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Rapamycin (sirolimus) in tuberous sclerosis associated pediatric central nervous system tumors
Author(s) -
Lam Catherine,
Bouffet Eric,
Tabori Uri,
Mabbott Donald,
Taylor Michael,
Bartels Ute
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.22298
Subject(s) - tuberous sclerosis , medicine , sirolimus , subependymal giant cell astrocytoma , subependymal zone , blood cancer , oncology , cancer , glioma , pathology , cancer research , astrocytoma
Tuberous sclerosis complex (TSC) is associated with hamartomatous growths including subependymal giant cell astrocytomas (SEGAs). Since chemo‐radiation therapies offer scant benefit, oncologists had traditionally been little involved in managing SEGAs. Recent evidence demonstrating rapamycin efficacy in adults and children with TSC‐associated tumors foresee a practice change. We summarize our institutional experience and literature review that highlight potential benefits and hazards of rapamycin therapy, for TSC patients with SEGA, and other syndromal brain tumors. Pediatr Blood Cancer 2010;54:476–479. © 2009 Wiley‐Liss, Inc.