Efficacy and safety of dose-dense gemcitabine and cisplatin as neoadjuvant chemotherapy for high-grade upper tract urothelial carcinoma (cT2-3N0M0).

Purpose: Neoadjuvant chemotherapy for upper tract urothelial carcinoma has shown favorable results. However, few studies have been conducted on dose-dense regimens that have demonstrated superior efficacy in bladder cancer. We aimed to retrospectively evaluate the efficacy and safety of dose-dense gemcitabine and cisplatin as neoadjuvant chemotherapy for upper tract urothelial carcinoma.

Materials And Methods: Ninety-five patients who underwent radical nephroureterectomy for high-grade upper tract urothelial carcinoma (cT2-3N0M0) with dose-dense gemcitabine and cisplatin (n = 33) or without neoadjuvant chemotherapy (n = 62, Control) were included. Propensity score matching was performed based on the patient and tumor demographics. Efficacy was evaluated by the pathological response rate defined as pathological downstaging to ≤ pT1N0 and complete response (pT0N0). Progression-free survival, cancer-specific survival, and overall survival were estimated. All adverse events and postoperative complications were assessed.

Results: Thirty-one matched patients were included in each cohort after adjusting for baseline propensity score matching. The pathological downstaging to ≤ pT1N0 rate of the neoadjuvant chemotherapy group was significantly higher than the Control group. The complete response rate was 6.5% in the neoadjuvant chemotherapy group; pT0N0 was not observed in the Control group. The 2-year progression-free survival and cancer-specific survival rates were significantly better in the neoadjuvant chemotherapy group. Of the 33 patients who received neoadjuvant chemotherapy, 7 severe adverse events (grade 3 or higher) were observed in 6 patients.

Conclusions: Dose-dense gemcitabine and cisplatin showed a favorable pathological response and oncological outcome with good tolerability.