Anti-Tumor Activity of Paclitaxel-Containing Regimens in Recurrent/Refractory Wilms Tumor

Authors

Alissa Groisser, Center For Cancer and Blood Disorders, Children's National Hospital and the Department of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, District of Columbia, USA.
Amy Frantz, Center For Cancer and Blood Disorders, Children's National Hospital and the Department of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, District of Columbia, USA.
James I. Geller, Division of Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Michael V. Ortiz, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
David Walterhouse, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.
Mark Ranalli, Nationwide Children's Hospital, Columbus, Ohio, USA.
Wendy Woods-Swafford, Blank Cancer and Blood Disorders Center, Blank Children's Hospital-UnityPoint Health, Des Moines, Iowa, USA.
Peter Schoettler, Division of Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Mayo Clinic, Rochester, Minnesota, USA.
Timothy Garrington, Department of Pediatric Hematology/Oncology/Bone Marrow Transplant, University of Colorado School of Medicine, Children's Hospital Colorado, Aurora, Colorado, USA.
Elizabeth A. Mullen, Dana-Farber/Boston Children's Blood Disorders and Cancer Center, Boston, Massachusetts, USA.
Shifra Ash, Division of Pediatric Hematology Oncology and Bone Marrow Transplantation, Ruth Rappaport Children's Hospital, Rambam Medical Center, Haifa, Israel.
Pooja Bardhan, Northside Hospital Cancer Institute, Atlanta, Georgia, USA.
Frank Balis, Division of Oncology, Children's Hospital of Philadelphia and University of Pennsylvania, Perelman School of Medicine, Philadelphia, Pennsylvania, USA.
Jeffrey S. Dome, Center For Cancer and Blood Disorders, Children's National Hospital and the Department of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, District of Columbia, USA.

Document Type

Journal Article

Publication Date

7-25-2025

Journal

Pediatric blood & cancer

DOI

10.1002/pbc.31915

Keywords

Wilms tumor; nephroblastoma; paclitaxel; recurrent; relapse; taxane

Abstract

BACKGROUND: The Pediatric Oncology Group P9262 Phase 2 study of single-agent paclitaxel demonstrated one complete response (CR), one partial response (PR), and four with stable disease (SD) among 15 patients with recurrent Wilms tumor (WT). Based on this activity, paclitaxel-containing regimens have been used as salvage therapy for treatment-refractory WT. We conducted a multi-institutional retrospective study to further characterize the clinical activity of paclitaxel-containing regimens in recurrent WT. METHODS: Twelve institutions submitted anonymized data from patients with relapsed WT treated with paclitaxel. Response after one or two cycles of a paclitaxel-containing regimen was reported according to Response Evaluation Criteria in Solid Tumors. RESULTS: Twenty-eight patients with a median age at diagnosis of 5.5 years (range: 3 months to 31.9 years) were reported. Twenty had non-anaplastic WT; eight had diffuse anaplasia (DA). Stage at diagnosis was I (n = 1), II (n = 1), III (n = 11), IV (n = 14), and V (n = 1). Patients received a mean of 8.5 anticancer drugs (range: 6-12) before paclitaxel, including eight who received high-dose therapy/stem cell transplant. Paclitaxel (n = 13) or nab-paclitaxel (n = 15) were predominantly administered in combination with other agents, most commonly doxorubicin and/or gemcitabine. Eleven of 26 patients with measurable disease had a partial response (7 = non-anaplastic; 4 = DA), four had stable disease, and 11 had progressive disease. Two patients without measurable disease maintained a complete response for 12 and 16 months. The mean duration without progression was 7.6 months (range: 1-35 months). CONCLUSIONS: Paclitaxel-containing regimens appear to be active against heavily pretreated WT with either non-anaplastic or DA histology. Prospective studies are warranted to formally evaluate the efficacy of paclitaxel-based therapies.

Department

Pediatrics

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