Michaud Tzeyu L, Kane Robert L, McCarten J Riley, Gaugler Joseph E, Nyman John A, Kuntz Karen M
Center for Reducing Health Disparities, College of Public Health, University of Nebraska Medical Center, Omaha, NE, USA.
Department of Health Promotion, Social and Behavioral Health, College of Public Health, University of Nebraska Medical Center, Omaha, NE, USA.
Pharmacoecon Open. 2018 Sep;2(3):309-323. doi: 10.1007/s41669-017-0054-z.
Cerebrospinal fluid (CSF) biomarkers are shown to facilitate a risk identification of patients with mild cognitive impairment (MCI) into different risk levels of progression to Alzheimer's disease (AD). Knowing a patient's risk level provides an opportunity for earlier interventions, which could result in potential greater benefits. We assessed the cost effectiveness of the use of CSF biomarkers in MCI patients where the treatment decision was based on patients' risk level.
We developed a state-transition model to project lifetime quality-adjusted life-years (QALYs) and costs for a cohort of 65-year-old MCI patients from a US societal perspective. We compared four test-and-treat strategies where the decision to treat was based on a patient's risk level (low, intermediate, high) of progressing to AD with two strategies without testing, one where no patients were treated during the MCI phase and in the other all patients were treated. We performed deterministic and probabilistic sensitivity analyses to evaluate parameter uncertainty.
Testing and treating low-risk MCI patients was the most cost-effective strategy with an incremental cost-effectiveness ratio (ICER) of US$37,700 per QALY. Our results were most sensitive to the level of treatment effectiveness for patients with mild AD and for MCI patients. Moreover, the ICERs for this strategy at the 2.5th and 97.5th percentiles were US$18,900 and US$50,100 per QALY, respectively.
Based on the best available evidence regarding the treatment effectiveness for MCI, this study suggests the potential value of performing CSF biomarker testing for early targeted treatments among MCI patients with a narrow range for the ICER.
脑脊液生物标志物已被证明有助于将轻度认知障碍（MCI）患者进展为阿尔茨海默病（AD）的不同风险水平进行风险识别。了解患者的风险水平为早期干预提供了机会，这可能带来更大的潜在益处。我们评估了在MCI患者中使用脑脊液生物标志物的成本效益，其中治疗决策基于患者的风险水平。
我们建立了一个状态转换模型，从美国社会视角预测一组65岁MCI患者的终身质量调整生命年（QALY）和成本。我们比较了四种检测与治疗策略，其中治疗决策基于患者进展为AD的风险水平（低、中、高），与两种不进行检测的策略，一种是在MCI阶段不治疗任何患者，另一种是所有患者都接受治疗。我们进行了确定性和概率性敏感性分析，以评估参数不确定性。
检测并治疗低风险MCI患者是最具成本效益的策略，每获得一个QALY的增量成本效益比（ICER）为37,700美元。我们的结果对轻度AD患者和MCI患者的治疗效果水平最为敏感。此外，该策略在第2.5百分位数和第97.5百分位数时的ICER分别为每QALY 18,900美元和50,100美元。
基于关于MCI治疗效果的现有最佳证据，本研究表明在ICER范围较窄的MCI患者中进行脑脊液生物标志物检测以进行早期靶向治疗具有潜在价值。
