Stanford University School of Medicine, Stanford, CA.
Baylor Simmons Transplant Institute, Fort Worth, TX.
Hepatology. 2017 Jul;66(1):46-56. doi: 10.1002/hep.29137. Epub 2017 May 27.
All-oral direct acting antivirals (DAAs) have been shown to have high safety and efficacy in treating patients with hepatitis C virus (HCV) awaiting liver transplant (LT). However, there is limited empirical evidence comparing the health and economic outcomes associated with treating patients pre-LT versus post-LT. The objective of this study was to analyze the cost-effectiveness of pre-LT versus post-LT treatment with an all-oral DAA regimen among HCV patients with hepatocellular carcinoma (HCC) or decompensated cirrhosis (DCC). We constructed decision-analytic Markov models of the natural disease progression of HCV in HCC patients and DCC patients waitlisted for LT. The model followed hypothetical cohorts of 1,000 patients with a mean age of 50 over a 30-year time horizon from a third-party US payer perspective and estimated their health and cost outcomes based on pre-LT versus post-LT treatment with an all-oral DAA regimen. Transition probabilities and utilities were based on the literature and hepatologist consensus. Sustained virological response rates were sourced from ASTRAL-4, SOLAR-1, and SOLAR-2. Costs were sourced from RedBook, Medicare fee schedules, and published literature. In the HCC analysis, the pre-LT treatment strategy resulted in 11.48 per-patient quality-adjusted life years and $365,948 per patient lifetime costs versus 10.39 and $283,696, respectively, in the post-LT arm. In the DCC analysis, the pre-LT treatment strategy resulted in 9.27 per-patient quality-adjusted life years and $304,800 per patient lifetime costs versus 8.7 and $283,789, respectively, in the post-LT arm. As such, the pre-LT treatment strategy was found to be the most cost-effective in both populations with an incremental cost-effectiveness ratio of $74,255 (HCC) and $36,583 (DCC). Sensitivity and scenario analyses showed that results were most sensitive to the utility of patients post-LT, treatment sustained virological response rates, LT costs, and baseline Model for End-Stage Liver Disease score (DCC analysis only).
The timing of initiation of antiviral treatment for HCV patients with HCC or DCC relative to LT is an important area of clinical and policy research; our results indicate that pre-LT treatment with a highly effective, all-oral DAA regimen provides the best health outcomes and is the most cost-effective strategy for the treatment of HCV patients with HCC or DCC waitlisted for LT. (Hepatology 2017;66:46-56).
本研究旨在分析在等待肝移植（LT）的丙型肝炎病毒（HCV）患者中，与 LT 后治疗相比，LT 前使用全口服直接作用抗病毒药物（DAA）方案进行治疗的成本效益。
我们构建了等待 LT 的 HCC 患者和失代偿性肝硬化（DCC）患者 HCV 自然疾病进展的决策分析 Markov 模型。该模型遵循从第三方美国支付方角度出发的 1000 名平均年龄为 50 岁的假设队列，在 30 年的时间范围内，根据 LT 前与 LT 后使用全口服 DAA 方案治疗的情况，估计他们的健康和成本结果。转移概率和效用基于文献和肝病专家共识。持续病毒学应答率来自 ASTRAL-4、SOLAR-1 和 SOLAR-2。成本来自 RedBook、医疗保险费用表和已发表的文献。在 HCC 分析中，与 LT 后治疗组相比，LT 前治疗策略使每位患者的质量调整生命年增加了 11.48 年，每位患者终生成本增加了 365948 美元；在 DCC 分析中，与 LT 后治疗组相比，LT 前治疗策略使每位患者的质量调整生命年增加了 9.27 年，每位患者终生成本增加了 304800 美元。因此，LT 前治疗策略在这两种人群中均具有成本效益，增量成本效益比分别为 74255 美元（HCC）和 36583 美元（DCC）。敏感性和情景分析表明，结果对 LT 后患者的效用、治疗持续病毒学应答率、LT 成本和基线终末期肝病模型（仅适用于 DCC 分析）评分的影响最大。
HCV 合并 HCC 或 DCC 患者相对于 LT 开始抗病毒治疗的时机是临床和政策研究的重要领域；我们的研究结果表明，对于等待 LT 的 HCC 或 DCC 患者，LT 前使用高效全口服 DAA 方案治疗可提供最佳的健康结果，并且是治疗 HCV 合并 HCC 或 DCC 患者的最具成本效益的策略。（Hepatology 2017;66:46-56）。
