Background
Although immune checkpoint inhibitors (ICIs) therapy has improved the prognosis of unresectable hepatocellular carcinoma (HCC), it has also resulted in the unique immune-related adverse events (irAE). The relationship between irAE and treatment outcomes in ICIs-treated advanced HCC patients remains unknown.
Methods
From March 2019 to February 2021, a total of 190 unresectable HCC (BCLC C) patients receiving Pembrolizumab treatment were retrospectively reviewed. Overall survival (OS) was the primary endpoint. objective response rate (ORR), disease control rate (DCR) and time to progression (TTP) were secondary evaluation indexes. We assessed demographics, irAE and outcomes by retrospective review.
Results
One hundred and forty-three male and 47 female were included in the study. The ORR and DCR were 12.1% (23/190) and 52.1% (99/190) respectively. The median OS was 376 days (95% CI 340 -411 days) and the median TTP was 98 days (95% CI 75 -124 days). The overall incidence of treatment-related adverse events was 72.6% (138/190) and 10.0% of them were severe irAEs (grade ≥ 3). Child-Pugh B class, PVTT, extrahepatic metastasis, and hypothyroidism were the independent risk factors of survival. Patients with hypothyroidism were observed a longer OS than those without irAE (517 days [95% CI 423-562] vs. 431 days, [95% CI 412-485], P=0.011) and a longer TTP (125 [95% CI 89-154] vs. 87 days [95% CI 61-98], P=0.004).
Conclusion
Unresectable HCC patients experienced hypothyroidism indicated a bettertherapeutic effect.Hypothyroidism, an immune-related adverse event may be used as a clinical evaluation parameter of HCC response to ICIs.