Delivering Effective Non-Invasive ventilation in Motor neuron disease using intensive remote support (DENIM)

Background

Non-invasive ventilation (NIV) is the only treatment demonstrated to improve quality of life and survival in people with motor neuron disease (MND). More than half of patients commencing NIV do not experience effective ventilation due to problems which should be identified and addressed early in the initiation process. Low adherence and ineffective ventilation are associated with reduced survival.

Optimal use of NIV requires effective patient preparation, initiation, ongoing support, monitoring and optimisation. We have developed a programme theory, consistent with the Consolidated Framework for Implementation Research and Normalization Process Theory, showing why these practices are not embedded into NIV services. We will determine if and how a tailored programme of intensive support and optimisation at the time of initiation will deliver more effective NIV to improve patients’ quality of life and survival.

Aims

To investigate the effectiveness and cost effectiveness of implementing an intensive, theory-informed goal-based approach to NIV initiation which aims to increase NIV effectiveness in people with MND.

To determine its impact on NIV use and effectiveness and to explore contextual barriers to normalising such a service within routine clinical practice. Objectives

WP1: A stepped wedge cluster randomised controlled trial, powered to detect if the addition of highly intensive, theory-informed goal-based approach during the first 12 weeks of NIV initiation increases NIV use and effectiveness;

WP2: A process evaluation showing how healthcare professionals’ self-reported normalisation of this new approach to NIV optimisation within clinical teams is associated with wear time and other measures of success, with pathways to outcome further elaborated through observations and interviews;

WP3: A Cost-Effectiveness Evaluation Alongside a Clinical Trial and long-term cost effectiveness modelling, from an NHS perspective Intervention

A theory-based package of staff education, highly intensive support, monitoring and optimisation using telemonitoring during the first 12 weeks of NIV use with the aim to optimise NIV use and effectiveness. Methods

A stepped-wedge cluster randomised trial at 12 UK sites (n=252 patients). Sites will start the trial enrolling participants receiving usual care. Sites will then switch to implementing the intervention at random intervals. Eligibility: patients initiating NIV expected to require optimisation by 12 weeks or earlier

Primary outcome: average NIV usage at week 9-12 (hr/day) Secondary outcomes WP1 & 3: Survival, NIV effectiveness, quality of life, respiratory symptoms, NIV comfort and health resource use

WP2: triangulation of NIV usage data with the perspectives of staff, patients and carers using interviews, ethnographic observations and NoMAD questionnaires Timelines for delivery 41m study: 8m set up, 24m recruitment including 6m roll in period, 27m maximum follow-up, 6m close out Anticipated impact and dissemination

DENIM will provide a blueprint for implementing a better standard of care for NIV initiation resulting in more patients experiencing longer survival and improved quality of life whilst making the best use of scarce resources. It will deliver a sustainable improvement in healthcare professionals’ skills, experience and knowledge of MND and develop a national network of research engaged staff able to deliver further innovative interventions in MND and respiratory care.