Natural History of Cardiac and Respiratory Function in Patients With Muscular Dystrophies on Home Mechanical Ventilation
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 350
- 试验地点
- 1
- 主要终点
- predictive factors for long term survival
研究概览
简要总结
Muscular dystrophies are inherited disorders that affect skeletal muscle. Cardiac and respiratory function may be affected in this group of diseases. The investigators sought to analyze the long term cardiac and respiratory function in patients with muscular dystrophies recquiring home mechnaical ventilation .
详细描述
Muscular dystrophies are neuromuscular disorders with disability and can affect respiratory muscle function and left ventricular function. Medical management of patients with cardiomyopathy relies mainly on angiotensin-converting-enzyme inhibitors and beta blockers. Respiratory management relies on home mechanical ventilation and couph assist.
The investigators aim
- to analyze cardiac and respiratory function over time in patients with muscular dystrophies followed at the home mechanical ventilation unit.
- to determine the long term impact of the home mechanical ventilation on cardiac and respiratory events and to define the predictive factors associated.
Patients will be included retrospectively. Tissue Doppler imaging and Tricuspid annular plane systolic excursion were used for the assessment of the right ventricle. Left ventricular ejection fraction was used for the analysis of the left ventricle. Respiratory function was assessed using the vital capacity, the maximal inspiratory pressure, the maximal expiratory pressure, the peak couph flow rate.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age higher or equal to 18 years
- •Duchenne muscular dystrophy
- •Becker muscular dystrophy
- •Sarcoglycanopathy
- •limb girdle muscular dystrophy
- •Pompe disease
- •Steinert disease
- •FSH disease
- •Others myopathies
排除标准
- •Minor patient
- •Sepsis condition
结局指标
主要结局
predictive factors for long term survival
时间窗: 10 years
次要结局
- predictuve factors for cardiac events(10 years)
- predictive factors for respiratory events(10 years)
- cumulative cardiac events incidence(10 years)
- cumulative respiratory events incidence(10 years)
- annual rate of maximal inspiratory pressure decline(10 years)
- annual rate of maximal expiratory pressure decline(10 years)
- annual rate of left ventricular ejection fraction decline(10 years)
- annual rate of respiratory vital capacity decline(10 years)