Health technology assessment最新文献

{"title":"A personalised health intervention to maintain independence in older people with mild frailty: a process evaluation within the HomeHealth RCT.","authors":"Rachael Frost, Yolanda Barrado-Martín, Louise Marston, Shengning Pan, Jessica Catchpole, Tasmin Rookes, Sarah Gibson, Jane Hopkins, Farah Mahmood, Benjamin Gardner, Rebecca L Gould, Claire Jowett, Rashmi Kumar, Rekha Elaswarapu, Christina Avgerinou, Paul Chadwick, Kalpa Kharicha, Vari M Drennan, Kate Walters","doi":"10.3310/MBCV1794","DOIUrl":"https://doi.org/10.3310/MBCV1794","url":null,"abstract":"<p><strong>Background: </strong>Frailty is common in later life and can lead to adverse health outcomes. Services aimed at preventing decline in early stages of frailty may support older people to remain independent for longer. We developed and tested a new service, HomeHealth, in a randomised controlled trial. HomeHealth was a multidomain behaviour change service based in the voluntary sector in England targeting mobility, socialising, nutrition and psychological well-being.</p><p><strong>Objective: </strong>To describe the population reach, fidelity, acceptability, context and mechanisms of impact of the HomeHealth service.</p><p><strong>Design and methods: </strong>Mixed-methods process evaluation of a randomised trial.</p><p><strong>Setting and participants: </strong>HomeHealth trial participants (older people aged 65+ years with mild frailty) and service providers.</p><p><strong>Data sources and analysis: </strong>Population reach was evaluated through comparison to local census data. Fidelity of audio-recorded appointments was assessed by two independent raters using a structured checklist. Using data from appointments attended, types of goals set and progress towards goals, we described appointment characteristics, goals and signposting, and evaluated three mechanisms of impact: (1) effect of appointment attendance on independence, (2) effect of goal progress on independence and (3) whether selecting a particular goal type led to improvements in the corresponding intermediate outcome. We thematically analysed qualitative interviews with 49 older people, 7 HomeHealth workers and 8 stakeholders to explore acceptability and context.</p><p><strong>Results: </strong>HomeHealth participants were similar with regards to deprivation, education and housing status to the local older population but with lower rates of minority ethnic groups. HomeHealth was delivered with good fidelity (81.7%) in voluntary sector organisations. Appointments were well attended (mean 5.33 out of the 6 intended), but attendance was not associated with better independence scores at 12 months [mean difference 1.29 (-8.20 to 10.78)]. Participants varied in progress towards goals within appointments (mean progress 1.15/2.00), but greater goal progress was not associated with improved independence scores at 12 months [mean difference -0.40 (-2.38 to 1.58)]. Mobility goals were most frequently selected (49%), but type of goal had no impact on independence and little impact on intermediate outcomes. Forty-one per cent were signposted or referred to other supportive services, with ongoing support where needed throughout this process. Qualitative data indicated that HomeHealth was acceptable, empowering for those who saw a need for change and fitted well within host voluntary sector organisations.</p><p><strong>Limitations: </strong>Census data were only available for all adults aged over 65 in local areas rather than a mildly frail population, who are likely to be older, female ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-23"},"PeriodicalIF":3.5,"publicationDate":"2025-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143788267","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":"Anti-VEGF drugs compared with laser photocoagulation for the treatment of proliferative diabetic retinopathy: a systematic review and individual participant data meta-analysis.","authors":"Mark Simmonds, Alexis Llewellyn, Ruth Walker, Helen Fulbright, Matthew Walton, Rob Hodgson, Laura Bojke, Lesley Stewart, Sofia Dias, Thomas Rush, João Pereira Figueira, Sobha Sivaprasad, John G Lawrenson, Tunde Peto, David Steel","doi":"10.3310/MJYP6578","DOIUrl":"https://doi.org/10.3310/MJYP6578","url":null,"abstract":"<p><strong>Background: </strong>Proliferative diabetic retinopathy is a major cause of sight loss in people with diabetes, with a high risk of vitreous haemorrhage, tractional retinal detachment and other complications. Panretinal photocoagulation is the primary established treatment for proliferative diabetic retinopathy. Anti-vascular endothelial growth factor drugs are used to treat various eye conditions and may be beneficial for people with proliferative diabetic retinopathy.</p><p><strong>Objective: </strong>To investigate the efficacy and safety of anti-vascular endothelial growth factor therapy for the treatment of proliferative diabetic retinopathy when compared to panretinal photocoagulation.</p><p><strong>Methods: </strong>A systematic review and network meta-analysis of randomised controlled trials comparing anti-vascular endothelial growth factor (alone or in combination) to panretinal photocoagulation in people with proliferative diabetic retinopathy. The database searches were updated in May 2023. Trials where the primary focus was treatment of macular oedema or vitreous haemorrhage were excluded. Key outcomes were best corrected visual acuity, diabetic macular oedema and vitreous haemorrhage. Individual participant data were obtained and analysed for three large, high-quality trials in combination with published data from other trials. Network meta-analyses of best corrected visual acuity and meta-analyses of other outcomes combined individual participant data with published data from other trials; regression analyses against patient covariates used just the individual participant data.</p><p><strong>Results: </strong>Twelve trials were included: one of aflibercept, five of bevacizumab and six of ranibizumab. Individual participant data were available from 1 aflibercept and 2 ranibizumab trials, representing 624 patients (33% of the total). When considered together, anti-vascular endothelial growth factors produced a modest, but not clinically meaningful, benefit over panretinal photocoagulation in best corrected visual acuity, after 1 year of follow-up (mean difference in logarithm of the minimum angle of resolution -0.116, 95% credible interval -0.183 to -0.038). There was no clear evidence of a difference in effectiveness between the anti-vascular endothelial growth factors. The benefit of anti-vascular endothelial growth factor appears to decline over time. Analysis of the individual participant data trials suggested that anti-vascular endothelial growth factor therapy may be more effective in people with poorer visual acuity, in those who have vitreous haemorrhage and, possibly, in people with poorer vision generally. Anti-vascular endothelial growth factor was superior to panretinal photocoagulation at preventing macular oedema after 1 year (relative risk 0.48, 95% confidence interval 0.28 to 0.83) and possibly at preventing vitreous haemorrhage (relative risk 0.72, 95% confidence interval 0.47 to 1.10). Anti-vascular endoth","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-75"},"PeriodicalIF":3.5,"publicationDate":"2025-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143788268","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":"Recent research in myalgic encephalomyelitis/chronic fatigue syndrome: an evidence map.","authors":"Alex Todhunter-Brown, Pauline Campbell, Cathryn Broderick, Julie Cowie, Bridget Davis, Candida Fenton, Sarah Markham, Ceri Sellers, Katie Thomson","doi":"10.3310/BTBD8846","DOIUrl":"10.3310/BTBD8846","url":null,"abstract":"<p><strong>Background: </strong>Myalgic encephalomyelitis/chronic fatigue syndrome is a chronic condition, classified by the World Health Organization as a nervous system disease, impacting around 17 million people worldwide. Presentation involves persistent fatigue and postexertional malaise (a worsening of symptoms after minimal exertion) and a wide range of other symptoms. Case definitions have historically varied; postexertional malaise is a core diagnostic criterion in current definitions. In 2022, a James Lind Alliance Priority Setting Partnership established research priorities relating to myalgic encephalomyelitis/chronic fatigue syndrome.</p><p><strong>Objective(s): </strong>We created a map of myalgic encephalomyelitis/chronic fatigue syndrome evidence (2018-23), showing the volume and key characteristics of recent research in this field. We considered diagnostic criteria and how current research maps against the James Lind Alliance Priority Setting Partnership research priorities.</p><p><strong>Methods: </strong>Using a predefined protocol, we conducted a comprehensive search of Cochrane, MEDLINE, EMBASE and Cumulative Index to Nursing and Allied Health Literature. We included all English-language research studies published between January 2018 and May 2023. Two reviewers independently applied inclusion criteria with consensus involving additional reviewers. Studies including people diagnosed with myalgic encephalomyelitis/chronic fatigue syndrome using any criteria (including self-report), of any age and in any setting were eligible. Studies with < 10 myalgic encephalomyelitis/chronic fatigue syndrome participants were excluded. Data extraction, coding of topics (involving stakeholder consultation) and methodological quality assessment of systematic reviews (using A MeaSurement Tool to Assess systematic Reviews 2) was conducted independently by two reviewers, with disagreements resolved by a third reviewer. Studies were presented in an evidence map.</p><p><strong>Results: </strong>Of the 11,278 identified studies, 742 met the selection criteria, but only 639 provided sufficient data for inclusion in the evidence map. These reported data from approximately 610,000 people with myalgic encephalomyelitis/chronic fatigue syndrome. There were 81 systematic reviews, 72 experimental studies, 423 observational studies and 63 studies with other designs. Most studies (94%) were from high-income countries. Reporting of participant details was poor; 16% did not report gender, 74% did not report ethnicity and 81% did not report the severity of myalgic encephalomyelitis/chronic fatigue syndrome. Forty-four per cent of studies used multiple diagnostic criteria, 16% did not specify criteria, 24% used a single criterion not requiring postexertional malaise and 10% used a single criterion requiring postexertional malaise. Most (89%) systematic reviews had a low methodological quality. Five main topics (37 subtopics) were included in the evidence map. Of","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-78"},"PeriodicalIF":3.5,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11973615/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143752305","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":"Gait Rehabilitation for Early rheumatoid Arthritis Trial (GREAT): lessons learnt from a mixed-methods feasibility study and internal pilot trial.","authors":"Gordon J Hendry, Lindsay Bearne, Linda Fenocchi, Nadine E Foster, Sally Gates, Emma Godfrey, Samantha Hider, Lisa Jolly, Helen Mason, Alex McConnachie, Iain McInnes, Aimie Patience, Yeliz Prior, Catherine Sackley, Mandeep Sekhon, Bethany Stanley, Jason Vickers, Jim Woodburn, Martijn Pm Steultjens","doi":"10.3310/XBDJ8546","DOIUrl":"10.3310/XBDJ8546","url":null,"abstract":"<p><strong>Background: </strong>People with rheumatoid arthritis experience foot and lower limb pain due to active synovitis, resulting in impaired lower limb function. Earlier intervention may help with prevention of functional decline. The aims of this research were to develop and evaluate a new gait rehabilitation intervention for people with early rheumatoid arthritis, evaluate its feasibility, and to test whether or not gait rehabilitation plus usual care is more clinically and cost-effective than usual care alone.</p><p><strong>Design and methods: </strong>We undertook a single-arm, repeated-measures, pre- and post-intervention, mixed-methods feasibility study with embedded qualitative components. We planned to undertake a pragmatic, two-arm, multicentre, superiority randomised controlled trial, with health economic evaluation, process evaluation and internal pilot.</p><p><strong>Setting and participants: </strong>Participants with early rheumatoid arthritis (< 2 years post diagnosis) were identified from early arthritis and rheumatology outpatient clinics and referred for intervention in either podiatry or physiotherapy clinics.</p><p><strong>Intervention(s): </strong>Participants were randomised to a gait rehabilitation programme (Gait Rehabilitation Early Arthritis Trial Strides) involving a six-task gait circuit. Sessions were underpinned by motivational interviewing to facilitate behaviour change, supported by trained physiotherapists or podiatrists for a minimum of two sessions. Both groups received their normal usual care from the rheumatology multidisciplinary team.</p><p><strong>Main outcome measures: </strong>Outcome measures for the feasibility study were intervention acceptability, adherence using the Exercise Adherence Rating Scale and fidelity using the Motivational Interviewing Treatment Integrity Scale. The main outcome measure for the internal pilot/randomised controlled trial was the Foot Function Index disability subscale. Outcomes were measured at baseline, 3 months, 6 months and 12 months. Other outcomes: intervention acceptability questionnaire, Exercise Adherence Rating Scale, exercise treatment beliefs via the Theory of Planned Behaviour Questionnaire, intervention fidelity (Motivational Interviewing Treatment Integrity Scale), health-related quality of life (EuroQol-5 Dimensions, five-level score).</p><p><strong>Results: </strong>Thirty-five participants were recruited for feasibility and 23 (65.7%) completed 12-week follow-up. Intervention acceptability was excellent: 21/23 were confident that it could help and would recommend it and 22/23 indicated it made sense to them. Adherence was good, with a median (interquartile range) Exercise Adherence Rating Scale score of 17/24 (12.5-22.5). Twelve participants' and nine therapists' interviews confirmed intervention acceptability, identified perceptions of benefit, but highlighted some barriers to completion. Motivational Interviewing Treatment Integrity Scale scores dem","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-48"},"PeriodicalIF":3.5,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11973904/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143752298","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":"Experiences of support to return to work after stroke: longitudinal case studies from RETAKE trial.","authors":"Diane Trusson, Katie Powers, Kathryn Radford, Audrey Bowen, Kristelle Craven, Jain Holmes, Rebecca Lindley, Christopher McKevitt, Julie Phillips, Ellen Thompson, Caroline Watkins, David J Clarke","doi":"10.3310/WRKS9661","DOIUrl":"10.3310/WRKS9661","url":null,"abstract":"<p><strong>Background: </strong>Returning to work after stroke has physical, psychological and financial benefits for stroke survivors. However, global evidence estimates return-to-work rates 1 year post stroke at < 50%. Although its importance is acknowledged by policy-makers and healthcare providers, vocational rehabilitation is not always part of National Health Service usual care post stroke. Currently, there is limited evidence of the effectiveness of return-to-work support interventions. RETurn to work After stroKE was a multicentre individually randomised controlled pragmatic trial, with embedded process and health economic evaluations. RETurn to work After stroKE aimed to establish whether Early Stroke Specialist Vocational Rehabilitation plus usual care improves the likelihood of return to work at 12 months post stroke compared to usual care alone. As part of an embedded process evaluation, longitudinal case studies enabled exploration of participants' experiences of support to return to work in the trial.</p><p><strong>Objectives: </strong>This article aims to understand participants' experiences of being supported to return to work and explores the social and structural factors which support, or act as barriers to, implementation of the Early Stroke Specialist Vocational Rehabilitation intervention.</p><p><strong>Method: </strong>A longitudinal case-study approach was used to compare experiences of post-stroke return-to-work support received over 12 months by 15 participants who received the Early Stroke Specialist Vocational Rehabilitation intervention plus usual care, and 11 participants who received usual care only. Data were gathered at three time points using follow-up questionnaires, health records, intervention delivery records and semistructured interviews with participants and seven nominated informal carers. Interviews were also conducted with 1 employer and 11 occupational therapists delivering the intervention.</p><p><strong>Setting: </strong>Sixteen National Health Service sites across England and Wales.</p><p><strong>Findings: </strong>In the intervention arm, stroke survivors, carers and employers reported benefits from information and support from the treating occupational therapist to facilitate acceptance of, and adaptation to, post-stroke abilities. Participants also valued occupational therapists' provision of sustained and tailored vocational rehabilitation, co-ordinating their care and advocating for them in return-to-work discussions with their employers. Those unable to return to their previous employment were supported to consider alternative options. In contrast, participants who received usual care only reported feeling abandoned when community rehabilitation support ended, typically after 2-8 weeks. Usual care largely focused on restoring physical function, leaving these participants struggling to find return-to-work information, advice and support. Longitudinal case studies enabled psychosocial and environm","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-27"},"PeriodicalIF":3.5,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11973616/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143752287","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":"Management of diarrhoea in patients with stable ulcerative colitis with low FODMAP diet, amitriptyline, ondansetron or loperamide: the MODULATE RCT.","authors":"Lauren A Moreau, Alexander Charles Ford, Matthew James Brookes, Sandra Graca, Elspeth Guthrie, Suzanne Hartley, Lesley Houghton, Karen Kemp, Nicholas A Kennedy, Yvonne McKenzie, Delia Muir, Pei Loo Ow, Christopher Probert, Emma Pryde, Christopher Taylor, Thomas A Willis, Alexandra Wright-Hughes, Amanda J Farrin","doi":"10.3310/GHFE4871","DOIUrl":"10.3310/GHFE4871","url":null,"abstract":"<p><strong>Background: </strong>Many patients with ulcerative colitis report ongoing diarrhoea even when their disease is stable and in remission.</p><p><strong>Design: </strong>MODULATE was a pragmatic, multicentre, seamless, adaptive, phase 2/3 open-label, parallel-group, multiarm multistage randomised controlled trial.</p><p><strong>Setting and participants: </strong>People aged over 18 years with stable ulcerative colitis who had diarrhoea, recruited from secondary care sites in the United Kingdom.</p><p><strong>Interventions: </strong>The control arm consisted of modified first-line dietary advice given to all patients with irritable bowel syndrome; the first interventional arm was amitriptyline, a tricyclic antidepressant, which at low doses slows colonic transit; the second intervention was loperamide, an antidiarrhoeal drug also thought to slow colonic transit; the third was ondansetron, an antiemetic thought to slow colonic transit; and the fourth was a diet low in fermentable oligo-, di-, and mono-saccharides and polyols, which is thought to reduce bloating and gas within the small intestine. All patients randomised to an interventional arm were to receive treatment for 6 months.</p><p><strong>Main outcome measures: primary outcome measures: </strong>Phase 2: Improvement in diarrhoea measured using the Gastrointestinal Symptom Rating Scale-irritable bowel syndrome questionnaire at 8 weeks post randomisation: improvement defined as those reporting minor discomfort from diarrhoea or less (scoring ≤ 2 on the diarrhoea subscale).</p><p><strong>Secondary outcome measures: </strong>Phases 2 and 3: Measured at both 8 weeks and 6 months: Improvement in diarrhoea measured using the Gastrointestinal Symptom Rating Scale-irritable bowel syndrome. Blood for C-reactive protein, stool for faecal calprotectin at 6 months only, reviewing case notes for escalation of medical therapy for ulcerative colitis. Anxiety and depression, via the Hospital Anxiety and Depression Scale.</p><p><strong>Results: </strong>The MODULATE trial opened in December 2021 and closed in January 2023. Of the eight secondary care sites that completed contracting, only four opened to recruitment during this time, and one person was randomised. Trial timelines coincided with the start of the COVID-19 pandemic, causing substantial delays and, ultimately, its early closure. During this time, the trial underwent two major redesign phases, enabling a fully remote participant pathway incorporating electronic consent, remote data capture, posted blood and stool sample kits for eligibility screening, delivery of the dietary intervention via telephone or video call platform, postage of trial investigational medicinal products directly to participants' homes and all trial follow-up appointments conducted via telephone. The second phase of redesign pushed the trial towards a fully decentralised model. However, this stage was not implemented due to the decision to close the trial early.</p><","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-30"},"PeriodicalIF":3.5,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11931405/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143624415","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":"The acceptability of blood spot screening and genome sequencing in newborn screening: a systematic review examining evidence and frameworks.","authors":"Duncan Chambers, Susan Baxter, Anastasios Bastounis, Katherine Jones, Burak Kundakci, Anna Cantrell, Andrew Booth","doi":"10.3310/RTPQ2268","DOIUrl":"10.3310/RTPQ2268","url":null,"abstract":"<p><strong>Background: </strong>Population-wide newborn blood spot screening programmes are a successful public health intervention used to detect whether the baby is at risk of certain rare conditions, with the aim of earlier diagnosis and provision of optimal care and treatment. Evaluating candidate conditions to include in newborn blood spot and genetic sequencing raises questions regarding acceptability to parents/carers.</p><p><strong>Methods: </strong>In the context of the possible expansion of the newborn blood spot screening programme in the United Kingdom, this review aimed to systematically review research on the acceptability to parents of newborn blood spot screening and genetic sequencing. A protocol was developed prior to commencing the review and was registered on the PROSPERO database. A team of researchers carried out the review, with checking at all stages carried out by at least two individuals. We included research published after 2013 with participants who were pregnant or a recent parent of a newborn and were resident in a high-income country. We included quantitative and qualitative studies that investigated the acceptability to parents/carers of newborn blood spot screening or genetic sequencing. Quantitative studies were narratively synthesised, and theories/frameworks identified and evaluated. Qualitative studies were analysed for recurring themes, and a meta-synthesis was carried out to compare and contrast these two types of data. We quality appraised included articles using tools appropriate for their study design.</p><p><strong>Results: </strong>Searches were carried out in September to November 2023 and screening identified 25 relevant research articles. Just over half were from North America, with four existing reviews and nine qualitative studies. Domains of acceptability described in the literature were: support for screening; level of anxiety, information and knowledge; consent; views of the procedure; and support after screening. The research indicated consensus support for blood spot screening, and for expanding to some other conditions, although some parental anxiety was reported. Parents/carers mostly perceived that they had received sufficient information, but the timing of this could be improved. While parents indicated interest in genomic screening, studies highlighted the need for clearer consent procedures and greater support for parents following genomic screening than for blood spot screening. Only three included studies reported using any kind of theoretical framework.</p><p><strong>Discussion: </strong>Most parents/carers found newborn blood spot screening programmes to be acceptable and favoured their large-scale implementation. A minority of parents/carers expressed concerns regarding the acceptability of processes underpinning newborn blood spot screening, such as consent, the timing of receiving information and support available after testing. More research is needed regarding the acceptability ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-53"},"PeriodicalIF":3.5,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11931406/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143630329","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":"Benefits of aldosterone receptor antagonism in chronic kidney disease: the BARACK-D RCT.","authors":"F D Richard Hobbs, Richard McManus, Clare Taylor, Nicholas Jones, Joy Rahman, Jane Wolstenholme, Louise Jones, Jennifer Hirst, Sam Mort, Ly-Mee Yu","doi":"10.3310/PYFT6977","DOIUrl":"10.3310/PYFT6977","url":null,"abstract":"<p><strong>Background: </strong>Chronic kidney disease affects around 10% of the global population and is associated with significant risk of progression to end-stage renal disease and vascular events. Aldosterone receptor antagonists such as spironolactone have shown prognostic benefits in patients with heart failure, but effects on patients with chronic kidney disease are uncertain.</p><p><strong>Objectives: </strong>To determine the effect of low-dose spironolactone on mortality and cardiovascular outcomes in people with chronic kidney disease stage 3b.</p><p><strong>Design: </strong>Prospective randomised open blinded end-point trial.</p><p><strong>Settings: </strong>Three hundred and twenty-nine general practitioner practices throughout the United Kingdom.</p><p><strong>Participants: </strong>Patients meeting the criteria for chronic kidney disease stage 3b (estimated glomerular filtration rate 30-44 ml/minute/1.73 m<sup>2</sup>) according to National Institute for Health and Care Excellence guidelines were recruited. Due to the higher than anticipated measurement error/fluctuations, the eligible range was extended to 30-50 ml/minute/1.73 m<sup>2</sup> following the initial recruitment period.</p><p><strong>Intervention: </strong>Participants were randomised 1 : 1 to receive either spironolactone 25 mg once daily in addition to standard care, or standard care only.</p><p><strong>Outcome measures: </strong>Primary outcome was the first occurring of all-cause mortality, first hospitalisation for heart disease (coronary heart disease, arrhythmia, atrial fibrillation, sudden death, failed sudden death), stroke, heart failure, transient ischaemic attack or peripheral arterial disease, or first occurrence of any condition not listed at baseline. Secondary outcome measures included changes in blood pressure, renal function, B-type natriuretic peptide, incidence of hyperkalaemia and treatment costs and benefits.</p><p><strong>Results: </strong>One thousand four hundred and thirty-four participants were randomised of the 3022 planned. We found no evidence of differences between the intervention and control groups in terms of effectiveness with the primary combined vascular end points, nor with the secondary clinical outcomes, including progression in renal decline. These results were similar for the total treatment periods or a 3-year follow-up period as originally planned. More adverse events were experienced and more participants discontinued treatment in the intervention group. Two-thirds of participants randomised to spironolactone stopped treatment within six months because they met pre-specified safety stop criteria. The addition of low-dose spironolactone was estimated to have a cost per quality-adjusted life-year gained value above the National Institute for Health and Care Excellence's threshold of £30,000.</p><p><strong>Limitations: </strong>Main limitations were difficulties in recruiting eligible participants resulting in an underpowered ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 5","pages":"1-130"},"PeriodicalIF":3.5,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11931407/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143663338","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 group psychological intervention for postnatal depression in British mothers of South Asian origin - the ROSHNI-2 RCT.","authors":"Nusrat Husain, Karina Lovell, Carolyn A Chew-Graham, Farah Lunat, Penny Bee, Matthias Pierce, Najia Atif, Saadia Aseem, Kamaldeep Bhui, Peter Bower, Traolach Brugha, Nasim Chaudhry, Akbar Ullah, Linda Davies, Nadeem Gire, Deepali Sharma, Jahanara Miah, Waqas Ahmed, Joe Kai, Ilyas Mirza, Jillian Morrison, Naeem Mohmed, Atif Rahman, Shanaya Rathod, Najma Siddiqi, Waquas Waheed, Christopher Williams, Nosheen Zaidi, Richard Emsley, Richard Morriss","doi":"10.3310/KKDS6622","DOIUrl":"10.3310/KKDS6622","url":null,"abstract":"<p><strong>Background: </strong>Postnatal depression is more common in British South Asian women than white women in the United Kingdom. Despite empirical evidence suggesting the effectiveness of cognitive-behavioural therapy as a first line of treatment, little evidence is available regarding its applicability to different minority ethnic groups.</p><p><strong>Objectives: </strong>Determining the clinical and cost-effectiveness of a culturally adapted group psychological intervention (Positive Health Programme) in primary care for British South Asian women with postnatal depression compared with treatment as usual.</p><p><strong>Setting: </strong>General practices and children's centres in the North West, East Midlands, Yorkshire, Glasgow and London.</p><p><strong>Participants: </strong>British South Asian women meeting the <i>Diagnostic and Statistical Manual of Mental Disorders</i> (Fifth Edition) depression criteria, aged 16 years or above, with infants up to 12 months.</p><p><strong>Design: </strong>A multicentre randomised controlled trial with an internal pilot and partially nested design to compare treatment as usual plus the Positive Health Programme with treatment as usual in British South Asian women with postnatal depression, with a qualitative study to examine the acceptability and feasibility of the intervention.</p><p><strong>Intervention: </strong>The Positive Health Programme, a culturally adapted group intervention based on the principles of cognitive-behavioural therapy delivered by facilitators over 12 sessions.</p><p><strong>Outcomes measures: </strong>The primary outcome was recovery from depression (Hamilton Depression Rating Scale ≤ 7) at end of intervention (approximately 4-6 months). Analysis of the primary outcome and the long-term follow-up (at 12 months) used a logistic random-effects model to estimate the odds ratio of caseness between treatments, adjusting for centre, severity of depression and education at baseline. Cost data were collected using an Economic Patient Questionnaire.</p><p><strong>Results: </strong>Seven hundred and thirty-two participants across four study centres were randomised by the Manchester Clinical Trials Unit. At 4 months, almost half of patients in the treatment (Positive Health Programme) group were recovered (138 or 49%), whereas 105 (37%) were recovered in the control (treatment as usual) group. By 12 months, the control (treatment as usual) and treatment (Positive Health Programme) group had over 50% recovery at 140 (54%) and 141 (54%), respectively. For the primary outcome, recovery from postnatal depression at end of intervention, we found a significant effect such that the odds of achieving recovery in the treatment group were almost twice as high compared to the treatment as usual group (odds ratio 1.97, 95% confidence interval 1.26 to 3.10). Between the two groups, there was no significant difference in the odds of recovery at 12 months (odds ratio 1.02, 95% confidence interval 0.6","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 6","pages":"1-113"},"PeriodicalIF":3.5,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11973614/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143752323","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":"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":"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":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11891625/pdf/","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":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}