Health technology assessment最新文献

{"title":"Sputum colour charts to guide antibiotic self-treatment of acute exacerbation of chronic obstructive pulmonary disease: the Colour-COPD RCT.","authors":"Eleni Gkini, Rachel L Adams, Daniella Spittle, Paul Ellis, Katherine Allsopp, Sanya Saleem, Matthew McKenna, Nick le Mesurier, Nicola Gale, Sarah Tearne, Peymane Adab, Rachel E Jordan, Nawar Diar Bakerly, Alice M Turner","doi":"10.3310/KPFD5558","DOIUrl":"https://doi.org/10.3310/KPFD5558","url":null,"abstract":"<p><strong>Background: </strong>Chronic obstructive pulmonary disease exacerbations (acute exacerbation of chronic obstructive pulmonary disease) are characterised by increased sputum volume, purulence and breathlessness. Patients are encouraged to recognise and treat acute exacerbation of chronic obstructive pulmonary disease as part of a self-management plan. Only half of acute exacerbation of chronic obstructive pulmonary disease are caused by bacterial infection, but self-management plans generally advocate use of antibiotics and steroids for all events, hence antibiotics may be overused. Sputum colour relates closely to bacterial load; thus it could determine whether antibiotics are appropriate. This pragmatic randomised controlled trial tested whether use of a sputum colour chart is safe and effective in United Kingdom primary care.</p><p><strong>Methods: </strong>Colour chronic obstructive pulmonary disease was a multicentre, randomised controlled trial in adults with chronic obstructive pulmonary disease who had ≥ 2 acute exacerbations of chronic obstructive pulmonary disease or ≥ 1 hospital admission for acute exacerbation of chronic obstructive pulmonary disease in the preceding year. The primary objective was to demonstrate that the Bronkotest<sup>®</sup> (London) sputum colour chart is non-inferior to usual care (safe). The primary outcome was rate of hospital admission for acute exacerbation of chronic obstructive pulmonary disease at 12 months; secondary outcomes included requirement for second courses of treatment and quality of life (chronic obstructive pulmonary disease assessment test score). Nested substudies examining daily symptoms via an e-diary and sputum culture assessed untreated acute exacerbation of chronic obstructive pulmonary disease rate and antibiotic resistance, respectively. A process evaluation examined trial fidelity and acceptability of the intervention, employing qualitative research methods incorporating patients as co-researchers.</p><p><strong>Limitations: </strong>The study was terminated early due to low recruitment (115/2954 planned sample size).</p><p><strong>Results: </strong>One hundred and fifteen patients were recruited and randomised 1 : 1 to colour chart use or usual care; they generally had severe Global Initiative for Chronic Obstructive Lung Disease D chronic obstructive pulmonary disease, with significant breathlessness (54% Medical Research Council score of 4 or 5) and poor quality of life (chronic obstructive pulmonary disease assessment test score at baseline 24). Comorbid respiratory and systemic disease was common. Self-management was delivered well in both arms, and the colour chart acceptable to patients and staff; no specific issues for patients with multiple long-term conditions were identified. Hospital admissions for acute exacerbation of chronic obstructive pulmonary disease tended to occur more in colour chart users [32 vs. 16%, relative risk 1.95 (0.92 to 4.18)], and antibiotic ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-42"},"PeriodicalIF":3.5,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144142392","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":"Technology-enabled CONTACT tracing in care homes in the COVID-19 pandemic: the CONTACT non-randomised mixed-methods feasibility study.","authors":"Carl A Thompson, Thomas A Willis, Amanda Farrin, Adam Gordon, Amrit Daffu-O'Reilly, Catherine Noakes, Kishwer Khaliq, Andrew Kemp, Tom Hall, Chris Bojke, Karen Spilsbury","doi":"10.3310/UHDN6497","DOIUrl":"https://doi.org/10.3310/UHDN6497","url":null,"abstract":"<p><strong>Background: </strong>Coronavirus disease 2019 devastated lives in care homes for older people, where residents faced higher mortality risks than the general population. Infection prevention and control decisions were critical to protect these vulnerable residents. Infection prevention and control measures like 'lockdowns' had their own risks, such as social isolation, alongside assumed benefits. A key non-pharmaceutical intervention for managing infections is contact tracing. Traditional contact tracing, which relies on recalling contacts, is not feasible in care homes where approximately 70% of residents have cognitive impairments. The CONtact TrAcing in Care homes using digital Technology intervention introduces Bluetooth-enabled wearable devices for automated contact tracing. We provided structured reports (scheduled regularly and in reaction to positive COVID-19 cases) on contact patterns to homes to support better-informed infection prevention and control decisions and potentially reduce blanket restrictive measures. We also partnered with the PROTECT COVID-19 research team to examine air quality in two of our homes.</p><p><strong>Methods: </strong>CONTACT was a non-randomised mixed-method feasibility study in four English care homes. Recruitment was via care home research networks, with individual consent. Data collection included routine device data, case report forms, qualitative interviews, field observations of care home activity and an adapted Normalisation Measure Development questionnaire survey to explore implementation using normalisation process theory. Quantitative data were analysed using descriptive statistical methods, and qualitative data were thematically analysed using normalisation process theory. Intervention and study delivery were evaluated against predefined progression criteria.</p><p><strong>Results: </strong>Of 156 eligible residents, 105 agreed to wear a device, with 102 (97%) starting the intervention. Of 225 eligible staff, 82.4% (<i>n</i> = 178) participated. Over 2 months, device loss and battery failure were significant: residents lost 11% of devices, with half replaced. Staff lost fewer devices, just 6.5%, but < 10% were replaced. Fob wearables needed more battery changes than card-type devices (15% vs. 0%). Homes variably understood structured and reactive feedback but were unlikely to act on it. Researcher support for interpreting reports was valued. Homes found information useful when it confirmed rather than challenged preconceived contact patterns. Staff privacy concerns were a barrier to adoption. Study procedures added to existing work, making participation burdensome. The perceived burden of participation, amplified by the pandemic context, outweighed the benefits. CONTACT did not meet its quantitative or qualitative progression criteria.</p><p><strong>Limitations: </strong>Researchers had to pragmatically adapt procedures, resulting in suboptimal implementation choices from an implementati","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-24"},"PeriodicalIF":3.5,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144019687","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 the best step-up treatments for children with uncontrolled asthma despite inhaled corticosteroids: the EINSTEIN systematic review, network meta-analysis and cost-effectiveness analysis using individual participant data.","authors":"Sofia Cividini, Ian Sinha, Giovanna Culeddu, Sarah Donegan, Michelle Maden, Katie Rose, Olivia Fulton, Dyfrig Hughes, Stephen Turner, Catrin Tudur Smith","doi":"10.3310/HGWT3617","DOIUrl":"10.3310/HGWT3617","url":null,"abstract":"<p><strong>Background: </strong>There is no clear preferential option for initial step-up of treatment for children with uncontrolled asthma on inhaled corticosteroid.</p><p><strong>Objectives: </strong>Evaluate the clinical effectiveness of pharmacological treatments to use in children with uncontrolled asthma on inhaled corticosteroid; identify and evaluate the potential for treatment effect modification to optimise treatment delivery; assess the cost-effectiveness of treatments.</p><p><strong>Methods: </strong>Systematic review and individual participant data network meta-analysis. Studies were eligible if they were parallel or crossover randomised controlled trials comparing at least one of the pharmacological treatments of interest in participants aged < 18 years with uncontrolled asthma on any dose inhaled corticosteroid alone. We searched MEDLINE<sup>®</sup>, Cochrane Library, Cochrane Central Register of Controlled Trials, EMBASE, National Institute for Health and Care Excellence Technology Appraisals, and the National Institute for Health and Care Research Health Technology Assessment series. Primary outcomes: exacerbation and asthma control. Secondary outcomes: health-related quality of life, mortality, forced expiratory volume in 1 second, adverse events, hospital admissions, symptoms (not analysed). We assessed the Risk Of Bias using the Cochrane Risk Of Bias tool and carried out Bayesian meta-analyses, network meta-analysis and network meta-regression, including treatment by covariate (age, sex, ethnicity, eczema, eosinophilia, asthma severity) interactions. A Markov decision-analytic model with a 12-month time horizon, which adopted the perspective of the National Health Service and Personal Social Services in the United Kingdom, was developed to compare alternative treatments. Cost-effectiveness was based on incremental costs per quality-adjusted life-years gained, with uncertainty considered in one-way, structural and probabilistic sensitivity analyses.</p><p><strong>Results: </strong>We identified and screened 4708 publications from the search and confirmed 144 randomised controlled trials as eligible. We obtained individual participant data from 29 trials (5381 participants) and extracted limited aggregate data from a further 19 trials. The majority of trials had low risk of bias. The network meta-analysis suggests that medium-dose inhaled corticosteroid + long-acting <i>β</i><sub>2</sub>-agonist is the preferred treatment for reducing odds of exacerbation [odds ratio 95% credibility interval: 0.43 (0.20 to 0.92) vs. low-dose inhaled corticosteroid; 40 studies, 8168 patients] and increasing forced expiratory volume in 1 second [mean difference 95% credibility interval: 0.71 (0.35 to 1.06) vs. low-dose inhaled corticosteroid; 23 studies, 2518 patients] while leukotriene receptor antagonist alone is the least preferred. No clear differences were found for asthma control (16 studies, 3027 patients). Limited pairwise analyses sugges","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 15","pages":"1-234"},"PeriodicalIF":3.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12104851/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144093426","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":"Ivacaftor-tezacaftor-elexacaftor, tezacaftor-ivacaftor and lumacaftor-ivacaftor for treating cystic fibrosis: a systematic review and economic evaluation.","authors":"Steven J Edwards, Benjamin G Farrar, Kate Ennis, Nicole Downes, Victoria Wakefield, Isaac Mackenzie, Archie Walters, Tracey Jhita","doi":"10.3310/CPLD8546","DOIUrl":"10.3310/CPLD8546","url":null,"abstract":"<p><strong>Background: </strong>Cystic fibrosis is a life-limiting genetic condition that affects over 9000 people in England. Cystic fibrosis is usually diagnosed through newborn screening and causes symptoms throughout the body, including the lungs and digestive system. Around 90% of individuals with cystic fibrosis have at least one copy of the <i>F508del</i> mutation on the cystic fibrosis transmembrane conductance regulator gene.</p><p><strong>Objectives: </strong>To appraise the clinical effectiveness and cost-effectiveness of elexacaftor-tezacaftor-ivacaftor, tezacaftor-ivacaftor and lumacaftor-ivacaftor within their expected marketing authorisations for treating people with cystic fibrosis and at least one <i>F508del</i> mutation, compared with each other and with established clinical management before these treatments.</p><p><strong>Methods: </strong>A de novo systematic literature review (search date February 2023) was conducted searching electronic databases (MEDLINE, EMBASE, Cochrane Central Register of Controlled Trials), bibliographies of relevant systematic literature reviews, clinical trial registers, recent conferences and evidence provided by Vertex Pharmaceuticals (Boston, MA, USA). Data on the following outcomes were summarised: acute change in per cent predicted forced expiratory volume in 1 second (change in weight-for-age <i>z</i>-score; and change in pulmonary exacerbation frequency requiring intravenous antibiotics. Network meta-analyses were conducted where head-to-head data were not available. Data from clinical trials and real-world evidence were examined to assess long-term effectiveness. A patient-level simulation model was developed to assess the cost-effectiveness of the three modulator treatments. The model employed a lifetime horizon and was developed from the perspective of the National Health Service.</p><p><strong>Results: </strong>Data from 19 primary studies and 7 open-label extension studies were prioritised in the systematic literature review. Elexacaftor/tezacaftor/ivacaftor was associated with a statistically significant increase in predicted forced expiratory volume in 1 second and weight-for-age <i>z</i>-score and a reduction in pulmonary exacerbations compared with established clinical management, lumacaftor/ivacaftor and tezacaftor/ivacaftor, and also led to a reduction in the rate of predicted forced expiratory volume in 1 second decline relative to established clinical management, although the magnitude of this decrease was uncertain. Lumacaftor/ivacaftor and tezacaftor/ivacaftor were also associated with a statistically significant increase in predicted forced expiratory volume in 1 second and reduction in pulmonary exacerbations relative to established clinical management, but with a smaller effect size than elexacaftor/tezacaftor/ivacaftor. There was some evidence that tezacaftor/ivacaftor reduced the rate of predicted forced expiratory volume in 1 second decline relative to established clinical","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 19","pages":"1-111"},"PeriodicalIF":3.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12127894/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144150156","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":"Behaviour change interventions to promote physical activity in people with intermittent claudication: the OPTIMA systematic review.","authors":"Ukachukwu O Abaraogu, Philippa Dall, Chris Seenan, Sarah Rhodes, Trish Gorely, Joanna McParland, Julie Brittenden, Ebuka M Anieto, Lorna Booth, Cathy Gormal, Jeremy Dearling, Candida Fenton, Sarah Audsley, Kimberley Fairer, Lindsay Bearne, Dawn A Skelton","doi":"10.3310/ZBNG5240","DOIUrl":"10.3310/ZBNG5240","url":null,"abstract":"<p><strong>Background: </strong>People with intermittent claudication are significantly less active compared to their peers without intermittent claudication, worsening future health outcomes. Supervised exercise therapy is not commonly available, but behaviour change techniques in unsupervised interventions can improve physical activity. Specific behaviour change techniques, theoretical mechanisms and contextual features linked to effectiveness remain unclear.</p><p><strong>Objectives: </strong>To conduct an integrative synthesis of: effectiveness of behaviour change technique-based interventions on daily physical activity and clinical-/patient-reported outcomes; behaviour change techniques and theoretical mechanisms within effective behaviour change technique-based interventions; feasibility and acceptability. Primary outcomes: short term (< 6 months) and maintenance (> 6 months) of daily physical activity. Secondary outcomes: clinical-/patient-reported outcomes.</p><p><strong>Data sources: </strong>Seven primary studies databases; Cochrane Database of Systematic Reviews, the Database of Abstracts of Reviews of Effects, Health Technology Assessment Database and Trial Registers to 31 August 2023.</p><p><strong>Review methods: </strong>Systematic review 1: interventions incorporating ≥ 1 behaviour change technique (coded using Behaviour Change Technique Taxonomy version 1, and Theoretical Domains Framework). Systematic review 2: quantitative, qualitative, mixed-methods research on patient/provider experiences. Study quality assessed using revised Cochrane risk-of-bias tool for randomised trials; Risk Of Bias In Non-randomised Studies - of Interventions and Mixed Methods Appraisal Tool.</p><p><strong>Results: </strong>Fifty-three articles (41 studies) were included in systematic review 1, and 28 articles (28 studies) in systematic review 2. Eleven randomised controlled trials demonstrated that behaviour change technique-based interventions increased daily physical activity in the short term [increase of 0.20 standardised mean difference (95% confidence interval 0.07 to 0.33), ~ 473 steps/day] with high certainty. Evidence of maintenance of daily physical activity is unclear (increase of 0.12 standardised mean difference; ~ 288 steps/day). Behaviour change techniques aimed at improving patients' intentions to engage in physical activity were most effective. Network analysis suggests that behaviour change technique-based interventions improved daily physical activity and may be better than supervised exercise therapy in maintaining daily physical activity. behaviour change technique-based interventions were acceptable and had short-medium-term benefits to initial/absolute claudication distance/time, walking impairment scores and disease-specific quality of life.</p><p><strong>Conclusions: </strong>The behaviour change technique-based interventions are effective, targeting intention to engage in physical activity, in improving daily physical activity","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 18","pages":"1-142"},"PeriodicalIF":3.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12127896/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144149931","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":"Bisoprolol for patients with chronic obstructive pulmonary disease at high risk of exacerbation: the BICS RCT.","authors":"Graham Devereux, Seonaidh Cotton, Mintu Nath, Nicola McMeekin, Karen Campbell, Rekha Chaudhuri, Gourab Choudhury, Anthony De Soyza, Shona Fielding, Simon Gompertz, John Haughney, Amanda Lee, Graeme MacLennan, Alyn Morice, John Norrie, David Price, Philip Short, Jorgen Vestbo, Paul Walker, Jadwiga Wedzicha, Andrew Wilson, Olivia Wu, Brian Lipworth","doi":"10.3310/TNDG8641","DOIUrl":"10.3310/TNDG8641","url":null,"abstract":"<p><strong>Background: </strong>Observational studies of people with chronic obstructive pulmonary disease using beta-blockers for cardiovascular disease indicate that beta-blocker use is associated with reduced risk of chronic obstructive pulmonary disease exacerbation. However, at the time this study was initiated, there had been no randomised controlled trials confirming or refuting this.</p><p><strong>Objective(s): </strong>To determine the clinical and cost-effectiveness of adding bisoprolol (maximal dose 5 mg once daily) to usual chronic obstructive pulmonary disease therapies in patients with chronic obstructive pulmonary disease at high risk of exacerbation.</p><p><strong>Design: </strong>A multicentre, pragmatic, double-blind, randomised, placebo-controlled clinical trial.</p><p><strong>Setting: </strong>Seventy-six United Kingdom primary and secondary care sites.</p><p><strong>Participants: </strong>People aged ≥ 40 years with a diagnosis of at least moderately severe chronic obstructive pulmonary disease with a history of at least two exacerbations in the previous year.</p><p><strong>Interventions: </strong>Participants were randomised (1 : 1) to receive either bisoprolol or placebo for 1 year. During a 4- to 7-week titration period, the maximum tolerated dose was established (1.25 mg, 2.5 mg, 3.75 mg, 5 mg once daily).</p><p><strong>Primary outcome: </strong>A number of participant-reported exacerbations during the 1-year treatment period.</p><p><strong>Results: </strong>In total, 519 participants were recruited and randomised. Four post-randomisation exclusions left 259 in the bisoprolol group and 256 in the placebo group. Treatment groups were balanced at baseline: mean (standard deviation) age 68 (7.9) years; 53% men; mean (standard deviation) pack year smoking history 45 (25.2); mean (standard deviation) 3.5 (1.9) exacerbations in previous year. Primary outcome data were available for 99.8% of participants (bisoprolol 259, placebo 255). The mean (standard deviation) number of exacerbations was 2.03 (1.91) in the bisoprolol group and 2.01 (1.75) in the placebo group (adjusted incidence rate ratio 0.97, 95% confidence interval 0.84 to 1.13), <i>p</i> = 0.72. The number of participants with serious adverse events was similar between the two groups (bisoprolol 37, placebo 36). The total number of adverse reactions was also similar between the two groups. As expected, bisoprolol was associated with a higher proportion of vascular adverse reactions (e.g. hypotension, cold peripheries) than placebo, but was not associated with an excess of other adverse reactions, including those classified as respiratory. Adding bisoprolol resulted in a statistically insignificant trend towards higher costs (£636, 95% confidence interval £118 to £1391) and fewer quality-adjusted life-years (0.035, 95% confidence interval 0.059 to 0.010) compared to placebo.</p><p><strong>Limitations: </strong>The study findings should be interpreted with caution as the ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 17","pages":"1-97"},"PeriodicalIF":3.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12107607/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144093428","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":"Pramipexole in addition to mood stabilisers for treatment-resistant bipolar depression: the PAX-BD randomised double-blind placebo-controlled trial.","authors":"Hamish McAllister-Williams, Nicola Goudie, Lumbini Azim, Victoria Bartle, Michael Berger, Chrissie Butcher, Thomas Chadwick, Emily Clare, Paul Courtney, Lyndsey Dixon, Nichola Duffelen, Tony Fouweather, William Gann, John Geddes, Sumeet Gupta, Beth Hall, Timea Helter, Paul Hindmarch, Eva-Maria Holstein, Ward Lawrence, Phil Mawson, Iain McKinnon, Adam Milne, Aisling Molloy, Abigail Moore, Richard Morriss, Anisha Nakulan, Judit Simon, Daniel Smith, Bryony Stokes-Crossley, Paul Stokes, Andrew Swain, Zoë Walmsley, Christopher Weetman, Allan H Young, Stuart Watson","doi":"10.3310/HBFC1953","DOIUrl":"10.3310/HBFC1953","url":null,"abstract":"<p><strong>Background: </strong>There are limited options currently recommended in National Institute for Health and Care Excellence guidelines for the treatment of bipolar depression. Pramipexole has been shown to improve mood symptoms in two small pilot studies in such patients.</p><p><strong>Objectives: </strong>Primary: to evaluate the clinical effectiveness of pramipexole versus placebo alongside routine mood-stabilising medications over 12 weeks in patients with treatment-resistant bipolar depression. Secondary: evaluate the impact of pramipexole on mood and anxiety, psychosocial function, cost-effectiveness, and safety and tolerability over 48 weeks.</p><p><strong>Design: </strong>Multicentre, randomised, placebo-controlled trial of pramipexole versus placebo in addition to standard-of-care mood stabilisers. Clinicians, researchers and participants were blinded throughout the duration of the study. Pre-randomisation stage (to adjust antipsychotics or commence mood stabilisers where required) before randomisation. Weekly online assessments of mood and anxiety from randomisation to week 52, with psychosocial function, quality of life and healthcare resource utilisation assessments conducted at regular intervals.</p><p><strong>Setting: </strong>Twenty-one National Health Service trusts and Health Boards across England and Scotland.</p><p><strong>Participants: </strong>Patients aged 18 years and over with a diagnosis of treatment-resistant bipolar depression currently under secondary care mental health services. Aim to randomise 290 participants.</p><p><strong>Interventions: </strong>Pramipexole or matched placebo orally once daily, titrated from 0.25 mg to maximum of 2.5 mg (salt weight) depending on efficacy and tolerability.</p><p><strong>Main outcome measures: </strong>Depression - Quick Inventory for Depressive Symptomology; anxiety - Generalised Anxiety Disorder-7-item scale; psychosocial functioning - Work and Social Adjustment Scale; hypomania/mania - Altman Self-rating Scale of Mania; tolerability - Treatment Satisfaction Questionnaire for Medication; well-being and quality of life - EuroQol-5 Dimensions, five-level version, ICEpop CAPability measure for Adults and Oxford CAPabilities questionnaire-Mental Health tools.</p><p><strong>Results: </strong>Thirty-nine participants randomised (18 to pramipexole and 21 to placebo) with 36 providing data for the primary analysis. Pramipexole led to greater reductions in depressive symptoms at 12 weeks compared to placebo [4.4 (4.8) vs. 2.1 (5.1)]: a medium-sized (<i>d</i> = -0.72) but not statistically significant difference (95% confidence interval -0.4 to 6.3; <i>p</i> = 0.087). There were some statistically significant positive effects of pramipexole on secondary outcomes (reduction in depressive symptoms at 36 weeks, response and remission rates at trial exit, psychosocial function). Pramipexole was associated with an increased rate of hypomania/manic symptoms, but this appeared to be redu","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 21","pages":"1-216"},"PeriodicalIF":3.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12146921/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144198956","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":"Anti-VEGF drugs compared with laser photocoagulation for the treatment of diabetic retinopathy: a systematic review and economic analysis.","authors":"Mark Simmonds, Matthew Walton, Rob Hodgson, Alexis Llewellyn, Ruth Walker, Helen Fulbright, Laura Bojke, Lesley Stewart, Sofia Dias, Thomas Rush, John Lawrenson, Tunde Peto, David Steel","doi":"10.3310/KRWP1264","DOIUrl":"10.3310/KRWP1264","url":null,"abstract":"<p><strong>Background: </strong>Diabetic retinopathy is a major cause of sight loss in people with diabetes, with a high risk of macular oedema, vitreous haemorrhage or other complications. Panretinal photocoagulation is the primary treatment for proliferative retinopathy. Anti-vascular endothelial growth factor drugs are used to treat various eye conditions and may be beneficial for people with proliferative or non-proliferative retinopathy.</p><p><strong>Methods: </strong>The Anti-VEGF In Diabetes project sought to investigate the clinical and cost-effectiveness of using anti-vascular endothelial growth factor to prevent retinopathy progression when compared to panretinal photocoagulation or no treatment. A systematic review with network meta-analysis of randomised controlled trials of anti-vascular endothelial growth factor (alone or in combination with panretinal photocoagulation) to treat retinopathy was conducted. The database searches were updated in May 2023. Individual participant data from larger trials were sought. A systematic review of non-randomised studies was performed. Existing cost-effectiveness analyses were reviewed, and a new economic model was developed, informed by the individual participant data meta-analysis. The model also estimated the value of undertaking further research to resolve decision uncertainty.</p><p><strong>Results: </strong>The review found that anti-vascular endothelial growth factors produced a slight, and not clinically meaningful, benefit over panretinal photocoagulation in best corrected visual acuity, after 1 year of follow-up in people with proliferative retinopathy (mean difference of 4.5 ETDRS letters; 95% credible interval -0.7 to 8.2). There was no evidence of a difference in effectiveness among the different anti-vascular endothelial growth factors. The benefit of anti-vascular endothelial growth factor appears to decline over time. Anti-vascular endothelial growth factor therapy may be more effective in people with poorer initial visual acuity. Anti-vascular endothelial growth factor had no impact on vision in people with non-proliferative retinopathy. Anti-vascular endothelial growth factor reduces rates of macular oedema and vitreous haemorrhage and may slow down the progression of retinopathy. Anti-vascular endothelial growth factors were predicted to be more costly but similarly effective to panretinal photocoagulation, with a net health benefit of -0.214 quality-adjusted life-years at a £20,000 willingness-to-pay threshold. Only under very select conditions might anti-vascular endothelial growth factors have the potential for cost-effectiveness to treat proliferative retinopathy. There is potentially significant value in reducing uncertainty through further primary research.</p><p><strong>Conclusions: </strong>Anti-vascular endothelial growth factor has no clinically meaningful benefit over panretinal photocoagulation for preserving visual acuity, but it may delay or prevent progression to","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-16"},"PeriodicalIF":3.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144019679","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":"Clinical and cost-effectiveness of detailed anomaly ultrasound screening in the first trimester: a mixed-methods study.","authors":"Jehan N Karim, Helen Campbell, Pranav Pandya, Edward C F Wilson, Zarko Alfirevic, Trish Chudleigh, Elizabeth Duff, Jane Fisher, Hilary Goodman, Lisa Hinton, Christos Ioannou, Edmund Juszczak, Louise Linsell, Heather L Longworth, Kypros H Nicolaides, Anne Rhodes, Gordon Smith, Basky Thilaganathan, Jim Thornton, Gillian Yaz, Oliver Rivero-Arias, Aris T Papageorghiou","doi":"10.3310/NLTP7102","DOIUrl":"10.3310/NLTP7102","url":null,"abstract":"<p><strong>Background: </strong>In the United Kingdom, pregnant women are offered two scans: at 11-14 and 18-20 weeks' gestation. Current guidance supports fetal anatomical screening at the second scan, but evidence suggests earlier detection is possible.</p><p><strong>Objectives: </strong>To determine clinical and cost-effectiveness of a detailed two-dimensional ultrasound scan in the first trimester for detection of fetal anomalies, in addition to usual practice.</p><p><strong>Design: </strong>Systematic review and meta-analysis. Nationwide survey. Analysis of National Congenital Anomaly Disease Registry data. Consensus procedure. Prospective survey of parental opinions. Probabilistic decision-analytic model for cost-effectiveness. Value-of-information analysis.</p><p><strong>Setting: </strong>United Kingdom National Health Service.</p><p><strong>Participants: </strong>Pregnant women and partners.</p><p><strong>Interventions: </strong>Detailed anomaly ultrasound at 11-14 weeks' gestation, in addition to usual practice.</p><p><strong>Main outcome measures: </strong>Diagnostic accuracy, protocol development, health economic modelling and value-of-information analysis.</p><p><strong>Data sources: </strong>MEDLINE (OvidSP), EMBASE (OvidSP), Science Citation Index and Conference Proceedings Citation Index-Science (Web of Science Core Collection); National Congenital Anomaly Disease Registry; European Congenital Anomalies Registry; Surveys of National Health Service Trusts; screening sonographers, midwives and doctors; and parents; National Schedule of National Health Service Costs (2019-20).</p><p><strong>Review methods: </strong>Systematic review and meta-analysis for diagnostic accuracy.</p><p><strong>Results: </strong>First-trimester ultrasound detects 93.3% (95% confidence interval 90.4% to 95.7%) of a pre-selected group of eight major anomalies with specificity of 99.99% (95% confidence interval 99.98% to 99.99%) and positive predictive value of 96.5% (95% confidence interval 93.3 to 98.8, 416,877 fetuses, 40 studies). For major cardiac anomalies, the respective data are 55.8% (95% confidence interval 45.9% to 65.5%), 99.98% (95% confidence interval 99.97% to 99.99%) and 94.85% (95% confidence interval 91.63% to 97.32%, 306,872 fetuses, 45 studies). Of NHS trusts surveyed, 77% currently perform first-trimester anatomy assessment, with evidence of inequity of care; earlier screening resulted in more diagnoses before 16 weeks' gestation. A consensus procedure (<i>n</i> = 172) developed an anatomical protocol and minimum targets for diagnosis. Parental survey (<i>n</i> = 1374) indicated that over 90% would opt for such screening. Modelling of singleton pregnancies undergoing earlier anomaly screening using two-dimensional ultrasound was associated with increased mean healthcare costs per woman (£11, 95% confidence interval £1 to £29) and maternal quality-adjusted life-years (0.002065, 95% confidence interval 0.000565 to 0.00358), an incremental co","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 22","pages":"1-250"},"PeriodicalIF":3.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12146947/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144198957","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":"Software with artificial intelligence-derived algorithms for detecting and analysing lung nodules in CT scans: systematic review and economic evaluation.","authors":"Julia Geppert, Peter Auguste, Asra Asgharzadeh, Hesam Ghiasvand, Mubarak Patel, Anna Brown, Surangi Jayakody, Emma Helm, Dan Todkill, Jason Madan, Chris Stinton, Daniel Gallacher, Sian Taylor-Phillips, Yen-Fu Chen","doi":"10.3310/JYTW8921","DOIUrl":"10.3310/JYTW8921","url":null,"abstract":"<p><strong>Background: </strong>Lung cancer is one of the most common types of cancer and the leading cause of cancer death in the United Kingdom. Artificial intelligence-based software has been developed to reduce the number of missed or misdiagnosed lung nodules on computed tomography images.</p><p><strong>Objective: </strong> To assess the accuracy, clinical effectiveness and cost-effectiveness of using  software with artificial intelligence-derived algorithms to assist in the detection and analysis of lung nodules in computed tomography scans of the chest compared with unassisted reading.</p><p><strong>Design: </strong>Systematic review and de novo cost-effectiveness analysis.</p><p><strong>Methods: </strong>Searches were undertaken from 2012 to January 2022. Company submissions were accepted until 31 August 2022. Study quality was assessed using the revised tool for the quality assessment of diagnostic accuracy studies (QUADAS-2), the extension to QUADAS-2 for assessing risk of bias in comparative accuracy studies (QUADAS-C) and the COnsensus-based Standards for the selection of health status Measurement INstruments (COSMIN) checklist. Outcomes were synthesised narratively. Two decision trees were used for cost-effectiveness: (1) a simple decision tree for the detection of actionable nodules and (2) a decision tree reflecting the full clinical pathways for people undergoing chest computed tomography scans. Models estimated incremental cost-effectiveness ratios, cost per correct detection of an actionable nodule, and cost per cancer detected and treated. We undertook scenario and sensitivity analyses.</p><p><strong>Results: </strong>Twenty-seven studies were included. All were rated as being at high risk of bias. Twenty-four of the included studies used retrospective data sets. Seventeen compared readers with and without artificial intelligence software. One reported prospective screening experiences before and after artificial intelligence software implementation. The remaining studies either evaluated stand-alone artificial intelligence or provided only non-comparative evidence. (1) Artificial intelligence assistance generally improved the detection of any nodules compared with unaided reading (three studies; average per-person sensitivity 0.43-0.68 for unaided and 0.79-0.99 for artificial intelligence-assisted reading), with similar or lower specificity (three studies; 0.77-1.00 for unaided and 0.81-0.97 for artificial intelligence-assisted reading). Nodule diameters were similar or significantly larger with semiautomatic measurements than with manual measurements. Intra-reader and inter-reader agreement in nodule size measurement and in risk classification generally improved with artificial intelligence assistance or were comparable to those with unaided reading. However, the effect on measurement accuracy is unclear. (2) Radiologist reading time generally decreased with artificial intelligence assistance in research settings. (3) Artific","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 14","pages":"1-234"},"PeriodicalIF":3.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12104856/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144086170","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}