title: Outcome of 11 children with ependymoblastoma treated within the prospective HIT-trials between 1991 and 2006.
abstract: Ependymoblastoma is a rare malignant brain tumor of early childhood. Data on clinical behavior and optimal treatment strategies are scarce. We report on 11 consecutively treated children with centrally confirmed diagnosis of CNS ependymoblastoma, registered between February 1994 and October 2006 to the prospective GPOH-HIT multicenter brain tumor trials, and treated by multimodal regimens. Median age at diagnosis was 3.5 years (range, 1.8-5.6 years), and the median follow-up of survivors was 5.9 years (range, 2.2-12.7 years). Initial stage was M0 in 9, and M0/1 (no cerebrospinal fluid examination done) in 2 patients. Gross-total tumor resection was achieved in 7 patients, incomplete resection in 4 patients. Further primary therapy included chemotherapy in all patients, craniospinal radiotherapy in 5 patients and high-dose chemotherapy in 2 patients. Tumor response to chemotherapy was observed in 1 of 4 evaluable patients. Tumor progression occurred in 7 patients after a median time of 5.0 months (range, 2.5-19.2 months). Five-year progression-free survival was 36.4% (±14.5%), 5-year overall survival 30.3% (±15.9%). Of 4 survivors, 3 had gross-total tumor resection, and all were treated by either craniospinal radiotherapy and/or high-dose chemotherapy with autologous blood stem cell rescue. Prognosis of children with ependymoblastoma is poor, but sustained remissions have been achieved after multimodal treatment. Considerable diagnostic discrepancies between local and central pathologists underscore the importance of central review. Further studies are needed to improve survival of children with this rare malignant central nervous system tumor.
authors.affiliation: Department of Oncology, University Children's Hospital, 8032 Zurich, Switzerland. nicolas.gerber@kispi.uzh.ch
authors.forename: Nicolas U
authors.initials: NU
authors.lastname: Gerber
authors.forename: Katja
authors.initials: K
authors.lastname: von Hoff
authors.forename: André O
authors.initials: AO
authors.lastname: von Bueren
authors.forename: Wiebke
authors.initials: W
authors.lastname: Treulieb
authors.forename: Monika
authors.initials: M
authors.lastname: Warmuth-Metz
authors.forename: Torsten
authors.initials: T
authors.lastname: Pietsch
authors.forename: Niels
authors.initials: N
authors.lastname: Soerensen
authors.forename: Andreas
authors.initials: A
authors.lastname: Faldum
authors.forename: Angela
authors.initials: A
authors.lastname: Emser
authors.forename: Paul G
authors.initials: PG
authors.lastname: Schlegel
authors.forename: Frank
authors.initials: F
authors.lastname: Deinlein
authors.forename: Rolf-Dieter
authors.initials: RD
authors.lastname: Kortmann
authors.forename: Stefan
authors.initials: S
authors.lastname: Rutkowski
mesh_terms: D000293:Adolescent; D000970:Antineoplastic Agents / Q000627:therapeutic use; D001932:Brain Neoplasms / Q000175:diagnosis / Q000401:mortality / Q000601:surgery; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D016371:Cranial Irradiation / Q000379:methods; D006801:Humans; D007223:Infant; D053208:Kaplan-Meier Estimate; D008137:Longitudinal Studies; D008279:Magnetic Resonance Imaging; D008297:Male; D018242:Neuroectodermal Tumors, Primitive / Q000175:diagnosis / Q000401:mortality / Q000601:surgery; D009493:Neurosurgery / Q000379:methods; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents