title: Phase II study of pre-irradiation chemotherapy for childhood intracranial ependymoma. Children's Cancer Group protocol 9942: a report from the Children's Oncology Group.
abstract: OBJECTIVE
  Standard therapy for childhood intracranial ependymoma is maximal tumor resection followed by involved-field irradiation. Although not used routinely, chemotherapy has produced objective responses in ependymoma, both at recurrence and in infants. Because the presence of residual tumor following surgery is consistently associated with inferior outcome, the potential impact of pre-irradiation chemotherapy was investigated.
  
  
  METHODS
  Between 1995 and 1999, the Children's Cancer Group undertook a Phase II trial of pre-irradiation chemotherapy in children 3-21 years of age with intracranial ependymoma and radiological evidence of post-operative residual tumor.
  
  
  RESULTS
  Of 84 patients, 41 had residual tumor, and were given four cycles of cisplatin-based chemotherapy prior to irradiation. Of 35 patients fully evaluable for response to chemotherapy, 14 (40%) demonstrated complete response, 6 (17%) partial response, 10 (29%) minor response or stable disease, and 5 (14%) demonstrated progressive tumor growth. For the entire group, 5-year overall survival (OS) and event-free survival (EFS) was 71 ± 6%, and 57 ± 6%, respectively. The pre-irradiation chemotherapy group demonstrated EFS comparable to that of patients with no residual tumor who received irradiation alone (55 ± 8% vs. 58 ± 9%, P = 0.45). Any benefit of chemotherapy was restricted to patients with greater than 90% tumor resection.
  
  
  CONCLUSIONS
  Children with near total resection of ependymoma may benefit from pre-irradiation chemotherapy. Patients with subtotal resection have inferior outcome despite responses to chemotherapy, and should be considered for second-look surgery prior to irradiation. Pediatr Blood Cancer 2012; 59: 1183-1189. © 2012 Wiley Periodicals, Inc.
authors.affiliation: Division of Pediatric Oncology, Columbia University Medical Center, New York, New York.
authors.forename: James H
authors.initials: JH
authors.lastname: Garvin
authors.forename: Michael T
authors.initials: MT
authors.lastname: Selch
authors.forename: Emi
authors.initials: E
authors.lastname: Holmes
authors.forename: Mitchell S
authors.initials: MS
authors.lastname: Berger
authors.forename: Jonathan L
authors.initials: JL
authors.lastname: Finlay
authors.forename: Ann
authors.initials: A
authors.lastname: Flannery
authors.forename: Joel W
authors.initials: JW
authors.lastname: Goldwein
authors.forename: Roger J
authors.initials: RJ
authors.lastname: Packer
authors.forename: Lucy B
authors.initials: LB
authors.lastname: Rorke-Adams
authors.forename: Tania
authors.initials: T
authors.lastname: Shiminski-Maher
authors.forename: Richard
authors.initials: R
authors.lastname: Sposto
authors.forename: Philip
authors.initials: P
authors.lastname: Stanley
authors.forename: Raymond
authors.initials: R
authors.lastname: Tannous
authors.forename: Ian F
authors.initials: IF
authors.lastname: Pollack
mesh_terms: D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy*; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D004806:Ependymoma / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy*; D005260:Female; D006801:Humans; D008297:Male; D020360:Neoadjuvant Therapy; D015996:Survival Rate; D055815:Young Adult