Treatment of Obstructive Sleep Apnea with Personalized Surgery in Children with Down Syndrome (TOPS-DS)

通过个性化手术治疗唐氏综合症儿童阻塞性睡眠呼吸暂停 (TOPS-DS)

• 批准号: 10518290
• 负责人: Derek J Lam
• 金额: $ 125.36万
• 依托单位: OREGON HEALTH & SCIENCE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-09-07 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10518290
• 关键词: Address; Adult; Affect; Age; Anatomy; Apnea; Birth; Cardiovascular system; Case Series; Child; Childhood; Clinical; Clinical Investigator; Cognitive; Collaborations; Complex; Data; Disease; Down Syndrome; Drowsiness; Drug usage; Endoscopes; Endoscopy; Evaluation; Failure; Future; Health; Intervention; Larynx; Medical; Muscle hypotonia; Nose; Obesity; Obstruction; Obstructive Sleep Apnea; Operative Surgical Procedures; Outcome; Participant; Pattern; Pharmaceutical Preparations; Pharyngeal structure; Physiological; Population; Prevalence; Problem behavior; Procedures; Prospective Studies; Quality of life; Randomized; Randomized Clinical Trials; Randomized Controlled Trials; Risk Factors; Sampling; Site; Sleep; Source; Standardization; Testing; Treatment Protocols; Uncertainty; airway obstruction; base; comparative effectiveness trial; craniofacial; design; effective therapy; effectiveness testing; executive function; experience; flexibility; improved; improved outcome; indexing; individualized medicine; intervention effect; novel; novel strategies; personalized approach; personalized medicine; pharyngeal patterning; positive airway pressure; precision drugs; randomized trial; standard care; surgery outcome; tongue root; trial comparing

Project Summary The overall objective of this randomized clinical trial is to test the effectiveness of a novel personalized approach to the surgical treatment of OSA in children with Down syndrome (DS). The estimated prevalence of obstructive sleep apnea (OSA) in children with Down syndrome (DS) ranges from 31-79%, compared to 1-5% in the general pediatric population. Adenotonsillectomy (AT) is the first line treatment for OSA in children, however, most studies of AT outcomes have excluded children with DS. Limited available evidence demonstrates that 50 to 90% of children with DS have persistent OSA after AT. Hypotonia and unfavorable craniofacial anatomy are commonly cited risk factors for failure of AT, however, there have been few attempts to characterize the pharyngeal anatomy or mechanisms of obstruction in this population. Drug-induced sleep endoscopy (DISE) enables direct observation of the sites and patterns of obstruction during sedated sleep using a flexible endoscope passed through the nose into the pharynx. Several small case series demonstrate that personalized DISE-directed surgery can be effective in treating OSA in children with DS. Because there have been few prospective studies and no randomized trials comparing different treatment options, there remains uncertainty about the most effective treatment for OSA in children with DS. It is our central hypothesis that a personalized DISE-directed surgical approach in children with DS will be superior to the recommended first line treatment of AT. To test this hypothesis, we propose an unprecedented randomized controlled trial of DISE-directed surgery vs AT in children with Down syndrome ages 2-18 years with the following specific aims: Aim 1: Compare the physiological outcomes of DISE-directed surgery vs AT in children with DS. Hypothesis 1: DISE-directed surgery will result in a greater improvement in the obstructive apnea-hypopnea index (OAHI) compared to the standard AT intervention after 6 months. Aim 2: Compare the clinical outcomes of DISE-directed surgery vs AT in children with DS. Hypothesis 2: DISE-directed surgery will result in a clinically significantly greater improvement in disease- specific QOL (OSA18) compared to the standard AT intervention after 6 months. This comparative effectiveness trial will be the first to directly compare a personalized surgical approach to the standard treatment in children with DS This novel approach may improve OSA outcomes and reduce the burden of unnecessary AT or secondary surgery for persistent OSA after an ineffective AT.

项目摘要 这项随机临床试验的总体目标是测试一种新的个性化的 唐氏综合征患儿阻塞性睡眠呼吸暂停的外科治疗探讨估计患病率为 唐氏综合征(DS)儿童的阻塞性睡眠呼吸暂停(OSA)发生率为31-79%，而唐氏综合征(DS)儿童的OSA为1-5% 在普通儿科人群中。腺体扁桃体切除术(AT)是儿童阻塞性睡眠呼吸暂停综合征的一线治疗方法， 然而，大多数关于AT结局的研究都排除了患有DS的儿童。可获得的证据有限 显示50%至90%的DS儿童在AT后有持续性OSA。 低眼压和不利的头面部解剖结构是房颤失败的常见危险因素， 然而，很少有人尝试描述咽部解剖或梗阻的机制。 在这群人中。药物诱导的睡眠内窥镜(DISE)可以直接观察部位和模式 在镇静睡眠期间使用柔性内窥镜通过鼻子进入咽部的梗阻。 几个小病例系列表明，个性化定向手术在治疗中可以有效 阻塞性睡眠呼吸暂停综合征患儿的临床研究因为很少有前瞻性研究，也没有随机试验 比较不同的治疗方案，对于#年阻塞性睡眠呼吸暂停最有效的治疗仍然存在不确定性 患有DS的儿童。 我们的中心假设是，对患有DS的儿童进行个性化的DISE定向手术将 优于推荐的AT一线治疗。为了检验这一假设，我们提出了一个 唐氏综合征儿童DISE定向手术与AT的史无前例的随机对照试验 年龄2-18岁，具体目标如下： 目的1：比较DS患儿DISE定向手术与AT手术的生理结局。 假设1：定向手术将大大改善阻塞性呼吸暂停低通气 6个月后与标准AT干预组比较。 目的：比较DISE定向手术与AT治疗儿童DS的临床疗效。 假设2：定向手术将在临床上显著改善疾病-- 6个月后将特定生活质量(OSA18)与标准AT干预进行比较。 这项比较有效性的试验将是第一次直接比较个性化手术 对DS儿童进行标准治疗的方法这种新方法可能会改善阻塞性睡眠呼吸暂停综合征的预后 并减轻不必要的AT或无效AT后持续OSA的二次手术的负担。