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REVIEW

Efficacy of pembrolizumab in urothelial cancer: A systematic review and meta-analysis

Vinay Eruvuri1 ,  Nitish Nichenametla2 ,  Monish Konda3 ,  Bala Sathvika Duggimpudi4 ,  Yethindra Vityala5*
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1 Department of General Medicine, Apollo Institute of Medical Sciences and Research, Chittoor, Andhra Pradesh 517127, India
2 Department of General Medicine, Kurnool Medical College, Kurnool, Andhra Pradesh 518002, India
3 Department of General Medicine, Shri B. M. Patil Medical College, Hospital and Research Centre, Bijapur, Karnataka 586103, India
4 Department of General Medicine, Government Medical College, Mahabubnagar, Telangana 509001, India
5 Department of Pathology, International Higher School of Medicine, Bishkek 720054, Kyrgyzstan
Bladder, e21200072 https://doi.org/10.14440/bladder.0249
Submitted: 11 August 2025 | Revised: 8 September 2025 | Accepted: 16 September 2025 | Published: 24 October 2025
© 2025 by the Author(s). This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution 4.0 International License ( https://creativecommons.org/licenses/by/4.0/ )
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Abstract

Background: Urothelial bladder cancer is a major health challenge owing to its high incidence, elevated mortality rates, and clinical diversity. Objective: This systematic review and meta-analysis assesses the efficacy of pembrolizumab in the treatment of non-muscle-invasive bladder cancer (NMIBC), muscle-invasive bladder cancer (MIBC), and metastatic urothelial carcinoma (mUC). After applying the inclusion and exclusion criteria, a literature search of PubMed, Scopus, and Web of Science (January 2020–June 2025) identified eight studies on NMIBC, MIBC, and mUC for the final analysis. Analysis revealed an advantage of pembrolizumab-based treatment (odds ratio [OR]: 1.86; 95% confidence interval [CI]: 1.70–2.03). Subgroup analyses showed the effectiveness in MIBC (OR: 1.88; 95% CI: 1.65–2.12) and mUC (OR: 1.94; 95% CI: 1.82–2.11). In contrast, the NMIBC subgroup lacked significance (OR: 1.28; 95% CI: 0.86–1.91), reflecting the evidence from small, early-phase studies. Pembrolizumab showed improved pathological complete response, progression-free survival, and disease-free survival in groups with elevated programmed death-ligand 1 expression and a high tumor mutational burden. Conclusion: Pembrolizumab is highly effective in treating MIBC and mUC; however, its impact on NMIBC remains uncertain. The limited number of studies and brief follow-up periods highlight the need for larger trials with long-term survival data.

Keywords
Urothelial bladder cancer
Pembrolizumab
PD-1/PD-L1
Muscle-invasive bladder cancer
Non-muscle-invasive bladder cancer
Funding
None.
Conflict of interest
The authors declare that they have no conflict of interest
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