Survival outcomes and prognostic factors in de novo advanced biliary tract cancer: insights from a Saudi multicenter cohort.

PURPOSE: Advanced biliary tract cancer (BTC) carries a poor prognosis, with limited regional data available. This study evaluated outcomes, treatment patterns, and prognostic factors in a multicenter cohort of patients with de novo advanced BTC.

METHODS

A retrospective cohort of 271 patients with de novo advanced biliary tract cancer diagnosed at five tertiary centers in Saudi Arabia (2016-2023) was analyzed. Clinical, pathological, treatment, and molecular data were extracted from electronic health records. Overall survival was estimated using the Kaplan-Meier analysis, and prognostic factors were assessed through multivariable Cox regression.

RESULTS

Systemic therapy was administered to 211 patients (77.9%). Among the entire cohort, 92 patients (33.9%) received second-line therapy, 25 (9.2%) received third-line therapy, and 8 (3.0%) received fourth-line therapy. Median overall survival (OS) was 9.5 months (95% CI, 8.4-10.6). On multivariable analysis, inferior OS was independently associated with poor ECOG performance status (PS 4: HR 4.08, P = 0.031), age 15-39 years (HR 4.44, P < 0.001), extrahepatic cholangiocarcinoma (HR 1.91, P = 0.017), cirrhosis (HR 3.13, P = 0.004), and elevated CEA (HR 1.92, P = 0.003). Patients with disease control had a 71% lower hazard of death (HR 0.29, 95% CI 0.18-0.47) and longer median OS (16.0 vs 7.7 months).

CONCLUSIONS

Advanced BTC in Saudi Arabia demonstrates poor outcomes, with survival strongly influenced by performance status, age, tumor site, cirrhosis, and biomarkers. Treatment exposure was limited, with only one-third receiving second-line therapy, highlighting the urgent need for expanded access to immunotherapy and molecular profiling.