title: Treatment of recurrent central nervous system primitive neuroectodermal tumours in children and adolescents: results of a Children's Cancer and Leukaemia Group study.
abstract: BACKGROUND
  The treatment of previously irradiated patients with recurrent central nervous system primitive neuroectodermal tumours (PNETs) is a considerable challenge. A study was undertaken to attempt to improve the outcome for such patients using a high dose chemotherapy (HDCT) based strategy.
  
  
  METHODS
  Between 2000 and 2007, 40 patients with relapsed medulloblastoma (MB) and 5 with relapsed supratentorial PNETs (StPNETs) were accrued. All but one had received prior craniospinal radiotherapy. Patients were initially treated with cyclophosphamide (4 g/m(2)) together with surgery or local radiotherapy where appropriate. If complete or near complete remission was achieved, the patient proceeded to receive two sequential courses of HDCT with stem cell rescue. The first course consisted of thiotepa (900 mg/m(2)) and the second carboplatin (AUC 21).
  
  
  RESULTS
  All five patients with StPNET died of tumour progression with a median OS of 0.4 years. Nineteen of the 40 patients with relapsed MB underwent surgery. Radiotherapy was administered to eight patients. All patients received at least one course of cyclophosphamide. Only 22 MB patients progressed to the HDCT phase; 10 patients received thiotepa only and 12 thiotepa and carboplatin. At a median follow-up of 7.4 years (Range 2.8-8.2 years), only three MB patients are still alive, one following a further relapse. Three and 5 year OS was 22.0% and 8.2%, respectively and 3 and 5 year EFS was 14.6% and 8.7%, respectively.
  
  
  CONCLUSIONS
  This national study based on a strategy including a particular tandem HDCT regimen showed no benefit for previously irradiated patients with relapsed StPNET and very limited benefit for patients with relapsed medulloblastoma.
authors.affiliation: Oncology Unit, Alder Hey Children's Hospital, Eaton Road, Liverpool L12 2AP, United Kingdom. barry.pizer@alderhey.nhs.uk
authors.forename: Barry
authors.initials: B
authors.lastname: Pizer
authors.forename: Paul H J
authors.initials: PH
authors.lastname: Donachie
authors.forename: Kathryn
authors.initials: K
authors.lastname: Robinson
authors.forename: Roger E
authors.initials: RE
authors.lastname: Taylor
authors.forename: Antony
authors.initials: A
authors.lastname: Michalski
authors.forename: Jonathan
authors.initials: J
authors.lastname: Punt
authors.forename: David W
authors.initials: DW
authors.lastname: Ellison
authors.forename: Susan
authors.initials: S
authors.lastname: Picton
mesh_terms: D000293:Adolescent; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D019540:Area Under Curve; D016543:Central Nervous System Neoplasms / Q000188:drug therapy* / Q000473:pathology / Q000532:radiotherapy*; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use; D018450:Disease Progression; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D008527:Medulloblastoma / Q000188:drug therapy* / Q000473:pathology / Q000532:radiotherapy*; D018242:Neuroectodermal Tumors, Primitive / Q000188:drug therapy* / Q000473:pathology / Q000532:radiotherapy*; D012008:Recurrence; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D003520:Cyclophosphamide