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<p>Biliary tract cancers (BTCs), including intrahepatic cholangiocarcinoma (iCCA), extrahepatic cholangiocarcinoma (eCCA), and gallbladder carcinoma (GBC), are rare but biologically aggressive malignancies with historically limited treatment options and dismal prognosis. The median overall survival in advanced stages remains below 13&#xa0;months despite chemotherapy and recent immunotherapy advances. However, molecular profiling has revealed targetable genetic alterations in up to 40% of BTCs, enabling the development and application of targeted therapies. This review provides an overview of the molecular landscape of BTC, describes the clinical relevance of key genomic alterations, discusses approved targeted agents, and highlights current implementation challenges and future directions in precision oncology.</p>

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Molecular profiling and targeted therapies in biliary tract cancer—a short review

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年7月23日

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  • Theresa Lentner,
  • Hossein Taghizadeh

摘要

Biliary tract cancers (BTCs), including intrahepatic cholangiocarcinoma (iCCA), extrahepatic cholangiocarcinoma (eCCA), and gallbladder carcinoma (GBC), are rare but biologically aggressive malignancies with historically limited treatment options and dismal prognosis. The median overall survival in advanced stages remains below 13 months despite chemotherapy and recent immunotherapy advances. However, molecular profiling has revealed targetable genetic alterations in up to 40% of BTCs, enabling the development and application of targeted therapies. This review provides an overview of the molecular landscape of BTC, describes the clinical relevance of key genomic alterations, discusses approved targeted agents, and highlights current implementation challenges and future directions in precision oncology.