title: High-dose thiotepa and etoposide in children with poor-prognosis brain tumors.
abstract: BACKGROUND
  Outcome data were analyzed for 27 patients who were affected with recurrent or newly diagnosed high-risk brain tumors and who underwent high-dose chemotherapy with triethylenethiophosphoramide (thiotepa) and etoposide in addition to autologous stem cell transplantation between May 1992 and September 2002.
  
  
  METHODS
  Fifteen males and 12 females (median age, 11 years) were included in the study. Twelve patients had newly diagnosed high-risk brain tumors, and 15 patients had recurrent brain tumors. The conditioning regimen consisted of thiotepa 900 mg/m2 and etoposide 1500 mg/m2 over 3 days starting on Day -5. Stem cell rescue was performed using bone marrow (BM) in 8 patients, peripheral blood stem cells (PBSCs) in 18 patients, and BM and PBSCs in 1 patient.
  
  
  RESULTS
  For the BM group, neutrophil (PMN) engraftment was achieved on Day +14 (median value), whereas platelet (PLT) engraftment was achieved on Day +68 (median value). One patient did not achieve PLT engraftment. For the PBSC group, the PMN engraftment was achieved on Day +10.0 (median value), and the PLT engraftment was achieved on Day +15.5 (median value). Transplantation-related toxicity (evaluated using the Bearman score) included Grade 2-3 mucositis in 16 patients, Grade 1 kidney toxicity in 6 patients, Grade 1 liver toxicity in 6 patients, and Grade 2 liver toxicity in 1 patient. Transplantation-related mortality was observed in 1 patient (3.6%), who died of Candida pneumonia. The 3-year overall survival (OS) rate was 44.6%, and the 3-year event-free survival (EFS) rate was 31%. There was a statistically significant difference in OS and EFS rates for patients who underwent ASCT and achieved complete remission compared with patients who had measurable disease.
  
  
  CONCLUSIONS
  The results of the current study suggest that high-dose chemotherapy followed by ASCT may be beneficial for patients who achieve complete remission before ASCT, whereas for other patients, new strategies are required.
authors.affiliation: Department of Pediatrics, Regina Margherita Children's Hospital, University of Turin, Turin, Italy. franca.fagioli@unito.it
authors.forename: Franca
authors.initials: F
authors.lastname: Fagioli
authors.forename: Eleonora
authors.initials: E
authors.lastname: Biasin
authors.forename: Luana
authors.initials: L
authors.lastname: Mastrodicasa
authors.forename: Alessandro
authors.initials: A
authors.lastname: Sandri
authors.forename: Ivana
authors.initials: I
authors.lastname: Ferrero
authors.forename: Massimo
authors.initials: M
authors.lastname: Berger
authors.forename: Elena
authors.initials: E
authors.lastname: Vassallo
authors.forename: Enrico
authors.initials: E
authors.lastname: Madon
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001254:Astrocytoma / Q000188:drug therapy* / Q000473:pathology; D001932:Brain Neoplasms / Q000188:drug therapy* / Q000473:pathology; D016190:Carboplatin / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000628:therapy; D011379:Prognosis; D011446:Prospective Studies; D012074:Remission Induction; D033581:Stem Cell Transplantation; D015996:Survival Rate; D013852:Thiotepa / Q000008:administration & dosage; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D013852:Thiotepa; D016190:Carboplatin