Nebulised Hypertonic Saline to Decrease Respiratory Exacerbations in Neuromuscular Disease or Neurodisability

Full courses of antibiotics as prescribed for respiratory infections, both oral and intravenous, (excluding prophylactic antibiotic prescriptions). 1 course of antibiotic would be the full treatment for one event of respiratory infection, irrespective of the number of days that the course was prescribed for.

measured by multiple breath washout

Respiratory resistance (Rrs)

Respiratory reactance (Xrs).

Global Lung ultrasound score. The global lung ultrasound score (LUS) quantifies lung aeration by translating lung ultrasound patterns into a numerical score across 12 lung regions (six areas on each side of the chest: two ventral regions, two lateral regions, and two posterolateral regions) and summing the results. The aeration pattern observed in each region is scored from 0 to 3 as follows: 0 = A pattern with ≤2 B lines; 1 = >2 separated B lines that cover ≤50% of the pleural line; 2 = B lines that cover >50% of the pleural line; or 3 = lung consolidation. In theory, the global LUS score can range from 0 (normal aeration in all regions) to 36 (severe abnormal aeration in all regions).

Electrical impedance tomography (EIT)-based global inhomogeneity index (quantification of homogeneity of the tidal volume distribution). The image matrix in EIT consists of 32 × 32 pixels. Global inhomogeneity (GI) is calculated as the sum of the absolute differences between the median value of tidal variation and every single pixel value, divided by the sum of all impedance values, to normalise the calculated values.The smaller the GI, the more homogeneous the tidal volume is distributed within the ventilated area. A GI of zero represents a perfectly homogeneous distribution of ventilation.

Levels of IL-8 in sputum or throat swab.

Levels of IL-6 in sputum or throat swab.

Levels of TNF-a in sputum or throat swab.

Levels of IL-1b in sputum or throat swab.

Operational taxonomic unit (OTU) Richness, defined as count of different species/OTUs.

Pielou's eveness index. Pielou's evenness is an index that measures diversity along with species richness.

Shannon diversity index. The index takes into account the number of species living in a habitat (richness) and their relative abundance (evenness).

Bray-Curtis dissimilarity index. Examines the abundances of microbes that are shared between two samples, and the number of microbes found in each.

0-10 Visual analogue scale, where 0 is most easy, and 10 is most difficult.

Pediatric Quality of Life Inventory (PedsQL)™. 0-100 scale, where higher scores indicate better HRQOL (Health-Related Quality of Life).

Treatment Satisfaction Questionnaire for Medication (TSQM Version 1.4). Scores range from 0 to 100, with higher scores indicating higher satisfaction.

PedsQL™ Family Impact Module. The scale has five Likert response options, 'never', 'almost never', 'sometimes', 'often' and 'almost always' (corresponding to scores of 100, 75, 50, 25 and 0). Regarding the interpretation of the scale, higher scores indicate better functioning (less negative impact).

Quality-adjusted life years

Number of participants recruited per centre per month.

Percentage of eligible participants who consented and were randomised.

Percentage of randomised participants retained with valid primary outcome data.

Mean percentage of adherence calculated from returned ampoules count

Pick-up rate as the percentage of picked up prescriptions from total prescribed doses

The Medication Adherence Report Scale (MARS) Score, where each of the 5 items are summed to give a scale score ranging from 5 to 25, where higher scores indicate higher levels of reported adherence.

Percentage of compliance with completion of monthly questionnaires.

Percentage of participants who provided a sputum sample or throat swab

Percentage of participants who completed acceptable measurements of Lung clearance index

Percentage of participants who completed acceptable measurements of Forced oscillation technique

Percentage of participants who completed acceptable measurements of Lung ulstrasound

Percentage of participants who completed acceptable measurements of Electrical impedance tomography

Time in minutes to complete acceptable measurements of Lung clearance index

Time in minutes to complete acceptable measurements of forced oscillation technique

Degree of agreement among independent observers using Cohen Kappa. Cohen suggested the Kappa result be interpreted as follows: values ≤ 0 as indicating no agreement and 0.01-0.20 as none to slight, 0.21-0.40 as fair, 0.41- 0.60 as moderate, 0.61-0.80 as substantial, and 0.81-1.00 as almost perfect agreement.

Degree of agreement among independent observers using Cohen Kappa. Cohen suggested the Kappa result be interpreted as follows: values ≤ 0 as indicating no agreement and 0.01-0.20 as none to slight, 0.21-0.40 as fair, 0.41- 0.60 as moderate, 0.61-0.80 as substantial, and 0.81-1.00 as almost perfect agreement.