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Survival outcomes with matched targeted therapy in advanced biliary tract cancer: a large global cohort analysis

Binbin Zheng-Lin1, Taro Shibuki2,3, Heidi E Kosiorek4

  • 1Earle A. Chiles Research Institute, Providence Cancer Institute, Portland, OR, USA.

NPJ Precision Oncology
|May 5, 2026
PubMed

Insights

Targeted therapy matching actionable alterations in advanced biliary tract cancer significantly improves patient survival. Universal molecular profiling and equitable access to these treatments are crucial for better outcomes.

Area of Science:

  • Oncology
  • Genomics
  • Translational Research

Background:

  • Advanced biliary tract cancer (BTC) has a poor prognosis.
  • A significant proportion of BTC tumors harbor actionable molecular alterations.
  • Current treatment strategies often do not fully leverage molecular profiling data.

Purpose of the Study:

  • To evaluate the clinical outcomes of advanced BTC patients based on tumor molecular profiling.
  • To compare survival rates between patients receiving matched targeted therapy, unmatched targeted therapy, and those with non-actionable alterations.
  • To determine the impact of matched therapy on overall survival in advanced BTC.

Main Methods:

  • International collaborative study involving 1049 patients with advanced BTC.
  • Tumor molecular profiling was performed to identify actionable alterations.
  • Patients were categorized into non-actionable, matched therapy, and unmatched therapy groups.
  • Clinical outcomes, including overall survival, were analyzed.

Main Results:

  • 34.1% of patients had actionable alterations, with 44.7% receiving matched therapy.
  • The matched therapy group showed significantly longer median overall survival (23.3 months) compared to unmatched (14.7 months) and non-actionable groups (17.1 months).
  • Matched therapy was independently associated with improved survival in patients receiving ≥2 lines of therapy.
  • Patients with actionable tumors not receiving matched therapy had worse outcomes than those with non-actionable disease.

Conclusions:

  • Matching targeted therapies to actionable molecular alterations significantly improves survival in advanced BTC.
  • Universal molecular profiling is essential for identifying eligible patients.
  • Equitable access to targeted therapies is critical for enhancing patient outcomes in advanced BTC.

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