Imedio Esteban Rodrigo, Beveridge Roberto Díaz, Urtasun Jorge Aparicio, Campos Gema Bruixola, Estellés David Lorente, Esparcia María Fonfría, Daroqui Javier Caballero, Huerta Ángel Segura, Ortiz Alejandra Giménez, Salcedo Joaquin Montalar
Department of Medical Oncology, University Hospital La fe, Valencia, Spain.
Med Oncol. 2014 May;31(5):948. doi: 10.1007/s12032-014-0948-x. Epub 2014 Apr 17.
Sorafenib is the standard treatment of patients with advanced hepatocellular carcinoma (HCC), with demonstrated outcome benefits in randomized clinical trials. We present a single-center experience with sorafenib with the aim to establish its efficacy and safety in daily clinical practice. A total of 62 patients were treated with sorafenib 400 mg/12 h until disease progression or unacceptable toxicity. Response rates, incidence of adverse events, actuarial disease-free survival, and overall survival (OS) were estimated. Univariate and multivariate analyses of prognostic factors for survival were also performed. Median treatment duration was 92 days. A 43% disease control rate was achieved (partial response, 15% and disease stabilization, 28%). After a median follow-up of 24.1 months, the median progression-free survival and OS for the overall population were 5.8 and 6.7 months, respectively, with survival rates of 27% at 1 year and 17 % at 2 years. The most common grade 3-4 adverse events were fatigue (19%), hand-foot syndrome (8%), hypertension (5%), and diarrhea (3%). The univariate analysis showed that patient performance status (PS), use of previous treatments, and albumin >3.5 g/dL were significant prognostic factors for survival. In the multivariate study, only PS, alcoholic etiology and albumin >3.5 g/dL remained as independent predictors of survival. Sorafenib is a safe and moderately effective drug in HCC, although patients must be properly selected before starting therapy. Baseline PS, Barcelona Clinic Liver Cancer staging, and liver function should be taken into account as prognostic factors. Results in daily practice are somewhat inferior than observed in clinical trials.
索拉非尼是晚期肝细胞癌（HCC）患者的标准治疗药物，在随机临床试验中已证明其具有疗效优势。我们介绍了单中心使用索拉非尼的经验，旨在确定其在日常临床实践中的疗效和安全性。共有62例患者接受索拉非尼400mg/12小时治疗，直至疾病进展或出现不可接受的毒性反应。评估了缓解率、不良事件发生率、无病生存概率和总生存期（OS）。还对生存预后因素进行了单因素和多因素分析。中位治疗持续时间为92天。疾病控制率达到43%（部分缓解15%，疾病稳定28%）。中位随访24.1个月后，总体人群的中位无进展生存期和OS分别为5.8个月和6.7个月，1年生存率为27%，2年生存率为17%。最常见的3-4级不良事件为乏力（19%）、手足综合征（8%）、高血压（5%）和腹泻（3%）。单因素分析显示，患者的体能状态（PS）、既往治疗的使用情况以及白蛋白>3.5g/dL是生存的重要预后因素。在多因素研究中，只有PS、酒精性病因和白蛋白>3.5g/dL仍然是生存的独立预测因素。索拉非尼在HCC治疗中是一种安全且疗效中等的药物，不过在开始治疗前必须对患者进行适当选择。应将基线PS、巴塞罗那临床肝癌分期和肝功能作为预后因素加以考虑。日常实践中的结果略逊于临床试验中的观察结果。
