{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,28]],"date-time":"2026-07-28T02:14:39Z","timestamp":1785204879749,"version":"3.55.0"},"reference-count":31,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2020,11,4]],"date-time":"2020-11-04T00:00:00Z","timestamp":1604448000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100006244","name":"Stichting Kinderen Kankervrij","doi-asserted-by":"publisher","award":["278"],"award-info":[{"award-number":["278"]}],"id":[{"id":"10.13039\/501100006244","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["acsjournals.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Cancer"],"published-print":{"date-parts":[[2021,2,15]]},"abstract":"<jats:sec><jats:title>Background<\/jats:title><jats:p>WAGR syndrome (Wilms tumor, aniridia, genitourinary anomalies, and range of developmental delays) is a rare contiguous gene deletion syndrome with a 45% to 60% risk of developing Wilms tumor (WT). Currently, surveillance and treatment recommendations are based on limited evidence.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Clinical characteristics, treatments, and outcomes were analyzed for patients with WAGR and WT\/nephroblastomatosis who were identified through International Society of Pediatric Oncology Renal Tumor Study Group (SIOP\u2010RTSG) registries and the SIOP\u2010RTSG network (1989\u20102019). Events were defined as relapse, metachronous tumors, or death.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Forty\u2010three patients were identified. The median age at WT\/nephroblastomatosis diagnosis was 22 months (range, 6\u201044 months). The overall stage was available for 40 patients, including 15 (37.5%) with bilateral disease and none with metastatic disease. Histology was available for 42 patients; 6 nephroblastomatosis without further WT and 36 WT, including 19 stromal WT (52.8%), 12 mixed WT (33.3%), 1 regressive WT (2.8%) and 2 other\/indeterminable WT (5.6%). Blastemal type WT occurred in 2 patients (5.6%) after prolonged treatment for nephroblastomatosis; anaplasia was not reported. Nephrogenic rests were present in 78.9%. Among patients with WT, the 5\u2010year event\u2010free survival rate was 84.3% (95% confidence interval, 72.4%\u201098.1%), and the overall survival rate was 91.2% (95% confidence interval, 82.1%\u2010100%). Events (n = 6) did not include relapse, but contralateral tumor development (n = 3) occurred up to 7 years after the initial diagnosis, and 3 deaths were related to hepatotoxicity (n = 2) and obstructive ileus (n = 1).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Patients with WAGR have a high rate of bilateral disease and no metastatic or anaplastic tumors. Although they can be treated according to existing WT protocols, intensive monitoring of toxicity and surveillance of the remaining kidney(s) are advised.<\/jats:p><\/jats:sec><jats:sec><jats:title>Lay Summary<\/jats:title><jats:p>\n<jats:list list-type=\"bullet\">\n\n<jats:list-item><jats:p>WAGR syndrome (Wilms tumor, aniridia, genitourinary anomalies, and range of developmental delays) is a rare genetic condition with an increased risk of developing Wilms tumor.<\/jats:p><\/jats:list-item>\n\n<jats:list-item><jats:p>In this study, 43 patients with WAGR and Wilms tumor (or Wilms tumor precursor lesions\/nephroblastomatosis) were identified through the international registry of the International Society of Pediatric Oncology Renal Tumor Study Group (SIOP\u2010RTSG) and the SIOP\u2010RTSG network. In many patients (37.5%), both kidneys were affected. Disease spread to other organs (metastases) did not occur.<\/jats:p><\/jats:list-item>\n\n<jats:list-item><jats:p>Overall, this study demonstrates that patients with WAGR syndrome and Wilms tumor can be treated according to existing protocols. However, intensive monitoring of treatment complications and surveillance of the remaining kidney(s) are advised.<\/jats:p><\/jats:list-item>\n<\/jats:list>\n<\/jats:p><\/jats:sec>","DOI":"10.1002\/cncr.33304","type":"journal-article","created":{"date-parts":[[2020,11,4]],"date-time":"2020-11-04T15:28:31Z","timestamp":1604503711000},"page":"628-638","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":41,"title":["Clinical characteristics and outcomes of children with WAGR syndrome and Wilms tumor and\/or nephroblastomatosis: The 30\u2010year SIOP\u2010RTSG experience"],"prefix":"10.1002","volume":"127","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3156-5229","authenticated-orcid":false,"given":"Janna A.","family":"Hol","sequence":"first","affiliation":[{"name":"Princess M\u00e1xima Center for Pediatric Oncology Utrecht the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marjolijn C. J.","family":"Jongmans","sequence":"additional","affiliation":[{"name":"Princess M\u00e1xima Center for Pediatric Oncology Utrecht the Netherlands"},{"name":"Department of Genetics University Medical Center Utrecht\/Wilhelmina Children's Hospital Utrecht the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H\u00e9l\u00e8ne","family":"Sudour\u2010Bonnange","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology Centre Oscar Lambret Lille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9986-5861","authenticated-orcid":false,"given":"Gema L.","family":"Ram\u00edrez\u2010Villar","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology Hospital Universitario Virgen del Roc\u00edo Seville Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tanzina","family":"Chowdhury","sequence":"additional","affiliation":[{"name":"Great Ormond Street Institute of Child Health University College London London United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine","family":"Rechnitzer","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology Rigshospitalet Copenhagen Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Niklas","family":"Pal","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology Karolinska University Hospital Solna Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gudrun","family":"Schleiermacher","sequence":"additional","affiliation":[{"name":"Department of Pediatric Oncology Institut Curie Hospital Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Axel","family":"Karow","sequence":"additional","affiliation":[{"name":"Department of Pediatrics and Adolescent Medicine Friedrich\u2010Alexander University Erlangen\u2010N\u00fcrnberg Erlangen 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