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Rapamycin and treatment of venous malformations

Seront, Emmanuela,b; Van Damme, Anb,c; Boon, Laurence M.b,d; Vikkula, Miikkab,d,e

Current Opinion in Hematology: May 2019 - Volume 26 - Issue 3 - p 185–192
doi: 10.1097/MOH.0000000000000498
VASCULAR BIOLOGY: Edited by M. Luisa Iruela-Arispe

Purpose of review The field of vascular anomalies has seen a fundamental change during the past 10 years. The identification of somatic genetic mutations as the explanation of sporadic vascular anomalies opened the doors to study prospectively and a posteriori the causes of various vascular malformations. This was helped by the rapidly evolving genetic techniques including the highly sensitive next generation sequencing. In parallel, knowledge on signaling alterations occurring in vascular endothelial cells because of the various mutations, development of in-vitro and especially the first in-vivo models, gave the possibility to test preclinically molecular therapies for vascular malformations.

Recent findings One of the first molecules, rapamycin, showed clear evidence of interrupting lesion growth. As its safety profile had been established in other conditions, it was quickly accepted for clinical trials on vascular anomalies. Now, with a few trials published and others ongoing, it is establishing itself as a gold standard for molecular therapy for recalcitrant lesions.

Summary Targeted molecular therapies are becoming interesting new additions to the management of vascular anomalies, and rapamycin is establishing itself as a gold standard for venous malformations.

aInstitut Roi Albert II, Department of Medical Oncology

bCenter for Vascular Anomalies, Division of Plastic Surgery

cInstitut Roi Albert II, Department of Pediatric Hematology and Oncology, Cliniques universitaires Saint Luc

dHuman Molecular Genetics, de Duve Institute

eWELBIO (Walloon Excellence in Lifesciences and Biotechnology), de Duve Institute, University of Louvain, Brussels, Belgium

Correspondence to Miikka Vikkula, MD, PhD, Professor of Human Genetics, Human Molecular Genetics (GEHU), de Duve Institute (DDUV), University of Louvain (UCLouvain), Avenue Hippocrate 74 (+5), bte B1.74.06, B-1200 Brussels, Belgium. Tel: +32 2 764 7490; fax: +32 2 764 7460; e-mail:

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