Zhang Pengfei, Yang Yu, Wen Feng, He Xiaofeng, Tang Ruilei, Du Zedong, Zhou Jing, Zhang Jian, Li Qiu
aState Key Laboratory of Biotherapy, Department of Medical Oncology, Cancer Center, West China Hospital, Sichuan University bDepartment of Medical Oncology, First People's Hospital of Longquanyi District cDepartment of Medical Oncology, 363 Hospital, Chengdu, Sichuan, China.
Eur J Gastroenterol Hepatol. 2015 Jul;27(7):853-9. doi: 10.1097/MEG.0000000000000373.
Sorafenib has been shown to significantly improve the overall survival of patients with advanced hepatocellular carcinoma (HCC). This study aimed to assess the cost-effectiveness of sorafenib as a first-line treatment for patients with advanced HCC.
To carry out the analysis, we collected the data on the efficacy and safety of patients treated with sorafenib from medical records and follow-up of these patients. A Markov model comprising three health states (progression-free survival, progressive disease, and death) was created to simulate the process of advanced HCC. We calculated the data on cost from the perspective of Chinese patients. Sensitivity analyses were also carried out to explore the impact of several essential variables.
Overall, 94 patients with advanced HCC were included in our study: 70 in the Child-Pugh A group and 24 in the Child-Pugh B group. The median overall survival was 8.0 months (95% confidence interval: 7.21-8.50). In general, treatment with sorafenib was estimated to increase costs by $18,251.84 compared with best supportive care, with a gain of 0.18 quality-adjusted life years (QALYs). Thus, the incremental cost-effective ratio was $101,399.11/QALY for sorafenib versus best supportive care. In addition, in patients with Child-Pugh A liver function, the total costs and effectiveness were $20,643.06 and 0.48 QALYs, respectively, whereas in the Child-Pugh class B group, the total costs and effectiveness were $15,844.33 and 0.28 QALYs.
On the basis of the commonly accepted willingness-to-pay threshold ($20,301.00/QALY in China), sorafenib is not a cost-effective option as a first-line treatment for patients with advanced HCC.
索拉非尼已被证明可显著提高晚期肝细胞癌（HCC）患者的总生存期。本研究旨在评估索拉非尼作为晚期HCC患者一线治疗的成本效益。
为进行分析，我们从病历及对这些患者的随访中收集了接受索拉非尼治疗患者的疗效和安全性数据。创建了一个包含三个健康状态（无进展生存期、疾病进展和死亡）的马尔可夫模型来模拟晚期HCC的病程。我们从中国患者的角度计算了成本数据。还进行了敏感性分析以探讨几个关键变量的影响。
总体而言，94例晚期HCC患者纳入我们的研究：Child-Pugh A组70例，Child-Pugh B组24例。中位总生存期为8.0个月（95%置信区间：7.21 - 8.50）。一般来说，与最佳支持治疗相比，索拉非尼治疗估计会使成本增加18,251.84美元，获得0.18个质量调整生命年（QALY）。因此，索拉非尼相对于最佳支持治疗的增量成本效益比为101,399.11美元/QALY。此外，在肝功能为Child-Pugh A级的患者中，总成本和效果分别为20,643.06美元和0.48 QALY，而在Child-Pugh B级组中，总成本和效果分别为15,844.33美元和0.28 QALY。
基于普遍接受的支付意愿阈值（中国为20,301.00美元/QALY），索拉非尼作为晚期HCC患者的一线治疗并非具有成本效益的选择。
