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

Multicenter Study

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

Lindsay A Renfro 2, Paul E Grundy 3, Elizabeth J Perlman 4, John A Kalapurakal 5, Peter F Ehrlich 6, Jackie Biegel 7 8, Vicki Huff 9, Anne B Warwick 10, Arnold Paulino 11, Elizabeth A Mullen 12, Najat C Daw 13, Fredric A Hoffer 14, Zelig Tochner 15, Kenneth Gow 16, Eric Gratias 17, Deborah A Ward 18, James R Anderson 19, Conrad V Fernandez 20, Jeffrey S Dome 21

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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.

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.

© 2024 Wiley Periodicals LLC.

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Figures

Figure 1:

Figure 1:

National Wilms’ Tumor Study V Regimen ‘RTK’ (A) and AREN0321 Regimen UH-1 (B)

Figure 2

Figure 2

Event Free Survival (A) and Overall Survival (B) by Study

Figure 2

Figure 2

Event Free Survival (A) and Overall Survival (B) by Study

Figure 3:

Figure 3:

Event Free Survival (A) and Overall Survival (B) for AREN0321 Renal vs Non-Renal Primary Site by Stage

Figure 3:

Figure 3:

Event Free Survival (A) and Overall Survival (B) for AREN0321 Renal vs Non-Renal Primary Site by Stage

Figure 4:

Figure 4:

Event Free Survival (A) and Overall Survival (B) for Stages 1–4 Enrolled on Regimen RTK vs UH-1

Figure 4:

Figure 4:

Event Free Survival (A) and Overall Survival (B) for Stages 1–4 Enrolled on Regimen RTK vs UH-1

References

    1. Fernandez C, Geller JI, Ehrlich PF, Hill DA, Van den Heuvel-Eibrink MM, Graf N, Mullen E, Hill DA, Kalapurakal JA, Dome JS. Chapter 24: Renal Tumors. Principles and Practice of Pediatric Oncology 8th Ed.861–85 (eds. Pizzo and Poplack): Lippincott-Williams & Wilkins. 2020.
    1. Tomlinson GE, Breslow NE, Dome J, Guthrie KA, Norkool P, Li S, et al. Rhabdoid tumor of the kidney in the National Wilms’ Tumor Study: age at diagnosis as a prognostic factor. J Clin Oncol 2005;23(30):7641–5. -PubMed
    1. Kodet R, Newton WA Jr, Sachs N, Hamoudi AB, Raney RB, Asmar L, Gehan EA. Rhabdoid tumors of soft tissues: a clinicopathologic study of 26 cases enrolled on the Intergroup Rhabdomyosarcoma Study. Hum Pathol. 1991. Jul;22(7):674–84. doi: 10.1016/0046-8177(91)90289-2. -DOI -PubMed
    1. Green DM, Breslow NE, Beckwith JB, Ritchey ML, Shamberger RC, Haase GM, et al. Treatment with nephrectomy only for small, stage I/favorable histology Wilms tumor. A report from the National Wilms Tumor Study Group. J Clin Oncol 19:3719–3724, 2001. -PubMed
    1. Daw NC, Chi YY, Kalapurakal JA, Kim Y, Hoffer FA, Geller JI, et al. ; AREN0321 Study Committee. Activity of vincristine and irinotecan in diffuse anaplastic Wilms tumor and therapy outcomes of stage II to IV disease: Results of the Children’s Oncology Group AREN0321 Study. J Clin Oncol. 2020. May 10;38(14):1558–1568.5. -PMC -PubMed

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