First analysis of the safety and efficacy of UGN-101 in the treatment of ureteral tumors

• Published in: Urologic oncology 2024-01, Vol.42 (1), p.20.e17-20.e23
• Main Authors: Jacob, Joseph M., Woldu, Solomon L., Linehan, Jennifer, Labbate, Craig, Rose, Kyle M., Sexton, Wade J., Tachibana, Isamu, Kaimakliotis, Hristos, Nieder, Alan, Bjurlin, Marc A., Humphreys, Mitchell, Ghodoussipor, Saum, Quek, Marcus L., Johnson, Brett, O'Donnell, Michael, Eisner, Brian H., Feldman, Adam S., Murray, Katie S., Matin, Surena F., Lotan, Yair, Dickstein, Rian J.
• Format: Article
• Language: English
• Subjects: Chemoablation; Jelmyto; UGN-101; Upper tract urothelial cancer; Ureter; Ureteral; Ureteral cancer; UTUC
• ISSN: 1078-1439; 1873-2496; 1873-2496
• DOI: 10.1016/j.urolonc.2023.07.004

•UGN-101 has been approved for the use based on data showing efficacy in the chemo-ablation of small upper tract tumors of pyelocaliceal system – but there is no data on potential efficacy in the setting of ureteral tumors.•This study aims to explore this for the first time and review outcomes in 47 ureteral tumors treated with UGN-101 .•The complete response rate in patients treated with chemo-ablative intent (n = 23) was 47.8%. UGN-101 has been approved for the chemoablation of low-grade upper tract urothelial cancer (UTUC) involving the renal pelvis and calyces. Herein is the first reported cohort of patients with ureteral tumors treated with UGN-101. We performed a retrospective review of patients treated with UGN-101 for UTUC at 15 high-volume academic and community centers focusing on outcomes of patients treated for ureteral disease. Patients received UGN-101 with either adjuvant or chemo-ablative intent. Response rates are reported for patients receiving chemo-ablative intent. Adverse outcomes were characterized with a focus on the rate of ureteral stenosis. In a cohort of 132 patients and 136 renal units, 47 cases had tumor involvement of the ureter, with 12 cases of ureteral tumor only (8.8%) and 35 cases of ureteral plus renal pelvic tumors (25.7%). Of the 23 patients with ureteral involvement who received UGN-101 induction with chemo-ablative intent, the complete response was 47.8%, which did not differ significantly from outcomes in patients without ureteral involvement. Fourteen patients (37.8%) with ureteral tumors had significant ureteral stenosis at first post-treatment evaluation, however, when excluding those with pre-existing hydronephrosis or ureteral stenosis, only 5.4% of patients developed new clinically significant stenosis. UGN-101 appears to be safe and may have similar efficacy in treating low-grade urothelial carcinoma of the ureter as compared to renal pelvic tumors.