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Recent studies have demonstrated that venous malformations (VMs) are associated with somatic, activating Pik3ca mutations resulting in a hyperproliferative phenotype. The Pik3ca gene encodes the p110a catalytic subunit of the PI3 kinase, which subsequently activates mTOR. While Sirolimus, an mTOR inhibitor, is widely used in the treatment of VMs, a complete clinical response has never been documented. Given sirolimus’s limited capacity, there is a need for alternative therapies. We therefore hypothesized that affected endothelial cells from venous malformations (VMECs) with Pik3ca variants could be used in a high-throughput screen (HTPS) to survey the efficacy of PI3K pathway inhibitors. Our data showed that the clinically available drugs are only partially effective against VMECs. Furthermore, our data suggested that dual inhibition of PI3K pathway proteins with a combined PI3K/mTOR inhibitor may be more efficacious in the treatment of VMs. High Throughput Screening (HTPS) Is A Powerful Tool To Identify Potential Therapeutic Agents In The Treatment Of Venous Malformations Noa Shapiro-Franklin, BS; Ajit Muley, PhD; Hai Li, PhD; Kate Stanley, PhD; Anna Alkelai, PhD; Vimla Aggarwal, MD, PhD; David Goldstein, PhD; Charles Karan, PhD; Carrie J. Shawber, PhD; June K. Wu, MD Columbia University Medical Center, New York

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