Division of Gastroenterology, University of Michigan, Ann Arbor, MI.
College of Pharmacy, University of Michigan, Ann Arbor, MI.
Hepatology. 2017 Jan;65(1):122-133. doi: 10.1002/hep.28881. Epub 2016 Nov 25.
Sorafenib is the only chemotherapeutic approved for treatment of advanced hepatocellular carcinoma (HCC). However, its effectiveness in patients with Child-Pugh class B cirrhosis and any moderating effects of health system characteristics are unclear. We examined the survival and cost-effectiveness associated with sorafenib in elderly patients with advanced HCC. We performed an analysis of Medicare beneficiaries with HCC diagnoses from 2007 to 2009. We compared advanced stage patients with HCC (American Joint Committee on Cancer stage III/IV) who received sorafenib within 6 months of diagnosis (and were otherwise untreated) to advanced stage patients with HCC who received no therapy (control). We performed univariate and multivariate analyses to identify predictors of survival. Incremental cost-effectiveness ratios (ICERs) were calculated for sorafenib-treated and control patients. We included 228 sorafenib-treated patients and 870 control patients. The median survival of the sorafenib-treated patients was 150.5 days versus 62 days for control patients. On multivariate analysis, significant predictors of improved survival were treatment with sorafenib (hazard ratio [HR], 0.66; 95% confidence interval [CI], 0.57-0.77), being seen at a National Cancer Institute-designated cancer center (HR, 0.77; 95% CI, 0.62-0.97), and being seen at a transplantation center (HR, 0.77; 95% CI, 0.65-0.93). Predictors of worse survival included stage IV disease (HR, 1.40; 95% CI, 1.24-1.58), decompensated cirrhosis (HR, 1.49; 95% CI, 1.30-1.70), and treatment in an urban setting (HR, 1.45; 95% CI, 1.21-1.73.) Although sorafenib use was associated with a survival benefit (HR, 0.61; 95% CI, 0.47-0.79) among patients with decompensated cirrhosis, the median survival benefit was 31 days, and it was not cost-effective (ICER, $224,914 per life year gained).
Sorafenib is associated with improved survival in elderly patients with advanced HCC; however, it is not cost-effective among those with hepatic decompensation. (Hepatology 2017;65:122-133).
索拉非尼是唯一被批准用于治疗晚期肝细胞癌(HCC)的化疗药物。然而,其在 Child-Pugh 分级 B 肝硬化患者中的疗效以及卫生系统特征的任何调节作用尚不清楚。我们研究了索拉非尼在老年晚期 HCC 患者中的生存和成本效益。我们对 2007 年至 2009 年期间患有 HCC 的医疗保险受益人的数据进行了分析。我们比较了在诊断后 6 个月内接受索拉非尼治疗(且未接受其他治疗)的晚期 HCC 患者(美国癌症联合委员会分期 III/IV 期)与未接受治疗的晚期 HCC 患者(对照组)。我们进行了单变量和多变量分析,以确定生存的预测因素。计算了索拉非尼治疗组和对照组患者的增量成本效益比(ICER)。我们纳入了 228 名接受索拉非尼治疗的患者和 870 名对照组患者。接受索拉非尼治疗的患者中位生存期为 150.5 天,而对照组为 62 天。多变量分析表明,生存改善的显著预测因素包括接受索拉非尼治疗(风险比 [HR],0.66;95%置信区间 [CI],0.57-0.77)、在国家癌症研究所指定的癌症中心就诊(HR,0.77;95%CI,0.62-0.97)和在移植中心就诊(HR,0.77;95%CI,0.65-0.93)。生存较差的预测因素包括 IV 期疾病(HR,1.40;95%CI,1.24-1.58)、失代偿性肝硬化(HR,1.49;95%CI,1.30-1.70)和在城市环境中接受治疗(HR,1.45;95%CI,1.21-1.73)。尽管失代偿性肝硬化患者使用索拉非尼治疗与生存获益相关(HR,0.61;95%CI,0.47-0.79),但中位生存获益仅为 31 天,且不具有成本效益(ICER,每获得 1 个生命年需花费 224914 美元)。
索拉非尼可改善老年晚期 HCC 患者的生存;然而,对于肝功能失代偿的患者,它并不具有成本效益。