Abstract
BACKGROUND: Hepatocellular carcinoma (HCC) is one of the most common malignant solid tumors. Its incidence is increasing worldwide due to the dissemination of hepatitis B infection, HCV infection and nonalcoholic steatohepatitis-related HCC. For patients with advanced HCC, the available treatments are extremely limited and the prognosis is very poor. Therefore, it is urgent to discover new innovative approaches. Programmed cell death protein-1-targeted immunotherapy has shown promising results in multicenter clinical trials. AIM: To evaluate the effectiveness and safety of anti-PD-1 agent in patients with advanced primary hepatocellular carcinoma. METHODS: A retrospective analysis of 55 patients with advanced primary hepatocellular carcinoma who had been administered anti-PD-1 agent. Tumor response was assessed according to the modified Response Evaluation Criteria in Solid Tumors and any adverse events were recorded. RESULTS: The median overall survival (OS) was 15 months. The median progression-free survival (PFS) was 10 months. No patient had complete response (CR) and 12 (22%) participants achieved partial response (PR), resulting in an overall response rate (ORR) of 22%. Thirty-seven (67%) patients showed stable disease (SD) and 6 (11%) subjects had progressive disease (PD) at first radiological evaluation. The disease control rate (DCR) was 89%. The total side effect rate was 61.8% and most were relieved after treatment. CONCLUSION: Programmed cell death protein-1-targeted immunotherapy is a safe and effective treatment for advanced primary hepatocellular carcinoma.