Circulating miRNAs as potentially predictive biomarkers for chemoimmunotherapy in advanced biliary tract cancer: a...

Circulating microRNAs (miRNAs) have not been investigated as predictive biomarkers in patients with advanced biliary tract cancer (ABTC) who received chemoimmunotherapy. In the single-arm phase II T1219 trial, 46 patients with ABTC received first-line nivolumab, gemcitabine, and S-1 treatment. Plasma samples were prospectively collected at baseline and 6 weeks after treatment for miRNA profiling. Among the 167 miRNAs analyzed, the baseline levels of hsa-miR-16-5p, hsa-miR-93-5p, and hsa-miR-126-3p were significantly elevated in the responders. High hsa-miR-16-5p expression was correlated with longer progression-free survival (hazard ratio [HR] = 0.44, 95% confidence interval [CI] = 0.12-0.85, p = 0.025) and overall survival (HR = 0.34, 95% CI = 0.07-0.7, p = 0.01). Post-treatment miRNA changes (e.g., hsa-miR-129-5p and hsa-miR-1254) were observed but were not significantly associated with survival. This study identified a three-miRNA signature with potential predictive value for chemoimmunotherapy in ABTC. Trial registration: NCT04172402, date of registration 2019/11/18.