Targeted treatment of billary tract tumors

Background: Biliary tract tumors are rare malignant neoplasms with 8000 new cases per year in Germany. The prognosis for patients with biliary tumors is still very poor. However, after many years of stagnation, the number of options regarding systemic therapy has been increasing in recent years: on the one hand, immuno-oncology has become part of first-line standard of care, and on the other hand, studies in genetically selected subgroups have shown the potential of targeted strategies.Objective: The aim of this paper is to provide an overview of targeted systemic therapy in biliary tumors.Materials and methods: This work is based on a selective literature search of the PubMed database on genetic alterations and targeted therapies in biliary tumors.Results: Genetic alterations can be found in up to 40% of cases of patients with biliary tumors, for which molecular therapies are already available today. However, many studies in the small genetic subgroups of a rare tumor entity have been performed as single-arm phase II studies without control arms.Conclusion: Comprehensive genetic analysis of biliary tumors should be performed at an early stage. For many genetic alterations, there is no approval for targeted therapy today, so these patients should be presented in a molecular tumor board. Moreover, the respective genetic subgroups are very small, so that it is paramount that suitable patients are consistently included in therapy studies and molecular registries.