Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV

Study Overview
=================
Brief Summary
-----------------
SMA (Spinal Muscular Atrophy) is a rare neuromuscular disease characterized by motoneuron damage. Symptoms consist of respiratory involvement with numerous respiratory infections and eventually respiratory failure, for which NIV (Non Invasive Ventilation) is often used. Ventilation machines are in close contact with the respiratory tract of patients. They contain heated water to humidify the circuit. These humid and warm environments are conducive to the development of bacteria such as Pseudomonas aeruginosa. In this context, it is interesting to look for the presence or absence of bacteria, in comparison with the respiratory ecology of the patients. The aim is to highlight the microbiological role of NIV on the occurrence of respiratory bacterial infections or secondary infections in patients with SMA. To do this, samples are taken from the machines, and ECBCs are performed on patients during respiratory physiotherapy sessions.

Official Title
-----------------
Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV

Conditions
-----------------
Non Invasive Ventilation, Spinal Muscular Atrophy, Respiratory Bacterial Infection

Intervention / Treatment
-----------------
* Other: non invasive ventilation samples
* Other: patient samples
* Other: parent questionnaire


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Diagnosis of AMS (previous diagnosis or new diagnosis during the inclusion period) Age from 0 to 12 years old Nusinersen treatment Benefiting from a NIV Current monitoring at the University Hospital of Amiens Exclusion Criteria: Invasive ventilation on tracheotomy No ventilation Other treatment than Nusinersen or no treatment Parental refusal to participate in the study

Ages Eligible for Study
-----------------
Maximum Age: 12 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: non invasive ventilation<br>SMA patients with NIV | Other: non invasive ventilation samples<br>* machine samples for bacterial culture will be performed<br>Other: patient samples<br>* Patient samples for sputum culture<br>Other: parent questionnaire<br>* parent questionnaire<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| number of hospitalization days for respiratory exacerbations with germs identical to protocol samples |  | one year | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
non invasive ventilation, Spinal Muscular Atrophy, Respiratory Bacterial Infection