Multicenter phase II study of neoadjuvant gemcitabine-cisplatin-pembrolizumab for MIBC

Muscle-invasive bladder cancer (MIBC) has poor outcomes despite aggressive treatment with cisplatin-based neoadjuvant chemotherapy followed by radical cystectomy (RC). A multicenter phase II study evaluated addition of the immune checkpoint inhibitor pembrolizumab to neoadjuvant gemcitabine and cisplatin before RC. This therapy resulted in a high rate (56%) of pathologic downstaging, meeting its primary end point for improvement over historical controls. The toxicity was manageable.