Title: Symptom-based pathways to earlier diagnosis: how to improve health for sufferers of chronic breathlessness with a cost-effective approach.
Funder: Department of Health and Social Care

Abstract: Background Chronic exertional breathlessness is a highly prevalent symptom, associated with long term sequelae, accounting for 5% of General Practitioner (GP visits) and 21% of medical admissions. Delays in diagnosis of long term conditions frequently occur leading to diagnosis when the disease is advanced and largely irreversible. 70% of the NHS budget is consumed by long term conditions and ways to minimise their impact are urgently needed. The five commonest causes of chronic breathlessness, accounting for 50% of the diagnoses for adults over 40 years old: Chronic Obstructive Pulmonary Disease (COPD), Chronic Heart Failure (CHF), Obesity, Anxiety, and Anaemia, can be diagnosed or excluded using inexpensive and accessible i.e. simple' investigations in primary care. Current practice involves multiple GP visits or referral to secondary care without utilising all simple' investigations first. Clinical guidelines, services and research tend to be designed around specific diseases or organ-specific specialities rather than symptoms. Therefore, GPs have limited evidence-based guidance on how to assess which patients with breathlessness are at risk of premature mortality and how to make a diagnosis effectively using a cost-effective approach. Overarching Hypothesis An earlier diagnosis could be achieved by utilising a simple panel of investigations' at presentation, earlier disease and symptom specific therapies would improve long term health status, and the approach would be cost-effective. Overarching Research Questions In adults over forty years old presenting for the first time with at least two months of exertional breathlessness: 1)What is the long term health status and prognosis? 2)Do the secondary consequences of breathlessness such as physical inactivity, mood disturbance and frailty predict the longer term outcomes independent of the specific causative disease? 3)Does investigating with a panel of simple investigations (aimed to diagnose or exclude the five most common diagnoses) lead to earlier diagnosis than usual care benefit individuals through earlier provision of disease specific treatment improving health status improve utility of healthcare services leading to a cost-effective approach enable earlier provision of interventions to enhance self-management of healthy behaviours to reduce the burden of the secondary consequences of breathlessness for individuals and the healthcare system To definitively answer the first two questions would require an observational cohort study over several years, and for the last: a large expensive randomised controlled trial (RCT). Plan of investigationIn adults over 40 years old presenting with chronic exertional breathlessness for the first time in primary care: 1)to conduct a longitudinal cohort study to quantify cardio-metabolic risk and investigate the relationship between variables of the secondary consequences of chronic breathlessness (potential biomarkers) and cardio-metabolic risk at one year 2)to conduct a feasibility study of a RCT comparing initial investigation with a simple panel of investigations' versus usual care clinician determined investigations' During the fellowship the aim is to apply for further funding: 1)to extend the follow up for individuals in the cohort study ' with mortality rate as the primary outcome. 2)to perform a RCT to determine whether early investigation with a panel of investigations leads to an earlier diagnosis and when combined with earlier intervention (both pharmacological and non-pharmacological) is clinically and cost effective. Summary of potential benefits to patients and NHS The aim of redesigning healthcare services around symptoms rather than diseases for chronic breathlessness is to achieve earlier diagnosis and treatment to improve health status for individuals, and improve efficiency of healthcare utilisation leading to cost effectiveness. The information from the cohort study could lead to validating a statistical model to predict individual risk of death to help tailor treatment decisions to the syndrome of chronic breathlessness (stratified medicine).

Lay abstract: Becoming short of breath doing everyday activities is a common problem. People often see their General Practitioner (GP). It can be difficult for a GP to know whether there is a serious problem and what the problem is. There are many reasons why people become more short of breath. Over half of cases are caused by five problems: a heart condition called Chronic Heart Failure (CHF)' a lung condition called Chronic Obstructive Pulmonary Disease (COPD)' obesity (being very overweight) anaemia (low red blood cells that carry oxygen around the body) anxiety We know many people wait a long time to find out what the problem is and to get the right treatment. In part because guidelines for GPs focus on specific diseases and specialist services are designed around specialities' such as cardiology (specialist heart services) not symptoms. Specialist exercise programmes are available for people with more severe heart and lung problems to improve shortness of breath. These programmes are known to be good for patients and improve their quality of life. Patients tell us they wish they had received this treatment earlier. This research involves people older than forty years old who go to their GP as they are becoming short of breath, and aims to: 1) Investigate their long term quality of life and how long they live. 2) Understand what features of becoming short of breath such as being less active affect their long term health. 3) Design a research trial to compare whether GPs using more investigations (focusing on the five common causes) early will lead to an earlier diagnosis and what this costs. 4) Understand whether combining an intervention to achieve an earlier diagnosis with earlier support to lead a healthy lifestyle would help prevent the longer term effects of being breathless and improve health To answer Questions 1 and 2 would need a large study of a group monitored yearly for a few years. Questions 3 and 4 need a large, expensive study to answer them. The aims of the fellowship are to conduct two smaller studies: the first to answer aspects of the first two questions using a measure of the risk of developing heart disease or diabetes as a surrogate of death rate. With this information, funding will be applied for to continue the study for longer to answer the questions completely. GPs will then have more information to discuss with a breathless person which could alter what treatment they receive and help them lead a more healthy life. The second study is to see whether and how the larger expensive study can be performed. Design and method In people older than forty years old who go to their GP as they are becoming short of breath doing everyday things for over two months: 1) A study will be undertaken where they are monitored at the beginning and end of one year. It will investigate how features caused by being short of breath influence their risk of heart disease and diabetes in future. 2) A study will be undertaken to see whether the larger study is practical. GP practices either continue usual care for these individuals (organising tests as they see fit) or to perform multiple specified tests straight away. Each practice will be allocated to one of these groups at random. Patients and GPs will be interviewed to understand their experience to inform the next trial. The main outcome will be how many patients can be recruited to the study in six months and to understand how many GP practices would be needed for the main trial. Whether the different parts of the study work together, whether having multiple tests was acceptable to patients, and whether patients would be willing to accept support to lead a healthier lifestyle will all be explored. Patient and Public Involvement The idea for this research came from patients with shortness of breath requesting earlier diagnosis and treatments. I have worked with patients to develop the research proposal and patients will be key members of the research steering group throughout the studies. Dissemination The information from the studies will be reported in different ways for peer reviewed journals, the relevant voluntary group sector, and patients. A film of how patient involvement shaped the research throughout will be made.

Research activity codes: 3.1 PRIMARY PREVENTION INTERVENTIONS TO MODIFY BEHAVIOURS OR PROMOTE WELL-BEING
Health categories: RESPIRATORY