Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive (DYVINE)

• Rate of patients having a complication (number of non expected hospitalization or death) at 5 years. [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
  Rate of patients having a complication (number of non expected hospitalization or death) at 5 years.
  
  

• Distribution of survival between the randomisation at 5 years [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
  Distribution of survival between the randomisation at 5 years
  
  
• Number of patients having a formal indication of ventilation [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
  Number of patients having a formal indication of ventilation
  
  
• Number of tracheostomized patients at 5 years [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
  Number of tracheostomized patients at 5 years
  
  
• Number of non expected hospitalizations at 5 years [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
  Number of non expected hospitalizations at 5 years
  
  
• Observance of the ventilation defined by an average minimal use of 4 am by 12:00 pm . [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
  Observance of the ventilation defined by an average minimal use of 4 am by 12:00 pm (determined in a objective way by the counter of the device).
  
  
• Degree of respiratory and sleep impairment at 5 years [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
  Degree of respiratory and sleep impairment at 5 years
  
  
• Quality of life SF36, scales of depression [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
  Quality of life SF36, scales of depression
  
  

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:

Multicenter, national, randomized, controlled, study on 2 parallel groups. The subjects presenting a theoretical indication following consensual criteria of ventilation will be randomized either for a start up of ventilation or for an annual monitoring.

Hypothesis: Early starting of non invasive ventilation allows a reduction of 20 % of the mortality or the number of non-programmed hospitalization compared to the control group for which the rate would be 40 %.