title: Atypical teratoid or rhabdoid tumors: improved outcome with high-dose chemotherapy.
abstract: OBJECTIVE
  To retrospectively review an institutional experience in managing atypical teratoid/rhabdoid tumors (AT/RT) of the Central Nervous System with high-dose chemotherapy in infants and children less than 4 years old.
  
  
  METHODS
  Eight AT/RT patients were identified during the study period 2003 to 2008. Tumor location was supratentorial in 3 cases, infratentorial in 3 cases, and multifocal in 2 patients. Five patients presented with leptomeningeal dissemination. Two of these patients did not receive any active therapy. After surgery, the 6 remaining patients received induction therapy followed by sequential high-dose chemotherapy with autologous stem cell rescue. Two patients receive focal irradiation.
  
  
  RESULTS
  At a median follow-up of 52 months, 4 patients are alive without evidence of tumor. Three of these patients, including 2 with metastatic disease were not irradiated. However, all surviving patients exhibit neuro-cognitive impairment, either at baseline assessment or upon follow-up.
  
  
  CONCLUSIONS
  This experience confirms that a subset of young AT/RT patients may achieve long-term survival with intensive and high-dose chemotherapy. The role of radiotherapy is still unclear. However, evidence of neuro-cognitive impairment even in the absence of radiotherapy in this young population suggests that systematic introduction of radiotherapy in current protocols should be carefully assessed.
authors.affiliation: Bone Marrow Transplant Program, Hospital for Sick Children, Toronto, Canada.
authors.forename: Tal
authors.initials: T
authors.lastname: Finkelstein-Shechter
authors.forename: Adam
authors.initials: A
authors.lastname: Gassas
authors.forename: Donald
authors.initials: D
authors.lastname: Mabbott
authors.forename: Annie
authors.initials: A
authors.lastname: Huang
authors.forename: Ute
authors.initials: U
authors.lastname: Bartels
authors.forename: Uri
authors.initials: U
authors.lastname: Tabori
authors.forename: Laura
authors.initials: L
authors.lastname: Janzen
authors.forename: Janzen
authors.initials: J
authors.lastname: Laura
authors.forename: Cynthia
authors.initials: C
authors.lastname: Hawkins
authors.forename: Michael
authors.initials: M
authors.lastname: Taylor
authors.forename: Eric
authors.initials: E
authors.lastname: Bouffet
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000188:drug therapy* / Q000473:pathology / Q000532:radiotherapy; D002675:Child, Preschool; D002945:Cisplatin / Q000008:administration & dosage; D003131:Combined Modality Therapy; D016371:Cranial Irradiation; D003520:Cyclophosphamide / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D009362:Neoplasm Metastasis; D011879:Radiotherapy Dosage; D012189:Retrospective Studies; D018335:Rhabdoid Tumor / Q000188:drug therapy* / Q000473:pathology / Q000532:radiotherapy; D015996:Survival Rate; D013724:Teratoma / Q000188:drug therapy* / Q000473:pathology / Q000532:radiotherapy; D016896:Treatment Outcome; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D014750:Vincristine; D005047:Etoposide; D003520:Cyclophosphamide; D002945:Cisplatin