title: Successful everolimus therapy for SEGA in pediatric patients with tuberous sclerosis complex.
abstract: OBJECTIVE
  Tuberous sclerosis complex (TSC) is associated with hamartomatous growths including subependymal giant cell astrocytomas (SEGAs). Although, SEGAs are slow-growing glioneuronal tumors, they represent a significant cause of morbidity and mortality due to the risk of sudden death from acute hydrocephalus. Neurosurgical resection has been the mainstay of therapy, since radiotherapy and chemotherapy were proved inefficient in those tumors. Recent studies support the use of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis and suggest it might represent a disease-modifying treatment for other aspects of tuberous sclerosis.
  
  
  METHODS
  We describe the clinical and radiological progression of three pediatric patients with definitive diagnosis of TSC and SEGA, which have been treated with everolimus.
  
  
  RESULTS
  Up to 34 % sustained SEGA decrease was observed in the three cases. All three patients have experienced seizure control and two of them have showed cognitive and behavioral improvement. Everolimus has been well tolerated by all. No severe adverse events have been observed to date.
  
  
  CONCLUSIONS
  Everolimus offers significant promise in treating SEGAs. Studies are required to explore optimal therapy duration and management upon discontinuing therapy.
authors.affiliation: Institute of Pediatric Oncology, GRAACC, Federal University of São Paulo, Rua Botucatu, 743 Vila Clementino, 04023-062, São Paulo, Brazil.
authors.forename: A M
authors.initials: AM
authors.lastname: Cappellano
authors.forename: A A
authors.initials: AA
authors.lastname: Senerchia
authors.forename: F
authors.initials: F
authors.lastname: Adolfo
authors.forename: P M
authors.initials: PM
authors.lastname: Paiva
authors.forename: R
authors.initials: R
authors.lastname: Pinho
authors.forename: A
authors.initials: A
authors.lastname: Covic
authors.forename: S
authors.initials: S
authors.lastname: Cavalheiro
authors.forename: N
authors.initials: N
authors.lastname: Saba
mesh_terms: D000293:Adolescent; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001254:Astrocytoma / Q000188:drug therapy* / Q000209:etiology; D001932:Brain Neoplasms / Q000188:drug therapy* / Q000209:etiology; D002675:Child, Preschool; D000068338:Everolimus; D005260:Female; D006801:Humans; D008297:Male; D020123:Sirolimus / Q000031:analogs & derivatives* / Q000627:therapeutic use; D014402:Tuberous Sclerosis / Q000150:complications*
chemical_list: D000970:Antineoplastic Agents; D000068338:Everolimus; D020123:Sirolimus