Health technology assessment最新文献

{"title":"Interventions that challenge established and accepted clinical practice: lessons learnt from a process evaluation of the STOP-APE trial.","authors":"Agnieszka Ignatowicz, Sheila Greenfield, Pooja Gaddu, Clare Prince, Mark Toshner, Graham Robinson, Jonathan Rodrigues, Susan Jowett, Simon Noble, Michael Newnham, Alice Turner, Daniel Lasserson","doi":"10.3310/PSDG7298","DOIUrl":"https://doi.org/10.3310/PSDG7298","url":null,"abstract":"<p><strong>Background: </strong>Developing and implementing interventions that change clinical practice can be challenging and complex. Such interventions can be particularly difficult when attempting to change established behaviours and practices. While extensive literature on implementation of interventions that focus on changing clinical practice exists, understanding of the difficulties involved in implementing interventions that go against accepted clinical practice is limited.</p><p><strong>Objectives: </strong>To describe the challenges involved in delivering a complex intervention that goes against established clinical practice, using a clinical trial assessing the balance of benefits and risks of withholding anticoagulation for subsegmental pulmonary embolism as an example.</p><p><strong>Design and methods: </strong>This study draws from a process evaluation conducted as part of a clinical trial. The evaluation utilised semistructured interviews with patients and clinicians during the trial's internal pilot phase to investigate the acceptability of withholding anticoagulant medication and participants' experiences within the trial. The data were analysed using the framework method.</p><p><strong>Setting and participants: </strong>Eight patients with subsegmental pulmonary embolism (six females and two males) and three acute care clinicians (two males and one female) from five trial sites were interviewed.</p><p><strong>Results: </strong>Our findings indicated that factors such as clinician equipoise, discomfort with certain patient characteristics, and effective patient communication are closely connected and significantly impact both the process of changing clinical practice and the conduct of the trial. Clinicians faced difficulties in approaching eligible patients for trial participation, especially when a diagnosis and treatment plan had already been provided by another clinician. The tension between maintaining clinical equipoise and addressing the needs of unwell patients further complicated decisions, particularly when withholding anticoagulation in those with severe symptoms or multiple comorbidities. Communication about the risks and benefits of non-medication strategies for pulmonary embolism was also challenging, with concerns about undermining patient trust. Patients, on the other hand, expressed considerable anxiety about not receiving anticoagulants, with their perspectives on study participation and treatment heavily influenced by their prior health experiences and ongoing medical conditions. The active involvement of clinicians in the consent process had a positive effect on patients' perceptions and experiences, with many feeling reassured in knowing they could contact clinical staff if needed.</p><p><strong>Limitations: </strong>Small sample size of patients and clinicians across limited study sites; single method of data collection.</p><p><strong>Conclusions: </strong>Our results highlight the multifaceted challenges w","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-11"},"PeriodicalIF":3.5,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143515439","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Learnings from the establishment and delivery of the UK Collaborative Paediatric Palliative Care Research Network.","authors":"George Peat, Emily Harrop, Anna-Karenia Anderson, Debbie Box, Fliss Murtagh, Richard Harding, Lucy Ziegler, Catherine Hewitt, Ian Wong, Joanna Elverson, Nicola Harris, Lorna K Fraser","doi":"10.3310/VRFT5679","DOIUrl":"https://doi.org/10.3310/VRFT5679","url":null,"abstract":"<p><strong>Background: </strong>There are increasing numbers of children living with conditions that may threaten or shorten their lives. While child mortality has decreased in recent decades, it is estimated 4500 infants, children and young people (0-19 years) die in the United Kingdom every year. Despite a growing increase in clinical provision, there is a clear paucity in research evidence underpinning paediatric palliative care. To support research delivery, a United Kingdom-wide network composed of paediatric palliative care-sector organisations and academics with expertise in the area was developed. The network had a clear vision of establishing partnerships between academia and services delivering paediatric palliative care that would support increased research capacity and delivery in the sector.</p><p><strong>Objective(s): </strong>The overarching aim of the network was to deliver national high-quality research studies, education and materials, and build research capacity. Specific objectives included working closely with seven paediatric palliative care sites to develop guidance on the appropriate methods for undertaking research, the delivery of information and educational resources including a webinar series, offering of mentor opportunities, and the development of a minimum of two bids to National Institute for Health and Care Research related funding pathways.</p><p><strong>Design: </strong>A collaborative design underpinned the network. Network activity included continued partner engagement through online meetings and newsletters, scoping activity to identify research priorities, establishment of research themes, and active engagement and support from national organisations. Patient and public involvement was core to all network activity.</p><p><strong>Setting: </strong>Network engagement largely took place online. The network has a web page hosted on the website of the organisation Together for Short Lives.</p><p><strong>Partners: </strong>Seven paediatric palliative care sites in England and Scotland (six hospices and one National Health Service hospital site) formed the network with input from several academic partners.</p><p><strong>Results: </strong>The network achieved several outputs related to objectives including the submission of two National Institute for Health and Care Research applications for funding, the delivery of five educational webinars, the establishment of an online research toolkit and the development of a research nurse group.</p><p><strong>Conclusions: </strong>Prior to the establishment of the network, there was no national mechanism for co-ordination for research in paediatric palliative care. In bringing together seven partner organisations and clinical and research expertise, the network has supported the foundations upon which to deliver high-quality research in the sector.</p><p><strong>Future work: </strong>Future work is required to support the sustainability of the network including obtaining nece","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-9"},"PeriodicalIF":3.5,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143523191","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Establishing palliative care research partnerships in Northern Ireland.","authors":"Julie McMullan, Clare McVeigh, Peter O'Halloran","doi":"10.3310/QUTP1946","DOIUrl":"https://doi.org/10.3310/QUTP1946","url":null,"abstract":"<p><strong>Background: </strong>The National Institute for Health and Care Research call for research partnerships was designed to build research capacity in palliative and end-of-life care and to ensure that the research of the National Institute for Health and Care Research is conducted in areas of greatest need and where there are historically low levels of research. Northern Ireland has high levels of need, relatively underdeveloped services, and comparatively low levels of research.</p><p><strong>Aims: </strong>To build palliative care and end-of-life research capacity in Northern Ireland, with a specific focus on less experienced sites, so that strong applications could be submitted to Part 2 of the National Institute for Health and Care Research Commissioned Call: building the evidence base. To create a sustained collaboration to support a programme of research focused on key areas of need in Northern Ireland that are also relevant to the rest of the United Kingdom.</p><p><strong>Methods: </strong>The Partners were: Queen's, Ulster and Open Universities; All Ireland Institute of Hospice and Palliative Care; Marie Curie Hospice Care; Patient and Client Council; Kidney Care UK; the three Health and Social Care Trusts covering areas with greatest need; Northern Ireland Clinical Trials Unit; Palliative Care Research Forum Northern Ireland; Public Health Agency; Department of Health; Health and Social Care Board. The Partnership was co-led by Drs Peter O'Halloran and Clare McVeigh, senior lecturers at Queen's University Belfast. A post-doctoral research assistant was employed 3 days a week in a support role. The Partners agreed the terms of reference for the Partnership and met six times over the following year. An expression of interest form was distributed to potential investigators, producing 13 responses. The Partnership then offered networking opportunities for investigators with specific partners, facilitated by the research assistant. The Partnership hosted a palliative care research conference on '<i>Cross-sector Partnerships for Palliative and End-of-life Care Research</i>'. This included presentations from the National Institute for Health and Care Research representatives on grant proposal preparation. A website and newsletter were published.</p><p><strong>Results: </strong>Nine introductory meetings took place, mostly with early career researchers. Topics included symptom management, accessing palliative care for vulnerable groups, perinatal bereavement care and advanced care planning. Draft proposals were reviewed by the Partnership and one was prepared for submission to Part 2 of the National Institute for Health and Care Research Commissioned Call: an evaluation of an intervention to improve the readiness of people with end-stage kidney disease, healthcare professionals, and surrogate decision-makers to engage with advance care planning.</p><p><strong>Conclusions: </strong>The Partnership took advantage of widespread interest and","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-11"},"PeriodicalIF":3.5,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143531327","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Stepping into day treatment approach versus inpatient treatment for adults with anorexia nervosa: the DAISIES RCT.","authors":"Başak İnce, Matthew D Phillips, Bethan Dalton, Madeleine Irish, Hannah Webb, Daniela Mercado, Catherine McCombie, Zohra Zenasni, James Shearer, Laura Potts, Gemma Peachey, Katie Au, Nikola Kern, Sam Clark-Stone, Frances Connan, A Louise Johnston, Stanimira Lazarova, Ewa Zadeh, Sophie Tomlin, Francesca Battisti, Konstantinos Ioannidis, Ciarán Newell, Tayeem Pathan, Jackie Wales, Rebecca Cashmore, Sandra Marshall, Jon Arcelus, Paul Robinson, Sarah Byford, Sabine Landau, Vanessa Lawrence, Hubertus Himmerich, Janet Treasure, Ulrike Schmidt","doi":"10.3310/FTJP6744","DOIUrl":"https://doi.org/10.3310/FTJP6744","url":null,"abstract":"<p><strong>Background: </strong>A substantial proportion of anorexia nervosa patients require intensive treatments, commonly inpatient or day-patient treatment. The relative merits of these treatments for adults with anorexia nervosa are unknown. Therefore, a trial investigating the clinical effectiveness and cost-effectiveness of inpatient treatment-as-usual versus a stepped-care day-patient approach in adults with anorexia nervosa (DAISIES) was commissioned. This trial terminated prematurely due to poor recruitment, mainly resulting from COVID-19's impact on service provision.</p><p><strong>Objective: </strong>We describe the rationale, methods and available outcomes of the DAISIES trial. Reasons behind the trial's failure and implications for future research are investigated.</p><p><strong>Design: </strong>A two-arm multicentre open-label parallel-group non-inferiority randomised controlled trial, evaluating the effectiveness, acceptability and cost-effectiveness of two intensive treatments for adults with severe anorexia nervosa.</p><p><strong>Setting: </strong>Specialist eating-disorder services in the United Kingdom with inpatient and/or day-patient treatment facilities.</p><p><strong>Participants: </strong>Adults (age 17 +) with severe anorexia nervosa (body mass index ≤ 16 kg/m<sup>2</sup>) requiring intensive treatment and (optionally) their carers. Intended sample size: 386.</p><p><strong>Interventions: </strong>Inpatient treatment-as-usual and a stepped-care day-patient treatment approach (with the option of initial inpatient treatment for medical stabilisation).</p><p><strong>Main outcome measures: </strong>The primary outcome was body mass index at 12 months post randomisation. Qualitative interviews conducted during the trial included semistructured interviews to investigate patients', families' and clinicians' views on treatments.</p><p><strong>Results: </strong>During the 16-month recruitment period (November 2020 to March 2022), 53 patients were approached. Of these, 15 were enrolled and randomly allocated to the inpatient treatment-as-usual (<i>n</i> = 7) or day-patient treatment (<i>n</i> = 8) treatment arms. All participants were female with a mean (standard deviation) age of 24.8 (9.1) years and a mean (standard deviation) body mass index of 14.4 (1.6) kg/m<sup>2</sup>. Patients' body mass indexes had increased similarly in both groups at 12 months. Participants perceived the stepped-care day-patient treatment approach to be more acceptable than inpatient treatment-as-usual. Qualitative interviews with patients, carers and clinicians suggested valued (e.g. multidisciplinary provision of care) and disliked (e.g. perceived over-focus on weight gain) aspects of treatment. Investigation of the reasons behind the trial's failure revealed strong treatment preferences among patients as the most common reason for non-participation, alongside the impact of COVID-19 on service provision.</p><p><strong>Limitations: </strong>The main tri","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-37"},"PeriodicalIF":3.5,"publicationDate":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143407138","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Understanding the effectiveness and underlying mechanisms of lifestyle modification interventions in adults with learning disabilities: a mixed-methods systematic review.","authors":"Dikshyanta Rana, Sophie Westrop, Nishant Jaiswal, Evi Germeni, Arlene McGarty, Louisa Ells, Phillippa Lally, Michael McEwan, Craig Melville, Leanne Harris, Olivia Wu","doi":"10.3310/BSTG4556","DOIUrl":"https://doi.org/10.3310/BSTG4556","url":null,"abstract":"<p><strong>Background: </strong>Adults with learning disabilities face increased risks of unhealthy lifestyle behaviours, including alcohol consumption, smoking, low physical activity, sedentary behaviour and poor diet. Lifestyle modification interventions that target health-risk behaviours can prevent or reduce their negative effects. The goal of this project was to investigate the effectiveness and underlying mechanisms of lifestyle modification interventions in adults with learning disabilities.</p><p><strong>Methods: </strong>A systematic review and meta-analysis were conducted to determine the effectiveness of lifestyle modification interventions and their components in targeting health risk behaviours in adults with learning disabilities. Major electronic databases, clinical trial registries, grey literature, and citations of systematic reviews and included studies were searched in January 2021 (updated in February 2022). We included randomised and non-randomised controlled trials targeting alcohol consumption, smoking, low physical activity only, sedentary behaviour and poor diet in adults (aged ≥ 18 years) with learning disabilities. Studies were also coded based on the extent of use of theories and behaviour change techniques in interventions. Risk of bias in studies was assessed using appropriate tools. A realist synthesis of qualitative, quantitative and mixed-methods literature was conducted to complement the systematic review findings by identifying key intervention mechanisms that are likely to improve the health of adults with learning disabilities. Data were synthesised in the form of a programme theory regarding complex causal mechanisms and how these interact with social context to produce outcomes. All findings were integrated into a logic model. A patient and public involvement group provided input and insights throughout the project.</p><p><strong>Results: </strong>A total of 80 studies with 4805 participants were included in the systematic review. The complexity of lifestyle modification interventions was dismantled by identifying six core components that influenced outcomes. These components could be present in interventions targeting single or multiple health risk behaviors, either as individual elements or in various combinations. Interventions on alcohol and smoking behaviours were found to be effective, but this was based on limited evidence. The effectiveness of interventions targeting low physical activity only or multiple behaviours (low physical activity only, sedentary behaviours and poor diet) was mixed. All interventions had a varying level of statistical significance. The intervention-level network meta-analysis for weight management outcomes showed none of the interventions was associated with a statistically significant change in outcomes when compared to treatment as usual and each other. Similar findings were observed in the component network meta-analysis. A variety of theories and behaviour change techniqu","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 4","pages":"1-168"},"PeriodicalIF":3.5,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143536998","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Public and patient involvement (PPI) in the design, execution and dissemination of a trial: the BISTRO trial.","authors":"David Coyle, Paula Ormandy, Nancy Fernandes da Silva, Simon Davies","doi":"10.3310/DOTR5903","DOIUrl":"10.3310/DOTR5903","url":null,"abstract":"<p><strong>Background: </strong>For people receiving haemodialysis, a balance has to be struck between removing sufficient but not too much fluid during a treatment session and maintaining any remaining kidney function they might have. In the BISTRO trial, this study sought to establish if getting the balance right might be improved by the additional use of bioimpedance, a device that measures body fluid composition to help decide how much fluid to remove during dialysis. Designing and executing this trial, which incorporated complex and repeated trial procedures that would be dependent on participant engagement, presented challenges that demanded effective public and patient involvement.</p><p><strong>Objectives: </strong>This study aimed to develop an effective public and patient involvement participation model, ensuring that the patient voice was heard by the Trial Management Group, with a Patient Advisory Group undertaking coproduction of all participant-facing documents and communications, including dissemination of the trial results, with the main purpose of maximising participant engagement in the study.</p><p><strong>Design: </strong>An open-label randomised controlled trial in which 439 participants from 34 centres were allocated for regular assessments of their bodily fluid content with or without the use of bioimpedance measurements.</p><p><strong>Interventions: </strong>Development of an effective public and patient involvement working model that was represented within the Trial Management Group, contributing to protocol design, selection of bioimpedance device, and coproduction of all participant-facing communications including dissemination of trial findings.</p><p><strong>Main outcome measures: </strong>Public and patient involvement contribution prior to trial initiation, description of the participant-facing communications, adherence to trial materials, dropout and dissemination of trial findings. Post-trial evaluation by research teams, Patient Advisory Group and co-applicants.</p><p><strong>Results: </strong>An effective working model was developed which relied on remuneration of the public and patient involvement patient lead and use of social media (e.g. WhatsApp) to maximise inclusivity. The Patient Advisory Group coproduced with the Trial Management Group a series of communication postcards and newsletters and a web page to support the participants and disseminate the trial results that were highly rated by research teams, but not always passed on to trial participants. Participant adherence to the main trial outcomes was excellent (113.6% urine collections obtained). Potentially avoidable dropout was 14.4%, with 3.6% being clearly attributable to inability or unwillingness to comply with the trial procedures. Reflections by the Patient Advisory Group indicated that they felt valued, involved and listened to but anticipated more direct involvement with the trial participants, recommending that barriers to this be addressed ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-18"},"PeriodicalIF":3.5,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11808441/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143064384","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A systematic review of the cost-effectiveness of anti-VEGF drugs for the treatment of diabetic retinopathy.","authors":"Robert Hodgson, Matthew Walton, Helen Fulbright, Laura Bojke, Ruth Walker, Alexis Llewellyn, Sofia Dias, Lesley Stewart, David Steel, John Lawrenson, Tunde Peto, Mark Simmonds","doi":"10.3310/NHYK3694","DOIUrl":"10.3310/NHYK3694","url":null,"abstract":"<p><strong>Background: </strong>Non-proliferative and proliferative diabetic retinopathy are common complications of diabetes and a major cause of sight loss. Anti-vascular endothelial growth factor drugs represent a treatment option for people with diabetic retinopathy and are routinely used to treat various other eye conditions. However, anti-vascular endothelial growth factor drugs are expensive relative to current care options, and it is unclear whether this additional cost is justified when the immediate risk of vision loss is lower compared to patients with more aggressive ophthalmological conditions.</p><p><strong>Objective: </strong>To systematically review the evidence supporting the cost-effectiveness of alternative treatments for diabetic retinopathy.</p><p><strong>Methods: </strong>A systematic review of all comparative cost-effectiveness studies evaluating any treatment for diabetic retinopathy was conducted. Bibliographic searches were carried out to identify studies reporting on the cost-effectiveness of treatments for diabetic retinopathy; the latest searches were conducted on 28 April 2023. Included studies were synthesised narratively and evaluated with reference to UK decision-making. Studies were grouped by population into non-proliferative diabetic retinopathy and proliferative diabetic retinopathy.</p><p><strong>Results: </strong>The review identified five studies in the proliferative diabetic retinopathy population, all of which examined the cost-effectiveness of anti-vascular endothelial growth factor treatments compared to pan-retinal photocoagulation. Results of these studies suggest that anti-vascular endothelial growth factor treatments offer some additional benefits in terms of preserved visual acuity but also incur substantial additional costs relative to pan-retinal photocoagulation. Most authors agreed that the additional costs outweigh the limited benefits, especially in certain patient subgroups without pre-existing oedema. As most of the identified evidence considered a US perspective, it is unclear how these results would translate to a UK setting. Two studies were identified in the non-proliferative diabetic retinopathy population. There was limited evidence to support the early use of anti-vascular endothelial growth factor treatment. However, one UK study suggested that early treatment of non-proliferative diabetic retinopathy with pan-retinal photocoagulation is cost-effective compared to delayed pan-retinal photocoagulation.</p><p><strong>Conclusions: </strong>Overall, there is a dearth of cost-effectiveness evidence considering the UK context. The identified studies raised doubts about the cost-effectiveness of anti-vascular endothelial growth factor treatments for proliferative diabetic retinopathy. No conclusions can be made regarding the cost-effectiveness of anti-vascular endothelial growth factor treatments for non-proliferative diabetic retinopathy. Future research should focus on developing rigorou","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-19"},"PeriodicalIF":3.5,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11808443/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143064382","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Multi-cancer early detection tests for general population screening: a systematic literature review.","authors":"Ros Wade, Sarah Nevitt, Yiwen Liu, Melissa Harden, Claire Khouja, Gary Raine, Rachel Churchill, Sofia Dias","doi":"10.3310/DLMT1294","DOIUrl":"10.3310/DLMT1294","url":null,"abstract":"<p><strong>Background: </strong>General population cancer screening in the United Kingdom is limited to selected cancers. Blood-based multi-cancer early detection tests aim to detect potential cancer signals from multiple cancers in the blood. The use of a multi-cancer early detection test for population screening requires a high specificity and a reasonable sensitivity to detect early-stage disease so that the benefits of earlier diagnosis and treatment can be realised.</p><p><strong>Objective: </strong>To undertake a systematic literature review of the clinical effectiveness evidence on blood-based multi-cancer early detection tests for screening.</p><p><strong>Methods: </strong>Comprehensive searches of electronic databases (including MEDLINE and EMBASE) and trial registers were undertaken in September 2023 to identify published and unpublished studies of multi-cancer early detection tests. Test manufacturer websites and reference lists of included studies and pertinent reviews were checked for additional studies. The target population was individuals aged 50-79 years without clinical suspicion of cancer. Outcomes of interest included test accuracy, number and proportion of cancers detected (by site and stage), time to diagnostic resolution, mortality, potential harms, health-related quality of life, acceptability and satisfaction. The risk of bias was assessed using the quality assessment of diagnostic accuracy studies-2 checklist. Results were summarised using narrative synthesis. Stakeholders contributed to protocol development, report drafting and interpretation of review findings.</p><p><strong>Results: </strong>Over 8000 records were identified. Thirty-six studies met the inclusion criteria: 1 ongoing randomised controlled trial, 13 completed cohort studies, 17 completed case-control studies and 5 ongoing cohort or case-control studies. Individual tests claimed to detect from 3 to over 50 different types of cancer. Diagnostic accuracy of currently available multi-cancer early detection tests varied substantially: Galleri<sup>®</sup> (GRAIL, Menlo Park, CA, USA) sensitivity 20.8-66.3%, specificity 98.4-99.5% (three studies); CancerSEEK (Exact Sciences, Madison, WI, USA) sensitivity 27.1-62.3%, specificity 98.9- 99.1% (two studies); SPOT-MAS™ (Gene Solutions, Ho Chi Minh City, Vietnam) sensitivity 72.4-100%, specificity 97.0-99.9% (two studies); Trucheck™ (Datar Cancer Genetics, Bayreuth, Germany) sensitivity 90.0%, specificity 96.4% (one study); Cancer Differentiation Analysis (AnPac Bio, Shanghai, China) sensitivity 40.0%, specificity 97.6% (one study). AICS<sup>®</sup> (AminoIndex Cancer Screening; Ajinomoto, Tokyo, Japan) screens for individual cancers separately, so no overall test performance statistics are available. Where reported, sensitivity was lower for detecting earlier-stage cancers (stages I-II) compared with later-stage cancers (stages III-IV). Studies of seven other multi-cancer early detection tests at an unclear stage of","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 2","pages":"1-105"},"PeriodicalIF":3.5,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11808444/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143079034","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development of a clinical decision support tool for Primary care Management of lower Urinary tract Symptoms in men: the PriMUS study.","authors":"Adrian Edwards, Ridhi Agarwal, Janine Bates, Alison Bray, Sarah Milosevic, Emma Thomas-Jones, Michael Drinnan, Marcus Drake, Peter Michell, Bethan Pell, Haroon Ahmed, Natalie Joseph-Williams, Kerenza Hood, Yemisi Takwoingi, Chris Harding","doi":"10.3310/RGTW5711","DOIUrl":"10.3310/RGTW5711","url":null,"abstract":"<p><strong>Background: </strong>Lower urinary tract symptoms particularly affect older men and their quality of life. General practitioners currently have no easily available assessment tools to diagnose lower urinary tract symptom causes. Referrals to urology specialists are increasing. General practitioner access to simple, accurate tests and clinical decision tools could facilitate management of lower urinary tract symptoms in primary care.</p><p><strong>Objectives: </strong>To determine which of several index tests in combination, best predicted three diagnoses (detrusor overactivity, bladder outlet obstruction and/or detrusor underactivity) in men presenting with lower urinary tract symptoms in primary care. To develop and validate three diagnostic prediction models, and a prototype primary care clinical decision support tool.</p><p><strong>Design: </strong>Prospective diagnostic accuracy study. Two participant cohorts, for <i>development</i> and <i>validation</i>, underwent simple index tests and a reference standard (invasive urodynamics).</p><p><strong>Setting: </strong>General practices in England and Wales.</p><p><strong>Participants: </strong>Men (16 years and over) consulting their general practitioner with lower urinary tract symptoms.</p><p><strong>Sample size: </strong>Separate calculations for model development and validation cohorts, from literature estimates of detrusor overactivity, bladder outlet obstruction and detrusor underactivity prevalences of 57%, 31% and 16%, respectively.</p><p><strong>Predictors and index tests: </strong>Twelve potential predictors considered for three diagnostic models.</p><p><strong>Main outcome measures: </strong>The primary outcome was diagnostic model sensitivity and specificity for detecting bladder outlet obstruction, detrusor underactivity and detrusor overactivity, with 75.0% considered minimum clinically useful performance.</p><p><strong>Statistical analysis: </strong>Three separate logistic regression models generated with index test variables to predict the presence of bladder outlet obstruction, detrusor overactivity, detrusor underactivity conditions in men with lower urinary tract symptoms.</p><p><strong>Results: </strong>One model each was developed and validated for bladder outlet obstruction and detrusor underactivity, two for detrusor overactivity (detrusor overactivity main, detrusor overactivity sensitivity analysis 2). Age, voiding symptoms subscore, prostate-specific antigen level, median maximum flow rate, median voided volume were predictors for bladder outlet obstruction. Median maximum flow rate and post-void residual volume were predictors for detrusor underactivity. Age, post-void residual volume and median voided volume were included in detrusor overactivity main model, while age and storage symptoms subscore predicted detrusor overactivity sensitivity analysis 2. For all four models, sensitivity of 75.0% could be achieved with a specificity of 74.2%, 47.3%, 45.6% and 46","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 1","pages":"1-140"},"PeriodicalIF":3.5,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11874884/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143079033","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Energetic activity for depression in young people aged 13-17 years: the READY feasibility RCT.","authors":"Megan Smith, Ryan James, Neil Howlett, Silvana Mengoni, Julia Jones, Erika Sims, David Turner, Kelly Grant, Allan Clark, Jamie Murdoch, Lindsay Bottoms, Jonathan Wilson, Shivani Sharma, Angel Chater, Cecile Guillard, Timothy Clarke, Andy Jones, Lee David, Solange Wyatt, Claire Rourke, David Wellsted, Daksha Trivedi","doi":"10.3310/KWNH4507","DOIUrl":"10.3310/KWNH4507","url":null,"abstract":"<p><strong>Background: </strong>Prevalence of depression is increasing in young people. Behaviour change interventions providing benefits equal to or greater than talking therapies or pharmacological alternatives are needed. Exercise could be beneficial for young people with depression, but we lack robust trials of its effectiveness.</p><p><strong>Objective: </strong>To test whether an exercise intervention targeting young people with depression is feasible, including recruitment and retention of young people, recruitment and training of exercise professionals and intervention delivery.</p><p><strong>Design: </strong>Three-arm cluster feasibility randomised controlled trial with embedded process evaluation and health economic data collection.</p><p><strong>Setting: </strong>Local community venues in Hertfordshire, Bedfordshire and Norfolk.</p><p><strong>Participants: </strong>Young people aged 13-17 years experiencing mild to moderate low mood or depression (indicated by scoring 17-36 on the Child Depression Inventory version 2) identified by mental health services, schools or self-referral.</p><p><strong>Interventions: </strong>Participants were randomised to one of three groups: high-intensity exercise, low-intensity exercise or a social activity control. Group sessions ran twice-weekly for 12 weeks delivered by registered exercise professionals, supported by mental health support workers.</p><p><strong>Main outcome measures: </strong>Referral, recruitment and retention rates; attendance at group sessions; adherence to and ability to reach intensity during exercise sessions; proportions of missing data, and adverse events measured at baseline and at 3 and 6 months; resource use; and reach and representativeness.</p><p><strong>Results: </strong>Of 321 referrals to the study, 173 were assessed for eligibility, and of the target sample size of 81, 15 were recruited and 14 were randomised (one withdrew). The retention rate was 71.4% and attendance at intervention sessions was > 67%; data completeness was > 80% for baseline assessments. Follow-up completion rate at 14 weeks was > 80% for most outcomes, with 50% for accelerometer data in the low-intensity group. Trial processes and the intervention were acceptable to young people. Barriers to and facilitators of intervention delivery were identified.</p><p><strong>Limitations: </strong>Findings highlighted challenges around recruitment, delivery of exercise interventions and informed ways of addressing barriers to recruitment for future studies. The study was conducted between October 2020 and August 2022 and consequently the COVID-19 pandemic had a disruptive impact on implementation.</p><p><strong>Conclusions: </strong>A large randomised trial of the effectiveness of the intervention is not feasible using the current study design, but issues relating to recruitment could be addressed with further work.</p><p><strong>Future work: </strong>Developing appropriate recruitment strategies via triage serv","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-26"},"PeriodicalIF":3.5,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11744430/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142871913","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}