Patient-Reported Outcome Measures for Patients With CKD: The Case for Patient-Reported Outcomes Measurement Information System (PROMIS) Tools.

Chronic kidney disease (CKD), kidney failure, and kidney replacement therapies are associated with high symptom burden and impaired health-related quality of life (HRQOL). Symptoms change with disease progression or transition between treatment modalities and frequently go unreported and unmanaged. Tools that reliably monitor symptoms may improve the management of patients with CKD. Patient-reported outcome measures (PROMs) assess symptom severity; physical, psychological, social, and cognitive functioning; treatment-related side effects; and HRQOL. Systematic use of PROMs can improve patient-provider communication, patient satisfaction, clinical outcomes, and HRQOL. Potential barriers to their use include a lack of engagement, response burden, and limited guidance about PROM collection, score interpretation, and workflow integration. Well-defined, acceptable, and effective clinical response pathways are essential for implementing PROMs. PROMs developed by the Patient-Reported Outcomes Measurement Information System (PROMIS) address some challenges and may be suitable for clinical use among patients with CKD. PROMIS tools assess multiple patient-valued, clinically actionable symptoms and functions. They can be administered as fixed-length, customized short forms or computer adaptive tests, offering precise measurement across a range of symptom severities or function levels, tailored questions to individuals, and reduced question burden. Here we provide an overview of the potential use of PROMs in CKD care, with a focus on PROMIS.

A patient-centred approach to measuring quality in kidney care: patient-reported outcome measures and patient-reported experience measures.

PURPOSE OF REVIEW: Chronic kidney disease (CKD) is associated with symptoms that can significantly reduce the health-related quality of life (HRQOL) of patients. Patient-reported outcome and experience measures (PROMs and PREMs) may assist with the evaluation of HRQOL and quality of care from the patient perspective. This review focuses on evidence from recent studies exploring the role of PROMs and PREMs in the measurement of quality in CKD care. RECENT FINDINGS: PROMs are increasingly used in CKD research as measures of clinical effectiveness, whereas the current use of PROMs in routine clinical settings and PREMs in all settings is more limited. Electronic PROMs may be sensitive enough to detect clinically relevant patient-reported outcomes changes. Patients on frequent shorter-hours daily haemodialysis may experience better HRQOL compared with those on conventional haemodialysis. PROM data may correlate significantly with clinical parameters. PREMs are being utilized by healthcare professionals to inform service improvements. SUMMARY: PROMs and PREMs may facilitate the measurement of quality in renal care and aid the tailoring of care to individual patients. PROMs may have a potential role as prognostic markers.

Patient-reported outcomes in integrated health and social care: A scoping review.

Background: Patient-reported outcomes (PROs) have potential to support integrated health and social care research and practice; however, evidence of their utilisation has not been synthesised. Objective: To identify PRO measures utilised in integrated care and adult social care research and practice and to chart the evidence of implementation factors influencing their uptake. Design: Scoping review of peer-reviewed literature. Data sources: Six databases (01 January 2010 to 19 May 2023). Study selection: Articles reporting PRO use with adults (18+ years) in integrated care or social care settings. Review methods: We screened articles against pre-specified eligibility criteria; 36 studies (23%) were extracted in duplicate for verification. We summarised the data using thematic analysis and descriptive statistics. Results: We identified 159 articles reporting on 216 PRO measures deployed in a social care or integrated care setting. Most articles used PRO measures as research tools. Eight (5.0%) articles used PRO measures as an intervention. Articles focused on community-dwelling participants (35.8%) or long-term care home residents (23.9%), with three articles (1.9%) focussing on integrated care settings. Stakeholders viewed PROs as feasible and acceptable, with benefits for care planning, health and wellbeing monitoring as well as quality assurance. Patient-reported outcome measure selection, administration and PRO data management were perceived implementation barriers. Conclusion: This scoping review showed increasing utilisation of PROs in adult social care and integrated care. Further research is needed to optimise PROs for care planning, design effective training resources and develop policies and service delivery models that prioritise secure, ethical management of PRO data.

"I just wanted to speak to someone- and there was no one ": using Burden of Treatment Theory to understand the impact of a novel ATMP on early recipients.

BACKGROUND: Advanced therapy medicinal products such as Chimeric antigen receptor T-cell therapy offer ground-breaking opportunities for the treatment of various cancers, inherited diseases, and chronic conditions. With development of these novel therapies continuing to increase it's important to learn from the experiences of patients who were among the first recipients of ATMPs. In this way we can improve the clinical and psychosocial support offered to early patient recipients in the future to support the successful completion of treatments and trials. STUDY DESIGN: We conducted a qualitative investigation informed by the principles of the key informant technique to capture the experience of some of the first patients to experience CAR-T therapy in the UK. A directed content analysis was used to populate a theoretical framework informed by Burden of Treatment Theory to determine the lessons that can be learnt in supporting their care, support, and ongoing self-management. RESULTS: A total of five key informants were interviewed. Their experiences were described within the three domains of the burden of treatment framework; (1) The health care tasks delegated to patients, Participants described the frequency of follow-up and the resources involved, the esoteric nature of the information provided by clinicians; (2) Exacerbating factors of the treatment, which notably included the lack of understanding of the clinical impacts of the treatment in the broader health service, and the lack of a peer network to support patient understanding; (3) Consequences of the treatment, in which they described the anxiety induced by the process surrounding their selection for treatment, and the feeling of loneliness and isolation at being amongst the very first recipients. CONCLUSIONS: If ATMPs are to be successfully introduced at the rates forecast, then it is important that the burden placed on early recipients is minimised. We have discovered how they can feel emotionally isolated, clinically vulnerable, and structurally unsupported by a disparate and pressured health service. We recommend that where possible, structured peer support be put in place alongside signposting to additional information that includes the planned pattern of follow-up, and the management of discharged patients would ideally accommodate individual circumstances and preferences to minimize the burden of treatment.

Identifying patient-valued outcomes for use in early phase trials of ocular surface disease interventions.

BACKGROUND: Patient-reported outcomes (PROs) can be used to evaluate the impact of dry eye symptoms (DES) on daily life. Early-phase clinical trials provide an opportunity to evaluate PRO strategies. Existing measures identified through systematic review omitted important concepts that mattered to patients. The aim of our work was to develop a conceptual map of DES and assess the relative importance of identified concepts. METHODS: Web-based group concept mapping software was used to develop a conceptual map. This semi-quantitative mixed-methods approach consists of three stages 1) statement generation, 2) thematic sorting, 3) rating of statements for importance [1 (not important), 2 (important), 3 (very important)] and relevance [1 (not my experience), 2 (sometimes my experience); 3 (definitely my experience)]. Thirty-nine participants were recruited from two UK-based patient support groups (British Sjögren's Syndrome Association, PemFriends). Three withdrew, two for health reasons and one struggled with the web-based format. RESULTS: 125 statements and six thematic clusters were generated. The Environmental Impacts cluster scored highest for importance (2.45), followed by Pain and Discomfort (2.35), Eye Treatments (2.32), Daily Impact (2.07), Psychosocial Issues (1.78) and Miscellaneous (1.78). Mapping statements against existing PRO measures confirmed a number of important missing issues including the impact of 'UV levels' (2.50), hot dry weather (2.33), the temporal aspects of pain (2.64), and issues with night-driving (2.59). CONCLUSIONS: Group concept mapping identified important issues for people living with DES not currently captured by existing PROs, highlighting the need for additional PRO items to be considered for use in clinical trials.

Development and validation of the symptom burden questionnaire for long covid (SBQ-LC): Rasch analysis.

OBJECTIVE: To describe the development and validation of a novel patient reported outcome measure for symptom burden from long covid, the symptom burden questionnaire for long covid (SBQ-LC). DESIGN: Multiphase, prospective mixed methods study. SETTING: Remote data collection and social media channels in the United Kingdom, 14 April to 1 August 2021. PARTICIPANTS: 13 adults (aged ≥18 years) with self-reported long covid and 10 clinicians evaluated content validity. 274 adults with long covid field tested the draft questionnaire. MAIN OUTCOME MEASURES: Published systematic reviews informed development of SBQ-LC's conceptual framework and initial item pool. Thematic analysis of transcripts from cognitive debriefing interviews and online clinician surveys established content validity. Consensus discussions with the patient and public involvement group of the Therapies for Long COVID in non-hospitalised individuals: From symptoms, patient reported outcomes and immunology to targeted therapies (TLC Study) confirmed face validity. Rasch analysis of field test data guided item and scale refinement and provided initial evidence of the SBQ-LC's measurement properties. RESULTS: SBQ-LC (version 1.0) is a modular instrument measuring patient reported outcomes and is composed of 17 independent scales with promising psychometric properties. Respondents rate their symptom burden during the past seven days using a dichotomous response or 4 point rating scale. Each scale provides coverage of a different symptom domain and returns a summed raw score that can be transformed to a linear (0-100) score. Higher scores represent higher symptom burden. After rating scale refinement and item reduction, all scales satisfied the Rasch model requirements for unidimensionality (principal component analysis of residuals: first residual contrast values <2.00 eigenvalue units) and item fit (outfit mean square values within 0.5 -1.5 logits). Rating scale categories were ordered with acceptable category fit statistics (outfit mean square values <2.0 logits). 14 item pairs had evidence of local dependency (residual correlation values >0.4). Across the 17 scales, person reliability ranged from 0.34 to 0.87, person separation ranged from 0.71 to 2.56, item separation ranged from 1.34 to 13.86, and internal consistency reliability (Cronbach's alpha) ranged from 0.56 to 0.91. CONCLUSIONS: SBQ-LC (version 1.0) is a comprehensive patient reported outcome instrument developed using modern psychometric methods. It measures symptoms of long covid important to people with lived experience of the condition and may be used to evaluate the impact of interventions and inform best practice in clinical management.

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.