Initiation of Home Mechanical Ventilation at Home in Patients With Chronic Hypercapnic Respiratory Failure

Study Overview
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Brief Summary
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Objective: To prove that initiation of chronic ventilatory support at home, in patients with chronic hypercapnic respiratory failure due to neuromuscular disease (NMD) or thoracic cage problem is not inferior compared to initiation in a hospital based setting. In addition we believe that the start at home is cheaper compared to an in-hospital start. Hypothesis: Initiation of chronic ventilatory support at home is effective, safe and cost effective compared to a hospital-based initiation. Study design: A nationwide non-inferiority multi-center randomized parallel active control study. Study population: Patients with chronic respiratory insufficiency due to a neuromuscular disease (NMD) or thoracic cage problem who are referred for chronic ventilator support. Intervention: The start of HMV at home Standard intervention to be compared to: The start of HMV is normally in a clinical setting as recommended in the national guideline. Outcome measures: Primary: PaCO2. Secondary: Health related quality of life; lung function; nocturnal transcutaneous carbon dioxide assessment and saturation, and costs Sample size calculation/data analysis: This is a non-inferiority trial based on PaCO2 as primary outcome. A difference in favor of the hospital care group of smaller than 0.5 kPa will be labeled as non-inferior. To retain 72 evaluable patients, and allow for drop-outs, we will include 96 patients in total. Cost-effectiveness analysis: A cost analysis will be conducted alongside the clinical trial. Costs of the initiation of HMV at home and in the hospital will be estimated form a societal perspective over a time horizon of 6 months. Time schedule: After an initial phase of 6 months recruitment will start and will take 24 months. Thirty-six months after the start of the study the last assessments will be done and analysis and writing of the papers will start. After 42 months the study will end.

Official Title
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Initiation of Home Mechanical Ventilation at Home in a Selectve Group of Patients With Chronic Hypercapnic Respiratory Failure in the Netherlands

Conditions
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Neuromuscular Diseases, Respiratory Therapy, Respiration, Artificial, Positive-Pressure Respiration, Noninvasive Ventilation, Cost-Benefit Analysis, Ambulatory Monitoring

Intervention / Treatment
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* Procedure: Initiation of mechanical ventilation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Indication to initiate non-invasive ventilatory support in patients with a neuromuscular disease or thoracic cage abnormality who suffer from complaints of alveolar hypoventilation (fatigue, headache of dyspnoea) combined with all following elements: arterial carbon dioxide > 6.0 kPa daytime or arterial or transcutaneous carbon dioxide > 6.0 kPa at night or orthopnea as a result of diaphragm paralysis Age > 18 years Existence, of a sufficient network (social or professional) according to the supervising HMV center making initiation of HMV at home possible and safe. Exclusion Criteria: Patients who already have had HMV due to acute respiratory failure Necessity for invasive ventilatory support Patients admitted to a nursing home

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Hospital initiation<br>Initiation of mechanical ventilation in hospital initiation of Home Mechanical ventilation takes place in a hospital; this makes this arm the standard care. | Procedure: Initiation of mechanical ventilation<br>* In patient with chronic respiratory insufficient chronic mechanical ventilation will be initiated in the patient his/her setting.<br>* Other names: start of home mechanical ventialtion with telemonitoring;|
| Experimental: Home initiation<br>Initiation of mechanical ventilation at home Initiation of mechanical ventilation in a patient's home setting with telemonitoring | Procedure: Initiation of mechanical ventilation<br>* In patient with chronic respiratory insufficient chronic mechanical ventilation will be initiated in the patient his/her setting.<br>* Other names: start of home mechanical ventialtion with telemonitoring;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Daytime arterial carbon dioxide | Daytime arterial carbon dioxide (PaCO2) assessed without oxygen supplementation and ventilatory support while the patient is in sitting position | 6 months after initiation of mechanical ventilation | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Quality of life | Health related quality of life by using the following questionnaires: SF 36, MRF 28, HADS, SRI | 6 months after initiation of mechanical ventilation | 
| Lung function | evaluate lungfunction in time; with measurments of flow/volume in a sitting and horizontal position | 6 months after initation of mechanical ventilation | 
| Nocturnal transcutaneous carbon dioxide and saturation | Nocturnal transcutaneous carbon dioxide and saturation in time | 6 months after initiation of mechanical ventilation | 
| costs | evaluation of the costs during initiation and the follow-up for 6 months. This will be objective by using the EuroQol (EQ-5D) questionnaire | 6 months after initiation of mechanical ventilation |