Self-Supporting Nasopharyngeal Airway (ssNPA) Treating Upper Airway Obstruction in Hypotonia

自支撑鼻咽气道 (ssNPA) 治疗肌张力低下的上气道阻塞

• 批准号: 10518836
• 负责人: LOUISE M O'BRIEN
• 金额: $ 67.24万
• 依托单位: UNIVERSITY OF MICHIGAN AT ANN ARBOR
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-09-01 至 2026-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10518836
• 关键词: Address; Adherence; Anatomy; Anoxic Encephalopathy; Apnea; Arousal; Asphyxia; Bypass; Caregivers; Caring; Cerebral Palsy; Child; Childhood; Clinical; Clinical Trials; Clinical assessments; Controlled Clinical Trials; Data; Device Approval; Devices; Diffuse; Discipline of Nursing; Down Syndrome; Effectiveness; Engineering; Evaluation; Failure; Family; Goals; Grant; Head and Neck Surgery; Healthcare; High Prevalence; Home; Incidence; Intervention; Literature; Maintenance; Masks; Medical Care Costs; Medicine; Molecular Abnormality; Morbidity - disease rate; Muscle; Muscle hypotonia; Neuromuscular Diseases; Obstruction; Obstructive Sleep Apnea; Operative Surgical Procedures; Otolaryngology; Outcome; Oxygen; Palliative Care; Patients; Pattern; Phase; Physiological; Polysomnography; Population; Preparation; Prevalence; Procedures; Protocol Compliance; Protocols documentation; Quality of life; Questionnaires; Randomized; Recurrence; Residual state; Resources; Risk; Safety; Severities; Sleep; Sleep Apnea Syndromes; Stents; Symptoms; Syndrome; Testing; Tracheostomy procedure; Translations; Treatment Efficacy; Vomiting; Waiting Lists; adherence rate; aged; airway obstruction; arm; cohort; desensitization; design; effective therapy; family burden; improved; inclusion criteria; indexing; meetings; mortality; multidisciplinary; novel; pediatric patients; positive airway pressure; sleep quality; standard of care; treatment duration

There is a critical need for safe and effective treatment options for persistent obstructive sleep apnea (OSA) in patients with Hypotonic Upper Airway Obstruction (HUAO). HUAO encompass conditions such as cerebral palsy, hypoxic encephalopathy, syndromic tone anomalies, Trisomy 21 or Down Syndrome, and neuromuscular disorders, and typically share a similar pattern of multisite upper airway collapse. OSA is characterized by recurrent episodes of partial or complete upper airway obstruction during sleep with associated arousals and/ or oxygen desaturations. In contrast to the incidence of OSA among children which is between 1-5%, there is a significantly higher prevalence of OSA among children with hypotonic conditions, cerebral palsy, and Trisomy 21/Down Syndrome. Of hypotonic patients undergoing polysomnography (PSG) for symptoms of sleep disordered breathing, 25% had moderate OSA, and 56.3% had severe OSA. Thus, not only are patients more likely to have OSA, but it is likely to be much more severe. Despite the obvious upper airway collapse seen in most of these patients, there is a paucity of data in literature, both on prevalence and severity of OSA in these conditions, but also on treatment efficacy particularly with regards to multi-level obstruction and moreover, with duration of treatment efficacy. Currently available treatment options, ranging from palliative care to tracheostomy, often fail to fully meet the needs of these patients. Our multidisciplinary team has developed a dramatically effective non-surgical nasopharyngeal airway stent that has demonstrated good tolerability in hypotonic patients. Treatment of OSA in HUAO presents an extremely difficult challenge, in part due to diffuse multilevel obstruction with lack of muscular airway tone. This renders many surgical approaches, other than tracheostomy, likely to fail. Although surgical options such as adenotonsillectomy are common, most children with HUAO have residual OSA after the procedure, due to residual anatomic and physiological abnormalities. This often necessitates attempted treatment with Positive Airway Pressure (PAP) or consideration for tracheostomy. However, the rate of adherence with PAP in typically developing children can be low, and even lower in those with hypotonic conditions. Moreover, inability to remove a mask during an emesis can pose a suffocation risk. Tracheostomy carries associated risks for morbidity and mortality, a substantial addition of care needs, family burden, nursing demands, and hundreds of thousands of dollars of medical costs. Our multidisciplinary team, led by Otolaryngology – Head and Neck Surgery, Sleep Medicine, and Engineering, has designed, developed, and successfully piloted a self-supported nasopharyngeal airway (ssNPA) device that bypasses challenging, diffuse, multilevel upper airway obstruction. The device has demonstrated initial safety and preliminary efficacy in a pilot group of patients, severe OSA. The Start-up phase (R61) of the grant will test an enhanced version of the device for acceptability and tolerability. Critically, insertion, adherence, and compliance protocols will be optimized for preparation of the R33 phase. The R33 phase will assess a powered cohort looking at PSG and quality of life outcomes.

持续性阻塞性睡眠呼吸暂停（OSA）的安全有效的治疗方案是一个关键的需求， 低张性上气道阻塞（HUAO）患者。HUAO包括诸如脑 麻痹、缺氧性脑病、综合征性音调异常、21三体或唐氏综合征和神经肌肉疾病，并且通常具有相似的多部位上气道塌陷模式。阻塞性睡眠呼吸暂停的特征是睡眠期间反复发作的部分或完全上呼吸道阻塞，伴有相关的觉醒和/或氧饱和度下降。与儿童中OSA的发病率在1- 5%之间相比，患有低渗状况、脑瘫和21三体/唐氏综合征的儿童中OSA的患病率显著更高。在因睡眠呼吸障碍症状而接受多导睡眠图（PSG）检查的低渗患者中，25%患有中度OSA，56.3%患有重度OSA。因此，患者不仅更有可能患有OSA，而且可能更加严重。尽管在这些患者中大多数可见明显的上呼吸道塌陷，但关于OSA在这些疾病中的患病率和严重程度以及治疗效果的文献数据很少 特别是关于多节段阻塞，此外，关于治疗效果的持续时间。目前 现有的治疗方案，从姑息治疗到气管切开术，往往不能完全满足患者的需要。 这些病人。我们的多学科团队已经开发出一种非常有效的非手术鼻咽 气道支架在低渗患者中表现出良好的耐受性。 在HUAO治疗OSA是一个非常困难的挑战，部分原因是弥漫性多水平的 由于缺乏肌肉气道张力而导致的阻塞。这使得许多手术方法，除了 气管切开术很可能会失败虽然手术选择，如腺样体切除术是常见的，大多数儿童 由于解剖和生理异常残留，HUAO患者术后存在残留OSA。 这通常需要尝试使用气道正压通气（PAP）进行治疗或考虑 气管切开术然而，在典型发育的儿童中，PAP的依从率可能很低，甚至 在低渗条件下更低。此外，在呕吐期间无法取下面罩可能会造成 窒息危险气管切开术具有相关的发病率和死亡率风险， 护理需求，家庭负担，护理需求，以及数十万美元的医疗费用。 我们的多学科团队由耳鼻喉科-头颈外科、睡眠医学和工程学领导， 设计、开发并成功试用了一种自支撑鼻咽气道（ssNPA）设备 绕过具有挑战性的、弥漫性的、多层次的上呼吸道阻塞。该器械已初步证明 安全性和初步疗效在一个试点组的患者，严重的OSA。赠款的启动阶段（R61） 将测试增强版器械的可接受性和耐受性。至关重要的是，插入、粘附和 将为R33阶段的准备工作优化合规协议。R33阶段将评估一个动力 观察PSG和生活质量结果的队列。