What is the role of immunotherapy in the treatment of bladder cancer?

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Last updated: April 30, 2025View editorial policy

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From the Guidelines

Immunotherapy is a highly effective treatment option for bladder cancer, and the most recent guidelines recommend its use for patients with non-muscle invasive bladder cancer (NMIBC) and advanced or metastatic disease, as stated in the 2024 NCCN guidelines 1.

Key Recommendations

  • For NMIBC, Bacillus Calmette-Guérin (BCG) therapy is the standard immunotherapy approach, typically administered as intravesical instillations once weekly for 6 weeks (induction), followed by maintenance therapy for 1-3 years with 3-week cycles at months 3,6,12,18,24,30, and 36.
  • For advanced or metastatic bladder cancer, immune checkpoint inhibitors like pembrolizumab (200mg IV every 3 weeks), nivolumab (240mg IV every 2 weeks), atezolizumab (1200mg IV every 3 weeks), durvalumab, and avelumab have shown efficacy, as reported in the 2020 NCCN guidelines 1.

Mechanism of Action

  • Immunotherapy works by enhancing the body's natural immune response against cancer cells, either by stimulating local inflammation (BCG) or by removing the brakes on the immune system (checkpoint inhibitors), allowing T-cells to recognize and attack cancer cells more effectively.

Side Effects and Monitoring

  • Common side effects include fatigue, rash, diarrhea, and potential immune-related adverse events affecting various organ systems.
  • Regular monitoring of liver and kidney function is essential during treatment, as recommended by the Society for Immunotherapy of Cancer consensus statement 1.

Recent Updates

  • The 2024 NCCN guidelines provide updated recommendations for the use of immunotherapy in bladder cancer, including the use of molecular/genomic testing to facilitate treatment decision-making and to prevent delays in administering later lines of therapy 1.

From the Research

Immunotherapy for Bladder Cancer

  • Immunotherapy has shown significant promise in the treatment of bladder cancer, with several clinical trials demonstrating its efficacy as a second-line treatment for metastatic urothelial cancer 2, 3, 4.
  • The US Food and Drug Administration has approved five drugs for the treatment of metastatic urothelial cancer, including three Programmed cell-death protein 1 (PD-1) inhibitors and two programmed cell-death ligand 1 (PD-L1) inhibitors 2.
  • Pembrolizumab, nivolumab, and atezolizumab are PD-1 inhibitors, while durvalumab and avelumab are PD-L1 inhibitors 2.
  • Atezolizumab and pembrolizumab are the only Food and Drug Administration-approved checkpoint inhibitors for cisplatin-ineligible patients 2.
  • The KEYNOTE-361 clinical trial demonstrated that pembrolizumab plus chemotherapy did not significantly improve efficacy compared to chemotherapy alone as a first-line treatment for advanced urothelial carcinoma 5.
  • A cost-effectiveness analysis of pembrolizumab versus carboplatin-based chemotherapy as first-line treatment for PD-L1-positive locally advanced or metastatic urothelial carcinoma ineligible for cisplatin-based therapy found that pembrolizumab was cost-effective 6.

Clinical Trials and Studies

  • The KEYNOTE-045, IMvigor211, CheckMate275, Javelin Solid Tumor, MEDI4736, and KEYNOTE-0528 clinical trials have investigated the use of immunotherapy in bladder cancer 2.
  • The KEYNOTE-361 clinical trial was a randomized, open-label, phase 3 trial that compared pembrolizumab alone or combined with chemotherapy versus chemotherapy as first-line therapy for advanced urothelial carcinoma 5.
  • A study published in the Journal of Clinical Advances in Hematology & Oncology discussed the recent development of T-cell checkpoint inhibitors in advanced and metastatic urothelial cancer and addressed their potential use in combination 4.

Treatment Options and Efficacy

  • Immunotherapy is approved as a second-line treatment for metastatic urothelial cancer, and its use as a first-line agent is limited to patients who are ineligible for cisplatin-based treatments 2.
  • Pembrolizumab has been shown to have meaningful, durable, and well-tolerated antitumor activity in patients with advanced or metastatic urothelial carcinoma 6.
  • The addition of pembrolizumab to first-line platinum-based chemotherapy did not significantly improve efficacy and should not be widely adopted for treatment of advanced urothelial carcinoma 5.

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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