Use of an electronic patient-reported outcome measure in the management of patients with advanced chronic kidney disease:the RePROM pilot trial protocol

INTRODUCTION Chronic kidney disease (CKD) affects up to 16% of adults in the UK. Patient quality of life is particularly reduced in end-stage renal disease and is strongly associated with increased hospitalisation and mortality. Thus, accurate and responsive healthcare is a key priority. Electronic patient-reported outcome measures (ePROMs) are online questionnaires which ask patients to self-rate their health status. Evidence in oncology suggests that the use of ePROM data within routine care, alongside clinical information, may enhance symptom management and improve patient outcomes. However, National Health Service (NHS)-based ePROM research in CKD is lacking. This pilot trial will assess the feasibility of undertaking a full-scale randomised controlled trial (RCT) in patients with CKD within the NHS. METHODS AND ANALYSIS The renal ePROM pilot trial is an investigator-led single-centre, open-label, two-arm randomised controlled pilot trial of 66 participants ≥18 years with advanced CKD. Participants will be randomised to receive either usual care or usual care supplemented with an ePROM intervention. Participants within the intervention arm will be asked to submit monthly self-reports of their health status using the ePROM system. The system will provide tailored information to patients in response to each report and notify the clinical team of patient deterioration. The renal clinical team will monitor for ePROM notifications and will respond with appropriate action, in line with standard clinical practice. Measures of study feasibility, participant quality of life and CKD severity will be completed at 3 monthly intervals. Health economic outcomes will be assessed. Clinicians will record treatment decision-making. Acceptability and feasibility of the protocol will be assessed alongside outcome measure and intervention compliance rates. Qualitative process evaluation will be conducted. ETHICS AND DISSEMINATION The findings will inform the design of a full-scale RCT and the results will be submitted for publication in peer-reviewed journals. The study has ethical approval. TRIAL REGISTRATION NUMBERS ISRCTN12669006; Pre-results

Using electronic patient-reported outcomes to promote quality of care and safety in the management of patients with end stage kidney disease requiring treatment with haemodialysis

Patients with Chronic Kidney Disease (CKD), particularly those with end-stage kidney disease (ESKD) undertaking haemodialysis (HD), often experience a high symptom burden and diminished health-related quality of life. Data on these constructs can be collected directly from patients and measured using electronic patient-reported outcomes (ePROs). Responses can be used at an individual level to facilitate optimal care and improve shared decision making; research from other clinical settings suggests ePROs use can improve clinician/patient communication, improve health related quality of life, reduce hospitalisations, and increase overall survival, while demonstrating cost effectiveness. At an aggregate level, these data can be used for audit and benchmarking to improve services. However, ePROs have not been implemented routinely and systematically in kidney care settings. The overall aim of this thesis was to explore and inform the evidence on how ePROs might be used to promote quality of care and safety in the management of patients with ESKD requiring treatment with HD. Firstly, a synthesis of current evidence on the use and implementation of PROs for patients with ESKD receiving HD was undertaken; identifying gaps in the evidence base and areas for investigation to deliver a roadmap for increasing the evidence base and introducing ePROs into mainstream HD clinical practice. Informed by these findings, a qualitative study was conducted, exploring the views, perceptions, and experiences of 22 patients receiving HD and 17 health care professionals (HCPs) in the HD multi-disciplinary team regarding the collection and use ePROs, in HD settings. This study highlighted the need to measure outcomes important to both patients and clinicians and supported the relevance of patient-reported outcomes included in HD-specific core outcome sets (COS). The study stressed the importance of building an empirical evidence base to support the use of ePROs and discussed key contextual implementation factors, including barriers and facilitators, to successful integration into clinical workflow. Leading on from this was a study to examine whether currently available patient-reported outcome measures (PROMs) used in HD adequately cover outcomes/domains recommended in HD-specific COS. A systematic search of published literature identified two HD-specific COS designed for use in HD research and routine clinical care and 46 validated PROMs currently being used in HD settings. Following assessment of COS coverage and measure characteristics, options for measure choice were outlined. Finally, to gain a deeper understanding of how ePROs might be successfully implemented, a mixed methods study was undertaken to comprehensively identify ePRO systems for CKD to explore system characteristics including methods of administration, levels of integration into existing workflow and electronic health records (EHR) and the reporting of ePROs to manage patient safety. Overall, 15 ePRO systems were identified allowing a comprehensive classification of core factors which contribute to successful implementation of ePROs in nephrology, including common facilitators and barriers. To conclude, this thesis describes the current and potential use of ePROs in CKD, with a focus on use in haemodialysis (HD) populations. It draws together a range of original research, including views on potential measures, existing and emerging ePRO systems and priority areas for future research to outline a roadmap to use ePROs to improve the quality of care delivered to patients with ESKD undergoing HD

Grounds for a New Normal: Integration of Telenephrology in Rural Communities

Patients with kidney disease represent a group of people who are known to have comorbidities, high costs of care, decreased quality of life, and invasive interventions. In rural communities of Western Washington State, many nephrology patients experience factors evidenced to impede access to quality and timely management of their disease, compounding the risk for poor outcomes. Telenephrology is a novel modality of service delivery with the potential to make care more efficient, cost-effective, flexible, and accessible. The author created this project to develop a telenephrology program plan and evaluation for rural settings in Western Washington, informed by relevant epidemiology, pathophysiology, standards of care, and evidence in the literature. As there are a paucity of program plans specific to this service delivery in rural Western Washington, development of this project aims to substantiate the need for evidence-based implementation in this setting. The appraisal of barriers to care for this patient population was used to guide aspects of telenephrology program planning and evaluation to ensure intervention outcomes are equitable. This project provides grounds to support the adoption a new normal for nephrology care and utilizes the existing evidence to inform the planning and evaluating of telenephrology services, implementation processes, budget, and outcomes to assist healthcare providers and their institutions in successful integration of telenephrology in rural communities of Western Washington State

ePROMs in the end-of-life and ethical decision making: An integrative review with narrative synthesis

Introdução: Os Patient-reported outcome measures são ferramentas poderosas que tentam assessar as necessidades dos pacientes de acordo com as suas perspectivas únicas. Permitem à equipa de cuidados de saúde monitorizar a condição e preocupações do paciente fora do ambiente clínico. Porém, a verdadeira inovação neste campo é a digitalização, os electronic patient-reported outcome measures (ePROM’s). Objetivo: O objectivo principal desta revisão é perceber se estas novas tecnologias estão a ser usadas como uma nova ajuda às equipas de cuidados paliativos na sua luta diária em oferecer cuidados de saúde e conforto aos seus pacientes. Métodos: Foi realizada uma revisão sistemática de literatura em que os reports foram recolhidos usando a PubMed e a Web of Science durante novembro de 2021. Durante todo o processo foi utilizada a guideline Preferred Reporting Items for Systematic Review and Meta-Analyses (PRISMA). A procura inicial resultou em 242 records, dos quais 13 cumpriam os critérios de inclusão e exclusão. Destes foi extraída toda a informação relevante no que toca ao uso dos ePROM’s. Resultados: Os principais usos dos ePROM’s no final de vida (end-of-life) são a avaliação da qualidade de vida (QOL assessment), a avaliação de carga sintomática e avaliações diretas e a decisão de introdução de cuidados paliativos. Em 61.5% dos casos, os ePROM’s tiveram um impacto positivo na qualidade de vida dos pacientes. Em 46.15% dos casos, os ePROM’s levaram as equipas de PC.Background: Patient-reported outcome measures (PROMS) are powerful tools that attempt to assess patients' needs from their unique perspectives. They allow the healthcare team to monitor the patient's condition and concerns outside the clinical setting. However, the real innovation in this field is their digitization: electronic patient-reported outcome measures (ePROMS). Objective: The main of this review is to assess whether these new technologies are being used as a novel aid to palliative care teams in their daily struggle to provide comfort to their patients. Methods: A systematic review was performed using PubMed and Web of Science. Through November 2021. Followed Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines at all stages. The search strategy yielded a total of 242 records, of which 13 met the predefined inclusion and exclusion criteria— extracted relevant information related to ePROM from each study. Results: The main uses of ePROMs in end-of-life scenarios are quality-of-life assessment, symptom burden and simple assessments, and the decision to introduce PC. In 61.5% of cases, ePROMs positively impacted patients' quality of life. In addition, in 46.15% of cases, the ePROMs led the PC teams to make an ethical decision; the same relative value as in the circumstances did not define the direction in ethical terms. Conclusion: Professionals and patients need to be reminded that these tools exist and can be applied in many situations. If used correctly, they can provide better QoL and QoC to patients in PC and better information provision to professionals

Using patient-reported outcome measures during the management of patients with end-stage kidney disease requiring treatment with haemodialysis (PROM-HD): a qualitative study

Objectives Patients undergoing haemodialysis report elevated symptoms and reduced health-related quality of life, and often prioritise improvements in psychosocial well-being over long-term survival. Systematic collection and use of patient-reported outcomes (PROs) may help support tailored healthcare and improve outcomes. This study investigates the methodological basis for routine PRO assessment, particularly using electronic formats (ePROs), to maximise the potential of PRO use, through exploration of the experiences, views and perceptions of patients and healthcare professionals (HCPs) on implementation and use of PROs in haemodialysis settings. Study design Qualitative study. Setting and participants Semistructured interviews with 22 patients undergoing haemodialysis, and 17 HCPs in the UK. Analytical approach Transcripts were analysed deductively using the Consolidated Framework for Implementation Research (CFIR) and inductively using thematic analysis. Results For effective implementation, the potential value of PROs needs to be demonstrated empirically to stakeholders. Any intervention must remain flexible enough for individual and aggregate use, measuring outcomes that matter to patients and clinicians, while maintaining operational simplicity. Any implementation must sit within a wider framework of education and support for both patients and clinicians who demonstrate varying previous experience of using PROs and often confuse related concepts. Implementation plans must recognise the multidimensionality of end-stage kidney disease and treatment by haemodialysis, while acknowledging the associated challenges of delivering care in a highly specialised environment. To support implementation, careful consideration needs to be given to barriers and facilitators including effective leadership, the role of champions, effective launch and ongoing evaluation. Conclusions Using the CFIR to explore the experiences, views and perceptions of key stakeholders, this study identified key factors at organisational and individual levels which could assist effective implementation of ePROs in haemodialysis settings. Further research will be required to evaluate subsequent ePRO interventions to demonstrate the impact and benefit to the dialysis community

Symptom burden and health-related quality of life in chronic kidney disease:A global systematic review and meta-analysis

BACKGROUND: The importance of patient-reported outcome measurement in chronic kidney disease (CKD) populations has been established. However, there remains a lack of research that has synthesised data around CKD-specific symptom and health-related quality of life (HRQOL) burden globally, to inform focused measurement of the most relevant patient-important information in a way that minimises patient burden. The aim of this review was to synthesise symptom prevalence/severity and HRQOL data across the following CKD clinical groups globally: (1) stage 1-5 and not on renal replacement therapy (RRT), (2) receiving dialysis, or (3) in receipt of a kidney transplant.METHODS AND FINDINGS: MEDLINE, PsycINFO, and CINAHL were searched for English-language cross-sectional/longitudinal studies reporting prevalence and/or severity of symptoms and/or HRQOL in CKD, published between January 2000 and September 2021, including adult patients with CKD, and measuring symptom prevalence/severity and/or HRQOL using a patient-reported outcome measure (PROM). Random effects meta-analyses were used to pool data, stratified by CKD group: not on RRT, receiving dialysis, or in receipt of a kidney transplant. Methodological quality of included studies was assessed using the Joanna Briggs Institute Critical Appraisal Checklist for Studies Reporting Prevalence Data, and an exploration of publication bias performed. The search identified 1,529 studies, of which 449, with 199,147 participants from 62 countries, were included in the analysis. Studies used 67 different symptom and HRQOL outcome measures, which provided data on 68 reported symptoms. Random effects meta-analyses highlighted the considerable symptom and HRQOL burden associated with CKD, with fatigue particularly prevalent, both in patients not on RRT (14 studies, 4,139 participants: 70%, 95% CI 60%-79%) and those receiving dialysis (21 studies, 2,943 participants: 70%, 95% CI 64%-76%). A number of symptoms were significantly (p &lt; 0.05 after adjustment for multiple testing) less prevalent and/or less severe within the post-transplantation population, which may suggest attribution to CKD (fatigue, depression, itching, poor mobility, poor sleep, and dry mouth). Quality of life was commonly lower in patients on dialysis (36-Item Short Form Health Survey [SF-36] Mental Component Summary [MCS] 45.7 [95% CI 45.5-45.8]; SF-36 Physical Component Summary [PCS] 35.5 [95% CI 35.3-35.6]; 91 studies, 32,105 participants for MCS and PCS) than in other CKD populations (patients not on RRT: SF-36 MCS 66.6 [95% CI 66.5-66.6], p = 0.002; PCS 66.3 [95% CI 66.2-66.4], p = 0.002; 39 studies, 24,600 participants; transplant: MCS 50.0 [95% CI 49.9-50.1], p = 0.002; PCS 48.0 [95% CI 47.9-48.1], p = 0.002; 39 studies, 9,664 participants). Limitations of the analysis are the relatively few studies contributing to symptom severity estimates and inconsistent use of PROMs (different measures and time points) across the included literature, which hindered interpretation.CONCLUSIONS: The main findings highlight the considerable symptom and HRQOL burden associated with CKD. The synthesis provides a detailed overview of the symptom/HRQOL profile across clinical groups, which may support healthcare professionals when discussing, measuring, and managing the potential treatment burden associated with CKD.PROTOCOL REGISTRATION: PROSPERO CRD42020164737.</p

The development and utility of electronic patient reported outcome measures in gynaecology

Aims and Objectives: Patient reported outcome measures (PROMs) are tools which seek to ascertain patients’ views of their symptoms, their functional status and their health- related quality of life, without interruption from a healthcare professional. Usually PROMs are questionnaires, they may include multiple choice components and/or free text answers. In order for the use of these tools to be considered reliable and valid in clinical practice, psychometric testing to provide evidence of reliability, validity and functionality should be undertaken. Electronic PROMs or ePROMs are such tools which are delivered electronically and completed on a computer or handheld device, often with responses uploaded onto the internet allowing remote completion and access of results. The ePAQ system is an ePROM platform which utilises an online electronic response system for completion of PROMs. The original ePROM for which the ePAQ system was developed is ePAQ-Pelvic Floor (ePAQ-PF). This is a pelvic floor questionnaire widely used in clinical practice with excellent evidence foe reliability, validity and functionality. This thesis aims to present a narrative on the development and utility of electronic patient reported outcome measures in gynaecology, including systematic literature reviews of existing tools prior to development/utilisation of ePROMs (papers 1 and 2), development and initial psychometric testing of two novel gynaecology ePROMs using the ePAQ system (papers 3 and 4) and discussion of the utility of ePROMs in gynaecology both as tools for clinical practice and in research (papers 5, 6 and 7). Methods: Two systematic reviews following PRISMA guidelines and registered prospectively on the PROSPERO database (CRD42017082508 and CRD42017084710) were undertaken. Paper 1 was a systematic review of non-invasive modalities used to identify women with anal incontinence following childbirth; to assess the existing PROMs for this purpose with a view to deploying the ePROM ePAQ-PF for this purpose in a future study. Paper 2 was a systematic review of PROMs, and their psychometric properties, used to assess body image in a urogynaecology patients. This was done to evaluate existing tools available, to inform development of a domain (area within a questionnaire) in ePAQ- PF to assess body image. The development and initial psychometric testing of a two novel gynaecology ePROMs based on the ePAQ system is described in papers 3 and three ethically approved retrospective cohort studies using data collected from ePAQ-PF as part of routine clinical practice are described in papers 5-7. Each of these papers deals with different symptom complexes and issues occurring in urogynaecology with the aim of presenting the utility of ePROMs in gynaecology both as clinical tools and research tools. In paper 5 the aim was to measure the prevalence of coital urinary incontinence (leakage of urine during sexual intercourse). In this study 2312 women completed ePAQ-PF in advance of their urogynaecology consultation. Logistic regression and Spearman’s rank correlation evaluated associations between types of coital incontinence and different types of urinary incontinence (urgency incontinence and stress incontinence). Mann-Witney test evaluated the relationship between coital incontinence and self-reported quality-of–sex-life and self -avoidance and partner-avoidance of sex using data from ePAQ-PF. A subgroup analysis was undertaken to analyse outcomes in 84 women with coital incontinence undergoing tension free vaginal tape (an operation to treat stress incontinence). In paper 6 the aim was to use data from ePAQ-PF to assess the relationship between pelvic organ prolapse symptoms, subjective outcomes of surgery and body mass index (BMI) in women undergoing vaginal hysterectomy for treatment of prolapse. Pre- and post-operative data from ePAQ-PF were collected prospectively from 60 women undergoing vaginal hysterectomy for prolapse. Of these, 20 were normal weight (BMI 18.5 – 24.9), 20 were overweight (BMI 25 – 29.9) and 20 were women with obesity (BMI 30 – 34.9). The relationship between BMI and symptom scores for prolapse, impact on vaginal symptoms on quality of life and ‘overall change in condition’ was assessed. Pre- and post-operative symptom scores were compared using repeated mixed ANOVA test for BMI as a categorical variable (Normal, Overweight and Obese). Spearman’s rank order correlation test was carried out to evaluate BMI as a continuous variable. All women underwent vaginal hysterectomy using a standardised technique. ePAQ-PF uses a free test question to assess the concerns and treatment goals of patients completing this ePROM. All patients are asked: ‘Considering the issues that currently concern you the most, what do you hope to achieve from any help, advice or treatment?’. The objectives of the study presented in paper 7 were to undertake a content analysis of the free-text concerns and goals recorded by patients completing this item in ePAQ-PF and measure how these related to self-reported symptoms and health-related quality-of-life (HRQOL) data also recorded using ePAQ-PF. In total, 1996 consenting patients completed ePAQ-PF. Content analysis was undertaken of the free-text item responses. Key content themes were identified by the lead researcher, and three researchers read and coded all recorded responses. Student’s t test was used to compare ePAQ-PF domain scores for patients reporting concerns in the relevant domain with those who did not. Results: Paper 1: 109 studies were included from 1602 screened articles. 36 studies utilising 15 different PROM instruments were identified. Mean response rates were 92% up to six weeks after childbirth. Non-personalised assessment modalities (PROMs) were associated with reporting of higher rates of anal incontinence compared to patient interview at all periods of follow up after childbirth, this was statistically significant between six weeks and one year after childbirth (p<0.05). Paper 2: 17 studies were included from 3207 screened articles. Seven different PROMs used to assess body image in a urogynaecological population were identified. Two of these PROMs (Genital Self-Image Scale-20 and Body Image in Pelvic Organ Prolapse questionnaire) had good psychometric evidence for use but this was only in the context of women with prolapse. Evidence for validity and reliability was limited for the other five PROMs identified. Paper 3 (development/testing of ePAQ-Vulva): Factor analysis identified five principal components. These were reviewed and amended to provide a putative domain structure of six domains. Internal reliability of these domains was assessed using Cronbach’s alpha, producing values of 0.715 to 0.917. Inter-rater reliability of the picture items produced a Kappa statistic of 0.405. Spearman’s rank showed moderate correlation between multiple choice answers and free-text concerns (r=0.364 – 0.462) in three of the six domains (Pain, Sex and Dyspareunia). Paper 4 (development/testing of ePAQ-Menstrual, Pain and Hormonal (ePAQ-MPH)): Exploratory factor analysis identified 18 domains (Cronbach’s α >0.7) and 30 redundant items. Test-retest analysis found acceptable intra-class correlations of 0.6–0.9 (p<0.05). Eight domains were compared with Menstrual Distress Questionnaire showing moderate or strong correlation in seven domains. Ten domains were compared with Women’s Health Questionnaire, six of which showed moderate correlation. Mean QQ-10 Value and Burden scores were 76 and 25, respectively (SD=15.8 and 15.5). The mean completion time for ePAQ-MPH was 31 minutes. Confirmatory factor analysis of the revised version 2 instrument with 15 domains showed good model fit. Paper 5: Prevalence of coital incontinence in the cohort was 30%. Symptoms of both urgency incontinence (p<0.005) and stress incontinence (p<0.005) were significantly and independently associated with both types of coital incontinence (orgasm & penetration). In women with coital incontinence compared with those without, there was significant self- avoidance of sex (p<0.0005), partner-avoidance of sex (p<0.0005) and impaired quality-of- sex-life due to sexual problems (p<0.005). Subgroup analysis of 84 women undergoing TVT showed significant improvement in all coital incontinence symptoms three months post- operatively. Paper 6: Overall, 93% of women reported improvement in their condition. The main finding was that ‘Overall change in condition’ was negatively correlated with increasing BMI (rs= - 0.324, p=0.028). Irrespective of BMI, significant improvements were observed in symptoms of prolapse and vaginal symptoms impact on health-related quality of life at three-months post operation. Paper 7: 63% of participants who completed the questionnaire, recorded at least one free- text item. Content analysis identified 1560 individual concerns coding into the 19 ePAQ-PF domains. Symptom scores were significantly higher for patients reporting free-text concerns in 18 domains (p<0.05) indicating convergent validity of ePAQ-PF. Additional concerns relating specifically to body image were recorded by 11% of patients. Conclusions: This narrative thesis presents good evidence for the use of ePROMs in gynaecology, both as clinical and research tools. Electronic patient reported outcome measures in gynaecology have an important role in supporting self- expression and increasing disclosure of potentially taboo or embarrassing symptoms. Their use as research tools is valuable allowing symptom complexes to be explored and outcomes after interventions measured accurately. Further research and development of ePROMs in gynaecology will be needed to support the NHS Long Term Plan

Routine self-reporting of symptoms and side effects during cancer treatment: The patient’s perspective

Introduction There has been a dramatic increase in web-based systems developed to support patients to report/manage cancer treatment side effects (ePROM systems). However, little is known about processes underpinning patient engagement and impact on experience. Aims To explore the patient perspective on using ePROM systems during chemotherapy. Mixed methods Preliminary work Interviews (n=87) and questionnaires (n=40) explored patient experience of chemotherapy and indicated that difficulty deciding when to seek medical support during treatment was common. Field usability testing of eRAPID (n=12) indicated potential to support patients but variable engagement. A systematic review of ePROM systems (n=41) indicated a scarcity of robust evidence with few RCTs, with patient engagement and psychosocial outcomes such as self-efficacy not routinely explored or assessed. Main studies Qualitative and quantitative evaluation of patient engagement/experience was integrated into an RCT to evaluate eRAPID (n=354). Engagement was evaluated by weekly symptom reports and use of website. Validated measures assessed impact of eRAPID on self-efficacy to manage side effects (CSES) and cope with cancer (CBI-B), and patient activation (PAM). Relationships between outcomes and engagement were explored. A subset of patients were interviewed (n=23) to explore patient engagement/experience. Triangulation techniques were used to compare and contrast findings. Results Engagement was generally high with few barriers to use reported. One of the main motivators for sustained patient engagement was providing information to clinicians for use in consultations. Patients reported eRAPID provided psychological benefits and improved care. There was a positive impact of eRAPID on CSES (p=.015) but not CBI-B or PAM. Engagement was a significant predictor of improvement in CSES (p<.001) and CBI-B (p<.01) but not PAM. Conclusion ePROM systems have potential to improve patients experience of chemotherapy. Further exploration using qualitative and quantitative assessments is needed to provide insights into motivators and barriers. Clinician engagement is intertwined with patient engagement and requires ongoing assessment to inform future development and implementation