Impact of the Type of Interface in Neuromuscular Patients Treated With Nocturnal Noninvasive Ventilation

Study Overview
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Brief Summary
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Nocturnal Non Invasive Ventilation (NIV) is the reference treatment for chronic alveolar hypoventilation in patients with neuro-muscular diseases. NIV can be provided by using different types of interfaces: Nasal masks are the most frequent type of interface used at home but oronasal masks are used by at least 25% of neuro-muscular patients mainly because of persistent unintentional mouth leaks. However, oronasal mask may cause persistent upper airway obstructive respiratory events because of the mechanical constraint on the chin induced by the traction of the straps that may push the mandible posteriorly during sleep. No randomized study has specifically addressed the question of the impact of type of interface in patients with neuromuscular diseases treated by nocturnal NIV. The investigators hypothesize that: the application of oronasal mask may jeopardize the pharyngeal patency in patients already proned to upper airway obstruction; the use of a nasal mask may improve upper airway stability and NIV efficacy while reducing side effects. Authors objective will be to compare the impact of nasal mask versus oronasal mask on NIV efficacy and side-effects. Eligible patients are those with nocturnal NIV and neuromuscular disease. After a scheduled hospital visit, patients willing to participate will undergo in random order 2 unattended nocturnal polygraphies under NIV at home: one polygraphy with nasal mask; one with an oronasal mask. Each polygraphy ans side effects assessment will be performed after one week of familiarization with each mask.

Official Title
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Impact of the Type of Interface in Neuromuscular Patients Treated With Nocturnal Noninvasive Ventilation: a Randomized Crossover Trial

Conditions
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Neuromuscular Diseases, Alveolar Hypoventilation

Intervention / Treatment
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* Device: Switch of nocturnal NIV interface


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Adult patients (>18 years old) Affected by slowly progressive neuro-muscular diseases (Becker muscular dystrophy, facio-scapulo-humeral dystrophy, limb-girdle dystrophy, myotonic dystrophy…) or relatively rapid progression (Duchenne muscular dystrophy). Treated with nocturnal non-invasive ventilation (<15 hours/day) In stable state (no cardiorespiratory or ear-nose-throat event for at least 1 month before inclusion) Exclusion Criteria: Rapidly progressive neuro-muscular diseases (such as ALS) Severe nasal obstruction, maxillofacial deformities or previous upper airway surgery preventing the usage of one type of mask (nasal or oronasal), or, at the discretion of investigator, any other contraindication for using the other type of mask NIV Daily use >15h/day Unwillingness or inability to provide consent to participation Curatorship Subject in exclusion period of another study Vulnerable person or legally protected adult.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 99 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: Supportive Care
Allocation: Randomized
Intervention Model: Crossover Assignment
Interventional Model Description: The patients will be randomized for two periods of one week to use NIV either with nasal or oronasal mask. The randomization will be stratified according to the type of interface previously used at home by the patient (nasal and oronasal). Such stratification will allow a balance between the number of patients that will begin with their usual interface versus with an alternative interface. In these patients, NIV withdrawal is not acceptable in terms of patient's security and ethics. Therefore, it is not possible to include a wash out period between both arms. The potential carry-over effect will be taken into account in the statistical analysis.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Usual interface<br>Patients which begin with their usual interface for one week, home polygraphy and side effect assessment at the end of the first week. Switch for alternative interface, seven days familiarisation, second polygraphy and side effect assessment at the end of the second week. | Device: Switch of nocturnal NIV interface<br>* test the alternative interfaces (either a nasal mask for the patient used to oronasal mask or inversely an oronasal mask if the usual mask is nasal) during a one-hour diurnal NIV session. SpO2 (polygraphy), PtcCO2 will be monitored continuously during this diurnal session. Patients will use their usual NIV device prescribed at home. NIV settings will be adapted if needed. Interface switch unattended nocturnal polygraphy under NIV (cf details below) will be performed at home with SomnoHolter® (Nomics, Liege, Belgium), synchronized with transcutaneous partial pressure in CO2 (PtcCO2) monitoring by SenTec V-Sign™ System. The PtcCO2 device will be calibrated before and at the end of each night to allow drift correction.<br>|
| Active Comparator: Alternative interface<br>Patients which begin with the alternative interface for one week, home polygraphy and side effect assessment at the end of the first week. Switch for usual interface, seven days with usual device, second polygraphy and side effect assessment at the end of the second week. | Device: Switch of nocturnal NIV interface<br>* test the alternative interfaces (either a nasal mask for the patient used to oronasal mask or inversely an oronasal mask if the usual mask is nasal) during a one-hour diurnal NIV session. SpO2 (polygraphy), PtcCO2 will be monitored continuously during this diurnal session. Patients will use their usual NIV device prescribed at home. NIV settings will be adapted if needed. Interface switch unattended nocturnal polygraphy under NIV (cf details below) will be performed at home with SomnoHolter® (Nomics, Liege, Belgium), synchronized with transcutaneous partial pressure in CO2 (PtcCO2) monitoring by SenTec V-Sign™ System. The PtcCO2 device will be calibrated before and at the end of each night to allow drift correction.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean nocturnal oxygen saturation (SpO2) | Mean nocturnal SpO2, measured by oximetry. | After one week with each type of mask | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| % sleep recording with SpO2<90% | Percentage of sleep recording spent with SpO2<90% between oronasal mask versus nasal mask | After one week with each type of mask | 
| Oxygen Desaturation Index | Oxygen desaturation index between oronasal mask versus nasal mask | After one week with each type of mask | 
| Mean nocturnal PtcCO2 | Mean nocturnal transcutaneous partial pressure in CO2 (PtcCO2) evaluated by capnography between oronasal mask versus nasal mask | After one week with each type of mask | 
| Mean mouth opening during sleep | Mean mouth opening during sleep between oronasal mask versus nasal mask | After one week with each type of mask | 
| Non-intentional leaks | Non-intentional leaks recorded by the NIV-device | After one week with each type of mask | 
| Side-effects of Continuous Positive Airway Pressure (CPAP) | Side-effects reported by patients using a modified version of Side Effect of CPAP inventory (SECI) questionnaire. A French translation will be done by two bilingual investigators (one medical doctor, one linguist). This questionnaire consists of a list of 15 commonly reported side effects under CPAP. For each side effect, the patient is asked to rate the frequency (0-5), magnitude (0-5) and perceived impact on adherence (0-5) on a five-point Likert-type scale. Total score range : 0 to 225, with the higher score associated with the worst tolerance. Range for each of the fifteen side effects: 0 to 15, higher values always represent a worse outcome (all subscales results are summed to compute the total score) | After one week with each type of mask | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Non-invasive ventilation, Nasal mask, Oronasal mask, Alveolar hypoventilation, leaks, Neuromuscular diseases, Sleep disorder breathing, Side effects