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

Authors

James I Geller
Lindsay A Renfro
Paul E Grundy
Elizabeth J Perlman
John A Kalapurakal
Peter F Ehrlich
Jackie Biegel
Vicki Huff
Anne B Warwick
Arnold Paulino
Elizabeth A Mullen
Najat C Daw
Fredric A Hoffer
Zelig Tochner
Kenneth Gow
Eric Gratias
Deborah A Ward
James R Anderson
Conrad V Fernandez
Jeffrey S Dome

Language

English

Publication Date

3-1-2025

Journal

Pediatric Blood & Cancer

DOI

10.1002/pbc.31490

PMID

39702900

PMCID

PMC11801664

PubMedCentral® Posted Date

3-1-2026

PubMedCentral® Full Text Version

Author MSS

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

Humans, Rhabdoid Tumor, Kidney Neoplasms, Female, Male, Child, Preschool, Antineoplastic Combined Chemotherapy Protocols, Infant, Child, Etoposide, Survival Rate, Soft Tissue Neoplasms, Cyclophosphamide, Vincristine, Prospective Studies, Carboplatin, Doxorubicin, Follow-Up Studies, Adolescent, Prognosis, Wilms Tumor, chemotherapy, pediatric oncology, renal, rhabdoid tumor, tumors

Published Open-Access

yes

Recommended Citation

Geller, James I; Renfro, Lindsay A; Grundy, Paul E; et al., "Rhabdoid Tumor of the Kidney and Soft Tissues: Results from National Wilms Tumor Study-5 and Children's Oncology Group Study AREN0321" (2025). Faculty, Staff and Student Publications. 5870.
https://digitalcommons.library.tmc.edu/uthgsbs_docs/5870