Document Detail


Characteristics and outcomes of children with the Wilms tumor-Aniridia syndrome: a report from the National Wilms Tumor Study Group.
MedLine Citation:
PMID:  14673045     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
PURPOSE: Children with the rare Wilms tumor (WT)-aniridia (WAGR) syndrome have not had systematic evaluation of their clinical and pathologic features. We compared demographics, disease characteristics, and treatment outcomes in a large cohort of WT patients who did or did not have the WAGR syndrome. PATIENTS AND METHODS: Clinical and pathology records were reviewed for 8,533 patients enrolled between 1969 and 2002 by the National Wilms Tumor Study Group. RESULTS: Sixty-four patients (0.75%) had the WAGR syndrome. For WAGR and non-WAGR patients, respectively, the average birth weights (2.94 and 3.45 kg), median ages at diagnosis (22 and 39 months), and the percentages with bilateral disease (17% and 6%), metastatic disease (2% and 13%), favorable histology (FH) tumors (100% and 92%), and intralobar nephrogenic rests (ILNR; 77% and 22%) all differed. Survival estimates for WAGR and non-WAGR patients were 95% +/- 3% and 92% +/- 0.3% at 4 years but 48% +/- 17% and 86% +/- 1.0%, respectively, at 27 years from diagnosis. Five late deaths in WAGR patients were from end-stage renal disease (ESRD). CONCLUSION: The excess of bilateral disease, ILNR-associated FH tumors of mixed cell type, and early ages at diagnosis in WAGR patients all fit the known phenotypic spectrum of constitutional deletion of chromosome 11p13. Despite a favorable response of their WT to treatment, WAGR patients have a high risk of ESRD as they approach adulthood. The renal pathology associated with this apparent late manifestation of WT1 deletion, and the explanation for the abnormally low birth weights in patients with del 11p13, have yet to be determined.
Authors:
Norman E Breslow; Robin Norris; Patricia A Norkool; Tammy Kang; J Bruce Beckwith; Elizabeth J Perlman; Michael L Ritchey; Daniel M Green; Kim E Nichols;
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Publication Detail:
Type:  Journal Article; Research Support, U.S. Gov't, P.H.S.    
Journal Detail:
Title:  Journal of clinical oncology : official journal of the American Society of Clinical Oncology     Volume:  21     ISSN:  0732-183X     ISO Abbreviation:  J. Clin. Oncol.     Publication Date:  2003 Dec 
Date Detail:
Created Date:  2003-12-15     Completed Date:  2004-02-13     Revised Date:  2007-11-14    
Medline Journal Info:
Nlm Unique ID:  8309333     Medline TA:  J Clin Oncol     Country:  United States    
Other Details:
Languages:  eng     Pagination:  4579-85     Citation Subset:  IM    
Affiliation:
Department of Biostatistics, Box 357232, University of Washington, Seattle, WA 98195-7232, USA. norm@u.washington.edu
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MeSH Terms
Descriptor/Qualifier:
Age Distribution
Analysis of Variance
Child
Child, Preschool
Female
Humans
Infant
Infant, Newborn
Male
Proportional Hazards Models
Retrospective Studies
Survival Analysis
Treatment Outcome
WAGR Syndrome / mortality,  pathology*,  therapy
Grant Support
ID/Acronym/Agency:
CA 42326/CA/NCI NIH HHS; CA 54498/CA/NCI NIH HHS

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