Treatment of childhood kaposiform hemangioendothelioma with sirolimus.
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.
Blatt
Julie
J
Division of Pediatric Hematology Oncology, Vascular Lesions Clinic, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA. jblat@med.unc.edu
Stavas
Joseph
J
Moats-Staats
Billie
B
Woosley
John
J
Morrell
Dean S
DS
D020533:Angiogenesis Inhibitors / Q000627:therapeutic use*; D000903:Antibiotics, Antineoplastic / Q000627:therapeutic use*; D005260:Female; D006390:Hemangioendothelioma / Q000097:blood / Q000188:drug therapy* / Q000473:pathology; D006801:Humans; D007223:Infant; D010976:Platelet Count; D020123:Sirolimus / Q000627:therapeutic use*; D012878:Skin Neoplasms / Q000097:blood / Q000188:drug therapy* / Q000473:pathology
D020533:Angiogenesis Inhibitors; D000903:Antibiotics, Antineoplastic; D020123:Sirolimus