Related Experiment Video
Updated: Jun 4, 2025
Tumorsphere Derivation and Treatment from Primary Tumor Cells Isolated from Mouse Rhabdomyosarcomas
Published on: September 13, 2019
Rhabdoid Tumor of the Kidney and Soft Tissues: Results from National Wilms Tumor Study-5 and Children's Oncology
James I Geller1, Lindsay A Renfro2, Paul E Grundy3
1Division of Pediatric Oncology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA.
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.
Insights
Children with rhabdoid tumors had improved survival on the AREN0321 intensive treatment regimen compared to NWTS-5. However, outcomes for rhabdoid tumor patients remain poor, necessitating novel therapeutic strategies.
Area of Science:
- Pediatric Oncology
- Medical Treatment Efficacy
- Cancer Research
Background:
- Rhabdoid tumors of the kidney (RTK) and soft tissues (MRT) are aggressive pediatric cancers.
- Previous treatment regimens have shown limited success in improving patient outcomes.
Purpose of the Study:
- To compare the efficacy of treatment regimens used in the National Wilms Tumor Study-5 (NWTS-5) and AREN0321 trials for children with RTK and MRT.
- To evaluate event-free survival (EFS) and overall survival (OS) in patients treated with different chemotherapy protocols.
Main Methods:
- Prospective enrollment of eligible patients with RTK on NWTS-5 (1995-2002) using carboplatin/etoposide alternating with cyclophosphamide (Regimen RTK).
- Enrollment of patients with RTK or MRT on AREN0321 (2005-2012) receiving vincristine/doxorubicin/cyclophosphamide alternating with carboplatin/cyclophosphamide/etoposide (Regimens UH-1 or dose-reduced Revised UH-1).
- Comparison of EFS and OS between the two study groups.
Main Results:
- Patients on AREN0321 demonstrated significantly improved 4-year EFS (23.1% vs. 16.7%) and OS (30.6% vs. 20.0%) compared to NWTS-5 (p=0.020 and p=0.014, respectively).
- The survival benefit was primarily observed in patients with Stage I/II disease.
- Median time to an event was longer on AREN0321 (7.2 months) versus NWTS-5 (3.6 months).
Conclusions:
- The more intensive AREN0321 treatment regimen significantly improved EFS and OS, particularly for Stage I/II rhabdoid tumors.
- Despite treatment advancements, outcomes for rhabdoid tumor patients remain unsatisfactory.
- Novel therapeutic strategies are urgently needed to improve survival rates for these aggressive pediatric cancers.
More Related Videos
Related Concept Videos
01:20The Retinoblastoma Gene
The first-ever tumor suppressor gene called Rb was identified in retinoblastoma - a rare eye tumor in children. In inherited forms of the disease, a child inherits one defective copy of the Rb gene, which predisposes them to retinoblastoma. However,...
01:03Rous Sarcoma Virus (RSV) and Cancer
RSV is a retrovirus that contains two copies of a plus-strand RNA genome. Its genome consists of four main open...
01:45Kidney Structure