Survival outcomes with matched targeted therapy in advanced biliary tract cancer: a large global cohort analysis.
Publication Title
NPJ Precis Oncol
Document Type
Article
Publication Date
5-5-2026
Keywords
oregon; chiles
Abstract
The prognosis of advanced biliary tract cancer remains poor despite up to 40% of tumors harboring actionable molecular alterations. This international collaborative study from Mayo Clinic, Duke Cancer Institute, National Cancer Center East (Japan), and the SCRUM-Japan GOZILA/MONSTAR-SCREEN projects evaluated clinical outcomes in 1049 patients with advanced BTC based on tumor molecular profiling and matched treatment selection. Patients were categorized into non-actionable, actionable with matched targeted therapy (matched), and actionable without matched therapy (unmatched) groups. Among 358 patients (34.1%) with actionable alterations, 160 (44.7%) received matched therapy. The matched group demonstrated significantly longer median overall survival (23.3 months; 95% CI: 19.5-30.4) compared to unmatched (14.7 months; 95% CI: 12.8-17.9) and non-actionable groups (17.1 months; 95% CI: 15.5-18.7; HR 0.57, 95% CI: 0.46-0.71; p 0.0001). In 700 patients receiving ≥2 lines of therapy, matched therapy remained independently associated with improved survival (HR 0.62; 95% CI 0.43-0.90). Notably, patients with actionable tumors who did not receive matched therapy experienced worse outcomes than those with non-actionable disease. These findings highlight the critical need for universal molecular profiling and equitable access to targeted therapies in advanced BTC.
Area of Special Interest
Cancer
Area of Special Interest
Digestive Health
Specialty/Research Institute
Oncology
Specialty/Research Institute
Gastroenterology
DOI
10.1038/s41698-026-01457-3
