Core outcome measurement set for research and clinical practice in post-COVID-19 condition (long COVID) in children and young people: an international Delphi consensus study "PC-COS Children".

The coronavirus disease 2019 (COVID-19) pandemic substantially impacted different age groups, with children and young people not exempted. Many have experienced enduring health consequences. Presently, there is no consensus on the health outcomes to assess in children and young people with post-COVID-19 condition. Furthermore, it is unclear which measurement instruments are appropriate for use in research and clinical management of children and young people with post-COVID-19. To address these unmet needs, we conducted a consensus study, aiming to develop a core outcome set (COS) and an associated core outcome measurement set (COMS) for evaluating post-COVID-19 condition in children and young people. Our methodology comprised of two phases. In phase 1 (to create a COS), we performed an extensive literature review and categorisation of outcomes, and prioritised those outcomes in a two-round online modified Delphi process followed by a consensus meeting. In phase 2 (to create the COMS), we performed another modified Delphi consensus process to evaluate measurement instruments for previously defined core outcomes from phase 1, followed by an online consensus workshop to finalise recommendations regarding the most appropriate instruments for each core outcome. In phase 1, 214 participants from 37 countries participated, with 154 (72%) contributing to both Delphi rounds. The subsequent online consensus meeting resulted in a final COS which encompassed seven critical outcomes: fatigue; post-exertion symptoms; work/occupational and study changes; as well as functional changes, symptoms, and conditions relating to cardiovascular, neuro-cognitive, gastrointestinal and physical outcomes. In phase 2, 11 international experts were involved in a modified Delphi process, selecting measurement instruments for a subsequent online consensus workshop where 30 voting participants discussed and independently scored the selected instruments. As a result of this consensus process, four instruments met a priori consensus criteria for inclusion: PedsQL multidimensional fatigue scale for "fatigue"; PedsQL gastrointestinal symptom scales for "gastrointestinal"; PedsQL cognitive functioning scale for "neurocognitive" and EQ-5D for "physical functioning". Despite proposing outcome measurement instruments for the remaining three core outcomes ("cardiovascular", "post-exertional malaise", "work/occupational and study changes"), a consensus was not achieved. Our international, consensus-based initiative presents a robust framework for evaluating post-COVID-19 condition in children and young people in research and clinical practice via a rigorously defined COS and associated COMS. It will aid in the uniform measurement and reporting of relevant health outcomes worldwide.

Depression as a risk factor for adverse outcomes and increased healthcare utilization in bariatric surgery patients.

INTRODUCTION: Depression is strongly associated with obesity and is common among patients undergoing bariatric surgery. Little is known about the impact of depression on early postoperative outcomes or its association with substance use. METHODS: The Michigan Bariatric Surgery Collaborative is a statewide quality improvement program that maintains a large clinical registry. We evaluated patients undergoing primary Roux-en-Y gastric bypass or sleeve gastrectomy between 2017 and 2022. Patients self-reported symptoms of depression (PHQ-8) and use of alcohol (AUDIT-C), smoking, prescription opiates, and marijuana at baseline. Preoperative PHQ-8 scores stratified patients based on severity: no depression (0-4), mild (5-9), moderate (10-14), or severe (15-24). We compared 30-day outcomes and substance use between patients with and without depression. RESULTS: Among 44,301 patients, 30.8% had some level of depression, with 19.8% mild, 7.5% moderate, and 3.5% severe. Patients with depression were more likely to have an extended length of stay (LOS) (> 3 days) than those without depression (no depression 2.1% vs. severe depression 3.0%, p = 0.0452). There were no significant differences between no depression and severe depression groups in rates of complications (5.7% vs. 5.2%, p = 0.1564), reoperations (0.9%, vs. 0.8%, p = 0.7394), ED visits (7.7% vs. 7.8%, p = 0.5353), or readmissions (3.2% vs. 3.9%, p = 0.3034). Patients with severe depression had significantly higher rates of smoking (9.7% vs. 12.5%, p < 0.0001), alcohol use disorder (8.6% vs. 14.0%, p < 0.0001), opiate use (14.5% vs. 22.4%, p < 0.0001) and marijuana use (8.4%, vs. 15.5%, p = 0.0008). CONCLUSIONS: This study demonstrated that nearly one-third of patients undergoing bariatric surgery have depression, with over 10% in the moderate to severe range. There was a significant association between preoperative depressive symptoms and extended LOS after bariatric surgery, as well as higher rates of smoking and use of marijuana, prescription opiates and alcohol. There was no significant effect on adverse events or other measures of healthcare utilization.

Rasch Analysis of the Listening Effort Questionnaire-Cochlear Implant.

OBJECTIVES: Listening effort may be defined as the attentional and cognitive resources needed to understand an auditory message, modulated by motivation. Despite the use of hearing devices such as hearing aids or cochlear implants (CIs), the requirement for high listening effort remains a challenge for individuals with hearing loss. The Listening Effort Questionnaire-Cochlear Implant (LEQ-CI) is a hearing-specific patient-reported outcome measure (PROM), which has been designed for use in the CI candidacy and rehabilitation process to assess perceived listening effort in everyday life in adults with severe-profound hearing loss. The LEQ-CI has been developed in line with international consensus-based standards for best practice in PROM construction. The aim of this study was to improve the measurement precision of the LEQ-CI and to assess its psychometric measurement properties. DESIGN: A field test was undertaken with 330 CI patients from five National Health Service auditory implant centers in the United Kingdom. Participants were adults (≥18 years of age), had a severe-profound hearing loss, and met the UK candidacy criteria for cochlear implantation specified by the National Institute for Health and Care Excellence (NICE). Participants completed and returned an anonymized 29-item (each with a 5- or 7-point response option), draft version of the LEQ-CI (LEQ-CI29) and a demographic questionnaire. Rasch analysis was undertaken using Winsteps software and the partial credit model to assess rating scale function and item fit. Results informed refinements to produce a 21-item version (LEQ-CI21), which underwent a further Rasch analysis. RESULTS: The sample was predominantly female: 60.3% (n = 191). Median age of participants was 66 (range 21 to 89) years, with 7.3% (n = 24) of respondents being CI candidates and 92.7% (n = 306) being CI recipients. Mean duration of implantation was 3.8 (SD = 4.8) years. Initial Rasch analysis of the LEQ-CI29 revealed poor rating scale functioning. Collapsing the 5- and 7-point rating scales to 3- and 4-point scales and removing eight items produced a 21-item PROM (LEQ-CI21). Rasch analysis of the LEQ-CI21 showed good fit to the Rasch measurement model. No items showed misfit and dimensionality analysis supported the existence of a single Rasch dimension, defined as perceived listening effort in daily life. Person reliability was 0.91 and the person separation index was 3.28, establishing four levels of person ability. The item separation index was 9.69, confirming the item hierarchy. No items showed differential item functioning for gender or age. The item difficulty range was -0.81 to 1.05, the person ability range for nonextreme persons was -3.54 to 2.49, and the mean person ability was -0.31. CONCLUSIONS: Overall, the LEQ-CI21 was found to meet the Rasch model criteria for interval-level measurement. The LEQ-CI21 is the first PROM to be developed specifically for the measurement of perceived listening effort and one of the first patient-reported outcome measures for use with CI patients to be developed using Rasch analysis. The LEQ-CI21 has the potential to be used as a research tool and in clinical practice to evaluate perceived listening effort in daily life. Further psychometric evaluation of the LEQ-CI21 is planned.

Frameworks for Change in Hearing Research: Valuing Qualitative Methods in the Real World.

In this article, we examine ecological validity in hearing science from a qualitative methodological perspective. We present an overview of qualitative methods, presenting their key characteristics and contrasting these techniques with quantitative approaches to enquiry. We argue that ecological validity sits at the heart of the qualitative paradigm and seek to clearly emphasize the methodological gap that could be effectively filled by qualitative or mixed methods. In doing so, we discuss qualitative methods that may work particularly well in enhancing ecological validity in hearing science and explore their range of applications in this field. These approaches can be applied to a wide range of hearing health research questions to present a unique understanding of people's experiences of disease and disability, indicating gradations of personal health and illness in nuanced ways. We acknowledge and commend the current expansion of qualitative methods within hearing science and present recommendations for increasing ecological validity, both in the design of future studies and in the context of the wider research cycle. We call on qualitative researchers to strive for transparency, rigor, and trustworthiness and highlight challenges to be overcome if qualitative methods are to contribute to effective, efficient research strategies. To facilitate the transference of high-quality research findings into practice, we stress the need for joined-up working to create a research culture that promotes coproduction of ecologically valid research designs, involving not only hearing researchers but also implementation scientists, hearing healthcare professionals and, most importantly, people with hearing loss for whom these efforts could make a difference.

Adults' cochlear implant journeys through care: a qualitative study.

BACKGROUND: Cochlear implants (CIs) can provide a sound sensation for those with severe sensorineural hearing loss (SNHL), benefitting speech understanding and quality of life. Nevertheless, rates of implantation remain low, and limited research investigates journeys from traditional hearing aids to implantable devices. METHOD: Fifty-five adults (≥ 50 years), hearing aid users and/or CI users, General Practitioners, and Australian and United Kingdom audiologists took part in a multi-methods study. Focus groups, interviews, and surveys were thematically analysed. RESULTS: One hundred forty-three data-capture events disclosed 2 themes: 1) "The burden of hearing loss and the impact of Cochlear Implants", and 2) "Professional Support and Practice, and HCPs Roles and Responsibilities". CONCLUSIONS: Care experience can include convoluted, complex journeys towards cochlear implantation. The significant impact of this, as hearing loss progresses, motivates people to consider implants, but they and healthcare professionals need clear supported with defined referral pathways, and less system complexity.

Hearing impairment and daily-life fatigue: a qualitative study.

Objective: Hearing impairment is linked to increased fatigue, yet little is known about the real-world impact of this fatigue. This qualitative study investigated the experience of daily-life fatigue in people with a hearing impairment. Design: Individual face-to-face semi-structured interviews were conducted. Thematic analysis was then used to analyse the data. Study sample: Fourteen hearing impaired participants (aged 44-70 years) who varied in terms of hearing loss, hearing aid status, age and gender. Results: The themes and sub-themes that emerged from the transcripts were: Fatigue (effort-driven fatigue, emotion-driven fatigue, breaks and recovery, the perceived relationship between hearing impairment and fatigue, and sleep), Effort (cognitive effort and physical effort), Coping Strategies (withdrawal, avoidance and planning), Relationships and Emotions, Hearing Aid Impact. Conclusions: The study highlights that hearing impairment-related fatigue is experienced by many but not all, and to different extents. Hearing aids were weakly linked to a beneficial effect on fatigue. In addition to the more widely researched effort-driven fatigue, participants described fatigue linked to the negative emotions related to having a hearing impairment. These findings, in conjunction with the widespread utilisation of different coping strategies, demonstrate that the experience of fatigue is varied and likely dependent on personal factors and lifestyle.

Social Connectedness and Perceived Listening Effort in Adult Cochlear Implant Users: A Grounded Theory to Establish Content Validity for a New Patient-Reported Outcome Measure.

OBJECTIVES: Individuals with hearing loss often report a need for increased effort when listening, particularly in challenging acoustic environments. Despite audiologists' recognition of the impact of listening effort on individuals' quality of life, there are currently no standardized clinical measures of listening effort, including patient-reported outcome measures (PROMs). To generate items and content for a new PROM, this qualitative study explored the perceptions, understanding, and experiences of listening effort in adults with severe-profound sensorineural hearing loss before and after cochlear implantation. DESIGN: Three focus groups (1 to 3) were conducted. Purposive sampling was used to recruit 17 participants from a cochlear implant (CI) center in the United Kingdom. The participants included adults (n = 15, mean age = 64.1 years, range 42 to 84 years) with acquired severe-profound sensorineural hearing loss who satisfied the UK's national candidacy criteria for cochlear implantation and their normal-hearing significant others (n = 2). Participants were CI candidates who used hearing aids (HAs) and were awaiting CI surgery or CI recipients who used a unilateral CI or a CI and contralateral HA (CI + HA). Data from a pilot focus group conducted with 2 CI recipients were included in the analysis. The data, verbatim transcripts of the focus group proceedings, were analyzed qualitatively using constructivist grounded theory (GT) methodology. RESULTS: A GT of listening effort in cochlear implantation was developed from participants' accounts. The participants provided rich, nuanced descriptions of the complex and multidimensional nature of their listening effort. Interpreting and integrating these descriptions through GT methodology, listening effort was described as the mental energy required to attend to and process the auditory signal, as well as the effort required to adapt to, and compensate for, a hearing loss. Analyses also suggested that listening effort for most participants was motivated by a need to maintain a sense of social connectedness (i.e., the subjective awareness of being in touch with one's social world). Before implantation, low social connectedness in the presence of high listening effort encouraged self-alienating behaviors and resulted in social isolation with adverse effects for participant's well-being and quality of life. A CI moderated but did not remove the requirement for listening effort. Listening effort, in combination with the improved auditory signal supplied by the CI, enabled most participants to listen and communicate more effectively. These participants reported a restored sense of social connectedness and an acceptance of the continued need for listening effort. CONCLUSIONS: Social connectedness, effort-reward balance, and listening effort as a multidimensional phenomenon were the core constructs identified as important to participants' experiences and understanding of listening effort. The study's findings suggest: (1) perceived listening effort is related to social and psychological factors and (2) these factors may influence how individuals with hearing loss report on the actual cognitive processing demands of listening. These findings provide evidence in support of the Framework for Understanding Effortful Listening a heuristic that describes listening effort as a function of both motivation and demands on cognitive capacity. This GT will inform item development and establish the content validity for a new PROM for measuring listening effort.

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.

Protocol for a mixed-methods study to develop and feasibility test a digital system for the capture of patient-reported outcomes (PROs) in patients receiving chimeric antigen receptor T-cell (CAR-T) therapies (the PRO-CAR-T study).

INTRODUCTION: Chimeric antigen receptor (CAR) T-cell therapies are novel, potentially curative therapies for haematological malignancies. CAR T-cell therapies are associated with severe toxicities, meaning patients require monitoring during acute and postacute treatment phases. Electronic patient-reported outcomes (ePROs), self-reports of health status provided via online questionnaires, can complement clinician observation with potential to improve patient outcomes. This study will develop and evaluate feasibility of a new ePRO system for CAR-T patients in routine care. METHODS AND ANALYSIS: Multiphase, mixed-methods study involving multiple stakeholder groups (patients, family members, carers, clinicians, academics/researchers and policy-makers). The intervention development phase comprises a Delphi study to select PRO measures for the digital system, a codesign workshop and consensus meetings to establish thresholds for notifications to the clinical team if a patient reports severe symptoms or side effects. Usability testing will evaluate how users interact with the digital system and, lastly, we will evaluate ePRO system feasibility with 30 CAR-T patients (adults aged 18+ years) when used in addition to usual care. Feasibility study participants will use the ePRO system to submit self-reports of symptoms, treatment tolerability and quality of life at specific time points. The CAR-T clinical team will respond to system notifications triggered by patients' submitted responses with actions in line with standard clinical practice. Feasibility measures will be collected at prespecified time points following CAR T-cell infusion. A qualitative substudy involving patients and clinical team members will explore acceptability of the ePRO system. ETHICS AND DISSEMINATION: Favourable ethical opinion was granted by the Health and Social Care Research Ethics Committee B(HSC REC B) (ref: 23/NI/0104) on 28 September 2023. Findings will be submitted for publication in high-quality, peer-reviewed journals. Summaries of results, codeveloped with the Blood and Transplant Research Unit Patient and Public Involvement and Engagement group, will be disseminated to all interested groups. TRIAL REGISTRATION NUMBER: ISCTRN11232653.

Recommendations to address respondent burden associated with patient-reported outcome assessment.

Patient-reported outcomes (PROs) are increasingly used in healthcare research to provide evidence of the benefits and risks of interventions from the patient perspective and to inform regulatory decisions and health policy. The use of PROs in clinical practice can facilitate symptom monitoring, tailor care to individual needs, aid clinical decision-making and inform value-based healthcare initiatives. Despite their benefits, there are concerns that the potential burden on respondents may reduce their willingness to complete PROs, with potential impact on the completeness and quality of the data for decision-making. We therefore conducted an initial literature review to generate a list of candidate recommendations aimed at reducing respondent burden. This was followed by a two-stage Delphi survey by an international multi-stakeholder group. A consensus meeting was held to finalize the recommendations. The final consensus statement includes 19 recommendations to address PRO respondent burden in healthcare research and clinical practice. If implemented, these recommendations may reduce PRO respondent burden.

Usability testing of an electronic patient-reported outcome system linked to an electronic chemotherapy prescribing and patient management system for patients with cancer.

Background: People affected by cancer experience a wide range of symptoms which have a major impact on their functioning and health-related quality of life (HRQoL). One way to measure the impact of cancer symptoms is through the use of patient-reported outcomes. Methods: An electronic patient-reported outcome (ePRO) application (ChemoPRO®) was designed to be used by cancer patients to report their symptoms and communicate with their clinical team. Usability testing sessions were conducted with people with lived experience of cancer to understand how real users interact with the ChemoPRO® system. One-to-one testing sessions were conducted to assess use of the system and identify areas for further refinement. User satisfaction was assessed using a brief satisfaction questionnaire previously used by Aiyegbusi et al. (date). Results: Ten people with lived experience of cancer took part in the usability study. Symptoms and HRQoL measures, including the Euroqol EQ5D5L and the PRO-CTCAE™ were included in the ePRO system. Participants: had a mean age of 62.3 years. Three critical errors and 21 non-critical errors were reported. All participants were enthusiastic about the app. Participants liked the simplicity and responsiveness of the patient-facing app and highlighted the potential for communicating with their clinical team. The overall usability and satisfaction score was 4.5 (sd = 0.09). Conclusion: This usability study suggests that people with lived experience of cancer found the ChemoPRO® app acceptable and easy to use. One of the key features of this particular ePRO system that should be developed further is system functionality to facilitate communication between patients and clinicians. Future testing should include testing in a clinical setting and testing with people from ethnic minorities.

Embedding patient-reported outcomes at the heart of artificial intelligence health-care technologies.

Integration of patient-reported outcome measures (PROMs) in artificial intelligence (AI) studies is a critical part of the humanisation of AI for health. It allows AI technologies to incorporate patients' own views of their symptoms and predict outcomes, reflecting a more holistic picture of health and wellbeing and ultimately helping patients and clinicians to make the best health-care decisions together. By positioning patient-reported outcomes (PROs) as a model input or output we propose a framework to embed PROMs within the function and evaluation of AI health care. However, the integration of PROs in AI systems presents several challenges. These challenges include (1) fragmentation of PRO data collection; (2) validation of AI systems trained and validated against clinician performance, rather than outcome data; (3) scarcity of large-scale PRO datasets; (4) inadequate selection of PROMs for the target population and inadequate infrastructure for collecting PROs; and (5) clinicians might not recognise the value of PROs and therefore not prioritise their adoption; and (6) studies involving PRO or AI frequently present suboptimal design. Notwithstanding these challenges, we propose considerations for the inclusion of PROs in AI health-care technologies to avoid promoting survival at the expense of wellbeing.

Reducing the pressures of outpatient care: the potential role of patient-reported outcomes.

The global demand for hospital treatment exceeds capacity.The COVID-19 pandemic has exacerbated this issue, leading to increased backlogs and longer wait times for patients. The amount of outpatient attendances undertaken in many settings is still below pre-pandemic levels and this, combined with delayed referrals, means that patients are facing delays in treatment and poorer health outcomes. Use of digital health technologies, notably the use of remote symptom monitoring systems based on patient-reported outcomes (PROs), may offer a solution to reduce outpatient waiting lists and tailor care to those in greatest need. Drawing on international examples, the authors explore the use of electronic PRO systems to triage clinical care. We summarise the key benefits of the approach and also highlight the challenges for implementation, which need to be addressed to promote equitable healthcare delivery.

Co-production of a feasibility trial of pacing interventions for Long COVID.

BACKGROUND: The high incidence of COVID-19 globally has led to a large prevalence of Long COVID but there is a lack of evidence-based treatments. There is a need to evaluate existing treatments for symptoms associated with Long COVID. However, there is first a need to evaluate the feasibility of undertaking randomised controlled trials of interventions for the condition. We aimed to co-produce a feasibility study of non-pharmacological interventions to support people with Long COVID. METHODS: A consensus workshop on research prioritisation was conducted with patients and other stakeholders. This was followed by the co-production of the feasibility trial with a group of patient partners, which included the design of the study, the selection of interventions, and the production of dissemination strategies. RESULTS: The consensus workshop was attended by 23 stakeholders, including six patients. The consensus from the workshop was to develop a clinical trial platform that focused on testing different pacing interventions and resources. For the co-production of the feasibility trial, patient partners selected three pacing resources to evaluate (video, mobile application, and book) and co-designed feasibility study processes, study materials and undertook usability testing of the digital trial platform. CONCLUSION: In conclusion, this paper reports the principles and process used to co-produce a feasibility study of pacing interventions for Long COVID. Co-production was effective and influenced important aspects of the study.

The World Health Organisation defines Long COVID as a condition which impacts people 3 months after they first had COVID-19. Some of the symptoms that characterise Long COVID symptoms include fatigue, breathlessness and brain fog. These symptoms have a major impact on people's health and quality of life. Today, over 2 million people in the United Kingdom suffer from Long COVID and there is a lack of drugs and non-drugs treatment. However, some non-drugs treatments which aim to manage fatigue in other conditions, such as pacing, could be used with people with Long COVID. In this paper, we report how we co-produced a study which tested whether or not it is feasible for people who have Long COVID to use a pacing resource and report their symptoms using an electronic platform. After a meeting to review existing non-drugs treatments, the research team and a group of patient partners agreed on co-developing a clinical trial platform to test different pacing resources. The research team then met with the patient partners twice a week to co-design the study during which people with Long COVID will use the pacing resources and report their symptoms. They also co-designed the study documents and how to report its results. Co-producing a study with patient partners was effective and influenced important aspects of the study.

Non-Pharmacological Therapies for Post-Viral Syndromes, Including Long COVID: A Systematic Review.

BACKGROUND: Post-viral syndromes (PVS), including Long COVID, are symptoms sustained from weeks to years following an acute viral infection. Non-pharmacological treatments for these symptoms are poorly understood. This review summarises the evidence for the effectiveness of non-pharmacological treatments for PVS. METHODS: We conducted a systematic review to evaluate the effectiveness of non-pharmacological interventions for PVS, as compared to either standard care, alternative non-pharmacological therapy, or placebo. The outcomes of interest were changes in symptoms, exercise capacity, quality of life (including mental health and wellbeing), and work capability. We searched five databases (Embase, MEDLINE, PsycINFO, CINAHL, MedRxiv) for randomised controlled trials (RCTs) published between 1 January 2001 to 29 October 2021. The relevant outcome data were extracted, the study quality was appraised using the Cochrane risk-of-bias tool, and the findings were synthesised narratively. FINDINGS: Overall, five studies of five different interventions (Pilates, music therapy, telerehabilitation, resistance exercise, neuromodulation) met the inclusion criteria. Aside from music-based intervention, all other selected interventions demonstrated some support in the management of PVS in some patients. INTERPRETATION: In this study, we observed a lack of robust evidence evaluating the non-pharmacological treatments for PVS, including Long COVID. Considering the prevalence of prolonged symptoms following acute viral infections, there is an urgent need for clinical trials evaluating the effectiveness and cost-effectiveness of non-pharmacological treatments for patients with PVS. REGISTRATION: The study protocol was registered with PROSPERO [CRD42021282074] in October 2021 and published in BMJ Open in 2022.

Considerations for patient and public involvement and engagement in health research.

Patient and public involvement and engagement (PPIE) can provide valuable insights into the experiences of those living with and affected by a disease or health condition. Inclusive collaboration between patients, the public and researchers can lead to productive relationships, ensuring that health research addresses patient needs. Guidelines are available to support effective PPIE; however, evaluation of the impact of PPIE strategies in health research is limited. In this Review, we evaluate the impact of PPIE in the 'Therapies for Long COVID in non-hospitalised individuals' (TLC) Study, using a combination of group discussions and interviews with patient partners and researchers. We identify areas of good practice and reflect on areas for improvement. Using these insights and the results of a survey, we synthesize two checklists of considerations for PPIE, and we propose that research teams use these checklists to optimize the impact of PPIE for both patients and researchers in future studies.

Development and usability testing of an electronic patient-reported outcome (ePRO) solution for patients with inflammatory diseases in an Advanced Therapy Medicinal Product (ATMP) basket trial.

BACKGROUND: Electronic patient-reported outcome (ePRO) systems are increasingly used in clinical trials to provide evidence of efficacy and tolerability of treatment from the patient perspective. The aim of this study is twofold: (1) to describe how we developed an electronic platform for patients to report their symptoms, and (2) to develop and undertake usability testing of an ePRO solution for use in a study of cell therapy seeking to provide early evidence of efficacy and tolerability of treatment and test the feasibility of the system for use in later phase studies. METHODS: An ePRO system was designed to be used in a single arm, multi-centre, phase II basket trial investigating the safety and activity of the use of ORBCEL-C™ in the treatment of patients with inflammatory conditions. ORBCEL-C™ is an enriched Mesenchymal Stromal Cells product isolated from human umbilical cord tissue using CD362+ cell selection. Usability testing sessions were conducted using cognitive interviews and the 'Think Aloud' method with patient advisory group members and Research Nurses to assess the usability of the system. RESULTS: Nine patient partners and seven research nurses took part in one usability testing session. Measures of fatigue and health-related quality of life, the PRO-CTCAE™ and FACT-GP5 global tolerability question were included in the ePRO system. Alert notifications to the clinical team were triggered by PRO-CTCAE™ and FACT-GP5 scores. Patient participants liked the simplicity and responsiveness of the patient-facing app. Two patients were unable to complete the testing session, due to technical issues. Research Nurses suggested minor modifications to improve functionality and the layout of the clinician dashboard and the training materials. CONCLUSION: By testing the effectiveness, efficiency, and satisfaction of our novel ePRO system (PROmicsR), we learnt that most people with an inflammatory condition found it easy to report their symptoms using an app on their own device. Their experiences using the PROmicsR ePRO system within a trial environment will be further explored in our upcoming feasibility testing. Research nurses were also positive and found the clinical dashboard easy-to-use. Using ePROs in early phase trials is important in order to provide evidence of therapeutic responses and tolerability, increase the evidence based, and inform methodology development. TRIAL REGISTRATION: ISRCTN, ISRCTN80103507. Registered 01 April 2022, https://www.isrctn.com/ISRCTN80103507.

More and more patients tell clinicians how they feel by completing questionnaires electronically. Therefore, it is important to assess how easy it is for patients to do this. In this study, we describe how we developed an electronic platform for patients to report their symptoms and how we tested the usability of this platform with patient partners and research nurses. Once the electronic platform was developed, quality of life and symptoms questionnaires were programmed onto it. Alerts were sent to the clinical team if specific scores were obtained on the symptoms questionnaires. Although two patient partners were not able to finish the testing session because of technical issues, the ones who completed the session liked its simplicity and responsiveness. The research nurses also liked the system and only suggested minor modifications. Following this testing, we refined the electronic platform to test it further in a larger study which investigates the safety and use of a drug. We hope that thanks to this electronic platform, we will obtain useful information on the safety and efficacy of treatment.

Protocol for a living evidence synthesis on variants of concern and COVID-19 vaccine effectiveness.

BACKGROUND: It is evident that COVID-19 will remain a public health concern in the coming years, largely driven by variants of concern (VOC). It is critical to continuously monitor vaccine effectiveness as new variants emerge and new vaccines and/or boosters are developed. Systematic surveillance of the scientific evidence base is necessary to inform public health action and identify key uncertainties. Evidence syntheses may also be used to populate models to fill in research gaps and help to prepare for future public health crises. This protocol outlines the rationale and methods for a living evidence synthesis of the effectiveness of COVID-19 vaccines in reducing the morbidity and mortality associated with, and transmission of, VOC of SARS-CoV-2. METHODS: Living evidence syntheses of vaccine effectiveness will be carried out over one year for (1) a range of potential outcomes in the index individual associated with VOC (pathogenesis); and (2) transmission of VOC. The literature search will be conducted up to May 2023. Observational and database-linkage primary studies will be included, as well as RCTs. Information sources include electronic databases (MEDLINE; Embase; Cochrane, L*OVE; the CNKI and Wangfang platforms), pre-print servers (medRxiv, BiorXiv), and online repositories of grey literature. Title and abstract and full-text screening will be performed by two reviewers using a liberal accelerated method. Data extraction and risk of bias assessment will be completed by one reviewer with verification of the assessment by a second reviewer. Results from included studies will be pooled via random effects meta-analysis when appropriate, or otherwise summarized narratively. DISCUSSION: Evidence generated from our living evidence synthesis will be used to inform policy making, modelling, and prioritization of future research on the effectiveness of COVID-19 vaccines against VOC.

Saving time and effort: Best practice for adapting existing patient-reported outcome measures in hepatology.

It is increasingly recognised that collecting patient reported outcome measures (PROMs) data is an important part of healthcare and should be considered alongside traditional clinical assessments. As part of a more holistic view of healthcare provision, there has been an increased drive to implement PROM collection as part of routine clinical care in hepatology. This drive has resulted in an increase in the number of PROMs currently developed to be used in various liver conditions. However, the development and validation of a new PROM is time-consuming and costly. Therefore, before deciding to develop a new PROM, researchers should consider identifying existing PROMs to assess their appropriateness and, if necessary, make adaptations to existing PROMs to ensure their rigour when used with the target population. Little is written in the literature on how to identify and adapt the existing PROMs in hepatology. This article aims to provide a summary of the current literature and guidance regarding identifying and adapting existing PROMs in clinical practice.

Adults' with hearing loss perceived listening ability in daily communication: protocol for a systematic review and qualitative meta-synthesis.

INTRODUCTION: Understanding how adults with hearing loss perceive their abilities when listening in daily communication situations is vital to understanding the functional listening challenges associated with hearing loss. The aim of this study is to explore how adults with hearing loss describe their own experiences of the processes, behaviours and components of listening in real-world communication through secondary analysis of published qualitative data. METHODS AND ANALYSIS: A systematic review and thematic meta-synthesis of qualitative research studies and qualitative components of mixed-methods studies will be conducted. Studies published in English will be identified through searching Medline, PsychInfo, Web of Science, Embase and Google Scholar databases from inception to November 2021. Handsearching of the included studies' reference lists will be completed. Included articles will be assessed for methodological quality using the Critical Appraisal Skills checklist for qualitative studies. Thematic synthesis will proceed as follows: (1) line-by-line coding to label concepts present in the 'results' or 'findings' section(s) of the included studies; (2) grouping of similar codes into descriptive themes; (3) development of higher level analytic themes to develop a new interpretation of the included studies' findings. The Grading of Recommendations Assessment, Development and Evaluation (GRADE) Confidence in the Evidence from Reviews of QUALitative (CerQUAL) research approach will be used to establish the degree of confidence that may be placed in synthesis findings and results will be reported alongside the synthesis. Two reviewers will independently undertake screening for eligibility, data extraction and quality appraisal, analysis and GRADE-CERQual assessments. Discrepancies will be resolved through discussion. ETHICS AND DISSEMINATION: As secondary data analysis of the published literature, ethical approval is not required. The results will be disseminated in peer-reviewed journals, conference presentations and other research and clinical meetings. This protocol is registered with PROSPERO prospective database of systematic review. PROSPERO REGISTRATION NUMBER: CRD42020213389.