Abstract
BACKGROUND: The prognosis is still dismal, although several tyrosine kinase inhibitors (TKIs) with/without immune checkpoint inhibitors (ICIs) have shown promising results in the treatment of biliary tract carcinoma (BTC). However, the combination of hepatic artery infusion chemotherapy (HAIC) with ICIs and TKIs may have potential in patients with BTC, according to the success of such a regimen for hepatocellular carcinoma. Hence, this study aimed to evaluate the preliminary efficacy and safety profile of combination therapy with HAIC plus ICI and lenvatinib in BTC patients. METHODS: This retrospective study included all BTC patients histologically diagnosed with combination therapy, which included HAIC with Gemox (Gemox-HAIC), programmed cell death-1 (PD-1)/programmed cell death-ligand 1 (PD-L1) inhibitor, and lenvatinib from July 2021 to October 2023. The outcomes were the objective response rate (ORR), disease control rate (DCR), progression-free survival (PFS), overall survival (OS), and safety profile. RESULTS: The median follow-up period was 7.0 months (range: 1.0-28.0 months). The ORR and DCR were 72.7% and 90.9%, respectively, with 0.0% CR, 72.7% PR, 18.2% SD, and 9.1% PD. The median PFS was 6.1 (4.3-8.0 (95% CI) months, and the 12-month accumulating PFS rate was 26.0%. The median OS was 10.3 (8.1-12.5 (95% CI) months, and the 12-month accumulating OS rate was 43.2%. The major adverse events included leukopenia (22.7%), thrombocytopenia (22.7%), vomiting (9.1%), etc. All AEs were grade 1-2 except for grade 3-4 leukopenia and 3-4 thrombocytopenia in one patient. CONCLUSION: The combination therapy of Gemox-HAIC with ICIs and lenvatinib shows promising efficacy and tolerable safety profiles in BTC patients.