Biomarkers associated with efficacy of first-line nivolumab vs. sorafenib in advanced hepatocellular carcinoma: results from the CheckMate 459 phase III trial.
Blood and tumor markers can help identify which liver cancer patients benefit most from nivolumab, potentially improving treatment selection.
Exploratory biomarker analysis from the Phase III CheckMate 459 trial identified Wnt/β-catenin pathway alteration as a key modifier of nivolumab efficacy in advanced HCC, with patients harboring unaltered pathway deriving significant OS benefit. High PD-L1 expression (CPS≥10), elevated T-cell inflammation gene signature, and circulating biomarkers including high IFN-γ further refined patient selection, providing a framework for prospective biomarker-stratified trials.
What the study was
- Study design
- Phase III RCT exploratory biomarker analysis
- Population
- Adults with advanced hepatocellular carcinoma
- Sample size
- 201
- Category
- Treatment Innovation
- Maturity
- Potentially Practice-Changing
- Journal
- JHEP Reports
Why it surfaced
Phase III trial exploratory biomarkers for immunotherapy in HCC; Wnt/β-catenin and PD-L1 CPS≥10 subgroups show OS benefit with nivolumab; large dataset (>200/arm); directly practice-relevant for patient selection.
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