Background: Biliary tract cancers (BTCs) are aggressive malignancies with limited treatment options, especially after first-line chemotherapy failure. FOLFIRINOX, though established for pancreatic cancer, has shown promise in advanced BTC, yet its role as a second-line treatment remains unclear. To address this gap, we conducted a retrospective cohort study to evaluate the efficacy and safety of FOLFIRINOX and performed a systematic review with meta-analysis to compare its outcomes with currently recommended regimens, including FOLFIRI, FOLFOX, and nal-IRI/FL.

Methods: We retrospectively analyzed 54 patients with BTC treated with FOLFIRINOX as second-line therapy after progression on first-line chemotherapy at a single tertiary hospital between 2011 and 2022. A systematic review and meta-analysis, registered with PROSPERO, incorporated 21 studies comparing second-line regimens for BTC, assessing progression-free survival (PFS), overall survival (OS), x-objective response rate (ORR), disease control rate (DCR), and safety.

Results: In our cohort, median PFS and OS were 2.7 and 8.9 months, respectively. ORR was 12.5%, and DCR was 52.1%, with 4.2% achieving complete response. Meta-analysis revealed pooled PFS and OS for FOLFIRINOX at 4.15 and 8.91 months, respectively, suggesting a potential benefit over FOLFIRI and FOLFOX. Grade 3–5 neutropenia occurred in 40.7% of patients, leading to dose reductions in 29.6% but low discontinuation rates (3.7%).

Conclusion: FOLFIRINOX demonstrates numerically favorable outcomes compared to current second-line regimens for advanced BTC, with manageable toxicities. These findings suggest FOLFIRINOX as a potential second-line option, warranting further prospective validation and patient selection refinement.

- Authors (Pusan National University)

 · First author: Kihun Kim (Department of Occupational and Environmental Medicine, Pusan National University Yangsan Hospital)

 · Corresponding author: Yun Hak Kim (Department of Anatomy and Department of Biomedical Informatics, Pusan National University)

- Title of original paper: Comparison of second-line chemotherapy regimens in advanced biliary tract cancer: a systematic review, meta-analysis, and single-center retrospective cohort study

- Journal: International Journal of Surgery

- Web link: https://doi.org/10.1097/JS9.0000000000003367 

- Contact e-mail: yunhak10510@pusan.ac.kr