Respiratory Insufficiency Clinical Trial
Official title:
Study of Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive Ventilation in Adults With Myotonic Dystrophy Type 1(DM1)
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.
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 %.
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