Health technology assessment最新文献

{"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":"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":"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":"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":"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":"2025-05-01","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":"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":"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":"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}

{"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":"Rapid tests to inform triage and antibiotic prescribing decisions for adults presenting with suspected acute respiratory infection: a rapid evidence synthesis of clinical effectiveness and cost-utility studies.","authors":"Katie Scandrett, Jill Colquitt, Rachel Court, Fiona Whiter, Bethany Shinkins, Yemisi Takwoingi, Emma Loveman, Daniel Todkill, Paramjit Gill, Daniel Lasserson, Lena Al-Khudairy, Amy Grove, Yen-Fu Chen","doi":"10.3310/KHGP7129","DOIUrl":"10.3310/KHGP7129","url":null,"abstract":"<p><strong>Background: </strong>This review assessed the clinical- and cost-effectiveness of point-of-care tests to guide the initial management of people presenting with suspected acute respiratory infection.</p><p><strong>Methods: </strong>Searches for systematic reviews, randomised controlled trials and cost-utility studies were conducted in May 2023. Sources included MEDLINE, Epistemonikos, EMBASE, Cochrane Central Register of Controlled Trials, the Cost-effectiveness Analysis Registry and reference checking. Eligible studies included people (≥ 16 years) making initial contact with the health system with symptoms suggestive of acute respiratory infection. Risk of bias in randomised controlled trials was assessed using the Cochrane risk-of-bias tool. The Drummond checklist was used for cost-utility studies. Meta-analyses of clinical outcomes were conducted to estimate summary risk ratios with 95% confidence intervals. Study characteristics and main results were summarised narratively and tabulated.</p><p><strong>Results: </strong>Fourteen randomised controlled trials were included; all had a high risk of bias. Ten randomised controlled trials analysed point-of-care tests for C-reactive protein. Compared with usual care, the effects on hospital admissions and mortality were highly uncertain due to sparse data. Three randomised controlled trials had heterogeneous findings on the resolution of symptoms/time to full recovery. The risk of re-consultations increased in patients receiving C-reactive protein point-of-care tests (pooled risk ratio 1.61, 95% confidence interval 1.07 to 2.41; four studies). There was a reduction in antibiotics initially prescribed (C-reactive protein point-of-care tests vs. usual care: pooled risk ratio 0.75, 95% confidence interval 0.68 to 0.84; nine studies). The effects of procalcitonin point-of-care tests compared with usual care on hospital admission, escalation of care, and duration of symptoms were very uncertain as only one randomised controlled trial was included. The study found a large reduction in antibiotic prescriptions within 7 days. Two studies revealed a large reduction in initial antibiotic prescriptions for Group A streptococcus point-of-care tests versus usual care. Only one study compared an influenza point-of-care test with usual care. The effect of the antibiotics prescribed was very uncertain. No deaths occurred in either treatment group.</p><p><strong>Cost-effectiveness: </strong>Six of the 17 included cost-utility studies were judged to be directly applicable to our review, 4 of which focused on the C-reactive protein point-of-care test. The results suggested that the C-reactive protein point-of-care test is potentially cost-effective; these studies were generally limited to capturing only short-term costs and consequences. One study evaluated 14 different point-of-care tests for Group A streptococcus; none were cost-effective compared with usual care. A further study evaluated two rapid tests (","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 13","pages":"1-114"},"PeriodicalIF":3.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12104858/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144086168","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":"Effects of pharmacological and non-pharmacological interventions for the management of sleep problems in people with fibromyalgia: a multi-methods evidence synthesis.","authors":"Mari Imamura, Clare Robertson, Jemma Hudson, Daniel Whibley, Lorna Aucott, Katie Gillies, Marcus Beasley, Martin J Stevens, Paul Manson, Debra Dulake, Abhishek Abhishek, Nicole Ky Tang, Gary J Macfarlane, Miriam Brazzelli","doi":"10.3310/GTBR7561","DOIUrl":"10.3310/GTBR7561","url":null,"abstract":"<p><strong>Background: </strong>Fibromyalgia is a chronic condition characterised by widespread musculoskeletal pain. Sleep problems are reported by 92% of people living with fibromyalgia.</p><p><strong>Objectives: </strong>To evaluate the effectiveness and safety of interventions for the management of fibromyalgia-related sleep problems; explore the experiences of people with fibromyalgia-related sleep problems and examine the content of patient-reported outcome measures for 'sleep quality'.</p><p><strong>Methods: </strong>We conducted: (1) a network meta-analysis of randomised controlled trials to compare the effectiveness of pharmacological and non-pharmacological interventions; (2) a systematic thematic synthesis of qualitative evidence; (3) a content analysis of existing patient-reported outcome measures validated in people with fibromyalgia. Major electronic databases were searched in November 2021.</p><p><strong>Results: </strong>One hundred and sixty-eight studies were included in the effectiveness synthesis. The network meta-analysis assessing sleep quality included 35 treatment categories from 65 studies (8247 participants). Most studies were at high overall risk of bias. There is some evidence that compared with placebo or sham treatments, some forms of exercise [i.e. land-based aerobic exercise training in combination with flexibility training (standardised mean difference -4.69, credible interval -8.14 to -1.28) and aquatic-based aerobic exercise training (standardised mean difference -2.63, credible interval -4.74 to -0.58)] may improve sleep. There is also a suggestion that land-based strengthening exercise, psychological and behavioural therapies with a focus on sleep, electrotherapy, weight loss, dental splints, antipsychotics and tricyclics may have a modest effect on sleep, but credible intervals are wide. For other interventions, there is no clear evidence of beneficial effects on sleep. Our certainty of current evidence was predominantly low to very low. The thematic synthesis highlighted the bidirectional relationship between sleep and pain. Twenty-one sleep domains were identified across five patient-reported outcome measures. The domain most frequently identified was sleep maintenance. The Pittsburgh Sleep Quality Index was the most comprehensive tool (15 domains), followed by the Medical Outcomes Study Sleep Scale (11 domains).</p><p><strong>Limitations: </strong>Quantitative studies varied considerably in terms of characteristics of interventions, control treatments and type of outcome measures. In the network, most interventions were compared with placebo, sham treatment or usual care and not with another active treatment. In general, studies were small, unblinded and of short duration (median 12 weeks). For the qualitative synthesis and patient-reported outcome measures analysis, it is unclear whether study participants are adequately representative of the wider population of fibromyalgia patients due to poor reporting","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 20","pages":"1-228"},"PeriodicalIF":3.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12146952/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144198955","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}