Effectiveness of early use of the mechanical insufflation- exsufflation compared to air stacking to reduce respiratory tract infections in patients with Duchenne muscular dystrophy.

Effectiveness of early use of the mechanical insufflation- exsufflation compared to air stacking to reduce respiratory tract infections in patients with Duchenne muscular dystrophy. - Cough

Status

Recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON53421

Enrollment

Registered

2023-04-03

Start date

2023-11-27

Completion date

Unknown

Last updated

2024-12-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

respiratory tract infections

Interventions

Both MI-E and AS with MAC should be used at least twice daily. For AS, the patients will be given a choice of using either an oronasal mask or a mouth piece. For MI-E an oronasal mask will be used.

Eligibility

Age

12 Years to 64 Years

Inclusion criteria

Inclusion criteria: Patientes with Duchenne muscular dystrofy older than 12 years Peak cough flow

Exclusion criteria

Exclusion criteria: recent pneumothorax, barotrauma and emphysema/bullae tracheostomy patients / legal representatives who do not speak Dutch of English

Design outcomes

Primary

Measure	Time frame
The primary endpoint of this study is the total number of RTIs during 2 year follow up. Definition of RTI is : The presence of one or more of the following symptoms or signs: deterioration of the clinical situation within one or a few days, characterised by worsening of dyspnoea or coughing, for which antibiotic treatment is initiated. Worsening of dyspnoea or coughing will be monitored in a diary by the patient, using visual analogue scale (VAS).	—

Measure

Time frame

The primary endpoint of this study is the total number of RTIs during 2 year follow up. Definition of RTI is : The presence of one or more of the following symptoms or signs: deterioration of the clinical situation within one or a few days, characterised by worsening of dyspnoea or coughing, for which antibiotic treatment is initiated. Worsening of dyspnoea or coughing will be monitored in a diary by the patient, using visual analogue scale (VAS).

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Secondary

Measure	Time frame
Secondary study parameters/endpoints Multiple secondary endpoints will be studied to compare MI-E treated participants to AS treated participants: 1. Severity of RTI a. Hospital admissions related to RTI. Need for extra oxygen, need for (non-) ventilation, or adjustment. b. Complains during RTI. Symptoms of RTI include: cough, fever, chest pain, headache, sputum production, nausea Patients with a RTI may also exhibit non-respiratory symptoms such as confusion, headache, myalgia, abdominal pain, nausea, vomiting and diarrhoea. c. Days of start RTI to recovery. Defined as: number of days to patient reported recovery with absence of symptoms and Borg Rating of Perceived Exertion Scale (RPE) back to baseline. 2. Compliance of therapy. Compliance is defined as the ability to perform MI-E or AS sessions at least twice a day. The reported compliance will be compared to the retrospective review of recorded data in the MI-E device and the LVR counter data in the AS group. 3. Patient reported satisfaction and fatigue related to the treatment. a. Comfort is a pleasant feeling of being relaxed and free form pain (Cambridge dictionary 30-8-2021). Relief is defined as a feeling of happiness that something unpleasant has not happened or has ended (Cambridge dictionary 30-08-2021). for this study we will use the following definition for comfort/relief after treatment: a feeling of relief from symptoms of secretions, coughing, dyspnoea. This is assessed by a visual analogue scale. b. Fatigue after treatment is defined as a feeling of weariness and tiredness after treatment with MI-E or AS. Assessed by the patient using visual analogue scale (VAS) between 1-10. Quality of life WHO defines Quality of Life as an individual's perception of their position in life in the context of the culture and value systems in which they live and in relation to their goals, expectations, standards and concerns. Qol will be assessed by the Severe Respiratory Insufficienc	—

Measure

Time frame

Secondary study parameters/endpoints Multiple secondary endpoints will be studied to compare MI-E treated participants to AS treated participants: 1. Severity of RTI a. Hospital admissions related to RTI. Need for extra oxygen, need for (non-) ventilation, or adjustment. b. Complains during RTI. Symptoms of RTI include: cough, fever, chest pain, headache, sputum production, nausea Patients with a RTI may also exhibit non-respiratory symptoms such as confusion, headache, myalgia, abdominal pain, nausea, vomiting and diarrhoea. c. Days of start RTI to recovery. Defined as: number of days to patient reported recovery with absence of symptoms and Borg Rating of Perceived Exertion Scale (RPE) back to baseline. 2. Compliance of therapy. Compliance is defined as the ability to perform MI-E or AS sessions at least twice a day. The reported compliance will be compared to the retrospective review of recorded data in the MI-E device and the LVR counter data in the AS group. 3. Patient reported satisfaction and fatigue related to the treatment. a. Comfort is a pleasant feeling of being relaxed and free form pain (Cambridge dictionary 30-8-2021). Relief is defined as a feeling of happiness that something unpleasant has not happened or has ended (Cambridge dictionary 30-08-2021). for this study we will use the following definition for comfort/relief after treatment: a feeling of relief from symptoms of secretions, coughing, dyspnoea. This is assessed by a visual analogue scale. b. Fatigue after treatment is defined as a feeling of weariness and tiredness after treatment with MI-E or AS. Assessed by the patient using visual analogue scale (VAS) between 1-10. Quality of life WHO defines Quality of Life as an individual's perception of their position in life in the context of the culture and value systems in which they live and in relation to their goals, expectations, standards and concerns. Qol will be assessed by the Severe Respiratory Insufficienc

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Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)