We conducted a comprehensive review of the current literature of published data, clinical trials (MEDLINE; ncbi.pubmed.com), congress contributions (asco.org; esmo.org), and active recruiting clinical trains (clinicaltrial.gov) on targeted therapies in cholangiocarcinoma. Palliative treatment regimens were analyzed as well as preoperative and perioperative treatment options. We summarized the current knowledge for each mutation and molecular pathway that is or has been under clinical evaluation and discussed the results on the background of current treatment guidelines. We established and recommended targeted treatment options that already exist for second-line settings, including IDH-, BRAF-, and NTRK-mutated tumors, as well as for FGFR2 fusion, HER2/neu-overexpression, and microsatellite instable tumors. Other options for targeted treatment include EGFR- or VEGF-dependent pathways, which are known to be overexpressed or dysregulated in this cancer type and are currently under clinical investigation. Targeted therapy in CCA is a hallmark of individualized medicine as these therapies aim to specifically block pathways that promote cancer cell growth and survival, leading to tumor shrinkage and improved patient outcomes based on the molecular profile of the tumor.
我们对胆管癌靶向治疗的现有文献、已发表数据、临床试验（MEDLINE；ncbi.pubmed.com）、学术会议报告（asco.org；esmo.org）以及正在招募的临床试验（clinicaltrial.gov）进行了全面综述。分析范围涵盖姑息治疗方案、术前及围手术期治疗策略。本文系统总结了目前处于或曾接受临床评估的各类基因突变及分子通路的研究现状，并结合现行治疗指南背景对相关结果展开讨论。我们确立并推荐了已应用于二线治疗的靶向治疗方案，包括针对IDH、BRAF和NTRK突变肿瘤，以及FGFR2融合、HER2/neu过表达和微卫星不稳定肿瘤的治疗方案。其他靶向治疗选择涉及EGFR或VEGF依赖通路——已知这些通路在该癌症类型中存在过表达或失调现象，目前正处于临床研究阶段。胆管癌的靶向治疗是个体化医疗的重要标志，这些疗法旨在特异性阻断促进癌细胞生长和存活的信号通路，根据肿瘤的分子特征实现肿瘤缩小并改善患者预后。
Current and Future Therapeutic Targets for Directed Molecular Therapies in Cholangiocarcinoma
肿瘤(癌症)患者之家