Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom

Background: Late-onset Pompe disease (LOPD) is a rare, progressive neuromuscular condition typically characterized by weakness of skeletal muscles, including those involved in respiration and diaphragmatic dysfunction. Individuals with LOPD typically eventually require mobility and/or ventilatory support. / Objectives: This study aimed to develop health state vignettes and estimate health state utility values for LOPD in the United Kingdom. / Methods: Vignettes were developed for 7 health states of LOPD with states defined in terms of mobility and/or ventilatory support. Vignettes were drafted based on patient-reported outcome data from the Phase 3 PROPEL trial (NCT03729362) and supplemented by a literature review. Qualitative interviews with individuals living with LOPD and clinical experts were conducted to explore the health-related quality-of-life (HRQoL) impact of LOPD and to review the draft vignettes. Vignettes were finalized following a second round of interviews with individuals living with LOPD and used in health state valuation exercises with people of the UK population. Participants rated the health states using the EQ-5D-5L, visual analog scale, and time trade-off interviews. / Results: Twelve individuals living with LOPD and 2 clinical experts were interviewed. Following the interviews, 4 new statements were added regarding dependence on others, bladder control problems, balance issues/fear of falling, and frustration. One hundred interviews with a representative UK population sample were completed. Mean time trade-off utilities ranged from 0.754 (SD = 0.31) (no support) to 0.132 (SD = 0.50) (invasive ventilatory and mobility support-dependent). Similarly, EQ- 5D-5L utilities ranged from 0.608 (SD = 0.12) to -0.078 (SD = 0.22). / Discussion: The utilities obtained in the study are consistent with utilities reported in the literature (0.670-0.853 for nonsupport state). The vignette content was based on robust quantitative and qualitative evidence and captured the main HRQoL impacts of LOPD. The general public rated the health states consistently lower with increasing disease progression. There was greater uncertainty around utility estimates for the severe states, suggesting that participants found it harder to rate them. / Conclusion: This study provides utility estimates for LOPD that can be used in economic modeling of treatments for LOPD. Our findings highlight the high disease burden of LOPD and reinforce the societal value of slowing disease progression

Speech Communication

Contains research objectives and three research projects.U. S. Air Force (Electronic Systems Division) under Contract AF 19(604)-6102National Science Foundation (Grant G-16526)National Institutes of Health (Grant MH-04737-02

Adult beginner distance language learner perceptions and use of assignment feedback

This qualitative study examines perceptions and use of assignment feedback among adult beginner modern foreign language learners on higher education distance learning courses. A survey of responses to feedback on assignments by 43 Open University students on beginner language courses in Spanish, French, and German indicated that respondents can be classified into three groups: those who use feedback strategically by integrating it into the learning process and comparing it with, for example, informal feedback from interaction with native speakers, those who take note of feedback, but seem not to use it strategically, and those who appear to take little account of either marks or feedback. The first group proved to be the most confident and most likely to maintain their motivation in the longer term. The conclusion discusses some of the pedagogical and policy implications of the findings

Additive Beneficial Effects of Beta-Blockers to Angiotensin-Converting Enzyme Inhibitors in the Survival and Ventricular Enlargement (SAVE) Study fn1fn1This study was supported by a University-Industry grant from the Medical Research Council, Ottawa, Ontario, Canada and Bristol Myers Squibb, Montreal, Quebec, Canada.

AbstractObjectives. This study assessed whether treatment with a beta-adrenergic blocking agent in addition to the use of the angiotensin-converting enzyme (ACE) inhibitor captopril decreases cardiovascular mortality and morbidity in patients with asymptomatic left ventricular dysfunction after myocardial infarction (MI) and whether the presence of neurohumoral activation at the time of hospital discharge predicts the effects of beta-blocker treatment in these patients.Background. Both beta-blockers and ACE inhibitors have been shown to have beneficial effects in patients with left ventricular dysfunction but no overt heart failure after MI. These patients often have persistent neurohumoral activation at the time of hospital discharge, and one would expect that patients with activation of the sympathetic nervous system derive the most benefit from treatment with beta-blockers. However, beta-blockers are underutilized in this high risk group of patients, and it is unknown whether their beneficial effects are additive to those of ACE inhibitors.Methods. We performed a retrospective analysis of data from the Survival and Ventricular Enlargement (SAVE) study and its neurohumoral substudy. The relations between beta-blocker use at the time of randomization and neurohumoral activation and the subsequent development of cardiovascular events were analyzed by use of Cox proportional hazards models controlling for covariates.Results. After adjustment for baseline imbalances, beta-blocker use was associated with a significant reduction in risk of cardiovascular death (30%, 95% confidence interval [CI] 12% to 44%) and development of heart failure (21%, 95% CI 3% to 36%), but the reduction in recurrent MI (11%, 95% CI 13% to 31%) was not significant. These reductions were independent of the use of captopril. Beta-blockers were not found to have a greater effect in patients with neurohumoral activation at the time of hospital discharge.Conclusions. The beneficial effects of beta-blocker use at the time of hospital discharge in patients with asymptomatic left ventricular dysfunction after MI appear to be additive to those of captopril and other interventions known to improve prognosis. Neurohumoral activation at the time of hospital discharge fails to identify those patients who will derive the greatest benefit from treatment with beta-blockers.(J Am Coll Cardiol 1997;29:229–36

Incidence of hyperoxia in trauma patients receiving pre-hospital emergency anaesthesia:results of a 5-year retrospective analysis

Abstract Background Previous studies have demonstrated an association between hyperoxia and increased mortality in various patient groups. Critically unwell and injured patients are routinely given high concentration oxygen in the pre-hospital phase of care. We aim to investigate the incidence of hyperoxia in major trauma patients receiving pre-hospital emergency anesthesia (PHEA) in the pre-hospital setting and determine factors that may help guide clinicians with pre-hospital oxygen administration in these patients. Methods A retrospective cohort study was performed of all patients who received PHEA by a single helicopter emergency medical service (HEMS) between 1 October 2014 and 1 May 2019 and who were subsequently transferred to one major trauma centre (MTC). Patient and treatment factors were collected from the electronic patient records of the HEMS service and the MTC. Hyperoxia was defined as a PaO2 > 16 kPA on the first arterial blood gas analysis upon arrival in the MTC. Results On arrival in the MTC, the majority of the patients (90/147, 61.2%) had severe hyperoxia, whereas 30 patients (20.4%) had mild hyperoxia and 26 patients (19.7%) had normoxia. Only 1 patient (0.7%) had hypoxia. The median PaO2 on the first arterial blood gas analysis (ABGA) after HEMS handover was 36.7 [IQR 18.5–52.2] kPa, with a range of 7.0–86.0 kPa. SpO2 pulse oximetry readings before handover were independently associated with the presence of hyperoxia. An SpO2 ≥ 97% was associated with a significantly increased odds of hyperoxia (OR 3.99 [1.58–10.08]), and had a sensitivity of 86.7% [79.1–92.4], a specificity of 37.9% [20.7–57.8], a positive predictive value of 84.5% [70.2–87.9] and a negative predictive value of 42.3% [27.4–58.7] for the presence of hyperoxemia. Conclusion Trauma patients who have undergone PHEA often have profound hyperoxemia upon arrival at hospital. In the pre-hospital setting, where arterial blood gas analysis is not readily available a titrated approach to oxygen therapy should be considered to reduce the incidence of potentially harmful tissue hyperoxia

Identifying where hospital and community trusts are managing general practices in England: a service mapping study

Background Organisations providing secondary care in the NHS in England have historically not also provided primary health care, but this is changing. Data on where this kind of ‘vertical integration’ is happening is lacking, making it difficult to evaluate its impact. Aim To compile a comprehensive list of instances of secondary care trusts running general practices in England, to enable evaluation of the impact of such arrangements. Design and setting Review, collation, synthesis and analysis of published information describing secondary and primary care provision in the NHS in England in March 2021. Method Desk-based collection, including hand-searching, of secondary care organisations’ statutory annual reports. Triangulation via comparison with national data on general practices, the general practice workforce and practice contracts. Results It was possible to construct a database of all instances of trusts running general practices in England as at end-March 2021. We have identified 26 trusts running a total of 85 general practices, operating across a total of 116 practice sites. These practices have on average fewer patients and fewer GP full-time equivalents than other general practices, and before becoming vertically integrated were performing less well in the Quality and Outcomes Framework. Conclusion We recommend that national statistics recording the details of general practices contracting with the NHS should include whether each practice is owned by another organisation and, whether that is an NHS trust, another public body or a private organisation. Such data are required to enable evaluation of the impacts of this kind of vertical integration

Undertaking rapid evaluations during the COVID-19 pandemic: Lessons from evaluating COVID-19 remote home monitoring services in England

Introduction: Rapid evaluations can offer evidence on innovations in health and social care that can be used to inform fast-moving policy and practise, and support their scale-up according to previous research. However, there are few comprehensive accounts of how to plan and conduct large-scale rapid evaluations, ensure scientific rigour, and achieve stakeholder engagement within compressed timeframes. / Methods: Using a case study of a national mixed-methods rapid evaluation of COVID-19 remote home monitoring services in England, conducted during the COVID-19 pandemic, this manuscript examines the process of conducting a large-scale rapid evaluation from design to dissemination and impact, and reflects on the key lessons for conducting future large-scale rapid evaluations. In this manuscript, we describe each stage of the rapid evaluation: convening the team (study team and external collaborators), design and planning (scoping, designing protocols, study set up), data collection and analysis, and dissemination. / Results: We reflect on why certain decisions were made and highlight facilitators and challenges. The manuscript concludes with 12 key lessons for conducting large-scale mixed-methods rapid evaluations of healthcare services. We propose that rapid study teams need to: (1) find ways of quickly building trust with external stakeholders, including evidence-users; (2) consider the needs of the rapid evaluation and resources needed; (3) use scoping to ensure the study is highly focused; (4) carefully consider what cannot be completed within a designated timeframe; (5) use structured processes to ensure consistency and rigour; (6) be flexible and responsive to changing needs and circumstances; (7) consider the risks associated with new data collection approaches of quantitative data (and their usability); (8) consider whether it is possible to use aggregated quantitative data, and what that would mean when presenting results, (9) consider using structured processes & layered analysis approaches to rapidly synthesise qualitative findings, (10) consider the balance between speed and the size and skills of the team, (11) ensure all team members know roles and responsibilities and can communicate quickly and clearly; and (12) consider how best to share findings, in discussion with evidence-users, for rapid understanding and use. / Conclusion: These 12 lessons can be used to inform the development and conduct of future rapid evaluations in a range of contexts and settings

Towards a better understanding of the dynamic role of the distance language learner: learner perceptions of personality, motivation, roles, and approaches

This study investigated the experience of learners enrolled on an Open University (UK) French course, and included personality factors, motivation, and tutor and student roles. The data gathered via multiple elicitation methods gave useful insights into issues of special relevance to distance language education, in particular the lack of fit between an inherently social discipline such as language learning and the distance context, whose main characterizing feature is remoteness from others. Motivation was seen to play a crucial role in success, along with tutor feedback, and personal responsibility for learning. Increased confidence and self?regulation were beneficial outcomes of the process of learning at a distance, and numerous suggestions for learning approaches based on personal experience were offered for language learners new to distance learning. The study concluded that the task for distance practitioners is to build on the insights shown by learners themselves, in order to target support where it is most needed

The Policy and Practice of Music Education in England, 2010-2020

Since the introduction of the National Plan for Music Education there have been significant changes in music education within England. Whilst some celebrate figures that report increased access and engagement, many teachers and others continue to have legitimate concerns regarding the quality of the music education on offer in schools and Music Education Hubs. There are concerns that the provision of music education is incoherent and patchy across the country. Many would argue that the opportunity to access high‐quality music education has become a ‘postcode lottery’. There is a sense that the fragmentation of music education as a result of curriculum reforms and the diversity of approaches taken by Music Education Hubs and other bodies has significantly enhanced this incoherence. This article seeks to review the policy and practice of music education in England over the last 10 years. It draws on recent research from various sources and maintains a particular focus on government policy and the consequences of this for the field as a whole. It reflects on how things could be improved in the future. It argues for a clearer focus on a practitioner‐led approach to research and advocacy, in particular one led by the notion of ‘policy as practice’ rather than continuing with the current approach and its intrinsic failings

Learning from the UK’s research impact assessment exercise: a case study of a retrospective impact assessment exercise and questions for the future

National governments spend significant amounts of money supporting public research. However, in an era where the international economic climate has led to budget cuts, policymakers increasingly are looking to justify the returns from public investments, including in science and innovation. The so-called ‘impact agenda’ which has emerged in many countries around the world is part of this response; an attempt to understand and articulate for the public what benefits arise from the research that is funded. The United Kingdom is the most progressed in implementing this agenda and in 2014 the national research assessment exercise, the Research Excellence Framework, for the first time included the assessment of research impact as a component. For the first time within a dual funding system, funding would be awarded not only on the basis of the academic quality of research, but also on the wider impacts of that research. In this paper we outline the context and approach taken by the UK government, along with some of the core challenges that exist in implementing such an exercise. We then synthesise, together for the first time, the results of the only two national evaluations of the exercise and offer reflections for future exercises both in the UK and internationally