Singal Amit G, Chhatwal Jagpreet, Parikh Neehar, Tapper Elliot
Department of Internal Medicine, UT Southwestern Medical Center, Dallas, TX, USA.
Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Liver Cancer. 2024 Jun 18;13(6):643-654. doi: 10.1159/000539895. eCollection 2024 Dec.
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.
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.
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.
Biomarker-based screening appears cost-effective for HCC screening, but results are sensitive to test sensitivity, adherence, and costs.
鉴于基于超声的监测在肝硬化患者早期肝细胞癌（HCC）检测中的表现欠佳，人们对包括血液生物标志物在内的替代监测策略产生了兴趣。我们旨在评估基于生物标志物的监测在肝硬化患者中的成本效益。
我们构建了一个决策分析模型，以比较超声/甲胎蛋白（AFP）和基于生物标志物的监测策略在100万例模拟的代偿期肝硬化患者中的效果。每种策略的依从性、益处和危害的模型输入均基于文献综述，成本则来自医疗保险费用表。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），成本效益评估的阈值为每QALY 150,000美元。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。
在基础病例中，超声/AFP和基于生物标志物的监测与不进行监测相比均具有成本效益，ICER分别为每QALY 105,620美元和101,295美元。基于生物标志物的监测与超声/AFP相比也具有成本效益，ICER为每QALY 14,800美元。基于生物标志物的筛查要比超声/AFP具有成本效益，生物标志物敏感性需超过80%、成本低于210美元或依从性超过58%。在双向敏感性分析中，生物标志物成本与检测敏感性和依从性直接相关，而敏感性和依从性则呈负相关。在概率敏感性分析中，在大多数（65%）模拟中，基于生物标志物的筛查是最具成本效益的策略。
基于生物标志物的筛查对于HCC筛查似乎具有成本效益，但结果对检测敏感性、依从性和成本较为敏感。
