title: Outcome of infants and young children with newly diagnosed ependymoma treated on the "Head Start" III prospective clinical trial.
abstract: This study investigates the outcome of children <10 years old with newly-diagnosed ependymoma treated on the prospective multinational "Head Start" III clinical trial. Between April 2004 and July 2009, 19 children with newly-diagnosed ependymoma were enrolled. All children were to receive five induction chemotherapy cycles followed by one consolidation cycle of myelo-ablative chemotherapy and autologous hematopoietic cell rescue. Children between 6 and 10 years of age or with residual tumor prior to consolidation were to receive irradiation thereafter. Median age of 19 children (8 female) was 20 months at diagnosis. Median follow up was 44 months. The primary site was infratentorial in 11 and supratentorial in 8 patients. Gross total resection was achieved in 10 patients. After induction chemotherapy, all three supratentorial ependymoma patients with residual disease achieved a complete response (CR), while only one of six infratentorial patients with residual disease achieved CR. Three infratentorial patients developed progressive disease during induction chemotherapy. All four infratentorial patients with residual disease who underwent autologous hematopoietic cell transplant, failed to achieve CR. Four patients received focal irradiation following chemotherapy. The 3-year event free survival (EFS) and overall survival (OS) for supratentorial ependymoma were 86 ± 13 % and 100 % respectively. The 3-year EFS and OS for infratentorial ependymoma were 27 ± 13 % and 73 ± 13 % respectively. The role of intensive induction and consolidation chemotherapy in deferring irradiation should be investigated further in children with supratentorial ependymoma with residual disease following surgery. This approach appears ineffective in children with infratentorial ependymoma in the absence of irradiation.
authors.affiliation: Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, CA 90027, USA. rvenkatramani@chla.usc.edu
authors.forename: Rajkumar
authors.initials: R
authors.lastname: Venkatramani
authors.forename: Lingyun
authors.initials: L
authors.lastname: Ji
authors.forename: Joseph
authors.initials: J
authors.lastname: Lasky
authors.forename: Kelley
authors.initials: K
authors.lastname: Haley
authors.forename: Alexander
authors.initials: A
authors.lastname: Judkins
authors.forename: Shengmei
authors.initials: S
authors.lastname: Zhou
authors.forename: Richard
authors.initials: R
authors.lastname: Sposto
authors.forename: Randal
authors.initials: R
authors.lastname: Olshefski
authors.forename: James
authors.initials: J
authors.lastname: Garvin
authors.forename: Tanya
authors.initials: T
authors.lastname: Tekautz
authors.forename: Gloria
authors.initials: G
authors.lastname: Kennedy
authors.forename: Shahrad Rod
authors.initials: SR
authors.lastname: Rassekh
authors.forename: Theodore
authors.initials: T
authors.lastname: Moore
authors.forename: Sharon
authors.initials: S
authors.lastname: Gardner
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Allen
authors.forename: Richard
authors.initials: R
authors.lastname: Shore
authors.forename: Christopher
authors.initials: C
authors.lastname: Moertel
authors.forename: Mark
authors.initials: M
authors.lastname: Atlas
authors.forename: Girish
authors.initials: G
authors.lastname: Dhall
authors.forename: Jonathan
authors.initials: J
authors.lastname: Finlay
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000175:diagnosis / Q000401:mortality* / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D002945:Cisplatin / Q000008:administration & dosage; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D004806:Ependymoma / Q000175:diagnosis / Q000401:mortality* / Q000628:therapy; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D002955:Leucovorin / Q000008:administration & dosage; D008297:Male; D008727:Methotrexate / Q000008:administration & dosage; D011379:Prognosis; D011446:Prospective Studies; D011879:Radiotherapy Dosage; D015996:Survival Rate; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D014750:Vincristine; D005047:Etoposide; D003520:Cyclophosphamide; D002945:Cisplatin; D002955:Leucovorin; D008727:Methotrexate