title: Treatment of early childhood medulloblastoma by postoperative chemotherapy alone.
abstract: BACKGROUND
  The prognosis for young children with medulloblastoma is poor, and survivors are at high risk for cognitive deficits. We conducted a trial of the treatment of this brain tumor by intensive postoperative chemotherapy alone.
  
  
  METHODS
  After surgery, children received three cycles of intravenous chemotherapy (cyclophosphamide, vincristine, methotrexate, carboplatin, and etoposide) and intraventricular methotrexate. Treatment was terminated if a complete remission was achieved. Leukoencephalopathy and cognitive deficits were evaluated.
  
  
  RESULTS
  Forty-three children were treated according to protocol. In children who had complete resection (17 patients), residual tumor (14), and macroscopic metastases (12), the five-year progression-free and overall survival rates (+/-SE) were 82+/-9 percent and 93+/-6 percent, 50+/-13 percent and 56+/-14 percent, and 33+/-14 percent and 38+/-15 percent, respectively. The rates in 31 patients without macroscopic metastases were 68+/-8 percent and 77+/-8 percent. Desmoplastic histology, metastatic disease, and an age younger than two years were independent prognostic factors for tumor relapse and survival. Treatment strategies at relapse were successful in 8 of 16 patients. There were no major instances of unexpected toxicity. In 19 of 23 children, asymptomatic leukoencephalopathy was detected by magnetic resonance imaging. After treatment, the mean IQ was significantly lower than that of healthy controls within the same age group but higher than that of patients in a previous trial who had received radiotherapy.
  
  
  CONCLUSIONS
  Postoperative chemotherapy alone is a promising treatment for medulloblastoma in young children without metastases.
authors.affiliation: Department of Pediatric Oncology, Children's Hospital, University of Würzburg, Würzburg, Germany. rutkowski@mail.uni-wuerzburg.de
authors.forename: Stefan
authors.initials: S
authors.lastname: Rutkowski
authors.forename: Udo
authors.initials: U
authors.lastname: Bode
authors.forename: Frank
authors.initials: F
authors.lastname: Deinlein
authors.forename: Holger
authors.initials: H
authors.lastname: Ottensmeier
authors.forename: Monika
authors.initials: M
authors.lastname: Warmuth-Metz
authors.forename: Niels
authors.initials: N
authors.lastname: Soerensen
authors.forename: Norbert
authors.initials: N
authors.lastname: Graf
authors.forename: Angela
authors.initials: A
authors.lastname: Emser
authors.forename: Torsten
authors.initials: T
authors.lastname: Pietsch
authors.forename: Johannes E A
authors.initials: JE
authors.lastname: Wolff
authors.forename: Rolf D
authors.initials: RD
authors.lastname: Kortmann
authors.forename: Joachim
authors.initials: J
authors.lastname: Kuehl
mesh_terms: D000704:Analysis of Variance; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D016190:Carboplatin / Q000008:administration & dosage; D002528:Cerebellar Neoplasms / Q000188:drug therapy* / Q000523:psychology / Q000601:surgery; D017024:Chemotherapy, Adjuvant; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D007360:Intelligence / Q000187:drug effects; D008527:Medulloblastoma / Q000188:drug therapy* / Q000523:psychology / Q000556:secondary / Q000601:surgery; D008727:Methotrexate / Q000008:administration & dosage; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy; D009483:Neuropsychological Tests; D011182:Postoperative Care; D016016:Proportional Hazards Models; D012074:Remission Induction; D016019:Survival Analysis; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D014750:Vincristine; D005047:Etoposide; D003520:Cyclophosphamide; D016190:Carboplatin; D008727:Methotrexate