Title: The use of an electronic Patient-Reported Outcome Measure (ePROM) in the Management of Patients with Advanced Chronic Kidney Disease – A Pilot/Feasibility Study for a Randomised Controlled Trial.
Funder: Department of Health and Social Care

Abstract: Background: Chronic Kidney Disease (CKD) affects 16% of adults in the UK, with an annual cost of 1.45 billion. Over half, 780m, is spent on patients undergoing Renal Replacement Therapy (RTT) for End Stage Renal Disease (ESRD), despite these patients comprising less than 2% of the total CKD population. Patients with ESRD experience significant symptom burden and reduced quality of life and are at increased risk of hospitalisation and mortality. Delaying/preventing the progression from advanced CKD to ESRD is therefore a key healthcare priority. Effective management of advanced CKD relies on the timely detection of deterioration. This is a particular challenge between scheduled clinic visits, when it is difficult to identify deterioration in the clinical condition unless a patient self-refers. Unfortunately, some patients self-refer too late because they have difficulty identifying the point at which they may require assistance. Without prompt recognition of advanced symptoms, such patients are at high risk of severe illness, emergency hospitalisation and death. A recent randomised controlled trial (RCT) in cancer, demonstrated that asking patients to remotely report symptoms between clinic visits using an electronic Patient-Reported Outcome Measure (ePROM) - which triggered alerts to a clinical response team upon signs of patient deterioration - significantly improved quality of life, reduced emergency hospitalisation, and improved quality-adjusted survival. Such an ePROM system could benefit patients with CKD, however, this has not yet been tested in a randomized controlled trial (RCT). Aims: Before a definitive RCT is undertaken, I will conduct essential pilot/feasibility work aiming to: Develop the CKD ePROM system with patient/clinician input (including translated/validated language versions). Determine if a RCT is possible, and to gather information to help plan the main trial. Develop a collaborative group to secure funding to run the main RCT, and begin follow-on research to examine the potential benefit of ePROMs in other long-term conditions. Plan of investigation: the project will involve 3 Work Packages (WPs): WP 1 - will finalise development of the ePROM system with patient/clinician input. The system will be integrated within an award winning patient portal in a large Birmingham hospital. I will conduct work to include translated/validated versions in Urdu, Punjabi and Bengali: the most common languages spoken/read by patients with CKD in the West Midlands region (one of the planned sites for the main RCT). WP 2- a two-arm randomized pilot/feasibility trial will be conducted, with participants (n=60) randomized to receive either the ePROM intervention or usual care. The study will assess the willingness of patients to be involved, the acceptability of outcome measures and will help estimation of the sample size and selection of the primary outcome measure for the main RCT. Key participation criteria will be identified and a survey of UK centres undertaken to see if they are willing/able to contribute to the main RCT.WP 3 I will host an international workshop aimed at showcasing the study results and developing a collaborative research group to: (i) secure funding for the main RCT, and (ii) begin ePROMs research across other long-term conditions managed in the NHS. Potential benefits to patients and the NHS: The full RCT will determine if ePROM use can prevent/delay the progression of advanced CKD to ESRD, improving patient's quality of life, reducing adverse outcomes, and reducing NHS cost. This study will determine whether the full RCT is possible (informing its design) and will produce new language ePROM versions to widen participation. ePROMs have the potential to become central to chronic disease management in the NHS: improving patient outcomes across a range of long-term conditions. Establishment of a dedicated ePROM research group will help realize these benefits.

Lay abstract: Background: Chronic Kidney Disease (CKD) affects around 1 in 7 people in the UK. Those with advanced CKD may go on to need demanding and costly treatments, such as dialysis, for the rest of their lives. These patients often experience a very poor quality of life and live less long than people without CKD. Patients with advanced CKD can also get worse very quickly. Unfortunately this can happen between visits to their doctor, meaning they need to go to hospital as an emergency and have an increased risk of dying. Some researchers and doctors believe it would be helpful to ask patients to use a computer or smartphone to provide regular information about their quality of life and symptoms, in between their hospital appointments. This information can be collected using questionnaires known as electronic Patient-Reported Outcome Measures' or ePROMs. Doctors believe they could use ePROMs to find out if the patient needs urgent care, so they can take action straight away. This could help slow the progress of the disease in patients with advanced CKD and reduce or delay the need for life-altering treatments such as dialysis or kidney transplantation. International research in cancer has shown that ePROMs can: Improve communication between patients and doctors, helping shared decision-making Improve patient quality of life Reduce the risk of hospital admission Increase patient survival However, ePROMs have not been tested in a full-scale randomised controlled trial (RCT) in CKD. Aims: A RCT is needed to assess whether ePROM use can improve patient outcomes and safety when compared to standard treatment alone in the NHS. Before a definitive trial is undertaken, however, I will conduct important pilot/feasibility work aiming to: Develop the ePROM system with patient/clinician input (including different language versions, so patients for whom English is not their first language can still take part in the study). Determine if a RCT is possible, and to gather information to help plan the trial. Develop a collaborative research group which will: (i) secure funding to run the main RCT, and (ii) begin follow-on research to examine the potential benefit of ePROMs in other long-term conditions managed in the NHS. Design & Methods: I will conduct this work across three projects: PROJECT 1 will focus on the development of the ePROM system, which will allow: (i) patients to input their data in multiple ways (via computer/smartphone/tablet/telephone/scanned paper questionnaires) and (ii) clinicians to view their patient's status in real time. The ePROM will also be translated/validated in Urdu, Punjabi and Bengali which, after English, are the most common languages spoken/read by patients with CKD in the West Midlands region (one of the planned sites for the main RCT). PROJECT 2 is a pilot/feasibility study, in which 60 consenting patients with advanced CKD will be randomly allocated to receive either standard treatment alone or standard treatment plus the ePROM. The study will assess: (a) the willingness of patients to be involved in the study, (b) the best way to measure the impact of the ePROM on patients' health status in the main RCT, (c) the willingness of hospitals across the UK to participate in the main RCT. PROJECT 3 will aim to create a new international collaborative research group who will work together to secure funding: (i) to deliver the main RCT, and (ii) conduct research to investigate the potential benefit of ePROMs in other long-term conditions treated in the NHS. To facilitate development of the group, I will invite expert national and international researchers, clinicians and patients to attend an international ePROM workshop. Establishing these links will help us to conduct this important research as quickly as possible in order to improve the outcomes of NHS patients. Patient and public involvement: A number of patients have helped develop this research proposal. A lived experience advisory panel (LEAP) of patients with CKD will support the research project. These patients will meet with me at key points during the study. The LEAP will assist in: maximizing recruitment, drawing-up study documentation, interpreting patients' acceptability of the ePROM intervention, and dissemination. Patients will also be invited to the ePROM workshop in Project 3. Dissemination: I will be supported in disseminating the findings by an advisory group of patients, clinicians and key academics. In addition to publishing the findings in journals and presenting at conferences, I will also write articles for Kidney charity newsletters, develop an online project web page, and organise an international workshop on ePROMs during project 3 to showcase the study results to leading experts in the field.

Research activity codes: 7.1 INDIVIDUAL CARE NEEDS
Health categories: RENAL AND UROGENITAL