Efficacy of Parent-implemented Treatment in Infant Siblings of Children With ASD

描述（由申请人提供）：一种预防或降低社交和语言沟通障碍高风险儿童（例如，患有自闭症谱系障碍的儿童的弟弟妹妹（Sibs-ASD）是在大脑仍在发育的年龄（即，婴儿期）。目前的筛查方法过度识别哪些兄弟姐妹ASD需要昂贵的治疗。“增量治疗方法”（1）对所有风险组成员使用具有成本效益的治疗，（2）确定那些在具有成本效益的治疗后在重要技能上没有改变的人（即，无应答者），和（3）为无应答者提供更密集（和昂贵）的治疗。该项目将涉及渐进式治疗方法的前两个步骤。实施前两个步骤可以避免对一组高危婴儿进行昂贵的治疗，这些婴儿在没有密集和昂贵的干预的情况下可能会改善。一种类型的具有成本效益的治疗，可以让我们完成第二步的目标是特定的儿童能力，促进沟通的发展（即，关键技能）。此外，确定最有可能从成本效益治疗中受益的同胞ASD与需要更密集干预的同胞ASD的特征，将促进增量治疗方法的有效和高效实施，以解决未来的社会和语言障碍。最后，评估成本效益的治疗改善语言延迟和ASD发育学的机制将提高我们对社会环境如何影响同胞ASD发展轨迹的理解。在120个同胞ASD样本中，（12-15个月），在两个研究中心招募（华盛顿大学，西雅图;范德比尔特大学，纳什维尔），该项目将实现以下目标：（a）测试一种具有成本效益的治疗方法的疗效，该方法有望治疗同胞ASD中的社会和语言障碍（ImPACT）;（B）确定最受益于ImPACT的亚组的特征;（c）评估关键技能的改善是否对更远的口语技能和ASD诊断产生预期的影响;（d）确定ImPACT中的活性成分，该治疗可改善关键技能。治疗疗效目标将通过对包括ASD在内的沟通障碍的初始累积风险状态对同胞ASD进行分层，然后将其随机分配到治疗组或对照组来实现。治疗将由父母在研究人员的监督下提供3个月。治疗后即刻评价后将进行3个月和6个月随访评估。检查员和编码员对治疗分配不知情。将进行意向治疗分析。将使用最先进的统计方法来确定与对ImPACT的应答相关的特征，并检测治疗的关键技能和活性成分是否介导治疗效果。该研究团队在方法方面经验丰富，并且有着富有成效的合作历史。

DESCRIPTION (provided by applicant): One way to prevent or reduce the severity of social and linguistic communication disorders in children at high risk for these disorders (e.g., younger siblings of children with autism spectrum disorders, Sibs-ASD) is to treat them at an age when the brain is still developing (i.e., infancy). Current screening methods over-identify which Sibs-ASD need expensive treatments. An "Incremental Treatment Approach" (1) uses a cost-effective treatment with all risk group members, (2) identifies those who do not change on important skills after cost- effective treatment (i.e., nonresponders), and (3) provides nonresponders with a more intensive (and expensive) treatment. This project will address the first two steps of the Incremental Treatment Approach. Implementing the first two steps may avoid costly treatment of a subgroup of at-risk infants who may improve without intensive and expensive intervention. One type of cost-effective treatment that may allow us to accomplish the 2nd step targets specific child abilities that promote communicative development (i.e., pivotal skills). Furthermore, identifying the characteristics that distinguish Sibs-ASD most likely to benefit from the cost-effective treatment from Sibs-ASD who will require more intensive intervention will promote effective and efficient implementation of the Incremental Treatment Approach to address social and linguistic disorders in the future. Finally, evaluating the mechanisms by which the cost-effective treatment ameliorates language delay and ASD symptomatology will improve our understanding of how the social environment can affect the developmental trajectories of Sibs-ASD. In a sample of 120 Sibs-ASD (12-15 mos), recruited at two sites (University of Washington, Seattle; Vanderbilt University, Nashville), this project will accomplish the following goals: (a) test the efficacy of a cost-effective treatment that shows promise for treatment of social and linguistic impairments in Sibs-ASD (ImPACT); (b) identify characteristics of the subgroup most benefiting from ImPACT; (c) evaluate whether improvements in pivotal skills produce anticipated effects on more distal spoken language skills and ASD symptomatology; (d) identify the active ingredients in ImPACT by which the treatment improves pivotal skills. Treatment efficacy goals will be addressed by stratifying Sibs-ASD on initial cumulative-risk status for communication disorder, including ASD, and then randomly assigning them to treatment or control groups. Treatment will be provided by parents under the supervision of research staff for 3 months. Immediate post-treatment evaluations will be followed by 3 month and 6 month follow-up assessments. Examiners and coders will be blind to treatment assignment. Intent to treat analyses will be conducted. State-of-the-art statistical methods will be used to identify the characteristics associated with response to ImPACT and to test whether pivotal skills and active ingredients of the treatment mediate treatment effects. The research team is experienced with the methods and has a productive collaborative history.