Faculty Collaboration Database

Bilateral Wilms tumor with anaplasia: A report from the Children's Oncology Group Study AREN0534. Pediatr Blood Cancer 2024 Jul;71(7):e30981

Date

04/19/2024

Pubmed ID

38637871

Pubmed Central ID

PMC11116047

DOI

10.1002/pbc.30981

Scopus ID

2-s2.0-85191696014 (requires institutional sign-in at Scopus site)   6 Citations

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.

Author List

Romao RLP, Aldrink JH, Renfro LA, Mullen EA, Murphy AJ, Brzezinski J, Malek MM, Benedetti DJ, Cost NG, Smith E, Dome JS, Davidoff AM, Treece A, Parsons LN, Fernandez CV, Tornwall B, Shamberger RC, Paulino A, Kalapurakal JA, Geller JI, Ehrlich PF

Author

Lauren Parsons MD Associate Professor in the Pathology and Laboratory Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Anaplasia
Child
Child, Preschool
Female
Follow-Up Studies
Humans
Infant
Kidney Neoplasms
Male
Nephrectomy
Prognosis
Survival Rate
Wilms Tumor