Rapamycin (sirolimus) in tuberous sclerosis associated pediatric central nervous system tumors.
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.
Division of Haematology/Oncology, Neuro-oncology Program, The Hospital for Sick Children, Toronto, Canada.
Catherine
C
Lam
Eric
E
Bouffet
Uri
U
Tabori
Donald
D
Mabbott
Michael
M
Taylor
Ute
U
Bartels
D000293:Adolescent; D000903:Antibiotics, Antineoplastic / Q000627:therapeutic use*; D016543:Central Nervous System Neoplasms / Q000188:drug therapy* / Q000473:pathology; D002648:Child; D005260:Female; D006801:Humans; D008297:Male; D020123:Sirolimus / Q000627:therapeutic use*; D014402:Tuberous Sclerosis / Q000188:drug therapy* / Q000473:pathology
D000903:Antibiotics, Antineoplastic; D020123:Sirolimus