Motor Neurone Disease, Respiratory Insufficiency
Conditions
Keywords
non invasive ventilation
Brief summary
Non-invasive ventilation (NIV) is commonly offered to people with Motor Neurone Disease (MND) who have breathing difficulties. It improves their quality of life and can prolong life by 6 months or more. It is initially used at night and typically set up during a hospital admission. By the time that they develop respiratory failure and need to start NIV, however, most patients require wheelchairs or have other significant health problems. Repeated travel to hospitals is increasingly difficult with increasing disability. It is possible to start and monitor NIV treatment at home. This may be more convenient for selected patients, though starting NIV is quite complex; it is not known if home treatment is as safe and effective as hospital-based treatment. To establish this, 60 patients with MND who have indications for NIV will be recruited. They will be randomly allocated to a home-based treatment (home NIV set up plus home visits supported by telemonitoring) or hospital-based care (inpatient NIV set up plus outpatient NIV monitoring) and followed up at 1, 4 and 7 months. Alongside measures of treatment effectiveness, assessment of patient and carer preferences, quality of life, and cost-effectiveness will be undertaken. In the additional qualitative part on this study, interviews with patients who took part in the main study and their carers will be conducted to understand in more depth their perspective on what makes for a good or bad experience with NIV, how the environment (home vs hospital) influences their NIV experience and what personal factors determine NIV use. Findings from the interviews will inform the design of a truly patient-centred NIV service.
Interventions
OTHERNon-invasive ventilation
Providing ventilation support through non-invasive means
Sponsors
University of Nottingham
MND Association
Papworth Hospital NHS Foundation Trust
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Random allocation to either home or hospital based treatment
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adults with a diagnosis of Motor Neurone Disease confirmed by a neurologist specialising in MND. * Indications to NIV: daytime pCO2 \>6.0 kPa or/and symptoms of ventilatory failure (e.g. orthopnoea, paroxysmal nocturnal dyspnoea, morning drowsiness) in the presence of significant diaphragmatic weakness confirmed by respiratory muscle tests
Exclusion criteria
* Cognitive impairment precluding understanding of the study protocol and valid consent * Severe co-morbidities (e.g. decompensated heart failure, severe chronic obstructive pulmonary disease (COPD), morbid obesity) causing or contributing to respiratory failure * Immediate need to start NIV (\<24hrs) and/or acute illness requiring inpatient treatment e.g. intravenous antibiotics for pneumonia (in addition to the need for NIV) * Lack of a sufficient social/professional network to support NIV application at home * Not wishing to accept home NIV
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Control of respiratory failure defined by daytime partial pressure of carbon dioxide (PaCO2) | measured at 1,4,7 months following NIV initiation | Daytime PaCO2 measured by arterial puncture |
| Patients and carers experiences of starting and managing NIV, and factors that relate to NIV concordance and acceptance. | measured at 4 weeks since starting NIV | Semi-structured interview with patients and their carers |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Survival | Survival since NIV initiation | Survival (in months) |
| Admissions | Up to 7 months from initiation of NIV | Number of additional planned and emergency hospital admissions |
| NIV setting changes | 1 , 4 and 7 months | Number of NIV setting changes required |
| Cost effectiveness | Up to 7 months from initiation of NIV | Absolute costs and costs in relation to quality-adjusted life-years (using EQ-5D) |
| Nocturnal oximetry | 1,4,7 months | time spent with oxygen saturation \<90%(TST90) |
| NIV acceptance | 1 month | Percent of patients using NIV for \>=4hrs/night |
| Quality of life assessment (EQ-5D-5L) | 1,4,7 ,months | Measurement of quality of life using EQ-5D-5L questionnaire |
| Quality of life (SRI) | 1,4, 7 months | Quality of life measured using Severe Respiratory Insufficiency (SRI) questionnaire |
| Quality of sleep | 1,4,7 ,months | Sleep quality measured with Pittsburgh Sleep Quality Index (PSQI) |
| Satisfaction with NIV and healthcare interactions | 7 months | Satisfaction with NIV and healthcare interactions measured with a visual analogue scale |
| Other blood gas analysis measurements of hypoventilation | 1,4,7 months | Bicarbonate |
| NIV concordance | 1 , 4 and 7 months | NIV adherence (machine usage in hours/24hours) |
Countries
United Kingdom
Contacts
Primary ContactDariusz Wozniak
Backup ContactVictoria Stoneman
Outcome results
None listed