Change-sensitive Measurement of Adult Functional Outcomes in Developmental Disabilities

发育障碍成人功能结果的变化敏感测量

• 批准号: 10565683
• 负责人: SHAUN M EACK
• 金额: $ 59.2万
• 依托单位: UNIVERSITY OF PITTSBURGH AT PITTSBURGH
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-05-19 至 2025-02-28
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10565683
• 关键词: Adaptive Behaviors; Adult; Age; Calibration; Caregivers; Categories; Child; Childhood; Clinical; Clinical Trials; Communities; Comprehension; Development; Developmental Disabilities; Diagnosis; Dimensions; Down Syndrome; Employment; Ensure; Equipment and supply inventories; Evaluation; Evidence based treatment; Factor Analysis; Family; Fragile X Syndrome; Group Homes; Independent Living; Individual; Information Systems; Inpatients; Intellectual functioning disability; Intervention; Intervention Studies; Knowledge; Life; Loneliness; Measurement; Measures; Methodology; Methods; Modeling; Monitor; Mood Disorders; Neurodevelopmental Disorder; Occupations; Outcome; Outcome Measure; Outpatients; Parents; Participant; Patient Outcomes Assessments; Patient Self-Report; Patients; Population; Property; Proxy; Psychometrics; Public Health; Reporter; Reporting; Research; Sampling; Schizophrenia; Severities; Social Functioning; Social isolation; Societies; Symptoms; Testing; Time; Underserved Population; United States National Institutes of Health; Validity and Reliability; adult with autism spectrum disorder; autism spectrum disorder; biological sex; cognitive interview; design; disability; emotion dysregulation; experience; functional disability; functional improvement; functional outcomes; improved; neuropsychiatry; novel; response; service providers; skills; social; social deficits; sound; success; theories; therapy development; treatment effect; treatment trial; verbal

7. PROJECT SUMMARY/ABSTRACT Developmental disabilities (DD), including autism spectrum disorder (ASD), Down syndrome, Fragile X syndrome, and other intellectual and developmental disabilities are heterogeneous neurodevelopmental disorders that place considerable burden on individuals, families, and society. Although most research on DD has focused on children, these conditions are life-long with few established treatments to support functioning in adulthood. As a result, many adults with DD experience significant functional impairment represented by low rates of employment, severe social dysfunction and isolation, and a limited ability to live independently and experience autonomy in adult life. The development of interventions to improve adult functional outcomes in social, employment, and independent living domains across DD has lagged far behind those developed for children. A key factor limiting the development of treatments to improve adult functioning in DD is the lack of validated assessments of functional outcome applicable to adulthood. Current studies either use measures relevant to childhood with limited applicability to and treatment sensitivity in adults, or fail to assess this important domain, greatly restricting knowledge on how adult functioning can be improved in DD. We have shown in preliminary studies with adults with ASD that it is possible to develop measures of functional outcome in adulthood that have greater validity and are more sensitive to treatment effects than existing measures adapted from childhood. In response to this major gap in adult outcome measurement in DD and PAR-18-039, “Outcome Measures for Use in Treatment Trials of Individuals with Intellectual and Developmental Disabilities”, this project proposes to use NIH PROMIS methods to develop and validate proxy- and self-report versions of the Adult Functioning Scale (AFS) for assessing functional outcomes in social, employment, and independent living domains in adults with DD. A pool of potential items will be generated based on our conceptual model, functional outcome measures used in other populations, and input from expert and stakeholder panels. This item pool will then be completed by two calibration samples: Proxy reporters (e.g., parents, clinicians, group home staff) for 1000 adults with DD representative of the full range of verbal and intellectual functioning in DD and 1000 self-reporting adults with DD (N = 500 with ASD, N = 500 with other DD). Advanced psychometric analytics employing exploratory and confirmatory factor analysis and item response theory models will be used to create final calibrated item banks (and static short forms) of the AFS suitable for broad use in clinical trials across heterogeneous DD. The reliability and validity of the AFS caregiver and self-report versions will be examined in the calibration samples, along with a 4-week retest subsample (N = 200). Sensitivity to treatment- related changes will be assessed in longitudinal intervention studies of inpatient and outpatient samples of adults with DD. The results will validate the first measure of functional outcome for use in clinical trials in adult DD and will pave the way for treatment advances to improve functioning in this underserved population.

7。项目摘要/摘要 发育障碍（DD），包括自闭症谱系障碍（ASD），唐氏综合症，脆弱x 综合征以及其他智力和发育障碍是异质性神经发育 尽管大多数DD研究 这些条件专注于儿童，是终身的，很少有既定的治疗方法来支持功能 成年。结果，许多DD的成年人经历了较低的功能障碍 就业率，严重的社会功能障碍和孤立以及独立生活的能力有限 在成人生活中体验自治。开发干预措施以改善成人功能结果 DD各地的社会，就业和独立生活领域远远落后于为 孩子们。限制治疗发展以改善DD成人功能的关键因素是缺乏 经过验证的适用于成年的功能结果的评估。当前的研究要么使用措施 与童年有关，适用于成人的适用性和治疗敏感性有限，或者无法评估这一点 重要的领域，极大地限制了DD中如何改善成人功能的知识。我们有 在与ASD的成年人的初步研究中显示了可以开发功能结果的测量值 在成年后具有更大的有效性，对治疗效果更敏感 从童年开始改编。响应DD和PAR-18-039中成人结局测量中的这一主要差距， “在智力和发育障碍的人的治疗试验中使用的结果措施”， 该项目建议使用NIH Promis方法来开发和验证代理和自我报告版本的 成人功能量表（AFS）用于评估社会，就业和独立的功能结果 DD成人的生活领域。根据我们的概念模型，将生成一批潜在项目 其他人群中使用的功能结果度量以及专家和利益相关者面板的投入。这 然后，项目池将通过两个校准样本完成：代理记者（例如，父母，临床医生，小组 家庭工作人员）1000名DD成年人，代表DD中的各种口头和智力功能 和1000名具有DD的自我报告的成年人（n = 500，ASD，n = 500与其他DD）。高级心理测量学 将使用采用探索性和确认因素分析和项目响应理论模型的分析（分析） 创建最终校准的物品库（和静态的简短形式），适用于临床试验的广泛使用 跨越异质的DD。 AFS护理人员和自我报告版本的可靠性和有效性将是 在校准样品中检查，以及4周的重新测试子样本（n = 200）。对治疗的敏感性 相关变化将在对住院和门诊样本的纵向干预研究中进行评估 成人DD。结果将验证在成人临床试验中使用功能结果的首次测量 DD并将为治疗方面的发展铺平道路，以改善服务不足的人群的功能。