Early experience with UGN-101 for the treatment of upper tract urothelial cancer – A multicenter evaluation of practice patterns and outcomes

Solomon L. Woldu, Craig Labbate, Katie S. Murray, Kyle Rose, Wade Sexton, Isamu Tachibana, Hristos Kaimakliotis, Joseph Jacob, Rian Dickstein, Jennifer Linehan, Alan Nieder, Marc A. Bjurlin, Mitchell Humphreys, Saum Ghodoussipour, Marcus L. Quek, Michael O'Donnell, Brian H. Eisner, Adam S. Feldman, Surena F. Matin, Yair Lotan

Research output: Contribution to journalArticlepeer-review


Background: UGN-101 is a novel delivery system for intracavitary treatment of upper tract urothelial cancer (UTUC). UGN-101 was approved based on a pivotal trial for small volume residual low-grade UTUC. Our aim was to report our experience with UGN-101 in a more heterogenous and real-world setting. Methods: We performed a retrospective review of all UGN-101 cases from 15 institutions with a focus on practice patterns, efficacy, and adverse effects. We include UGN-101 utilization in both the chemoablative and adjuvant setting. Results: There were a total 136 renal units treated from 132 patients. The majority of cases were biopsy proven low-grade UTUC. Practice patterns varied considerably - the most common administration technique was antegrade instillation via a percutaneous nephrostomy. When utilized in the adjuvant setting, 69% of patients were disease free at the time of their first endoscopic evaluation, while in the chemoablative setting, 37% were endoscopically clear on the first evaluation (P < 0.001). Complete response was higher in patients with smaller tumor size prior to UGN-101 induction; low volume (<1 cm) residual disease was associated with a 70% complete response, similar to disease free rate at first endoscopic evaluation when UGN-101 was used in the adjuvant setting. The use of maintenance doses of UGN-101 was reported in 27% of cases. The overall incidence of new onset, clinically significant ureteral stenosis was 23%. Conclusions: This study represents the largest review of patients treated with UGN-101 and can serve as a basis of ongoing hypotheses regarding treatment with UGN-101 for UTUC.

Original languageEnglish (US)
Pages (from-to)147.e15-147.e21
JournalUrologic Oncology: Seminars and Original Investigations
Issue number3
StatePublished - Mar 2023


  • Chemoablation
  • Jelmyto
  • UGN-101
  • UTUC
  • Upper tract urothelial cancer

ASJC Scopus subject areas

  • Oncology
  • Urology


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