Kaposiform hemangioendothelioma further broadens the phenotype of <scp>PIK3CA</scp> ‐related overgrowth spectrum

Diana Carli, Silvia Kalantari, Rosaria Manicone, Paola Coppo, Paola Francia di Celle, Roberta La Selva, Federica Santoro, Carlotta Ranieri, Simona Cardaropoli, Franca Fagioli,

Clinical Genetics · 2021 · 21 citations · 15 references

Concepts

Abstract

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.

References

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