FDA Approves Pembrolizumab for BCG-Unresponsive, High-Risk Bladder Cancer

The FDA approved pembrolizumab (Keytruda) for the treatment of patients who are unresponsive to Bacillus Calmette-Guerin (BCG) treatment with high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

The approval was based off the multicenter, single-arm KEYNOTE-057 trial, which included 148 patients with high-risk NMIBC, 96 of whom had BCG-unresponsive CIS with or without papillary tumors. Participants were given 200 mg of pembrolizumab every 3 weeks (the recommended dose) until unacceptable toxicity, persistent/recurrent progressive disease, or 24 months.

The complete response (CR) rates in the patients with high-risk BCG-unresponsive NMIBC with CIS was 41% (95% CI, 31-51). The average response duration was 16.2 months, and 46% of patients who responses had a complete response that lasted 12 months or longer.

Key endpoints were complete response — measured by cyctoscopy with TURBT/biopsies as applicable—as well as urine cytology, and computed tomography urography (CTY) imaging, and duration of response.

Common adverse events that nurses should look out for include: fatigue, diarrhea, rash, pruritis, musculoskeletal pain, hematuria, cough, arthralgia, nausea, constipation, urinary tract infection, peripheral edema, hypothyroidism, and nasopharyngitis.