Clinical Outcomes of Patients with MIBC Treated with Adjuvant Nivolumab - Expert Commentary

Muscle-invasive urothelial carcinoma is associated with high recurrence rates despite standard radical surgery, with over 50% of patients experiencing disease recurrence within 2-3 years. A recent retrospective cohort study examined real-world effectiveness of adjuvant nivolumab, which received FDA approval based on the CheckMate 274 trial.

The investigators reviewed the records of 253 patients with clinical stage II-IIIB muscle-invasive urothelial carcinoma who initiated adjuvant nivolumab between September 2021 and November 2022. The median follow-up was 12.8 (9.6-15.4) months. Median age at diagnosis was 67.8 (61.5-72.4) years, with 169 patients (66.8%) male. The study population was more diverse than CheckMate 274, including 62 Black or African American patients (24.5%) and 45 patients (18.0%) with ECOG performance status ≥2.

Overall, 141 patients (55.7%) received neoadjuvant chemotherapy, predominantly gemcitabine plus cisplatin (119 patients, 84.4%). Among 112 patients who did not receive neoadjuvant chemotherapy, 88 (78.6%) were cisplatin-ineligible, primarily due to creatinine clearance <60 mL/min (79 patients, 70.5%). Most patients initiated nivolumab at 480 mg every 4 weeks (175 patients, 69.2%). The median treatment duration was 11.2 (8.4-12.0) months, with 220 patients (87.0%) discontinuing therapy primarily due to completion of scheduled duration (163 patients, 74.1%).

Survival outcomes were favorable, with median disease-free survival and overall survival not reached. Twelve-month disease-free survival was 86.3% (95% CI, 81.0%-90.2%), and 12-month overall survival was 90.8% (95% CI, 86.0%-94.0%). Patients who received neoadjuvant chemotherapy had 12-month disease-free survival of 90.0% (95% CI, 82.9%-94.3%) and overall survival of 95.6% (95% CI, 89.6%-98.2%). Among patients who did not receive neoadjuvant chemotherapy, 12-month disease-free survival was 81.6% (95% CI, 72.6%-87.9%) and overall survival was 85.0% (95% CI, 76.3%-90.7%). Adverse events occurred in 52 patients (20.6%), with diarrhea (18 patients, 7.1%) and hypothyroidism (15 patients, 5.9%) most common. Only 10 patients (4.5%) discontinued treatment due to adverse events. Recurrence occurred in 26 patients (10.3%), predominantly distant metastases (25 patients). At last follow-up, 226 patients (89.3%) were alive, with 209 (92.5%) disease-free.

This important study demonstrated clinical outcomes consistent with the CheckMate 274 trial, even with a more diverse population with worse performance status. These results support efficacy of adjuvant nivolumab use in patients who did not receive neoadjuvant chemotherapy, addressing a key clinical question from the original trial. Study limitations include potential selection bias, lack of standardized surveillance protocols, and absence of central imaging review. Extended follow-up is needed to assess long-term clinical outcomes.

Written by: Bishoy M. Faltas, MD, Chief Research Officer, Englander Institute for Precision Medicine, Gellert Family - John P. Leonard, MD, Research Scholar, Associate Professor of Medicine, Cell and Developmental Biology, Weill Cornell Medicine, New York- Presbyterian Hospital, NY

References:

  1. Barragán-Carrillo R, Ebrahimi H, John WS, Lucht S, Miller TA, Pathak P, Bland E, Gordon S, Laney J, Klink AJ, Feinberg B, Singh N, Alonso C, Patel MY, Rosenblatt L, Yin X, Chehrazi-Raffle A. Clinical outcomes in patients with muscle-invasive urothelial carcinoma treated with nivolumab. JAMA Netw Open. 2025;8(6):e2514427. doi:10.1001/jamanetworkopen.2025.14427
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