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Updated: Jul 27, 2025

A Patient-Derived Xenograft Model for Venous Malformation
Published on: June 15, 2020
Case Report: Kaposiform hemangioendothelioma with PIK3CA mutation successfully treated with sirolimus
Zuopeng Wang1, Hanlei Yan1, Yangyang Ma2
1Department of Pediatric Surgery, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Abstract:
Kaposiform hemangioendothelioma (KHE) is an extremely rare, locally aggressive vascular neoplasm. The etiopathogenesis of KHE is still poorly understood. In the present study, we found a new mutation in KHE (c.685delA, p.Thr229fs). The KHE patient with the PIK3CA mutation showed complete regression after sirolimus treatment. We propose that the presence of the PIK3CA mutation in KHE may correlate with good response to sirolimus.
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