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).
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.
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.
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.
Pediatric blood & cancer. 2024 Dec 19 [Epub ahead of print]
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
Division of Pediatric Oncology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA., Division of Biostatistics, University of Southern California and Children's Oncology Group, Los Angeles, California, USA., Division of Immunology, Hematology, Oncology, Palliative Care, and Environmental Interactions, University of Alberta, Edmonton, Alberta, Canada., 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., Department of Radiation Oncology, Northwestern University, Chicago, Illinois, USA., Section of Pediatric Surgery, CS Mott Children's Hospital, University of Michigan, Ann Arbor, Michigan, USA., Keck School of Medicine, University of Southern California, Los Angeles, California, USA., Department of Genetics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA., Department of Pediatrics, F. Edward Hébert School of Medicine, Uniformed Services University, Bethesda, Maryland, USA., MD Anderson Cancer Center, Houston, Texas, USA., Boston Children's Hospital and Dana Farber Cancer Institute, Boston, Massachusetts, USA., Division of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA., Department of Radiology, Fred Hutchison Cancer Center, University of Washington, Seattle, Washington, USA., Department of Radiation Oncology, Perelman Center for Advanced Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA., Division of Pediatric General and Thoracic Surgery, Seattle Children's Hospital, University of Washington, Seattle, Washington, USA., eviCore Healthcare, Bluffton, South Carolina, USA., Department of Pharmaceutical Services, St. Jude Children's Research Hospital, Memphis, Tennessee, USA., University of Nebraska Medical Center, Omaha, Nebraska, USA., Division of Pediatric Hematology Oncology, IWK Health Centre, Dalhousie University, Halifax, Nova Scotia, Canada., 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.