Multicenter phase 2 trial of organ-sparing chemo-immunotherapy for patients with muscle-invasive bladder cancer

Cystectomy is a standard treatment for muscle-invasive bladder cancer (MIBC), but it is life-altering. A multicenter phase 2 trial in the USA evaluated four cycles of gemcitabine, cisplatin, and nivolumab, followed by clinical restaging. Patients achieving a clinical compete response (cCR) could proceed without cystectomy. Co-primary objectives were cCR rate and the positive predictive value of cCR for a) 2-year metastatisc-free survival in patients forgoing immediate cystectomy, or b)<ypT1N0 in patients electing immediate cystectomy. The cCR rate was 43% (95% CI 32-55), and the positive predictive value was 0.97 (95% CI 0.91-1.00), meeting the primary objective. The most common adverse events werd fatigue, anemia, neutropenia, and nausea. The authors conclude that cCR after gemcitabine, cisplatine, plus nivolumab facilitated bladder sparing and warrants further study.