Faculty Collaboration Database

Impact of Pulmonary Tumor Burden in Favorable Histology Wilms Tumor Outcomes: A Report From the Children's Oncology Group Study AREN0533. J Clin Oncol 2025 Dec 20;43(36):3822-3832

Date

11/12/2025

Pubmed ID

41223336

Pubmed Central ID

PMC12646512

DOI

10.1200/JCO-25-00532

Scopus ID

2-s2.0-105025241481 (requires institutional sign-in at Scopus site)   1 Citation

Abstract

PURPOSE: Children with favorable histology Wilms tumor (FHWT) with pulmonary metastases have inferior outcomes compared with those with localized disease. We evaluated the impact of pulmonary tumor burden within subgroups of similarly treated children with stage IV FHWT.

METHODS: Children with FHWT with pulmonary-only metastasis enrolled in AREN0533 were included. Lung nodule response assessment, by chest computed tomography after two cycles of vincristine/dactinomycin/doxorubicin (Regimen DD-4A) chemotherapy, identified rapid complete responses (RCRs) and slow incomplete pulmonary nodule responses (SIRs). Event-free survival (EFS) and overall survival (OS) were compared by number and size of pulmonary metastases within two cohorts: (1) RCR treated with additional DD-4A without lung radiation therapy (RT) and (2) SIR treated with the original three drugs plus cyclophosphamide/etoposide (Regimen M) with lung RT. The multivariable Cox proportional hazards model for EFS and OS stratified by treatment assessed the impact of the number and size of pulmonary metastases adjusted for tumor 1q gain.

RESULTS: AREN0533 enrolled 288 children with stage IV pulmonary-only metastases, of whom 251 met inclusion criteria for outcome analyses. In the RCR cohort (n = 105), EFS and OS were not significantly different based on the number of lung metastases, whereas size of pulmonary metastases was significantly associated with EFS (P = .022), but not OS. In the SIR cohort (n = 146), EFS and OS did not differ by the number or size of lung metastases. In multivariable models, neither number nor size of lung metastases was significantly associated with EFS or OS although 1q gain was significant (EFS P = .0015; OS P = .039) after adjustment for these factors.

CONCLUSION: 1q gain is a superior prognostic indicator to pulmonary tumor burden in patients with FHWT with pulmonary-only metastasis.

Author List

Dix DB, Khanna G, Renfro LA, Tfirn IC, Smith EA, Artunduaga M, Eklund MJ, Sandberg JK, Parsons LN, Kalapurakal JA, Ehrlich PF, Aldrink JH, Glick RD, Benedetti DJ, Fernandez CV, Dome JS, Mullen EA, Geller JI, COG Renal Tumor Committee

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

Antineoplastic Combined Chemotherapy Protocols
Child
Child, Preschool
Cyclophosphamide
Dactinomycin
Doxorubicin
Female
Humans
Infant
Kidney Neoplasms
Lung Neoplasms
Male
Tumor Burden
Vincristine
Wilms Tumor