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. 项目概要/摘要 发育障碍 (DD)，包括自闭症谱系障碍 (ASD)、唐氏综合症、脆性 X 病 综合征以及其他智力和发育障碍是异质性神经发育障碍 给个人、家庭和社会带来沉重负担的疾病。尽管大多数研究都是关于 DD 重点关注儿童，这些病症是终生的，几乎没有既定的治疗方法来支持功能 成年期。结果，许多患有 DD 的成年人经历了严重的功能障碍，表现为低 就业率、严重的社会功能失调和孤立以及独立生活和生活能力有限 体验成年生活的自主性。开发改善成人功能结果的干预措施 DD 的社会、就业和独立生活领域远远落后于为 孩子们。限制开发改善 DD 成人功能的治疗方法的一个关键因素是缺乏 适用于成年期的功能结果的经过验证的评估。目前的研究要么使用措施 与童年相关，但对成人的适用性和治疗敏感性有限，或未能评估这一点 重要领域，极大地限制了如何在 DD 中改善成人功能的知识。我们有 对患有自闭症谱系障碍的成年人进行的初步研究表明，开发功能结果的衡量标准是可能的 在成年后，与现有措施相比，这些措施具有更高的有效性，并且对治疗效果更敏感 从小就改编的。针对 DD 和 PAR-18-039 中成人结果测量的这一重大差距， “智力和发育障碍人士治疗试验中使用的结果衡量标准”， 该项目建议使用 NIH PROMIS 方法来开发和验证代理报告版本和自我报告版本 成人功能量表 (AFS)，用于评估社交、就业和独立方面的功能结果 DD 成人的生活领域。将根据我们的概念模型生成潜在项目池， 其他人群中使用的功能性成果衡量标准，以及专家和利益相关者小组的投入。这 然后，项目池将由两个校准样本完成：代理报告者（例如，父母、临床医生、团体 家庭工作人员）针对 1000 名患有 DD 的成年人，代表了 DD 的全部言语和智力功能 以及 1000 名自我报告患有 DD 的成年人（N = 500 患有 ASD，N = 500 患有其他 DD）。高级心理测量学 将使用采用探索性和验证性因素分析以及项目响应理论模型的分析 创建适合在临床试验中广泛使用的 AFS 最终校准项目库（和静态简短形式） 跨异构DD。 AFS 护理人员和自我报告版本的可靠性和有效性将 在校准样品中进行检查，以及 4 周重新测试子样品 (N = 200)。对治疗的敏感性- 相关变化将在住院和门诊样本的纵向干预研究中进行评估 患有 DD 的成人。结果将验证用于成人临床试验的第一个功能结果测量 DD 将为治疗进步铺平道路，以改善这一服务不足人群的功能。