Adjuvant Nivolumab Therapy for Upper Tract Urothelial Carcinoma: A Single-Institution Study

While immune checkpoint inhibitors have transformed adjuvant therapy for urothelial carcinoma, data focused on upper tract urothelial carcinoma (UTUC) remain scarce. In the CheckMate 274 trial, the UTUC subgroup did not demonstrate a definitive survival benefit. This study aimed to evaluate the clinical outcomes of adjuvant nivolumab in patients with high-risk UTUC.

Of the 41 patients who underwent radical nephroureterectomy for UTUC at our institution between April 2022 and April 2024, 11 received adjuvant nivolumab, thereby retrospectively analyzed. Their treatment adherence and immune-related adverse events (irAEs) and survival outcomes were assessed.

Of the 11 patients, eight patients (72.7%) received neoadjuvant chemotherapy. Seven (63.6%) completed the 12-month nivolumab regimen. Four discontinued the treatment because of irAEs (n = 2) and disease recurrence (n = 2). None experienced treatment-related death. The one- and two-year metastasis-free survival rates were 81.8% and 70.1%, respectively, with a median of 30 months. The two-year disease-free and overall survival rates were 40.9% and 79.5%, respectively.

This is one of the first real-world experiences focused exclusively on patients receiving adjuvant nivolumab for UTUC. The observed delay in distant progression may reflect the systemic therapeutic activity of immune checkpoint blockade in this setting.