Pharmaceutical Health Services Research, University of Maryland School of Pharmacy, 220 Arch Street, 12th floor, Baltimore, MD, 21201, USA,
Pharmacoeconomics. 2014 Jan;32(1):63-74. doi: 10.1007/s40273-013-0109-7.
The incidence of hepatocellular carcinoma (HCC) is increasing in the USA and worldwide. Several treatments are available for patients diagnosed at any disease stage. It remains unclear how medical expenditures vary across patients who remain untreated or undergo different modes of therapy. We evaluate the comparative and cost effectiveness of treatment modalities for HCC from a Medicare perspective.
The Surveillance, Epidemiology, and End Results (SEER) registries and linked Medicare database with claims from Parts A/B were used to identify Medicare enrollees with initial diagnosis of HCC between 2000 and 2007 and followed through 2009. Patients were assigned to treatment modalities based on HCC staging systems: transplant, resection, liver directed, radiation, chemotherapy or no treatment. Survival benefits and cumulative Medicare expenditures were estimated in multivariate models, stratified by initial disease stage, to control for confounding. Cost-effectiveness ratios compared costs and benefits of the modalities across initial stages.
Cancer stages I, II, III, IV and unstaged represented 24, 9, 14, 17 and 37 % of 11,047 patients, respectively. Fewer than 40 % received any treatment. Relative to no treatment, transplant was most effective in reducing mortality, followed by resection, liver directed, and radiation or chemotherapy. Resection tended to be most cost effective in early staged and unstaged patients; transplant was least cost effective. In stage IV patients, liver directed therapy was more cost effective than chemotherapy or radiation.
Survival benefit was attributable to all treatment modalities. More effective treatments incurred greater Medicare expenditures, but resection patients incurred the least expenditures per year of life gained.
在美国和全球范围内，肝细胞癌（HCC）的发病率正在增加。对于任何疾病阶段诊断出的患者，都有多种治疗方法。目前尚不清楚未接受治疗或接受不同治疗模式的患者的医疗支出如何有所不同。我们从医疗保险的角度评估 HCC 的治疗方式的比较和成本效益。
使用监测、流行病学和最终结果（SEER）登记处和与 A/B 部分索赔相关联的医疗保险数据库，来确定在 2000 年至 2007 年间初次诊断为 HCC 并在 2009 年之前接受随访的医疗保险参保者。根据 HCC 分期系统将患者分配至治疗方式：移植、切除术、肝脏定向治疗、放射治疗、化学疗法或不治疗。在多变量模型中，根据初始疾病阶段分层，估计生存获益和累积医疗保险支出，以控制混杂因素。在初始阶段，对成本效益比进行了比较，以比较各模式的成本和效益。
癌症 I 期、II 期、III 期、IV 期和未分期分别占 11047 例患者的 24%、9%、14%、17%和 37%。不到 40%的患者接受了任何治疗。与不治疗相比，移植在降低死亡率方面最有效，其次是切除术、肝脏定向治疗和放射治疗或化学疗法。在早期分期和未分期患者中，切除术倾向于最具成本效益；移植最不具成本效益。在 IV 期患者中，肝脏定向治疗比化学疗法或放射治疗更具成本效益。
生存获益归因于所有治疗方式。更有效的治疗方法会产生更高的医疗保险支出，但每年每增加一年生命，切除术患者的支出最少。
