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Cholangiocarcinoma in the Era of Precision Medicine: Emerging Insights and Therapeutic Strategies

Research output: Contribution to journalReview articlepeer-review

Abstract

Introduction/Objective: Cholangiocarcinoma is an aggressive malignancy of the biliary tract, with increasing global incidence and poor prognosis due to frequent late-stage diagnosis and nonspecific symptoms. It encompasses intrahepatic, perihilar, and distal subtypes, each with distinct clinical characteristics. This review outlines current and emerging therapeutic strategies for the treatment of resectable and advanced cholangiocarcinoma, with a focus on adjuvant therapies, systemic chemotherapy, immunotherapy, and targeted molecular approaches. Methods: A comprehensive literature search was conducted across major medical databases. Relevant randomized controlled trials, meta-analyses, clinical guidelines, and high-quality observational studies were reviewed. Emphasis was placed on recent evidence and emerging treatment modalities with clinical significance. Results: Capecitabine remains the standard adjuvant chemotherapy regimen following resection, although optimal regimens are currently under investigation. Gemcitabine-cisplatin is the first-line chemotherapy regimen for advanced disease. Immunotherapy has shown benefit, especially in patients with microsatellite instability. Promising advances lie in targeted therapies, including fibroblast growth factor receptor inhibitors and isocitrate dehydrogenase 1 inhibitors, which have demonstrated improved outcomes in molecularly selected patients. Discussion: The treatment landscape of cholangiocarcinoma continues to evolve. While chemotherapy remains foundational, increasing molecular characterization may offer improved disease control in selected subgroups. Conclusion: Advances in molecular profiling and immunotherapy are reshaping the management of cholangiocarcinoma, enabling more personalized treatment strategies. Continued integration of precision oncology into clinical practice and prospective trials will be critical to improving outcomes for cholangiocarcinoma moving forward.

Original languageEnglish (US)
JournalCurrent pharmaceutical design
DOIs
StateAccepted/In press - 2026

Keywords

  • Cholangiocarcinoma
  • FGFR3 mutations
  • IDH-1 mutations
  • adjuvant treatment in cholangiocarcinoma
  • immunotherapy
  • metstatic treatment in cholangiocarcinoma

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery

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