Title: Enhancing the efficacy of non-invasive ventilation for patients with motor neurone disease: Synthesising the evidence, mapping the services and translating the evidence into clinical practice.
Funder: Department of Health and Social Care

Abstract: Background Motor neurone disease (MND) is an incurable neurodegenerative disease causing progressive, and eventually fatal, muscle weakness with an average life expectancy of only two to four years. When patients with MND (pwMND) develop respiratory failure, non-invasive ventilation (NIV) is the only intervention shown to be effective. NIV improves survival by on average, seven months and improves quality of life and symptoms such as breathlessness. Nevertheless, evidence suggests that many patients with respiratory failure do not use NIV, and many of those that do are not gaining the full benefit. Previous RfPB funded work, undertaken by our team, improved our understanding of the patient/informal carer-relating obstacles to tolerance and adherence to NIV. However, the NIV service/pathway across the UK varies and there is a lack evidence to determine how NIV should be used. NIV is a complex intervention and many uncertainties remain, including how to initiate and monitor NIV; how often it should be used; how and what equipment should be used and how it should be adjusted and how common problems should be addressed. There is, therefore, an urgent need for clear evidence for how HCPs should best deliver NIV in order to maximise patient benefit. Aims The aim of this research is to determine how NIV can best be used and delivered in order to ensure that every patient gains the most benefit. For this, we will systematically gather and synthesise the evidence relating to the current and optimal use of non-invasive ventilation in pwMND and translate this evidence into tools to improve clinical practice and optimise NIV services for patients. Plan of investigation This is a 27-month project, which contains four overlapping and complementary work packages. Work package 1 (WP1) A systematic review of quantitative and qualitative evidence to identify the factors associated with effective use of NIV at all stages of the care pathway for pwMND. Work package 2 (WP2) A service mapping/survey of UK and European centres who provide respiratory support to pwMND to determine the current available services, standards, guidelines and practices, as well as barriers to, and enablers of, effective NIV use from the perspective of HCPs. Work package 3 (WP3) Stakeholder consultations using focus groups and semi-structured narrative interviews with. It will explore their views on the research findings and determine how best to translate this knowledge into improved services for patients. Although the emphasis will be on HCPs, PPI members, including patients/informal carers will be also consulted. Work package 4 (WP4) Combining the knowledge identified develop a package of outputs which will support MND services, HCPs, pwMND and their informal carers to optimise the use of NIV at all stages of the care pathway. The outputs will depend on the needs of stakeholders, determined during WP3 and will employ strategies based on theoretical frameworks to ensure that the outputs support change in behaviours and clinical practice. This may include tools to guide NHS services on how to best deliver NIV and to compare current services against best practice. A web-based educational platform will be developed to present the evidence-based guidelines targeted at HCPs but also accessible to patients and carers. It will use iterative, user-centred design techniques to work collaboratively with stakeholders in workshops to design, develop and test resources. Potential benefits to patients and the NHS This research will develop evidence-based tools which can be directly translated into clinical practice in the NHS. The research will enable patients to gain the most benefit from NIV which, ultimately should lead to increasing survival and quality of life for pwMND.

Lay abstract: Aim of the research This research aims to identify the best ways to provide non-invasive ventilation (NIV) services to people with motor neurone disease (MND) and to use this information to develop resources to ensure patients get the best service possible. Background MND is a progressive, and eventually fatal condition causing muscle weakness. Weakness of the breathing muscles causes symptoms such as breathlessness and disturbed sleep. Breathing difficulties are the most common cause of death in MND. NIV uses a machine to support patients own breathing. It can enable patients to live longer and can improve their quality of life. However, many patients find it difficult to use NIV and some may not receive the full benefit if it is not used correctly. Services offering NIV vary widely and patients may not always be getting the right support, at the right time. Accordingly, patients with MND have identified the need to prioritise research that can improve the quality of NIV services. Methods We will look at the research in a systematic way to see what best practice might be. We will carry out a survey of NIV services in UK and Europe to see what practices, standards and policies exist. We will ask policy-makers, staff, patients and carers to give their views on how the results might be used to help improve services. We will develop resources, such as online information and guidelines, to help improve the way in which NIV is delivered. Patient and Public Involvement We will involve those living with MND at every stage of the research through advisory groups to help us understand what the study findings mean, develop new information tools and publicise our findings. Dissemination We will publicise our findings to researchers, staff, patients and carers via open access publications, conferences, talks and workshops.

Research activity codes: 8.1 ORGANISATION AND DELIVERY OF SERVICES
Health categories: NEUROLOGICAL