Evaluating Assessment and Medication Treatment of ADHD in Children with Down Syndrome

项目摘要 唐氏综合症（DS）儿童的注意力缺陷多动症患病率是其他儿童的3-5倍。 注意力缺陷多动症（ADHD）比一般发育中的儿童。尽管他们患多动症的风险更高， 建议治疗儿童智力残疾（ID）和共病ADHD与兴奋剂（最 有效的ADHD治疗），DS+ADHD儿童的兴奋剂使用率不成比例地低， 药物治疗DS+ADHD中兴奋剂治疗利用不足的可能原因包括：1） 关于如何准确诊断DS儿童ADHD的诊断不确定性， 倾向于“诊断遮蔽”（即，将ADHD归因于他们的ID）; 2）没有任何先前的临床 检查兴奋剂药物在DS+ADHD儿童中的安全性和有效性的试验;以及3）关注 考虑到DS中先天性心脏病或缺陷（CHD）的高发病率， 人口基于R61先导试验的结果，我们提出了第一个随机临床试验的兴奋剂 DS+ADHD儿童的药物治疗，以提供有关短期和长期安全性的证据， 兴奋剂对伴有和不伴有CHD的DS+ADHD儿童的疗效100名儿童 6-17岁的DS+ADHD患者将参加。患有DS+ADHD的儿童将完成多阶段的 一项随机、双盲临床试验，交叉至安慰剂组，并进行长期随访，以评估短期 刺激性药物治疗ADHD症状和障碍的长期有效性和安全性 DS的孩子R33的研究目标将集中在1）评估兴奋剂的短期和长期安全性 DS+ADHD儿童的治疗，特别关注心脏安全性; 2）确定短期和 兴奋剂治疗对认知、行为和功能障碍的长期疗效 患有DS+ADHD的儿童;以及3）探索调节者（例如，智商，ADHD亚型/表现，执行 功能水平，共病内化障碍，CHD）的刺激反应和不良反应。结果 这项研究将提供急需的诊断和治疗数据，这些数据将直接影响结果。 在全国大约45,000名患有DS+ADHD的儿童中。

PROJECT SUMMARY Children with Down syndrome (DS) have a 3-5 times greater prevalence of Attention Deficit Hyperactivity Disorder (ADHD) than typically developing children. Despite their higher risk of ADHD and consensus guideline recommendations to treat children with intellectual disability (ID) and comorbid ADHD with stimulants (the most efficacious ADHD treatment), children with DS+ADHD have disproportionately low rates of stimulant medication treatment. Possible reasons for under-utilization of stimulant treatment in DS+ADHD include: 1) diagnostic uncertainty regarding how to accurately diagnose ADHD in children with DS, making providers prone to “diagnostic overshadowing” (i.e., attributing ADHD to their ID); 2) the absence of any prior clinical trials examining the safety and efficacy of stimulant medication in children with DS+ADHD; and 3) concerns about cardiac safety, given the high incidence of congenital heart disease or defects (CHD) in the DS population. Based on R61 pilot trial findings, we propose the first randomized clinical trial of stimulant medication in children with DS+ADHD to provide evidence regarding the short and long-term safety and efficacy of stimulant use in children with DS+ADHD, both with and without CHD. One hundred (100) children with DS+ADHD aged 6-17 years will participate. Children with DS+ADHD will complete a multi-phased randomized, double-blind clinical trial with crossover to placebo and long-term follow-up to assess the short- and long-term efficacy and safety of stimulant medications for treating ADHD symptoms and impairment in children with DS. Study aims of the R33 will focus on 1) Assessing the short- and long-term safety of stimulant treatment in children with DS+ADHD with a specific focus on cardiac safety; 2) Determining the short- and long-term efficacy of stimulant treatment at remediating cognitive, behavioral, and functional impairments in children with DS+ADHD; and 3) Exploring moderators (e.g., IQ, ADHD subtype/presentation, executive functioning levels, comorbid internalizing disorders, CHD) of stimulant response and adverse effects. Results from this study will provide much needed diagnostic and treatment data that will directly impact the outcomes of the approximately 45,000 children with DS+ADHD nationwide.