A Personalized Surgical Approach for the Treatment of Children with Obstructive Sleep Apnea and Small Tonsils

项目摘要 阻塞性睡眠呼吸暂停(OSA)在儿童中很常见，估计患病率为1-6%。未经治疗 儿童阻塞性睡眠呼吸暂停综合征与高血压、自主神经功能障碍、注意力缺陷/多动障碍、 神经认知缺陷，生活质量差。扁桃体肥大是儿童高血压的主要危险因素 OSA，腺扁桃体切除术(AT)是推荐的一线治疗方法。然而，目前还不清楚是否 扁桃体小的儿童将从AT中受益。扁桃体大小的临床评估并不一致 OSA的严重程度或对AT的反应，导致对如何最好地治疗小OSA儿童的困惑 扁桃体。药物诱导睡眠内窥镜(DISE)可直接观察 镇静睡眠时的阻塞。DISE越来越多地帮助指导成人阻塞性睡眠呼吸暂停综合征的手术决策，并且 也可用于指导儿童阻塞性睡眠呼吸暂停的手术决定和改善预后。总的目标是 本研究旨在测试一种新的个性化手术治疗阻塞性睡眠呼吸暂停低通气综合征的疗效。 孩子们。我们的中心假设是针对儿童的个体化定向手术方法 用小扁桃体将优于标准的一线治疗AT。为了检验这一假设，我们 提出一项史无前例的2-18岁儿童DISE定向手术与AT的随机对照试验 几年来，扁桃体临床较小，具体目标如下： 目的1：比较儿童小扁桃体直视手术与电切术的生理结果。 假设1：DISE定向手术将导致临床上更大程度的改善 阻塞性呼吸暂停低通气指数(OAHI)与标准AT干预的比较。 目的：比较DISE定向手术与AT手术治疗儿童小扁桃体的临床疗效。 假设2：定向手术将在临床上显著改善疾病-- 特定生活质量(OSA18)与标准AT干预的比较。 目的3：确定小扁桃体儿童OSA的严重程度是否与特定的解剖内型有关。 假设3：OSA基线严重程度(由OAHI定义)与梗阻程度的增加相关 (即DISE分级标准中完全梗阻的解剖部位的数量)。 目标4：确定基线患者特征(例如年龄、肥胖)和特定患者特征之间的潜在关联 严重鼻塞、重度舌根阻塞儿童的解剖内型 临床上扁桃体较小(探查目标)。 这项比较有效性的试验将是第一次直接比较个性化手术 在AT结果不确定的情况下，接近标准治疗。这部小说 该方法可以改善OSA的预后，并减轻不必要的AT或二次手术的负担 无效AT后持续性阻塞性睡眠呼吸暂停。

Project Summary Obstructive sleep apnea (OSA) is common in children with an estimated prevalence of 1-6%. Untreated pediatric OSA is associated with hypertension, autonomic dysfunction, attention deficit/hyperactivity disorder, neurocognitive deficits, and poor quality of life. Adenotonsillar hypertrophy is the primary risk factor for pediatric OSA, and adenotonsillectomy (AT) is the recommended first-line treatment. However, it is unclear whether children with small tonsils will benefit from AT. Clinic assessments of tonsil size do not consistently correlate with OSA severity or response to AT, resulting in confusion about how best to treat OSA in children with small tonsils. Drug-induced sleep endoscopy (DISE) enables direct observation of the sites and patterns of obstruction during sedated sleep. DISE increasingly helps guide surgical decision-making in adult OSA, and can also be used to guide surgical decisions in pediatric OSA and improve outcomes. The overall objective of this study is to test the effectiveness of a novel personalized approach to the surgical treatment of OSA in children. It is our central hypothesis that a personalized DISE-directed surgical approach in children with small tonsils will be superior to the standard first line treatment of AT. To test this hypothesis, we propose an unprecedented randomized controlled trial of DISE-directed surgery vs AT in children ages 2-18 years with clinically small tonsils with the following specific aims: Aim 1: Compare the physiological outcomes of DISE-directed surgery vs AT in children with small tonsils. Hypothesis 1: DISE-directed surgery will result in a clinically significantly greater improvement in the obstructive apnea-hypopnea index (OAHI) compared to the standard AT intervention. Aim 2: Compare the clinical outcomes of DISE-directed surgery vs AT in children with small tonsils. Hypothesis 2: DISE-directed surgery will result in a clinically significantly greater improvement in disease- specific QOL (OSA18) compared to the standard AT intervention. Aim 3: Determine if OSA severity is associated with specific anatomic endotypes in children with small tonsils. Hypothesis 3: Baseline OSA severity (defined by OAHI) is correlated with increasing severity of obstruction (i.e., number of anatomic sites with complete obstruction on DISE rating scale). Aim 4: Identify potential associations between baseline patient characteristics (e.g. age, obesity) and specific anatomic endotypes (e.g., severe nasal obstruction, severe base of tongue obstruction) in children with clinically small tonsils (exploratory aim). This comparative effectiveness trial will be the first to directly compare a personalized surgical approach to the standard treatment in children where the outcome of AT is uncertain. This novel approach may improve OSA outcomes and reduce the burden of unnecessary AT or secondary surgery for persistent OSA after an ineffective AT.