Hepatic arterial infusion chemotherapy with Folfox 4 regimen versus cisplatin and gemcitabine for locally advanced intrahepatic cholangiocarcinoma.

For locally advanced intrahepatic cholangiocarcinoma (ICC), the combination of cisplatin plus gemcitabine (CisGem) is the standard first-line treatment. However, the outcome remains unsatisfied with the median overall survival (OS) of 11.7 months. We aimed to compare the effect of CisGem regimen and hepatic arterial infusion chemotherapy (HAIC) with Folfox 4 for locally advanced ICC. 97 Locally advanced ICC patients treated by CisGem regimen or HAIC with Folfox 4 in our institution from 2017 to 2019 were studied as training group. 43 locally advanced ICCs receiving CisGem chemotherapy or HAIC with Folfox 4 were investigated as validation group. The median OS was 14.5 months among 37 ICC patients from the HAIC group and 10.3 months among 60 ICC cases in the CisGem group. The median PFS in the HAIC group was 8.2 months in contrast to 5.3 months in the CisGem group. Additionally, objective response rate (ORR) in the HAIC group was markedly better than one in the CisGem group (29.7% v 5.0%). Patients from the HAIC group suffered from less AE (particularly 3-4 grade AE) than those in the CisGem group. The prediction nomogram models for OS and PFS were built respectively after Cox multivariate analysis, which were confirmed to be clinically useful by external validation cohort. These data here suggested HAIC with Folfox 4 was a potential first-line treatment option for local advanced ICC.