A perspective on molecular therapy in cholangiocarcinoma: present status and future directions.
A perspective on molecular therapy in cholangiocarcinoma: present status and future directions.
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DOI：
10.2217/hep.13.4
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发表时间：
2014-01-01
期刊：
影响因子：
5
通讯作者：
Thorgeirsson SS
中科院分区：
文献类型：
--
作者：
Andersen JB;Thorgeirsson SS
胆管癌（CCA）是一种孤儿癌，其遗传和基因组发病机制的了解有限。通常，它是高度治疗难治性的，患者结局令人沮丧。目前，没有批准的CCA治疗方法，手术切除仍然是唯一的治疗选择。目前正在对胆道癌的混合队列进行临床试验，包括肝内CCA、肝外/肝门周围CCA、远端肝外CCA、胆囊癌，在极少数情况下甚至包括胰腺癌。今天，临床试验失败主要是因为它们是动力不足的混合队列，并且设计时没有意图富集标记物以优化靶向治疗的成功。本文综述了CCA靶向治疗的临床研究现状，并重点介绍了翻译基因组学研究发现的有前景的新的候选途径。
Cholangiocarcinoma (CCA) is an orphan cancer with limited understanding of its genetic and genomic pathogenesis. Typically, it is highly treatment-refractory and patient outcome is dismal. Currently, there are no approved therapeutics for CCA and surgical resection remains the only option with curative intent. Clinical trials are currently being performed in a mixed cohort of biliary tract cancers that includes intrahepatic CCA, extrahepatic/perihilar CCA, distal extrahepatic CCA, gallbladder carcinoma and, in rare cases, even pancreatic cancers. Today, clinical trials fail primarily because they are underpowered mixed cohorts and designed without intent to enrich for markers to optimize success for targeted therapy. This review aims to emphasize current clinical attempts for targeted therapy of CCA, as well as highlight promising new candidate pathways revealed by translational genomics.