Abstract
BACKGROUND AND OBJECTIVES: Intrahepatic cholangiocarcinoma (iCCA) is a highly malignant biliary tumor associated with a poor prognosis. Here, we explored conversion therapy (CT) for the treatment of locally advanced iCCA (LA-iCCA). METHODS: We retrospectively enrolled 115 patients with LA-iCCA at our single center from January 2019 to June 2024, with the final follow-up conducted in January 2025. The primary outcome measured was overall survival (OS), while the secondary outcome focused on progression-free survival (PFS). We utilized a propensity score matching (PSM) approach to adjust for potential confounders between the CT group and the systemic therapy (ST) group. RESULTS: Before PSM, the median OS was 34.57 months (95% CI: 19.22-49.91), while the median PFS was 17.30 months (95% CI: 12.89-21.71) in the CT group. Both OS and PFS were significantly longer than those in the ST group (both p < 0.001). After PSM, both groups were successfully matched, resulting in 31 patients in each group. In the CT group, the median OS and PFS were 45.53 months (95% CI: 29.19-61.87) and 18.23 months (95% CI: 7.02-29.45), respectively. Both values were significantly higher than those in the ST group, with P values of less than 0.001 for OS and 0.002 for PFS. Furthermore, CT was identified as an independent factor associated with improved outcomes, both before and after PSM. CONCLUSIONS: CT was an effective approach for improving outcomes in patients with LA-iCCA.