Executive Function Outcome Measures for Young Children with Down Syndrome

唐氏综合征(DS)是与智力相关的最常见的神经遗传学综合征 残疾，大约发生在1：691个活产婴儿中(Parker等人，2010年)。最近的方法 对DS和其他神经遗传性疾病的治疗已经开始集中在最早的部分 生命的长短。这种有针对性的早期治疗工作将需要高度敏感和 心理测量学结果指标对早期发育的影响 在DS中的轨迹。然而，患有DS的幼儿表现出表型特有的运动发育迟缓。 发展和早期沟通，这可能会混淆早期的可靠性和效度 执行功能(EF)关键认知领域的发展评估。因此， 需要对EF评估进行严格的心理测量验证，以促进治疗科学 患有DS的儿童。在这项研究中，210名患有DS的幼儿(年龄在2.5到7.99岁之间) 将在四波数据收集中进行评估，以建立可靠性、效度、内部 一致性，以及对拟议评估组合变化的敏感度。一套前景看好的 已选择实验室评估来衡量EF的各个方面。建议数 措施将最大限度地减少与表型相关的混淆，这些混淆威胁到可靠性和/或 DS患者早期认知功能评定的效度研究利用先进的定量分析方法 结构方程建模框架内的潜变量模型将用于 调查拟议措施的心理测量学特性。项目成果将 加强对患有DS的幼儿的治疗科学，并为关键的努力做出贡献 改善这一人群的发展成果。

Down syndrome (DS) is the most common neurogenetic syndrome associated with intellectual disability, occurring in approximately 1:691 live births (Parker et al., 2010). Recent approaches to treatment in DS and other neurogenetic disorders have begun to focus on the earliest part of the lifespan. This type of targeted early treatment work will require highly sensitive and psychometrically sound outcome measures to demonstrate effects on early developmental trajectories in DS. However, young children with DS show phenotype-specific delays in motor development and early communication, which could confound the reliability and validity of early developmental assessments in the critical cognitive domain of executive function (EF). Therefore, rigorous psychometric validation of EF assessments is needed to advance treatment science for young children with DS. In this study, 210 young children with DS (between 2.5 and 7.99 years) will be assessed at four waves of data collection to establish reliability, validity, internal consistency, and sensitivity to change in the proposed battery of assessments. A promising set of laboratory assessments has been selected to measure various aspects of EF. The proposed measures will minimize phenotype-related confounds that threaten the reliability and/or the validity of early cognitive assessment in DS. Advanced quantitative analytic approaches utilizing latent variable models within a structural equation modeling framework will be used to investigate the psychometric properties of the proposed measures. Project outcomes will strengthen treatment science for young children with DS and contribute to the critical effort to improve developmental outcomes in this population.