1 Department of Radiology and the Comparative Effectiveness, Cost, and Outcomes Research Center, University of Washington, 1959 NE Pacific St, Seattle, WA 98195.
2 Department of Medicine, University of Washington, Seattle, WA.
AJR Am J Roentgenol. 2018 Jun;210(6):1359-1365. doi: 10.2214/AJR.17.19094. Epub 2018 Apr 9.
The purpose of this study was to compare the clinical effectiveness of embolization with that of sorafenib in the management of hepatocellular carcinoma as practiced in real-world settings.
This population-based observational study was conducted with the Surveillance, Epidemiology, and End Results-Medicare linked database. Patients 65 years old and older with a diagnosis of primary liver cancer between 2007 and 2011 who underwent embolization or sorafenib treatment were identified. Patients were excluded if they had insufficient claims records, a diagnosis of intrahepatic cholangiocarcinoma, or other primary cancer or had undergone liver transplant or combination therapy. The primary outcome of interest was overall survival. Inverse probability of treatment weighting models were used to control for selection bias.
The inclusion and exclusion criteria were met by 1017 patients. Models showed good balance between treatment groups. Compared with those who underwent embolization, patients treated with sorafenib had significantly higher hazard of earlier death from time of treatment (hazard ratio, 1.87; 95% CI, 1.46-2.37; p < 0.0001) and from time of cancer diagnosis (hazard ratio, 1.87; 95% CI, 1.46-2.39; p < 0.0001). The survival advantage after embolization was seen in both intermediate- and advanced-stage disease.
This comparative effectiveness study of Medicare patients with hepatocellular carcinoma showed significantly longer overall survival after treatment with embolization than with sorafenib. Because these findings conflict with expert opinion-based guidelines for treatment of advanced-stage disease, prospective randomized comparative trials in this subpopulation would be justified.
本研究旨在比较栓塞治疗与索拉非尼治疗在真实世界环境中对肝细胞癌的临床疗效。
本基于人群的观察性研究使用了监测、流行病学和最终结果-医疗保险数据库进行。在 2007 年至 2011 年间诊断为原发性肝癌且接受栓塞或索拉非尼治疗的年龄在 65 岁及以上的患者被纳入研究。如果患者的索赔记录不足、患有肝内胆管癌或其他原发性癌症、接受过肝移植或联合治疗,则将其排除在外。主要观察终点为总生存期。采用逆概率治疗加权模型来控制选择偏倚。
1017 名患者符合纳入和排除标准。模型显示两组之间具有良好的平衡。与接受栓塞治疗的患者相比,接受索拉非尼治疗的患者从治疗开始时(风险比,1.87;95%置信区间,1.46-2.37;p<0.0001)和从癌症诊断开始时(风险比,1.87;95%置信区间,1.46-2.39;p<0.0001)更早死亡的风险显著更高。栓塞治疗后的生存优势在中晚期疾病中均可见。
这项针对医疗保险患者肝细胞癌的比较有效性研究表明,栓塞治疗后的总生存期明显长于索拉非尼治疗。由于这些发现与基于专家意见的晚期疾病治疗指南相冲突,因此在该亚人群中进行前瞻性随机对照试验是合理的。