Abstract
Background: Pembrolizumab is approved for patients with metastatic urothelial carcinoma (UC) who progressed under platinum therapy. The aim of this study was to assess the efficacy and safety of pembrolizumab in a cohort of real-life UC patients. Methods: This retrospective, observational study included advanced UC patients treated with pembrolizumab in a single institution in France. The co-primary endpoints were overall survival (OS) and progression-free survival (PFS) at 6 months. Secondary endpoints were objective response rate (ORR), duration of response (DOR), disease control rate (DCR) and safety. Results: 78 patients were included in the study. The median OS was 7.3 months (3.8–12.2). The estimated OS rate at 6 months was 61.5% (50.5–72.6). The median PFS was 3.1 months (1.4–7.2). The estimated PFS rate at 6 months was 42.3% (31.1–53.5). The best ORR was 35.9%. The mean DOR was 95.5 days. The DCR was 30.8%. The most common treatment-related adverse events (AEs) of any grade were fatigue (46.2%), diarrhea (11.5%), pruritus (10.3%) and nausea (9.0%). There were no grade 3 AEs that occurred with an incidence of 5% or more. Conclusion: Our results confirmed those of randomized clinical trials concerning the treatment with pembrolizumab in patients with advanced UC that progressed after platinum-based chemotherapy.
Highlights
Metastatic urothelial carcinoma (UC) is a highly lethal disease with a poor prognosis and a 5-year overall survival (OS) of less than 5% [1]
Intravesical instillation of Bacillus of Calmette-Guérin (BCG) in high-risk, nonmuscle invasive disease has shown that UC could be a suitable candidate for modern immunotherapy [4]; UC has a high immunogenicity, an element which supports the application of immune checkpoint inhibitors (ICIs) in the advanced or metastatic setting [5]
In the phase 1b Keynote-012 study [9] and in the phase 2 Keynote-052 study [10]. The results of these two trials led to the randomized, phase 3 Keynote-045 trial [11], an open label study in which 542 selected patients who progressed under platinum therapy were randomized to receive pembrolizumab at a dose of 200 mg every 3 weeks or chemotherapy
Summary
Metastatic urothelial carcinoma (UC) is a highly lethal disease with a poor prognosis and a 5-year overall survival (OS) of less than 5% [1]. Pembrolizumab is a humanized IgG4 monoclonal antibody against PD-1, blocking its engagement with its ligands (PD-L1 and PD-L2) It has been recently approved by the European Medicines Agency as monotherapy for the treatment of locally advanced or metastatic UC in adults who have received prior platinum-based chemotherapy and for treatment of locally advanced or metastatic UC in adults who are not eligible for cisplatincontaining chemotherapy and whose tumors express PD-L1 with a combined positive score (CPS) ≥ 10. Pembrolizumab has shown a robust antitumor activity and a good safety profile in UC in the phase 1b Keynote-012 study [9] and in the phase 2 Keynote-052 study [10] The results of these two trials led to the randomized, phase 3 Keynote-045 trial [11], an open label study in which 542 selected patients who progressed under platinum therapy were randomized to receive pembrolizumab at a dose of 200 mg every 3 weeks or chemotherapy. We provided complementary information to previous randomized controlled trials (RCTs) in a real-world study, whose aim was to assess the efficacy and safety of pembrolizumab in a cohort of real-life patients with UC treated in a single hospital in France
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