Abstract
BACKGROUND: Anti-programmed cell death protein-1 immunotherapy has been approved as a new treatment option for advanced hepatocellular carcinoma (HCC) based on the promising results of several studies. METHODS: This retrospective study included 71 patients with advanced HCC treated with anti-programmed cell death protein-1 immunotherapy between June 1, 2017 and September 30, 2020 at the First Affiliated Hospital of Anhui Medical University. Responses to pulmonary metastases were evaluated. RESULTS: The median follow-up duration was 7.73 months (95% confidence interval (CI), 4.48-10.98). Of 71 patients, the overall response rate (ORR) and disease control rate (DCR) were 32% (23/71) and 73% (52/71), respectively. The median progression-free survival (PFS) and overall survival (OS) were 4.90 (95% CI, 2.71-7.09) and 20.23 (95% CI, 6.87-33.59) months, respectively. Forty-two patients had HCC pulmonary metastases, whereas 29 did not have pulmonary metastasis. No significant differences were observed in the ORR (38% [16/42] vs. 24% [7/29], P = 0.22) and DCR (74% [31/42] vs. 72% [21/29], P = 0.90) between groups. In patients with pulmonary metastases, the median disease control duration of pulmonary lesions was significantly longer than extrapulmonary lesions (Not Reached vs. 12.37 months, P = 0.048). Pulmonary metastases were not associated with an increased incidence of adverse events (67% vs. 62%, P = 0.69). CONCLUSIONS: Anti-programmed cell death protein-1 immunotherapy showed promising efficacy and safety in patients with advanced HCC, with good responses observed in pulmonary metastases. The mechanism underlying the differences in responses between pulmonary and extrapulmonary metastases requires further investigation.