S232 Efficacy and Safety of FOLFIRINOX as First-Line Treatment for Advanced Biliary Tract Cancers: A Single-Arm Meta-Analysis

Introduction: Biliary tract cancer (BTC) is a rare, aggressive malignancy with poor prognosis and limited treatment options. Gemcitabine and cisplatin (GemCis) have remained the standard first-line regimen for over a decade. However, response rates are modest, prompting interest in more active regimens. FOLFIRINOX, a triplet chemotherapy regimen originally developed for pancreatic cancer, has shown potential in BTC, but its role is not well defined. This study aimed to evaluate the efficacy and safety of first-line FOLFIRINOX in advanced BTC through a single-arm meta-analysis. Methods: A systematic review and meta-analysis were conducted, including studies that evaluated FOLFIRINOX as first-line therapy in advanced BTC. A total of 184 patients were analyzed across 4 studies. The sample included 80 women and 104 men with mean ages ranging from 49.9 to 65.6 years. Primary outcomes were complete response, overall survival, and progression-free survival. Secondary outcomes included treatment-related adverse events such as anemia, fatigue, and thrombocytopenia. Pooled estimates were generated using a random-effects model, and forest plots were used to assess consistency across studies. Results: The pooled complete response rate was 2.9% (95% CI: 1.2–6.7%), with all studies showing statistically significant contributions to the overall estimate (Z-values from –2.29 to –5.81, P < 0.05). Mean progression-free survival was 6.66 months (95% CI: 6.59–6.74), with a narrow confidence interval and minimal variance across studies, suggesting consistent disease control. Mean overall survival was 13.1 months (95% CI: 13.25–21.97). Hematologic toxicity was common: anemia occurred in 71.4% (95% CI: 63–78.6%), fatigue in 67.7% (95% CI: 58.3–75.9%), and thrombocytopenia in 61.8% (95% CI: 53.5–69.5%). These adverse events were largely grade ≥2, highlighting a need for careful supportive care during treatment. Conclusion: FOLFIRINOX demonstrates modest but consistent survival benefits in patients with advanced BTC, with a low rate of complete response but meaningful disease stabilization. However, the high incidence of hematologic toxicity emphasizes the importance of patient selection and monitoring. These findings suggest that FOLFIRINOX may be a viable first-line option for fit patients with advanced BTC, but further prospective studies are needed to confirm these results and identify which patients are most likely to benefit.