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)

基本信息

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
项目类别：
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

项目摘要

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新型个性化的有效性唐氏综合症儿童（DS）中OSA手术治疗的方法。估计的患病率唐氏综合症（DS）儿童的阻塞性睡眠呼吸暂停（OSA）范围为31-79％，而1-5％在一般的小儿人群中。腺孔切除术（AT）是儿童OSA的第一行治疗，但是，大多数对结局的研究都排除了DS的儿童。有限的可用证据证明50％至90％的DS儿童在AT之后具有持久的OSA。低调和不利的颅面解剖结构通常是引用AT失败的风险因素但是，很少有尝试表征咽解剖或阻塞机制的尝试在这个人群中。药物诱导的睡眠内窥镜检查（DISE）可以直接观察站点和模式镇静睡眠期间使用柔性内窥镜穿过鼻子进入咽部的障碍物。几个小病例系列表明，个性化疾病指导的手术可以有效治疗 DS儿童的OSA。因为前瞻性研究很少，没有随机试验比较不同的治疗方案，关于OSA最有效的治疗仍然不确定性患有DS的孩子。我们的核心假设是，DS儿童的个性化疾病指导的手术方法要优于推荐的第一行处理。为了检验这一假设，我们提出了一个疾病儿童的疾病指导手术的前所未有的随机对照试验年龄在2-18岁之间，以下具体目标： AIM 1：比较DS儿童的疾病指导手术的生理结果。假设1：指导的手术将导致阻塞性呼吸暂停呼吸症的改善与6个月后干预时的标准相比，指数（OAHI）。 AIM 2：比较DS儿童的疾病指导手术的临床结果。假设2：疾病指导的手术将在临床上显着改善疾病 - 特定的QOL（OSA18）与6个月后干预时的标准相比。该比较有效性试验将是第一个直接比较个性化手术的试验 DS儿童的标准治疗方法可能会改善OSA结果并减轻无效的OSA的不必要或继发手术的负担。