Clinical Genetics · 2021 · 21 citations · 15 references
MedicineOncologyPathologyMolecular OncologyNeoplasiaPediatric OncologyAngiogenesisKaposi SarcomaOncogenic Signaling PathwaysPediatric TumorsVascular AnomaliesVascular MalformationsKaposiform HemangioendotheliomaPhosphatidylinositol 3-Kinase-akt PathwayPhosphatidylinositol 3-Kinase-akt SignalingPi3 Kinase-akt SignalingPi3k SignalingPi3k-akt Signaling PathwayPi3k-akt-mtor SignalingPik3ca Mutations
Kaposiform hemangioendothelioma (KHE) is a rare locally aggressive mixed vascular tumor, with typical onset in early childhood and characterized by progressive angio- and lymphangiogenesis. Its etiopathogenesis and molecular bases are still unclear. Here, we report the first case of congenital KHE harboring a PIK3CA mosaic pathogenic variant (c.323G > A, p.Arg108His) in a boy with very subtle PIK3CA-related overgrowth spectrum (PROS) features. This finding provides insights into the pathophysiology of KHE, offering targeted therapeutic options by inhibition of the PI3K/Akt/mTOR pathway. We propose the inclusion of this mixed lymphatic and vascular anomaly within the PROS.
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Targeted therapy in patients with PIK3CA-related overgrowth syndrome
Quitterie Venot, Thomas Blanc, Laureline Berteloot et al. · Nature · 2018 · 575 citations · Full text