Genomic profiling unlocks new treatment opportunities for ampullary carcinoma.

ABSTRACT:

Background

Ampullary carcinoma (AC) is a rare disease with an abysmal prognosis and few treatment options. The molecular landscape and its therapeutic implications remain inadequately understood. This study aims to provide a clinical and genomic characterization of AC and explore opportunities for precision oncology.

Materials and methods

We carried out a retrospective analysis of clinical and genomic features in patients with AC treated in our institution. Gene mutations were categorized into molecular pathways, and potentially targetable alterations were classified according to the European Society for Medical Oncology (ESMO) Scale for Clinical Actionability of Molecular Targets (ESCAT). Key molecular findings were validated in an external cohort.

Results

We include

SUBMITTER: Fabregat-Franco C 

PROVIDER: S-EPMC12141931 | biostudies-literature | 2025 May

REPOSITORIES: biostudies-literature