Rapamycin (sirolimus) in tuberous sclerosis associated pediatric central nervous system tumors

Pediatr Blood Cancer. 2010 Mar;54(3):476-9. doi: 10.1002/pbc.22298.

Abstract

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.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Antibiotics, Antineoplastic / therapeutic use*
  • Central Nervous System Neoplasms / drug therapy*
  • Central Nervous System Neoplasms / pathology
  • Child
  • Female
  • Humans
  • Male
  • Sirolimus / therapeutic use*
  • Tuberous Sclerosis / drug therapy*
  • Tuberous Sclerosis / pathology

Substances

  • Antibiotics, Antineoplastic
  • Sirolimus