Myotonic dystrophy type 1 is a myopathy with complex respiratory pattern and at risk to develop respiratory failure. Classical mode of ventilation are sometimes not tolerated or ineffective in this population. New modes of nocturnal ventilation by combining both volumetric and barometric advantages. The aim of this study is to compare effect of AVAPS mode to bilevel pressure support.
Justification of study Respiratory abnormalities are complex in Myotonic dystrophy type 1. Some patients presented with isolated alveolar hypoventilation and breathe rhythm irregularity. Nocturnal ventilation is usually proposed but usual modes of ventilation can't provide enough respiratory assistance for patients especially during REM sleep or too much respiratory assistance increasing the risk of asynchrony. The goal of this study is to evaluate the effect of the mode AVAPS (a mode permitting a pressure support with guaranteed volume and offering advantage of volume and pressure support with Bipap A30 Phillips Respironics) compared to bilevel pressure support. Main Objective To evaluate efficacy of AVAPS Mode at day 7 on arterial PCO2 under ventilation after launching ventilation. Secondary Objectives To evaluate efficacy of AVAPS Mode at day 90 on daytime arterial PCO2 after launching ventilation. To evaluate compliance to ventilation at day 7 and 90. To evaluate clinical efficacy on respiratory symptoms, dyspnea and sleepiness at day 1 and 90. To evaluate quality of life at day 1 and 90. To evaluate effect of AVAPS on polysomnography, nocturnal SaO2, nocturnal PtCO2. To evaluate Multiple sleep latency and Maintenance of wakefulness tests at day 90. To evaluate effect of AVAPS on respiratory parameters VC and mouth maximal pressures. Type of study: Prospective, monocentre, randomized, controlled single blind study on 2 parallel group. Number of subjects: 32 patients recruited in home ventilation unit of Raymond Poincaré hospital. Selection criteria : Patients with Myotonic dystrophy presenting at least one clinical signs : effort or rest dyspnea, orthopnea, sleepiness, morning headache or VC\<50% or Pi max\< 60 cm H2O or time of SaO2\<90% more than 5 minutes and Hypercapnia \> 6.0 kPa. Study process Preceding screening period within the 3 months before inclusion. Day 1 to day 3 baseline evaluation. Day 3 Inclusion and Randomisation Day 3 to 8 Launch of ventilation Day 8 Home discharge Day 90 Evaluation of efficacy (secondary objectives) and observance. Duration Participation of a patient 3 months. Period of inclusion 24 months. Total duration of study 30 months.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
32
Home ventilation only the mode AVAPS will be used if the patient is randomized in the experimental group.
Home ventilation Unit , Raymond Poincaré hospital
Garches, Paris Area, France
arterial PCO2 under ventilation
To evaluate efficacy of AVAPS Mode versus bilevel pressure mode at day 7 on arterial PCO2 under ventilation after launching ventilation
Time frame: 7 days
daytime arterial PCO2 after launching ventilation.
To evaluate efficacy of AVAPS Mode at day 90 on daytime arterial PCO2 after launching ventilation.
Time frame: 90 days
Compliance to ventilation
To evaluate compliance (h/24h) to ventilation at days 7 and 90.
Time frame: 7 and 90 days
Symptoms
To evaluate clinical efficacy on respiratory symptoms, dyspnea and sleepiness at day 1 and 90.
Time frame: 90 days
Sleep studies
To evaluate effect of AVAPS on polysomnography, nocturnal SaO2, nocturnal PtCO2 at day 90.
Time frame: 90 days
OBJECTIVE SLEEPINESS
To evaluate Multiple sleep latency and Maintenance of wakefulness tests at day 90.
Time frame: 90 days
Respiratory parameters
To evaluate effect of AVAPS on respiratory parameters VC and mouth maximal pressures.
Time frame: 90 days
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