Abstract
BACKGROUND: Intrahepatic cholangiocarcinoma (iCCA) is the second most prevalent primary liver cancer, and there are limited treatment options when resection is not eligible. Systemic chemotherapy (SYS) offers modest survival benefits, highlighting the need for more effective approaches. This study aimed to evaluate and compare hepatic arterial infusion chemotherapy (HAIC) with SYS in patients with unresectable iCCA in terms of efficacy and safety. METHODS: A propensity score-matched analysis was conducted on 111 patients with unresectable iCCA from March 2019 to October 2023. The cohort comprised 37 HAIC-treated and 74 SYS-treated patients. The primary endpoint was overall survival (OS), while the secondary endpoints included progression-free survival (PFS), objective response rate (ORR), disease control rate (DCR), and adverse events (AEs). RESULTS: The HAIC group demonstrated comparable survival outcomes to those of the SYS group, with a median OS of 23.7 and 19.3 months [hazard ratio (HR) =0.84, 95% confidence interval (CI): 0.53-1.35; P=0.487] and median PFS of 10.7 vs. 10.3 months (HR =0.75, 95% CI: 0.46-1.22; P=0.246), respectively. However, HAIC showed superior tumor control, achieving a significantly higher ORR (35.13% vs. 12.16%, P<0.05) and DCR (83.78% vs. 64.86%, P<0.05) as compared to SYS. Safety analysis revealed markedly lower grade 3-4 AEs in the HAIC group. CONCLUSIONS: This study demonstrated that HAIC can achieve comparable survival outcomes with superior local tumor control and reduced systemic toxicity as compared to SYS, suggesting its potential as an alternative treatment option for select patients.