Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada.
Institute for Clinical Evaluative Sciences, Toronto, Ontario, Canada.
Cancer Med. 2017 Sep;6(9):2017-2033. doi: 10.1002/cam4.1119. Epub 2017 Aug 8.
Patients with early-stage hepatocellular carcinoma (HCC) are potential candidates for curative treatments such as radiofrequency ablation (RFA), surgical resection (SR), or liver transplantation (LT), which have demonstrated a significant survival benefit. We aimed to estimate the cost-effectiveness of curative and combination treatment strategies among patients diagnosed with HCC during 2002-2010. This study used Ontario Cancer Registry-linked administrative data to estimate effectiveness and costs (2013 USD) of the treatment strategies from the healthcare payer's perspective. Multiple imputation by logistic regression was used to handle missing data. A net benefit regression approach of baseline important covariates and propensity score adjustment were used to calculate incremental net benefit to generate incremental cost-effectiveness ratio (ICER) and uncertainty measures. Among 2,222 patients diagnosed with HCC, 10.5%, 14.1%, and 10.3% received RFA, SR, and LT monotherapy, respectively; 0.5-3.1% dual treatments; and 0.5% triple treatments. Compared with no treatment (53.2%), transarterial chemoembolization (TACE) + RFA (average $2,465, 95% CI: -$20,000-$36,600/quality-adjusted life years [QALY]) or RFA monotherapy ($15,553, 95% CI: $3,500-$28,500/QALY) appears to be the most cost-effective modality with lowest ICER value. The cost-effectiveness acceptability curve showed that if the relevant threshold was $50,000/QALY, RFA monotherapy and TACE+ RFA would have a cost-effectiveness probability of 100%. Strategies using LT delivered the most additional QALYs and became cost-effective at a threshold of $77,000/QALY. Our findings found that TACE+ RFA dual treatment or RFA monotherapy appears to be the most cost-effective curative treatment for patients with potential early stage of HCC in Ontario. These findings highlight the importance of identifying and measuring differential benefits, costs, and cost-effectiveness of alternative HCC curative treatments in order to evaluate whether they are providing good value for money in the real world.
患有早期肝细胞癌 (HCC) 的患者是接受根治性治疗（如射频消融 (RFA)、手术切除 (SR) 或肝移植 (LT)）的潜在候选者，这些治疗方法已显示出显著的生存获益。我们旨在评估 2002 年至 2010 年间诊断为 HCC 的患者接受根治性和联合治疗策略的成本效益。本研究使用安大略省癌症登记处链接的行政数据，从医疗保健支付者的角度估计治疗策略的有效性和成本（2013 年美元）。使用逻辑回归的多重插补来处理缺失数据。使用基线重要协变量的净收益回归方法和倾向评分调整来计算增量净收益，以生成增量成本效益比 (ICER) 和不确定性度量。在 2222 名诊断为 HCC 的患者中，分别有 10.5%、14.1%和 10.3%接受了 RFA、SR 和 LT 单药治疗；0.5-3.1%接受了双重治疗；0.5%接受了三重治疗。与不治疗（53.2%）相比，经动脉化疗栓塞 (TACE)+RFA（平均 2465 美元，95%CI：-20000 美元至 36600 美元/质量调整生命年 [QALY]）或 RFA 单药治疗（15553 美元，95%CI：3500 美元至 28500 美元/QALY）似乎是最具成本效益的治疗方法，ICER 值最低。成本效益可接受性曲线表明，如果相关阈值为 50000 美元/QALY，则 RFA 单药治疗和 TACE+RFA 的成本效益概率为 100%。使用 LT 的策略提供了最多的额外 QALYs，并在阈值为 77000 美元/QALY 时具有成本效益。我们的研究结果发现，TACE+RFA 双重治疗或 RFA 单药治疗似乎是安大略省患有潜在早期 HCC 患者最具成本效益的根治性治疗方法。这些发现强调了确定和衡量替代 HCC 根治性治疗方法的差异效益、成本和成本效益的重要性，以便评估它们在现实世界中是否提供了物有所值。
