Phase 3, randomized study of pembrolizumab (pembro) vs best supportive care (BSC) for second-line advanced hepatocellular carcinoma (HCC): KEYNOTE-240.

TPS4143Background: The tyrosine kinase inhibitor sorafenib is the standard of care for first-line HCC; currently, there is no clear standard of care after disease progression on sorafenib or for patients (pts) with intolerance to sorafenib. Because most HCC is driven by inflammation, there is a strong rationale to evaluate immunotherapy in pts with this type of cancer. The randomized, double-blind, placebo-controlled phase 3 KEYNOTE-240 study (ClinicalTrials.gov, NCT02702401) was designed to compare the efficacy and safety of the anti–PD-1 antibody pembro + BSC vs placebo + BSC in pts with previously treated advanced HCC. Methods: Eligibility criteria include age ≥ 18 years, histologically or cytologically confirmed diagnosis of HCC, documented progression after stopping treatment with sorafenib or intolerance to sorafenib, disease not amenable to a curative treatmentapproach (eg, transplantation, surgery, or ablation), measurable disease confirmed by central imaging vendor review per RECIST v1.1, Child-P...