There is a paucity of information about the clinical characteristics and long‐term outcomes of pediatric epithelioid hemangioendothelioma (EHE), a rare vascular neoplasm commonly presenting in adulthood. In our case series of 24 patients with EHE aged 2‐26 years, the majority presented with multi‐organ disease. Progression was seen in 63% of patients with a mean time to progression of 18.4 months (range: 0‐72). Three patients treated with sirolimus achieved stable disease or partial response for >2.5 years. Longitudinal prospective pediatric studies are needed to develop standardized approaches to surgical and medical management.
Epithelioid hemangioendothelioma (EHE) is a rare malignant vascular tumor with no standardized treatment. The mammalian target of rapamycin inhibitor, sirolimus, has been used successfully in adult EHE and other vascular tumors in children but has not been studied in pediatric EHE. The aim of this retrospective case series is to discuss the results of sirolimus for treatment in 6 pediatric patients with EHE. Four of 6 patients demonstrated partial response or disease stabilization with sirolimus treatment. No treatment dosing, trough goals, or duration of treatment recommendations can be made. Prospective studies are warranted to further investigate the use of sirolimus in treatment of EHE.
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