Saunders Stina, Jannati Ali, Sheehan Shane, Toro-Serey Claudio, Tobyne Sean, McManus Killian, Bates David, Showalter John, Pascual-Leone Álvaro
Linus Health, Inc. Boston Massachusetts USA.
Usher Institute University of Edinburgh Edinburgh UK.
Alzheimers Dement (N Y). 2025 May 13;11(2):e70088. doi: 10.1002/trc2.70088. eCollection 2025 Apr-Jun.
While a limited number of disease-modifying treatments for Alzheimer's disease (AD) have been approved in the United States, there is caution in adopting these treatments in clinical use. The electronic Person-Specific Outcome Measure (ePSOM) tool was recently developed to establish whether, besides modifying underlying AD pathology, new treatments exerted sufficient beneficial effects in areas that matter the most to an individual.
We conducted a study to understand how findings from the ePSOM UK study applied in the United States. The ePSOM US survey (May 2023 to January 2024) collected primarily free-text responses in personally defined brain health priorities, alongside self-reported confidence in managing these priorities. We used natural language processing (k-means clustering of GloVe vectors) and chi-squared tests to examine differences between the US and UK populations' answers. We used a Mann-Whitney test to compare the confidence ratings between participants with and without a self-reported diagnosis of neurodegenerative disease.
Our analysis included 764 participants in the United States (68.8% female; 74.2% high educational attainment) with a total of 9010 free-text responses, of whom 53 individuals (6.90%) reported neurodegenerative disease diagnosis. The comparable sample from the UK survey included 4529 participants with a total of 38,056 responses. There were statistically significant differences in the proportion of responses between the US and UK populations. The diagnosis group showed a significant difference in average total scores of self-reported confidence compared with those without a diagnosis (median score 21, interquartile range [IQR] = 17 to 23 vs median score 24, IQR = 22 to 25, = 8908, < .01).
Our study demonstrates heterogeneity in individual-level brain health priorities in the US and differences between the US and UK populations. The diagnosis group was significantly less confident in managing personally meaningful priorities. These findings support our hypothesis that what constitutes meaningful treatment benefits should be determined at an individual rather than group level, and cultural context needs to be considered.
The study captured individually defined treatment priorities.A self-reported confidence rating was used to assess how people manage their most meaningful areas of lifeThe study found differences between US and UK participants' priorities.Neurodegenerative disease is associated with lower confidence in managing priorities.The ePSOM tool offers a method to assess meaningful treatment benefits.
虽然美国已批准了有限数量的用于治疗阿尔茨海默病（AD）的疾病修饰疗法，但在临床应用中采用这些疗法时需谨慎。最近开发了电子个人特定结果测量（ePSOM）工具，以确定除了改变AD潜在病理外，新疗法是否在对个体最重要的领域产生了足够的有益效果。
我们开展了一项研究，以了解ePSOM英国研究的结果如何应用于美国。ePSOM美国调查（2023年5月至2024年1月）主要收集了关于个人定义的大脑健康优先事项的自由文本回复，以及自我报告的管理这些优先事项的信心。我们使用自然语言处理（GloVe向量的k均值聚类）和卡方检验来检查美国和英国人群答案之间的差异。我们使用曼-惠特尼检验来比较有和没有自我报告神经退行性疾病诊断的参与者之间的信心评分。
我们的分析纳入了美国的764名参与者（68.8%为女性；74.2%具有高学历），共有9010条自由文本回复，其中53人（6.90%）报告了神经退行性疾病诊断。英国调查的可比样本包括4529名参与者，共有38056条回复。美国和英国人群的回复比例存在统计学上的显著差异。诊断组与未诊断组相比，自我报告信心的平均总分存在显著差异（中位数分数21，四分位间距[IQR]=17至23，而中位数分数24，IQR=22至25，Z=8908，P<0.01）。
我们的研究表明美国个体层面大脑健康优先事项存在异质性，以及美国和英国人群之间存在差异。诊断组在管理个人有意义的优先事项方面信心明显较低。这些发现支持了我们的假设，即构成有意义治疗益处的因素应在个体而非群体层面确定，并且需要考虑文化背景。
该研究获取了个人定义的治疗优先事项。使用自我报告的信心评分来评估人们如何管理他们生活中最有意义的领域。该研究发现美国和英国参与者的优先事项存在差异。神经退行性疾病与管理优先事项的信心较低有关。ePSOM工具提供了一种评估有意义治疗益处的方法。
