School of Graduate, Bengbu Medical College, Bengbu, 233030, People's Republic of China.
Department of Interventional Radiology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, 230001, People's Republic of China.
Drug Des Devel Ther. 2023 Feb 16;17:507-518. doi: 10.2147/DDDT.S400533. eCollection 2023.
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.
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.
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.
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.
瑞戈非尼是治疗肝细胞癌（HCC）的标准二线治疗药物。本研究旨在评估瑞戈非尼作为一线治疗失败后的巴塞罗那临床肝癌（BCLC）C 期 HCC 患者的二线治疗的疗效和安全性，并分析影响瑞戈非尼疗效的因素。
对 103 例接受瑞戈非尼二线治疗的 BCLC 期 C 期 HCC 患者进行回顾性分析。其中，51 例患者接受瑞戈非尼联合肝动脉化疗栓塞（TACE）治疗，52 例患者接受瑞戈非尼单药治疗。比较两组患者的无进展生存期（PFS）、总生存期（OS）和不良事件，并分析影响瑞戈非尼疗效的因素。
在一线治疗失败后的 BCLC 期 C 期 HCC 患者中，瑞戈非尼联合 TACE 组与瑞戈非尼组的中位 PFS 无统计学差异（5.3 与 4.0 个月，P=0.432）。瑞戈非尼联合 TACE 组的中位 OS 明显长于瑞戈非尼组（11.3 与 8.2 个月，P=0.034）。两组患者不良事件发生率分别为 78.43%和 75%，Ⅲ/Ⅳ级严重不良事件发生率分别为 19.61%和 13.46%。手足皮肤反应、乏力、腹痛和高血压是瑞戈非尼常见的不良反应。单因素和多因素 Cox 回归分析显示，肿瘤数量是 PFS 的独立预后因素，而东部肿瘤协作组（ECOG）体能状态（ECOG-PS）评分、肿瘤大小、肿瘤数量和联合局部治疗是 OS 的独立预后因素。与瑞戈非尼单药治疗相比，瑞戈非尼联合 TACE 治疗可改善 ECOG-PS 评分为 0-1、肿瘤大小<5cm 和肿瘤数量≥3 的患者的 OS。
瑞戈非尼作为一线治疗失败后的 BCLC 期 C 期 HCC 患者的二线治疗安全有效，联合 TACE 可提高疗效。ECOG-PS 评分、肿瘤大小、肿瘤数量和联合局部治疗是影响接受瑞戈非尼治疗的 BCLC 期 C 期 HCC 患者预后的因素。
