Timothy S Walsh, Richard A Parker, Leanne M Aitken, Cathrine A McKenzie, Robert Glen, Christopher J Weir
<p><strong>Background: </strong>Optimising comfort and ability to communicate for mechanically ventilated intensive care unit patients is a priority for clinicians, intensive care unit patients and their relatives. Current usual care is propofol-based sedation plus an opioid analgesic. The alpha2-agonists dexmedetomidine and clonidine are potential alternative sedatives.</p><p><strong>Objective(s): </strong>To explore whether nurses and relatives perceive patients sedated with dexmedetomidine and/or clonidine appear more awake, comfortable and co-operative than patients receiving only propofol-based sedation.</p><p><strong>Design and methods: </strong>Substudy within an open-label, three-arm trial.</p><p><strong>Setting and participants: </strong>Forty-one intensive care units in the United Kingdom. One thousand four hundred and thirty-seven adults receiving propofol ± opioid for sedation-analgesia within 48 hours of starting mechanical ventilation, expected to require ≥ 48 total hours of mechanical ventilation.</p><p><strong>Interventions: </strong>Light sedation was targeted in all patients unless clinicians requested deeper sedation. In intervention groups, algorithms promoted alpha2-agonist up-titration and propofol down-titration, followed by sedation primarily with allocated alpha2-agonist. Usual care was propofol-based sedation. Intervention continued until patients were successfully extubated (primary outcome), or other pre-defined end points.</p><p><strong>Outcomes: </strong>For each 12-hour care period, nurses responded to two 'yes/no' questions: <i>is the patient able to communicate pain? Is the patient able to co-operate with care?</i> When the patients' personal legal representative visited, they were asked for 'yes/no' responses to three questions: <i>does the patient appear awake? Does the patient appear comfortable? Does the visitor feel they can communicate with the patient?</i> Intervention versus propofol group responses were compared fitting a generalised linear mixed model, with results expressed as odds ratios (95% confidence intervals); odds ratios > 1 indicated greater probability of a 'yes' response.</p><p><strong>Results: </strong>Nurse responses were available for > 90% of trial patients [mean (standard deviation) 12 (12) care periods per patient]. Comparing dexmedetomidine versus propofol groups, the odds ratio for a 'yes' response to '<i>communicate pain</i>' was 1.38 (95% confidence interval 1.08 to 1.75), and for clonidine versus propofol, it was 1.13 (0.89 to 1.43). For '<i>co-operate with care</i>' comparing dexmedetomidine versus propofol groups, the odds ratio was 1.14 (95% confidence interval 0.98 to 1.32), and for clonidine versus propofol, it was 0.96 (95% confidence interval 0.83 to 1.12). Relative responses were available for 32-34% of trial patients across groups [mean (standard deviation) 3 (3) days per patient]. For the '<i>appear awake</i>' question, the dexmedetomidine versus propofol group odds ratio
{"title":"Relative and bedside nurse assessment of comfort and communication during propofol, dexmedetomidine, or clonidine-based sedation: pre-planned analysis within the A2B RCT.","authors":"Timothy S Walsh, Richard A Parker, Leanne M Aitken, Cathrine A McKenzie, Robert Glen, Christopher J Weir","doi":"10.3310/GJTW2718","DOIUrl":"10.3310/GJTW2718","url":null,"abstract":"<p><strong>Background: </strong>Optimising comfort and ability to communicate for mechanically ventilated intensive care unit patients is a priority for clinicians, intensive care unit patients and their relatives. Current usual care is propofol-based sedation plus an opioid analgesic. The alpha2-agonists dexmedetomidine and clonidine are potential alternative sedatives.</p><p><strong>Objective(s): </strong>To explore whether nurses and relatives perceive patients sedated with dexmedetomidine and/or clonidine appear more awake, comfortable and co-operative than patients receiving only propofol-based sedation.</p><p><strong>Design and methods: </strong>Substudy within an open-label, three-arm trial.</p><p><strong>Setting and participants: </strong>Forty-one intensive care units in the United Kingdom. One thousand four hundred and thirty-seven adults receiving propofol ± opioid for sedation-analgesia within 48 hours of starting mechanical ventilation, expected to require ≥ 48 total hours of mechanical ventilation.</p><p><strong>Interventions: </strong>Light sedation was targeted in all patients unless clinicians requested deeper sedation. In intervention groups, algorithms promoted alpha2-agonist up-titration and propofol down-titration, followed by sedation primarily with allocated alpha2-agonist. Usual care was propofol-based sedation. Intervention continued until patients were successfully extubated (primary outcome), or other pre-defined end points.</p><p><strong>Outcomes: </strong>For each 12-hour care period, nurses responded to two 'yes/no' questions: <i>is the patient able to communicate pain? Is the patient able to co-operate with care?</i> When the patients' personal legal representative visited, they were asked for 'yes/no' responses to three questions: <i>does the patient appear awake? Does the patient appear comfortable? Does the visitor feel they can communicate with the patient?</i> Intervention versus propofol group responses were compared fitting a generalised linear mixed model, with results expressed as odds ratios (95% confidence intervals); odds ratios > 1 indicated greater probability of a 'yes' response.</p><p><strong>Results: </strong>Nurse responses were available for > 90% of trial patients [mean (standard deviation) 12 (12) care periods per patient]. Comparing dexmedetomidine versus propofol groups, the odds ratio for a 'yes' response to '<i>communicate pain</i>' was 1.38 (95% confidence interval 1.08 to 1.75), and for clonidine versus propofol, it was 1.13 (0.89 to 1.43). For '<i>co-operate with care</i>' comparing dexmedetomidine versus propofol groups, the odds ratio was 1.14 (95% confidence interval 0.98 to 1.32), and for clonidine versus propofol, it was 0.96 (95% confidence interval 0.83 to 1.12). Relative responses were available for 32-34% of trial patients across groups [mean (standard deviation) 3 (3) days per patient]. For the '<i>appear awake</i>' question, the dexmedetomidine versus propofol group odds ratio ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-18"},"PeriodicalIF":4.0,"publicationDate":"2026-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12862536/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145997903","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}
Dawn Lee, Madhusubramanian Muthukumar, Alan Lovell, Caroline Farmer, Darren Burns, Justin Matthews, Helen Coelho, Brian O'Toole, Laura A Trigg, Tristan M Snowsill, Maxwell S Barnish, Thalia Nikoglou, Amanda Brand, Zain Ahmad, Ahmed Abdelsabour, Louise Crathorne, Sophie Robinson, Edward Cf Wilson, G J Melendez-Torres
<p><strong>Background: </strong>The National Institute for Health and Care Excellence is piloting a new approach to evaluating health technologies, which takes into consideration the full treatment pathway for a condition. This report describes the first pilot topic for the pathways process, which evaluated systemic treatments for advanced renal cell carcinoma.</p><p><strong>Objectives: </strong>This pilot aimed to develop a decision model representing the treatment pathway that will be used to evaluate new technologies for advanced renal cell carcinoma. The pilot also evaluated a new treatment for renal cell carcinoma: cabozantinib (Cabometyx®; Ipsen, Slough, UK) plus nivolumab (Opdivo®; Bristol Myers Squibb, Princeton, NJ, USA).</p><p><strong>Review methods: </strong>A systematic literature review was conducted to identify evidence to inform effectiveness, safety and economic model development, including systematic literature reviews, randomised controlled trials, economic evaluations, utility studies and cost and resource use data. Real-world evidence was sought following the recommendations of the National Institute for Health and Care Excellence real-world evidence framework. Structured expert elicitation informed assumptions about overall survival and progression-free survival. Network meta-analyses were conducted to evaluate the clinical effectiveness of treatments. A de novo state transition model that was constructed with a partitioned survival analysis structure was also presented. The cost perspective of the model was that of the National Health Service and Personal Social Services; the time horizon was 40 years, costs and outcomes were discounted at 3.5% per annum and a 2022 price year was used. The model allowed sequences of up to four active lines of treatment.</p><p><strong>Information sources: </strong>The review included 118 systematic literature reviews, 30 randomised controlled trials, 122 economic evaluations, 82 studies reporting utility data and 13 studies reporting cost and/or resource use data. A total of 21 real-world evidence sources were identified. Unpublished data were provided by the manufacturer and other stakeholders (competitor companies, patient and clinical organisations). The expert elicitation recruited nine United Kingdom-based oncologists.</p><p><strong>Results: </strong>Cabozantinib plus nivolumab was associated with better progression-free survival and overall survival than existing tyrosine kinase inhibitors as first-line treatment in the all-risk group. Using the list price of the evaluated interventions, the incremental cost-effectiveness ratio for cabozantinib plus nivolumab compared to the next non-dominated tyrosine kinase inhibitor monotherapy (pazopanib [Votrient®; Novartis, Slough, UK]) was £275,106 per quality-adjusted life-year in the all-risk population and was £379,222 in the favourable-risk population. Incremental cost-effectiveness ratios were relatively consistent across the base-case and s
{"title":"Treatments for renal cell carcinoma: NICE Pilot Treatment Pathways Appraisal.","authors":"Dawn Lee, Madhusubramanian Muthukumar, Alan Lovell, Caroline Farmer, Darren Burns, Justin Matthews, Helen Coelho, Brian O'Toole, Laura A Trigg, Tristan M Snowsill, Maxwell S Barnish, Thalia Nikoglou, Amanda Brand, Zain Ahmad, Ahmed Abdelsabour, Louise Crathorne, Sophie Robinson, Edward Cf Wilson, G J Melendez-Torres","doi":"10.3310/GJDL0327","DOIUrl":"10.3310/GJDL0327","url":null,"abstract":"<p><strong>Background: </strong>The National Institute for Health and Care Excellence is piloting a new approach to evaluating health technologies, which takes into consideration the full treatment pathway for a condition. This report describes the first pilot topic for the pathways process, which evaluated systemic treatments for advanced renal cell carcinoma.</p><p><strong>Objectives: </strong>This pilot aimed to develop a decision model representing the treatment pathway that will be used to evaluate new technologies for advanced renal cell carcinoma. The pilot also evaluated a new treatment for renal cell carcinoma: cabozantinib (Cabometyx®; Ipsen, Slough, UK) plus nivolumab (Opdivo®; Bristol Myers Squibb, Princeton, NJ, USA).</p><p><strong>Review methods: </strong>A systematic literature review was conducted to identify evidence to inform effectiveness, safety and economic model development, including systematic literature reviews, randomised controlled trials, economic evaluations, utility studies and cost and resource use data. Real-world evidence was sought following the recommendations of the National Institute for Health and Care Excellence real-world evidence framework. Structured expert elicitation informed assumptions about overall survival and progression-free survival. Network meta-analyses were conducted to evaluate the clinical effectiveness of treatments. A de novo state transition model that was constructed with a partitioned survival analysis structure was also presented. The cost perspective of the model was that of the National Health Service and Personal Social Services; the time horizon was 40 years, costs and outcomes were discounted at 3.5% per annum and a 2022 price year was used. The model allowed sequences of up to four active lines of treatment.</p><p><strong>Information sources: </strong>The review included 118 systematic literature reviews, 30 randomised controlled trials, 122 economic evaluations, 82 studies reporting utility data and 13 studies reporting cost and/or resource use data. A total of 21 real-world evidence sources were identified. Unpublished data were provided by the manufacturer and other stakeholders (competitor companies, patient and clinical organisations). The expert elicitation recruited nine United Kingdom-based oncologists.</p><p><strong>Results: </strong>Cabozantinib plus nivolumab was associated with better progression-free survival and overall survival than existing tyrosine kinase inhibitors as first-line treatment in the all-risk group. Using the list price of the evaluated interventions, the incremental cost-effectiveness ratio for cabozantinib plus nivolumab compared to the next non-dominated tyrosine kinase inhibitor monotherapy (pazopanib [Votrient®; Novartis, Slough, UK]) was £275,106 per quality-adjusted life-year in the all-risk population and was £379,222 in the favourable-risk population. Incremental cost-effectiveness ratios were relatively consistent across the base-case and s","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 1","pages":"1-221"},"PeriodicalIF":4.0,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12833715/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146003346","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}
Matthew Prescott, Michelle Collinson, Abi J Hall, Rebecca Bestwick, Victoria A Goodwin, Ellen Thompson, Chris Bojke, David Clarke, Florence Day, Anne Forster, Claire Hulme, Julie Peacock, Friederike Ziegler, Amanda J Farrin, Andrew Clegg
<p><strong>Background: </strong>Half of older people in hospital have frailty and are at increased risk of re-admission or death following discharge. Although short-term rehabilitation can reduce early re-admissions, benefits are attenuated over time. It is unknown whether extended rehabilitation for older people with frailty can improve outcomes.</p><p><strong>Trial design: </strong>Pragmatic, multicentre, individually randomised controlled parallel-group superiority trial with economic evaluation and embedded process evaluation.</p><p><strong>Methods: </strong>Participants: Eligible participants were 65 years or older with mild/moderate/severe frailty (score of 5-7 on Clinical Frailty Scale) admitted to hospital with acute illness or injury, then discharged home directly or from intermediate care (post-acute care) rehabilitation services. People with significant cognitive impairment and care home residents were among those ineligible. Recruitment took place from December 2017 to August 2021, with follow-up till August 2022. Interventions: Participants were randomly assigned (1.28 : 1) to the Home-based Older People's Exercise programme - a 24-week home-based manualised, progressive exercise intervention delivered by National Health Service therapists as extended rehabilitation, or usual care (control). Randomisation occurred after the participant had been discharged from hospital or intermediate care. Participants were not masked to allocation. Main outcome measures: The primary outcome was physical health-related quality of life, measured using the physical component score of the modified Short Form 36-item health questionnaire at 12 months. Secondary outcomes at 6 and 12 months included physical and mental health-related quality of life, functional independence, death, hospitalisations and care home admissions. Researchers involved in data collection were masked to allocation. Data sources: Primary and secondary outcomes were obtained via self-report questionnaire at 6 and 12 months. Hospitalisations and deaths were collected from routine healthcare data.</p><p><strong>Results: </strong>We randomised 740 participants (410 Home-based Older People's Exercise, 330 control) across 15 sites. Four hundred and seventy-nine (64.7%) participants completed 12-month follow-up. One hundred and eighty-eight Home-based Older People's Exercise participants (45.9%) completed 24 weeks of intervention delivery. Over half of participants completed more than 75% of prescribed exercises. Intention-to-treat analyses (258 Home-based Older People's Exercise participants, 208 control participants for primary outcome) showed no evidence that Home-based Older People's Exercise was superior to control for 12-month physical component score (adjusted mean difference -0.22, 95% confidence interval -1.47 to 1.03; <i>p</i> = 0.73). There was some evidence of a higher rate of all-cause hospitalisations in the control arm (incidence rate ratio 1.12, 95% confidence interval 1.0
{"title":"Home-based extended rehabilitation for older people with frailty (HERO): a multicentre randomised controlled trial with health economic analysis and process evaluation.","authors":"Matthew Prescott, Michelle Collinson, Abi J Hall, Rebecca Bestwick, Victoria A Goodwin, Ellen Thompson, Chris Bojke, David Clarke, Florence Day, Anne Forster, Claire Hulme, Julie Peacock, Friederike Ziegler, Amanda J Farrin, Andrew Clegg","doi":"10.3310/GJAC1602","DOIUrl":"10.3310/GJAC1602","url":null,"abstract":"<p><strong>Background: </strong>Half of older people in hospital have frailty and are at increased risk of re-admission or death following discharge. Although short-term rehabilitation can reduce early re-admissions, benefits are attenuated over time. It is unknown whether extended rehabilitation for older people with frailty can improve outcomes.</p><p><strong>Trial design: </strong>Pragmatic, multicentre, individually randomised controlled parallel-group superiority trial with economic evaluation and embedded process evaluation.</p><p><strong>Methods: </strong>Participants: Eligible participants were 65 years or older with mild/moderate/severe frailty (score of 5-7 on Clinical Frailty Scale) admitted to hospital with acute illness or injury, then discharged home directly or from intermediate care (post-acute care) rehabilitation services. People with significant cognitive impairment and care home residents were among those ineligible. Recruitment took place from December 2017 to August 2021, with follow-up till August 2022. Interventions: Participants were randomly assigned (1.28 : 1) to the Home-based Older People's Exercise programme - a 24-week home-based manualised, progressive exercise intervention delivered by National Health Service therapists as extended rehabilitation, or usual care (control). Randomisation occurred after the participant had been discharged from hospital or intermediate care. Participants were not masked to allocation. Main outcome measures: The primary outcome was physical health-related quality of life, measured using the physical component score of the modified Short Form 36-item health questionnaire at 12 months. Secondary outcomes at 6 and 12 months included physical and mental health-related quality of life, functional independence, death, hospitalisations and care home admissions. Researchers involved in data collection were masked to allocation. Data sources: Primary and secondary outcomes were obtained via self-report questionnaire at 6 and 12 months. Hospitalisations and deaths were collected from routine healthcare data.</p><p><strong>Results: </strong>We randomised 740 participants (410 Home-based Older People's Exercise, 330 control) across 15 sites. Four hundred and seventy-nine (64.7%) participants completed 12-month follow-up. One hundred and eighty-eight Home-based Older People's Exercise participants (45.9%) completed 24 weeks of intervention delivery. Over half of participants completed more than 75% of prescribed exercises. Intention-to-treat analyses (258 Home-based Older People's Exercise participants, 208 control participants for primary outcome) showed no evidence that Home-based Older People's Exercise was superior to control for 12-month physical component score (adjusted mean difference -0.22, 95% confidence interval -1.47 to 1.03; <i>p</i> = 0.73). There was some evidence of a higher rate of all-cause hospitalisations in the control arm (incidence rate ratio 1.12, 95% confidence interval 1.0","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 4","pages":"1-40"},"PeriodicalIF":4.0,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12833716/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146010043","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}
Gwenllian Wynne-Jones, Martyn Lewis, Gail Sowden, Ira Madan, Karen Walker-Bone, Carolyn A Chew-Graham, Kieran Bromley, Sue Jowett, Vaughan Parsons, Gemma Mansell, Kendra Cooke, Benjamin Saunders, Rosie Harrison, Sarah A Lawton, Simon Wathall, John Pemberton, Julia Hammond, Cyrus Cooper, And Nadine E Foster
Background and objectives: To describe exploratory findings and lessons learned from the discontinued WAVE trial, which sought to determine the effectiveness and costs of adding an early vocational advice intervention to usual primary care on number of days of sickness absence over 6 months.
Methods: Pragmatic, multicentre, two-parallel arm, superiority, randomised controlled trial with health economic analysis in 10 general practices in England, with nested qualitative interviews. Population: Adults with fit notes for any health condition, absent from work ≥ 2 weeks and ≤ 6 months were invited to participate. Intervention and comparator: Participants were randomised (1 : 1) to usual primary care with/without vocational advice delivered by trained Vocational Support Workers. The planned sample size was 720, the first 4 months of recruitment served as an internal pilot phase and the primary outcome was self-reported days of work absence over 6 months.
Results: One hundred and thirty participants were recruited from 7955 invitations (May 2022-May 2023) before trial closure (64 usual care, 66 usual care plus vocational advice). Exploratory analysis of 125 participants (with outcome data) indicated small additional benefits of the vocational advice intervention over usual care [mean days absence = 37.86 (standard deviation = 48.76) vs. usual care = 42.66 (standard deviation = 57.67), incidence rate ratio = 0.913, 80% confidence interval (0.653 to 1.276)]. The vocational advice intervention was delivered remotely [mean = 4.8 contacts (range 1-12)]. Partial health economic evaluation found lower work productivity losses at 6 months after vocational advice intervention (£5513.84, standard deviation = £7101.43) compared to usual care (£6146.21, standard deviation = £8431.88).
Conclusions, limitations and future work: Exploratory analysis indicated a signal of effect, with differences in the number of days absent from work, costs and secondary outcomes. Key lessons learned included the need for closer working with primary care teams and more flexible recruitment methods. A future fully powered randomised controlled trial of vocational advice intervention added to usual primary care is needed to determine the effectiveness and cost-effectiveness.
Funding: This article presents independent research funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme as award number 17/94/49.
{"title":"Addition of early vocational advice to usual primary care on sickness absence in employed adults: exploratory findings from the discontinued WAVE Randomised Controlled Trial.","authors":"Gwenllian Wynne-Jones, Martyn Lewis, Gail Sowden, Ira Madan, Karen Walker-Bone, Carolyn A Chew-Graham, Kieran Bromley, Sue Jowett, Vaughan Parsons, Gemma Mansell, Kendra Cooke, Benjamin Saunders, Rosie Harrison, Sarah A Lawton, Simon Wathall, John Pemberton, Julia Hammond, Cyrus Cooper, And Nadine E Foster","doi":"10.3310/SVEG8456","DOIUrl":"10.3310/SVEG8456","url":null,"abstract":"<p><strong>Background and objectives: </strong>To describe exploratory findings and lessons learned from the discontinued WAVE trial, which sought to determine the effectiveness and costs of adding an early vocational advice intervention to usual primary care on number of days of sickness absence over 6 months.</p><p><strong>Methods: </strong>Pragmatic, multicentre, two-parallel arm, superiority, randomised controlled trial with health economic analysis in 10 general practices in England, with nested qualitative interviews. Population: Adults with fit notes for any health condition, absent from work ≥ 2 weeks and ≤ 6 months were invited to participate. Intervention and comparator: Participants were randomised (1 : 1) to usual primary care with/without vocational advice delivered by trained Vocational Support Workers. The planned sample size was 720, the first 4 months of recruitment served as an internal pilot phase and the primary outcome was self-reported days of work absence over 6 months.</p><p><strong>Results: </strong>One hundred and thirty participants were recruited from 7955 invitations (May 2022-May 2023) before trial closure (64 usual care, 66 usual care plus vocational advice). Exploratory analysis of 125 participants (with outcome data) indicated small additional benefits of the vocational advice intervention over usual care [mean days absence = 37.86 (standard deviation = 48.76) vs. usual care = 42.66 (standard deviation = 57.67), incidence rate ratio = 0.913, 80% confidence interval (0.653 to 1.276)]. The vocational advice intervention was delivered remotely [mean = 4.8 contacts (range 1-12)]. Partial health economic evaluation found lower work productivity losses at 6 months after vocational advice intervention (£5513.84, standard deviation = £7101.43) compared to usual care (£6146.21, standard deviation = £8431.88).</p><p><strong>Conclusions, limitations and future work: </strong>Exploratory analysis indicated a signal of effect, with differences in the number of days absent from work, costs and secondary outcomes. Key lessons learned included the need for closer working with primary care teams and more flexible recruitment methods. A future fully powered randomised controlled trial of vocational advice intervention added to usual primary care is needed to determine the effectiveness and cost-effectiveness.</p><p><strong>Funding: </strong>This article presents independent research funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme as award number 17/94/49.</p>","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-32"},"PeriodicalIF":4.0,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12884355/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146100155","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}
<p><strong>Background: </strong>Bariatric surgery can improve health outcomes but high-quality comparative evidence about different procedures is limited.</p><p><strong>Objective: </strong>To compare the effectiveness and cost-effectiveness of Roux-en-Y gastric bypass (Bypass), adjustable gastric banding (Band) and sleeve gastrectomy (Sleeve) for people living with severe obesity.</p><p><strong>Design, setting and participants: </strong>Multicentre, parallel-group, randomised controlled trial conducted in 12 National Health Service hospitals. Adults with a body mass index ≥ 35 kg/m<sup>2</sup> with comorbidity or body mass index ≥ 40 kg/m<sup>2</sup> without comorbidity were eligible. Participants were initially randomised 1 : 1 to Bypass or Band. After 32 months of recruitment, the trial was adapted to include Sleeve, and participants were randomised to Bypass, Band or Sleeve thereafter. Participants were followed up for 3 years.</p><p><strong>Interventions: </strong>Bypass, Band and Sleeve surgery.</p><p><strong>Main outcome measures: </strong>Primary outcomes were self-reported quality of life (EQ-5D-5L utility score) and weight (at least 50% excess weight lost) at 3 years. Sleeve and Bypass were each considered superior to Band if there was non-inferior excess weight loss (< 12% difference between groups) and superior quality of life. Sleeve was considered superior to Bypass by the same criteria. Secondary outcomes included comorbidities, adverse health events, generic and disease-specific quality of life at 6, 12, 24 and 36 months post randomisation, dietary intake, binge eating behaviour and cost-effectiveness.</p><p><strong>Results: </strong>One thousand three hundred and fifty-one participants were randomised between December 2012 and September 2019. Five participants withdrew consent to use their data, leaving 1346 (462 Bypass, 464 Band, 420 Sleeve). The mean age was 47.3 years, 1020 (75.9%) were women and the mean weight and body mass index was 129.7 kg and 46.4 kg/m<sup>2</sup>, respectively. Overall, 1183 (87.5%) of participants underwent surgery within 3 years, with a median waiting time of 5 months (interquartile range 2.5-10.1 months). At least 50% excess weight loss at 3 years was achieved for 276/405 (68.1%) participants randomised to Bypass, 97/383 (25.3%) randomised to Band and 142/342 (41.5%) randomised to Sleeve [adjusted risk difference (Bypass-Band) + 40.7%, 98% confidence interval (+ 33.9% to + 47.5%); (Sleeve-Band) + 14.7% (+ 5.2% to + 24.2%), (Sleeve-Bypass) -26.0% (-35.8% to -16.3%)]. Mean EQ-5D scores at 3 years were 0.72 (standard deviation 0.29), 0.62 (0.33) and 0.68 (0.30) for participants randomised to Bypass, Band and Sleeve, respectively [adjusted mean difference (Bypass-Band) + 0.079 (+ 0.040 to + 0.117), (Sleeve-Band) + 0.045 (+ 0.006 to + 0.085), (Sleeve-Bypass) -0.033 (-0.072 to + 0.006)]. Secondary outcomes showed similar trends. The adverse event rate was highest in the Band group and lowest with Sleeve. By
{"title":"Roux-en-Y gastric bypass, adjustable gastric banding or sleeve gastrectomy for severe obesity: The By-Band-Sleeve randomised controlled trial.","authors":"","doi":"10.3310/CXSE2951","DOIUrl":"10.3310/CXSE2951","url":null,"abstract":"<p><strong>Background: </strong>Bariatric surgery can improve health outcomes but high-quality comparative evidence about different procedures is limited.</p><p><strong>Objective: </strong>To compare the effectiveness and cost-effectiveness of Roux-en-Y gastric bypass (Bypass), adjustable gastric banding (Band) and sleeve gastrectomy (Sleeve) for people living with severe obesity.</p><p><strong>Design, setting and participants: </strong>Multicentre, parallel-group, randomised controlled trial conducted in 12 National Health Service hospitals. Adults with a body mass index ≥ 35 kg/m<sup>2</sup> with comorbidity or body mass index ≥ 40 kg/m<sup>2</sup> without comorbidity were eligible. Participants were initially randomised 1 : 1 to Bypass or Band. After 32 months of recruitment, the trial was adapted to include Sleeve, and participants were randomised to Bypass, Band or Sleeve thereafter. Participants were followed up for 3 years.</p><p><strong>Interventions: </strong>Bypass, Band and Sleeve surgery.</p><p><strong>Main outcome measures: </strong>Primary outcomes were self-reported quality of life (EQ-5D-5L utility score) and weight (at least 50% excess weight lost) at 3 years. Sleeve and Bypass were each considered superior to Band if there was non-inferior excess weight loss (< 12% difference between groups) and superior quality of life. Sleeve was considered superior to Bypass by the same criteria. Secondary outcomes included comorbidities, adverse health events, generic and disease-specific quality of life at 6, 12, 24 and 36 months post randomisation, dietary intake, binge eating behaviour and cost-effectiveness.</p><p><strong>Results: </strong>One thousand three hundred and fifty-one participants were randomised between December 2012 and September 2019. Five participants withdrew consent to use their data, leaving 1346 (462 Bypass, 464 Band, 420 Sleeve). The mean age was 47.3 years, 1020 (75.9%) were women and the mean weight and body mass index was 129.7 kg and 46.4 kg/m<sup>2</sup>, respectively. Overall, 1183 (87.5%) of participants underwent surgery within 3 years, with a median waiting time of 5 months (interquartile range 2.5-10.1 months). At least 50% excess weight loss at 3 years was achieved for 276/405 (68.1%) participants randomised to Bypass, 97/383 (25.3%) randomised to Band and 142/342 (41.5%) randomised to Sleeve [adjusted risk difference (Bypass-Band) + 40.7%, 98% confidence interval (+ 33.9% to + 47.5%); (Sleeve-Band) + 14.7% (+ 5.2% to + 24.2%), (Sleeve-Bypass) -26.0% (-35.8% to -16.3%)]. Mean EQ-5D scores at 3 years were 0.72 (standard deviation 0.29), 0.62 (0.33) and 0.68 (0.30) for participants randomised to Bypass, Band and Sleeve, respectively [adjusted mean difference (Bypass-Band) + 0.079 (+ 0.040 to + 0.117), (Sleeve-Band) + 0.045 (+ 0.006 to + 0.085), (Sleeve-Bypass) -0.033 (-0.072 to + 0.006)]. Secondary outcomes showed similar trends. The adverse event rate was highest in the Band group and lowest with Sleeve. By","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 6","pages":"1-224"},"PeriodicalIF":4.0,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12884357/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146113041","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}
Dwayne Boyers, Moira Cruickshank, Mohammad Azharuddin, Paul Manson, Diane Swallow, Carl Counsell, Miriam Brazzelli
<p><strong>Background: </strong>Chronic tic disorders and Tourette syndrome typically present around age 5, with peak severity between ages 10 and 12. Treatment approaches vary by country and service availability and include psychoeducation, behavioural therapy, pharmacological therapies and deep brain stimulation. Digitally enabled interventions may improve outcomes.</p><p><strong>Objectives: </strong>We evaluate the clinical and cost-effectiveness of two digital technologies, Online Remote Behavioural Treatment for Tics and Neupulse and identify evidence gaps for future research.</p><p><strong>Methods: </strong>We searched major electronic databases (MEDLINE, EMBASE, Cochrane Library, Web of Science, and Cumulative Index to Nursing and Allied Health Literature) for published studies on clinical and cost-effectiveness. Data were extracted, assessed for bias using Cochrane risk-of-bias tool (version 2), and pooled using random-effects meta-analysis where appropriate. Cost-effectiveness was evaluated using a Markov cohort model with five tic severity states based on the Yale Global Tic Severity Scale - Total Tic Severity Score scale, from a United Kingdom National Health Service perspective. Model inputs were obtained from the Online Remote Behavioural Treatment for Tics study, company data, expert opinion and additional literature.</p><p><strong>Results: </strong>We identified three trials reported across 14 publications: 2 comparing Online Remote Behavioural Treatment for Tics with online psychoeducation, and 1 comparing Neupulse with sham stimulation and a waitlist control. All were assessed as low risk of bias. Meta-analysis of the 2 Online Remote Behavioural Treatment for Tics studies (445 participants) showed significantly lower Yale Global Tic Severity Scale - Total Tic Severity Score at 3 and 12 months compared to online psychoeducation. Results of secondary outcomes were mixed. Neupulse showed significantly lower Yale Global Tic Severity Scale - Total Tic Severity Score, and improvements in motor and phonic tic scores at 4 weeks compared to sham, but no differences in Yale Global Tic Severity Scale-Impairment or Premonitory Urge for Tics Scale - Revised scores. A definitive base-case incremental cost-effectiveness ratio could not be determined due to limited long-term data and uncertainty around long-term combinations of effectiveness and intervention costs in United Kingdom National Health Service practice. Probabilistic incremental cost-effectiveness ratios ranged from £642 per quality-adjusted life-year gained to Online Remote Behavioural Treatment for Tics being dominated. The probability of Online Remote Behavioural Treatment for Tics being cost-effective at a threshold value of £20,000 per quality-adjusted life-year ranged from 52% to 89% across a range of scenarios. Cost-effectiveness results for Neupulse were even more uncertain due to a lack of published data, only a 4-week follow-up and uncertainty surrounding the intervention c
背景：慢性抽动障碍和抽动秽语综合征通常出现在5岁左右，严重程度在10 - 12岁之间达到高峰。治疗方法因国家和服务的可得性而异，包括心理教育、行为治疗、药物治疗和深部脑刺激。数字化干预可以改善结果。目的：我们评估两种数字技术的临床和成本效益，抽搐和神经脉冲的在线远程行为治疗，并为未来的研究确定证据差距。方法：我们检索了主要的电子数据库（MEDLINE， EMBASE, Cochrane Library, Web of Science, and Cumulative Index to Nursing and Allied Health Literature），以获取已发表的临床和成本效益方面的研究。提取数据，使用Cochrane风险-偏倚工具（版本2）评估偏倚，并在适当情况下使用随机效应荟萃分析进行汇总。成本-效果评估采用基于耶鲁全球抽动严重程度量表-总抽动严重程度评分量表的马尔可夫队列模型，从英国国家卫生服务的角度出发。模型输入来自tic在线远程行为治疗研究、公司数据、专家意见和其他文献。结果：我们确定了14份出版物中报道的3项试验：2项比较抽搐症的在线远程行为治疗与在线心理教育，1项比较神经脉冲与假刺激和候补对照。所有被评估为低偏倚风险。对两项抽动症在线远程行为治疗研究（445名参与者）的荟萃分析显示，与在线心理教育相比，3个月和12个月的耶鲁全球抽动症严重程度量表-总抽动症严重程度评分显着降低。次要结局的结果是混合的。与假手术相比，新脉冲手术在4周时的运动和语音抽动评分明显降低，但在耶鲁全球抽动严重程度量表-缺陷或抽动先兆冲动量表-修订评分方面没有差异。由于长期数据有限以及联合王国国民保健服务实践中有效性和干预成本长期组合的不确定性，无法确定确定的基本情况增量成本效益比。概率增量成本效益比从每质量调整生命年获得642英镑到抽动症的在线远程行为治疗占主导地位。在各种情况下，抽搐症在线远程行为治疗在每个质量调整生命年2万英镑的阈值下具有成本效益的可能性在52%到89%之间。由于缺乏公开的数据，只有4周的随访，以及干预成本的不确定性，nepulse的成本效益结果更加不确定。局限性：抽搐和神经性搏动的在线远程行为治疗证据有限，评估结果不一致。比较方法不包括面对面的行为治疗，也不可能区分在线提供的效果与接触和反应预防的效果。由于缺乏长期数据，成本效益结果不确定。结论：抽动症的在线远程行为治疗和神经脉冲治疗似乎都能显著降低耶鲁全球抽动严重程度量表-总抽动严重程度评分，但耶鲁全球抽动严重程度量表-损伤评分没有改善，其他次要结局的结果也不一致，这意味着抽动严重程度评分的改善在多大程度上可以转化为生活质量的改善尚不清楚。由于缺乏长期证据，成本效益估计极不确定。未来研究：需要进行重复性研究来证实研究结果。长期随访——特别是对nepulse的随访——对于了解持续效果和长期成本效益至关重要。更多关于抽动症严重程度的自然过程和治疗效果的可持续性的数据将减少经济模型中的不确定性。未来的研究还应优先考虑反映现实生活对人们日常功能影响的结果。研究注册：本研究注册号为PROSPERO CRD42024508045。资助：该奖项由美国国家卫生与保健研究所（NIHR）证据综合计划（NIHR奖励编号：NIHR136022）资助，全文发表在《卫生技术评估》上；第30卷第8期有关进一步的奖励信息，请参阅美国国立卫生研究院资助和奖励网站。
{"title":"Digitally enabled therapy for chronic tic disorders and Tourette Syndrome: a systematic review and economic evaluation.","authors":"Dwayne Boyers, Moira Cruickshank, Mohammad Azharuddin, Paul Manson, Diane Swallow, Carl Counsell, Miriam Brazzelli","doi":"10.3310/QLAS8524","DOIUrl":"10.3310/QLAS8524","url":null,"abstract":"<p><strong>Background: </strong>Chronic tic disorders and Tourette syndrome typically present around age 5, with peak severity between ages 10 and 12. Treatment approaches vary by country and service availability and include psychoeducation, behavioural therapy, pharmacological therapies and deep brain stimulation. Digitally enabled interventions may improve outcomes.</p><p><strong>Objectives: </strong>We evaluate the clinical and cost-effectiveness of two digital technologies, Online Remote Behavioural Treatment for Tics and Neupulse and identify evidence gaps for future research.</p><p><strong>Methods: </strong>We searched major electronic databases (MEDLINE, EMBASE, Cochrane Library, Web of Science, and Cumulative Index to Nursing and Allied Health Literature) for published studies on clinical and cost-effectiveness. Data were extracted, assessed for bias using Cochrane risk-of-bias tool (version 2), and pooled using random-effects meta-analysis where appropriate. Cost-effectiveness was evaluated using a Markov cohort model with five tic severity states based on the Yale Global Tic Severity Scale - Total Tic Severity Score scale, from a United Kingdom National Health Service perspective. Model inputs were obtained from the Online Remote Behavioural Treatment for Tics study, company data, expert opinion and additional literature.</p><p><strong>Results: </strong>We identified three trials reported across 14 publications: 2 comparing Online Remote Behavioural Treatment for Tics with online psychoeducation, and 1 comparing Neupulse with sham stimulation and a waitlist control. All were assessed as low risk of bias. Meta-analysis of the 2 Online Remote Behavioural Treatment for Tics studies (445 participants) showed significantly lower Yale Global Tic Severity Scale - Total Tic Severity Score at 3 and 12 months compared to online psychoeducation. Results of secondary outcomes were mixed. Neupulse showed significantly lower Yale Global Tic Severity Scale - Total Tic Severity Score, and improvements in motor and phonic tic scores at 4 weeks compared to sham, but no differences in Yale Global Tic Severity Scale-Impairment or Premonitory Urge for Tics Scale - Revised scores. A definitive base-case incremental cost-effectiveness ratio could not be determined due to limited long-term data and uncertainty around long-term combinations of effectiveness and intervention costs in United Kingdom National Health Service practice. Probabilistic incremental cost-effectiveness ratios ranged from £642 per quality-adjusted life-year gained to Online Remote Behavioural Treatment for Tics being dominated. The probability of Online Remote Behavioural Treatment for Tics being cost-effective at a threshold value of £20,000 per quality-adjusted life-year ranged from 52% to 89% across a range of scenarios. Cost-effectiveness results for Neupulse were even more uncertain due to a lack of published data, only a 4-week follow-up and uncertainty surrounding the intervention c","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 8","pages":"1-67"},"PeriodicalIF":4.0,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12884836/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146113089","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}
Zoe Ward, Ruth Simmons, Hannah Fraser, Adam Trickey, Joanna Kesten, Andrew Gibson, Leila Reid, Sean Cox, Fiona Gordon, Stuart McPherson, Stephen Ryder, Francisco Javier Vilar, Alec Miners, Jack Williams, Beatrice Emmanouil, Monica Desai, Laura Coughlan, Ross Harris, Graham R Foster, Matthew Hickman, Sema Mandal, Peter Vickerman
<p><strong>Background: </strong>People who inject drugs are disproportionately affected by hepatitis C virus. The emergence of direct-acting antiviral treatments for hepatitis C virus motivated England to achieve the World Health Organization's elimination target of decreasing hepatitis C virus incidence among people who inject drugs to < 2 per 100 person-years (/100 person-years) by 2030. We determined whether existing testing and treatment strategies will reach this target in England, or whether improved strategies are needed and whether they are cost-effective.</p><p><strong>Methods: </strong>A dynamic hepatitis C virus transmission model among people who inject drugs was developed for four English regions. The model included the pathway from testing to treatment in prisons, drug treatment centres and other settings. Each pathway was parameterised using region-specific data, with yearly bio-behavioural surveys among people who inject drugs being used to parameterise and calibrate the model in a Bayesian framework. The model projected whether each region will reach the hepatitis C virus incidence target or what improvements (in testing and linkage to treatment) are needed from 2024 to achieve it. Hepatitis C virus care pathway costs were collated through interviews with practitioners and the published literature. The mean incremental cost-effectiveness ratio (per quality-adjusted life-year saved) was estimated for any 'improved' strategy that reached the incidence target compared to the baseline strategy. Incremental cost-effectiveness ratios were compared to a willingness-to-pay threshold of £20,000/quality-adjusted life-year saved over a 50-year time horizon (3.5% annual discount rate).</p><p><strong>Results: </strong>Across the four regions over 2016-22, an estimated 8831-9689 treatments occurred among 37,230 people who inject drugs, with the annual number treated increasing in prisons (7.8 times) and drug treatment centres (3.6 times). Model projections suggest that hepatitis C virus incidence among people who inject drugs has decreased across the regions by 56.1-85.4% (range of medians) over 2015-22, with incidence decreasing by 79.7-98.6% to 0.2-2.2/100 person-years by 2030. The World Health Organization incidence target (< 2/100 person-years) will be reached with > 80% probability in three regions and 40% probability in the other region. The probability of reaching the incidence target increases to > 65% in this region if screening is increased in drug treatment centres (80% screened annually) or prisons (75% of people get tested during their prison stay), with these screening strategies being cost-effective.</p><p><strong>Conclusion: </strong>Numerous England regions may be on target to decrease hepatitis C virus incidence among people who inject drugs to < 2/100 person-years. In regions that are not on target, further scale-up of testing in drug treatment centre or prison from 2024 could enable them to reach the World Health Organizati
{"title":"The impact and cost-effectiveness of scaling up HCV treatment for achieving elimination among people who inject drugs in England: a synopsis including evidence synthesis and economic modelling.","authors":"Zoe Ward, Ruth Simmons, Hannah Fraser, Adam Trickey, Joanna Kesten, Andrew Gibson, Leila Reid, Sean Cox, Fiona Gordon, Stuart McPherson, Stephen Ryder, Francisco Javier Vilar, Alec Miners, Jack Williams, Beatrice Emmanouil, Monica Desai, Laura Coughlan, Ross Harris, Graham R Foster, Matthew Hickman, Sema Mandal, Peter Vickerman","doi":"10.3310/GJPV1707","DOIUrl":"10.3310/GJPV1707","url":null,"abstract":"<p><strong>Background: </strong>People who inject drugs are disproportionately affected by hepatitis C virus. The emergence of direct-acting antiviral treatments for hepatitis C virus motivated England to achieve the World Health Organization's elimination target of decreasing hepatitis C virus incidence among people who inject drugs to < 2 per 100 person-years (/100 person-years) by 2030. We determined whether existing testing and treatment strategies will reach this target in England, or whether improved strategies are needed and whether they are cost-effective.</p><p><strong>Methods: </strong>A dynamic hepatitis C virus transmission model among people who inject drugs was developed for four English regions. The model included the pathway from testing to treatment in prisons, drug treatment centres and other settings. Each pathway was parameterised using region-specific data, with yearly bio-behavioural surveys among people who inject drugs being used to parameterise and calibrate the model in a Bayesian framework. The model projected whether each region will reach the hepatitis C virus incidence target or what improvements (in testing and linkage to treatment) are needed from 2024 to achieve it. Hepatitis C virus care pathway costs were collated through interviews with practitioners and the published literature. The mean incremental cost-effectiveness ratio (per quality-adjusted life-year saved) was estimated for any 'improved' strategy that reached the incidence target compared to the baseline strategy. Incremental cost-effectiveness ratios were compared to a willingness-to-pay threshold of £20,000/quality-adjusted life-year saved over a 50-year time horizon (3.5% annual discount rate).</p><p><strong>Results: </strong>Across the four regions over 2016-22, an estimated 8831-9689 treatments occurred among 37,230 people who inject drugs, with the annual number treated increasing in prisons (7.8 times) and drug treatment centres (3.6 times). Model projections suggest that hepatitis C virus incidence among people who inject drugs has decreased across the regions by 56.1-85.4% (range of medians) over 2015-22, with incidence decreasing by 79.7-98.6% to 0.2-2.2/100 person-years by 2030. The World Health Organization incidence target (< 2/100 person-years) will be reached with > 80% probability in three regions and 40% probability in the other region. The probability of reaching the incidence target increases to > 65% in this region if screening is increased in drug treatment centres (80% screened annually) or prisons (75% of people get tested during their prison stay), with these screening strategies being cost-effective.</p><p><strong>Conclusion: </strong>Numerous England regions may be on target to decrease hepatitis C virus incidence among people who inject drugs to < 2/100 person-years. In regions that are not on target, further scale-up of testing in drug treatment centre or prison from 2024 could enable them to reach the World Health Organizati","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 7","pages":"1-14"},"PeriodicalIF":4.0,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12884358/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146113131","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}
David Cottrell, Rebecca Walwyn, Amanda Farrin, Donna Irving, Peter Fonagy, Dennis Ougrin, Daniel Stahl, Judy Wright, Alex Wright-Hughes
<p><strong>Background: </strong>Self-harm is common in adolescents and a major public health concern. Evidence for effective interventions is lacking. An individual participant data meta-analysis has potential to provide more reliable estimates of the effects of therapeutic interventions than conventional meta-analyses and to explore which treatments are best suited to certain groups.</p><p><strong>Methods: </strong>A systematic review and individual participant data meta-analysis of randomised controlled trials of therapeutic interventions to reduce repeat self-harm in adolescents with a history of self-harm and who had presented to clinical services. We searched Cochrane Library, EMBASE, trial registers and other databases for randomised controlled trials published in January 2022. Eligible randomised controlled trials compared any therapeutic intervention against a control, aimed to reduce self-harm in adolescents (11-18 years old), with past self-harm presenting to clinical services, and collected outcome data on self-harm or suicide attempts. Interventions reviewed were grouped into nine categories: cognitive-behavioural therapy; dialectical behaviour therapy; family therapy; group therapy; mentalisation based, psychodynamic, cognitive analytic therapy; multisystemic therapy; problem-solving, psychoeducation, support; postcards, tokens, documents (postcards/tokens); and other single session, brief interventions. Control interventions were all either treatment as usual or enhanced treatment as usual and were not usually well described. There were no 'no treatment' controls except in the postcard/document/token studies. Primary outcome was repetition of self-harm at 12 months. Other outcomes included repetition of self-harm at other time points, overall mental health, depressive symptoms, thoughts of suicide, quality of life and death. Two-stage random-effects individual participant data meta-analyses were conducted overall and by intervention, and to examine interaction between treatment received and participant characteristics. Secondary analyses incorporated aggregate data from randomised controlled trials without individual participant data. Metaregression explored moderating study effects.</p><p><strong>Results: </strong>We identified 39 eligible studies, from 10 countries, where we sought individual participant data (18 studies with full sample eligibility, 21 with partial sample eligibility). We obtained individual participant data from 26 studies of 3448 eligible participants. We used published data from a further seven studies where individual participant data were not available for a combined individual participant data aggregate data meta-analysis (698 participants). For our primary outcome, repetition of self-harm, only six studies were rated as low risk of bias. There was no evidence that intervention/s were more or less effective than controls at preventing repeat self-harm by 12 months using individual participant data (odds rat
{"title":"Reducing self-harm in adolescents: the RISA-IPD comprehensive synopsis.","authors":"David Cottrell, Rebecca Walwyn, Amanda Farrin, Donna Irving, Peter Fonagy, Dennis Ougrin, Daniel Stahl, Judy Wright, Alex Wright-Hughes","doi":"10.3310/KKBB1164","DOIUrl":"10.3310/KKBB1164","url":null,"abstract":"<p><strong>Background: </strong>Self-harm is common in adolescents and a major public health concern. Evidence for effective interventions is lacking. An individual participant data meta-analysis has potential to provide more reliable estimates of the effects of therapeutic interventions than conventional meta-analyses and to explore which treatments are best suited to certain groups.</p><p><strong>Methods: </strong>A systematic review and individual participant data meta-analysis of randomised controlled trials of therapeutic interventions to reduce repeat self-harm in adolescents with a history of self-harm and who had presented to clinical services. We searched Cochrane Library, EMBASE, trial registers and other databases for randomised controlled trials published in January 2022. Eligible randomised controlled trials compared any therapeutic intervention against a control, aimed to reduce self-harm in adolescents (11-18 years old), with past self-harm presenting to clinical services, and collected outcome data on self-harm or suicide attempts. Interventions reviewed were grouped into nine categories: cognitive-behavioural therapy; dialectical behaviour therapy; family therapy; group therapy; mentalisation based, psychodynamic, cognitive analytic therapy; multisystemic therapy; problem-solving, psychoeducation, support; postcards, tokens, documents (postcards/tokens); and other single session, brief interventions. Control interventions were all either treatment as usual or enhanced treatment as usual and were not usually well described. There were no 'no treatment' controls except in the postcard/document/token studies. Primary outcome was repetition of self-harm at 12 months. Other outcomes included repetition of self-harm at other time points, overall mental health, depressive symptoms, thoughts of suicide, quality of life and death. Two-stage random-effects individual participant data meta-analyses were conducted overall and by intervention, and to examine interaction between treatment received and participant characteristics. Secondary analyses incorporated aggregate data from randomised controlled trials without individual participant data. Metaregression explored moderating study effects.</p><p><strong>Results: </strong>We identified 39 eligible studies, from 10 countries, where we sought individual participant data (18 studies with full sample eligibility, 21 with partial sample eligibility). We obtained individual participant data from 26 studies of 3448 eligible participants. We used published data from a further seven studies where individual participant data were not available for a combined individual participant data aggregate data meta-analysis (698 participants). For our primary outcome, repetition of self-harm, only six studies were rated as low risk of bias. There was no evidence that intervention/s were more or less effective than controls at preventing repeat self-harm by 12 months using individual participant data (odds rat","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 3","pages":"1-51"},"PeriodicalIF":4.0,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12869895/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146010035","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}
Katie Kirkham, Eleni Gkini, Catherine Moakes, Sue Tohill, Jennifer A Hutcheon, Joel Singer, Jon Dorling, Clive Stubbs, Marcus Green, Mishal Javed, Jesse Kigozi, Ben Mol, Pollyanna Hardy, Jim Thornton, Peter von Dadelszen, Laura A Magee
<p><strong>Background: </strong>For women with chronic or gestational hypertension who remain well, early term birth (at 37-38 weeks' gestation) may reduce maternal complications, caesareans and stillbirths, but it may increase neonatal morbidity compared with expectant care. Expectant care may increase costs. There are no high-quality data to guide care, which currently involves maternal-fetal surveillance and intervention for maternal or fetal compromise, which may be rapid or unexpected.</p><p><strong>Objective: </strong>To investigate optimal timing of birth for women with chronic or gestational hypertension who reach term and remain well.</p><p><strong>Design: </strong>Pragmatic, unmasked, multicentre randomised trial with a health economic analysis.</p><p><strong>Setting: </strong>Fifty United Kingdom hospitals.</p><p><strong>Participants: </strong>Inclusion: maternal age ≥ 16 years, chronic or gestational hypertension, singleton pregnancy, live fetus, 36<sup>+0</sup>-37<sup>+6</sup> weeks' gestation and able to give documented informed consent. Exclusion: contraindication to either trial arm (e.g. pre-eclampsia), blood pressure ≥ 160/110 mmHg until controlled, major fetal anomaly anticipated to require neonatal care unit admission or participation in another timed birth trial.</p><p><strong>Interventions: </strong>Planned early term birth at 38<sup>+0-3</sup> weeks' (intervention) or 'usual care at term' (control, revised from 'expectant care until at least 40<sup>+0</sup> weeks', August 2022).</p><p><strong>Main outcome measures: </strong>Maternal coprimary: composite of 'poor maternal outcome' (severe hypertension, maternal death or maternal morbidity and superiority hypothesis). Neonatal coprimary: neonatal care unit admission ≥ 4 hours (non-inferiority hypothesis). Each coprimary is measured until primary hospital discharge or 28 days post birth (whichever is earlier). Key secondary: caesarean birth.</p><p><strong>Randomisation: </strong>1 : 1 ratio, minimised for key prognostic variables: site, hypertension type and prior caesarean.</p><p><strong>Blinding: </strong>It was not possible to mask care providers or participants to the intervention. For the coprimary maternal outcome, there was local site principal investigator/delegate sign-off based on review, masked to allocated group, of primary case notes.</p><p><strong>Results: </strong>From 2019 to 2022, 403 participants were randomised (37% of target 1080) to intervention (<i>n</i> = 201) or control (<i>n</i> = 202). The funder stopped the trial during the coronavirus disease discovered in 2019 pandemic for delayed recruitment. In the intervention (vs. control) group, birth was a median of 0.9 weeks earlier (38.4, interquartile range 38.3-38.6 vs. 39.3, interquartile range 38.7-39.9 weeks). There was no evidence of a difference in 'poor maternal outcome' (13% vs. 12%, respectively; adjusted risk ratio 1.16, 95% confidence interval 0.72 to 1.87). For 'neonatal care unit admission ≥ 4
{"title":"Timing of birth to improve outcomes in chronic or gestational hypertension: the WILL RCT.","authors":"Katie Kirkham, Eleni Gkini, Catherine Moakes, Sue Tohill, Jennifer A Hutcheon, Joel Singer, Jon Dorling, Clive Stubbs, Marcus Green, Mishal Javed, Jesse Kigozi, Ben Mol, Pollyanna Hardy, Jim Thornton, Peter von Dadelszen, Laura A Magee","doi":"10.3310/AAVV3131","DOIUrl":"10.3310/AAVV3131","url":null,"abstract":"<p><strong>Background: </strong>For women with chronic or gestational hypertension who remain well, early term birth (at 37-38 weeks' gestation) may reduce maternal complications, caesareans and stillbirths, but it may increase neonatal morbidity compared with expectant care. Expectant care may increase costs. There are no high-quality data to guide care, which currently involves maternal-fetal surveillance and intervention for maternal or fetal compromise, which may be rapid or unexpected.</p><p><strong>Objective: </strong>To investigate optimal timing of birth for women with chronic or gestational hypertension who reach term and remain well.</p><p><strong>Design: </strong>Pragmatic, unmasked, multicentre randomised trial with a health economic analysis.</p><p><strong>Setting: </strong>Fifty United Kingdom hospitals.</p><p><strong>Participants: </strong>Inclusion: maternal age ≥ 16 years, chronic or gestational hypertension, singleton pregnancy, live fetus, 36<sup>+0</sup>-37<sup>+6</sup> weeks' gestation and able to give documented informed consent. Exclusion: contraindication to either trial arm (e.g. pre-eclampsia), blood pressure ≥ 160/110 mmHg until controlled, major fetal anomaly anticipated to require neonatal care unit admission or participation in another timed birth trial.</p><p><strong>Interventions: </strong>Planned early term birth at 38<sup>+0-3</sup> weeks' (intervention) or 'usual care at term' (control, revised from 'expectant care until at least 40<sup>+0</sup> weeks', August 2022).</p><p><strong>Main outcome measures: </strong>Maternal coprimary: composite of 'poor maternal outcome' (severe hypertension, maternal death or maternal morbidity and superiority hypothesis). Neonatal coprimary: neonatal care unit admission ≥ 4 hours (non-inferiority hypothesis). Each coprimary is measured until primary hospital discharge or 28 days post birth (whichever is earlier). Key secondary: caesarean birth.</p><p><strong>Randomisation: </strong>1 : 1 ratio, minimised for key prognostic variables: site, hypertension type and prior caesarean.</p><p><strong>Blinding: </strong>It was not possible to mask care providers or participants to the intervention. For the coprimary maternal outcome, there was local site principal investigator/delegate sign-off based on review, masked to allocated group, of primary case notes.</p><p><strong>Results: </strong>From 2019 to 2022, 403 participants were randomised (37% of target 1080) to intervention (<i>n</i> = 201) or control (<i>n</i> = 202). The funder stopped the trial during the coronavirus disease discovered in 2019 pandemic for delayed recruitment. In the intervention (vs. control) group, birth was a median of 0.9 weeks earlier (38.4, interquartile range 38.3-38.6 vs. 39.3, interquartile range 38.7-39.9 weeks). There was no evidence of a difference in 'poor maternal outcome' (13% vs. 12%, respectively; adjusted risk ratio 1.16, 95% confidence interval 0.72 to 1.87). For 'neonatal care unit admission ≥ 4","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 2","pages":"1-28"},"PeriodicalIF":4.0,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12862530/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146003378","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}
Abril Seyahian, Martin Taylor-Rowan, Clareece Nevill, Ryan Mulholland, Campbell Roxburgh, Susan Brunskill, Nicola Cooper, Anna Noel-Storr, Alex J Sutton, Olivia Wu, Terry J Quinn
<p><strong>Background: </strong>United Kingdom blood shortages necessitate better prediction of surgical blood requirement. We sought to assess the predictive accuracy of tools designed to identify those patients requiring blood transfusion within the perioperative period.</p><p><strong>Methods: </strong>We searched the Cochrane library, EMBASE, MEDLINE, ClinicalTrials.gov and WHO trials portal, 2000-July 2023. We included studies that developed and/or validated prediction tools for blood requirement during the early perioperative period (48 hours). Risk of bias was evaluated using the Prediction model Risk Of Bias Assessment Tool. We pooled area under receiver operating curve and calibration data via random effects meta-analysis. We evaluated certainty of evidence of any estimates using the Grading of Recommendations Assessment, Development and Evaluation framework. We used meta-regression to describe associations between included variables/tool characteristics with tool accuracy.</p><p><strong>Results: </strong>We included 50 papers, describing 67 unique prediction tools. Most tools were at high risk of bias, with limited external validation. Discrimination (area under receiver operating curve) of prognostic models ranged from 0.49 to 0.96. Only two surgery-specific tools, the McClusky Index (liver transplant surgery) and Papworth Bleeding Risk Score (cardiothoracic surgery), had sufficient data to enable pooling of discrimination measures. The McClusky Index's pooled area under receiver operating curve: 0.74 (95% CI 0.61 to 0.84) and Bleeding Risk Score's area under receiver operating curve: 0.68 (95% CI 0.49 to 0.82) were both rated 'very low' certainty by Grading of Recommendations Assessment, Development and Evaluation. Pooling calibration data was not possible for any prediction tools. Meta-regression suggested that fewer included variables, longer time from surgery and independent validation studies were all associated with lower accuracy.</p><p><strong>Limitations: </strong>There were insufficient studies to assess overall tool performance via meta-analysis in other surgical subgroups beyond cardiothoracic surgery and liver transplant. Our study population is also predominantly made up of elective surgeries which may make our results less generalisable to emergency settings.</p><p><strong>Future work: </strong>Implementation and cost-effectiveness studies are needed to evaluate how promising tools could be applied to clinical practice and the economic impact such tools could have upon the service.</p><p><strong>Conclusions: </strong>Despite the availability of multiple potential tools, available data suggest none are currently suitable for predicting blood transfusion in surgical practice. Our summary of the data comes with caveats around the quality of the included papers and the limited number of tools with more than one reported external validation.</p><p><strong>Funding: </strong>This article presents independent research funded by t
背景：英国血液短缺需要更好地预测手术用血需求。我们试图评估用于识别围手术期需要输血的患者的工具的预测准确性。方法：检索Cochrane图书馆、EMBASE、MEDLINE、ClinicalTrials.gov和WHO试验门户网站，检索时间为2000- 2023年7月。我们纳入了开发和/或验证围手术期早期（48小时）血液需求预测工具的研究。使用预测模型偏倚风险评估工具评估偏倚风险。我们通过随机效应荟萃分析合并了受试者工作曲线下的面积和校准数据。我们使用建议分级评估、发展和评估框架评估任何估计证据的确定性。我们使用元回归来描述包含变量/工具特征与工具精度之间的关联。结果：我们纳入了50篇论文，描述了67种独特的预测工具。大多数工具存在高偏倚风险，外部验证有限。预后模型的鉴别（受试者工作曲线下面积）范围为0.49 ~ 0.96。只有麦克卢斯基指数（McClusky Index，肝移植手术）和帕普沃斯出血风险评分（Papworth Bleeding Risk Score，心胸外科手术）这两种手术专用工具有足够的数据来汇集区分措施。麦克拉斯基指数在受者操作曲线下的合并面积：0.74 （95% CI 0.61至0.84）和出血风险评分在受者操作曲线下的合并面积：0.68 （95% CI 0.49至0.82）均被推荐评估、发展和评估分级评为“非常低”确定性。任何预测工具都不可能汇集校准数据。meta回归表明，纳入变量较少、手术时间较长和独立验证研究均与较低的准确性相关。局限性：没有足够的研究通过荟萃分析来评估除心胸外科和肝移植以外的其他手术亚组的总体工具性能。我们的研究人群也主要由选择性手术组成，这可能使我们的结果不太适用于急诊情况。未来工作：需要进行实施和成本效益研究，以评估有希望的工具如何应用于临床实践以及这些工具对服务的经济影响。结论：尽管有多种潜在的工具可用，但现有数据表明，目前没有一种工具适合预测外科手术中的输血。我们的数据总结附带了关于所纳入论文质量的警告，以及具有多个外部验证报告的有限数量的工具。资助：本文介绍了由国家卫生与保健研究所（NIHR）卫生技术评估计划资助的独立研究，奖励号为NIHR159933。
{"title":"Risk assessment tools for predicting transfusion in surgery: a systematic review and meta-analysis.","authors":"Abril Seyahian, Martin Taylor-Rowan, Clareece Nevill, Ryan Mulholland, Campbell Roxburgh, Susan Brunskill, Nicola Cooper, Anna Noel-Storr, Alex J Sutton, Olivia Wu, Terry J Quinn","doi":"10.3310/GJAS1620","DOIUrl":"10.3310/GJAS1620","url":null,"abstract":"<p><strong>Background: </strong>United Kingdom blood shortages necessitate better prediction of surgical blood requirement. We sought to assess the predictive accuracy of tools designed to identify those patients requiring blood transfusion within the perioperative period.</p><p><strong>Methods: </strong>We searched the Cochrane library, EMBASE, MEDLINE, ClinicalTrials.gov and WHO trials portal, 2000-July 2023. We included studies that developed and/or validated prediction tools for blood requirement during the early perioperative period (48 hours). Risk of bias was evaluated using the Prediction model Risk Of Bias Assessment Tool. We pooled area under receiver operating curve and calibration data via random effects meta-analysis. We evaluated certainty of evidence of any estimates using the Grading of Recommendations Assessment, Development and Evaluation framework. We used meta-regression to describe associations between included variables/tool characteristics with tool accuracy.</p><p><strong>Results: </strong>We included 50 papers, describing 67 unique prediction tools. Most tools were at high risk of bias, with limited external validation. Discrimination (area under receiver operating curve) of prognostic models ranged from 0.49 to 0.96. Only two surgery-specific tools, the McClusky Index (liver transplant surgery) and Papworth Bleeding Risk Score (cardiothoracic surgery), had sufficient data to enable pooling of discrimination measures. The McClusky Index's pooled area under receiver operating curve: 0.74 (95% CI 0.61 to 0.84) and Bleeding Risk Score's area under receiver operating curve: 0.68 (95% CI 0.49 to 0.82) were both rated 'very low' certainty by Grading of Recommendations Assessment, Development and Evaluation. Pooling calibration data was not possible for any prediction tools. Meta-regression suggested that fewer included variables, longer time from surgery and independent validation studies were all associated with lower accuracy.</p><p><strong>Limitations: </strong>There were insufficient studies to assess overall tool performance via meta-analysis in other surgical subgroups beyond cardiothoracic surgery and liver transplant. Our study population is also predominantly made up of elective surgeries which may make our results less generalisable to emergency settings.</p><p><strong>Future work: </strong>Implementation and cost-effectiveness studies are needed to evaluate how promising tools could be applied to clinical practice and the economic impact such tools could have upon the service.</p><p><strong>Conclusions: </strong>Despite the availability of multiple potential tools, available data suggest none are currently suitable for predicting blood transfusion in surgical practice. Our summary of the data comes with caveats around the quality of the included papers and the limited number of tools with more than one reported external validation.</p><p><strong>Funding: </strong>This article presents independent research funded by t","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-38"},"PeriodicalIF":4.0,"publicationDate":"2025-12-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12703565/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145687386","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}
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