Long-Term Outcomes of Primary Chemoablation of Low-Grade Upper Tract Urothelial Carcinoma With UGN-101, a Mitomycin Reverse Thermal Gel

Abstract

Purpose: We evaluate long-term outcomes of primary chemoablation using a mitomycin reverse thermal gel (UGN-101) in patients with low-grade upper tract urothelial carcinoma.

Materials and methods: Patients who participated in the OLYMPUS trial (IRB No. TC-UT-03, NCT02793128) and achieved a complete response (CR) after 6 weekly doses of UGN-101 were followed up to 12 months after initial CR. Those with CR at study completion were eligible for long-term follow-up for up to 5 years or until disease recurrence, progression, or death.

Results: Of the 71 patients enrolled in the OLYMPUS trial, 42 patients achieved CR 4 to 6 weeks after completing ≥ 1 instillation of UGN-101. Among the 41 patients followed after initial CR, median follow-up was 28.1 months (95% CI, 13.1-57.5), and median duration of response was 47.8 months (95% CI, 13.0-not estimable [NE]). Twenty patients (49%) had long-term follow-up (median 53.3 months [95% CI, 27.9-65.3]). Seventy-five percent of patients had no evidence of recurrence at the last follow-up, with median duration of response NE (95% CI, 43.5-NE) because of a low event rate.

Conclusions: Primary intracavitary chemoablation with UGN-101 for low-grade upper tract urothelial carcinoma is associated with favorable long-term durability.

Clinical trial registration no.: NCT02793128.

Keywords: carcinoma; clinical trial; mitomycin; transitional cell.

Figure.

Kaplan-Meier estimation of duration of response in all patients with complete response in the OLYMPUS trial (n = 41) and the subset of patients in the long-term follow-up (LTFU) cohort analysis set (n = 20). NE indicates not estimable.

Figure 1.

CONSORT (Consolidated Standards of Reporting Trials) diagram for patients enrolled in BL007. CR indicates complete response.

Figure 2.

Swimmer plot for 41 patients with complete response in the OLYMPUS parent trial and additional long-term follow-up for 20 patients with complete response enrolled in BL007. DOR indicates duration of response.

Figure 3.

Kaplan-Meier estimation of duration of response in all patients with complete response in the OLYMPUS trial (n = 41) and the subset of patients in the long-term follow-up (LTFU) cohort analysis set (n = 20). NE indicates not estimable.

Figure 4.

Weibull predicted curve and Kaplan-Meier estimation of duration of response cohort analysis set (n = 41). A Weibull curve (red lines) was fit to the data and overlaid on the Kaplan-Meier curve (black lines). CIs are displayed as dotted lines (respective colors).