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

项目总结 患有唐氏综合症(DS)的儿童注意力缺陷多动的患病率高出3-5倍 儿童多动症(ADHD)比一般发育中的儿童更严重。尽管他们患ADHD的风险更高，但他们的共识指南 治疗智力残疾儿童(ID)和与兴奋剂共病的ADHD的建议(最多 有效的ADHD治疗)，患有DS ADHD的儿童兴奋剂发生率低得不成比例 药物治疗。DS ADHD患者未充分利用兴奋剂治疗的可能原因包括：1) 关于如何准确诊断患有DS的儿童的ADHD的诊断不确定性，使提供者 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表现 研究DS ADHD儿童服用兴奋剂的安全性和有效性的试验；3)关注的问题 关于心脏安全，鉴于DS中先天性心脏病或先天缺陷(CHD)的高发病率 人口。基于R61先导试验的结果，我们提出了第一个兴奋剂的随机临床试验 DS ADHD儿童的药物治疗提供关于短期和长期安全性和 伴和不伴CHD的DS ADHD儿童使用兴奋剂的疗效。一百(100)个孩子 患有DS、ADHD的6-17岁儿童将参加。患有DS ADHD的儿童将完成一个多阶段的 随机双盲临床试验，交叉使用安慰剂，并进行长期随访，以评估短期和 兴奋剂治疗儿童多动症症状和损害的远期疗效和安全性 患有DS的儿童。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；2)确定短期和 刺激性治疗在纠正认知、行为和功能障碍方面的长期疗效 患有多动症的儿童；和3)探索主持人(例如，智商、ADHD亚型/陈述、执行者 刺激性反应和不良反应的功能水平、共病内化障碍、冠心病。结果 这项研究将提供直接影响结果的急需的诊断和治疗数据 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.