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<p>Kaposiform hemangioendothelioma (KHE) is a rare, locally aggressive vascular tumour of childhood. Due to its infiltrative growth and possible development 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 the management of vascular anomalies (VAs) in children.</p><p>We present our experience with a 7-month-old male patient with KHE of the lower limb and significant motor function impairment who was successfully treated with sirolimus.</p><p>This case provides 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.</p>

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Sirolimus to the rescue: rapid therapeutic response in kaposiform hemangioendothelioma - case report and literature overview

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年7月8日

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  • Aleksandra Filipiuk,
  • Łukasz Hutnik,
  • Dominik Nguyen,
  • Paweł Łaguna

摘要

Kaposiform hemangioendothelioma (KHE) is a rare, locally aggressive vascular tumour of childhood. Due to its infiltrative growth and possible development 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 the management of vascular anomalies (VAs) in children.

We present our experience with a 7-month-old male patient with KHE of the lower limb and significant motor function impairment who was successfully treated with sirolimus.

This case provides 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.