Early childhood communication outcome measures for DS

项目总结/摘要 据报道，各种药物治疗都能成功地挽救或部分挽救， 唐氏综合征小鼠模型Ts 65 Dn中的学习和记忆缺陷。这些进步导致人们呼吁 为DS制定更强大的国家转化研究议程。然而，对疗效的评价 DS的靶向治疗由于缺乏已被验证的结果指标而受到阻碍， DS.对于生命的最初几年，对DS的有效结局指标的需求尤其迫切，因为， 据推测，开始早的干预措施最有可能产生一连串积极的事件 可以累积、普及、长时间的激光效益。不幸的是，这些措施实际上 在学龄前不存在。在本项目中，我们建议评估可行性， 在自然语言学中收集的各种前语言和口语度量的心理测量特性 背景，已用于研究和临床实践，以跟踪发展的改善，在一个 儿童人群的数量，用于治疗研究，重点是年轻的DS。重点 语言和沟通作为DS治疗研究的终点特别有吸引力， 这些领域的困难是DS表型最显著的特征。本课题 建议（1）评估五种语言前沟通技能的心理测量特性，（2） 评估早期口语技能的四个指标的心理测量特性，以及（3）表征 这些发展轨迹的各种指标的前语言和口语在幼儿与 通过横截面的方法。这些目标将通过收集两个自然的解决 来自2.50-7.99岁DS儿童的沟通样本：早期社会沟通量表 （ESCS）和一个半结构化的发挥会议与考官。从样本中得出的测量结果将包括 那些索引语言前交流的（例如，ESCS启动共同注意，启动行为 请求和发起社会互动评分;沟通复杂性量表评分;表达性 沟通指数-加权沟通得分）和口语（例如，多话，词汇 多样性、句法复杂性、可理解性）。将在2周（+/-3天）内评估重测信度。 标准化测量将被用作结构效度的指标。1年的纵向随访将产生 对各种措施变化的相对敏感性的估计。多地点实施的可行性 将通过在关键心理测量指标上比较各研究中心的样本进行评估。发育 这些指标的轨迹将通过描述作为受试者特征函数的表现来探索 (e.g.,实足年龄、非言语认知和言语认知）。

Project Summary/Abstract A diverse array of pharmaceutical treatments has been reported to successfully rescue, or partially rescue, learning and memory deficits in the Down syndrome mouse model Ts65Dn. These advances have led to calls for a more robust national translational research agenda for DS. However, the evaluation of the efficacy of targeted treatments for DS has been hampered by a lack of outcome measures that have been validated for DS. The need for validated outcome measures for DS is particularly acute for the earliest years of life because, presumably, interventions that begin early have the greatest potential to create a cascade of positive events that can have cumulative, pervasive, long-lasing benefits. Unfortunately, such measures are virtually nonexistent prior to the school-age years. In the present project, we propose to evaluate the feasibility and psychometric properties of a variety of prelinguistic and spoken language metrics collected in naturalistic contexts, which have been used in research and clinical practice to track developmental improvements in a number of populations of children, for use in treatment studies focused on young with DS. The focus on language and communication as endpoints for treatment studies of DS is particularly attractive given that difficulties in these domains are among the most salient features of the DS phenotype. In this project, we propose to (1) evaluate the psychometric properties of five metrics of prelinguistic communication skills, (2) evaluate the psychometric properties of four metrics of early spoken language skills, and (3) characterize the developmental trajectories of these various metrics of prelinguistic and spoken language in young children with DS through cross-sectional approaches. These aims will be addressed by collecting two naturalistic communication samples from children with DS aged 2.50-7.99 years: Early Social Communication Scales (ESCS) and a semi-structured play session with an examiner. Measures derived from the samples will include those indexing prelinguistic communication (e.g., ESCS Initiating Joint Attention, Initiating Behavioral Requests, and Initiating Social Interaction scores; Communication Complexity Scale score; Expressive Communication Index-Weighted Communication score) and spoken language (e.g., talkativeness, lexical diversity, syntactic complexity, intelligibility). Test-retest reliability will be assessed over 2 weeks (+/-3 days). Standardized measures will be used as indicators of construct validity. A 1-year longitudinal follow-up will yield an estimate of relative sensitivity to change of the various measures. Feasibility of multiple-site implementation will be evaluated by comparing samples across sites on key psychometric indicators. The developmental trajectories of these metrics will be explored by describing performance as a function of subject characteristics (e.g., chronological age, nonverbal cognition, and verbal cognition).