title: Tandem high-dose chemotherapy and autologous stem cell rescue in children with newly diagnosed high-risk medulloblastoma or supratentorial primitive neuro-ectodermic tumors.
abstract: BACKGROUND
  To assess the feasibility and effectiveness of high-dose chemotherapy (HDC) with stem cell support followed by conventional craniospinal radiotherapy (RT) as treatment for children older than 5 years of age with newly diagnosed high-risk medulloblastoma (MB) or supratentorial PNET (sPNET).
  
  
  METHODS
  Between May 2001 and April 2010, 24 children older than 5 years of age (MB = 21; sPNET = 3), fulfilling inclusion criteria at diagnosis, were treated at Gustave Roussy. After conventional chemotherapy, they received two courses of high-dose thiotepa (600 mg/m(2)) followed by craniospinal RT.
  
  
  RESULTS
  The median follow-up was 4.4 years (range, 0.8-11.3 years). For children with metastatic MB, the 5-year event-free survival (EFS) and overall survival (OS) were 72% and 83%, respectively. The toxicity was manageable. No toxic death occurred. At the most recent evaluation, among the 24 children who had at least one Full Scale Intellectual Quotient (FSIQ) examination at a median follow-up of 3.79 years after diagnosis, the mean estimated FSIQ was 82 (range, 56-114).
  
  
  CONCLUSIONS
  In children with metastatic MB, tandem HDCT with ASCT followed by conventional craniospinal RT proved its feasibility without jeopardizing survival.
authors.affiliation: Department of Pediatric and Adolescent Oncology, Gustave Roussy, Villejuif, France.
authors.forename: Christelle
authors.initials: C
authors.lastname: Dufour
authors.forename: Virginie
authors.initials: V
authors.lastname: Kieffer
authors.forename: Pascale
authors.initials: P
authors.lastname: Varlet
authors.forename: Marie Anne
authors.initials: MA
authors.lastname: Raquin
authors.forename: Frederic
authors.initials: F
authors.lastname: Dhermain
authors.forename: Stephanie
authors.initials: S
authors.lastname: Puget
authors.forename: Dominique
authors.initials: D
authors.lastname: Valteau-Couanet
authors.forename: Jacques
authors.initials: J
authors.lastname: Grill
mesh_terms: D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage*; D064592:Autografts; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008527:Medulloblastoma / Q000175:diagnosis* / Q000628:therapy*; D018242:Neuroectodermal Tumors, Primitive / Q000175:diagnosis* / Q000628:therapy*; D012189:Retrospective Studies; D033581:Stem Cell Transplantation
keywords: childhood; high-dose chemotherapy; medulloblastoma