Immune Checkpoint Inhibitors Redefine First-Line Cholangiocarcinoma Therapy

This systematic review consolidates efficacy, safety, and emerging biomarker data across immune checkpoint inhibitor regimens in advanced cholangiocarcinoma, informing regimen selection and toxicity anticipation for oncology pharmacists.

durvalumabpembrolizumabnivolumabtoripalimabatezolizumabtislelizumablenvatinibimmune checkpoint inhibitorsPD-1PD-L1CTLA-4cholangiocarcinomabiliary tract cancergemcitabine-cisplatinsystematic reviewbiomarkersHRDctDNA Oncology Pharmacy

Background Information

Immune checkpoint inhibitors redefine advanced cholangiocarcinoma treatment.

Cholangiocarcinoma (CCA) has limited treatment options and poor prognosis; new therapies are urgently needed. Immune checkpoint inhibitors (ICIs) plus chemotherapy improved overall survival in advanced CCA, with KEYNOTE-966 and TOPAZ-1 trials showing significant survival benefits. First-line chemo-immunotherapy is now recommended for advanced CCA, with promising results. Biomarker-driven approaches could further optimize treatment efficacy, although further validation is needed. ICIs combined with chemotherapy establish a new first-line standard for advanced cholangiocarcinoma.

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Authors: Nisa FU, Ali M, Khan T, Lohana M, Asif A, Kumari N, Ali M

Citation: Nisa FU, Ali M, Khan T, et al. Immune checkpoint inhibitors in advanced cholangiocarcinoma: a systematic review of efficacy, safety, and emerging biomarkers. Journal of the Egyptian National Cancer Institute. 2026;38(1). doi:10.1186/s43046-026-00381-8

Article Links: PubMed · Journal / DOI

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Executive Summary

Study Design

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Secondary Outcomes & Safety Profile

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