Incorporating individually defined brain health priorities in clinical trial outcomes: the electronic Person-Specific Outcome Measure approach in the United States

IF 4.9 Q1 CLINICAL NEUROLOGY

Alzheimer''s and Dementia: Translational Research and Clinical Interventions Pub Date : 2025-05-13 DOI:10.1002/trc2.70088

Stina Saunders, Ali Jannati, Shane Sheehan, Claudio Toro-Serey, Sean Tobyne, Killian McManus, David Bates, John Showalter, Álvaro Pascual-Leone

{"title":"Incorporating individually defined brain health priorities in clinical trial outcomes: the electronic Person-Specific Outcome Measure approach in the United States","authors":"Stina Saunders, Ali Jannati, Shane Sheehan, Claudio Toro-Serey, Sean Tobyne, Killian McManus, David Bates, John Showalter, Álvaro Pascual-Leone","doi":"10.1002/trc2.70088","DOIUrl":null,"url":null,"abstract":"<div>\n \n \n <section>\n \n <h3> INTRODUCTION</h3>\n \n <p>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.</p>\n </section>\n \n <section>\n \n <h3> METHODS</h3>\n \n <p>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 <i>U</i> test to compare the confidence ratings between participants with and without a self-reported diagnosis of neurodegenerative disease.</p>\n </section>\n \n <section>\n \n <h3> RESULTS</h3>\n \n <p>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, <i>U</i> = 8908, <i>p</i> < .01).</p>\n </section>\n \n <section>\n \n <h3> DISCUSSION</h3>\n \n <p>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.</p>\n </section>\n \n <section>\n \n <h3> Highlights</h3>\n \n <div>\n <ul>\n \n <li>The study captured individually defined treatment priorities.</li>\n \n <li>A self-reported confidence rating was used to assess how people manage their most meaningful areas of life</li>\n \n <li>The study found differences between US and UK participants’ priorities.</li>\n \n <li>Neurodegenerative disease is associated with lower confidence in managing priorities.</li>\n \n <li>The ePSOM tool offers a method to assess meaningful treatment benefits.</li>\n </ul>\n </div>\n </section>\n </div>","PeriodicalId":53225,"journal":{"name":"Alzheimer''s and Dementia: Translational Research and Clinical Interventions","volume":"11 2","pages":""},"PeriodicalIF":4.9000,"publicationDate":"2025-05-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/trc2.70088","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Alzheimer''s and Dementia: Translational Research and Clinical Interventions","FirstCategoryId":"1085","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1002/trc2.70088","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

INTRODUCTION

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.

METHODS

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 U test to compare the confidence ratings between participants with and without a self-reported diagnosis of neurodegenerative disease.

RESULTS

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, U = 8908, p < .01).

DISCUSSION

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.

Highlights

The study captured individually defined treatment priorities.
A self-reported confidence rating was used to assess how people manage their most meaningful areas of life
The 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.

Abstract Image

查看原文本刊更多论文

将个体定义的脑健康优先级纳入临床试验结果：美国电子个人特定结果测量方法

虽然美国已经批准了数量有限的阿尔茨海默病（AD）的疾病改善治疗，但在临床应用中采用这些治疗方法仍需谨慎。电子个人特异性结果测量（ePSOM）工具最近被开发出来，以确定除了改变潜在的阿尔茨海默病病理外，新的治疗方法是否在对个体最重要的领域发挥了足够的有益作用。方法我们进行了一项研究，以了解ePSOM英国研究的结果如何在美国应用。ePSOM美国调查（2023年5月至2024年1月）主要收集了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-means聚类）和卡方检验来检验美国和英国人群的答案之间的差异。我们使用Mann-Whitney U测试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级。结果：我们的分析纳入了764名美国参与者(68.8%为女性；74.2%高学历)，共有9010份自由文本回复，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，总共有38,056份回复。美国和英国人口的回答比例在统计学上存在显著差异。诊断组与未诊断组相比，自我报告自信的平均总分有显著差异(中位数得分21，四分位间距[IQR] = 17 ~ 23 vs中位数得分24，IQR = 22 ~ 25, U = 8908, p <；. 01)。我们的研究证明了美国个体层面的大脑健康优先级的异质性以及美国和英国人群之间的差异。诊断组在管理个人有意义的优先事项方面明显缺乏信心。这些发现支持了我们的假设，即什么构成有意义的治疗效益应该在个人而不是群体水平上确定，并且需要考虑文化背景。该研究捕获了个体定义的治疗优先级。一项自我报告的信心评级被用来评估人们如何管理生活中最有意义的领域。研究发现，美国和英国参与者在优先事项上存在差异。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的治疗效果的方法。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Alzheimer''s and Dementia: Translational Research and Clinical Interventions Medicine-Psychiatry and Mental Health

CiteScore

10.10

自引率

2.10%

发文量

134

审稿时长

10 weeks

期刊介绍： Alzheimer''s & Dementia: Translational Research & Clinical Interventions (TRCI) is a peer-reviewed, open access,journal from the Alzheimer''s Association®. The journal seeks to bridge the full scope of explorations between basic research on drug discovery and clinical studies, validating putative therapies for aging-related chronic brain conditions that affect cognition, motor functions, and other behavioral or clinical symptoms associated with all forms dementia and Alzheimer''s disease. The journal will publish findings from diverse domains of research and disciplines to accelerate the conversion of abstract facts into practical knowledge: specifically, to translate what is learned at the bench into bedside applications. The journal seeks to publish articles that go beyond a singular emphasis on either basic drug discovery research or clinical research. Rather, an important theme of articles will be the linkages between and among the various discrete steps in the complex continuum of therapy development. For rapid communication among a multidisciplinary research audience involving the range of therapeutic interventions, TRCI will consider only original contributions that include feature length research articles, systematic reviews, meta-analyses, brief reports, narrative reviews, commentaries, letters, perspectives, and research news that would advance wide range of interventions to ameliorate symptoms or alter the progression of chronic neurocognitive disorders such as dementia and Alzheimer''s disease. The journal will publish on topics related to medicine, geriatrics, neuroscience, neurophysiology, neurology, psychiatry, clinical psychology, bioinformatics, pharmaco-genetics, regulatory issues, health economics, pharmacoeconomics, and public health policy as these apply to preclinical and clinical research on therapeutics.