E-monitoring of Pulmonary Function in Patients With Duchenne Muscular Dystrophy Undergoing Respiratory Rehabilitation at Home"
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 2
- 主要终点
- Change from Baseline of the mean Forced Vital Capacity in Liters measured by home and hospital spirometry in DMD participants with vs without respiratory telerehabilitation
研究概览
简要总结
Duchenne muscular dystrophy (DMD) is the most common, progressive, irreversible muscular dystrophy. The pulmonary function is crucial for the duration of life in this disease. The European Respiratory Society is currently focused on digital health, seeking to define the realistic innovations for digital respiratory medicine to support professionals and patients during the COVID-19 pandemic. This study aimed to investigate whether it is possible to monitor pulmonary function at home by using an individual electronical spirometry system in children with Duchenne muscular dystrophy DMD. The second aim of the study is the implementation of respiratory telerehabilitation and the assessment of its impact on pulmonary function (FVC).
详细描述
Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy. DMD is a genetically determined, progressive, irreversible disease in which dystrophin dysfunction in skeletal and multiple organ muscles is fatal before the age of 20 years. Respiratory muscle failure is the most common cause of death.
In the first years of life, the respiratory system of children with DMD is efficient and does not differ from that of healthy peers. From the age of 7 years, the parameters of lung function no longer increase, and between the ages of 10 to 12 years, when the child loses the ability to walk independently, the lung function rapidly deteriorates.
Currently, it is not possible to cure the disease, but appropriate medical management may improve the quality of life and prolong the survival of patients with DMD.
The basic tasks include the initiation of early monitoring of respiratory system functions. It is recommended that measurement of lung function is started from the age of 5 years. Taking the measurement at such an early age is aimed at familiarizing and teaching the child about this type of systematic examination and determining the individual maximum parameters of lung function in each child. Thanks to systematic measurements, it is possible to detect any sharp deterioration as well as the moment when the decreasing lung function requires respiratory support, so-called non-invasive ventilation (NIV).
According to the standards, lung function is assessed by spirometry, which should be performed at least once a year, at least every 6 months after losing independent walking, and every 3 months after starting non-invasive ventilation.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Factorial
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 7 Years 至 17 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •male, ≥7 years and <18 years of age at the time of enrollment in the study;
- •ability to perform spirometry;
- •stated willingness to comply with all study procedures and availability for the duration of the study.
排除标准
- •no consent to participate in the study;
- •patients under 7 years of age or above 18 years of age;
- •inability to perform spirometry
结局指标
主要结局
Change from Baseline of the mean Forced Vital Capacity in Liters measured by home and hospital spirometry in DMD participants with vs without respiratory telerehabilitation
时间窗: 12 months
Some studies showed that respiratory function declines at a rate of 6-11% annually in patients with DMD. A major component of respiratory dysfunction seems to be a decline of inspiratory muscle weakness. Methods of improving the functioning of the muscles of the respiratory system are constantly sought. One of the key factors that can improve the function of respiratory muscles is proper rehabilitation. The proposition is implementation of telerehabilitation of respiratory muscle together with e-monitoring pulmonary function at home.
Change from Baseline of the mean Forced Vital Capacity in %predicted value measured by home and hospital spirometry in DMD participants with vs without respiratory telerehabilitation
时间窗: 12 months
Some studies showed that respiratory function declines at a rate of 6-11% annually in patients with DMD. A major component of respiratory dysfunction seems to be a decline of inspiratory muscle weakness. Methods of improving the functioning of the muscles of the respiratory system are constantly sought. One of the key factors that can improve the function of respiratory muscles is proper rehabilitation. The proposition is implementation of telerehabilitation of respiratory muscle together with e-monitoring pulmonary function at home.
次要结局
- The number of the participants who performed at least one correct spirometry examination(4 weeks)
- the difference in the value of spirometry results (FVC %pv, L) between home spirometry and spirometry in the hospital(4 weeks)
- Possibility of home e-monitoring of pulmonary function in patients with Duchenne Muscular Dystrophy in the Covid-19 pandemic(4 weeks)