MATCHED targeted treatment significantly improved overall survival in patients with advanced cholangiocarcinoma, according to results from the large Italian ANITA dataset.
In this observational study, researchers evaluated the real-world impact of extended molecular profiling and matched targeted treatment in 621 patients with cholangiocarcinoma treated across 10 tertiary Italian cancer centres between 2017 and 2023. While molecular profiling became increasingly available during the study period, access to targeted treatment remained limited.
Cholangiocarcinoma Outcomes Improved with Targeted Treatment
Extended molecular profiling was performed in 79.9% of patients with advanced cholangiocarcinoma, with uptake increasing by 25.8% between 2017 and 2023. Among those tested, 139 patients were found to harbour ESCAT I to III actionable alterations, including FGFR2 fusions and IDH1 mutations. However, only 18.7% of these patients received matched targeted treatment.
Importantly, no significant differences in overall survival or progression-free survival were observed based solely on whether molecular profiling had been performed. In contrast, patients who received targeted treatment experienced significantly longer overall survival compared with those who did not receive matched therapy or those without molecular profiling available. The hazard ratio for death was 0.49 compared with untreated patients and 0.34 compared with patients without profiling.
Among molecular subgroups, FGFR2 fusions retained prognostic significance for overall survival when patients receiving targeted treatment were excluded, whereas IDH1 mutations did not independently confer a survival advantage in this analysis.
Cholangiocarcinoma, a form of biliary tract cancer arising from the bile ducts, is often diagnosed at an advanced stage and is associated with poor prognosis. According to GLOBOCAN data, biliary tract cancers account for a substantial proportion of hepatobiliary cancer mortality worldwide.
The authors concluded that although access to molecular profiling in cholangiocarcinoma had improved, timely access to targeted treatment remained suboptimal in routine practice. They emphasised that strategies to accelerate availability and uptake of new agents were warranted to translate molecular findings into meaningful survival gains.
Reference
International Agency for Research on Cancer. GLOBOCAN cancer statistics. 2026. Available at: https://gco.iarc.fr/. Last accessed: 03 February 2026.
