title: High-dose carboplatin, thiotepa, and etoposide with autologous stem-cell rescue for patients with recurrent medulloblastoma. Children's Cancer Group.
abstract: OBJECTIVE
  Medulloblastoma is a highly lethal disease when it recurs. Very few patients survive with conventional treatment. This study evaluated the use of high-dose carboplatin, thiotepa, and etoposide with autologous stem-cell rescue (ASCR) in patients with recurrent medulloblastoma.
  
  
  METHODS
  Chemotherapy consisted of carboplatin 500 mg/m2 (or area under the curve = 7 mg/mL x min via Calvert formula) on days -8, -7, and -6; and thiotepa 300 mg/m2 and etoposide 250 mg/m2 on days -5, -4, and -3; followed by ASCR on day 0. In addition to the study-prescribed therapy, 21 patients received other treatment: neurosurgical resection in seven, conventional chemotherapy in 17, and external-beam irradiation in 11 cases.
  
  
  RESULTS
  Twenty-three patients with recurrent medulloblastoma, aged two to 44 years (median, 13 years) at ASCR, were treated. Three patients died of treatment-related toxicities within 21 days of ASCR; multiorgan system failure in two, and Aspergillus infection with venoocclusive disease in one. Seven of 23 patients (30%) are event-free survivors at a median of 54 months post-ASCR (range, 24 to 78 months). Kaplan-Meier estimates of event-free (EFS) and overall survival are 34% +/- 10% and 46% +/- 11%, respectively, at 36 months post-ASCR.
  
  
  CONCLUSIONS
  This strategy may provide long-term survival for some patients with recurrent medulloblastoma.
authors.affiliation: Memorial Sloan-Kettering Cancer Center, New York, NY, USA. dunkeli@mskcc.org
authors.forename: I J
authors.initials: IJ
authors.lastname: Dunkel
authors.forename: J M
authors.initials: JM
authors.lastname: Boyett
authors.forename: A
authors.initials: A
authors.lastname: Yates
authors.forename: M
authors.initials: M
authors.lastname: Rosenblum
authors.forename: J H
authors.initials: JH
authors.lastname: Garvin
authors.forename: B C
authors.initials: BC
authors.lastname: Bostrom
authors.forename: S
authors.initials: S
authors.lastname: Goldman
authors.forename: L S
authors.initials: LS
authors.lastname: Sender
authors.forename: S L
authors.initials: SL
authors.lastname: Gardner
authors.forename: H
authors.initials: H
authors.lastname: Li
authors.forename: J C
authors.initials: JC
authors.lastname: Allen
authors.forename: J L
authors.initials: JL
authors.lastname: Finlay
mesh_terms: D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D016190:Carboplatin / Q000008:administration & dosage / Q000009:adverse effects; D002528:Cerebellar Neoplasms / Q000188:drug therapy* / Q000401:mortality; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005047:Etoposide / Q000008:administration & dosage / Q000009:adverse effects; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D008527:Medulloblastoma / Q000188:drug therapy* / Q000401:mortality; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy* / Q000401:mortality; D016019:Survival Analysis; D013852:Thiotepa / Q000008:administration & dosage / Q000009:adverse effects; D014182:Transplantation, Autologous
chemical_list: D005047:Etoposide; D013852:Thiotepa; D016190:Carboplatin