Sezione di Gastroenterologia, DIBIMIS, University of Palermo, Italy.
Hepatology. 2013 Mar;57(3):1046-54. doi: 10.1002/hep.26221. Epub 2013 Feb 12.
The purpose was to assess the cost-effectiveness of sorafenib in the treatment of hepatocellular carcinoma (HCC) patients incorporating current prices and the results of the recent published field practice SOraFenib Italian Assessment (SOFIA) study. We created a Markov Decision Model to evaluate, in a hypothetical cohort of Caucasian male patients, aged 67 years with Barcelona Clinic Liver Cancer (BCLC) C HCC, or BCLC B HCC who were unfit or failed to respond to locoregional therapies, well compensated cirrhosis, and with performance status 0-1 according to Eastern Cooperative Oncology Group (ECOG), the cost-effectiveness of the following strategies: (1) full or dose-adjusted sorafenib for BCLC B and C patients together; (2) full or dose-adjusted sorafenib for BCLC B patients; (3) full or dose-adjusted sorafenib for BCLC C patients. Outcomes include quality-adjusted life years (QALYs), costs, and incremental cost-effectiveness ratio (ICER). In the base-case analysis dose-adjusted sorafenib was the most effective of the evaluated strategies. For dose-adjusted sorafenib, QALY was 0.44 for BCLC B and C patients together, 0.44 for BCLC C patients, and 0.38 for BCLC B patients. The ICER of dose-adjusted sorafenib compared with BSC was €34,534 per QALY gained for BCLC B and C patients together, €27,916 per QALY gained for BCLC C patients, and €54,881 per QALY gained for BCLC B patients. Results were sensitive to BSC survival rate, and sorafenib treatment duration.
In daily practice dose-adjusted, but not full-dose, sorafenib is a cost-effective treatment compared to BSC in intermediate and advanced HCC.
目的是评估索拉非尼治疗肝细胞癌（HCC）患者的成本效益，纳入当前价格和最近发表的实地实践 SOraFenib 意大利评估（SOFIA）研究的结果。我们创建了一个马尔可夫决策模型，以评估在一个假设的高加索男性患者队列中，年龄为 67 岁，巴塞罗那临床肝癌（BCLC）C 期 HCC 或 BCLC B 期 HCC，或不适合或对局部区域治疗无反应、代偿良好的肝硬化，以及根据东部合作肿瘤学组（ECOG）的表现状态 0-1，以下策略的成本效益：（1）BCLC B 和 C 期患者联合使用全剂量或剂量调整的索拉非尼；（2）BCLC B 期患者使用全剂量或剂量调整的索拉非尼；（3）BCLC C 期患者使用全剂量或剂量调整的索拉非尼。结果包括质量调整生命年（QALYs）、成本和增量成本效益比（ICER）。在基本分析中，剂量调整的索拉非尼是评估策略中最有效的。对于剂量调整的索拉非尼，BCLC B 和 C 期患者联合使用的 QALY 为 0.44，BCLC C 期患者为 0.44，BCLC B 期患者为 0.38。与 BSC 相比，剂量调整的索拉非尼的 ICER 为 BCLC B 和 C 期患者联合使用时每 QALY 增加 34534 欧元，BCLC C 期患者每 QALY 增加 27916 欧元，BCLC B 期患者每 QALY 增加 54881 欧元。结果对 BSC 生存率和索拉非尼治疗持续时间敏感。
在日常实践中，与 BSC 相比，剂量调整但非全剂量的索拉非尼是一种治疗中晚期 HCC 的具有成本效益的治疗方法。
