Hepatic Arterial Infusion Pump Chemotherapy in Patients With Unresectable Intrahepatic Cholangiocarcinoma—PUMP-2 Trial

PURPOSE: The 3-year overall survival (OS) for advanced intrahepatic cholangiocarcinoma (iCCA) confined to the liver after systemic chemotherapy with gemcitabine-cisplatin (gem-cis) is only 3%. Hepatic arterial infusion pump (HAIP) chemotherapy with floxuridine aims to control the liver disease and improve survival. The aim of this study was to assess the effectiveness of HAIP chemotherapy with floxuridine and concurrent systemic gem-cis in patients with unresectable liver-confined iCCA in the Netherlands. METHODS: We performed a nonrandomized multicenter phase II trial. Treatment-naïve patients and patients previously treated with systemic therapy were eligible. Up to six cycles of HAIP floxuridine and eight cycles of concurrent systemic gem-cis were administered. The primary endpoint was 1-year OS compared with a historical cohort. RESULTS: < .001). The 3-year OS was 31.5% (95% CI, 20.4% to 48.6%). CONCLUSION: Combining HAIP chemotherapy with floxuridine and systemic gem-cis in patients with unresectable liver-confined iCCA had a 1- and 3-year OS superior to gem-cis alone in historical cohorts.