Balloon-occluded hepatic arterial infusion for unresectable hepatocellular carcinoma: a phase II trial interim analysis

Background: Hepatic arterial infusion chemotherapy (HAIC) is increasingly used to treat unresectable hepatocellular carcinoma (HCC), yet some HCC patients remain unresponsive or show disease progression. Purpose: This phase II study (ChiCTR2300077718) aimed to assess the efficacy and safety of balloon-occluded HAIC (bHAIC) based on the FOLFOX (oxaliplatin plus fluorouracil and leucovorin) regimen (bHAIC-FO) in unresectable HCC. Materials and methods: The present work is an investigator-initiated, multicenter, single-arm, prospective cohort trial. Unresectable HCC patients who had undergone a successful bHAIC procedure were included in the efficacy and safety analysis. After all patients received at least one post-bHAIC follow-up, an interim analysis was conducted in accordance with the predefined protocol. The primary endpoint of this interim analysis was the objective response rate (ORR). Secondary endpoints included changes in alpha-fetoprotein (AFP) levels and the occurrence of adverse events (AEs). Interim data were collected and analyzed using SPSS, version 27.0. Results: 50 patients received at least one successful bHAIC procedure and were included in the efficacy and safety analysis. The best ORR for 45 evaluable patients was 53.3% according to Response Evaluation Criteria in Solid Tumors 1.1 (RECIST 1.1) criteria, and 82.2% according to modified RECIST (mRECIST) criteria. In the patients population, 36 treatment-naive individuals achieved an 88.9% ORR according to mRECIST criteria, with 15 cases achieving radiological complete response (CR). Overall, 77.3% of patients with elevated AFP levels experienced a reduction of over 50% in their AFP levels. All AEs were manageable by medication or dose modifications. Conclusion: bHAIC-FO demonstrates encouraging efficacy and acceptable safety profiles in unresectable HCC treatment, particularly in treatment-naive patients. Clinical trial registration: www.chictr.org.cn, identifier ChiCTR2300077718.