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.
Keywords: Wilms tumor; nephroblastoma; paclitaxel; recurrent; relapse; taxane.
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