Treatment of childhood kaposiform hemangioendothelioma with sirolimus

Pediatr Blood Cancer. 2010 Dec 15;55(7):1396-8. doi: 10.1002/pbc.22766.

Abstract

Sirolimus (Rapamune), a mammalian target of Rapamycin (mTOR) inhibitor, which has been used extensively in children following solid organ transplantation, has been demonstrated to have anti-angiogenic activity in pre-clinical models. Limited experience suggests that it may have application to the treatment of vascular lesions. We describe our experience with a 1-year-old female with a kaposiform hemangioendothelioma and Kasabach-Merritt phenomenon who had rapid and dramatic response to sirolimus (0.1 mg/kg/day). This case provides further rationale for clinical trials of sirolimus in the treatment of vascular lesions.

Publication types

  • Case Reports

MeSH terms

  • Angiogenesis Inhibitors / therapeutic use*
  • Antibiotics, Antineoplastic / therapeutic use*
  • Female
  • Hemangioendothelioma / blood
  • Hemangioendothelioma / drug therapy*
  • Hemangioendothelioma / pathology
  • Humans
  • Infant
  • Platelet Count
  • Sirolimus / therapeutic use*
  • Skin Neoplasms / blood
  • Skin Neoplasms / drug therapy*
  • Skin Neoplasms / pathology

Substances

  • Angiogenesis Inhibitors
  • Antibiotics, Antineoplastic
  • Sirolimus