Passive stretch of the chest wall in patients with Congential Muscular Dystrophy
先天性肌营养不良症患者的胸壁被动拉伸
基本信息
- 批准号:8445541
- 负责人:
- 金额:$ 35.32万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2013
- 资助国家:美国
- 起止时间:2013-08-01 至 2015-05-31
- 项目状态:已结题
- 来源:
- 关键词:AdherenceAdoptedAdverse eventAffectAmericanBirthCaregiversCharacteristicsChestChest wall structureChildChildhoodCollagen Type VIContractureControl GroupsDataDeformityDevelopmentDiseaseEconomic InflationElasticityEnrollmentEnvironmental air flowExhalationFailureFutureGeneticHealth Care CostsIndividualInvestigationLeadLifeLungMeasuresMechanicsMorbidity - disease rateMotorMuscleMuscle WeaknessMuscular DystrophiesMyopathyNamesNeuromuscular DiseasesPatientsProcessProtocols documentationQuality of lifeRandomizedRandomized Controlled TrialsRare DiseasesRegimenResearchRespiratory FailureRespiratory MusclesRespiratory SystemSecondary toSocietiesStretchingTelephoneTestingTimeTissuesVital capacityVital capacity measurementabstractingbaseburden of illnesscongenital muscular dystrophydesigndiariesgroup interventionimprovedlung volumemeetingsmortalityneuromuscularpressurepublic health relevancepulmonary functionrespiratoryskeletalstandard of caretooltreatment duration
项目摘要
DESCRIPTION (provided by applicant):
Congenital muscular dystrophy (CMD) secondary to collagen VI deficiency is a distinct genetic neuromuscular disorder presenting from birth with progressive loss of motor function, skeletal deformities and debilitating contractures. Respiratory failure is the principal cause of morbidity and mortality from the disease. As CMD has no existing treatment, the majority of affected children develop respiratory failure and require non-invasive ventilation between the first and second decades of life. There is evidence that in muscular dystrophy the progressive decrease in lung volume is disproportionally greater than anticipated for the degree of respiratory muscle weakness. Chest wall stiffness and decreased lung elasticity which collectively diminish compliance of the respiratory system are believed to be important contributors to the disproportionate decrease in lung volume. Our research aims at testing the hypothesis that in subjects with CMD, daily passive stretch of the chest wall through lung hyperinsufflation therapy could slow down the annual rate of decline in lung volume, delay the onset of respiratory failure and improve quality of life. We will demonstrate that 1) By measuring in each subject the respiratory pressure volume relationship, maximum lung inflation by hyperinsufflation could be attained and the corresponding pressure estimated, 2) Subjects with CMD will be able to adhere to a twice-a-day 15 minute hyperinsufflation regimen over a period of 5 months and 3) Demonstrate that by studying for a period of one year 17 subjects enrolled in the intervention group and 17 in the observational group, the difference in the rate of decline of vital capacity between the 2 groups will yield an effect size of > 0.5. The results of this application will pave the way for the development of a future randomized control trial of daily passive stretch of the chest wall through lung hyperinsuflation in patients with neuromuscular disorders.
描述(由申请人提供):
先天性肌营养不良症(CMD)继发于VI型胶原缺乏,是一种独特的遗传性神经肌肉疾病,从出生起就表现为进行性的运动功能丧失、骨骼畸形和衰弱的肌挛缩。呼吸衰竭是该病发病率和死亡率的主要原因。由于CMD目前没有治疗方法,大多数受影响的儿童会出现呼吸衰竭,需要在生命的第一到第二个十年之间进行无创呼吸机。有证据表明,在肌营养不良症中,肺体积的进行性减少不成比例地大于呼吸肌无力程度的预期。胸壁僵硬和肺弹性降低共同降低了呼吸系统的顺应性,被认为是导致肺体积不成比例减少的重要因素。我们的研究旨在验证这一假设,即在CMD患者中,通过肺过度充气治疗每天被动拉伸胸壁可以减缓肺容量下降的年率,推迟呼吸衰竭的发生,并提高生活质量。我们将证明:1)通过在每个受试者中测量呼吸压力与容量的关系,可以通过过度充气获得最大肺膨胀并估计相应的压力;2)CMD受试者将能够在5个月的时间内坚持每天两次15分钟的过度充气方案;3)通过为期一年的研究,进入干预组的17名受试者和观察组的17名受试者,两组肺活量下降速度的差异将产生>;0.5的有效大小。这项应用的结果将为未来通过肺高胰岛素对神经肌肉疾病患者进行每日被动胸壁拉伸的随机对照试验的发展铺平道路。
项目成果
期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
数据更新时间:{{ journalArticles.updateTime }}
{{
item.title }}
{{ item.translation_title }}
- DOI:
{{ item.doi }} - 发表时间:
{{ item.publish_year }} - 期刊:
- 影响因子:{{ item.factor }}
- 作者:
{{ item.authors }} - 通讯作者:
{{ item.author }}
数据更新时间:{{ journalArticles.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ monograph.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ sciAawards.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ conferencePapers.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ patent.updateTime }}
Raouf S. Amin其他文献
Upper Airway Volume Segmentation Analysis Using Cine MRI Findings in Children with Tracheostomy Tubes
使用气管切开插管儿童的电影 MRI 结果进行上气道体积分割分析
- DOI:
- 发表时间:
2007 - 期刊:
- 影响因子:4.8
- 作者:
Bradley L. Fricke;M. Bret Abbott;Lane F. Donnelly;B. Dardzinski;S. Poe;M. Kalra;Raouf S. Amin;Robin T. Cotton - 通讯作者:
Robin T. Cotton
Chapter 14 – Chronic Respiratory Failure
第 14 章 – 慢性呼吸衰竭
- DOI:
- 发表时间:
2006 - 期刊:
- 影响因子:0
- 作者:
Raouf S. Amin - 通讯作者:
Raouf S. Amin
Obliterative bronchiolitis in a 13-year-old pre-transplant cystic fibrosis patient
- DOI:
10.1016/j.jcf.2007.05.006 - 发表时间:
2008-01-01 - 期刊:
- 影响因子:
- 作者:
Patrick O. Sobande;James D. Acton;Raouf S. Amin;Jeanne Weiland - 通讯作者:
Jeanne Weiland
Raouf S. Amin的其他文献
{{
item.title }}
{{ item.translation_title }}
- DOI:
{{ item.doi }} - 发表时间:
{{ item.publish_year }} - 期刊:
- 影响因子:{{ item.factor }}
- 作者:
{{ item.authors }} - 通讯作者:
{{ item.author }}
{{ truncateString('Raouf S. Amin', 18)}}的其他基金
Randomized Control Trial of oxygen therapy in Children and Adolescents with Down Syndrome and Obstructive Sleep Apnea
唐氏综合症和阻塞性睡眠呼吸暂停儿童和青少年氧疗的随机对照试验
- 批准号:
10838939 - 财政年份:2022
- 资助金额:
$ 35.32万 - 项目类别:
Randomized Control Trial of oxygen therapy in Children and Adolescents with Down Syndrome and Obstructive Sleep Apnea
唐氏综合症和阻塞性睡眠呼吸暂停儿童和青少年氧疗的随机对照试验
- 批准号:
10518497 - 财政年份:2022
- 资助金额:
$ 35.32万 - 项目类别:
Personalized Cystic Fibrosis Therapy and Research Center
个性化囊性纤维化治疗和研究中心
- 批准号:
10672703 - 财政年份:2018
- 资助金额:
$ 35.32万 - 项目类别:
Personalized Cystic Fibrosis Therapy and Research Center
个性化囊性纤维化治疗和研究中心
- 批准号:
10672708 - 财政年份:2018
- 资助金额:
$ 35.32万 - 项目类别:
Regional monitoring of CF lung disease after changes in mechanical airway-clearance treatment
机械气道清除治疗改变后 CF 肺部疾病的区域监测
- 批准号:
10737221 - 财政年份:2016
- 资助金额:
$ 35.32万 - 项目类别:
UTE MRI to monitor CF lung disease and response to CFTR modulation in young children
UTE MRI 用于监测幼儿 CF 肺部疾病和对 CFTR 调节的反应
- 批准号:
9896865 - 财政年份:2016
- 资助金额:
$ 35.32万 - 项目类别:
Passive stretch of the chest wall in patients with Congential Muscular Dystrophy
先天性肌营养不良症患者的胸壁被动拉伸
- 批准号:
8708194 - 财政年份:2013
- 资助金额:
$ 35.32万 - 项目类别:
Cincinnati Children's Summer Medical Student Respiratory Research Fellowship
辛辛那提儿童夏季医学生呼吸研究奖学金
- 批准号:
10397502 - 财政年份:2012
- 资助金额:
$ 35.32万 - 项目类别:
Cincinnati Children's Summer Medical Student Respiratory Research Fellowship
辛辛那提儿童夏季医学生呼吸研究奖学金
- 批准号:
10630069 - 财政年份:2012
- 资助金额:
$ 35.32万 - 项目类别:
Dynamic Computational Modeling of Obstructive Sleep Apnea in Down Syndrome
唐氏综合症阻塞性睡眠呼吸暂停的动态计算模型
- 批准号:
8323924 - 财政年份:2010
- 资助金额:
$ 35.32万 - 项目类别:
相似海外基金
How novices write code: discovering best practices and how they can be adopted
新手如何编写代码:发现最佳实践以及如何采用它们
- 批准号:
2315783 - 财政年份:2023
- 资助金额:
$ 35.32万 - 项目类别:
Standard Grant
One or Several Mothers: The Adopted Child as Critical and Clinical Subject
一位或多位母亲:收养的孩子作为关键和临床对象
- 批准号:
2719534 - 财政年份:2022
- 资助金额:
$ 35.32万 - 项目类别:
Studentship
A comparative study of disabled children and their adopted maternal figures in French and English Romantic Literature
英法浪漫主义文学中残疾儿童及其收养母亲形象的比较研究
- 批准号:
2633211 - 财政年份:2020
- 资助金额:
$ 35.32万 - 项目类别:
Studentship
A material investigation of the ceramic shards excavated from the Omuro Ninsei kiln site: Production techniques adopted by Nonomura Ninsei.
对大室仁清窑遗址出土的陶瓷碎片进行材质调查:野野村仁清采用的生产技术。
- 批准号:
20K01113 - 财政年份:2020
- 资助金额:
$ 35.32万 - 项目类别:
Grant-in-Aid for Scientific Research (C)
A comparative study of disabled children and their adopted maternal figures in French and English Romantic Literature
英法浪漫主义文学中残疾儿童及其收养母亲形象的比较研究
- 批准号:
2436895 - 财政年份:2020
- 资助金额:
$ 35.32万 - 项目类别:
Studentship
A comparative study of disabled children and their adopted maternal figures in French and English Romantic Literature
英法浪漫主义文学中残疾儿童及其收养母亲形象的比较研究
- 批准号:
2633207 - 财政年份:2020
- 资助金额:
$ 35.32万 - 项目类别:
Studentship
The limits of development: State structural policy, comparing systems adopted in two European mountain regions (1945-1989)
发展的限制:国家结构政策,比较欧洲两个山区采用的制度(1945-1989)
- 批准号:
426559561 - 财政年份:2019
- 资助金额:
$ 35.32万 - 项目类别:
Research Grants
Securing a Sense of Safety for Adopted Children in Middle Childhood
确保被收养儿童的中期安全感
- 批准号:
2236701 - 财政年份:2019
- 资助金额:
$ 35.32万 - 项目类别:
Studentship
A Study on Mutual Funds Adopted for Individual Defined Contribution Pension Plans
个人设定缴存养老金计划采用共同基金的研究
- 批准号:
19K01745 - 财政年份:2019
- 资助金额:
$ 35.32万 - 项目类别:
Grant-in-Aid for Scientific Research (C)
Structural and functional analyses of a bacterial protein translocation domain that has adopted diverse pathogenic effector functions within host cells
对宿主细胞内采用多种致病效应功能的细菌蛋白易位结构域进行结构和功能分析
- 批准号:
415543446 - 财政年份:2019
- 资助金额:
$ 35.32万 - 项目类别:
Research Fellowships