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.
Lam
Catherine
C
Division of Haematology/Oncology, Neuro-oncology Program, The Hospital for Sick Children, Toronto, Canada.
Bouffet
Eric
E
Tabori
Uri
U
Mabbott
Donald
D
Taylor
Michael
M
Bartels
Ute
U
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