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

唐氏综合症儿童 ADHD 的评估和药物治疗

• 批准号: 10754339
• 负责人: Anna J. Esbensen
• 金额: $ 54.93万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-30 至 2025-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10754339
• 关键词: Academic achievement; Address; Adverse effects; Area; Attention deficit hyperactivity disorder; Attenuated; Behavior; Behavioral; Cardiac; Catecholamines; Child; Clinical; Clinical Trials; Cognitive; Cognitive remediation; Conduct Clinical Trials; Congenital Heart Defects; Consensus; Data; Defect; Developmental Disabilities; Diagnosis; Diagnostic; Differential Diagnosis; Disease; Dose; Double-Blind Method; Down Syndrome; Electrocardiogram; Enrollment; General Population; Genetic Diseases; Genotype; Goals; Guidelines; Heart Abnormalities; Heart Diseases; Hyperactivity; Impaired cognition; Impairment; Impulsivity; Incidence; Intellectual functioning disability; Language; Longterm Follow-up; Monitor; Motor; Outcome; Outcome Measure; Pharmaceutical Preparations; Pharmacotherapy; Phase; Placebos; Population; Prevalence; Protocols documentation; Provider; Quality of life; Randomized; Recommendation; Research; Risk; Ritalin; Safety; Sample Size; Stimulant; Symptoms; Treatment Efficacy; Uncertainty; Underserved Population; Update; Vulnerable Populations; accurate diagnosis; aged; behavioral impairment; behavioral phenotyping; clinically relevant; comorbidity; congenital heart disorder; diagnostic criteria; evidence based guidelines; executive function; functional disability; functional status; heart function; high risk; improved; improved outcome; inattention; medication safety; neurophysiology; pilot trial; randomized, clinical trials; response; side effect; stimulant use; treatment strategy

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.

项目概要 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 与正常发育儿童相比，注意力缺陷多动症（ADHD）更加严重。尽管他们患注意力缺陷多动症的风险较高并且共识指南 建议使用兴奋剂（最常见的药物）治疗患有智力障碍 (ID) 和合并多动症 (ADHD) 的儿童 有效的 ADHD 治疗），患有 DS+ADHD 的儿童接受兴奋剂的比例极低 药物治疗。 DS+ADHD 中兴奋剂治疗使用不足的可能原因包括：1) 关于如何准确诊断患有 DS 的儿童 ADHD 的诊断不确定性，使得提供者 容易出现“诊断掩盖”的情况（即，将多动症归因于他们的 ID）； 2) 没有任何既往临床经验 检查兴奋剂药物治疗 DS+ADHD 儿童的安全性和有效性的试验； 3）担忧 鉴于 DS 中先天性心脏病或缺陷 (CHD) 的高发病率，有关心脏安全的问题 人口。根据 R61 试点试验结果，我们提出了第一个兴奋剂随机临床试验 DS+ADHD 儿童的药物治疗提供有关短期和长期安全性的证据 兴奋剂使用对患有 DS+ADHD 的儿童（无论是否患有先心病）的疗效。一百 (100) 名儿童 6-17岁患有DS+ADHD的人士将参加。患有 DS+ADHD 的儿童将完成多阶段的 随机、双盲临床试验，交叉安慰剂和长期随访，以评估短期疗效 兴奋剂药物治疗多动症症状和损伤的长期疗效和安全性 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）确定短路和 兴奋剂治疗在修复认知、行为和功能障碍方面的长期疗效 患有 DS+ADHD 的儿童； 3) 探索调节因素（例如智商、多动症亚型/表现、执行力 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 全国大约 45,000 名患有 DS+ADHD 的儿童。