Expressive Language Sampling as an Outcome Measure

描述(由申请人提供)：为智障人士提供的新治疗方法(ID)越来越多地针对具体情况。针对脆性X综合征(FXS)和唐氏综合征(DS)的许多靶向药物的临床试验目前正在进行中。针对具体情况的行为治疗也在不断涌现。由于缺乏足够的认知和行为终点，对所有这些治疗方法的评估都受到了阻碍。在这个项目中，我们建议评估表达语言样本对于得出与语言相关的临床终点的充分性。在这个过程中，富有表现力的语言样本是在高度结构化和脚本化的、但又自然的互动中收集的。然后这些样本可以 被分析以得出反映语言技能和非典型语言行为的重要维度的临床终点。尽管表现性语言采样对FXS或DS(更广泛地说是ID)的临床试验的适用性尚未确定，但该程序特别有希望，因为它们产生了临床相关和功能终点，已被证明捕获了ID常见的以及FXS或DS特有的损害，并已被证明在典型和其他语言障碍人群中产生了强有力的发育变化指标。在这个项目中，我们建议：(1)检验来自表达语言采样技术的测量的基本心理测量学特性，包括建立它们的重测信度、内部一致性、效度和敏感度；(2)评估表达语言采样技术的心理测量特性随被试的病因、年龄、性别、自闭症症状严重程度和ID水平的变化而存在的差异；(3)比较三种不同表达语言采样技术的心理测量特性；以及(4)评估典型临床试验所要求的跨多个地点实施表达语言采样的可行性。这些目标将通过收集儿童、青少年和患有FXS或DS的年轻人的富有表现力的语言样本来实现。样本将以三种互动形式收集：对话、叙述和构成自闭症诊断观察计划(ADOS)的结构化互动。从样本中得出的衡量标准将包括那些索引句法(DS中损害特别严重的领域)和持久化(FXS中损害特别严重的领域)。重测信度将在4周(+/-1周)使用不同版本的抽样材料进行评估。内部一致性将通过在抽样技术内部和跨抽样技术计算阿尔法系数来评估。标准化测试和举报人报告将作为有效性的指标。两年的纵向跟踪将产生对变化敏感性的估计。参与者将在多个地点接受测试，每个地点都有相当丰富的评估FXS或DS患者的经验。将通过比较各地点关键指标的语言样本来评估多地点实施的可行性。转录、编码和分析将只在加州大学戴维斯分校心智研究所进行。

DESCRIPTION (provided by applicant): New treatments for people with intellectual disabilities (ID) are increasingly condition-specific in nature. Numerous clinical trials of targeted pharmacological agents are now in process for fragile X syndrome (FXS) and Down syndrome (DS). Condition-specific behavioral treatments also are emerging. Evaluation of all such treatments is being hampered by a lack of adequate cognitive and behavioral endpoints. In this project, we propose to evaluate the adequacy of expressive language sampling for deriving language-relevant clinical endpoints. In this procedure, expressive language samples are collected in highly structured and scripted, yet naturalistic, interactions. These samples can then be analyzed to derive clinical endpoints reflecting important dimensions of language skill and atypical language behavior. Although the suitability of expressive language sampling for clinical trials with FXS or DS (or ID more generally) has yet to be determined, the procedures are especially promising because they yield clinically relevant and functional endpoints, have been shown to capture impairments that are common to ID as well as those specific to FXS or DS, and have been shown to yield robust indicators of developmental change within typical and other language-impaired populations. In this project, we propose: (1) to examine the basic psychometric properties of measures derived from expressive language sampling techniques, including establishing their test-retest reliability, internal consistency, validity, and sensitiviy; (2) to evaluate differences in the psychometric properties of expressive language sampling techniques as a function of variations in participant etiology, age, gender, autism symptom severity, and level of ID; (3) to compare the psychometric properties of three different expressive language sampling techniques; and (4) to evaluate the feasibility of implementing the expressive language sampling across multiple sites, as would be required in a typical clinical trial. These aims will be addressed by collecting expressive language samples from children, adolescents, and young adults with FXS or DS. Samples will be collected within three interaction formats: conversation, narration, and the structured interactions comprising the Autism Diagnostic Observation Schedule (ADOS). Measures derived from the samples will include those indexing syntax (an area of especially severe impairment in DS) and perseveration (an area of especially severe impairment for FXS). Test-retest reliability will be assessed at 4 weeks (+/- 1 week) using alternate versions of sampling materials. Internal consistency will be assessed by computing alpha coefficients within and across sampling techniques. Standardized tests and informant report will be used as indicators of validity. A two-year longitudinal follow-up will yield an estimate of sensitivity to change. Participants will be tested at multiple sites, each with considerable experience in the evaluation of individuals with FXS or DS. Feasibility of multiple-site implementation will be evaluated by comparing language samples across sites on key indicators. Transcription, coding, and analysis will be conducted only at the UC Davis MIND Institute site.