For the treatment of patients with advanced urothelial cancer, pembrolizumab demonstrated anti-tumor activity and an acceptable safety profile, according to a study published in The Lancet Oncology.1

As part of the non-randomized, multi-cohort, open-label phase 1b KEYNOTE-012 basket trial ( Identifier: NCT01848834), researchers evaluated data from 33 patients who had histologically or cytologically confirmed locally advanced or metastatic urothelial cancer with at least 1% PD-L1 expression.

To determine the safety and activity of pembrolizumab, researchers administered the drug intravenously every 2 weeks until study termination or until disease progression or toxic effects.

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While all observed patients received at least 1 dose of pembrolizumab and were subsequently analyzed for safety, 27 patients were assessed for activity in the full analysis. Three patients discontinued treatment with pembrolizumab due to non-treatment-related adverse events before first post-baseline scan, 2 withdrew before the first post-baseline scan, and 1 had “no measurable disease” at baseline.

Of 33 patients, 6 (18%) experienced fatigue and 4 (12%) experienced peripheral edema. Three patients (9%) experienced 5 serious treatment-related adverse events. While 5 patients (15%) experienced 11 grade 3 treatment-related adverse events, no individual event occurred in more than 1 patient.

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Overall response was achieved in 7 patients (26%) among the 27 who were assessed for activity upon median follow-up of 13 months, 3 of which were complete and 4 of which were partial. While there were 4 deaths during the course of the study, the researchers determined that none were related to treatment with pembrolizumab.

The authors concluded that their findings support “ongoing phase 2 and 3 studies of pembrolizumab in this population.”


  1. Plimack ER, Bellmunt J, Gupta S, et al. Safety and activity of pembrolizumab in patients with locally advanced or metastatic urothelial cancer (KEYNOTE-012): a non-randomised, open-label, phase 1b study. Lancet Oncol. 2017 Jan 9. doi: 10.1016/S1470-2045(17)30007-4 [Epub ahead of print]