Introduction Given suboptimal performance of ultrasound-based surveillance for early hepatocellular carcinoma (HCC) detection in patients with cirrhosis, there is interest in alternative surveillance strategies, including blood-based biomarkers. We aimed to evaluate the cost-effectiveness of biomarker-based surveillance in patients with cirrhosis. Methods We constructed a decision-analytic model to compare ultrasound/alpha-fetoprotein (AFP) and biomarker-based surveillance strategies in 1,000,000 simulated patients with compensated cirrhosis. Model inputs for adherence, benefits, and harms of each strategy were based on literature review, and costs were derived from the Medicare fee schedule. Primary outcomes were quality-adjusted life-years (QALY) and incremental cost-effectiveness ratio (ICER) of the surveillance strategies, with cost-effectiveness assessed at a threshold of USD 150,000 per QALY. We performed sensitivity analyses for HCC incidence, test performance characteristics, surveillance adherence, and biomarker costs. Results In the base case, both ultrasound/AFP and biomarker-based surveillance were cost-effective versus no surveillance, with ICERs of USD 105,620, and USD 101,295, per QALY, respectively. Biomarker-based surveillance was also cost-effective versus ultrasound/AFP, with an ICER of USD 14,800 per QALY. Biomarker sensitivity exceeding 80%, cost below USD 210, or adherence exceeding 58% were necessary for biomarker-based screening to be cost-effective versus ultrasound/AFP. In two-way sensitivity analyses, biomarker costs were directly related with test sensitivity and adherence, whereas sensitivity and adherence were inversely related. In a probabilistic sensitivity analysis, biomarker-based screening was the most cost-effective strategy in most (65%) simulations. Conclusion Biomarker-based screening appears cost-effective for HCC screening, but results are sensitive to test sensitivity, adherence, and costs.

中文翻译：

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基于生物标志物的肝细胞癌筛查策略的肝硬化患者成本效益。


引言 鉴于基于超声的监测对肝硬化患者早期肝细胞癌 （HCC） 检测的性能不佳，人们对替代监测策略感兴趣，包括基于血液的生物标志物。我们旨在评估基于生物标志物的肝硬化患者监测的成本效益。方法 我们构建了一个决策分析模型，以比较 1,000,000 名模拟代偿期肝硬化患者的超声/甲胎蛋白 （AFP） 和基于生物标志物的监测策略。每种策略的依从性、益处和危害的模型输入基于文献综述，成本来自 Medicare 费用表。主要结局是监测策略的质量调整生命年 （QALY） 和增量成本效益比 （ICER），成本效益评估为每个 QALY 150,000 美元的阈值。我们对 HCC 发病率、检测性能特征、监测依从性和生物标志物成本进行了敏感性分析。结果 在基本情况下，超声/AFP 和基于生物标志物的监测与无监测相比都具有成本效益，每个 QALY 的 ICER 分别为 105,620 美元和 101,295 美元。与超声/AFP 相比，基于生物标志物的监测也具有成本效益，每个 QALY 的 ICER 为 14,800 美元。与超声/AFP 相比，基于生物标志物的筛查需要生物标志物敏感性超过 80%、成本低于 210 美元或依从性超过 58%，才能具有成本效益。在双向敏感性分析中，生物标志物成本与测试敏感性和依从性直接相关，而敏感性和依从性呈负相关。 在概率敏感性分析中，基于生物标志物的筛选是大多数 （65%） 模拟中最具成本效益的策略。结论 基于生物标志物的筛查对于 HCC 筛查似乎具有成本效益，但结果对测试敏感性、依从性和成本敏感。