title: Intensive chemotherapy and bone marrow rescue for young children with newly diagnosed malignant brain tumors.
abstract: OBJECTIVE
  To evaluate a strategy that avoids radiotherapy in children less than 6 years of age with newly diagnosed malignant brain tumors, by administering myeloablative consolidation chemotherapy with autologous bone marrow reconstitution (ABMR) after maximal surgical resection and conventional induction chemotherapy.
  
  
  METHODS
  Between March 1991 and April 1995, 62 children (median age, 30 months) with newly diagnosed malignant brain tumors were enrolled onto this trial. Children received conventional induction chemotherapy with vincristine, cisplatin, cyclophosphamide, and etoposide, repeated every 3 weeks for five cycles. Children without disease progression on induction chemotherapy were offered consolidation with myeloablative chemotherapy that incorporated carboplatin, thiotepa, and etoposide followed by ABMR. Irradiation was used only for residual tumor at consolidation or for progressive/recurrent disease.
  
  
  RESULTS
  Induction chemotherapy was well tolerated by most patients; however, progression was noted in 17 children (27%) and four (6%) died of treatment complications. Of 37 children who received consolidation chemotherapy with ABMR, 15 are free of disease progression (median post-ABMR without further treatment, >44 months). The remaining 22 all progressed within 15 months of ABMR; three of 37 (8%) died of treatment-related complications. The 3-year overall survival (OS) and event-free survival (EFS) rates from diagnosis for all children are 40% (95% confidence interval [CI], 28% to 52%) and 25% (95% CI, 13% to 37%), respectively. Radiotherapy was administered to 19 of 62 children: 17 for progressive disease (PD) and two for residual disease at the time of ABMR.
  
  
  CONCLUSIONS
  A significant proportion of children with malignant brain tumors can avoid radiotherapy and prolonged maintenance chemotherapy yet still achieve durable remission with this brief intensive chemotherapy regimen.
authors.affiliation: Memorial Sloan-Kettering Cancer Center, New York, NY 10021, USA.
authors.forename: W P
authors.initials: WP
authors.lastname: Mason
authors.forename: A
authors.initials: A
authors.lastname: Grovas
authors.forename: S
authors.initials: S
authors.lastname: Halpern
authors.forename: I J
authors.initials: IJ
authors.lastname: Dunkel
authors.forename: J
authors.initials: J
authors.lastname: Garvin
authors.forename: G
authors.initials: G
authors.lastname: Heller
authors.forename: M
authors.initials: M
authors.lastname: Rosenblum
authors.forename: S
authors.initials: S
authors.lastname: Gardner
authors.forename: D
authors.initials: D
authors.lastname: Lyden
authors.forename: S
authors.initials: S
authors.lastname: Sands
authors.forename: D
authors.initials: D
authors.lastname: Puccetti
authors.forename: K
authors.initials: K
authors.lastname: Lindsley
authors.forename: T E
authors.initials: TE
authors.lastname: Merchant
authors.forename: B
authors.initials: B
authors.lastname: O'Malley
authors.forename: L
authors.initials: L
authors.lastname: Bayer
authors.forename: M M
authors.initials: MM
authors.lastname: Petriccione
authors.forename: J
authors.initials: J
authors.lastname: Allen
authors.forename: J L
authors.initials: JL
authors.lastname: Finlay
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation; D001932:Brain Neoplasms / Q000188:drug therapy* / Q000601:surgery*; D002675:Child, Preschool; D002945:Cisplatin / Q000008:administration & dosage; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D018450:Disease Progression; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D009364:Neoplasm Recurrence, Local; D009503:Neutropenia / Q000139:chemically induced; D012074:Remission Induction; D013921:Thrombocytopenia / Q000139:chemically induced; D014182:Transplantation, Autologous; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D014750:Vincristine; D005047:Etoposide; D003520:Cyclophosphamide; D002945:Cisplatin