title: Outcome for children <4 years of age with malignant central nervous system tumors treated with high-dose chemotherapy and autologous stem cell rescue.
abstract: BACKGROUND
  Children <4 years of age (yo) with malignant central nervous system (CNS) tumors have a dismal prognosis. In an attempt to delay or obviate radiation therapy (XRT) and improve outcome, our institution has treated children <4 yo with newly diagnosed malignant CNS tumors with high-dose chemotherapy (HDC) and autologous stem cell rescue (ASCR) followed by selective XRT.
  
  
  METHODS
  Fifteen children (age 4-38 months) with malignant CNS tumors have completed treatment with HDC/ASCR. All patients received three cycles of induction chemotherapy (cisplatin 3.5 mg/kg- day 0, cyclophosphamide 60 mg/kg- day 1 and 2, etoposide 2.5 mg/kg- day 0-2, vincristine 0.05 mg/kg, day 0, 7, 14) followed by three cycles of HDC (carboplatin 17 mg/kg and thiotepa 6 mg/kg, day 0 and 1) with ASCR. Histology included five medulloblastomas, four primitive neuroectodermal tumors (PNET), five malignant gliomas, and one ependymoma. Outcome and treatment toxicities were evaluated by retrospective chart review.
  
  
  RESULTS
  Median follow-up time of the 15 patients is 22 months (range 8-82 months). The 1- and 2-year progression-free survival (PFS) is 86.1% and 52.2% and overall survival (OS) 91.6% and 72.1%, respectively. Ten patients are alive and disease free 3-77 months (median 18 months) after having completed HDC/ASCR, thereoff five received XRT. Toxicity was primarily myelosuppression. There was no treatment mortality.
  
  
  CONCLUSIONS
  We are encouraged by the outcome of 15 children <4 yo with malignant CNS tumors treated with tandem cycles of HDC and ASCR at our institution. The treatment regimen is relatively well tolerated.
authors.affiliation: Division of Hematology and Oncology, Children's National Medical Center, Washington, District of Columbia, USA.
authors.forename: Halldora K
authors.initials: HK
authors.lastname: Thorarinsdottir
authors.forename: Brian
authors.initials: B
authors.lastname: Rood
authors.forename: Naynesh
authors.initials: N
authors.lastname: Kamani
authors.forename: Debbie
authors.initials: D
authors.lastname: Lafond
authors.forename: Evelio
authors.initials: E
authors.lastname: Perez-Albuerne
authors.forename: Brett
authors.initials: B
authors.lastname: Loechelt
authors.forename: Roger J
authors.initials: RJ
authors.lastname: Packer
authors.forename: Tobey J
authors.initials: TJ
authors.lastname: MacDonald
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000150:complications / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000601:surgery / Q000628:therapy*; D016190:Carboplatin / Q000008:administration & dosage; D002675:Child, Preschool; D002945:Cisplatin / Q000008:administration & dosage; D003072:Cognition Disorders / Q000209:etiology; D016371:Cranial Irradiation / Q000009:adverse effects; D003520:Cyclophosphamide / Q000008:administration & dosage; D002658:Developmental Disabilities / Q000209:etiology; D018572:Disease-Free Survival; D004806:Ependymoma / Q000188:drug therapy / Q000401:mortality / Q000601:surgery; D005047:Etoposide / Q000008:administration & dosage; D005500:Follow-Up Studies; D018303:Ganglioglioma / Q000188:drug therapy / Q000378:metabolism / Q000532:radiotherapy / Q000601:surgery; D005910:Glioma / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000601:surgery; D006801:Humans; D007223:Infant; D008527:Medulloblastoma / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000601:surgery; D008940:Mitotic Index; D018242:Neuroectodermal Tumors, Primitive / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000601:surgery; D009837:Oligodendroglioma / Q000188:drug therapy / Q000401:mortality / Q000601:surgery / Q000628:therapy*; D036102:Peripheral Blood Stem Cell Transplantation / Q000009:adverse effects; D011782:Quadriplegia / Q000209:etiology; D012189:Retrospective Studies; D012678:Sensation Disorders / Q000209:etiology; D013120:Spinal Cord Neoplasms / Q000188:drug therapy / Q000601:surgery; D013852:Thiotepa / Q000008:administration & dosage; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D014750:Vincristine; D005047:Etoposide; D003520:Cyclophosphamide; D013852:Thiotepa; D016190:Carboplatin; D002945:Cisplatin