Introduction: Hepatocellular carcinoma (HCC) is a major challenge in oncology. It ranks fourth in most common causes of cancer death worldwide. Despite advancements in cancer treatment, limited effective treatment options exist for advanced HCC. Immune checkpoint inhibitors have become an irreplaceable tool in the treatment of many metastatic cancers. Early phase trials have demonstrated superior efficacy and good safety profile of immune checkpoint inhibitors and its combination with other drugs in the treatment of advanced HCC.
Areas covered: The scientific rationale and the current state-of-the-art of treatment in HCC involving immune checkpoint inhibitors, either as monotherapy or in combination with other drugs are reviewed and discussed.
Expert opinion: Immune checkpoint inhibitors have shown clinically relevant benefits as monotherapy in advanced HCC. These agents have shown superior survival benefits, durable response and manageable safety profiles in advanced HCC. Recent triumph of combination strategy with immune checkpoint inhibitor and anti-VEGF agent will likely bring a paradigm shift in systemic treatment of advanced HCC. Further research are needed to identify predictive biomarkers for response and best treatment sequence prioritization. Financial cost remains a major impediment for the widespread use of these novel treatments.
Keywords: Hepatocellular carcinoma; ctla-4; immune checkpoint inhibitor; pd-1; pd-l1.