Positive Airway Pressure For The Treatment Of The Obstructive Sleep Apnea Syndrome In Children With Down Syndrome

气道正压通气治疗唐氏综合症儿童阻塞性睡眠呼吸暂停综合症

• 批准号: 10912150
• 负责人: Ignacio Esteban Tapia
• 金额: $ 6.39万
• 依托单位: UNIVERSITY OF MIAMI SCHOOL OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-09-01 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10912150
• 关键词: Address; Adherence; Adolescent; Attention; Behavior; Behavior Therapy; Behavioral; Birth; Child; Childhood; Cognitive; Congenital Heart Defects; Craniofacial Abnormalities; Data; Diagnosis; Disease; Down Syndrome; Drowsiness; Evaluation; Failure; Family; General Population; Growth; Health; Healthcare; Hypothyroidism; Individual; Infrastructure; Intellectual functioning disability; Intervention; Life Expectancy; Measures; Mediating; Methods; Moods; Morbidity - disease rate; Muscle hypotonia; Neurobehavioral Manifestations; Obesity; Obstructive Sleep Apnea; Outcome; Participant; Perception; Performance; Phase; Philadelphia; Polysomnography; Population; Prevalence; Pulmonary Hypertension; Quality of life; Randomized; Randomized, Controlled Trials; Schools; Self Efficacy; Site; Sleep; Sleep Apnea Syndromes; Standardization; Syndrome; Testing; Translating; Treatment Efficacy; Uncertainty; Universities; Youth; aged; arm; clinical care; comorbidity; craniofacial; design; disability; efficacy evaluation; empowerment; endothelial dysfunction; experience; health care service utilization; health related quality of life; improved; mortality; neurobehavioral; positive airway pressure; reduce symptoms; treatment arm

Abstract Individuals with Down syndrome (DS) are predisposed to OSAS due to craniofacial features (midface hypoplasia, glossoptosis) and studies have shown that the prevalence of OSAS in this population is markedly increased compared to that of typically developing children. Adenotonsillectomy (AT) is considered first-line treatment for childhood OSAS. However, OSAS resolves in only a portion of children with DS after AT. In fact, many children with DS are referred for positive airway pressure (PAP) therapy initiation due to persistent OSAS after AT, and PAP appears to be an important feature of the experience of living with DS. PAP has been shown to be highly effective at treating OSAS and improve OSAS-associated neurobehavioral symptoms, such as quality of life, behavior, mood, daytime sleepiness and school performance. However, PAP as a treatment for OSAS has not been well-studied in children with DS. Therefore, we propose to leverage the HEalthy SLeeP for Children with Down Syndrome (HELP-DS, U01HL125295-S1 and U01HL123507-S1) infrastructure and conduct in two HELP-DS sites (Philadelphia and Cincinnati) plus the University of Miami, a randomized controlled trial to test a 6-month intensive behavioral intervention (INT) to improve PAP adherence vs standard clinical care (CON). Participants with DS and OSAS, aged 6-17.9 years, referred to PAP initiation for the treatment of OSAS will be included. They will undergo standardized evaluations of quality of life, behavior, attention, and family-relevant outcomes, PAP adherence, and health care utilization at baseline, 6, and 12 months. This rigorous design and comprehensive study will resolve existing uncertainties on initial management approaches for children with DS and OSAS treated with PAP by addressing critical issues: a) determine the efficacy of INT vs CON in promoting PAP adherence, b) elucidate which factors mediate or moderate adherence to PAP in children with DS and OSAS, c) determine the effect of PAP use on neurobehavioral and family-relevant outcomes, and quality of life.

摘要 唐氏综合征（DS）患者由于颅面特征（面中部）而易患OSAS 发育不全，舌下垂），研究表明，OSAS在这一人群中的患病率明显高于其他人群。 与一般发育中的儿童相比有所增加。腺样体切除术（AT）被认为是一线 儿童OSAS的治疗然而，OSAS解决只有一部分儿童DS后AT。事实上， 许多患有DS的儿童由于持续的呼吸困难而被转诊进行气道正压通气（PAP）治疗。 AT和PAP后的OSAS似乎是DS生活经历的一个重要特征。PAP已经 在治疗阻塞性睡眠呼吸暂停综合征和改善阻塞性睡眠呼吸暂停综合征相关的神经行为症状方面非常有效， 如生活质量、行为、情绪、白天嗜睡和学校表现。然而，PAP作为治疗 在患有DS的儿童中尚未进行充分研究。因此，我们建议利用健康睡眠 为唐氏综合症儿童（HELP-DS，U 01 HL 125295-S1和U 01 HL 123507-S1）提供基础设施， 在两个HELP-DS研究中心（费城和辛辛那提）加上迈阿密大学进行， 一项对照试验，旨在测试6个月强化行为干预（INT）与标准治疗相比改善PAP依从性 临床护理（CON）。患有DS和OSAS的参与者，年龄6-17.9岁， OSAS的治疗将包括在内。他们将接受生活质量，行为， 基线、6和12时的注意力、家庭相关结局、PAP依从性和卫生保健利用 个月这种严格的设计和全面的研究将解决现有的不确定性， 通过解决关键问题，为接受PAP治疗的DS和OSAS儿童提供管理方法：a） 确定INT与CON在促进PAP粘附方面的功效，B）阐明哪些因素介导或 在患有DS和OSAS的儿童中中度坚持PAP，c）确定PAP使用对 神经行为和家庭相关结果以及生活质量。