Desmoplastic small round cell tumors with EWS-WT1 fusion transcript in children and young adults.

BACKGROUND: The presence of the EWS-WT1 gene fusion transcript (GFT) is characteristic of desmoplastic small round cell tumor (DSRCT), a rare and very aggressive disease for which the treatment has not yet been clearly standardized. METHODS: This was a retrospective national multicenter analysis of...

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Published in:Pediatric Blood & Cancer Vol. 58; no. 6; pp. 891 - 898
Main Authors: Philippe-Chomette P, Kabbara N, Andre N, Pierron G, Coulomb A, Laurence V, Blay JY, Delattre O, Schleiermacher G, Orbach D
Format: research Journal Article
Published: Wiley-Blackwell Jun2012
Online Access:View this record in EBSCOhost
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      dt: Jun2012
      vid: 58
      iid: 6
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      pub: Wiley-Blackwell
      place: Malden, Massachusetts
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        2011493951
        10.1002/pbc.23403
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        atl: Desmoplastic small round cell tumors with EWS-WT1 fusion transcript in children and young adults.
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          Philippe-Chomette P
          Kabbara N
          Andre N
          Pierron G
          Coulomb A
          Laurence V
          Blay JY
          Delattre O
          Schleiermacher G
          Orbach D
        affil: Department of Pediatric Surgery, Hôpital Robert Debré - Assistance Publique, Paris, France.
      sug:
        subj:
          Sarcoma
          Sarcoma Mortality
          Sarcoma Surgery
          Proteins
          Adolescence
          Adult
          Antineoplastic Agents, Combined
          Child
          Child, Preschool
          Combined Modality Therapy
          Prognosis
          Female
          Human
          Kaplan-Meier Estimator
          Male
          Radiotherapy
          Polymerase Chain Reaction
          Retrospective Design
          Reverse Transcriptase Polymerase Chain Reaction
          Treatment Outcomes
          Young Adult
          Adolescent: 13-18 years
          Adult: 19-44 years
          Child: 6-12 years
          Child, Preschool: 2-5 years
          Female
          Male
      ab: BACKGROUND: The presence of the EWS-WT1 gene fusion transcript (GFT) is characteristic of desmoplastic small round cell tumor (DSRCT), a rare and very aggressive disease for which the treatment has not yet been clearly standardized. METHODS: This was a retrospective national multicenter analysis of young patients <30 years with tumors expressing the EWS-WT1-GFT, designed to determine whether extensive surgery had an impact on survival. RESULTS: Between 1995 and 2006, a EWS-WT1-GFT was detected in the tumors of 38 patients, 17 (44.7%) of whom had had a different initial pathologic diagnosis prior to molecular testing. Mean age was 13.2 years (range: 4-29.7 years). Only 9 patients (24%) had localized disease. Treatment was heterogeneous. Nine patients had 'limited' surgical resections and 22 underwent 'extensive' surgery. Two-year event-free survival and overall survival were 14.4% and 50%, respectively. Among the five patients who were alive in complete remission, four had undergone extensive and complete surgery. CONCLUSIONS: Detection of the EWS-WT1-GFT plays a major role in the diagnosis of DSRCT. No survival difference was observed according to extent of surgery, but complete surgery seemed to offer the best chance of long-term survival. High-dose chemotherapy or local radiotherapy did not appear to improve survival in this retrospective analysis, but larger prospective studies are needed to provide definitive conclusions on the role of these treatments. Pediatr Blood Cancer 2012; 58: 891-897. © 2011 Wiley Periodicals, Inc.
      pubtype: Academic Journal
      doctype:
        research
        Journal Article
      ougenre: Article
    language: English
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