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

治疗患有阻塞性睡眠呼吸暂停和小扁桃体的儿童的个性化手术方法

• 批准号: 10634395
• 负责人: Derek J Lam
• 金额: $ 33.21万
• 依托单位: OREGON HEALTH & SCIENCE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-09-20 至 2025-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10634395
• 关键词: Address; Adult; Age; Anatomy; Apnea; Attention deficit hyperactivity disorder; Autonomic Dysfunction; Case Series; Characteristics; Child; Childhood; Clinic; Clinical; Confusion; Data; Decision Making; Disease; Effectiveness; Endoscopes; Endoscopy; Event; Funding; High Prevalence; Hypertension; Hypertrophy; Intervention; Knowledge; Larynx; Neurocognitive Deficit; Nose; Obesity; Obstruction; Obstructive Sleep Apnea; Operative Surgical Procedures; Outcome; Palate; Patients; Pattern; Performance; Pharmaceutical Preparations; Pharyngeal structure; Physiological; Prevalence; Principal Investigator; Procedures; Publishing; Quality of life; Randomized, Controlled Trials; Recommendation; Reporting; Research; Risk Factors; Schools; Sedation procedure; Severities; Site; Sleep; Symptoms; Testing; Tonsil; airway obstruction; comparative effectiveness trial; comparison control; effective therapy; effectiveness testing; executive function; flexibility; health care service utilization; improved; improved outcome; improvement on sleep; indexing; individualized medicine; nasal obstruction; novel; novel strategies; optimal treatments; personalized approach; personalized medicine; precision drugs; response; standard care; study population; surgery outcome; tongue root; Address; Adult; Age; Anatomy; Apnea; Attention deficit hyperactivity disorder; Autonomic Dysfunction; Case Series; Characteristics; Child; Childhood; Clinic; Clinical; Confusion; Data; Decision Making; Disease; Effectiveness; Endoscopes; Endoscopy; Event; Funding; High Prevalence; Hypertension; Hypertrophy; Intervention; Knowledge; Larynx; Neurocognitive Deficit; Nose; Obesity; Obstruction; Obstructive Sleep Apnea; Operative Surgical Procedures; Outcome; Palate; Patients; Pattern; Performance; Pharmaceutical Preparations; Pharyngeal structure; Physiological; Prevalence; Principal Investigator; Procedures; Publishing; Quality of life; Randomized, Controlled Trials; Recommendation; Reporting; Research; Risk Factors; Schools; Sedation procedure; Severities; Site; Sleep; Symptoms; Testing; Tonsil; airway obstruction; comparative effectiveness trial; comparison control; effective therapy; effectiveness testing; executive function; flexibility; health care service utilization; improved; improved outcome; improvement on sleep; indexing; individualized medicine; nasal obstruction; novel; novel strategies; optimal treatments; personalized approach; personalized medicine; precision drugs; response; standard care; study population; surgery outcome; tongue root

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.

项目摘要 阻塞性睡眠呼吸暂停（OSA）在估计患病率为1-6％的儿童中很常见。未经处理 小儿OSA与高血压，自主神经功能障碍，注意力缺陷/多动障碍有关， 神经认知缺陷和生活质量差。腺刺激性肥大是小儿的主要危险因素 OSA和腺孔切除术（AT）是建议的一线治疗。但是，目前尚不清楚是否 扁桃体小的孩子将从AT中受益。扁桃体大小的诊所评估不一致地相关 OSA的严重程度或对AT的反应，导致了如何最好地治疗小儿童的OSA 扁桃体。药物诱导的睡眠内窥镜检查（DISE）可以直接观察到地点和模式 镇静睡眠期间的阻塞。越来越多地帮助指导成人OSA的手术决策，以及 还可以用于指导小儿OSA中的手术决策并改善结果。总体目标 这项研究是为了测试一种新型个性化方法的有效性，以在OSA中进行手术治疗 孩子们。我们的中心假设是儿童的个性化指导的手术方法 用小扁桃体将优于AT的标准第一行处理。为了检验这一假设，我们 提出了一项前所未有的疾病指导手术的随机对照试验，而在2-18岁的儿童中 临床上小扁桃体的年份，具有以下特定目的： AIM 1：比较小扁桃体的儿童的疾病指导手术的生理结果。 假设1：指导的手术将在临床上显着改善 与干预时的标准相比，阻塞性呼吸暂停 - 海型指数（OAHI）。 AIM 2：比较小扁桃体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）与干预时的标准相比。 AIM 3：确定OSA严重程度是否与小扁桃体儿童的特定解剖学内型有关。 假设3：基线OSA严重程度（由OAHI定义）与障碍的严重程度增加相关 （即，具有完全阻塞的疾病评级量表的解剖部位数量）。 目标4：确定基线患者特征（例如年龄，肥胖）与特定之间的潜在关联 患有儿童 临床上的扁桃体（探索目的）。 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 AT结果不确定的儿童的标准治疗方法。这本小说 方法可能会改善OSA的结果，并减轻或减轻次要手术的不必要的负担 无效之后，OSA持久。