Neuromuscular Diseases in Children
Conditions
Brief summary
Neuromuscular diseases (NMD) in children are severe, possibly life-threatening orphan conditions. The children are vulnerable and often subject to rapid deterioration of pulmonary function due to impaired ability to clear airway secretions. The use of mechanical cough augmentation with insufflation-exsufflation (MIE) is a strategy to treat and prevent. Possible major benefits are described, but optimal settings for best efficacy and comfort in children are not established. The project aim to improve the quality of the treatment with MIE in children with NMD and weak cough. On the basis of a bench study a clinical trial aims to examine the most effective MIE settings when used in stable state and when respiratory tract infections are present.
Detailed description
On the basis of a prevalence study and a bench study different settings will be studied in children with NMD and weak cough to examine if settings derived from the lung model, are optimal to increase the peak cough flow (PCF) and comfort measured by visual analogue scale (VAS) in children with NMD and weak cough.
Interventions
OTHERSymmetric settings (high pressures/fast rate)
Describes settings on the MI-E device
OTHERAsymmetric settings (Pi<Pe/Ti>Te)
Lower insufflation pressures at longer times
OTHERPreinclusion settings
Settings in use at start of study
Sponsors
Oslo University Hospital
Study design
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Participant, care provider, investigator and outcomes assessor are blinded from the order of the settings tested. Only a technician change settings and know the order.
Intervention model description
Three different settings are tested on the same patients but in randomized order.
Eligibility
Sex/Gender
ALL
Age
6 Months to 18 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosed neuromuscular disease \< 18 years * Established use (\> 3mnd) of MI-E. * Reduced PCF * PCF \< 270 l/min (when \> 12 years) * \< 5th percentiles for PCF 16 (when 4 - 12 years) * Clinical indication (difficulty to clear secretions, audible weak cough, history of pneumonia or frequent or prolonged respiratory tract infections).
Exclusion criteria
* age \< 6 mnd * obstructive lung disease (hyperinflation or emphysema on x-ray.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| PCF in the MI-E circuit | 30 minutes. | Recording of maximal value produced by the MI-E device during Cough |
| Patient reported comfort | Total time use 30 min | Comfort rated on a Visual Analog Scale (VAS) (0-100) where 100 is very uncomfortable and 0 is very comfortable. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Carbon dioxide | maximal time use is 30 min | Transcutaneous trend measurement of CO2 during data collection |
| Oxygen | maximal time use is 30 min | Transcutaneous trend measurement of peripheral O2 during data collection |
| Hart rate | Total max 30 minutes (During three MI-E trials) | Transcutaneous trend measurement of hart rate during data collection |
| Patient reported efficacy | The VAS is recorded after each of the three trials. Total time use 30 min | Efficacy rated on a Visual Analog Scale (0-100) where 100 is not efficient at all and 0 is very efficient. |
Countries
Norway
Outcome results
None listed