Rhabdoid Tumor of the Kidney and Soft Tissues: Results from National Wilms Tumor Study-5 and Children's Oncology Group Study AREN0321

James I Geller¹, Lindsay A Renfro², Paul E Grundy³, Elizabeth J Perlman⁴, John A Kalapurakal⁵, Peter F Ehrlich⁶, Jackie Biegel^{7

8}, Vicki Huff⁹, Anne B Warwick¹⁰, Arnold Paulino¹¹, Elizabeth A Mullen¹², Najat C Daw¹³, Fredric A Hoffer¹⁴, Zelig Tochner¹⁵, Kenneth Gow¹⁶, Eric Gratias¹⁷, Deborah A Ward¹⁸, James R Anderson¹⁹, Conrad V Fernandez²⁰, Jeffrey S Dome²¹

Affiliations

¹ Division of Pediatric Oncology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA.
² Division of Biostatistics, University of Southern California and Children's Oncology Group, Los Angeles, California, USA.
³ Division of Immunology, Hematology, Oncology, Palliative Care, and Environmental Interactions, University of Alberta, Edmonton, Alberta, Canada.
⁴ Department of Pathology and Laboratory Medicine, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.
⁵ Department of Radiation Oncology, Northwestern University, Chicago, Illinois, USA.
⁶ Section of Pediatric Surgery, CS Mott Children's Hospital, University of Michigan, Ann Arbor, Michigan, USA.
⁷ Keck School of Medicine, University of Southern California, Los Angeles, California, USA.
⁸ Department of Pathology and Laboratory Medicine, Children's Hospital Los Angeles, Los Angeles, California, USA.
⁹ Department of Genetics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
¹⁰ Department of Pediatrics, F. Edward Hébert School of Medicine, Uniformed Services University, Bethesda, Maryland, USA.
¹¹ MD Anderson Cancer Center, Houston, Texas, USA.
¹² Boston Children's Hospital and Dana Farber Cancer Institute, Boston, Massachusetts, USA.
¹³ Division of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
¹⁴ Department of Radiology, Fred Hutchison Cancer Center, University of Washington, Seattle, Washington, USA.
¹⁵ Department of Radiation Oncology, Perelman Center for Advanced Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
¹⁶ Division of Pediatric General and Thoracic Surgery, Seattle Children's Hospital, University of Washington, Seattle, Washington, USA.
¹⁷ eviCore Healthcare, Bluffton, South Carolina, USA.
¹⁸ Department of Pharmaceutical Services, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
¹⁹ University of Nebraska Medical Center, Omaha, Nebraska, USA.
²⁰ Division of Pediatric Hematology Oncology, IWK Health Centre, Dalhousie University, Halifax, Nova Scotia, Canada.
²¹ Division of Oncology, Children's National Hospital and Department of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, District of Columbia, USA.

PMID: 39702900
PMCID: PMC11801664 (available on 2026-03-01)
DOI: 10.1002/pbc.31490

Multicenter Study

Rhabdoid Tumor of the Kidney and Soft Tissues: Results from National Wilms Tumor Study-5 and Children's Oncology Group Study AREN0321

James I Geller et al. Pediatr Blood Cancer. 2025 Mar.

. 2025 Mar;72(3):e31490.

doi: 10.1002/pbc.31490. Epub 2024 Dec 19.

Authors

Affiliations

¹ Division of Pediatric Oncology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA.
² Division of Biostatistics, University of Southern California and Children's Oncology Group, Los Angeles, California, USA.
³ Division of Immunology, Hematology, Oncology, Palliative Care, and Environmental Interactions, University of Alberta, Edmonton, Alberta, Canada.
⁴ Department of Pathology and Laboratory Medicine, Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.
⁵ Department of Radiation Oncology, Northwestern University, Chicago, Illinois, USA.
⁶ Section of Pediatric Surgery, CS Mott Children's Hospital, University of Michigan, Ann Arbor, Michigan, USA.
⁷ Keck School of Medicine, University of Southern California, Los Angeles, California, USA.
⁸ Department of Pathology and Laboratory Medicine, Children's Hospital Los Angeles, Los Angeles, California, USA.
⁹ Department of Genetics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
¹⁰ Department of Pediatrics, F. Edward Hébert School of Medicine, Uniformed Services University, Bethesda, Maryland, USA.
¹¹ MD Anderson Cancer Center, Houston, Texas, USA.
¹² Boston Children's Hospital and Dana Farber Cancer Institute, Boston, Massachusetts, USA.
¹³ Division of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
¹⁴ Department of Radiology, Fred Hutchison Cancer Center, University of Washington, Seattle, Washington, USA.
¹⁵ Department of Radiation Oncology, Perelman Center for Advanced Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
¹⁶ Division of Pediatric General and Thoracic Surgery, Seattle Children's Hospital, University of Washington, Seattle, Washington, USA.
¹⁷ eviCore Healthcare, Bluffton, South Carolina, USA.
¹⁸ Department of Pharmaceutical Services, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
¹⁹ University of Nebraska Medical Center, Omaha, Nebraska, USA.
²⁰ Division of Pediatric Hematology Oncology, IWK Health Centre, Dalhousie University, Halifax, Nova Scotia, Canada.
²¹ Division of Oncology, Children's National Hospital and Department of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, District of Columbia, USA.

PMID: 39702900
PMCID: PMC11801664 (available on 2026-03-01)
DOI: 10.1002/pbc.31490

Abstract

Purpose: National Wilms Tumor Study-5 (NWTS-5) and AREN0321 evaluated the outcomes of children with rhabdoid tumor of the kidney (RTK) and malignant rhabdoid tumor of soft tissues (MRT).

Patients and methods: Eligible patients with RTK were enrolled prospectively on NWTS-5 (1995-2002) and treated with carboplatin and etoposide alternating with cyclophosphamide (Regimen RTK). Patients with RTK or MRT were enrolled on AREN0321 (2005-2012) and received vincristine, doxorubicin, and cyclophosphamide alternating with carboplatin, cyclophosphamide, and etoposide (Regimens UH-1 or dose-reduced Revised UH-1). We report event-free survival (EFS) and overall survival (OS) from each study.

Results: Thirty patients received Regimen RTK on NWTS-5; on AREN0321, 20 received UH-1 and 19 received Revised UH-1. Patient and disease characteristics were statistically similar between studies. Patients on AREN0321 had significantly improved EFS and OS compared to those on NWTS-5 (4-year EFS = 23.1% vs. 16.7%; p = 0.020; 4-year OS = 30.6% vs. 20.0%; p = 0.014), mostly driven by patients with Stage I/II disease (p = 0.05). Median time to an event was 3.6 months on NWTS-5 compared to 7.2 months on AREN0321. There were no differences in EFS or OS by revised versus original Regimen UH-1 on AREN0321, or by renal versus extra-renal primary disease when the studies were pooled.

Conclusions: The more intensive treatment regimen used on AREN0321 improved EFS and OS overall, a result driven by patients with Stage I/II disease. Despite this improvement, outcomes for patients with rhabdoid tumor remain unsatisfactory and there is a need for novel therapeutic strategies.

Keywords: chemotherapy; pediatric oncology; renal; rhabdoid tumor; tumors.

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References

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Rhabdoid Tumor of the Kidney and Soft Tissues: Results from National Wilms Tumor Study-5 and Children's Oncology Group Study AREN0321

Affiliations

Rhabdoid Tumor of the Kidney and Soft Tissues: Results from National Wilms Tumor Study-5 and Children's Oncology Group Study AREN0321

Authors

Affiliations

Abstract

References

Publication types

MeSH terms

Substances

Grants and funding

LinkOut - more resources

Full Text Sources

Medical