Impact of Pulmonary Tumor Burden in Favorable Histology Wilms Tumor Outcomes: A Report From the Children's Oncology Group Study AREN0533

David B Dix ¹, Geetika Khanna ², Lindsay A Renfro ³

¹British Columbia Children's Hospital, Vancouver, BC, Canada.
²Children's Healthcare of Atlanta, Atlanta, GA.
³University of Southern California and Children's Oncology Group, Los Angeles, CA.
⁴Department of Radiology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
⁵UT Southwestern/Simmons Cancer Center, Dallas, TX.
⁶Medical University of South Carolina, Charleston, SC.
⁷Lucile Packard Children's Hospital Stanford University, Palo Alto, CA.
⁸Children's Hospital of Wisconsin, Milwaukee, WI.
⁹Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, IL.
¹⁰C S Mott Children's Hospital, Ann Arbor, MI.
¹¹Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus, OH.
¹²Cohen Children's Medical Center, Zucker School of Medicine at Hofstra/Northwell, New Hyde Park, NY.
¹³Vanderbilt University Medical Center, Nashville, TN.
¹⁴IWK Health Centre, Dalhousie University, Halifax, NS, Canada.
¹⁵Children's National Medical Center, George Washington University School of Medicine and Health Sciences, Washington, DC.
¹⁶Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA.
¹⁷Peckham Center for Cancer and Blood Disorders, Rady Children's Hospital, San Diego, CA.
¹⁸Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of California San Diego, La Jolla, CA.

⁰British Columbia Children's Hospital, Vancouver, BC, Canada.

Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology +

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November 12, 2025

View abstract on PubMed

Summary

Pulmonary tumor burden does not significantly impact outcomes in children with favorable histology Wilms tumor and lung metastases. Instead, 1q gain is a better predictor of event-free and overall survival in these patients.

Area Of Science

Pediatric Oncology
Wilms Tumor Research
Cancer Metastasis Studies

Background

Children with favorable histology Wilms tumor (FHWT) and pulmonary metastases face poorer outcomes than those with localized disease.
Pulmonary tumor burden is a key factor in assessing prognosis for stage IV FHWT.

Purpose Of The Study

To evaluate the impact of pulmonary tumor burden on outcomes in children with stage IV FHWT and pulmonary-only metastases.
To compare the prognostic significance of tumor burden versus 1q gain in this patient group.

Main Methods

Analysis of children with FHWT and pulmonary-only metastases from the AREN0533 trial.
Classification into rapid complete response (RCR) and slow incomplete response (SIR) cohorts based on lung nodule response to initial chemotherapy.
Comparison of event-free survival (EFS) and overall survival (OS) by number and size of pulmonary metastases, adjusted for 1q gain using multivariable Cox models.

Main Results

In the RCR cohort (n=105), metastasis size, but not number, was associated with EFS.
In the SIR cohort (n=146), neither metastasis number nor size affected EFS or OS.
Multivariable models showed 1q gain significantly predicted EFS and OS, while tumor burden metrics did not.

Conclusions

Pulmonary tumor burden is not a significant prognostic indicator for event-free or overall survival in children with stage IV FHWT and pulmonary-only metastases.
1q gain is a superior prognostic marker compared to pulmonary tumor burden in this patient population.