Burke James F, Langa Kenneth M, Hayward Rodney A, Albin Roger L
Dept. of Neurology, University of Michigan, Ann Arbor, Michigan, United States of America; Robert Wood Johnson Clinical Scholars Program, University of Michigan, Ann Arbor, Michigan, United States of America; Center for Clinical Management Research, VAAAHS, Ann Arbor, Michigan, United States of America.
Dept. of Internal Medicine, University of Michigan, Ann Arbor, Michigan, United States of America; Center for Clinical Management Research, VAAAHS, Ann Arbor, Michigan, United States of America; Institute for Social Research, University of Michigan, Ann Arbor, Michigan, United States of America.
PLoS One. 2014 Dec 4;9(12):e114339. doi: 10.1371/journal.pone.0114339. eCollection 2014.
In this study, we developed a model of presymptomatic treatment of Alzheimer disease (AD) after a screening diagnostic evaluation and explored the circumstances required for an AD prevention treatment to produce aggregate net population benefit.
Monte Carlo simulation methods were used to estimate outcomes in a simulated population derived from data on AD incidence and mortality. A wide variety of treatment parameters were explored. Net population benefit was estimated in aggregated QALYs. Sensitivity analyses were performed by individually varying the primary parameters.
In the base-case scenario, treatment effects were uniformly positive, and net benefits increased with increasing age at screening. A highly efficacious treatment (i.e. relative risk 0.6) modeled in the base-case is estimated to save 20 QALYs per 1000 patients screened and 221 QALYs per 1000 patients treated.
Highly efficacious presymptomatic screen and treat strategies for AD are likely to produce substantial aggregate population benefits that are likely greater than the benefits of aspirin in primary prevention of moderate risk cardiovascular disease (28 QALYS per 1000 patients treated), even in the context of an imperfect treatment delivery environment.
在本研究中，我们在进行筛查诊断评估后建立了阿尔茨海默病（AD）症状前治疗模型，并探讨了AD预防治疗产生总体净人群效益所需的条件。
采用蒙特卡罗模拟方法，根据AD发病率和死亡率数据估计模拟人群的结果。探讨了各种治疗参数。用累计质量调整生命年（QALY）估计净人群效益。通过单独改变主要参数进行敏感性分析。
在基础病例情景中，治疗效果始终为阳性，净效益随筛查年龄的增加而增加。基础病例中模拟的一种高效治疗方法（即相对风险0.6）估计每筛查1000例患者可节省20个QALY，每治疗1000例患者可节省221个QALY。
即使在治疗实施环境不完善的情况下，AD的高效症状前筛查和治疗策略也可能产生显著的总体人群效益，这可能大于阿司匹林在中度风险心血管疾病一级预防中的效益（每治疗1000例患者28个QALY）。
