Adjuvant Immune Checkpoint Therapy for Muscle-Invasive Urothelial Cancer

Patients with muscle-invasive urothelial cancer following optimal therapy with cisplatin-based neoadjuvant chemotherapy and cystectomy remain at high risk for systemic failure, and those who are not candidates for perioperative chemotherapy are at even higher risk. To evaluate adjuvant pembrolizumab in this setting, investigators randomized 702 patients to either receive pembrolizumab (200 mg every three weeks) or undergo observation for one year.

At a median follow-up of 45 months, disease-free survival (DFS), one of the coprimary endpoints, was significantly longer in the pembrolizumab arm than the observation arm (median, 29.6 vs. 14.2 months). Three-year overall survival (OS), the other coprimary endpoint, did not differ significantly between groups (60.8% and 61.9%). Grade 3 or higher adverse events occurred in 50.6% of patients in the pembrolizumab arm compared with 31.6% in the observation arm.