Department of Medical Oncology and Hematology, Istituto Clinico Humanitas, Via Manzoni 56, 20089 Rozzano, Milan, Italy.
Dig Liver Dis. 2010 Jul;42 Suppl 3:S273-80. doi: 10.1016/S1590-8658(10)60516-6.
Hepatocellular carcinoma (HCC) is a highly lethal disease that is resistant to conventional cytotoxic drugs. Historically, effective systemic treatment options have not been available for patients with unresectable advanced disease. However, an improved understanding of the molecular mechanisms that regulate tumor initiation and progression over the past few years has led to the development of novel molecularly-targeted therapies that specifically block the different cellular signaling pathways involved. Recently, the antiangiogenesis and Raf kinase inhibitor, sorafenib showed a survival advantage in advanced stage HCC in two randomized, double-blind, controlled trials. These positive results were the first to demonstrate the efficacy of molecularly-targeted therapies in advanced HCC. Moreover, results from phase I and II trials evaluating other agents in this disease are promising and are under active clinical development. In the near future, we expect to have more data, knowledge, and evidence regarding the use of molecularly-targeted therapies in advanced HCC, both as single agents and in combination regimens. In this review, we will summarize the data concerning the present standard treatment for advanced HCC and discuss the newest, most promising clinical research that may affect the future treatment of this disease.
肝细胞癌（HCC）是一种高度致命的疾病，对传统细胞毒性药物有耐药性。从历史上看，对于不可切除的晚期疾病患者，尚无有效的系统治疗选择。然而，过去几年中对调节肿瘤起始和进展的分子机制的深入了解，导致了新型的分子靶向治疗的发展，这些治疗方法专门阻断了涉及的不同细胞信号通路。最近，抗血管生成和 Raf 激酶抑制剂索拉非尼在两项随机、双盲、对照试验中显示出晚期 HCC 的生存优势。这些积极的结果首次证明了分子靶向疗法在晚期 HCC 中的疗效。此外，在该疾病中评估其他药物的 I 期和 II 期试验结果令人鼓舞，并且正在积极进行临床开发。在不久的将来，我们预计将有更多关于晚期 HCC 中分子靶向治疗的使用的数据、知识和证据，无论是单药治疗还是联合治疗方案。在这篇综述中，我们将总结有关晚期 HCC 目前标准治疗的现有数据，并讨论可能影响这种疾病未来治疗的最新、最有前途的临床研究。
