Prognostic factors and survival in non-central nervous system rhabdoid tumors Journal Article


Authors: Farber, B. A.; Shukla, N.; Lim, I. I. P.; Murphy, J. M.; La Quaglia, M. P.
Article Title: Prognostic factors and survival in non-central nervous system rhabdoid tumors
Abstract: Introduction: 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 < 22 y) with non-CNS rhabdoid tumor at our institution between 1980 and 2014. Variables evaluated for correlation with survival included: age > or < 1.5 years (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 3.2 years (median 1.5 y, range 1.3 mo–21.8 y). 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 1.2 years. Age greater than the median and radiation therapy were associated with better outcome, with a median overall survival of 2.7 years (P = 0.049 and P = 0.003, respectively). Conclusion: 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. © 2016
Keywords: child; preschool child; child, preschool; survival rate; retrospective studies; young adult; mortality; retrospective study; kidney neoplasms; kidney tumor; infant; head and neck neoplasms; kaplan meier method; onset age; age of onset; mediastinum tumor; mediastinal neoplasms; head and neck tumor; rhabdoid tumor; kaplan-meier estimate; pediatric cancer; secondary; smarcb1; humans; prognosis; human; male; female
Journal Title: Journal of Pediatric Surgery
Volume: 52
Issue: 3
ISSN: 0022-3468
Publisher: W.B. Saunders Co-Elsevier Inc.  
Date Published: 2017-03-01
Start Page: 373
End Page: 376
Language: English
DOI: 10.1016/j.jpedsurg.2016.08.017
PUBMED: 27639430
PROVIDER: scopus
PMCID: PMC5535760
DOI/URL:
Notes: Article -- Export Date: 1 May 2018 -- Source: Scopus
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MSK Authors
  1. Neerav Shukla
    93 Shukla
  2. Jennifer Michelle Murphy
    11 Murphy
  3. Benjamin Asher Farber
    12 Farber