Efficacy and Prognostic Factors of Regorafenib in the Treatment of BCLC Stage C Hepatocellular Carcinoma After Failure of the First-Line Therapy.

ABSTRACT

Purpose: Regorafenib is a standard second-line treatment for hepatocellular carcinoma (HCC). This study aimed to evaluate the efficacy and safety of regorafenib in the treatment of patients with Barcelona clinic liver cancer (BCLC) stage C HCC after failure of the first-line therapy and to analyze factors affecting the efficacy of regorafenib as the second-line treatment. Methods: A retrospective analysis was conducted on 103 BCLC stage C HCC patients who received regorafenib as the second-line treatment. Among them, 51 patients received regorafenib plus transarterial chemoembolization (TACE) and 52 patients received regorafenib alone. Progression-free survival (PFS), overall survival (OS), and adverse events were compared between the two groups, and factors influencing the efficacy of regorafenib were analyzed. Results: In patients with BCLC stage C HCC after failure of the first-line therapy, there was no statistically significant difference in median PFS between regorafenib plus TACE group and regorafenib group (5.3 vs 4.0 months, P=0.432). The median OS was significantly longer in the regorafenib plus TACE group than that in the regorafenib group (11.3 vs 8.2 months, P=0.034). Patients in both groups experienced adverse events at rates of 78.43% and 75%, respectively. Rates of grade III-IV serious adverse events were 19.61% and 13.46%, respectively. Hand-foot skin reactions, fatigue, abdominal pain, and hypertension were common side effects of regorafenib. The number of tumors was noted as an independent prognostic factor for PFS in the univariate and multivariate Cox regression analyses, while Eastern Cooperative Oncology Group (ECOG) performance status (ECOG-PS) score, tumor size, the number of tumors, and combined local therapy were independent prognostic factors for OS. Regorafenib combined with TACE treatment improved OS for patients with ECOG-PS scores of 0-1, tumor size < 5 cm, and the number of tumors ≥ 3 compared with regorafenib alone. Conclusion: Regorafenib exhibited to be a safe and effective sequential therapy for patients with BCLC stage C HCC after failure of the first-line treatment, and its combination with TACE could achieve a higher efficacy. ECOG-PS score, tumor size, the number of tumors, and combined local therapy were noted as prognostic factors affecting patients with BCLC stage C HCC who were treated with regorafenib.