The Cholangiocarcinoma in the Young (CITY) Study: Tumor Biology, Treatment Patterns, and Survival Outcomes in Adolescent Young Adults With Cholangiocarcinoma

Cancer research · Cholangiocarcinoma · BRAF

Young patients with cholangiocarcinoma exhibit more advanced disease at resection, harbor actionable genetic alterations such as FGFR2 fusions, BRAF, and ATM mutations, receive more adjuvant and palliative therapies, enroll more frequently in clinical trials, and demonstrate improved overall survival compared with older patients.

The Cholangiocarcinoma in the Young (CITY) Study is the largest analysis to date examining disease biology, treatment patterns, and survival outcomes in adolescent and young adults (AYAs) with cholangiocarcinoma (CCA). In a multi‑institutional cohort of 847 patients, 124 were 50 years or younger. Young patients had larger tumors, more node‑positive disease, and were more likely to receive adjuvant and palliative systemic therapy, including targeted therapy (23% vs 8%) and clinical‑trial enrollment (31% vs 19%). Molecular profiling revealed higher frequencies of FGFR2 fusions, BRAF mutations, and ATM mutations in young patients. In patients presenting with advanced disease, young patients achieved a median overall survival of 17.7 months compared to 13.5 months for older patients. These findings suggest that young CCA patients possess distinct biology with actionable alterations and experience improved survival despite a more advanced presentation.

Understanding the biological and clinical differences in adolescent and young adult cholangiocarcinoma enables the design of age‑specific therapeutic strategies and may improve outcomes in a historically underserved population through targeted therapy and increased clinical‑trial participation.

Evidence level: Korai humán adat. Kis vagy feltáró emberi adat.

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