Clinical trial NCT03203577: Initiation of Home Mechanical Ventilation at Home in Patients With Chronic Hypercapnic Respiratory Failure
Official title: Initiation of Home Mechanical Ventilation at Home in a Selectve Group of Patients With Chronic Hypercapnic Respiratory Failure in the Netherlands
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Respiratory Therapy
Respiration, Artificial
Positive-Pressure Respiration
Neuromuscular Diseases
Noninvasive Ventilation
Cost-Benefit Analysis
Ambulatory Monitoring
Lead sponsor: University Medical Center Groningen
Sponsor class: OTHER
First posted: 2017-06-29
Enrollment: 96
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Daytime arterial carbon dioxide
timeFrame: 6 months after initiation of mechanical ventilation
Secondary outcomes:
measure: Quality of life
timeFrame: 6 months after initiation of mechanical ventilation
measure: Lung function
timeFrame: 6 months after initation of mechanical ventilation
measure: Nocturnal transcutaneous carbon dioxide and saturation
timeFrame: 6 months after initiation of mechanical ventilation
measure: costs
timeFrame: 6 months after initiation of mechanical ventilation
Other outcomes:
Locations:
city: Groningen
country: Netherlands
facility: University Medical Center Groningen
Study officials:
affiliation: University Medical Center Groningen
name: P.J. Wijkstra, Prof.
role: PRINCIPAL_INVESTIGATOR
References:
citation: van den Biggelaar RJM, Hazenberg A, Cobben NAM, Gaytant MA, Vermeulen KM, Wijkstra PJ. A Randomized Trial of Initiation of Chronic Noninvasive Mechanical Ventilation at Home vs In-Hospital in Patients With Neuromuscular Disease and Thoracic Cage Disorder: The Dutch Homerun Trial. Chest. 2020 Dec;158(6):2493-2501. doi: 10.1016/j.chest.2020.07.007. Epub 2020 Jul 16.