title: Assessment of the treatment approach and survival outcomes in a modern cohort of patients with atypical teratoid rhabdoid tumors using the National Cancer Database.
abstract: Atypical teratoid rhabdoid tumors (ATRTs) are rare brain tumors that occur primarily in children under the age of 3 years. This report evaluates the treatment approach and survival outcomes in a large cohort of patients treated in the United States.
  
  
  
  Using the National Cancer Database, the analysis included all ATRT patients aged 0 to 18 years who were diagnosed between 2004 and 2012 and had complete treatment data.
  
  
  
  Three hundred sixty-one ATRT patients were evaluated. The 5-year overall survival (OS) rate was 29.9%, and it was significantly lower for children who were less than 3 years old (5-year OS, 27.7%) versus those who were 3 years old or older (5-year OS, 37.5%; P < .001). The best outcome was seen for patients with localized disease who received trimodality therapy (surgery, chemotherapy, and radiation therapy [RT]) with a 5-year OS rate of 46.8%. The utilization of trimodality therapy significantly increased during the study period (27.7% in 2004-2008 vs 45.1% in 2009-2012; P < .01), largely because of the increased use of RT. In a multivariate analysis, treatment that did not utilize trimodality therapy was associated with significantly worse OS (hazard ratio, 2.52; 95% confidence interval (1.82-3.51). Children aged 0 to 2 years were significantly less likely to receive trimodality therapy because of decreased utilization of RT in this age group.
  
  
  
  The use of trimodality therapy significantly increased during the study period and was associated with improved outcomes. For patients with localized disease who received trimodality therapy, the OS rate at 5 years approached 50%. However, further research into the optimal management of children less than 3 years old is needed because of their significantly worse OS in comparison with older children. Cancer 2017;123:682-687. © 2016 American Cancer Society.
authors.affiliation: Department of Radiation Oncology, Washington University School of Medicine, St. Louis, Missouri.
authors.forename: Benjamin W
authors.initials: BW
authors.lastname: Fischer-Valuck
authors.affiliation: Department of Radiation Oncology, Washington University School of Medicine, St. Louis, Missouri.
authors.forename: Ishita
authors.initials: I
authors.lastname: Chen
authors.affiliation: Department of Radiation Oncology, Washington University School of Medicine, St. Louis, Missouri.
authors.forename: Amar J
authors.initials: AJ
authors.lastname: Srivastava
authors.affiliation: Department of Radiation Oncology, Washington University School of Medicine, St. Louis, Missouri.
authors.forename: John M
authors.initials: JM
authors.lastname: Floberg
authors.affiliation: Department of Radiation Oncology, Washington University School of Medicine, St. Louis, Missouri.
authors.forename: Yuan James
authors.initials: YJ
authors.lastname: Rao
authors.affiliation: Department of Pediatric Hematology and Oncology, Washington University School of Medicine, St. Louis, Missouri.
authors.forename: Allison A
authors.initials: AA
authors.lastname: King
authors.affiliation: Department of Radiation Oncology, Vanderbilt University, Nashville, Tennessee.
authors.forename: Eric T
authors.initials: ET
authors.lastname: Shinohara
authors.affiliation: Department of Radiation Oncology, Washington University School of Medicine, St. Louis, Missouri.
authors.forename: Stephanie M
authors.initials: SM
authors.lastname: Perkins
mesh_terms: D000293:Adolescent; D016543:Central Nervous System Neoplasms / Q000188:drug therapy / Q000453:epidemiology* / Q000532:radiotherapy / Q000601:surgery; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D016208:Databases, Factual; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D016016:Proportional Hazards Models; D018335:Rhabdoid Tumor / Q000188:drug therapy / Q000453:epidemiology* / Q000532:radiotherapy / Q000601:surgery; D016019:Survival Analysis; D013724:Teratoma / Q000188:drug therapy / Q000453:epidemiology* / Q000532:radiotherapy / Q000601:surgery; D016896:Treatment Outcome
keywords: National Cancer Database (NCDB); atypical teratoid rhabdoid tumor (ATRT); pediatric oncology