Advances in Management of Vascular Anomalies (ASH 2026) - Sep 26, 2026 - "Because many of these genes are established oncogenes, cancer drugs have been repurposed for these conditions, exemplified by the PI3Kα inhibitor, alpelisib, now approved for PIK3CA-related overgrowth spectrum (PROS). Ricci synthesizes the genomic landscape across phenotypes and reviews the evidence for MEK, KRAS, and BRAF inhibition, with trametinib as the most established agent. This presentation then offers a genotype-guided framework for diagnosis and management, highlighting the expanding role of the hematologist-oncologist and the barriers to delivering equitable precision care."
CNS Disorders • Rare Diseases • AKT1 • KRAS • PIK3CA • PIK3R1 • PTEN
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