PD-1 targeted immunotherapy in advanced hepatocellular carcinoma: efficacy and safety data from an international multicenter real-world cohort

Effectiveness of nivolumab versus regorafenib in hepatocellular carcinoma patients who failed sorafenib treatment

“…The objective response rate with nivolumab was 16.7% in the current study, a finding that is consistent with previous studies reporting objective response rates of 14.3-16.7%. 10,14,25 However, the objective response rate with regorafenib (5.9%) was lower compared to the previous studies (10.0-10.6%). 5,15 In the RESORCE trial, the objective response rate was 10.6%, and 68% of patients experienced drug interruption or dose modification during the treatment.…”

“…9,10 In contrast, recent real-world evidence studies evaluating the efficacy of nivolumab reported that median OS with nivolumab ranged from <2 months to 11.0 months. [11][12][13][14] Considering the major causes of death of advanced HCC patients were intrahepatic tumor progression and complications of cirrhosis, the discrepancy between the results of clinical trials and real-world studies can be explained by the differences in baseline characteristics of the study population. 16,18 However, even though regorafenib treatment demonstrated efficacy over placebo in prolonging OS after sorafenib failure in the RESORCE trial, and the ability of nivolumab to improve OS compared to regorafenib was demonstrated in the current study, survival gain with nivolumab treatment is still unsatisfactory.…”

“…The safety profiles of regorafenib and nivolumab in the present study were generally consistent with those reported in previous studies. 5,6,[13][14][15]25 Whilst both treatments showed comparable safety in terms of drug discontinuation rate, the proportion of patients with impaired hepatic function was significantly higher among patients receiving nivolumab compared to regorafenib at baseline. Furthermore, three of four patients who discontinued nivolumab treatment due to hepatic decompensation had extensive intrahepatic tumor burden (>50%) at the start of treatment.…”

“…Previous studies including Child-Pugh class B patients with HCC showed similar safety profile of nivolumab between Child-Pugh class A and Child-Pugh Class B subgroups. 14,25 Moreover, one patient with Child-Pugh class C disease at baseline showed complete response during the treatment with nivolumab in a small study involving 14 HCC patients. 31 The safety of nivolumab in patients with hepatic impairment needs to be validated in future studies.…”

“…Though regorafenib and nivolumab have been proven effective in pivotal clinical trials, study results in real-world clinical practice are limited. [11][12][13][14][15] In addition, a direct comparison of the efficacy of regorafenib and nivolumab has not been conducted to date. As sorafenib failed patients raised importance in clinical practice, we also included sorafenib intolerant patients.…”