Biomarker-Guided ICI Therapy Provides Greater Benefit in Cancer Patients

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Cancer patients with biomarker-positive tumors have better outcomes on immune checkpoint inhibitor therapy, a meta-analysis suggests.

Biomarkers should be used when selecting cancer patients for immune checkpoint inhibitor (ICI) therapy, according to researchers. 

They found that cancer patients who have biomarker-positive tumors have better objective response rates (ORR), progression-free survival (PFS), and overall survival (OS) on ICI treatment.

These findings, from a meta-analysis, were published in the European Journal of Cancer.

The meta-analysis included 174 phase 1 and 2 trials that encompassed 19,178 cancer patients. The most common cancer types were melanoma (27 studies), non-small cell lung cancer (n=26), esophageal or gastric cancer (n=17), and breast cancer (n=14). 

Correlative predictive biomarkers were investigated in 132 studies. Biomarkers included PD-L1 expression (n=111), tumor mutational burden (n=20), mutations (n=17), tumor-infiltrating lymphocytes (n=15), microsatellite instability/mismatch repair deficiency (n=10), gene expression/RNA-based signatures (n=10), and other biomarkers.

Patients with biomarkers had a significantly higher ORR than patients without biomarkers; 30% and 16%, respectively (odds ratio [OR}, 2.15; 95% CI, 1.79-2.58, P <.0001).

Patients with biomarkers also had significantly better PFS (hazard ratio [HR], 0.55; 95% CI, 0.45-0.67; P <.0001) and OS (HR, 0.65; 95% CI, 0.53-0.80; P <.0001).

In a multivariate analysis, patients with biomarkers had significantly better ORRs (OR, 2.11; 95% CI, 1.77-2.51, P <.001) and PFS (HR, 0.56; 95% C.I, 0.43-0.72; P <.001). Due to a small number of studies, OS was not included in the multivariate analysis.

“This meta-analysis demonstrated that the use of immune-related biomarkers is important for selection of patients who will benefit from ICIs,” the researchers concluded. 

Disclosures: Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.


Fountzilas E, Vo HH, Mueller P, et al. Correlation between biomarkers and treatment outcomes in diverse cancers: A systematic review and meta-analysis of phase I and II immunotherapy clinical trials. Eur J Cancer. Published online May 22, 2023. doi:10.1016/j.ejca.2023.05.015

This article originally appeared on Cancer Therapy Advisor