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Predictive biomarkers for immune checkpoint inhibitor response in urothelial cancer
Pauline Parent1, Gautier Marcq2, Sola Adeleke3,4,5
1Medical Oncology Department, Centre Hospitalier Universitaire de Lille (CHU Lille), University of Lille, Hôpital Huriez, Lille 59037, France.
Abstract:
Immune checkpoint inhibitors (ICIs) are commonly used to treat patients with advanced urothelial cancer. However, a significant number of patients do not respond to ICI, and the lack of validated predictive biomarkers impedes the success of the ICI strategy alone or in combination with chemotherapy or targeted therapies. In addition, some patients experience potentially severe adverse events with limited clinical benefit. Therefore, identifying biomarkers of response to ICI is crucial to guide treatment decisions. The most evaluated biomarkers to date are programmed death ligand 1 expression, microsatellite instability/defective mismatch repair phenotype, and tumor mutational burden. Other emerging biomarkers, such as circulating tumor DNA and microbiota, require evaluation in clinical trials. This review aims to examine these biomarkers for ICI response in urothelial cancer and assess their analytical and clinical validation.
Insights
Predictive biomarkers are crucial for immune checkpoint inhibitor (ICI) therapy in advanced urothelial cancer. This review examines current and emerging biomarkers to improve treatment selection and patient outcomes.
Area of Science:
- Oncology
- Immunology
- Genetics
Background:
- Immune checkpoint inhibitors (ICIs) are a key treatment for advanced urothelial cancer.
- Many patients do not respond to ICIs, and lack of predictive biomarkers limits treatment success.
- Adverse events with limited clinical benefit highlight the need for better patient selection.
Purpose of the Study:
- To review and assess biomarkers predicting response to ICIs in urothelial cancer.
- To evaluate the analytical and clinical validation of established and emerging biomarkers.
- To guide treatment decisions for improved ICI efficacy and safety.
Main Methods:
- Literature review of biomarkers for ICI response in urothelial cancer.
- Analysis of programmed death ligand 1 (PD-L1) expression, microsatellite instability (MSI)/mismatch repair deficiency (dMMR), and tumor mutational burden (TMB).
- Evaluation of emerging biomarkers including circulating tumor DNA (ctDNA) and microbiota.
Main Results:
- PD-L1 expression, MSI/dMMR, and TMB are the most studied predictive biomarkers.
- Emerging biomarkers like ctDNA and microbiota show potential but require further clinical validation.
- No single biomarker currently guarantees prediction of ICI response.
Conclusions:
- Validated predictive biomarkers are essential for optimizing ICI therapy in urothelial cancer.
- Further clinical trials are needed to validate emerging biomarkers.
- Biomarker-guided treatment selection can improve patient outcomes and reduce unnecessary toxicity.
