Expressive Language Sampling as an Outcome Measure

 描述（由申请人提供）：R 01 HD 074346的拟议修订旨在将该项目扩展到一种新的疾病：自闭症谱系障碍（ASD）。正在开发有前途的新的药理学和行为治疗方法，用于许多智力和发育障碍（IDD），包括ASD。然而，由于缺乏足够的结果测量，这些治疗的评价受到阻碍。从2013年1月3日开始，我们的团队获得资助，以评估表达性语言采样（ELS）是否足以获得结果指标。在ELS程序中，口语样本是在高度结构化但自然的互动中收集的。样本进行了分析，以得出反映语言技能和非典型语言行为的重要方面的结果措施。ELS程序产生临床相关和功能终点，捕获IDD常见的语言障碍以及特定IDD特有的障碍，并在典型和语言障碍人群中产生发育变化的稳健指标。在已经资助的项目中，我们专注于患有脆性X综合征（FXS）和唐氏综合征（DS）的儿童，青少年和年轻人。在具体目标方面，我们（1）检查ELS程序的基本心理测量学特性，包括重测信度、内部一致性、效度和敏感性：（2）评估ELS程序的心理测量学特性的差异，作为参与者病因、年龄、性别、自闭症症状严重程度和智商的函数;（3）比较三种不同ELS情境（对话、叙述和自闭症诊断观察表-2）的心理测量学特性;（4）评估在多个地点实施ELS程序的可行性。使用ELS材料的替代版本在4周（+/- 1周）时评估重测信度。通过计算抽样范围内和抽样范围之间的阿尔法系数，评估内部一致性。常模参照标准化测验和被试报告是结构效度的指标。纵向随访将产生一个相对于现有措施的变化敏感性的估计。在本次修订中，我们建议增加ASD参与者，这是一个与FXS和DS重叠的人群（例如，许多患有ASD的个体具有共病的智力残疾）。然而，与此同时，ASD与比FXS或DS更广泛的语言和认知能力相关，这使得识别有效结果测量的任务更具挑战性。ASD参与者将在两个新的地点（明尼苏达大学和华盛顿大学）以及加州大学戴维斯分校进行测试。收集的数据将与已资助项目的所有四个具体目标相关，但对ELS程序的普遍性提供了更全面的评估，并增加了ASD，这是一种非常普遍的IDD疾病，迫切需要新的工具来评估治疗效果。

 DESCRIPTION (provided by applicant): The proposed revision to R01HD074346 is designed to extend the project to a new disorder: autism spectrum disorder (ASD). Promising new pharmacological and behavioral treatments are being developed for numerous intellectual and developmental disabilities (IDD), including ASD. Evaluation of these treatments, however, is hampered by a lack of adequate outcome measures. Beginning 03-01-2013, our team was funded to evaluate the adequacy of expressive language sampling (ELS) for deriving outcome measures. In ELS procedures, samples of spoken language are collected in highly structured, yet naturalistic, interactions. The samples are analyzed to derive outcome measures reflecting important dimensions of language skill and atypical language behavior. ELS procedures yield clinically relevant and functional endpoints, capture language impairments common to IDD as well as impairments specific to particular IDDs, and yield robust indicators of developmental change within typical and language-impaired populations. In the already funded project, we are focused on children, adolescents, and young adults with fragile X syndrome (FXS) and Down syndrome (DS). In terms of specific aims, we are (1) examining the basic psychometric properties of measures derived from ELS procedures, including test-retest reliability, internal consistency, validity, and sensitivity; (2) evaluating differences in the psychometric properties o ELS procedures as a function of variations in participant etiology, age, gender, autism symptom severity, and IQ; (3) comparing the psychometric properties of three different ELS contexts (conversation, narration, and the Autism Diagnostic Observation Schedule-2); and (4) evaluating the feasibility of implementing ELS procedures across multiple sites. Test-retest reliability is being assessed at 4 weeks (+/- 1 week) using alternate versions of ELS materials. Internal consistency is being assessed by computing alpha coefficients within and across sampling contexts. Norm-referenced standardized tests and informant report are the indicators of construct validity. Longitudinal follow-up will yield an estimate of sensitivity to change relatve to existing measures. In this revision, we propose to add participants with ASD, which is a population that overlaps with FXS and DS (e.g., many individuals with ASD have a comorbid intellectual disability). At the same time, however, ASD is associated with a wider range of language and cognitive abilities than is FXS or DS, which has made the task of identifying valid outcome measures more challenging. ASD participants will be tested at two new sites (University of Minnesota and University of Washington), as well as at UC Davis. The data collected will be relevant to all four specific aims of the already funded project, but offers a fuler evaluation of the generalizability of ELS procedures and adds ASD, a highly prevalent IDD condition in desperate need of new tools for evaluating treatment efficacy.