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

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.

• non invasive mechanical ventilation (Device): instrumental increase of inspiratory capacity and cough

Primary outcomes

• 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]: Change in the maximal assisted vital capacity (maximal insufflation capacity) between the begin of the treatment period and the 3-months follow up

Secondary outcomes

• spontaneous vital capacity [15 minutes]: spontaneous vital capacity change at 3 months
• peak flow [15 minutes]: peak cough flow (spontaneous / assisted) change at 3 months
• optoelectronic plethysmography [1 hour]: change at 3 months in the distribution in ventilation (assessed by optoelectronic plethysmography), spontaneous/assisted

Sex: All, ages: 18 Years to any

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

• David Orlikowski, MD, PhD, Principal investigator, Assistance Publique - Hôpitaux de Paris