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ID
Source
Brief title
Health condition
Myotonic dystrophy type 1
Neuromuscular disorder
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary objective is to investigate the effect of NIV on different factors including gas exchange, lung function, daily functioning, fatigue and sleepiness, sleep quality, quality of life and compliance to the therapy.
Secondary outcome
Secondary objectives are to define possible determinants for development of sleep disordered breathing (disease severity, disease duration, age, weight, lung function parameters).
Background summary
Patients with myotonic dystrophy (DM1) frequently develop sleep-disordered breathing (SDB) with or without alveolar hypoventilation, which may lead to fatigue, excessive daytime sleepiness, and impaired sleep quality, negatively influencing daily and cognitive functioning, and quality of life. Furthermore, SDB is associated with cardiac conduction disorders, and can lead to cardiac arrhythmias. The benefit of non-invasive ventilation (NIV) for patients with DM1 varies to a great extent, from a burdensome experience to a beneficial treatment. This also leads to difficulty motivating patients for NIV, and this seems a bigger problem in DM1 compared to other patient groups. Therefore, identification of patients with DM1 that do (or do not) respond well to NIV will become necessary to improve its cost-effectiveness and reduce the burden for patients and their loved ones. This requires consensus about the key performance measures of NIV. The study is a longitudinal observational study to profile a multidimensional response to NIV in patients with DM1. We will profile a multidimensional response to NIV in patients with DM1, enabling us to identify patients with DM1 that do (or do not) respond well to NIV.
In this prospective research, DM1 patients with SDB will be included when their treating physician at the local centre of home mechanical ventilation decides that there is an indication to start with NIV treatment. To determine the indication for NIV, physicians use the 'Veldnorm chronische beademing', a document which has been developed by and for physicians of the centres of home mechanical ventilation in the Netherlands. Data will be collected regarding: lung function test, blood gas, polysomnography or overnight measurement of O2 and CO2. Follow-up will take place up to 6 months after start with the NIV treatment. Follow-up data will be collected systematically and registered in an eCRF. We aim to include our patients within a timeframe of 2.5 years.
Study objective
DM1 patients that start with NIV will respond differently in terms of gas exchange, exercise capacity, daily functioning. fatigue, sleep quality, excessive daytime sleepiness and quality of life. This research will enable us to identify the different factors that influence the response to NIV.
Study design
T0: baseline measurements
T1: start non-invasive ventilation
T2: follow-up after 2 weeks
T3: follow-up after 6 weeks
T4: follow-up after 6 months (and end of study)
Inclusion criteria
- diagnosis myotonic dystrophy type 1
- age >18 years
- daytime pCO2 >6.0 kPa and complaints of alveolar hypoventilation OR daytime pCO2 >7.0 kPa without complaints of alveolar hypoventilation
- the general criteria regarding home mechanical ventilation ('dutch veldnorm 'Chronische beademing' versie 1.0, 2012) are met
Exclusion criteria
- previous episode of non-invasive home mechanical ventilation or CPAP (continuous positive airway pressure) therapy in de last 5 years
- other medical condition leading to hypercapnia
- severe heart failure, NYHA IV
- instable angina pectoris
Design
Recruitment
IPD sharing statement
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
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In other registers
| Register | ID |
|---|---|
| NTR-new | NL7972 |
| Other | METC azM/UM : METC 2018-0853 |