Identifying Measures of Pulmonary Morbidity for Clinical Trials in Children with Down Syndrome and Aspiration

唐氏综合症和误吸儿童临床试验中确定肺部发病率的指标

• 批准号: 10020429
• 负责人: Emily M DeBoer
• 金额: $ 15.75万
• 依托单位: UNIVERSITY OF COLORADO DENVER
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-20 至 2022-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10020429
• 关键词: 6 year old; Affect; Airway Resistance; Allergic inflammation; Aspirate substance; Asthma; Blood; Breathing; Bronchiectasis; Bronchodilator Agents; C-reactive protein; Caregivers; Case-Control Studies; Cause of Death; Characteristics; Child; Childhood; Childhood Asthma; Chronic; Clinical; Clinical Trials; Clinical Trials Design; Collaborations; Colorado; Conduct Clinical Trials; Data; Deglutition; Diagnosis; Down Syndrome; Evaluation; Exercise Tolerance; Future; General Population; Goals; Gold; Health; IgE; Individual; Inflammation; Inflammatory; Institutes; Institution; Intellectual functioning disability; Lead; Liquid substance; Longevity; Lung; Lung Inflammation; Lung diseases; Measurable; Measurement; Measures; Morbidity - disease rate; Muscle hypotonia; Oscillometry; Outcome; Outcome Measure; Participant; Patients; Pediatric Hospitals; Phenotype; Physician Executives; Pneumonia; Population; Positioning Attribute; Prevalence; Prevention; Principal Investigator; Pulmonary Inflammation; Quality of life; Quality-of-Life Assessment; Questionnaires; Readiness; Reporting; Research Personnel; Respiratory Signs and Symptoms; Respiratory physiology; Spirometry; Structural defect; Symptoms; Testing; Walking; Work; airway inflammation; case control; clinical care; clinical center; eosinophil; epidemiology study; high risk; immune function; improved; inflammatory marker; mortality; multidisciplinary; neutrophil; prevent; pulmonary function; respiratory health; response; vocal cord

Respiratory disease is the most common cause of death in people with Down syndrome. Children with Down syndrome have a high risk of aspiration, and chronic aspiration can lead to respiratory diseases including pneumonitis, pneumonia, and bronchiectasis. Epidemiological studies in children without Down syndrome have shown that respiratory health in childhood is a strong predictor of respiratory health across the lifespan, with trajectories of lung health established by age 6 years. However, little is known about lung health trajectories in DS or how aspiration and lung disease in childhood leads to morbidity in Down syndrome across the lifespan. To improve respiratory health over the lifespan, this study aims to determine the effect of aspiration on lung function and inflammation in children with Down syndrome. This study is a case-control evaluation of children with Down syndrome who aspirate and those who do not aspirate. The overarching goal of this project is to identify quantifiable measures of pulmonary morbidity that can be used as outcome measures in clinical trials designed to treat and prevent aspiration in children with DS. This will be tested through AIM 1: Determine the effect of aspiration on lung function measured by oscillometry and spirometry with bronchodilator response and by six-minute walk in children with Down syndrome, and AIM 2: Determine the effect of aspiration on inflammatory blood markers in children with DS. AIM 3: Determine the effect of aspiration on caregiver-reported respiratory symptoms and quality of life. The hypothesis is that aspiration causes measurable changes in lung function, inflammatory markers, and caregiver-reported symptom and quality of life outcomes distinct from asthma in children with DS. The study will include 75 children between 3 and 18 years with DS who have had a clinical swallow study diagnosing aspiration or no aspiration. The lung function, respiratory symptom, and quality of life assessments will be repeated after 3-12 months for a subset of 40 participants. The proposed study will characterize objective lung function and airway inflammation measurements in children with DS. Establishing these measures for participant identification and clinical outcomes will enable the design of clinical trials that test treatments to diminish the effects of aspiration on lung function and inflammation and reduce respiratory disease in children with DS, which will improve respiratory health across their lifespan.

呼吸道疾病是唐氏综合症患者最常见的死亡原因。患有唐氏病的儿童 综合症有很高的误吸风险，慢性误吸可导致呼吸道疾病，包括 肺炎、肺炎和支气管扩张。无唐氏综合症儿童的流行病学研究 研究表明，儿童时期的呼吸系统健康是整个一生中呼吸系统健康的有力预测因素， 6 岁时建立的肺部健康轨迹。然而，人们对肺部健康轨迹知之甚少。 DS 或儿童时期的误吸和肺部疾病如何导致整个生命周期中唐氏综合症的发病。 为了改善一生中的呼吸系统健康，本研究旨在确定误吸对肺部的影响 唐氏综合症儿童的功能和炎症。本研究是一项针对儿童的病例对照评估 患有唐氏综合症的人有误吸和无误吸。该项目的总体目标是 确定可量化的肺部发病率指标，可用作临床试验的结果指标 旨在治疗和预防 DS 儿童误吸。这将通过 AIM 1 进行测试：确定 通过示波法和肺活量测定法以及支气管扩张剂反应测量误吸对肺功能的影响 唐氏综合症儿童步行六分钟，目标 2：确定抽吸对唐氏综合症儿童的影响 DS 儿童的炎症血液标志物。目标 3：确定误吸对护理人员报告的影响 呼吸道症状和生活质量。假设是误吸会导致肺部发生可测量的变化 功能、炎症标志物以及护理人员报告的症状和生活质量结果与 DS 儿童哮喘。该研究将包括 75 名 3 至 18 岁患有 DS 的儿童，他们曾接受过 诊断误吸或无误吸的临床吞咽研究。肺功能、呼吸系统症状和质量 3-12 个月后，将对 40 名参与者进行重复的生活评估。拟议的研究将 描述 DS 儿童的客观肺功能和气道炎症测量结果。建立 这些用于参与者识别和临床结果的措施将使临床试验的设计能够测试 减少误吸对肺功能和炎症影响并减少呼吸道疾病的治疗 患有 DS 的儿童，这将改善他们一生中的呼吸系统健康。

项目成果

Airwave oscillometry to measure lung function in children with Down syndrome.

气波示波法测量唐氏综合症儿童的肺功能。

• 发表时间: 2022-06
• 影响因子: 3.6
• 作者: Vielkind, Monica L;Hamlington, Katharine L;Wolter;Meier, Maxene R;Liu, Andrew H;Hickey, Francis J;Brown, Mark A;DeBoer, Emily M
• 通讯作者: DeBoer, Emily M