Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Multicenter Study
. 2024 Jul;71(7):e30981.
doi: 10.1002/pbc.30981. Epub 2024 Apr 18.

Bilateral Wilms tumor with anaplasia: A report from the Children's Oncology Group Study AREN0534

Rodrigo L P Romao  1 , Jennifer H Aldrink  2 , Lindsay A Renfro  3 , Elizabeth A Mullen  4 , Andrew J Murphy  5 , Jack Brzezinski  1 , Marcus M Malek  6 , Daniel J Benedetti  7 , Nicholas G Cost  8 , Ethan Smith  9 , Jeffrey S Dome  10 , Andrew M Davidoff  5 , Amy Treece  11 , Lauren N Parsons  12 , Conrad V Fernandez  13 , Brett Tornwall  14 , Robert C Shamberger  4 , Arnold Paulino  15 , John A Kalapurakal  16 , James I Geller  9 , Peter F Ehrlich  17
Affiliations
Multicenter Study

Bilateral Wilms tumor with anaplasia: A report from the Children's Oncology Group Study AREN0534

Rodrigo L P Romao et al. Pediatr Blood Cancer. 2024 Jul.

Abstract

Introduction: The purpose of this study is to examine the outcomes in children with anaplastic bilateral Wilms tumor (BWT) from study AREN0534 in order to define potential prognostic factors and areas to target in future clinical trials.

Methods: Demographic and clinical data from AREN0534 study patients with anaplasia (focal anaplasia [FA], or diffuse anaplasia [DA]) were compared. Event-free survival (EFS) and overall survival (OS) were reported using Kaplan-Meier estimation with 95% confidence bands, and differences in outcomes between FA and DA compared using log-rank tests. The impact of margin status was analyzed.

Results: Twenty-seven children who enrolled on AREN0534 had evidence of anaplasia (17 DA, 10 FA) in at least one kidney and were included in this analysis. Twenty-six (96%) had BWT. Nineteen percent had anaplastic histology in both kidneys (four of 17 DA, and one of 10 FA). Forty-six percent with BWT had bilateral nephron-sparing surgery (NSS); one child who went off protocol therapy, eventually required bilateral completion nephrectomies. Median follow-up for EFS and OS was 8.6 and 8.7 years from enrollment. Four- and 8-year EFS was 53% [95% confidence interval (CI): 34%-83%] for DA; 4-year EFS was 80% [95% CI: 59%-100%], and 8-year EFS 70% [95% CI: 47%-100%] for FA. Three out of 10 children with FA and eight out of 17 children with DA had events. EFS did not differ statistically by margin status (p = .79; HR = 0.88). Among the six children who died (five DA, one FA), all experienced prior relapse or progression within 18 months.

Conclusion: Events in children with DA/FA in the setting of BWT occurred early. Caution should be taken about interpreting the impact of margin status outcomes in the context of contemporary multimodal therapy. Future targeted investigations in children with BWT and DA/FA are needed.

Keywords: Wilms tumor; anaplasia; bilateral.

PubMed Disclaimer

Conflict of interest statement

The authors have NO conflicts of interest

Conflict of interest statement: The authors report no conflicts of interest.

Figures

Figure 1 –
Figure 1 –
Event-free survival (EFS) in children enrolled on AREN0534 with evidence of anaplasia on at least one kidney
Figure 2 –
Figure 2 –
Event-free survival (EFS) in children enrolled on AREN0534 with evidence of anaplasia on at least one kidney based on surgical margin status
See this image and copyright information in PMC

References

    1. Perlman EJ. Pediatric renal tumors: practical updates for the pathologist. Pediatr Dev Pathol. 2005;8(3):32–338. - PubMed
    1. Dome JS, Mullen EA, Dix DB, Gratias EJ, Ehrlich PF, Daw NC, et al. Impact of the First Generation of Children’s Oncology Group Clinical Trials on Clinical Practice for Wilms Tumor. J Natl Compr Canc Netw. 2021;19(8):978–85. - PMC - PubMed
    1. Ehrlich P, Chi YY, Chintagumpala MM, Hoffer FA, Perlman EJ, Kalapurakal JA, et al. Results of the First Prospective Multi-institutional Treatment Study in Children With Bilateral Wilms Tumor (AREN0534): A Report From the Children’s Oncology Group. Ann Surg. 2017;266(3):470–8. - PMC - PubMed
    1. Ehrlich PF. Bilateral Wilms’ tumor: the need to improve outcomes. Expert Rev Anticancer Ther. 2009;9(7):963–73. - PubMed
    1. Grundy PE, Breslow N, Li S, et al. Loss of heterozygosity for chromosomes 1p and 16q is an adverse prognostic factor in favorable-histology Wilms tumor: a report from the National Wilms Tumor Study Group. J Clin Oncol. 2005;23(29):7312–21. - PubMed

Publication types