Clinical Characteristics of Long Term Survivors Following Sorafenib Treatment for Unresectable Hepatocellular Carcinoma: Korean National Multi-Center Retrospective Cohort Study = Clinical Characteristics of Long Term Survivors Following Sorafenib Treatment for Unresectable Hepatocellular Carcinoma: Korean National Multi-Center Retrospective Cohort Study

다국어 초록 (Multilingual Abstract)

Aims: Sorafenib is the standard systemic therapy for treatment of advanced stage hepatocellular carcinoma (HCC), and progressive HCC after locoregional therapy. The efficacy and safety of the drug in clinical practice have been addressed by several real world studies. However, little data are available regarding clinical factors of good responders following sorafenib treatment. This study aimed to evaluate prognostic factors of long term survivors.
Methods: This multi-center retrospective cohort study included 1,605 unresectable HCC patients who received sorafenib treatment between 2007 and 2014 in 9 tertiary centers in Korea. The patients were classified into long-term survivor group (survival longer than 2 years, n=246) or control group (n=1,359). The primary endpoint was prognostic factors on survival for overall patients. Secondary endpoints included time-to-progression and other safety profiles.
Results: The patients were predominantly male (83.8%), chronic hepatitis B (77.3%), and Barcelona Clinic of Liver Cancer stage C (78.3%). The median overall survival was 9.0 months. After treatment, 12 patients (0.7%) achieved complete response, and 146 patients (9.1%) achieved partial response by mRECIST criteria. The prognostic factors predicting long-term survival were metformin use (hazard ratio [HR]=2.44; P=0.004), hand foot skin reaction (HR=1.77; P=0.001), and concomittent treatment with chemoembolization or radiotherapy (HR =2.59; P<0.001). Prognostic factors against long-term survival were child pugh score B (HR=0.375; P< 0.001), presence of extrahepatic metastasis (HR=0.625; P=0.002), main portal vein invasion (HR=0.625; P=0.002), and elevated AFP ( >1,000 ng/mL; HR=0.393; P<0.001).
Conclusions: This large multi-center retrospective study showed that the objective response was 9.8%, and the proportion of long-term survivors was 15.3% in Korean patients. The prognostic factors predicting long-term survival after sorafenib treatment were hand foot skin reaction, metformin use, concomittent treatment, good liver function, absence of extrahepatic metastasis, low degree of portal vein thrombosis, and low AFP.