title: Prognostic factors and survival in non-central nervous system rhabdoid tumors.
abstract: BACKGROUND
  Non-central nervous system (non-CNS) rhabdoid tumors tend to present at a young age and have an extremely aggressive course, with dismal overall survival rates. Inactivation of the tumor suppressor gene SMARCB1 has been shown in rhabdoid tumors regardless of anatomic location, suggesting a common genetic basis. We retrospectively analyzed our institutional experience with non-CNS rhabdoid tumors to determine overall survival and prognostic variables.
  
  
  METHODS
  We reviewed records of pediatric patients (age<22y) with non-CNS rhabdoid tumor at our institution between 1980 and 2014. Variables evaluated for correlation with survival included: age > or <1.5years (median) at diagnosis, M1 status, and radiation therapy. The log-rank test was used to compare Kaplan-Meier probability distributions with P values adjusted for multiple testing using the false discovery rate approach.
  
  
  RESULTS
  Nineteen consecutive patients (10 female) with histologically verified rhabdoid tumor were identified. Mean age at diagnosis was 3.2years (median 1.5y, range 1.3mo-21.8y). Primary tumors were located in the kidney (n=10), head and neck (n=5), and in the liver, thigh, mediastinum and retroperitoneum (n=1 each). SMARCB1 expression was absent in all 10 patients tested. Eight patients had distant metastases at diagnosis. Median overall survival was 1.2years. Age greater than the median and radiation therapy were associated with better outcome, with a median overall survival of 2.7years (P=0.049 and P=0.003, respectively).
  
  
  CONCLUSIONS
  Survival rates for rhabdoid tumor remain poor, but prognosis is better in older children, regardless of primary tumor location. Because of its rarity, clinical trials with present agents are difficult to conduct. Further progress will require a focus on therapies targeted at tumor biology rather than anatomic location for non-CNS rhabdoid tumors.
authors.affiliation: Pediatric Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10065.
authors.forename: Benjamin A
authors.initials: BA
authors.lastname: Farber
authors.affiliation: Department of Pediatrics, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10065.
authors.forename: Neerav
authors.initials: N
authors.lastname: Shukla
authors.affiliation: Pediatric Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10065.
authors.forename: Irene Isabel P
authors.initials: II
authors.lastname: Lim
authors.affiliation: Pediatric Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10065.
authors.forename: Jennifer M
authors.initials: JM
authors.lastname: Murphy
authors.affiliation: Pediatric Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10065. Electronic address: laquaglm@mskcc.org.
authors.forename: Michael P
authors.initials: MP
authors.lastname: La Quaglia
mesh_terms: D017668:Age of Onset; D002648:Child; D002675:Child, Preschool; D005260:Female; D006258:Head and Neck Neoplasms / Q000401:mortality*; D006801:Humans; D007223:Infant; D053208:Kaplan-Meier Estimate; D007680:Kidney Neoplasms / Q000401:mortality*; D008297:Male; D008479:Mediastinal Neoplasms / Q000401:mortality; D011379:Prognosis; D012189:Retrospective Studies; D018335:Rhabdoid Tumor / Q000401:mortality* / Q000556:secondary; D015996:Survival Rate; D055815:Young Adult
keywords: Pediatric cancer; Rhabdoid tumor; SMARCB1