Related Experiment Video
Updated: Jun 23, 2026

An Oncogenic Hepatocyte-Induced Orthotopic Mouse Model of Hepatocellular Cancer Arising in the Setting of Hepatic Inflammation and Fibrosis
Published on: September 12, 2019
Selection of patients with hepatocellular carcinoma for sorafenib
1Memorial Sloan-Kettering Cancer Center, New York, New York 10022, USA. abou-alg@mskcc.org
Abstract:
Sorafenib, a multitargeted anti-VEGF receptor and raf kinase inhibitor, was recently approved by the FDA for treating unresectable hepatocellular carcinoma (HCC) based on 2 randomized phase III studies. In addition, a phase II study evaluating sorafenib in patients with HCC and Child-Pugh A and B and a phase I study evaluating sorafenib in patients with organ dysfunction have provided insight about the safety and efficacy of sorafenib in patients with HCC and more advanced cirrhosis, and any difference in outcome based on etiology of HCC. The lack of objective responses observed in the sorafenib arm in the SHARP study also raises practical issues about how to assess response or efficacy of the therapy and thus how long a patient should receive sorafenib. This article addresses these questions on the use of sorafenib in HCC, both in the locally advanced and metastatic settings, in addition to the potential future applications and uses of sorafenib.
Insights
Sorafenib is an FDA-approved treatment for unresectable hepatocellular carcinoma (HCC). This review discusses its efficacy, safety, and practical considerations for HCC patients with advanced cirrhosis.
Area of Science:
- Oncology
- Hepatology
- Pharmacology
Background:
- Sorafenib, a multi-targeted kinase inhibitor, is FDA-approved for unresectable hepatocellular carcinoma (HCC).
- Previous studies evaluated sorafenib in patients with advanced cirrhosis and organ dysfunction.
- Efficacy and safety data in diverse HCC patient populations are crucial.
Purpose of the Study:
- To review the safety and efficacy of sorafenib in unresectable HCC.
- To address practical issues regarding sorafenib use in HCC patients, including response assessment.
- To explore potential future applications of sorafenib in HCC treatment.
Main Methods:
- Review of randomized phase III, phase II, and phase I studies of sorafenib in HCC.
- Analysis of safety and efficacy data in patients with varying stages of cirrhosis and organ dysfunction.
- Discussion of response evaluation criteria and treatment duration.
Main Results:
- Sorafenib demonstrated efficacy in unresectable HCC, with safety data available for advanced cirrhosis.
- Variations in outcomes based on HCC etiology were investigated.
- Practical challenges in assessing sorafenib efficacy, such as lack of objective responses, were highlighted.
Conclusions:
- Sorafenib is a key therapeutic option for unresectable HCC, including in patients with advanced cirrhosis.
- Careful consideration of response assessment and treatment duration is necessary for optimal sorafenib use.
- Further research may expand sorafenib's role in HCC management.
