Centre de Recherche des Cordeliers, Functional Genomics of Solid Tumors Laboratory, Sorbonne Université, Inserm, USPC, Université Paris Descartes, Université Paris Diderot, Université Paris 13, Paris, France.
Liver Unit, Hôpital Jean Verdier, Hôpitaux Universitaires Paris-Seine-Saint-Denis, Assistance-Publique Hôpitaux de Paris, Bondy, France.
Hepatology. 2021 Jan;73 Suppl 1:115-127. doi: 10.1002/hep.31175. Epub 2020 Nov 29.
The clinical management of primary liver cancers such as hepatocellular carcinoma (HCC) and cholangiocarcinoma (CCA) has significantly changed in the last 3 years. The introduction of systemic therapies, including immune-based therapies and biomarker-driven therapies, has significantly improved survival, particularly in patients at advanced stages of disease. Survival is still poor, and projections from the World Health Organization underscore the need to improve outcomes in these patients. Biomarkers have emerged as powerful tools for the diagnosis, prognosis, and prediction of treatment responses to improve patient stratification and maximize clinical benefits. Recent advances in understanding the molecular alterations of HCC have not yet translated into biomarkers. Some reasons for the lack of progress are the absence of druggable mutations in the majority of liver cancers and the significant heterogeneity of the disease. In contrast, several therapeutic targets have been identified in CCA, and biomarker-driven therapies are currently under evaluation in phase 2/3 clinical trials. Here, we summarize the status on biomarker development for HCC and CCA.
在过去的 3 年中，原发性肝癌（如肝细胞癌[HCC]和胆管癌[CCA]）的临床管理发生了重大变化。系统治疗方法的引入，包括免疫治疗和基于生物标志物的治疗方法，显著提高了生存率，特别是在疾病晚期的患者中。但生存情况仍然不佳，世界卫生组织的预测强调需要改善这些患者的预后。生物标志物已成为用于诊断、预后和预测治疗反应的有力工具，以改善患者分层并最大程度地提高临床获益。最近对 HCC 分子改变的理解方面并没有转化为生物标志物。缺乏进展的一些原因是大多数肝癌中缺乏可用药突变，以及疾病的显著异质性。相比之下，CCA 中已经确定了几个治疗靶点，并且基于生物标志物的治疗方法目前正在进行 2/3 期临床试验评估。在这里，我们总结了 HCC 和 CCA 的生物标志物开发现状。
