Department of Medicine, School of Clinical Medicine, The University of Hong Kong, Hong Kong, Hong Kong.
State Key Laboratory of Liver Research, The University of Hong Kong, Hong Kong, Hong Kong.
F1000Res. 2024 May 7;13:104. doi: 10.12688/f1000research.145493.2. eCollection 2024.
Advanced hepatocellular carcinoma (HCC) is traditionally associated with limited treatment options and a poor prognosis. Sorafenib, a multiple tyrosine kinase inhibitor, was introduced in 2007 as a first-in-class systemic agent for advanced HCC. After sorafenib, a range of targeted therapies and immunotherapies have demonstrated survival benefits in the past 5 years, revolutionizing the treatment landscape of advanced HCC. More recently, evidence of novel combinations of systemic agents with distinct mechanisms has emerged. In particular, combination trials on atezolizumab plus bevacizumab and durvalumab plus tremelimumab have shown encouraging efficacy. Hence, international societies have revamped their guidelines to incorporate new recommendations for these novel systemic agents. Aside from treatment in advanced HCC, the indications for systemic therapy are expanding. For example, the combination of systemic therapeutics with locoregional therapy (trans-arterial chemoembolization or stereotactic body radiation therapy) has demonstrated promising early results in downstaging HCC. Recent trials have also explored the role of systemic therapy as neoadjuvant treatment for borderline-resectable HCC or as adjuvant treatment to reduce recurrence risk after curative resection. Despite encouraging results from clinical trials, the real-world efficacy of systemic agents in specific patient subgroups (such as patients with advanced cirrhosis, high bleeding risk, renal impairment, or cardiometabolic diseases) remains uncertain. The effect of liver disease etiology on systemic treatment efficacy warrants further research. With an increased understanding of the pathophysiological pathways and accumulation of clinical data, personalized treatment decisions will be possible, and the field of systemic treatment for HCC will continue to evolve.
晚期肝细胞癌(HCC)传统上与有限的治疗选择和预后不良相关。索拉非尼是一种多靶点酪氨酸激酶抑制剂,于 2007 年作为晚期 HCC 的首个系统治疗药物问世。索拉非尼之后,在过去的 5 年中,一系列靶向治疗和免疫疗法已显示出生存获益,彻底改变了晚期 HCC 的治疗格局。最近,出现了具有不同机制的新型系统药物联合治疗的证据。特别是,阿替利珠单抗联合贝伐珠单抗和度伐利尤单抗联合替西木单抗的联合试验显示出令人鼓舞的疗效。因此,国际社会已修订其指南,纳入了这些新型系统药物的新建议。除了晚期 HCC 的治疗外,系统治疗的适应证正在扩大。例如,系统治疗与局部区域治疗(经动脉化疗栓塞或立体定向体部放射治疗)联合在 HCC 降期方面显示出有前途的早期结果。最近的试验还探讨了系统治疗作为边界可切除 HCC 的新辅助治疗或作为根治性切除后降低复发风险的辅助治疗的作用。尽管临床试验取得了令人鼓舞的结果,但系统药物在特定患者亚组(如晚期肝硬化、高出血风险、肾功能损害或心脏代谢疾病患者)中的实际疗效仍不确定。肝脏疾病病因对系统治疗疗效的影响需要进一步研究。随着对病理生理途径的理解加深和临床数据的积累,将有可能做出个性化的治疗决策,HCC 的系统治疗领域将继续发展。