Duchenne Muscular Dystrophy (DMD)
Conditions
Keywords
Duchenne Muscular Dystrophy, Non-invasive ventilation, Feasibility, Home unattended full-video polysomnography, Transcutaneous capnography
Brief summary
The aim of this study is to verify whether full video home polysomnography (sleep study) with nocturnal transcutaneous CO2 monitoring is feasible in the follow-up of non-invasive ventilation (NIV) in patients with Duchenne muscular dystrophy. The researchers would like to investigate whether they could perform sufficient measurements in the home situation and see if correct decision for further treatment could be made. Furthermore, the researchers will make an analysis on the minimal requirements of measurements to make a correct decision for the treatment of these patients.
Interventions
DIAGNOSTIC_TESTMonitoring of NIV at home
Monitoring of NIV at home
Sponsors
Universitaire Ziekenhuizen KU Leuven
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients with DMD \> 18 years using NIV * Signing the written informed consent
Exclusion criteria
* /
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The feasibility of full video home polysomnography (PSG) and follow-up at home in patients with DMD on NIV, identical to the sophisticated PSG in-hospital | Yearly home polysomnopraphy and Transcutaneous CO2 measurement, for a period of 3 years | The primary endpoint is the feasibility of a home based full-video polysomnography for follow-up of DMD patients on NIV. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Cost-benefit analysis of follow-up of NIV at home as a preparation to create a file for the Belgian health insurance (registration of time and material) | Cost-benifit analysis will be carried out at study completion, after 3 years. | During 3 years, the costs for performing a home based full-video PSG (relocation costs, material, physiotherapist wage) will be weighed against the benifits (patient comfort, relieving hospital ward occupation) |
| Amount of technical failures, i.e. PSG signals, PtcCO2 measurement, O2 saturation measurement and video monitoring. | Performed immediately after each PSG or PtcCO2 measurement, during a period of 3 years. | Checklist after each PSG |
| Patient Reported Outcomes: Experience between in-hospital PSG vs PSG at home | The PROM will be completed by the patient at each yearly PSG, during the 3 years of the ongoing study | Both patient and caregiver report on a 10 point likert scale, ranging from totally not to very, how mor comfortable the PSG at home was compared to the hospital PSG. |
| Analysis of the minimal equipment to assure a correct follow-up of NIV at home (full video PSG vs PtcCO2) measurement combined with ventilator software data vs ventilator software data vs PtcCO2 measurement) | 3 years | — |
| Home-NIV PROM: S3-NIV | The S3-NIV questionnaire will be completed by the patient at each yearly PSG and at each yearly PTCO2 measurement, during 3 years. | The S3-NIV questionnaire is a short, valid and repeatable self-completed tool for the routine clinical assessment of patients undergoing home NIV. The S3-NIV questionnaire provides clinicians and patients with a simple and reliable tool to assess important domains (symptoms, sleep quality and NIV-related side effects) as a complement to physiological monitoring. The S3-NIV questionnaire has 11 items. Each item is scored using a 5-point Likert scale from always true to never true. The total score and the two sub scores range from 0 to 10 points. Lower scores represent a higher, negative impact of the disease and NIV on the individual. |
| Daytime sleepiness questionnaire: Epworth Sleepiness Scale (ESS) | The ESS questionnaire will be completed by the patient at each yearly PSG and each yearly PtcCO2 measurement, during 3 years. | The ESS is a self-administered questionnaire with 8 questions. Respondents are asked to rate, on a 4-point scale (0-3), their usual chances of dozing off or falling asleep while engaged in eight different activities. |
| Sleep quality questionnaire: The Pittsburgh Sleep Quality Index (PSQI) | The PSQI will be completed by the patient at each yearly PSG and at each yearly PtcCO2 measurment, during 3 years. | The Pittsburgh Sleep Quality Index (PSQI) is a widely used self-report questionnaire that assesses sleep quality over a one-month time interval. Each component score of the PSQI ranges from 0 to 3, with 3 indicating the greatest dysfunction or disturbance. The seven component scores are then summed to obtain a global PSQI score, which ranges from 0 to 21. Higher scores indicate poorer sleep quality, with a score greater than 5 suggesting significant sleep difficulties |
Countries
Belgium
Contacts
Primary ContactDries Testelmans, MD PHD
Backup ContactBart Vrijsen, PT PHD
Outcome results
None listed