Connecting the Dots: An RCT Integrating Standardized ASD Screening, High-Quality Treatment, and Long-Term Outcomes

连接点：整合标准化 ASD 筛查、高质量治疗和长期结果的随机对照试验

• 批准号: 9975220
• 负责人: Diana L Robins
• 金额: $ 224.11万
• 依托单位: DREXEL UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-09-07 至 2022-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9975220
• 关键词: 2 year old; 4 year old; Academy; Address; Advisory Committees; Affect; Age; Age of Onset; American; Attitude; Behavior Therapy; Belief; California; Child; Childhood; Clinical; Cognition; Communities; Conflict (Psychology); Connecticut; Control Groups; Coupled; Data; Detection; Development; Diagnosis; Diagnostic; Early Diagnosis; Early Intervention; Early identification; Early treatment; Educational Intervention; Eligibility Determination; Enrollment; Ethics; Evaluation; Geographic Locations; Goals; Guidelines; Happiness; Health Services Accessibility; Heterogeneity; Individual; Institutes; Intervention; Knowledge; Literature; Longevity; Measures; Medical; Methods; Monitor; Outcome; Outcome Measure; Parent-Child Relations; Parents; Pediatrics; Personal Satisfaction; Physicians; Positioning Attribute; Preventive service; Process; Productivity; Publishing; Randomized; Randomized Controlled Trials; Readiness; Recommendation; Research; Risk; Sampling; Science Policy; Screening procedure; Severities; Site; Social Functioning; Socioeconomic Status; Standardization; Stress; Symptoms; System; Testing; Toddler; Training; Universities; Work; autism spectrum disorder; autistic children; base; cognitive ability; cognitive function; disability; early onset; early screening; empowerment; evidence base; experience; experimental group; improved; improved outcome; intervention effect; kindergarten; primary outcome; satisfaction; screening; screening guidelines; secondary outcome; service delivery; social engagement; social reciprocity; societal costs; targeted treatment; treatment as usual; treatment response; trial design; visual tracking

A growing body of evidence demonstrates that younger age of onset for ASD treatment improves outcomes. Universal toddler screening for autism spectrum disorder (ASD) has been shown to lower the age of ASD diagnosis by two years compared to the national median; this in turn lowers the age of access to ASD-specific treatment. Yet on February 17, 2016, the US Preventive Services Task Force (USPSTF) published a final statement on autism screening in JAMA, in which they found insufficient evidence to recommend universal ASD screening. One of the primary gaps they identified was a lack of randomized controlled trials (RCTs) in which children detected through screening received treatment for ASD, with their outcomes compared to those of children not screened. The current proposal aims to fill this gap, connecting the dots between the screening and treatment literatures, and demonstrating that standardized, high-fidelity, universal screening lowers the age of diagnosis and treatment onset, leading to improved short- and long-term outcomes. The intervention in this RCT is supported deployment of such screening, with immediate evaluation of at-risk children, compared to a control group receiving usual care approaches to early detection of ASD, namely physician surveillance and/or unstandardized screening. In a sample of 8,000 toddlers enrolled through participating pediatric practices near Drexel University, the University of Connecticut, and the University of California Davis MIND Institute, we expect that cognitive functioning and ASD symptom severity will show greater improvements in the experimental group, in which most children will be detected at 18 m screening, compared to the control group, in which children are expected to be detected significantly later. In both groups, missed ASD cases will be detected through screening at 48 m. Across both groups, all children identified as at risk for ASD will receive a diagnostic evaluation, and all children diagnosed with ASD will be enrolled in one year of manualized early intensive behavioral treatment (EIBI). In addition to the primary outcome measures of cognition and symptom severity, exploratory outcomes will include group differences in adaptive functioning, kindergarten readiness, and social reciprocity as measured by experimental eye tracking and parent-child interaction ratings. We also will examine the impact of the screening intervention on physician attitudes and on parent empowerment and stress. Finally, we will examine potential moderators of group differences, to determine whether initial symptom severity, cognitive ability, or SES affects the expected superior response to treatment in the experimental group. Our Autism Centers of Excellence Network is uniquely positioned to carry out this study; our prior work demonstrates our extensive experience and productivity in the field of early screening and treatment in ASD. This study will be the first RCT of toddler ASD screening, and will use rigorous methods that have provided evidence for universal screening protocols in other medical fields. This study will have a significant impact on science, policy, and practice. By demonstrating that universal early screening leads to earlier treatment and better outcomes, this project will provide evidence for the benefits of universal ASD screening, including better social and cognitive functioning, mitigating lifespan disability, reducing societal costs, and improving personal well-being and productivity of individuals with ASD.

越来越多的证据表明，自闭症谱系障碍（ASD）治疗的年轻发病年龄可以改善预后。通用幼儿 研究表明，与自闭症谱系障碍 (ASD) 筛查相比，自闭症谱系障碍 (ASD) 筛查可将 ASD 诊断年龄降低两年 全国中位数；这反过来又降低了获得 ASD 特异性治疗的年龄。然而2016年2月17日，美国 预防服务工作组 (USPSTF) 在《美国医学会杂志》上发表了关于自闭症筛查的最终声明，其中他们发现 没有足够的证据推荐普遍的 ASD 筛查。他们发现的主要差距之一是缺乏 随机对照试验 (RCT)，其中通过筛查发现的儿童接受了自闭症谱系障碍 (ASD) 治疗， 与未接受筛查的儿童的结果进行比较。当前的提案旨在填补这一空白，将各个点连接起来 筛选和治疗文献之间的关系，并证明标准化、高保真度、通用性 筛查降低了诊断和治疗开始的年龄，从而改善短期和长期结果。这 这项随机对照试验的干预措施是支持部署此类筛查，立即评估高危儿童，比较 对照组接受常规护理方法以早期发现 ASD，即医生监测和/或 筛查不规范。通过参与德雷克塞尔附近儿科诊所招募的 8,000 名幼儿样本 大学、康涅狄格大学和加州大学戴维斯分校 MIND 研究所，我们预计认知 实验组的功能和 ASD 症状严重程度将显示出更大的改善，其中大多数儿童 筛查时将在 18 m 时检出，与对照组相比，儿童预计会显着检出 之后。在两组中，漏检的 ASD 病例都将通过 48 m 的筛查来发现。在两组中，所有儿童都发现 有自闭症谱系障碍风险的儿童将接受诊断评估，所有被诊断患有自闭症谱系障碍的儿童将参加为期一年的 手动早期强化行为治疗（EIBI）。除了认知和认知的主要结果测量之外 症状严重程度、探索性结果将包括适应性功能、幼儿园准备情况和 通过实验性眼球追踪和亲子互动评级来衡量社会互惠。我们还将检查 筛查干预对医生态度以及家长赋权和压力的影响。最后，我们将检查 群体差异的潜在调节因素，以确定初始症状严重程度、认知能力或 SES 是否会影响 预期实验组对治疗有更好的反应。我们的自闭症卓越中心网络是独一无二的 有能力开展这项研究；我们之前的工作展示了我们在早期领域的丰富经验和生产力 ASD 的筛查和治疗。这项研究将是第一个针对幼儿 ASD 筛查的随机对照试验，并将使用严格的方法 这为其他医学领域的通用筛查方案提供了证据。这项研究将产生重大影响 关于科学、政策和实践。通过证明普遍的早期筛查可以带来更早的治疗和更好的结果 结果，该项目将为普遍 ASD 筛查的好处提供证据，包括更好的社会和 认知功能、减轻终身残疾、降低社会成本、改善个人福祉和 自闭症谱系障碍患者的生产力。