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Treatment of childhood kaposiform hemangioendothelioma with sirolimus
Author(s) -
Blatt Julie,
Stavas Joseph,
MoatsStaats Billie,
Woosley John,
Morrell Dean S.
Publication year - 2010
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.22766
Subject(s) - sirolimus , medicine , discovery and development of mtor inhibitors , blood cancer , hemangioendothelioma , clinical trial , cancer , pathology , pi3k/akt/mtor pathway , apoptosis , biochemistry , chemistry
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. Pediatr Blood Cancer. 2010;55:1396–1398. © 2010 Wiley‐Liss, Inc.