Division of Gastroenterology and Hepatology, University of Missouri, Kansas City, MO, USA.
Division of Gastroenterology and Hepatology, University of Michigan, 1500 E. Medical Center Dr., Ann Arbor, MI, 48109, USA.
Pharmacoeconomics. 2020 Jan;38(1):5-24. doi: 10.1007/s40273-019-00839-9.
The incidence of hepatocellular carcinoma (HCC) is increasing worldwide, with significant morbidity and associated costs. Treatment allocation depends on the stage of diagnosis; however, resource utilization can be significant across all stages. We aimed to summarize the available data on the cost effectiveness of surveillance of and treatments for HCC in the context of current treatment guidelines. We performed a focused review of studies investigating the economic burden and cost effectiveness of HCC surveillance treatment modalities published between January 2000 and January 2019. The overall economic burden of HCC is increasing in the USA and in several countries worldwide due to its rising incidence and the proliferation of therapies. Liver transplantation is a cost-effective strategy for early-stage HCC treatment in selected patients. In settings where liver transplantation is not available or in patients awaiting transplant, ablative or locoregional therapies are cost effective with increases in quality-adjusted life-years. First-line therapy with sorafenib for advanced stage HCC is cost effective in the treatment of compensated cirrhosis. The cost effectiveness of recently approved systemic therapies for advanced HCC require further investigation. Existing studies have shown that guideline-recommended surveillance techniques and several available therapies for the treatment of HCC are cost effective; however, there are limitations in the literature, including reliance on suboptimal modeling with incomplete/simplified model structure or inadequate inputs. With increasing therapeutic options in patients with HCC, understanding their relative value is critical in designing HCC treatment algorithms.
肝细胞癌 (HCC) 的发病率在全球范围内呈上升趋势，其发病率和相关费用都很高。治疗方案的选择取决于诊断的阶段；然而，在所有阶段，资源的利用都可能非常重要。我们旨在总结目前治疗指南背景下，用于 HCC 监测和治疗的成本效益的现有数据。我们对 2000 年 1 月至 2019 年 1 月期间发表的有关 HCC 监测治疗模式的经济负担和成本效益的研究进行了重点回顾。由于 HCC 的发病率上升和治疗方法的增多，HCC 的总体经济负担在美国和世界上的几个国家都在增加。肝移植是治疗早期 HCC 的一种具有成本效益的策略，适用于特定患者。在肝移植不可用或等待移植的情况下，消融或局部区域治疗具有成本效益，可以提高质量调整生命年。索拉非尼作为晚期 HCC 的一线治疗药物，对代偿性肝硬化具有成本效益。最近批准的晚期 HCC 全身治疗药物的成本效益需要进一步研究。现有研究表明，推荐的 HCC 监测技术和几种现有的 HCC 治疗方法具有成本效益；然而，文献中有一些局限性，包括依赖于不完整/简化模型结构或输入不充分的次优建模。随着 HCC 患者治疗选择的增加，了解它们的相对价值对于设计 HCC 治疗方案至关重要。
