Efficacy of Intermittent Abdominal Pressure Ventilation in Neuromuscular Patients

Study Overview
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Brief Summary
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Non-Invasive Ventilation (NIV) is an established treatment to manage respiratory muscles dysfunction in neuromuscular disease, preventing the progression of respiratory failure to intubation and/or a tracheotomy. NIV is commonly needed at first during the night, but when the disease worsens, it is required during the day. It is provided via nasal or oronasal masks, causing discomfort and/or aesthetic issues that result in poor compliance. Intermittent Abdominal Pressure Ventilation (IAPV) is a valid, though unconventional, alternative to daytime NIV: it consists of a portable ventilator with an internal battery and a corset as interface. The IAPV corset is lightweight, comfortable and, thanks to velcro fasteners, easier and better fitting than a face mask. Cyclical inflation of a rubber bladder inside the corset moves the diaphragm upwards like a pneumobelt causing air to enter in the lungs via the upper airways as gravity draws the diaphragm back to its resting position. IAPV is indicated in neuromuscular disease and has already been tested in few preliminary studies and case reports. This study wants to verify the hypothesis of its application in population of neuromuscular patients.

Official Title
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Efficacy of Intermittent Abdominal Pressure Ventilation in Neuromuscular Patients: Multicentre, Randomised Controlled Trial

Conditions
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Neuromuscular Diseases

Intervention / Treatment
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* Device: Intermittent Abdominal Pressure Ventilation
* Device: Usual ventilation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of Neuromuscular Disease (Amyotrophic Lateral Sclerosis, Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Pompe Disease) Non Invasive Ventilation > 16 hours/day Informed consent signed Exclusion Criteria: - Diagnosis of kyphoscoliosis
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
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Intermittent Abdominal Pressure Ventilation<br>Patients in experimental group will be adapted to Intermittent Abdominal Pressure Ventilation (IAPV). Abdominal ventilation replaces part of usual ventilation (non invasive ventilation with mouthpiece or nasal-pillow) | Device: Intermittent Abdominal Pressure Ventilation<br>* Patients of experimental group will use IAPV in daytime ventilation. Intermittent abdominal pressure ventilation (IAPV) is a portable ventilator with an internal battery and PneumoBelt corset as an interface.<br>* Other names: IAPV;|
| Active Comparator: Usual ventilation<br>Patients in control group continue with usual ventilation (NIV through mouthpiece or nasal-pillow) | Device: Usual ventilation<br>* NIV<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Blood Gas Analysis | Changes of respiratory parameters in terms of normalization of oxyemia (Arterial Pressure of oxygen 80-100 mmHg) and normalization of capnia (Arterial Pressure of carbon dioxide 35-45 mmHg), assessed by blood gas analysis | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Therapy adherence | Adherence to IAPV tested with Beliefs about Ventilation Questionnaire (BVQ). BVQ is a qualitative questionnaire: it consist of 25 items, where higher scores mean a better outcome. | 12 months | 
| Patients satisfaction to IAPV | Satisfaction to IAPV tested with Visual Analog Scale (VAS), with a score that ranges between 0 and 10, where a higher score means a worse outcome. | 12 months | 
| Caregivers satisfaction to IAPV | Satisfaction to IAPV tested with Visual Analog Scale (VAS), with a score that ranges between 0 and 10, where a higher score means a worse outcome. | 12 months | 
| Amelioration of Quality of life | Improvement of quality of life tested with World Health Organization Quality of Life-Brief (WHOQOL-Brief), with a score ranging from 0 to 100 for each of the four domain and where a higher score means a better outcome | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Neuromuscular Diseases;, Intermittent Abdominal Pressure Ventilation;, IAPV;