Cholangiocarcinoma, a cancer arising from bile duct epithelia, is an increasingly prevalent malignancy worldwide. To address longstanding clinical challenges, an international expert panel has established recommendations and strategic priorities covering disease classification, biomarker implementation and therapeutic interventions.
Standardizing Classification and Treatment Pathways
Precise anatomical classification under ICD-11 is a fundamental step in distinguishing intrahepatic, perihilar and distal cholangiocarcinoma. Accurate classification helps establish the basis for appropriate treatment decisions.
Surgical resection remains the only curative-intent treatment option, but it is feasible in only 20% to 30% of newly diagnosed cases. Given the high risk of recurrence, six months of adjuvant capecitabine is recommended following surgery to reduce that risk.
For patients with unresectable lesions, identifying candidates for liver transplantation or locoregional therapies at an early stage offers important potential survival advantages. The recommendations also strongly advocate multidisciplinary tumor board discussions for every patient to help navigate complex cholangiocarcinoma management pathways.
Genomic Profiling Expands Targeted Treatment Options
Comprehensive molecular profiling through next-generation sequencing is now considered standard clinical practice for all advanced cases, helping guide individualized targeted treatment.
Several actionable oncogenic drivers offer established targets for approved systemic agents, including IDH1 mutations, FGFR2 fusions, HER2 amplifications and BRAF mutations. Identifying these alterations can help determine appropriate treatment options for patients with advanced disease.
For first-line palliative treatment, combining cisplatin and gemcitabine with immune-checkpoint inhibitors, including durvalumab or pembrolizumab, significantly improves survival compared with chemotherapy alone.
When actionable molecular alterations are absent, FOLFOX remains the preferred evidence-based second-line treatment following disease progression. These recommendations emphasize the role of molecular testing and treatment selection in managing advanced cholangiocarcinoma.
Research Priorities Target Unmet Clinical Needs
Despite advances in treatment, significant clinical gaps remain. The expert panel has highlighted the need for rapid validation of novel liquid biopsy platforms, increased clinical trial participation and dedicated research into the complex tumor immune microenvironment.
Further progress in cholangiocarcinoma management will also depend on addressing acquired drug resistance through novel treatment combinations and implementing proactive supportive care protocols. These priorities aim to address persistent barriers to effective treatment and support continued innovation in biliary oncology.
The consensus statement also recognizes the clinical legacy of coauthor Dr. Rachna Shroff, whose pioneering leadership transformed modern biliary cancer clinical trials.
Her vision continues through the Conquer Cancer Rachna Shroff, MD, MS, FASCO Endowed Young Investigator Award, which funds promising early-career researchers dedicated to conquering biliary tract cancers.
The recommendations, published in Nature Reviews Gastroenterology & Hepatology, outline priorities across classification, molecular testing, treatment and research, providing a framework for addressing continuing challenges in cholangiocarcinoma care.
Reference: Banales JM et al. “Cholangiocarcinoma 2026: status quo, unmet needs and priorities.” Nature Reviews Gastroenterology & Hepatology. 2026;23(1):65-96
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