations has just been published in the American Journal of Clinical Dermatology. It is co-authored by Vascular Anomalies Working Group (VASCA WG) Chairs Professors Miikka Vikkula and Laurence Boon along with members of their Healthcare Provider (HCP) at Saint Luc University Hospital in Brussels, Belgium.

Access the full article here

While vascular anomalies have historically been managed by surgery and sclerotherapy, improved molecular and pathophysiological understanding of vascular anomalies in recent years has opened the door to new treatment possibilities thanks to the finding that most are due to inherited or somatic mutations in genes that hyperactivate two major intracellular signaling pathways: phosphatidylinositol 3 kinase/protein kinase B/mammalian target of rapamycin (mTOR) or the RAS/ MAPK/ERK pathway.

Targeted molecular therapies that target these pathways (initially used for treating cancers that harbor mutations in the same pathways) are therefore now being tested in vascular malformation patients. This review gives an overview of the various therapies currently being tested or soon to be tested in vascular anomaly patients (including sirolimus, everolimus alpelisib, trametinib, and bevacizumab) and the disease outcomes reported to date as well as ongoing trials underway in this rapidly evolving field.

Reference: New and Emerging Targeted Therapies for Vascular Malformations.Van Damme A, Seront E, Dekeuleneer V, Boon LM, Vikkula M. Am J Clin Dermatol. 2020 Jun 15. doi: 10.1007/s40257-020-00528-w. Online ahead of print.PMID: 32557381