Introduction
Recent advances in information technology and improved access to the internet have led to a rapid increase in the adoption and ownership of electronic devices such as touch screen ...smartphones and tablet computers. This has also led to a renewed interest in the field of digital health also referred to as telehealth or electronic health (eHealth). There is now a drive to collect these PROs electronically using ePRO systems.
Method
However, the user interfaces of ePRO systems need to be adequately assessed to ensure they are not only fit for purpose but also acceptable to patients who are the end users. Usability testing is a technique that involves the testing of systems, products or websites with participants drawn from the target population. Usability testing can assist ePRO developers in the evaluation of ePRO user interface. The complexity of ePRO systems; stage of development; metrics to measure; and the use of scenarios, moderators and appropriate sample sizes are key methodological issues to consider when planning usability tests.
Conclusion
The findings from usability testing may facilitate the improvement of ePRO systems making them more usable and acceptable to end users. This may in turn improve the adoption of ePRO systems post-implementation. This article highlights the key methodological issues to consider and address when planning usability testing of ePRO systems.
Cell and gene therapies offer opportunities for treating disease with potential to restore function, and cure disease. However, they are not without risk and pose complex logistical, economic, ...ethical and social challenges for health systems. Here we report our systematic review of the current evidence on patient and public knowledge and perspectives of cell and gene therapies, to inform future research, education and awareness raising activities. We screened 10,735 titles and abstracts, and evaluated the full texts of 151 publications. The final selection was 35 publications. Four themes were generated from the narrative synthesis of the study findings namely: (1) Knowledge and understanding of cell and gene therapies, (2) Acceptance of cell and gene therapies (3) Understanding of risk and benefits of therapy, and (4) Information needs and current sources of information. As potential funders or future recipients, it is important that the public and patients are aware of these therapies, understand the issues involved, and can contribute to the debate. This review highlights the need for appropriate patient and public education on the various aspects of cell and gene therapies. High quality studies exploring patient and public opinions and experiences of cell and gene therapy are required. Patient and public perceptions of these therapies, alongside evidence of clinical and cost-effectiveness, will be central to their uptake and use.
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.
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 < 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.
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.
PROSPERO CRD42020164737.
Wong and colleagues’ findings highlight the fact that rare kidney disease patients, although a smaller portion of the CKD population (5–10%), constitute a substantial fraction (at least 25%) of those ...receiving kidney replacement therapy due to a lower mortality rate, higher risk of kidney failure, and better survival on dialysis. ...addressing the unmet need for effective therapies in rare kidney diseases could substantially reduce the long-term demand for kidney replacement therapy and the associated economic impact on health-care systems. Peakstock/Science Photo Library Furthermore, the study highlights the importance of distinguishing patients with rare kidney diseases from those with more common causes of CKD, to facilitate early specialist referral and treatment aimed at delaying the progression to kidney failure as recommended by the National Institute for Health and Care Excellence guideline for CKD.6 Early diagnosis of rare kidney diseases requires increased education and awareness among health-care providers and more extensive use of genetic or advanced diagnostic testing, particularly in patients with unexplained CKD. Clinicians might find the insights from the RaDaR data on the natural history of individual rare diseases valuable for discussions about care and trial participation with patients and their caregivers.7 However, it is essential to recognise that the findings represent the prognosis in the context of the standard of care at the time of the study. ...the prognosis will probably improve with the wider implementation of supportive kidney therapies, such as SGLT2 inhibitors,8 and the development of novel treatments such as cell and gene therapies for specific rare kidney diseases.9 The application of big data analytics—including machine-learning algorithms for the analyses of data collected by registries such as RaDaR and the European Rare Kidney Disease Registry—could provide valuable insights into rare kidney diseases and facilitate personalised medicine in future.10 OLA declares consulting fees from Innovate UK, Merck, and GlaxoSmithKline outside the submitted work and receives funding from the National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, NIHR Applied Research Collaboration West Midlands, NIHR Blood and Transplant Research Unit in Precision Transplant and Cellular Therapeutics at the University of Birmingham, University Hospitals Birmingham NHS Foundation, Innovate UK (part of UK Research and Innovation), Gilead Sciences, Anthony Nolan, and Sarcoma UK.
Globally, there are now over 160 million confirmed cases of COVID-19 and more than 3 million deaths. While the majority of infected individuals recover, a significant proportion continue to ...experience symptoms and complications after their acute illness. Patients with ‘long COVID’ experience a wide range of physical and mental/psychological symptoms. Pooled prevalence data showed the 10 most prevalent reported symptoms were fatigue, shortness of breath, muscle pain, joint pain, headache, cough, chest pain, altered smell, altered taste and diarrhoea. Other common symptoms were cognitive impairment, memory loss, anxiety and sleep disorders. Beyond symptoms and complications, people with long COVID often reported impaired quality of life, mental health and employment issues. These individuals may require multidisciplinary care involving the long-term monitoring of symptoms, to identify potential complications, physical rehabilitation, mental health and social services support. Resilient healthcare systems are needed to ensure efficient and effective responses to future health challenges.
Abstract
Background
Patient-reported adverse events may be a useful adjunct for assessing a drug’s tolerability in dose-finding oncology trials (DFOT). We conducted surveys of international ...stakeholders and the National Cancer Research Institute (NCRI) Consumer Forum to understand attitudes about patient-reported outcome (PRO) use in DFOT.
Methods
A 35-question survey of clinicians, trial managers, statisticians, funders, and regulators of DFOT was distributed via professional bodies examining experience using PROs, benefits/barriers, and their potential role in defining tolerable doses. An 8-question survey of the NCRI Consumer Forum explored similar themes.
Results
International survey: 112 responses from 15 September–30 November 2020; 103 trialists 48 clinicians (42.9%), 38 statisticians (34.0%), 17 trial managers (15.2%), 7 regulators (6.3%), 2 funders (1.8%). Most trialists had no experience designing (73, 70.9%), conducting (52, 50.5%), or reporting (88, 85.4%) PROs in DFOT. Most agreed that PROs could identify new toxicities (75, 67.0%) and provide data on the frequency (86, 76.8%) and duration (81, 72.3%) of toxicities. The top 3 barriers were lack of guidance regarding PRO selection (73/103, 70.9%), missing PRO data (71/103, 68.9%), and overburdening staff (68/103, 66.0%). NCRI survey: 57 responses on 21 March 2021. A total of 28 (49.1%) were willing to spend <15 min/day completing PROs. Most (55, 96.5%) preferred to complete PROs online. 61 (54.5%) trialists and 57 (100%) consumers agreed that patient-reported adverse events should be used to inform dose-escalation decisions.
Conclusion
Stakeholders reported minimal experience using PROs in DFOT but broadly supported their use. Guidelines are needed to standardize PRO selection, analysis, and reporting in DFOT.
Severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) infection is associated with a range of persistent symptoms impacting everyday functioning, known as post-COVID-19 condition or long ...COVID. We undertook a retrospective matched cohort study using a UK-based primary care database, Clinical Practice Research Datalink Aurum, to determine symptoms that are associated with confirmed SARS-CoV-2 infection beyond 12 weeks in non-hospitalized adults and the risk factors associated with developing persistent symptoms. We selected 486,149 adults with confirmed SARS-CoV-2 infection and 1,944,580 propensity score-matched adults with no recorded evidence of SARS-CoV-2 infection. Outcomes included 115 individual symptoms, as well as long COVID, defined as a composite outcome of 33 symptoms by the World Health Organization clinical case definition. Cox proportional hazards models were used to estimate adjusted hazard ratios (aHRs) for the outcomes. A total of 62 symptoms were significantly associated with SARS-CoV-2 infection after 12 weeks. The largest aHRs were for anosmia (aHR 6.49, 95% CI 5.02-8.39), hair loss (3.99, 3.63-4.39), sneezing (2.77, 1.40-5.50), ejaculation difficulty (2.63, 1.61-4.28) and reduced libido (2.36, 1.61-3.47). Among the cohort of patients infected with SARS-CoV-2, risk factors for long COVID included female sex, belonging to an ethnic minority, socioeconomic deprivation, smoking, obesity and a wide range of comorbidities. The risk of developing long COVID was also found to be increased along a gradient of decreasing age. SARS-CoV-2 infection is associated with a plethora of symptoms that are associated with a range of sociodemographic and clinical risk factors.
An application of telemedicine of growing interest and relevance is the use of personal computers and mobile devices to collect patient-reported outcomes (PROs). PROs are self-reports of patients’ ...health status without interpretation by anyone else. The tools developed to assess PROs are known as patient-reported outcomes measures (PROMs). The technological innovations that have led to an increased ownership of electronic devices have also facilitated the development of electronic PROMs (ePROMs). ePROMs are a conduit for telemedicine in the care of patients with chronic diseases. Various studies have demonstrated that the use of ePROMs in routine clinical practice is both acceptable and feasible with patients increasingly expressing a preference for an electronic mode of administration. There is increasing evidence that the use of electronic patient-reported outcome (ePROMs) could have significant impacts on outcomes valued by patients, healthcare providers and researchers. Whilst the development and implementation of these systems may be initially costly and resource-intensive, patient preferences and existing evidence to support their implementation suggests the need for continued research prioritisation in this area. This narrative review summarises and discusses evidence of the impact of ePROMs on clinical parameters and outcomes relevant to chronic diseases. We also explore recently published literature regarding issues that may influence the robust implementation of ePROMs for routine clinical practice.
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