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Sirolimus therapy in the treatment of pseudomyogenic hemangioendothelioma
Author(s) -
Gabor Krisztina Mita,
Sapi Zoltan,
Tiszlavicz Lilla Gyorgyi,
Fige Anita,
Bereczki Csaba,
Bartyik Katalin
Publication year - 2018
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.26781
Subject(s) - medicine , sirolimus , chemotherapy , discovery and development of mtor inhibitors , amputation , hemangioendothelioma , pi3k/akt/mtor pathway , oncology , surgery , pathology , apoptosis , biochemistry , chemistry
Pseudomyogenic hemangioendothelioma (PMH) is a rare, mostly indolent vascular tumor. Extensive cases are treated with amputation as chemotherapy seems to be ineffective. Recently, promising results were published using mammalian target of rapamycin (mTOR) inhibitors in tumors of vascular origin. Here, we present a case of a child with advanced PMH relapsing after surgery and chemotherapy. Sirolimus achieved significant clinical improvement and stabilization of the lesions without any remarkable toxicity. This case contributes to the growing evidence regarding the efficacy of mTOR inhibitors, such as sirolimus, in multifocal PMH.