Study of Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive Ventilation in Adults With Myotonic Dystrophy Type 1(DM1)
试验速览
- 阶段
- 3 期
- 入组人数
- 77
- 试验地点
- 1
- 主要终点
- Rate of patients having a complication (number of non expected hospitalization or death) at 5 years.
研究概览
简要总结
This is a multicenter randomized controlled open labeled study testing efficacy and tolerance of early launching of night non invasive ventilation in patients with myotonic dystrophy type 1(DM1). The object of this project is to estimate the effects of the early introduction of non invasive ventilation on the arisen of complication (non expected hospitalization, tracheostomy even death) with regard to a simple respiratory follow-up in patients affected by myotonic dystrophy.
详细描述
DM1 is the most frequent genetic myopathy in the adult. Actually there is no curative treatment, and symptomatic cares are essentials. The respiratory impairment is the main cause of morbid-mortality at these patients. The median of survival of the patients affected by DM1 with respiratory failure is of 59 years. Mechanisms of disease are complex implying a central and a direct impairment of respiratory muscles. These patients can present an alveolar hypoventilation, notably during night, not correlated to the muscular weakness. These patients often present a cognitive impairment complicating the interpretation of the clinical symptoms and their compliance to treatments. International recommendations for launching mechanical ventilation in neuromuscular diseases are the presence: 1) at least a clinical sign of alveolar hypoventilation, and one of the following criteria 2) diurnal hypercapnia (> 45 mmHg), 3) restrictive syndrome (vital capacity < 50 % and\or maximal inspiratory pressure < 60 cmH20), 4) the existence of a oxygen night-desaturation (SaO2 < 88 %) of more than 5 minutes. However, a Cochrane meta analyzes underline the absence of randomised study estimating the profit risk in the long term of the night-mechanical ventilation for progressive myopathies such as the DM1. The validity of these criteria and the effect of the ventilation on the survival and the complications were never estimated in DM1. On a retrospective series, the compliance is inferior and the observance is only 20 % a year and the incidence of the complications (death or resort to a tracheostomy) was 6 times as important in non observant patients.
Objective (s) of the clinical study To estimate the efficiency and the tolerance of long term night-non invasive mechanical ventilation in patients affected by DM1.
Main judgment criteria:
Mortality and non programmed hospitalization.
Experimental plan:
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Man or woman of age ≥ 18 years
- •Preliminary medical examination
- •Enlightened and written consent
- •Genetically proved Steinert disease
- •Presenting at least one of the following 3 criteria
- •A hypercapnia: PaCO2 > 45 mmHg or
- •A night-desaturation: SaO2 < 88 % more than consecutive 5 minutes or
- •Apnea syndrome with significant sleep:index of apnea / hypopnea> a 30 / hour
- •And with presence of at least a clinical sign: dyspnoea, orthopnea, headaches, asthenia, diurnal sleepiness, or any other sign suggestive of disturbance of the sleep or of respiratory dysfunction
排除标准
- •Age inferior to 18
- •Regime of legal protection
- •Pregnancy
- •Absolute indication for ventilation: clinical signs (dyspnoea, orthopnea, headaches, asthenia, diurnal sleepiness), AND PaCO2 > 60 mmHg, AND night-desaturation < 88 % AND one CV < 50 % of the theoretical or the PIMAX < 60 cm H2O
- •Acute respiratory failure
- •Already ventilated patient
- •Patient under oxygen
- •Not (beneficiary to a regime of Social Security or legal successor)
研究组 & 干预措施
bipap ventilation
干预措施: Bilevel pressure ventilator (Device)
Standard care
Standard care and ventilation if occurrence of absolute criteria of ventilation (cf infra).
干预措施: Bilevel pressure ventilator (Device)
结局指标
主要结局
Rate of patients having a complication (number of non expected hospitalization or death) at 5 years.
时间窗: 5 YEARS
Rate of patients having a complication (number of non expected hospitalization or death) at 5 years.
次要结局
- Quality of life SF36, scales of depression(5 YEARS)
- Number of non expected hospitalizations at 5 years(5 YEARS)
- Distribution of survival between the randomisation at 5 years(5 YEARS)
- Number of patients having a formal indication of ventilation(5 YEARS)
- Number of tracheostomized patients at 5 years(5 YEARS)
- Observance of the ventilation defined by an average minimal use of 4 am by 12:00 pm .(5 YEARS)
- Degree of respiratory and sleep impairment at 5 years(5 YEARS)