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: 8445541
• 负责人: Raouf S. Amin
• 金额: $ 35.32万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 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: https://reporter.nih.gov/project-details/8445541
• 关键词: Adherence; Adopted; Adverse event; Affect; American; Birth; Caregivers; Characteristics; Chest; Chest wall structure; Child; Childhood; Collagen Type VI; Contracture; Control Groups; Data; Deformity; Development; Disease; Economic Inflation; Elasticity; Enrollment; Environmental air flow; Exhalation; Failure; Future; Genetic; Health Care Costs; Individual; Investigation; Lead; Life; Lung; Measures; Mechanics; Morbidity - disease rate; Motor; Muscle; Muscle Weakness; Muscular Dystrophies; Myopathy; Names; Neuromuscular Diseases; Patients; Process; Protocols documentation; Quality of life; Randomized; Randomized Controlled Trials; Rare Diseases; Regimen; Research; Respiratory Failure; Respiratory Muscles; Respiratory System; Secondary to; Societies; Stretching; Telephone; Testing; Time; Tissues; Vital capacity; Vital capacity measurement; abstracting; base; burden of illness; congenital muscular dystrophy; design; diaries; group intervention; improved; lung volume; meetings; mortality; neuromuscular; pressure; public health relevance; pulmonary function; respiratory; skeletal; standard of care; tool; treatment duration

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）： 先天性肌肉营养不良（CMD）继发于胶原蛋白VI缺乏症是一种独特的遗传神经肌肉疾病，该疾病从出生中出现，逐渐丧失运动功能，骨骼畸形和使人衰弱的闭合。呼吸衰竭是疾病发病率和死亡率的主要原因。由于CMD没有现有治疗，因此大多数受影响的儿童会出现呼吸衰竭，并且在生命的第一十年和第二十年之间需要无创的通风。有证据表明，在肌肉营养不良中，肺部体积的逐渐减少量比预期的呼吸肌无力较低。胸壁的刚度和肺部弹性降低，据信，呼吸系统的依从性统一是肺部体积不成比例下降的重要因素。我们的研究旨在检验以下假设：在患有CMD的受试者中，每天通过肺部超弹药治疗的胸壁被动延伸可能会降低肺部体积下降的年龄，延迟呼吸衰竭的发作并改善生活质量。我们将证明1）通过在每个受试者中测量呼吸压力之间的关系，可以达到高度填充的最大肺通货膨胀，并估计的相应压力，2）CMD的受试者能够遵守15天的15分钟15分钟的高度填充度，以表明在5个月内进行一年的间隔，并在17年中进行了17年的时间，并在17年中进行了17年的学位，并在17年中进行了17次介绍。两组之间生命力下降率的差异将产生效果大小> 0.5。该应用的结果将为未来的神经肌肉疾病患者的肺部过度膨胀的每日被动控制试验的发展铺平道路。