Long-Term Effect of LIAM on Respiratory Performance in NIV Patients Suffering From Neuromuscular Disease

Study Overview
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Brief Summary
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The long-term effect of LIAM (Lung Insufflation Assist Maneuver) on respiratory performance in home non-invasively ventilated (NIV) patients suffering from neuromuscular disease will be assessed in a prospective, randomized, cross over, open label study

Detailed Description
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In advanced neuromuscular disorders, respiratory complications represent the main cause of morbidity and mortality. Beside chronic respiratory insufficiency, necessitating a ventilatory support, mostly performed by non-invasive ventilation (NIV), cough is impaired due to the muscle weakness, and respiratory physiotherapy becomes an essential part of the management. Various techniques have been proposed to improve lung recruitment and cough in neuromuscular patients. The assisted techniques based on a positive pressure insufflation maneuver have shown an improvement in physiological variables on the short term, but there is to date no good-quality prospective study allowing to evaluate the long term efficacy of mechanical cough assistance devices in neuromuscular patients. We designed a randomized, cross over, open label study to assess the long-term effect of LIAM (Lung Insufflation Assist Maneuver) on respiratory performance in NIV patients suffering from neuromuscular disease.

Official Title
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Long-Term Effect of LIAM (Lung Insufflation Assist Maneuver) on Respiratory Performance in Non-invasive Ventilated Patients Suffering From Neuromuscular Disease

Conditions
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Neuromuscular Disease

Intervention / Treatment
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* Device: non invasive mechanical ventilation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: o neuromuscular disease ( progressive muscular dystrophy, for example; Duchenne muscular dystrophy, spinal muscular atrophy) age ≥ 18 years indication to NIV (ongoing ventilation or new patients) vital capacity ≤ 50% pred peak cough flow < 270 l/min LIAM Responder = increased insufflation capacity (≥ + 50%) and Peak Cough Flow (PCF ≥+ 50%) with LIAM Exclusion Criteria: o acute respiratory failure (respiratory acidosis) home treatment by instrumental cough assistance in the preceding 12 months ongoing medical treatment of the neuromuscular disease (for ex: corticosteroids in Duchenne disease, enzyme therapy in Pompe disease) previous pneumothorax plan of legal protection pregnant or breastfeeding women failure to cooperate no affiliation to a social security scheme

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: non invasive mechanical ventilation<br>Patients suffering from neuromuscular disease with NIV indication and cough inefficiency | Device: non invasive mechanical ventilation<br>* instrumental increase of inspiratory capacity and cough<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in the maximal assisted vital capacity (maximal insufflation capacity) between the begin of the treatment period and the 3-months follow up | Change in the maximal assisted vital capacity (maximal insufflation capacity) between the begin of the treatment period and the 3-months follow up | 15 minutes | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| spontaneous vital capacity | spontaneous vital capacity change at 3 months | 15 minutes | 
| peak flow | peak cough flow (spontaneous / assisted) change at 3 months | 15 minutes | 
| optoelectronic plethysmography | change at 3 months in the distribution in ventilation (assessed by optoelectronic plethysmography), spontaneous/assisted | 1 hour | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Instrumental cough assistance, positive pressure insufflation maneuver, home mechanical ventilation, prospective evaluation