Health technology assessment最新文献

{"title":"A systematic review of the cost-effectiveness of anti-VEGF drugs for the treatment of diabetic retinopathy.","authors":"Robert Hodgson, Matthew Walton, Helen Fulbright, Laura Bojke, Ruth Walker, Alexis Llewellyn, Sofia Dias, Lesley Stewart, David Steel, John Lawrenson, Tunde Peto, Mark Simmonds","doi":"10.3310/NHYK3694","DOIUrl":"https://doi.org/10.3310/NHYK3694","url":null,"abstract":"<p><strong>Background: </strong>Non-proliferative and proliferative diabetic retinopathy are common complications of diabetes and a major cause of sight loss. Anti-vascular endothelial growth factor drugs represent a treatment option for people with diabetic retinopathy and are routinely used to treat various other eye conditions. However, anti-vascular endothelial growth factor drugs are expensive relative to current care options, and it is unclear whether this additional cost is justified when the immediate risk of vision loss is lower compared to patients with more aggressive ophthalmological conditions.</p><p><strong>Objective: </strong>To systematically review the evidence supporting the cost-effectiveness of alternative treatments for diabetic retinopathy.</p><p><strong>Methods: </strong>A systematic review of all comparative cost-effectiveness studies evaluating any treatment for diabetic retinopathy was conducted. Bibliographic searches were carried out to identify studies reporting on the cost-effectiveness of treatments for diabetic retinopathy; the latest searches were conducted on 28 April 2023. Included studies were synthesised narratively and evaluated with reference to UK decision-making. Studies were grouped by population into non-proliferative diabetic retinopathy and proliferative diabetic retinopathy.</p><p><strong>Results: </strong>The review identified five studies in the proliferative diabetic retinopathy population, all of which examined the cost-effectiveness of anti-vascular endothelial growth factor treatments compared to pan-retinal photocoagulation. Results of these studies suggest that anti-vascular endothelial growth factor treatments offer some additional benefits in terms of preserved visual acuity but also incur substantial additional costs relative to pan-retinal photocoagulation. Most authors agreed that the additional costs outweigh the limited benefits, especially in certain patient subgroups without pre-existing oedema. As most of the identified evidence considered a US perspective, it is unclear how these results would translate to a UK setting. Two studies were identified in the non-proliferative diabetic retinopathy population. There was limited evidence to support the early use of anti-vascular endothelial growth factor treatment. However, one UK study suggested that early treatment of non-proliferative diabetic retinopathy with pan-retinal photocoagulation is cost-effective compared to delayed pan-retinal photocoagulation.</p><p><strong>Conclusions: </strong>Overall, there is a dearth of cost-effectiveness evidence considering the UK context. The identified studies raised doubts about the cost-effectiveness of anti-vascular endothelial growth factor treatments for proliferative diabetic retinopathy. No conclusions can be made regarding the cost-effectiveness of anti-vascular endothelial growth factor treatments for non-proliferative diabetic retinopathy. Future research should focus on developing rigorou","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-19"},"PeriodicalIF":3.5,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143064382","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":"Public and patient involvement (PPI) in the design, execution and dissemination of a trial: the BISTRO trial.","authors":"David Coyle, Paula Ormandy, Nancy Fernandes da Silva, Simon Davies","doi":"10.3310/DOTR5903","DOIUrl":"https://doi.org/10.3310/DOTR5903","url":null,"abstract":"<p><strong>Background: </strong>For people receiving haemodialysis, a balance has to be struck between removing sufficient but not too much fluid during a treatment session and maintaining any remaining kidney function they might have. In the BISTRO trial, this study sought to establish if getting the balance right might be improved by the additional use of bioimpedance, a device that measures body fluid composition to help decide how much fluid to remove during dialysis. Designing and executing this trial, which incorporated complex and repeated trial procedures that would be dependent on participant engagement, presented challenges that demanded effective public and patient involvement.</p><p><strong>Objectives: </strong>This study aimed to develop an effective public and patient involvement participation model, ensuring that the patient voice was heard by the Trial Management Group, with a Patient Advisory Group undertaking coproduction of all participant-facing documents and communications, including dissemination of the trial results, with the main purpose of maximising participant engagement in the study.</p><p><strong>Design: </strong>An open-label randomised controlled trial in which 439 participants from 34 centres were allocated for regular assessments of their bodily fluid content with or without the use of bioimpedance measurements.</p><p><strong>Interventions: </strong>Development of an effective public and patient involvement working model that was represented within the Trial Management Group, contributing to protocol design, selection of bioimpedance device, and coproduction of all participant-facing communications including dissemination of trial findings.</p><p><strong>Main outcome measures: </strong>Public and patient involvement contribution prior to trial initiation, description of the participant-facing communications, adherence to trial materials, dropout and dissemination of trial findings. Post-trial evaluation by research teams, Patient Advisory Group and co-applicants.</p><p><strong>Results: </strong>An effective working model was developed which relied on remuneration of the public and patient involvement patient lead and use of social media (e.g. WhatsApp) to maximise inclusivity. The Patient Advisory Group coproduced with the Trial Management Group a series of communication postcards and newsletters and a web page to support the participants and disseminate the trial results that were highly rated by research teams, but not always passed on to trial participants. Participant adherence to the main trial outcomes was excellent (113.6% urine collections obtained). Potentially avoidable dropout was 14.4%, with 3.6% being clearly attributable to inability or unwillingness to comply with the trial procedures. Reflections by the Patient Advisory Group indicated that they felt valued, involved and listened to but anticipated more direct involvement with the trial participants, recommending that barriers to this be addressed ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-18"},"PeriodicalIF":3.5,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143064384","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":"Multi-cancer early detection tests for general population screening: a systematic literature review.","authors":"Ros Wade, Sarah Nevitt, Yiwen Liu, Melissa Harden, Claire Khouja, Gary Raine, Rachel Churchill, Sofia Dias","doi":"10.3310/DLMT1294","DOIUrl":"https://doi.org/10.3310/DLMT1294","url":null,"abstract":"<p><strong>Background: </strong>General population cancer screening in the United Kingdom is limited to selected cancers. Blood-based multi-cancer early detection tests aim to detect potential cancer signals from multiple cancers in the blood. The use of a multi-cancer early detection test for population screening requires a high specificity and a reasonable sensitivity to detect early-stage disease so that the benefits of earlier diagnosis and treatment can be realised.</p><p><strong>Objective: </strong>To undertake a systematic literature review of the clinical effectiveness evidence on blood-based multi-cancer early detection tests for screening.</p><p><strong>Methods: </strong>Comprehensive searches of electronic databases (including MEDLINE and EMBASE) and trial registers were undertaken in September 2023 to identify published and unpublished studies of multi-cancer early detection tests. Test manufacturer websites and reference lists of included studies and pertinent reviews were checked for additional studies. The target population was individuals aged 50-79 years without clinical suspicion of cancer. Outcomes of interest included test accuracy, number and proportion of cancers detected (by site and stage), time to diagnostic resolution, mortality, potential harms, health-related quality of life, acceptability and satisfaction. The risk of bias was assessed using the quality assessment of diagnostic accuracy studies-2 checklist. Results were summarised using narrative synthesis. Stakeholders contributed to protocol development, report drafting and interpretation of review findings.</p><p><strong>Results: </strong>Over 8000 records were identified. Thirty-six studies met the inclusion criteria: 1 ongoing randomised controlled trial, 13 completed cohort studies, 17 completed case-control studies and 5 ongoing cohort or case-control studies. Individual tests claimed to detect from 3 to over 50 different types of cancer. Diagnostic accuracy of currently available multi-cancer early detection tests varied substantially: Galleri<sup>®</sup> (GRAIL, Menlo Park, CA, USA) sensitivity 20.8-66.3%, specificity 98.4-99.5% (three studies); CancerSEEK (Exact Sciences, Madison, WI, USA) sensitivity 27.1-62.3%, specificity 98.9- 99.1% (two studies); SPOT-MAS™ (Gene Solutions, Ho Chi Minh City, Vietnam) sensitivity 72.4-100%, specificity 97.0-99.9% (two studies); Trucheck™ (Datar Cancer Genetics, Bayreuth, Germany) sensitivity 90.0%, specificity 96.4% (one study); Cancer Differentiation Analysis (AnPac Bio, Shanghai, China) sensitivity 40.0%, specificity 97.6% (one study). AICS<sup>®</sup> (AminoIndex Cancer Screening; Ajinomoto, Tokyo, Japan) screens for individual cancers separately, so no overall test performance statistics are available. Where reported, sensitivity was lower for detecting earlier-stage cancers (stages I-II) compared with later-stage cancers (stages III-IV). Studies of seven other multi-cancer early detection tests at an unclear stage of","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 2","pages":"1-105"},"PeriodicalIF":3.5,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143079034","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":"Development of a clinical decision support tool for Primary care Management of lower Urinary tract Symptoms in men: the PriMUS study.","authors":"Adrian Edwards, Ridhi Agarwal, Janine Bates, Alison Bray, Sarah Milosevic, Emma Thomas-Jones, Michael Drinnan, Marcus Drake, Peter Michell, Bethan Pell, Haroon Ahmed, Natalie Joseph-Williams, Kerenza Hood, Yemisi Takwoingi, Chris Harding","doi":"10.3310/RGTW5711","DOIUrl":"https://doi.org/10.3310/RGTW5711","url":null,"abstract":"<p><strong>Background: </strong>Lower urinary tract symptoms particularly affect older men and their quality of life. General practitioners currently have no easily available assessment tools to diagnose lower urinary tract symptom causes. Referrals to urology specialists are increasing. General practitioner access to simple, accurate tests and clinical decision tools could facilitate management of lower urinary tract symptoms in primary care.</p><p><strong>Objectives: </strong>To determine which of several index tests in combination, best predicted three diagnoses (detrusor overactivity, bladder outlet obstruction and/or detrusor underactivity) in men presenting with lower urinary tract symptoms in primary care. To develop and validate three diagnostic prediction models, and a prototype primary care clinical decision support tool.</p><p><strong>Design: </strong>Prospective diagnostic accuracy study. Two participant cohorts, for <i>development</i> and <i>validation</i>, underwent simple index tests and a reference standard (invasive urodynamics).</p><p><strong>Setting: </strong>General practices in England and Wales.</p><p><strong>Participants: </strong>Men (16 years and over) consulting their general practitioner with lower urinary tract symptoms.</p><p><strong>Sample size: </strong>Separate calculations for model development and validation cohorts, from literature estimates of detrusor overactivity, bladder outlet obstruction and detrusor underactivity prevalences of 57%, 31% and 16%, respectively.</p><p><strong>Predictors and index tests: </strong>Twelve potential predictors considered for three diagnostic models.</p><p><strong>Main outcome measures: </strong>The primary outcome was diagnostic model sensitivity and specificity for detecting bladder outlet obstruction, detrusor underactivity and detrusor overactivity, with 75.0% considered minimum clinically useful performance.</p><p><strong>Statistical analysis: </strong>Three separate logistic regression models generated with index test variables to predict the presence of bladder outlet obstruction, detrusor overactivity, detrusor underactivity conditions in men with lower urinary tract symptoms.</p><p><strong>Results: </strong>One model each was developed and validated for bladder outlet obstruction and detrusor underactivity, two for detrusor overactivity (detrusor overactivity main, detrusor overactivity sensitivity analysis 2). Age, voiding symptoms subscore, prostate-specific antigen level, median maximum flow rate, median voided volume were predictors for bladder outlet obstruction. Median maximum flow rate and post-void residual volume were predictors for detrusor underactivity. Age, post-void residual volume and median voided volume were included in detrusor overactivity main model, while age and storage symptoms subscore predicted detrusor overactivity sensitivity analysis 2. For all four models, sensitivity of 75.0% could be achieved with a specificity of 74.2%, 47.3%, 45.6% and 46","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 1","pages":"1-140"},"PeriodicalIF":3.5,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143079033","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":"Energetic activity for depression in young people aged 13-17 years: the READY feasibility RCT.","authors":"Megan Smith, Ryan James, Neil Howlett, Silvana Mengoni, Julia Jones, Erika Sims, David Turner, Kelly Grant, Allan Clark, Jamie Murdoch, Lindsay Bottoms, Jonathan Wilson, Shivani Sharma, Angel Chater, Cecile Guillard, Timothy Clarke, Andy Jones, Lee David, Solange Wyatt, Claire Rourke, David Wellsted, Daksha Trivedi","doi":"10.3310/KWNH4507","DOIUrl":"10.3310/KWNH4507","url":null,"abstract":"<p><strong>Background: </strong>Prevalence of depression is increasing in young people. Behaviour change interventions providing benefits equal to or greater than talking therapies or pharmacological alternatives are needed. Exercise could be beneficial for young people with depression, but we lack robust trials of its effectiveness.</p><p><strong>Objective: </strong>To test whether an exercise intervention targeting young people with depression is feasible, including recruitment and retention of young people, recruitment and training of exercise professionals and intervention delivery.</p><p><strong>Design: </strong>Three-arm cluster feasibility randomised controlled trial with embedded process evaluation and health economic data collection.</p><p><strong>Setting: </strong>Local community venues in Hertfordshire, Bedfordshire and Norfolk.</p><p><strong>Participants: </strong>Young people aged 13-17 years experiencing mild to moderate low mood or depression (indicated by scoring 17-36 on the Child Depression Inventory version 2) identified by mental health services, schools or self-referral.</p><p><strong>Interventions: </strong>Participants were randomised to one of three groups: high-intensity exercise, low-intensity exercise or a social activity control. Group sessions ran twice-weekly for 12 weeks delivered by registered exercise professionals, supported by mental health support workers.</p><p><strong>Main outcome measures: </strong>Referral, recruitment and retention rates; attendance at group sessions; adherence to and ability to reach intensity during exercise sessions; proportions of missing data, and adverse events measured at baseline and at 3 and 6 months; resource use; and reach and representativeness.</p><p><strong>Results: </strong>Of 321 referrals to the study, 173 were assessed for eligibility, and of the target sample size of 81, 15 were recruited and 14 were randomised (one withdrew). The retention rate was 71.4% and attendance at intervention sessions was > 67%; data completeness was > 80% for baseline assessments. Follow-up completion rate at 14 weeks was > 80% for most outcomes, with 50% for accelerometer data in the low-intensity group. Trial processes and the intervention were acceptable to young people. Barriers to and facilitators of intervention delivery were identified.</p><p><strong>Limitations: </strong>Findings highlighted challenges around recruitment, delivery of exercise interventions and informed ways of addressing barriers to recruitment for future studies. The study was conducted between October 2020 and August 2022 and consequently the COVID-19 pandemic had a disruptive impact on implementation.</p><p><strong>Conclusions: </strong>A large randomised trial of the effectiveness of the intervention is not feasible using the current study design, but issues relating to recruitment could be addressed with further work.</p><p><strong>Future work: </strong>Developing appropriate recruitment strategies via triage serv","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-26"},"PeriodicalIF":3.5,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11744430/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142871913","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Anti-VEGF drugs compared with laser photocoagulation for the treatment of diabetic retinopathy: a systematic review and meta-analysis.","authors":"Mark Simmonds, Alexis Llewellyn, Ruth Walker, Helen Fulbright, Matthew Walton, Rob Hodgson, Laura Bojke, Lesley Stewart, Sofia Dias, Thomas Rush, John G Lawrenson, Tunde Peto, David Steel","doi":"10.3310/PCGV5709","DOIUrl":"https://doi.org/10.3310/PCGV5709","url":null,"abstract":"<p><strong>Background: </strong>Diabetic retinopathy is a major cause of sight loss in people with diabetes. The most severe form, proliferative diabetic retinopathy, carries a high risk of vision loss, vitreous haemorrhage, macular oedema and other harms. Panretinal photocoagulation is the primary treatment for proliferative diabetic retinopathy. Anti-vascular endothelial growth factor drugs are used to treat various eye conditions and may be beneficial for people with diabetic retinopathy.</p><p><strong>Objective: </strong>To investigate the efficacy and safety of anti-vascular endothelial growth factor therapy for the treatment of diabetic retinopathy when compared to panretinal photocoagulation.</p><p><strong>Methods: </strong>A systematic review and network meta-analysis of all published randomised controlled trials comparing anti-vascular endothelial growth factor (alone or in combination with panretinal photocoagulation) to panretinal photocoagulation in people with diabetic retinopathy. The database searches were updated in May 2023. Trials where the primary focus was treatment of macular oedema or vitreous haemorrhage were excluded.</p><p><strong>Results: </strong>A total of 14 trials were included: 3 of aflibercept, 5 of bevacizumab and 6 of ranibizumab. Two trials were of patients with non-proliferative diabetic retinopathy; all others were in proliferative diabetic retinopathy. Overall, anti-vascular endothelial growth factor was slightly better than panretinal photocoagulation at preventing vision loss, measured as best corrected visual acuity, at up to 2 years follow-up [mean difference in the logarithm of the minimum angle of resolution -0.089 (or 3.6 Early Treatment Diabetic Retinopathy Study letters), 95% confidence interval -0.180 to -0.019]. There was no clear evidence of any difference between the anti-vascular endothelial growth factors, but the potential for bias complicated the comparison. One trial found no benefit of anti-vascular endothelial growth factor over panretinal photocoagulation after 5 years. Anti-vascular endothelial growth factor was superior to panretinal photocoagulation at preventing macular oedema (relative risk 0.29, 95% confidence interval 0.18 to 0.49) and vitreous haemorrhage (relative risk 0.77, 95% confidence interval 0.61 to 0.99). There was no clear evidence that the effectiveness of anti-vascular endothelial growth factor varied over time.</p><p><strong>Conclusions: </strong>Anti-vascular endothelial growth factor injections reduce vision loss when compared to panretinal photocoagulation, but the benefit is small and unlikely to be clinically meaningful. Anti-vascular endothelial growth factor may have greater benefits for preventing complications such as macular oedema. Observational studies extending follow-up beyond the 1-year duration of most trials are needed to investigate the longer-term effects of repeated anti-vascular endothelial growth factor injections.</p><p><strong>Funding: </strong","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-71"},"PeriodicalIF":3.5,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142824064","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":"Collagenase injection versus limited fasciectomy surgery to treat Dupuytren's contracture in adult patients in the UK: DISC, a non-inferiority RCT and economic evaluation.","authors":"Joseph Dias, Puvan Tharmanathan, Catherine Arundel, Charlie Welch, Qi Wu, Paul Leighton, Maria Armaou, Belen Corbacho, Nick Johnson, Sophie James, John Cooke, Christopher Bainbridge, Michael Craigen, David Warwick, Samantha Brady, Lydia Flett, Judy Jones, Catherine Knowlson, Michelle Watson, Ada Keding, Catherine Hewitt, David Torgerson","doi":"10.3310/KGXD8528","DOIUrl":"10.3310/KGXD8528","url":null,"abstract":"<p><strong>Background: </strong>Dupuytren's contracture is caused by nodules and cords which pull the fingers towards the palm of the hand. Treatments include limited fasciectomy surgery, collagenase injection and needle fasciotomy. There is limited evidence comparing limited fasciectomy with collagenase injection.</p><p><strong>Objectives: </strong>To compare whether collagenase injection is not inferior to limited fasciectomy when treating Dupuytren's contracture.</p><p><strong>Design: </strong>Pragmatic, two-arm, unblinded, randomised controlled non-inferiority trial with a cost-effectiveness evaluation and nested qualitative and photographic substudies.</p><p><strong>Setting: </strong>Thirty-one National Health Service hospitals in England and Scotland.</p><p><strong>Participants: </strong>Patients with Dupuytren's contracture of ≥ 30 degrees who had not received previous treatment in the same digit.</p><p><strong>Interventions: </strong>Collagenase injection with manipulation 1-7 days later was compared with limited fasciectomy.</p><p><strong>Main outcome measures: </strong>The primary outcome was the Patient Evaluation Measure score, with 1 year after treatment serving as the primary end point. A difference of 6 points in the primary end point was used as the non-inferiority margin. Secondary outcomes included: Unité Rhumatologique des Affections de la Main scale; Michigan Hand Outcomes Questionnaire; recurrence; extension deficit and total active movement; further care/re-intervention; complications; quality-adjusted life-year; resource use; and time to function recovery.</p><p><strong>Randomisation and blinding: </strong>Online central randomisation, stratified by the most affected joint, and with variable block sizes allocates participants 1 : 1 to collagenase or limited fasciectomy. Participants and clinicians were not blind to treatment allocation.</p><p><strong>Results: </strong>Between 31 July 2017 and 28 September 2021, 672 participants were recruited (<i>n</i> = 336 per group), of which 599 participants contributed to the primary outcome analysis (<i>n</i> = 285 limited fasciectomy; <i>n</i> = 314 collagenase). At 1 year (primary end point) there was little evidence to support rejection of the hypothesis that collagenase is inferior to limited fasciectomy. The difference in Patient Evaluation Measure score at 1 year was 5.95 (95% confidence interval 3.12 to 8.77; <i>p</i> = 0.49), increasing to 7.18 (95% confidence interval 4.18 to 10.88) at 2 years. The collagenase group had more complications (<i>n</i> = 267, 0.82 per participant) than the limited fasciectomy group (<i>n</i> = 177, 0.60 per participant), but limited fasciectomy participants had a greater proportion of 'moderate'/'severe' complications (5% vs. 2%). At least 54 participants (15.7%) had contracture recurrence and there was weak evidence suggesting that collagenase participants recurred more often than limited fasciectomy participants (odds ratio 1.39, 95% confidence","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 78","pages":"1-262"},"PeriodicalIF":3.5,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647555/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791513","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hybrid closed-loop systems for managing blood glucose levels in type 1 diabetes: a systematic review and economic modelling.","authors":"Asra Asgharzadeh, Mubarak Patel, Martin Connock, Sara Damery, Iman Ghosh, Mary Jordan, Karoline Freeman, Anna Brown, Rachel Court, Sharin Baldwin, Fatai Ogunlayi, Chris Stinton, Ewen Cummins, Lena Al-Khudairy","doi":"10.3310/JYPL3536","DOIUrl":"10.3310/JYPL3536","url":null,"abstract":"<p><strong>Background: </strong>Hybrid closed-loop systems are a new class of technology to manage type 1 diabetes mellitus. The system includes a combination of real-time continuous glucose monitoring from a continuous glucose monitoring device and a control algorithm to direct insulin delivery through an insulin pump. Evidence suggests that such technologies have the potential to improve the lives of people with type 1 diabetes mellitus and their families.</p><p><strong>Aim: </strong>The aim of this appraisal was to assess the clinical effectiveness and cost-effectiveness of hybrid closed-loop systems for managing glucose in people who have type 1 diabetes mellitus and are having difficulty managing their condition despite prior use of at least one of the following technologies: continuous subcutaneous insulin infusion, real-time continuous glucose monitoring or flash glucose monitoring (intermittently scanned continuous glucose monitoring).</p><p><strong>Methods: </strong>A systematic review of clinical effectiveness and cost-effectiveness evidence following predefined inclusion criteria informed by the aim of this review. An independent economic assessment using iQVIA CDM to model cost-effectiveness.</p><p><strong>Results: </strong>The clinical evidence identified 12 randomised controlled trials that compared hybrid closed loop with continuous subcutaneous insulin infusion + continuous glucose monitoring. Hybrid closed-loop arm of randomised controlled trials achieved improvement in glycated haemoglobin per cent [hybrid closed loop decreased glycated haemoglobin per cent by 0.28 (95% confidence interval -0.34 to -0.21), increased per cent of time in range (between 3.9 and 10.0 mmol/l) with a MD of 8.6 (95% confidence interval 7.03 to 10.22), and significantly decreased time in range (per cent above 10.0 mmol/l) with a MD of -7.2 (95% confidence interval -8.89 to -5.51), but did not significantly affect per cent of  time below range (< 3.9 mmol/l)]. Comparator arms showed improvements, but these were smaller than in the hybrid closed-loop arm. Outcomes were superior in the hybrid closed-loop arm compared with the comparator arm. The cost-effectiveness search identified six studies that were included in the systematic review. Studies reported subjective cost-effectiveness that was influenced by the willingness-to-pay thresholds. Economic evaluation showed that the published model validation papers suggest that an earlier version of the iQVIA CDM tended to overestimate the incidences of the complications of diabetes, this being particularly important for severe visual loss and end-stage renal disease. Overall survival's medium-term modelling appeared good, but there was uncertainty about its longer-term modelling. Costs provided by the National Health Service Supply Chain suggest that hybrid closed loop is around an annual average of £1500 more expensive than continuous subcutaneous insulin infusion + continuous glucose monitoring, this being a ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 80","pages":"1-190"},"PeriodicalIF":3.5,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11664472/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142824073","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessing long-term effectiveness and cost-effectiveness of statin therapy in the UK: a modelling study using individual participant data sets.","authors":"Borislava Mihaylova, Runguo Wu, Junwen Zhou, Claire Williams, Iryna Schlackow, Jonathan Emberson, Christina Reith, Anthony Keech, John Robson, Richard Parnell, Jane Armitage, Alastair Gray, John Simes, Colin Baigent","doi":"10.3310/KDAP7034","DOIUrl":"10.3310/KDAP7034","url":null,"abstract":"<p><strong>Background: </strong>Cardiovascular disease has declined but remains a major disease burden across developed countries.</p><p><strong>Objective: </strong>To assess the effectiveness and cost-effectiveness of statin therapy across United Kingdom population categories.</p><p><strong>Design: </strong>The cardiovascular disease microsimulation model, developed using Cholesterol Treatment Trialists' Collaboration data and the United Kingdom Biobank cohort, projected cardiovascular events, mortality, quality of life and healthcare costs using participant characteristics.</p><p><strong>Setting: </strong>United Kingdom primary health care.</p><p><strong>Participants: </strong>A total of 117,896 participants in 16 statin trials in the Cholesterol Treatment Trialists' Collaboration; 501,854 United Kingdom Biobank participants by previous cardiovascular disease status, sex, age (40-49, 50-59 and 60-70 years), 10-year cardiovascular disease risk [QRISK<sup>®</sup>3 (%): < 5, 5-10, 10-15, 15-20 and ≥ 20] and low-density lipoprotein cholesterol level (< 3.4, 3.4-4.1 and ≥ 4.1 mmol/l); 20,122 United Kingdom Biobank and Whitehall II participants aged ≥ 70 years by previous cardiovascular disease status, sex and low-density lipoprotein cholesterol (< 3.4, 3.4-4.1 and ≥ 4.1 mmol/l).</p><p><strong>Interventions: </strong>Lifetime standard (35-45% low-density lipoprotein cholesterol reduction) or higher-intensity (≥ 45% reduction) statin.</p><p><strong>Main outcome measures: </strong>Quality-adjusted life-years and incremental cost per quality-adjusted life-year gained from the United Kingdom healthcare perspective.</p><p><strong>Data sources: </strong>Cholesterol Treatment Trialists' Collaboration and United Kingdom Biobank data informed risk equations. United Kingdom primary and hospital care data informed healthcare costs (2020-1 Great British pounds); £1.10 standard or £1.68 higher-intensity generic statin therapy per 28 tablets; and Health Survey for England data informed health-related quality of life. Meta-analyses of trials and cohort studies informed the effects of statin therapies on cardiovascular events, incident diabetes, myopathy and rhabdomyolysis.</p><p><strong>Results: </strong>Across categories of participants 40-70 years old, lifetime use of standard statin therapy resulted in undiscounted 0.20-1.09 quality-adjusted life-years gained per person, and higher-intensity statin therapy added a further 0.03-0.20 quality-adjusted life-years per person. Among participants aged ≥ 70 years, lifetime standard statin was estimated to increase quality-adjusted life-years by 0.24-0.70 and higher-intensity statin by a further 0.04-0.13 quality-adjusted life-years per person. Benefits were larger among participants at higher cardiovascular disease risk or with higher low-density lipoprotein cholesterol. Standard statin therapy was cost-effective across all categories of people 40-70 years old, with incremental costs per quality-adjusted life-year gaine","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 79","pages":"1-134"},"PeriodicalIF":3.5,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647556/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791515","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cervical ripening at home or in hospital during induction of labour: the CHOICE prospective cohort study, process evaluation and economic analysis.","authors":"Mairead Black, Cassandra Yuill, Mairi Harkness, Sayem Ahmed, Linda Williams, Kathleen A Boyd, Maggie Reid, Amar Bhide, Neelam Heera, Jane Huddleston, Neena Modi, John Norrie, Dharmintra Pasupathy, Julia Sanders, Gordon C S Smith, Rosemary Townsend, Helen Cheyne, Christine McCourt, Sarah Stock","doi":"10.3310/LPYT7894","DOIUrl":"10.3310/LPYT7894","url":null,"abstract":"<p><strong>Background: </strong>Around one in three pregnant women undergoes induction of labour in the United Kingdom, usually preceded by in-hospital cervical ripening to soften and open the cervix.</p><p><strong>Objectives: </strong>This study set out to determine whether cervical ripening at home is within an acceptable safety margin of cervical ripening in hospital, is effective, acceptable and cost-effective from both National Health Service and service user perspectives.</p><p><strong>Design: </strong>The CHOICE study comprised a prospective multicentre observational cohort study using routinely collected data (CHOICE cohort), a process evaluation comprising a survey and nested case studies (qCHOICE) and a cost-effectiveness analysis. The CHOICE cohort set out to compare outcomes of cervical ripening using dinoprostone (a prostaglandin) at home with in-hospital cervical ripening from 39 weeks of gestation. Electronic maternity record data were collected from 26 maternity units. Following pilot analysis, the primary comparison was changed to ensure feasibility and to reflect current practice, comparing home cervical ripening using a balloon catheter with in-hospital cervical ripening using any prostaglandin from 37 weeks of gestation. Analysis involved multiple logistic regression for the primary outcome and descriptive statistics for all other outcomes. The qCHOICE study reported descriptive statistics of quantitative survey data and thematic analysis of focus group and interview data. The economic analysis involved a decision-analytic model from a National Health Service and Personal Social Services perspective, populated with CHOICE cohort and published data. Secondary analysis explored the patient perspective utilising cost estimates from qCHOICE data.</p><p><strong>Setting: </strong>Twenty-six United Kingdom maternity units.</p><p><strong>Participants: </strong>Women with singleton pregnancies at or beyond 37 weeks of gestation having induction with details of cervical ripening method and location recorded.</p><p><strong>Main outcome measures: </strong>Neonatal unit admission within 48 hours of birth for 48 hours or more.</p><p><strong>Qchoice: </strong>Maternal and staff experience of cervical ripening.</p><p><strong>Economic analysis: </strong>Incremental cost per neonatal unit admission within 48 hours of birth avoided.</p><p><strong>Data sources: </strong>Electronic maternity records from 26 maternity units; survey and interviews with service users/maternity staff; focus groups with maternity staff; published literature on economic aspects.</p><p><strong>Results: </strong>CHOICE cohort: A total of 515 women underwent balloon cervical ripening at home and 4332 underwent in-hospital cervical ripening using prostaglandin in hospitals that did not offer home cervical ripening. Neonatal unit admission within 48 hours of birth for 48 hours or more following home cervical ripening with balloon was not increased compared with in-hospital c","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 81","pages":"1-142"},"PeriodicalIF":3.5,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11683663/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142871914","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}