Department of Gastroenterology, Transplantation Unit, Universidade de São Paulo, São Paulo 05403-000, Brazil.
Int J Mol Sci. 2023 Dec 29;25(1):461. doi: 10.3390/ijms25010461.
Intrahepatic cholangiocarcinoma (ICC) is a relatively uncommon but highly aggressive primary liver cancer that originates within the liver. The aim of this study is to review the molecular profile of intrahepatic cholangiocarcinoma and its implications for prognostication and decision-making. This comprehensive characterization of ICC tumors sheds light on the disease's underlying biology and offers a foundation for more personalized treatment strategies. This is a narrative review of the prognostic and therapeutic role of the molecular profile of ICC. Knowing the molecular profile of tumors helps determine prognosis and support certain target therapies. The molecular panel in ICC helps to select patients for specific therapies, predict treatment responses, and monitor treatment responses. Precision medicine in ICC can promote improvement in prognosis and reduce unnecessary toxicity and might have a significant role in the management of ICC in the following years. The main mutations in ICC are in (), (), (), and (). The rate of mutations varies significantly for each population. Targeting and is challenging due to the natural characteristics of these genes. Different stages of clinical studies have shown encouraging results with inhibitors of mutated and target therapy for downstream effectors. () fusions are an important target in patients with ICC. Immune checkpoint blockade can be applied to a small percentage of ICC patients. Molecular profiling in ICC represents a groundbreaking approach to understanding and managing this complex liver cancer. As our comprehension of ICC's molecular intricacies continues to expand, so does the potential for offering patients more precise and effective treatments. The integration of molecular profiling into clinical practice signifies the dawn of a new era in ICC care, emphasizing personalized medicine in the ongoing battle against this malignancy.
肝内胆管癌(ICC)是一种相对罕见但高度侵袭性的原发性肝癌,起源于肝脏。本研究旨在综述肝内胆管癌的分子特征及其对预后和决策的影响。这种对 ICC 肿瘤的全面特征描述揭示了该疾病的潜在生物学,并为更个性化的治疗策略提供了基础。这是一篇关于 ICC 分子特征的预后和治疗作用的叙述性综述。了解肿瘤的分子特征有助于确定预后并支持某些靶向治疗。ICC 的分子谱有助于为特定疗法选择患者,预测治疗反应,并监测治疗反应。ICC 的精准医学可以促进预后的改善,减少不必要的毒性,并可能在未来几年ICC 的管理中发挥重要作用。ICC 的主要突变发生在 ()、()、() 和 ()。每个群体的突变率差异很大。由于这些基因的自然特性,靶向 () 和 () 具有挑战性。不同阶段的临床研究表明,突变 () 抑制剂和针对 () 下游效应器的靶向治疗具有令人鼓舞的结果。ICC 患者中的 () 融合是一个重要的靶点。免疫检查点阻断可应用于一小部分 ICC 患者。ICC 的分子谱分析代表了理解和管理这种复杂肝癌的突破性方法。随着我们对 ICC 分子复杂性的理解不断深入,为患者提供更精确和有效的治疗的潜力也在不断增加。分子谱分析纳入临床实践标志着 ICC 治疗新时代的开始,强调了个性化医学在对抗这种恶性肿瘤的持续斗争中的重要性。