Cognitive Outcome Measures in School Age Children with Down Syndrome

学龄唐氏综合症儿童的认知结果测量

• 批准号: 10453735
• 负责人: Anna J. Esbensen
• 金额: $ 49.98万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-07-20 至 2024-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10453735
• 关键词: Accounting; Adaptive Behaviors; Address; Affect; Age; Area; Attention; Basic Science; Behavior; Behavioral; Caring; Characteristics; Child; Child Care; Chronology; Clinical; Clinical Trials; Cognition; Cognitive; Complex; Computers; Data; Development; Down Syndrome; Evaluation; Floor; Future; Gender; Genetic; Goals; Hippocampus (Brain); Human; Individual; Informal Social Control; Intellectual functioning disability; Intervention; Investigation; Language; Learning; Measurement; Measures; Medical; Memory; Methods; Motor; National Institute of Child Health and Human Development; Neuroanatomy; Neuropsychology; Outcome; Outcome Assessment; Outcome Measure; Outcome Study; Parents; Participant; Pathway interactions; Pattern; Performance; Pharmacologic Substance; Phenotype; Population; Positioning Attribute; Prefrontal Cortex; Problem behavior; Property; Psychometrics; Recommendation; Reporting; Research; Research Priority; School-Age Population; Short-Term Memory; Site; Testing; Time; Translational Research; United States; United States National Institutes of Health; Validity and Reliability; Variant; Work; base; behavioral phenotyping; care outcomes; clinical outcome measures; cognitive function; comorbidity; computerized; demographics; design; effectiveness trial; efficacy trial; emotional functioning; evidence base; executive function; follow-up; improved; individual variation; maladaptive behavior; member; memory process; mental age; mouse model; neurobehavioral; processing speed; skills; social; social cognition; sound; standardize measure; teacher; treatment trial; working group

PROJECT SUMMARY There is a fundamental gap in the availability of cognitive outcome measures that are reliable and sensitive to detecting change among children with Down syndrome (DS). Lack of such outcome measures represents an important problem to interpreting clinical trials aimed at improving the lives of individuals with DS. Without evidence-based cognitive outcome measures, future treatment trials in this population will remain suboptimal due to poor study measures. Despite some promising findings in currently recommended assessment batteries, the evidence base for cognitive outcome measures in DS is limited. Multi-site studies evaluating the reliability and validity of outcome measures are the needed next step towards supporting the evaluation of new pharmaceutical and clinical interventions for children with DS. Working groups convened by NICHD of leading experts in DS led to recommendations of promising measures for use in this population. The current study proposes to evaluate the psychometric properties of recommended promising measures and expands upon these recommendations by evaluating individual variability and accounting for the method of assessment (verbal, nonverbal, computer, parent-report) to support our understanding of performance within a cognitive domain. The overall objective of this application is to establish the psychometric properties of clinical outcome measures in children with DS across the cognitive domains of executive functioning (inhibition, working memory, set-shifting), learning and memory, and processing speed. Our rationale for working with this population is that DS is associated with a distinct pattern of cognitive strengths and weaknesses related to their neuroanatomy. Thus the selection of outcome measures to be evaluated needs to take into account the DS behavioral phenotype. We propose three specific aims: 1) To examine the psychometric properties of measures in the assessment battery with children with DS. 2) To evaluate differences in the psychometric properties of the measures as a function of variations in demographics and medical comorbidities. 3) To characterize the developmental trajectories of cognition and executive functioning. To achieve these aims, 160 children ages 6-17 years with DS will participate in repeated neuropsychological assessments with follow-up evaluations at 2 weeks, 3 months, 6 months, and 1 year. Parents and teachers will complete ratings of executive functioning, adaptive behavior and behavioral concerns. Our research team is uniquely positioned to conduct this work, combining expertise in DS, neuropsychological assessment, medical conditions and measurement evaluation. We anticipate that this measurement study will provide critical guidance for future efficacy and effectiveness trials. Our goals are in line with NICHD PAR-16-216 research priority to assess the reliability, validity and change over time of measures for use in clinical trials in ID, in this case specifically targeted to DS. As our field continues to develop new pharmaceutical and clinical interventions, our project will support this work and have an impact on the 400,000 individuals with DS living in the United States.

项目概要 可靠且对认知结果敏感的认知结果测量的可用性存在根本性差距 检测唐氏综合症 (DS) 儿童的变化。缺乏此类成果衡量标准意味着 解释旨在改善 DS 患者生活的临床试验的一个重要问题。没有 基于证据的认知结果测量，未来对该人群的治疗试验仍将不理想 由于学习措施不力。尽管目前推荐的评估中有一些有希望的发现 电池，DS 认知结果测量的证据基础是有限的。多地点研究评估 结果测量的可靠性和有效性是支持新的评估的下一步所需的步骤 DS 儿童的药物和临床干预措施。 NICHD 领导召集的工作组 DS 专家提出了针对这一人群使用的有希望的措施的建议。目前的研究 提议评估推荐的有希望的措施的心理测量特性，并扩展 通过评估个体差异并考虑评估方法来提出这些建议 （语言、非语言、计算机、家长报告）以支持我们对认知范围内表现的理解 领域。该应用程序的总体目标是建立临床结果的心理测量特性 对 DS 儿童执行功能认知领域（抑制、工作 记忆、设定转换）、学习和记忆以及处理速度。我们开展此工作的理由 总体而言，DS 与与其相关的认知优势和劣势的独特模式有关。 神经解剖学。因此，选择要评估的结果指标需要考虑 DS 行为表型。我们提出三个具体目标：1）检查心理测量特性 DS 儿童评估电池的措施。 2）评估心理测量的差异 测量的特性作为人口统计和医疗合并症变化的函数。 3) 至 描述认知和执行功能的发展轨迹。为了实现这些目标，160 6-17 岁患有 DS 的儿童将参加重复的神经心理学评估和随访 2周、3个月、6个月和1年时进行评估。家长和老师将完成评分 执行功能、适应性行为和行为问题。我们的研究团队具有独特的定位 结合 DS、神经心理学评估、医疗条件和 测量评估。我们预计这项测量研究将为未来提供重要指导 功效和有效性试验。我们的目标与 NICHD PAR-16-216 研究优先级一致，以评估 ID 临床试验中使用的措施的可靠性、有效性和随时间的变化，在本例中具体为 针对 DS。随着我们的领域不断开发新的药物和临床干预措施，我们的项目将 支持这项工作并对生活在美国的 400,000 名 DS 患者产生影响。

项目成果

Psychometric Evaluation of Social Cognition and Behavior Measures in Children and Adolescents with Down Syndrome.

• DOI: 10.3390/brainsci11070836
• 发表时间: 2021-06-24
• 期刊: Brain sciences
• 影响因子: 3.3
• 作者: Schworer EK;Hoffman EK;Esbensen AJ
• 通讯作者: Esbensen AJ

Evaluating working memory outcome measures for children with Down syndrome.

• DOI: 10.1111/jir.12833
• 发表时间: 2022-01
• 期刊: Journal of intellectual disability research : JIDR
• 作者: Schworer EK;Esbensen AJ;Fidler DJ;Beebe DW;Carle A;Wiley S
• 通讯作者: Wiley S

Evaluating Verbal Fluency Outcome Measures in Children With Down Syndrome.

• DOI: 10.1352/1944-7558-127.4.328
• 发表时间: 2022-07-01
• 期刊: AJIDD-AMERICAN JOURNAL ON INTELLECTUAL AND DEVELOPMENTAL DISABILITIES
• 影响因子: 2.1
• 作者: Smeyne, Catelyn N.;Esbensen, Anna J.;Schworer, Emily K.;Belizaire, Shequanna;Hoffman, Emily K.;Beebe, Dean W.;Wiley, Susan
• 通讯作者: Wiley, Susan

Short-term memory outcome measures: Psychometric evaluation and performance in youth with Down syndrome.

• DOI: 10.1016/j.ridd.2021.104147
• 发表时间: 2022-01
• 期刊: Research in developmental disabilities
• 影响因子: 3.1
• 作者: Schworer EK;Voth K;Hoffman EK;Esbensen AJ
• 通讯作者: Esbensen AJ

Semantic Verbal Fluency in Youth with Down Syndrome: Analysis of Conventional and Contextual Cluster Formation.

• DOI: 10.3390/brainsci12010009
• 发表时间: 2021-12-23
• 期刊: Brain sciences
• 影响因子: 3.3
• 作者: Schworer EK;Belizaire S;Hoffman EK;Esbensen AJ
• 通讯作者: Esbensen AJ