S2801 Efficacy and Safety of Immune Checkpoint Inhibitors in Hepatocellular Carcinoma: A Systematic Review and Meta-Analysis

Introduction: Immune checkpoint inhibitors (ICIs) are now central to advanced hepatocellular carcinoma (HCC) therapy. A previous meta-analysis demonstrated significant improvements in tumor response and survival outcomes. Since that publication, 6 randomized controlled trials (RCTs) with updated data have been released. We conducted an updated systematic review and meta-analysis to incorporate these additional trials and evaluate contemporary ICI efficacy and safety in HCC. Methods: PubMed, Google Scholar, and Cochrane Library were searched following PRISMA guidelines for RCTs comparing ICIs with standard therapy or placebo in HCC patients. Eligible trials reported objective response rate (ORR), disease control rate (DCR), survival, and adverse-event outcomes. Pooled odds ratios (ORs) for response metrics and hazard ratios (HRs) for survival endpoints were calculated using a random-effects model. Results: Eighteen RCTs involving 9,244 patients met inclusion criteria. ICIs significantly improved ORR (OR 3.20; 95% CI 2.44–4.20; P < 0.00001), DCR (OR 1.40; 95% CI 1.08–1.81; P = 0.01), and stable disease rates (OR 2.15; 95% CI 1.16–3.98; P = 0.02). Survival analysis favored ICIs for overall survival (HR 0.79; 95% CI 0.73–0.86; P < 0.00001) and progression-free survival (HR 0.77; 95% CI 0.69–0.87; P < 0.00001). ICIs were associated with higher odds of all-cause grade ≥3 adverse events (OR 1.36; 95% CI 1.10–1.67; P = 0.005), with no other significant safety differences observed. Conclusion: This updated meta-analysis confirms that ICIs significantly improve tumor response and survival outcomes in HCC while maintaining an acceptable safety profile. These findings reinforce the role of ICIs as a key component of systemic HCC therapy and highlight the need for further research to optimize treatment combinations and patient selection.