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 (0.1mg/kg/day). This case provides further rationale for clinical trials of sirolimus in the treatment of vascular lesions. © 2010 Wiley-Liss, Inc.
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Blatt, J., Stavas, J., Moats-Staats, B., Woosley, J., & Morrell, D. S. (2010). Treatment of childhood kaposiform hemangioendothelioma with sirolimus. Pediatric Blood and Cancer, 55(7), 1396–1398. https://doi.org/10.1002/pbc.22766