title: Intensive multimodality treatment for children with newly diagnosed CNS atypical teratoid rhabdoid tumor.
abstract: OBJECTIVE
  Atypical teratoid rhabdoid tumor (ATRT) of the CNS is a highly malignant neoplasm primarily affecting young children, with a historic median survival ranging from 6 to 11 months. Based on a previous pilot series, a prospective multi-institutional trial was conducted for patients with newly diagnosed CNS ATRT.
  
  
  METHODS
  Treatment was divided into five phases: preirradiation, chemoradiation, consolidation, maintenance, and continuation therapy. Intrathecal chemotherapy was administered, alternating intralumbar and intraventricular routes. Radiation therapy (RT) was prescribed, either focal (54 Gy) or craniospinal (36 Gy, plus primary boost), depending on age and extent of disease at diagnosis.
  
  
  RESULTS
  Between 2004 and 2006, 25 patients were enrolled; 20 were eligible for evaluation. Median age at diagnosis was 26 months (range, 2.4 months to 19.5 years). Gross total resection of the primary tumor was achieved in 11 patients. Fourteen patients had M0 disease at diagnosis, one patient had M2 disease, and five patients had M3 disease. Fifteen patients received radiation therapy: 11 focal and four craniospinal. Significant toxicities, in addition to the expected, included radiation recall (n = 2) and transverse myelitis (n = 1). There was one toxic death. Of the 12 patients who were assessable for chemotherapeutic response (pre-RT), the objective response rate was 58%. The objective response rate observed after RT was 38%. The 2-year progression-free and overall survival rates are 53% +/- 13% and 70% +/- 10%, respectively. Median overall survival has not yet been reached.
  
  
  CONCLUSIONS
  This intensive multimodality regimen has resulted in a significant improvement in time to progression and overall survival for patients with this previously poor-prognosis tumor.
authors.affiliation: Dana-Farber Cancer Institute, Children's Hospital Boston, Boston, MA, USA. susan_chi@dfci.harvard.edu
authors.forename: Susan N
authors.initials: SN
authors.lastname: Chi
authors.forename: Mary Ann
authors.initials: MA
authors.lastname: Zimmerman
authors.forename: Xiaopan
authors.initials: X
authors.lastname: Yao
authors.forename: Kenneth J
authors.initials: KJ
authors.lastname: Cohen
authors.forename: Peter
authors.initials: P
authors.lastname: Burger
authors.forename: Jaclyn A
authors.initials: JA
authors.lastname: Biegel
authors.forename: Lucy B
authors.initials: LB
authors.lastname: Rorke-Adams
authors.forename: Michael J
authors.initials: MJ
authors.lastname: Fisher
authors.forename: Anna
authors.initials: A
authors.lastname: Janss
authors.forename: Claire
authors.initials: C
authors.lastname: Mazewski
authors.forename: Stewart
authors.initials: S
authors.lastname: Goldman
authors.forename: Peter E
authors.initials: PE
authors.lastname: Manley
authors.forename: Daniel C
authors.initials: DC
authors.lastname: Bowers
authors.forename: Anne
authors.initials: A
authors.lastname: Bendel
authors.forename: Joshua
authors.initials: J
authors.lastname: Rubin
authors.forename: Christopher D
authors.initials: CD
authors.lastname: Turner
authors.forename: Karen J
authors.initials: KJ
authors.lastname: Marcus
authors.forename: Liliana
authors.initials: L
authors.lastname: Goumnerova
authors.forename: Nicole J
authors.initials: NJ
authors.lastname: Ullrich
authors.forename: Mark W
authors.initials: MW
authors.lastname: Kieran
mesh_terms: D000293:Adolescent; D001932:Brain Neoplasms / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D006801:Humans; D015192:Infratentorial Neoplasms; D011379:Prognosis; D011446:Prospective Studies; D018335:Rhabdoid Tumor / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000628:therapy*; D015173:Supratentorial Neoplasms; D013724:Teratoma / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000628:therapy*; D055815:Young Adult