Efficacy of adjuvant nivolumab in patients with upper tract predominant urothelial carcinoma: A single-center real-world study.

Background: Upper tract urothelial carcinoma (UTUC) is more common in Taiwan than in other regions. Although adjuvant nivolumab showed efficacy in high-risk urothelial carcinoma in the CheckMate 274 trial, its benefit in UTUC remains unclear. In this study, the real-world effectiveness and safety of adjuvant nivolumab were evaluated in Taiwanese patients with UC.

Methods: This retrospective, single-center study included 42 patients with UC who underwent radical resection and received adjuvant nivolumab at Taipei Veterans General Hospital. Survival outcomes were analyzed using the Kaplan-Meier method and Cox regression.

Results: The cohort had a median age of 70.5 years, and 54.8% of enrollees were male. UTUC accounted for 85.7% of all cases. The median follow-up was 17.3 months, and 26.2% of patients experienced recurrence or died during their follow-up. At 6, 12, and 24 months, the DFS rate was 89.9%, 78.0%, and 68.4%, respectively. The favorable 24-month DFS was more favorable in patients with UTUC (73.1%) vs. those with bladder UC (66.7%). PD-L1 expression ≥1% was associated with a nonsignificant DFS benefit (hazard ratio = 0.50, P = 0.272). Adjuvant nivolumab was well tolerated, with 40.5% of patients experiencing largely mild treatment-related adverse events.

Conclusion: Adjuvant nivolumab was effective and well tolerated in Taiwanese patients with UTUC, resulting in promising DFS and OS. These findings suggest potential benefits of nivolumab in patients with UTUC. Further research on genomic and environmental influences and a prospective validation to refine patient selection for adjuvant immunotherapy are needed.