Title: A pilot study to assess the safety, effectiveness and, practical utility of remote patient monitoring to guide the timing of valve intervention in patients with asymptomatic severe aortic stenosis (APRAISE-AS)
Funder: Department of Health and Social Care

Abstract: Research question Does proactive surveillance of symptoms, quality of life and frailty using a remote monitoring/early warning support system (using a novel smart device app) in addition to standard care lead to timelier valve intervention in people with asymptomatic severe aortic stenosis? Background Aortic stenosis (AS) is the most common presentation of heart valve disease encountered in clinical practice and severe symptomatic AS carries a significant mortality burden, similar to many cancers. In the absence of proven pharmacological treatments, early aortic intervention (surgical or transcatheter) is the guideline mandated in patients with symptomatic severe AS. In asymptomatic patients with severe AS the timing of valve intervention is not well defined; currently a &ldquo;watchful waiting&rdquo; strategy with a 6-monthly clinic-based review is advocated with intervention at the point of symptom development. However, the progressive nature of AS combined with the advanced age of the affected population result in underreporting and/or underestimation of symptoms and often irreversible myocardial damage, leading to poorer outcomes following intervention. Aims and objectives To assess the impact of proactive app-based symptom reporting, disease-specific patient reported outcome measures and frailty assessments on the treatment pathway of patients with asymptomatic severe AS. We will assess whether this approach, in addition to standard care (watchful waiting), leads to timelier appropriate valve intervention and an improved patient experience. The pilot study will allow for refinement of trial design prior to undertaking a larger multi-centre randomised controlled trial which will address clinical outcome measures and health economic analysis before possibly leading to clinical service redesign. Methods A single centre, prospective, two-arm, randomised controlled trial in which 66 participants with asymptomatic severe AS will be recruited and assigned to either the intervention arm (app based proactive surveillance plus standard care) or control arm (current standard of care, clinic based 'watchful waiting'). Outcomes The primary outcome is to pilot the trial protocol to inform the development of a larger multi-centre clinical and healthcare economic endpoint based randomised controlled trial. The pilot study will test the trial protocol in a representative population and assess the utility of the app, recruitment, retention rates, completeness of data collection and adherence to the intervention. Timelines for delivery App/clinician s portal development and end user testing has already been undertaken. The trial itself will last for 26 months; the timeframe for recruitment, randomisation and follow-up will cover 16 months. Data monitoring will take place over the duration of the trial and further 3 months will be required for final data analysis and report writing. Anticipated impact and dissemination Our proposal will increase patient engagement, empowerment and improved self-management of their condition, in addition to enhanced access to the healthcare team. The findings of our pilot trial will help to inform the selection of the most appropriate outcome measures for a full-scale randomised controlled trial. The findings of our work will be shared with our patients through local newsletters and via our links with patient groups, they will be published in peer reviewed journals and presented at national/international conferences.

Lay abstract: Aims of the research To find out whether using an app at home to monitor symptoms in between hospital visits can improve the health of people with asymptomatic severe aortic stenosis. Background to the research Heart valve disease is caused by damage to one or more of the four valves in the heart. The commonest form is a narrowing of the aortic valve known as aortic stenosis. The process which causes this narrowing develops over years, which is why it affects mainly older adults. Narrowing of the valve affects the way blood is pumped out of the heart to the body. Because the heart adapts and works harder to overcome the narrowing, people may not notice any symptoms until the narrowing is severe. Common symptoms include tiredness, shortness of breath, chest tightness, dizziness and collapse. The only treatment for aortic stenosis when symptoms develop is aortic valve replacement. Some people with severe aortic stenosis don t have symptoms; they are &ldquo;asymptomatic&rdquo;. In these people, 6-monthly hospital-based clinic visits are recommended and once symptoms appear, it is recommended that the aortic valve is replaced. In clinic symptoms are often confused with &ldquo;getting older&rdquo; and may not be reported to the doctor, which can lead to delays in valve replacement. We want to find out whether using a smart device app at home, in addition to routine hospital visits, will allow us to pick up these important symptoms sooner and take action. Earlier valve replacement may lead to improved survival, well-being and prevent unwanted hospital admissions. Design and methods used As part of a pilot trial, adults with asymptomatic severe aortic stenosis will be randomised to either usual care (6 monthly outpatient clinic appointments) or to usual care plus the app which asks about symptoms and exercise capacity. This information will be reviewed regularly by the clinical team over a 12-month period and used to help make treatment decisions. Patient and public involvement Our patient advisory group have experience of aortic stenosis and feel that home monitoring which can lead to timelier valve replacement is important. They worked with us to co-design and test the app and have helped to develop our research plan. They will also play a role in the trial and its delivery. Dissemination With input from our patient members, we will produce lay information and disseminate our findings using newsletters and social media to our patients. We will also share our findings in peer reviewed journals and at national/international conferences.

Research activity codes: 4.2 EVALUATION OF MARKERS AND TECHNOLOGIES;7.3 MANAGEMENT AND DECISION MAKING
Health categories: CARDIOVASCULAR