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Updated: Apr 13, 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
Systemic therapy of hepatocellular carcinoma: current and promising
Aparna Kalyan1, Halla Nimeiri1, Laura Kulik2
1Northwestern Medicine Developmental Therapeutics Institute (NMDTI), Chicago, IL, USA; Division of Hematology and Oncology, Robert H. Lurie Medical Research Center, Room 5-121, 303 East Superior, Chicago, IL 60611, USA.
Abstract:
Treatment of advanced hepatocellular carcinoma (HCC) remains challenging, particularly with the limited systemic therapy options. Sorafenib remains the only approved, targeted molecule for the treatment of advanced HCC. Although a survival benefit was demonstrated with sorafenib, it remains only true in the population of patients with Child-Turcotte-Pugh class A disease. Sorafenib also has distinct side effects that require close monitoring. Newer tyrosine kinase inhibitors and angiogenic inhibitors have been evaluated with disappointing results, particularly in phase III trials. Herein we review the pertinent trials for targeted therapy in HCC to date.
Insights
Advanced hepatocellular carcinoma (HCC) treatment is difficult, with limited options. Sorafenib is the only approved targeted therapy, but its benefits are mainly for Child-Turcotte-Pugh class A patients and it has side effects.
Area of Science:
- Hepatobiliary cancers
- Medical oncology
- Translational research
Background:
- Advanced hepatocellular carcinoma (HCC) presents significant treatment challenges.
- Systemic therapy options for HCC are currently limited.
- Sorafenib is the sole approved targeted therapy for advanced HCC.
Purpose of the Study:
- To review pivotal clinical trials of targeted therapies for HCC.
- To evaluate the efficacy and limitations of current advanced HCC treatments.
- To provide an overview of targeted therapy research in HCC.
Main Methods:
- Systematic review of published clinical trials.
- Analysis of data from phase III trials of targeted agents.
- Evaluation of sorafenib's efficacy and side effect profile.
Main Results:
- Sorafenib demonstrated a survival benefit, primarily in Child-Turcotte-Pugh class A patients.
- Newer tyrosine kinase inhibitors and angiogenic inhibitors have yielded disappointing results in phase III trials.
- Sorafenib's side effects necessitate careful patient monitoring.
Conclusions:
- Despite sorafenib's approval, effective systemic treatments for advanced HCC remain limited.
- Further research into novel targeted therapies is crucial for improving HCC patient outcomes.
- Understanding patient stratification is key for optimizing treatment response in HCC.
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