Hepatobiliary Surgery and Nutrition · 3 authors, 7 centres
This summary was generated by AI from a single paper. It has not been reviewed by a clinician and is not clinical advice. Verify against the source before acting on it.
This narrative review evaluates the current state and future directions for biomarker-driven precision medicine in cholangiocarcinoma, highlighting advances in targeted therapies and immunotherapy while noting challenges in trial design, biomarker validation, and patient access.
This narrative review discusses the evolving landscape of precision medicine for cholangiocarcinoma (CCA), focusing on biomarkers, targeted therapies, and immunotherapy. It notes that conventional chemotherapy offers limited survival benefit, with 5-year survival of 5–10%. The review summarizes progress in identifying molecular targets like FGFR2 alterations (revised to ~9% prevalence in iCCA) and IDH1 mutations, and reports on clinical trial results, including the phase III TOPAZ-1 and KEYNOTE-966 trials establishing frontline chemo-immunotherapy. It highlights the development of new agents like third-generation FGFR inhibitors with response rates of 40% and 80% and discusses challenges such as low prevalence of some alterations, difficulty recruiting for trials, and limited access to targeted treatments (e.g., <10% of patients with targetable alterations received matched therapy). Limitations include the narrative nature of the review and the reliance on data from mostly non-randomized studies for many targeted therapies.
