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World J Gastroenterol. Mar 28, 2014; 20(12): 3135-3145
Published online Mar 28, 2014. doi: 10.3748/wjg.v20.i12.3135
Development of systemic therapy for hepatocellular carcinoma at 2013: Updates and insights
Stephen L Chan, Winnie Yeo
Stephen L Chan, Winnie Yeo, Department of Clinical Oncology, The Chinese University of Hong Kong, Prince of Wales Hospital, Hong Kong, China
Stephen L Chan, Winnie Yeo, State Key Laboratory in Oncology in South China, Hong Kong, China
Author contributions: Chan SL and Yeo W wrote the paper.
Correspondence to: Winnie Yeo, MD, FRCP, Department of Clinical Oncology, The Chinese University of Hong Kong, Prince of Wales Hospital, Shatin, Hong Kong, China. winnieyeo@cuhk.edu.hk
Telephone: +86-852-26322118 Fax: +86-852-26487097
Received: October 24, 2013
Revised: December 23, 2013
Accepted: January 19, 2014
Published online: March 28, 2014
Processing time: 154 Days and 0.1 Hours
Abstract

A growing number of multi-targeted tyrosine kinase inhibitor (TKI) has undergone testing for hepatocellular carcinoma (HCC). Unfortunately, this enthusiasm has recently been discouraged by a number of negative phase III studies on several anti-angiogenic TKIs in HCC. Several postulations have been made to account for this phenomenon, namely the plateau effects of anti-angiogenesis approach, the heterogeneity of HCC in terms of background hepatitis/cirrhosis and tumor biology, as well as the way how clinical trials are designed. Regardless of the underlying reasons, these results suggested that alternative strategies are necessary to further develop systemic therapy for HCC. Several new strategies are currently evaluated: for examples, molecular agents with activities against targets other than vascular endothelial growth factor receptor are being evaluated in on-going clinical trials. In addition, different approaches of targeted agents in combination with various treatment modalities, such as concurrently with another molecular agent, cytotoxic chemotherapy or transarterial chemoembolization, are being developed. This review aims to give a summary on the results of recently released clinical trials on TKIs, followed by discussion on some of the potential novel agents and combinational approaches. Future directions for testing innovative systemic agents for HCC will also be discussed.

Keywords: Liver neoplasms; Systemic treatment; Biologics; Staging; Clinical trial

Core tip: This review article aims to provide an update and vision on the development of novel targeted agents for liver cancers. Recently released phase III clinical trial results as well as important future focus will be discussed.