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<p>Cholangiocarcinoma (CCA) comprises a&#xa0;heterogeneous group of malignancies of the intra- and extrahepatic bile ducts. Despite low incidence rates, CCA is responsible for a&#xa0;relevant proportion of deaths from liver neoplasms. As most patients present with advanced disease without the option of curative resection, systemic therapy is the cornerstone of treatment. Based on the ABC-02 trial, gemcitabine plus cisplatin has long been the standard first-line regimen and has recently been successfully combined with immune checkpoint inhibitors such as durvalumab and pembrolizumab; randomized phase&#xa0;III trials (TOPAZ‑1; KEYNOTE-966) have shown a&#xa0;significant survival advantage. In the second-line setting, chemotherapy with mFOLFOX or irinotecan-based regimens confers only modest benefit in unselected populations (ABC-06, NIFTY). Parallel to these developments, comprehensive molecular profiling has revealed a&#xa0;broad spectrum of targetable genomic alterations, particularly IDH1 mutations and FGFR2 fusions in intrahepatic CCA and HER2 amplification in extrahepatic tumors and gallbladder cancer. Targeted agents, including ivosidenib for IDH1 mutations (ClarIDHy), pemigatinib and futibatinib for FGFR2 fusions (FIGHT-202, FOENIX-CCA2), and the bispecific HER2 antibody zanidatamab for HER2-amplified advanced CCA (HERIZON-BTC-01), have demonstrated clinically meaningful activity in pivotal trials and are increasingly integrated into routine care. This review summarizes the current evidence on systemic treatment of CCA, with a&#xa0;focus on the evolving role of precision oncology.</p>

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Systemtherapie beim Cholangiokarzinom

  • 期刊论文

  • 发表日期：

    2026年2月25日

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  • Markus Sebastian Jördens,
  • Tom Luedde,
  • Christoph Roderburg

摘要

Cholangiocarcinoma (CCA) comprises a heterogeneous group of malignancies of the intra- and extrahepatic bile ducts. Despite low incidence rates, CCA is responsible for a relevant proportion of deaths from liver neoplasms. As most patients present with advanced disease without the option of curative resection, systemic therapy is the cornerstone of treatment. Based on the ABC-02 trial, gemcitabine plus cisplatin has long been the standard first-line regimen and has recently been successfully combined with immune checkpoint inhibitors such as durvalumab and pembrolizumab; randomized phase III trials (TOPAZ‑1; KEYNOTE-966) have shown a significant survival advantage. In the second-line setting, chemotherapy with mFOLFOX or irinotecan-based regimens confers only modest benefit in unselected populations (ABC-06, NIFTY). Parallel to these developments, comprehensive molecular profiling has revealed a broad spectrum of targetable genomic alterations, particularly IDH1 mutations and FGFR2 fusions in intrahepatic CCA and HER2 amplification in extrahepatic tumors and gallbladder cancer. Targeted agents, including ivosidenib for IDH1 mutations (ClarIDHy), pemigatinib and futibatinib for FGFR2 fusions (FIGHT-202, FOENIX-CCA2), and the bispecific HER2 antibody zanidatamab for HER2-amplified advanced CCA (HERIZON-BTC-01), have demonstrated clinically meaningful activity in pivotal trials and are increasingly integrated into routine care. This review summarizes the current evidence on systemic treatment of CCA, with a focus on the evolving role of precision oncology.