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Updated: Sep 3, 2026

Comprehensive Endovascular and Open Surgical Management of Cerebral Arteriovenous Malformations
Published on: October 20, 2017
Medical Management of Vascular Anomalies
1Department of Pediatric Hematology-Oncology, Dana Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA, USA.
Abstract:
Most vascular anomalies are caused by mutations in the PI3K-AKT-mTOR or RAS-RAF-MEK-ERK signaling pathways. This knowledge, along with increasing availability of genetic testing for molecular diagnosis, has made medical management an important component of treatment of vascular anomalies. We are now able to use mTOR inhibitors like sirolimus, PI3K inhibitors like alpelisib and MEK inhibitors like trametinib for targeted therapy of vascular anomalies, often in conjunction with interventional and surgical management. Awareness of these treatment options can help provide optimal care to all patients with vascular anomalies.
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