Article
Alpelisib for treatment of patients with PIK3CA-related overgrowth spectrum (PROS).
Genetics in medicine : official journal of the American College of Medical Genetics - 1 Dec 2023
Canaud Guillaume, Lopez Gutierrez Juan Carlos, Irvine Alan D, Vabres Pierre, Hansford Jordan R, Ankrah Nii, Branle Fabrice, Papadimitriou Athanasia, Ridolfi Antonia, O'Connell Paul, Turner Stuart, Adams Denise M
Abstract excerpt
PURPOSE: PIK3CA-related overgrowth spectrum (PROS) encompasses several rare conditions resulting from activating variants in PIK3CA. Alpelisib, a PI3Kα-selective inhibitor, targets the underlying etiology of PROS, offering a novel therapeutic approach to current management strategies. This study evaluated the safety and efficacy of alpelisib in pediatric and adult patients with PROS. METHODS: EPIK-P1...
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