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<p>Intrahepatic cholangiocarcinoma (ICC) is the second most prevalent liver cancer after hepatocellular carcinoma and is characterized by high malignancy and poor prognosis. Gemcitabine combined with cisplatin is the standard first-line therapy for metastatic or unresectable ICC. The combination of immunotherapy and targeted therapy represents a promising new direction for ICC treatment. Common genetic mutations in ICC include those in TP53, FGFR2, IDH1/2, and KRAS. MET alterations such as fusions and amplifications are rare in ICC. However, limited research has been conducted on the efficacy of specific MET inhibitors. We present two cases: the first with refractory ICC treated with a combination of immunotherapy and targeted therapy, harboring a ZKSCAN1-MET fusion and the second with a metastatic ICC with MET amplification. Both patients demonstrated a significant clinical response to crizotinib, a MET-specific tyrosine kinase inhibitor.</p>

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Response to crizotinib in advanced intrahepatic cholangiocarcinoma with ZKSCAN1-MET fusion and MET amplification: case reports and literature review

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年6月14日

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  • Jian-Hui Wu,
  • Yu-ze Fan,
  • Jing Sun,
  • Xue-Zhang Duan

摘要

Intrahepatic cholangiocarcinoma (ICC) is the second most prevalent liver cancer after hepatocellular carcinoma and is characterized by high malignancy and poor prognosis. Gemcitabine combined with cisplatin is the standard first-line therapy for metastatic or unresectable ICC. The combination of immunotherapy and targeted therapy represents a promising new direction for ICC treatment. Common genetic mutations in ICC include those in TP53, FGFR2, IDH1/2, and KRAS. MET alterations such as fusions and amplifications are rare in ICC. However, limited research has been conducted on the efficacy of specific MET inhibitors. We present two cases: the first with refractory ICC treated with a combination of immunotherapy and targeted therapy, harboring a ZKSCAN1-MET fusion and the second with a metastatic ICC with MET amplification. Both patients demonstrated a significant clinical response to crizotinib, a MET-specific tyrosine kinase inhibitor.