Characterization of Patients with Lymph Node Only Metastatic Urothelial Carcinoma Treated with Nivolumab Plus Gemcitabine-Cisplatin Versus Gemcitabine-Cisplatin Alone from the CheckMate 901 Trial.

Nivolumab (NIVO) plus gemcitabine-cisplatin (GC) demonstrated deep, durable responses in patients with untreated unresectable or metastatic urothelial carcinoma (mUC) with superior overall (OS) and progression-free (PFS) survival versus GC alone. We report post hoc exploratory analyses with 33.6 mo of median follow-up in patients with lymph node (LN) only disease from CheckMate 901. In this phase 3 randomized trial (n = 608), cisplatin-eligible patients received NIVO 360 mg plus GC every 3 wk (Q3W) for six or fewer cycles followed by NIVO 480 mg every 4 wk for ≤2 yr, or GC Q3W for six or fewer cycles. The primary endpoints were OS and PFS by a blinded independent central review (BICR). The objective response rate (ORR) as per the BICR and safety were exploratory. This post hoc analysis evaluated patients with LN only mUC (n = 110). In this subgroup, OS (hazard ratio [HR], 0.58; 95% confidence interval [CI] 0.34-1.00) and PFS (HR, 0.38; 95% CI 0.22-0.66) improved with NIVO + GC versus GC. The ORR was 81% with NIVO + GC versus 64% with GC; complete response rates were 63% versus 34%. The incidence of treatment-related adverse events was similar between arms. In conclusion, clinically meaningful improvements in OS, PFS, and ORR, including twice the rate of complete response and more patients with treatment-free intervals, were observed with NIVO + GC versus GC in patients with LN only disease, providing additional support for first-line NIVO plus cisplatin-based chemotherapy in mUC.