Idarubicin versus Epirubicin in Transarterial Chemoembolization for Barcelona Clinic Liver Cancer Stage B Hepatocellular Carcinoma: An Open-label, Randomized, Phase IV Trial.

Abstract

Background Transarterial chemoembolization (TACE) is regarded as the first-line treatment for patients with Barcelona Clinic Liver Cancer (BCLC) stage B hepatocellular carcinoma (HCC). However, the optimal chemotherapeutic agent loaded in TACE remains controversial. Purpose To compare the efficacy and safety of idarubicin and epirubicin as loaded drugs in drug-eluting bead (DEB)-TACE in patients with BCLC stage B HCC. Materials and Methods In this open-label, phase IV trial, patients with BCLC stage B HCC were recruited from four centers from August 2020 to October 2022 and randomly assigned (at a one-to-one ratio) to undergo idarubicin DEB-TACE or epirubicin DEB-TACE. The primary end point was progression-free survival (PFS), which was measured from the time of randomization to the time of progression or death from any cause. The efficacy analysis was conducted on an intention-to-treat basis, and only participants who received treatment were included in the safety analysis. Results A total of 239 participants (median age, 57 years; IQR, 50-66 years; 210 male) were randomly assigned to the idarubicin group (<i>n</i> = 120) or the epirubicin group (<i>n</i> = 119). The primary analysis cutoff for PFS was March 1, 2023, with 167 events observed (70%; idarubicin group, 85 events; epirubicin group, 82 events). The median PFS was 10.8 months and 8.7 months in the idarubicin and epirubicin groups, respectively (hazard ratio [HR], 0.61; 95% CI: 0.44, 0.84; <i>P</i> = .002). The HR for median overall survival (OS) was 0.53 (95% CI: 0.31, 0.88), with OS rates of 81.5% and 77.3% at 12 months and 71.8% and 54.0% at 24 months for the idarubicin and epirubicin groups, respectively. The objective response rates were 70.8% and 57.1% for the idarubicin and epirubicin groups, respectively (<i>P</i> = .03). There was no evidence of a between-group difference in incidence of adverse events, including hematologic toxicity. No treatment-related deaths were observed. Conclusion Idarubicin DEB-TACE increased survival in participants with BCLC stage B HCC, without an increase in the incidence of any adverse events. Clinical trial registration no. ChiCTR2000034758 © RSNA, 2025 <i>Supplemental material is available for this article.</i>