Case report reveals sirolimus improves symptoms in a child with kaposiform hemangioendothelioma, suggesting effective management for vascular anomalies.
Abstract Kaposiform hemangioendothelioma (KHE) is a rare, locally aggressive vascular tumour of childhood. Due to infiltrative growing and possible development of symptoms of Kasabach-Merritt phenomenon (KMP) if untreated, early intervention is mandatory. Sirolimus, an mTOR (mammalian target of rapamycin) inhibitor that has been used extensively in children following solid organ transplantation. Due to its pleiotropic effects, including the downregulation of angiogenic processes, immunomodulation, and reduction of platelet consumption, it has also shown promise in managing vascular anomalies (VAs) in children.We present our experience with a 7-month-old male patient with KHE and significant motor function impairment, who was successfully treated with sirolimus. This case gives evidence of the growing significance of the high efficacy of sirolimus in the treatment of vascular tumours. It also highlights the need for the development of standardized treatment guidelines for VAs in the paediatric population.
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Filipiuk et al. (2025) studied this question.
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