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.

📅 Journal publication: Jul 6, 2026 · Last updated: Jul 16, 2026

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 · Free Full Text (PMC)

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Executive Summary

Study Design

Patient Population

Primary Outcomes

Secondary Outcomes & Safety Profile

Pharmacist Implications

Clinical Pearls

Limitations

Controversies & Evidence Gaps

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