Health technology assessment最新文献

{"title":"Identifying optimal primary prevention interventions for major cardiovascular disease events and all-cause mortality: a systematic review and hierarchical network meta-analysis of RCTs.","authors":"Olalekan A Uthman, Rachel Court, Jodie Enderby, Chidozie Nduka, Lena Al-Khudairy, Seun Anjorin, Hema Mistry, G J Melendez-Torres, Sian Taylor-Phillips, Aileen Clarke","doi":"10.3310/RLDH7432","DOIUrl":"https://doi.org/10.3310/RLDH7432","url":null,"abstract":"<p><strong>Background: </strong>Cardiovascular disease accounts for substantial mortality and healthcare costs worldwide. Numerous interventions exist for primary prevention but lack head-to-head comparisons on long-term impacts.</p><p><strong>Objective: </strong>To determine the comparative effectiveness of interventions for primary cardiovascular disease prevention through network meta-analysis of randomised trials.</p><p><strong>Data sources: </strong>MEDLINE, EMBASE, Cochrane Central Register of Controlled Trials, conference abstracts and trial registries from inception to March 2021.</p><p><strong>Review methods: </strong>Randomised controlled trials of pharmacologic therapies, nutritional supplements, lifestyle changes, behavioural approaches and health policies with at least 6 months' follow-up were included. Pairwise and network meta-analyses were conducted for all-cause mortality, cardiovascular disease events, coronary heart disease and cardiovascular disease mortality.</p><p><strong>Results: </strong>Data from 139 randomised trials, including 1,053,772 participants, proved suitable for quantitative synthesis. Blood pressure-lowering medications (risk ratio 0.82, 95% confidence interval 0.71 to 0.94), tight blood pressure control (risk ratio 0.66, 95% confidence interval 0.46 to 0.96), statins (risk ratio 0.81, 95% confidence interval 0.71 to 0.91) and multifactorial lifestyle interventions (risk ratio 0.75, 95% confidence interval 0.61 to 0.92) reduced composite cardiovascular events and mortality.</p><p><strong>Limitations: </strong>Residual confounding may exist. Few direct head-to-head comparisons limited differentiation between some specific modalities.</p><p><strong>Conclusions: </strong>We found evidence that blood pressure treatments, intense blood pressure targets, statins when appropriate and multifactorial lifestyle changes are the most effective strategies for primary prevention of cardiovascular disease, with unclear effects from other interventions. These findings can inform clinical guidelines and health policies prioritising interventions.</p><p><strong>Funding: </strong>This research article presents independent research funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme as award number 17/148/05.</p>","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-65"},"PeriodicalIF":3.5,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144560003","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":"Ambulatory Oxygen for Pulmonary Fibrosis (OxyPuF): a randomised controlled trial and acceptability study.","authors":"Rachel L Adams, Alisha Maher, Nicola Gale, Anjali Crawshaw, David Thickett, Alice M Turner","doi":"10.3310/TWKS4194","DOIUrl":"https://doi.org/10.3310/TWKS4194","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Introduction: &lt;/strong&gt;Idiopathic pulmonary fibrosis is a devastating condition of unknown cause that results in progressive, irreversible scarring of the lung, manifesting as breathlessness and dry cough. Idiopathic pulmonary fibrosis is thought to be responsible for as many as 1 in 100 deaths in the United Kingdom, killing 5300 people a year. Ambulatory oxygen therapy is commonly used in idiopathic pulmonary fibrosis to relieve exertional breathlessness, although evidence to support this strategy is lacking. This pragmatic randomised controlled trial was planned to test whether use of ambulatory oxygen therapy is beneficial in people with idiopathic pulmonary fibrosis.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We planned a randomised controlled trial in 260 patients with idiopathic pulmonary fibrosis who are breathless on exertion and do not meet criteria for long-term oxygen therapy, randomising in a 1 : 1 ratio between ambulatory oxygen therapy and best supportive care. Primary outcome was a quality-of-life questionnaire validated in pulmonary fibrosis, the King's Brief Interstitial Lung Disease questionnaire, measured at 6 months. We calculated our sample size based on the minimum clinically important difference of four units and standard deviation equal to 8.85 in King's Brief Interstitial Lung Disease questionnaire; assuming power of 90% and 5% two-sided significance level, thus required 130 per arm, after accounting for 20% dropout. The trials unit's web-based randomisation algorithm minimises on factors potentially influencing response to ambulatory oxygen therapy, such as severity of idiopathic pulmonary fibrosis, desaturation to &lt; 88% present on walking, current or recent (within 6 months) pulmonary rehabilitation, and recruitment centre. Secondary outcomes included symptoms, exercise capacity and cost-effectiveness. A process evaluation included assessment of trial fidelity and acceptability of the intervention with use of qualitative research methods and arts approaches with patients and staff. Qualitative interviews were conducted with patients from the Ambulatory Oxygen for Pulmonary Fibrosis trial and the idiopathic pulmonary fibrosis patient support group Action for Pulmonary Fibrosis, and stakeholders: healthcare professionals and policy-makers. Interviews were audio-recorded, transcribed clean verbatim. Photovoice methodology was conducted with patients. A workshop prior to data collection informed and guided data collection and analysis. Traditional qualitative analysis and arts-based coproduction analysis approaches were used to produce a short film. An economic model was planned but could not occur due to early termination.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The trial was stopped prematurely due to low recruitment. This was due to a combination of the impact of COVID-19 on research infrastructure, financial issues for sites with the payment structure for the trial and lack of equipoise which limited site recruitment. Seven out","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-33"},"PeriodicalIF":3.5,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144560001","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":"Challenges to overcome in a randomised trial for Proper Understanding of Recurrent Stress Urinary Incontinence Treatment in women: the PURSUIT RCT.","authors":"Caroline Pope, Madeleine Cochrane, Clare Clement, Yumeng Liu, Sangeetha Paramasivan, Sian Noble, Stephanie J MacNeill, Amanda L Lewis, Jodi Taylor, Bethanie Fitzgerald, Nikki Cotterill, Tamsin Greenwell, Hashim Hashim, Swati Jha, Nikesh Thiruchelvam, Philip Toozs-Hobson, Alison White, Wael Agur, J Athene Lane, Marcus Drake","doi":"10.3310/AKAK8992","DOIUrl":"https://doi.org/10.3310/AKAK8992","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Recurrence or persistence of symptoms after interventions to treat stress urinary incontinence in women is common, but without robust evidence to base treatment recommendations.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;To investigate whether endoscopic or surgical treatments for stress urinary incontinence in women are effective and cost-effective.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;A multicentre, unblinded, parallel-group randomised controlled trial.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Fifteen centres across the United Kingdom.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Adult women with recurrent or persistent stress urinary incontinence.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Intervention: &lt;/strong&gt;Individual randomisation to endoscopic (urethral bulking) or surgical (autologous sling, colposuspension, artificial urinary sphincter) interventions. Women randomised to surgery chose their operative intervention.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcomes: &lt;/strong&gt;Primary outcome self-reported International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form at 1 year post randomisation. Secondary outcomes included International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form, Patient Global Impression of Improvement and Pelvic Organ Prolapse/Urinary Incontinence Sexual questionnaires up to 3 years post randomisation, operative assessment measures and adverse events, cost-effectiveness from National Health Service and societal perspectives (quality-adjusted life-years and International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form) at 1 year, and a secondary care perspective (quality-adjusted life-years) at 3 years. Semistructured qualitative interviews at baseline (post randomisation), follow-up (3-6 months) and longer-term (12 and 36 months), to explore stress urinary incontinence generally, the acceptability and attitudes to treatments and to improve understanding of outcomes. Qualitative interviews with clinicians at baseline were focused on potential difficulties of recruitment and optimising patient-facing information and training materials for clinicians.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Fifty-five women were deemed eligible after screening (&lt;i&gt;n&lt;/i&gt; = 328 screened) from October 2019 to June 2022. Twenty-four eligible women consented, and 23 were randomised (between January 2020 and July 2022) from 8 sites with the average age of 57 years (standard deviation: 10.7) and all self-reported 'white' ethnicity. Participants reported a median International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form score at baseline of 16 (interquartile range: 13-19) and mean post-void residual volume of 4.64 ml (standard deviation: 8.45). Eleven participants received their allocated intervention, 2 participants withdrew prior to receiving their intervention and 10 were waiting for their intervention when the study closed. The most common reason for declining participation was a trea","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-43"},"PeriodicalIF":3.5,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144560002","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":"Melatonin versus midazolam in the premedication of anxious children attending for elective surgery under general anaesthesia: the MAGIC non-inferiority RCT.","authors":"Christopher Deery, Robert Bolt, Diana Papaioannou, Matthew Wilson, Marie Hyslop, Esther Herbert, Nikki Totton, Zoe Marshman, Tracey Young, Jennifer Kettle, Sondos Albadri, Simon Atkins, Katie Biggs, Janet Clarkson, Chris Evans, Laura Flight, Jacqui Gath, Fiona Gilchrist, Kate Hutchence, Nicholas Ireland, Amanda Loban, Amy Norrington, Hamish Paton, Jaydip Ray, Helen Rodd, Elena Sheldon, Richard Simmonds, Christopher Vernazza","doi":"10.3310/CWKF1987","DOIUrl":"10.3310/CWKF1987","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Anxiety in children prior to general anaesthesia is common, with up to half displaying distress. Anxiety and distress may lead to unsuccessful anaesthesia, together with greater postoperative pain, agitation and behavioural changes after surgery including sleep disturbances. Midazolam is the current standard premedication; however, it has adverse effects such as the potential for respiratory suppression and unpredictable effects which may result in agitation rather than anxiolysis. Melatonin is an alternative preoperative anxiolytic; however, previous trials have delivered conflicting results. The aim of this non-inferiority trial was to evaluate the effectiveness of melatonin compared to midazolam in reducing anxiety in children undergoing general anaesthesia.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We undertook a randomised-controlled, parallel-group, double-blind, non-inferiority trial in 20 United Kingdom National Health Service trusts, with an embedded qualitative study and health economic evaluation. Anxious children having day case elective surgery under general anaesthesia were randomly assigned to either control (standard of care) group: midazolam; or intervention group: melatonin. The primary outcome was preoperative distress (non-inferiority hypothesis) as assessed by modified Yale Preoperative Anxiety Scale Short Form. Secondary outcomes included safety and efficacy objectives. Analyses were by intention to treat, with an additional per-protocol analysis. The sample size of the trial was 624 children.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The trial was stopped early due to recruitment futility. Between 30 July 2019 and 9 November 2022, 110 children were recruited; 55 allocated to midazolam and 55 allocated to melatonin. Pre-planned analyses showed an adjusted mean difference of 13.1 (95% confidence interval 3.7 to 22.4) for the intention-to-treat population and 12.9 (95% confidence interval 3.1 to 22.6) for the per-protocol population, in favour of midazolam. In both analyses, the upper limit of the 95% confidence interval exceeds the predefined margin of 4.3; therefore, melatonin is not non-inferior to midazolam. The lower limit of the 95% confidence intervals excludes zero and thus melatonin is inferior to midazolam; the difference found is considered to be clinically meaningful. Adverse events in the midazolam arm (26%) were slightly higher than melatonin (18%); there were no serious adverse events in either arm. Challenges to recruitment included study-related factors (eligibility criteria and trial design), participant factors (caregiver stress on the day of treatment) and practitioner factors (valuing predictability). In terms of acceptability, preferences of the anaesthetist, patient and caregiver factors and medication side effects profile were influential and suggest the choice of preoperative anxiolytic is more complex than previously described. On average, costs over the 14 days post surgery were lower for","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 29","pages":"1-25"},"PeriodicalIF":3.5,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144575364","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":"Invasive urodynamic investigations in the management of women with refractory overactive bladder symptoms: FUTURE, a superiority RCT and economic evaluation.","authors":"Mohamed Abdel-Fattah, Christopher Chapple, Suzanne Breeman, David Cooper, Helen Bell-Gorrod, Preksha Kuppanda, Karen Guerrero, Simon Dixon, Nikki Cotterill, Karen Ward, Hashim Hashim, Ash Monga, Karen Brown, Marcus Drake, Andrew Gammie, Alyaa Mostafa, Rebecca Bruce, Victoria Bell, Christine Kennedy, Suzanne Evans, Graeme MacLennan, John Norrie","doi":"10.3310/UKYW4923","DOIUrl":"10.3310/UKYW4923","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Overactive bladder is a common problem affecting the United Kingdom adult female population. Symptoms include urinary urgency, with or without urgency incontinence, increased daytime urinary frequency and nocturia. Initial conservative treatments for overactive bladder are unsuccessful in 25-40% of women (refractory overactive bladder). Before considering invasive treatments, such as botulinum toxin injection-A or sacral neuromodulation, guidelines recommend urodynamics to confirm diagnosis of detrusor overactivity. However, the clinical and cost effectiveness of urodynamics has never been robustly assessed.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;To compare the clinical and cost effectiveness of urodynamics plus comprehensive clinical assessment versus comprehensive clinical assessment only in the management of refractory overactive bladder in women.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;Parallel-group, multicentre, superiority, open-label, randomised controlled trial. Allocation by remote web-based randomisation (1 : 1 ratio). The cost-effectiveness analysis took the National Health Service perspective with a model-based lifetime time horizon, as informed by a within-trial analysis.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Sixty-three United Kingdom secondary and tertiary hospitals.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Women aged ≥ 18 years with refractory overactive bladder or urgency-predominant mixed urinary incontinence who had failed conservative management and pharmacological treatment and were being considered for invasive treatment. Women were excluded if any of the following criteria were met: predominant stress urinary incontinence; previous urodynamics in last 12 months; current pelvic malignancy or clinically significant pelvic mass; bladder pain syndrome; neurogenic bladder; urogenital fistulae; previous treatment with botulinum toxin injection-A or sacral neuromodulation for urinary incontinence; previous pelvic radiotherapy; prolapse beyond introitus; pregnant or planning pregnancy; recurrent urinary tract infection where a significant pathology has not been excluded; and inability to give an informed consent.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Urodynamics plus comprehensive clinical assessment (urodynamics arm) versus comprehensive clinical assessment only.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;Participant-reported success at the last follow-up time point as measured by the Patient Global Impression of Improvement. Primary economic outcome was incremental cost per quality-adjusted life-year gained as modelled over the lifetime of participants.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;A total of 1099 participants were included: 550 randomised to the urodynamics arm and 549 to the comprehensive clinical assessment only arm. At the final follow-up time point, participant-reported success rates of 'very much improved' and 'much improved' were not superior in the urodynamics arm (117 participants; 23.6%) compared to the ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 27","pages":"1-139"},"PeriodicalIF":3.5,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144575363","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":"Variation within and between digital pathology and light microscopy for the diagnosis of histopathology slides: blinded crossover comparison study.","authors":"David Rj Snead, Ayesha S Azam, Jenny Thirlwall, Peter Kimani, Louise Hiller, Adam Bickers, Clinton Boyd, David Boyle, David Clark, Ian Ellis, Kishore Gopalakrishnan, Mohammad Ilyas, Paul Kelly, Maurice Loughrey, Desley Neil, Emad Rakha, Ian Sd Roberts, Shatrughan Sah, Maria Soares, YeeWah Tsang, Manuel Salto-Tellez, Helen Higgins, Donna Howe, Abigail Takyi, Yan Chen, Agnieszka Ignatowicz, Jason Madan, Henry Nwankwo, George Partridge, Janet Dunn","doi":"10.3310/SPLK4325","DOIUrl":"10.3310/SPLK4325","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Digital pathology refers to the conversion of histopathology slides to digital image files for examination on computer workstations as opposed to conventional microscopes. Prior to adoption, it is important to demonstrate pathologists provide equivalent reports when using digital pathology in comparison to bright-field and immunofluorescent light microscopy, the current standard of care.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;A multicentre comparison of digital pathology with light microscopy for reporting of histopathology slides, measuring variation within and between pathologists on both modalities.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;A blinded crossover 2000-case study estimating clinical management concordance (identical diagnoses plus differences not affecting patient management). Each sample was assessed twice by four pathologists (once using light microscopy, once using digital pathology, the order randomly assigned and a 6-week gap between viewings). Random-effects logistic regression models, including crossed random-effects terms for case and pathologist, estimated percentage clinical management concordance. Findings were interpreted with reference to 98.3% concordance (Azam AS, Miligy IM, Kimani PKU, Maqbool H, Hewitt K, Rajpoot NM, Snead DRJ. Diagnostic concordance and discordance in digital pathology: a systematic review and meta-analysis. &lt;i&gt;J Clin Pathol&lt;/i&gt; 2021;&lt;b&gt;74&lt;/b&gt;:448-55. https://doi.org/10.1136/jclinpath-2020-206764).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Sixteen consultant pathologists, four for each specialty, from six National Health Service laboratories. Experience ranged from 3 to 35 years. Some were early adopters of digital pathology, but the majority were new to digital pathology.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Eight viewings per sample (four pathologists with light microscopy and with digital pathology), culminating in a consensus ground truth, enabling measurement of agreement within and between readers. Samples enrolled reflected routine practice, included cancer screening biopsies, and were enriched for areas of difficulty [e.g. dysplasia (7, 10, 11)]. State-of-the-art digital pathology equipment designed for diagnosis, and holding either Conformité Européene or Food and Drug Administration approval, was used.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome: &lt;/strong&gt;Intra-pathologist variation between reports issued on digital pathology and light microscopy, inter-pathologist variation against ground-truth diagnosis using light microscopy and digital pathology.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Secondary outcomes: &lt;/strong&gt;Pathologist-recorded reporting times, along with their confidence in diagnosis, analysis of eye-tracking evaluating examination techniques, and a qualitative study examining attitudes of pathologists and laboratory staff to digital pathology adoption.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Two thousand and twenty-four cases (608 breast, 607 gastrointestinal, 609 skin, 200 renal) were recruited, with breast and gast","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 30","pages":"1-75"},"PeriodicalIF":3.5,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144626064","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":"The risks, benefits, and resource implications of different diets in gastrostomy-fed children: The YourTube mixed method study.","authors":"Lorna Fraser, Andre Bedendo, Mark O'Neill, Johanna Taylor, Julia Hackett, Karen Horridge, Janet Cade, Gerry Richardson, Thai Han Phung, Bryony Beresford, Alison McCarter, Catherine Hewitt","doi":"10.3310/RRREF7741","DOIUrl":"https://doi.org/10.3310/RRREF7741","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Many children receive some or all their nutritional intake via a gastrostomy. More parents are using home-blended meals to feed their children, reporting beneficial effects, such as improved gastro-oesophageal reflux and less distress.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Aim: &lt;/strong&gt;To compare safety, outcomes and resource use of those on home-blended diets compared to formula diets.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;A mixed-methods study of gastrostomy-fed children.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Workstream 1: &lt;/strong&gt;Qualitative study involving semistructured interviews with parents (&lt;i&gt;n &lt;/i&gt;≈ 20) and young people (&lt;i&gt;n &lt;/i&gt;≈ 2) and focus groups with health professionals (&lt;i&gt;n &lt;/i&gt;≈ 41).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Workstream 2: &lt;/strong&gt;Cohort study; data were collected on 180 children at months 0, 12 and 18 from parents and clinicians using standardised measures. Data included gastrointestinal symptoms, quality of life, sleep (child and parent), dietary intake, anthropometry, healthcare usage, safety outcomes and resource use. Outcomes were compared using propensity scored weighted multiple regression analyses.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: workstream 1: &lt;/strong&gt;Participants believed the type of diet would most likely affect gastrointestinal symptoms, time spent on feeding, sleep and physical health.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Workstream 2: &lt;/strong&gt;&lt;b&gt;Baseline&lt;/b&gt;: Children receiving a home-blended diet and those receiving a formula diet were similar in terms of diagnoses and age, but those receiving a home-blended diet were more likely to live in areas of lower deprivation and their parents had higher levels of education. They also had a higher dietary fibre intake and demonstrated significantly better gastrointestinal symptom scores compared to those receiving a formula diet (beta 13.8, &lt;i&gt;p&lt;/i&gt; &lt; 0.001). The number of gut infections and tube blockages were similar between the two groups, but stoma site infections were lower in those receiving a home-blended diet. &lt;b&gt;Follow-up&lt;/b&gt;: There were 134 (74%) and 105 (58%) children who provided follow-up data at 12 and 18 months. Gastrointestinal symptoms were lower at all time points in the home-blended diet group, but there was no difference in change over time within or between the groups. The nutritional intake of those on a home-blended diet had higher calories/kg and fibre, and both home-blended and formula-fed children have values above the Dietary Reference Values for most micronutrients. Safety outcomes were similar between groups and over time. Total costs to the statutory sector were higher among children who were formula fed, but costs of purchasing special equipment for home-blended food and the total time spent on child care were higher for families with home-blended diet.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;Findings show that home-blended diets for children who are gastrostomy fed should be seen as a safe alternative to formula feeding for children unless there is a clinical contraindication.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Limitations: ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 25","pages":"1-21"},"PeriodicalIF":3.5,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144575361","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":"Urodynamics tests for the diagnosis and management of male bladder outlet obstruction: long-term follow-up of the UPSTREAM non-inferiority RCT.","authors":"Madeleine Clout, Amanda L Lewis, Madeleine Cochrane, Grace J Young, Paul Abrams, Peter S Blair, Christopher Chapple, Gordon T Taylor, Sian Noble, Tom Steuart-Feilding, Jodi Taylor, J Athene Lane, Marcus J Drake","doi":"10.3310/SLPT4675","DOIUrl":"10.3310/SLPT4675","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Lower urinary tract symptoms are common in older men and can be bothersome, leading to treatment. The UPSTREAM randomised controlled trial (Phase I) investigated whether assessment of these symptoms with invasive urodynamic testing could improve symptoms when guiding treatment options.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To assess the long-term lower urinary tract symptoms and the rates of surgery for bladder outlet obstruction in men participating in the UPSTREAM study (Phase I).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;Pragmatic, multicentre, parallel-group, two-group open randomised controlled study, with outcome assessors blinded to aggregate data.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Urology departments of 26 National Health Service hospitals in England.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Men ≥ 18 years, seeking further treatment for their bothersome lower urinary tract symptoms, which may include surgery, who were existing participants of the UPSTREAM study (Phase I). Men were excluded if they were unable to pass urine without a catheter, had a relevant neurological disease, were currently undergoing treatment for prostate or bladder cancer, had previous prostate surgery or were unfit for surgery.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Routine care plus invasive urodynamics (intervention) or non-invasive routine care.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;The primary outcome was a patient-reported International Prostate Symptom Score 5 years post randomisation. Rates of surgery was the key secondary outcome. Patient-reported outcomes included measures of lower urinary tract symptoms, sexual function, overall quality of life and cost-effectiveness from a secondary care perspective.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Data sources: &lt;/strong&gt;Questionnaires to participants for patient-reported outcome measures, and National Health Service England Hospital Episode Statistics and mortality data.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Of 820 men randomised in UPSTREAM (Phase I) between October 2014 and December 2016, 211/427 men randomised to the intervention group completed Phase II questionnaires (49.4%) and 205/363 in the routine care group (56.5%). There was no difference found between International Prostate Symptom Scores in the two groups at 5 years (adjusted difference 0.41, 95% confidence interval -1.10 to 1.93). There was also no difference in other lower urinary tract symptoms, sexual function or quality of life. Routine data were received for 98% of men. Three hundred and forty-seven (43.3%) men with routine data available had received at least one related surgical procedure for the treatment of lower urinary tract symptoms. Over the 5-year time horizon, incremental mean costs were slightly higher (£176.63, 95% confidence interval -£464.06 to £817.32) in the intervention group and incremental mean QALYs were slightly lower (-0.039, 95% confidence interval -0.152 to 0.073) in the intervention group. This suggests that routine care is the cost","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 26","pages":"1-57"},"PeriodicalIF":3.5,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144575362","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":"The quantity, quality and findings of network meta-analyses evaluating the effectiveness of GLP-1 RAs for weight loss: a scoping review.","authors":"Michael Nunns, Samantha Febrey, Jill Buckland, Rebecca Abbott, Rebecca Whear, Alison Bethel, Kate Boddy, Liz Shaw, Jo Thompson Coon, G J Melendez-Torres","doi":"10.3310/SKHT8119","DOIUrl":"https://doi.org/10.3310/SKHT8119","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Glucagon-like peptide 1 receptor agonists are a class of drug originally developed to treat type 2 diabetes but now increasingly used for weight loss, especially in people living with obesity. Despite an abundance of evidence about the effectiveness and safety of glucagon-like peptide 1 receptor agonists for weight loss, network meta-analyses are inconsistent in their quality and scope, and this is a fast-moving field.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;We sought to identify the most recent network meta-analyses evaluating the effectiveness of glucagon-like peptide 1 receptor agonists for weight loss; critically appraise included network meta-analyses; provide an overview of the quality and findings of existing network meta-analyses, and identify any pertinent gaps in the evidence; and consider the value of updating the most recent, comprehensive and high-quality network meta-analyses.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;On 6 June 2023, we searched MEDLINE, EMBASE, the Cochrane Database of Systematic Reviews and Epistemonikos for systematic reviews with network meta-analyses published since 2020 in adults (18 or above) with body mass index ≥ 25 (or ≥ 23 for Asian populations), including at least one relevant glucagon-like peptide 1 receptor agonist and weight loss outcomes. We screened and selected reviews in duplicate and independently, and appraised reviews using a modified A MeaSurement Tool to Assess systematic Reviews 2 (AMSTAR-2) and a network meta-analysis reliability checklist. The highest-quality reviews were then extracted in depth, and the most relevant network meta-analysis models identified, focusing on weight loss and safety outcomes. A top-up search for trials published since October 2022 was also undertaken to identify relevant trials not included in published network meta-analyses. A further search for new network meta-analyses was conducted on 26 September 2024.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Of 22 systematic reviews identified, 14 were prioritised for analysis as the remaining 8 reviews were rated as low or critically low quality. We focused on network meta-analyses of weight loss outcomes measured at 6 months, 12 months, longer than 12 months or over a mix of time points. At 6 months, subcutaneous tirzepatide was the most effective drug associated with 9 kg (at 5 mg) to 12 kg (at 15 mg) of weight loss. However, the largest effects were seen for subcutaneous semaglutide 2.4 mg, which was associated with between 11.5 and 12.5 kg of weight loss, though this came from two network meta-analyses, both informed by six trials, and both merging findings across multiple time points. The relative effectiveness among glucagon-like peptide 1 receptor agonists followed a pattern suggested by their performance against placebo, with tirzepatide and semaglutide standing out as the most effective drugs for weight loss. No network meta-analyses compared tirzepatide and semaglutide 2.4 mg. The drugs associated with th","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-73"},"PeriodicalIF":3.5,"publicationDate":"2025-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144527680","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":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;There is no clear preferential option for initial step-up of treatment for children with uncontrolled asthma on inhaled corticosteroid.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;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 &lt; 18 years with uncontrolled asthma on any dose inhaled corticosteroid alone. We searched MEDLINE&lt;sup&gt;®&lt;/sup&gt;, 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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;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 &lt;i&gt;β&lt;/i&gt;&lt;sub&gt;2&lt;/sub&gt;-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}