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Can Biomarkers Predict Outcomes With Nivolumab in Refractory Biliary Tract Cancer?

By: Sarah Campen, PharmD
Posted: Wednesday, November 2, 2022

Richard D. Kim, MD, of Moffitt Cancer Center, Tampa, Florida, and colleagues have shared data on several biomarkers that may predict outcomes in patients with advanced refractory biliary tract cancer who are treated with nivolumab. Published in the European Journal of Cancer, their analysis identified tumor PD-L1 expression, CD8 T-cell infiltration, and high ratio of CD8 to regulatory T cells as potential predictive biomarkers. The authors also developed a six-gene prediction model to attempt to forecast response to nivolumab in patients with cholangiocarcinoma. However, the authors cautioned that further large-scale studies are warranted to verify these study findings, particularly given the recent approvals of durvalumab plus chemotherapy in advanced biliary tract cancer as well as durvalumab plus tremelimumab in hepatocellular carcinoma.

This study incorporated data from a phase II multi-institutional study of nivolumab in patients with advanced refractory biliary tract cancer ( identifier NCT02829918), including baseline clinicopathologic characteristics and pretreatment tumor samples. The tumor samples were assessed for whole-exome sequencing, RNA sequencing, and immunohistochemistry, and then they were compared with clinical outcomes.

The pretreatment tumor evaluation found that PD-L1 expression on the tumor (P = .0001), CD8 T-cell infiltration (P = .01), and a high ratio of CD8 T cells to regulatory T cells in the tumor microenvironment (P = .02) were significantly associated with prolonged progression-free survival. There was no apparent correlation observed between progression-free survival and either PD-1 expression on lymphocytes or CD68-expressing macrophage infiltration in the tumor microenvironment.

The authors also constructed a six-gene predictive model by screening 23,550 candidate genes from RNA sequencing of the baseline tumor samples. A high score on the predictive model was associated with a significant improvement in progression-free survival with nivolumab compared with a low score (6.8 vs. 1.8 months, P = .0005).

Disclosure: For full disclosures of the study authors, visit

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