Characteristics and outcome of children with Beckwith-Wiedemann syndrome and Wilms' tumor: A report from the National Wilms Tumor Study Group

Purpose: Children with Beckwith-Wiedemann syndrome (BWS) are at increased r isk for developing Wilms' tumor (WT), We reviewed the National Wilms Tumor Study Group (NWTSG) records to assess clinical characteristics and outcome of patients with WT and BWS. Methods: In the NWTSG, treating clinicians were asked to report, for each e nrolled patient, whether the patient had BWS. Between 1980 and 1995, 4,669 patients were treated on two consecutive NWTSG protocols (NWTS 3 and NWTS 4 ). We retrospectively reviewed the clinical characteristics and treatment o utcomes of BWS patients compared with patients with WT without BWS. Results: Fifty-three children enrolled onto NWTS 3 and 4 were reported to h ave BWS. BWS patients were more likely to present with lower-stage tumors ( P =.0001), with more than half (27 of 53) presenting with stage I disease. The overall treatment outcomes for the BWS patients were nearly identical t o those without BWS, with overall survival at 4 years from diagnosis at 89% and 90%, respectively. Overall, 21% of the patients with BWS had bilateral disease, either at diagnosis (nine of 53) or as metachronous contralateral recurrence (two of 53). BWS patients enrolled onto NWTS 4 had smaller tumo rs than those enrolled onto NWTS 3 (P =.02), a trend not seen in the non-BW S patients. Conclusion: Like children without BWS, children with BWS and WT have an exc ellent prognosis with modern treatment regimens. There is a high risk of bi lateral disease, and increasingly smaller rumors are being detected. This s uggests that a national trial assessing the role of ultrasound screening fo llowed by nephron-sparing surgery for some patients may be appropriate. J C lin Oncol 18:2026-2031. (C) 2000 by American Society of Clinical Oncology.