title: Postoperative chemotherapy in children less than 4 years of age with malignant brain tumors: promising initial response to a VETOPEC-based regimen. A Study of the Australian and New Zealand Children's Cancer Study Group (ANZCCSG).
abstract: OBJECTIVE
  Postoperative chemotherapy with indefinite postponement of radiation therapy in children < 4 years old with brain tumors was investigated in a multi-institutional study.
  
  
  METHODS
  From 1991 to 1995, 42 patients aged 3 to 47 months (median 20) with brain tumors were enrolled in a 2-phase chemotherapy protocol: 16 patients had medulloblastoma (MB); 8 had supratentorial primitive neuroectodermal tumor (PNET); 14 had ependymoma; and 4 had other tumors. The initial phase was comprised of 4 courses of the 3-drug regimen: vincristine (VCR), etoposide (VP-16), and intensive cyclophosphamide (CPA) in a previously reported schedule (VETOPEC). The continuation phase was comprised of 2-drug courses: A, CPA + VCR; B, cisplatin + VP-16; and C, carboplatin + VP-16, for a total duration of 64 weeks.
  
  
  RESULTS
  Response to VETOPEC was evaluable in 28 patients with postresection residual (25) and/or metastatic (1 M2, 6 M3) tumor. There were 9 complete responses (CR) and 9 partial responses (PR) with a combined CR + PR of 64% (95% confidence interval [CI] 44 to 81). In 12 evaluable patients with MB, CR + PR was 82% (48 to 98); in 6 patients with PNET, 50% (12 to 88); and, in 8 patients with ependymoma, 86% (42 to 99). Of 40 patients eligible for further analysis, 6 remain progression-free at a median of 30 months, 14 are alive at a median of 38 months, 29 have progressed at a median of 7 months (range, 2 to 37 months), and 26 have died. The progression-free and overall survival rates at 36 months are estimated to be 11% (95% CI 1 to 22) and 34% (18 to 50), respectively.
  
  
  CONCLUSIONS
  The initial response to the VETOPEC regimen is encouraging and warrants study of further dose escalation. Survival remains poor with current strategies in this high-risk population.
authors.affiliation: Sydney Children's Hospital, NSW, Australia.
authors.forename: L
authors.initials: L
authors.lastname: White
authors.forename: S
authors.initials: S
authors.lastname: Kellie
authors.forename: E
authors.initials: E
authors.lastname: Gray
authors.forename: I
authors.initials: I
authors.lastname: Toogood
authors.forename: K
authors.initials: K
authors.lastname: Waters
authors.forename: L
authors.initials: L
authors.lastname: Lockwood
authors.forename: S
authors.initials: S
authors.lastname: Macfarlane
authors.forename: H
authors.initials: H
authors.lastname: Johnston
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000188:drug therapy* / Q000473:pathology / Q000601:surgery; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D004806:Ependymoma / Q000188:drug therapy / Q000601:surgery; D005047:Etoposide / Q000008:administration & dosage; D006801:Humans; D007223:Infant; D018242:Neuroectodermal Tumors, Primitive / Q000188:drug therapy / Q000601:surgery; D011182:Postoperative Care; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D014750:Vincristine; D005047:Etoposide; D003520:Cyclophosphamide