Pembrolizumab in patients with Bacillus Calmette Guérin (BCG)-unresponsive, high-risk non–muscle-invasive bladder cancer (NMIBC): Phase 2 KEYNOTE-057 study

Abstract

Background: Despite standard-of-care therapy with transurethral resection of bladder tumor (TURBT) and intravesical BCG instillation, a large percentage of patients with NMIBC have disease recurrence/progression. PD-L1 is widely expressed in urothelial tumors, providing a therapeutic rationale for targeting the PD-1/PD-L1 pathway in NMIBC. KEYNOTE-057 (NCT02625961) is an open-label, phase 2 study designed to evaluate the efficacy of the anti-PD-1 antibody pembrolizumab (pembro) in patients with high-risk, BCG-unresponsive NMIBC. Trial design: Eligible patients must be age >=18 years; have histologically confirmed diagnosis of high-risk, BCG-unresponsive NMIBC (high-grade Ta, T1, and/or carcinoma in situ [CIS] despite adequate BCG treatment); be ineligible for or have declined radical cystectomy; and have ECOG PS 0-2. Patients must have undergone >=2 cystoscopic procedures with the most recent