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

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治疗的有效性。 探讨手术治疗唐氏综合征（DS）儿童OSA的方法。的估计患病率 患有唐氏综合征（DS）的儿童中阻塞性睡眠呼吸暂停（OSA）的比例为31- 79%，而患有DS的儿童中OSA的比例为1-5%。 在一般儿科人群中。腺样体切除术（AT）是儿童OSA的一线治疗方法， 然而，大多数AT结果的研究都排除了DS儿童。现有证据有限 表明50 - 90%的DS儿童在AT后有持续的OSA。 张力减退和不利的颅面解剖结构是AT失败的常见风险因素， 然而，很少有人尝试描述咽部解剖结构或阻塞机制 在这个人群中。药物诱导睡眠内窥镜检查（DISE）可以直接观察部位和模式 在镇静剂睡眠期间使用一个柔性内窥镜通过鼻子进入咽部。 几个小病例系列表明，个性化DISE定向手术可有效治疗 DS患儿的OSA。因为几乎没有前瞻性研究，也没有随机试验， 比较不同的治疗方案，仍然存在不确定性的最有效的治疗OSA， DS的孩子 我们的中心假设是，DS儿童的个性化DISE定向手术方法将 上级推荐的AT一线治疗。为了验证这一假设，我们提出了一个 在唐氏综合征儿童中进行DISE定向手术与AT的前所未有的随机对照试验 2-18岁，具体目标如下： 目的1：比较DS儿童中DISE定向手术与AT的生理结局。 假设1：DISE定向手术将导致阻塞性呼吸暂停低通气的更大改善 6个月后，与标准AT干预相比，OAHI指数。 目的2：比较儿童DS中DISE定向手术与AT的临床结局。 假设2：DISE定向手术将导致疾病的临床显著改善- 6个月后与标准AT干预相比的特定QOL（OSA 18）。 这项比较有效性的试验将是第一次直接比较个性化的手术治疗， DS儿童标准治疗的方法这种新方法可能改善OSA的结局 并减少无效AT后不必要的AT或持续性OSA的二次手术的负担。

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.