title: Outcome for young children newly diagnosed with ependymoma, treated with intensive induction chemotherapy followed by myeloablative chemotherapy and autologous stem cell rescue.
abstract: BACKGROUND
  The purpose of this study is to investigate the efficacy of an intensive chemotherapy induction regimen followed by myeloablative chemotherapy and autologous hematopoietic stem cell rescue (AHSCR) in children with newly diagnosed ependymoma.
  
  
  METHODS
  Twenty-nine children less than 10 years of age at diagnosis of ependymoma were enrolled on the "Head Start" studies. Twenty-four patients with localized disease received an induction regimen including five cycles of chemotherapy (cisplatin, vincristine, etoposide cyclophosphamide, and high dose methotrexate for patients with metastatic disease). Following induction, individuals without evidence of disease proceeded to marrow-ablative chemotherapy (thiotepa, carboplatin, and etoposide) with AHSCR.
  
  
  RESULTS
  The estimated 5-year event free survival (EFS) and overall survival (OS) from diagnosis were 12% (+/-6%) and 38% (+/-10%), respectively. The toxic mortality amongst this group of 29 patients was 10.3%. Younger age (less than 18 months at diagnosis) was the only statistically significant prognostic factor. The estimated 5-year OS rate for the five patients with metastatic disease at presentation was 80% (+/-18%). Overall, radiation-free survival at 5 years from diagnosis was 8% (+/-5%).
  
  
  CONCLUSIONS
  The use of an intensive induction chemotherapy regimen including myeloablative chemotherapy followed by AHSCR in newly diagnosed young children with ependymoma is not superior to other previously reported chemotherapeutic strategies.
authors.affiliation: Department of Pediatric Hematology/Oncology, Children's Hospital Los Angdes, Neural Tumors Program, Los Angles, CA 900 27, USA. szacharoulis@chla.usc.edu
authors.forename: Stergios
authors.initials: S
authors.lastname: Zacharoulis
authors.forename: Adam
authors.initials: A
authors.lastname: Levy
authors.forename: Susan N
authors.initials: SN
authors.lastname: Chi
authors.forename: Sharon
authors.initials: S
authors.lastname: Gardner
authors.forename: Marc
authors.initials: M
authors.lastname: Rosenblum
authors.forename: Douglas C
authors.initials: DC
authors.lastname: Miller
authors.forename: Ira
authors.initials: I
authors.lastname: Dunkel
authors.forename: Blanca
authors.initials: B
authors.lastname: Diez
authors.forename: Richard
authors.initials: R
authors.lastname: Sposto
authors.forename: Lingyun
authors.initials: L
authors.lastname: Ji
authors.forename: Shahab
authors.initials: S
authors.lastname: Asgharzadeh
authors.forename: Juliette
authors.initials: J
authors.lastname: Hukin
authors.forename: Jean
authors.initials: J
authors.lastname: Belasco
authors.forename: Ronald
authors.initials: R
authors.lastname: Dubowy
authors.forename: Stewart
authors.initials: S
authors.lastname: Kellie
authors.forename: Amanda
authors.initials: A
authors.lastname: Termuhlen
authors.forename: Jonathan
authors.initials: J
authors.lastname: Finlay
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000175:diagnosis / Q000628:therapy*; D016190:Carboplatin / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D002945:Cisplatin / Q000008:administration & dosage; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D018572:Disease-Free Survival; D004806:Ependymoma / Q000175:diagnosis / Q000628:therapy*; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D008727:Methotrexate / Q000008:administration & dosage; D009364:Neoplasm Recurrence, Local / Q000628:therapy; 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; D008727:Methotrexate