Health technology assessment最新文献

{"title":"Evaluation of prognostic models to improve prediction of metastasis in patients following potentially curative treatment for primary colorectal cancer: the PROSPECT trial.","authors":"Vicky Goh, Susan Mallett, Manuel Rodriguez-Justo, Victor Boulter, Rob Glynne-Jones, Saif Khan, Sarah Lessels, Dominic Patel, Davide Prezzi, Stuart Taylor, Steve Halligan","doi":"10.3310/BTMT7049","DOIUrl":"https://doi.org/10.3310/BTMT7049","url":null,"abstract":"<p><strong>Background: </strong>Despite apparently curative treatment, many patients with colorectal cancer develop subsequent metastatic disease. Current prognostic models are criticised because they are based on standard staging and omit novel biomarkers. Improved prognostication is an unmet need.</p><p><strong>Objectives: </strong>To improve prognostication for colorectal cancer by developing a baseline multivariable model of standard clinicopathological predictors, and to then improve prediction via addition of promising novel imaging, genetic and immunohistochemical biomarkers.</p><p><strong>Design: </strong>Prospective multicentre cohort.</p><p><strong>Setting: </strong>Thirteen National Health Service hospitals.</p><p><strong>Participants: </strong>Consecutive adult patients with colorectal cancer.</p><p><strong>Interventions: </strong>Collection of prespecified standard clinicopathological variables and more novel imaging, genetic and immunohistochemical biomarkers, followed by 3-year follow-up to identify postoperative metastasis.</p><p><strong>Main outcome: </strong>Best multivariable prognostic model including perfusion computed tomography compared with tumour/node staging. Secondary outcomes: Additive benefit of perfusion computed tomography and other biomarkers to best baseline model comprising standard clinicopathological predictors; measurement variability between local and central review; biological relationships between perfusion computed tomography and pathology variables.</p><p><strong>Results: </strong>Between 2011 and 2016, 448 participants were recruited; 122 (27%) were withdrawn, leaving 326 (226 male, 100 female; mean ± standard deviation 66 ± 10.7 years); 183 (56%) had rectal cancer. Most cancers were locally advanced [≥ T3 stage, 227 (70%)]; 151 (46%) were node-positive (≥ N1 stage); 306 (94%) had surgery; 79 (24%) had neoadjuvant therapy. The resection margin was positive in 15 (5%); 93 (28%) had venous invasion; 125 (38%) had postoperative adjuvant chemotherapy; 81 (25%, 57 male) developed recurrent disease. Prediction of recurrent disease by the baseline clinicopathological time-to-event Weibull multivariable model (age, sex, tumour/node stage, tumour size and location, treatment, venous invasion) was superior to tumour/node staging: sensitivity: 0.57 (95% confidence interval 0.45 to 0.68), specificity 0.74 (95% confidence interval 0.68 to 0.79) versus sensitivity 0.56 (95% confidence interval 0.44 to 0.67), specificity 0.58 (95% confidence interval 0.51 to 0.64), respectively. Addition of perfusion computed tomography variables did not improve prediction significantly: <i>c</i>-statistic: 0.77 (95% confidence interval 0.71 to 0.83) versus 0.76 (95% confidence interval 0.70 to 0.82). Perfusion computed tomography parameters did not differ significantly between patients with and without recurrence (e.g. mean ± standard deviation blood flow of 60.3 ± 24.2 vs. 61.7 ± 34.2 ml/minute/100 ml). Furthermore, baseline model pr","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 8","pages":"1-91"},"PeriodicalIF":3.5,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12010235/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144003180","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 proliferative diabetic retinopathy: a systematic review and individual participant data meta-analysis.","authors":"Mark Simmonds, Alexis Llewellyn, Ruth Walker, Helen Fulbright, Matthew Walton, Rob Hodgson, Laura Bojke, Lesley Stewart, Sofia Dias, Thomas Rush, João Pereira Figueira, Sobha Sivaprasad, John G Lawrenson, Tunde Peto, David Steel","doi":"10.3310/MJYP6578","DOIUrl":"10.3310/MJYP6578","url":null,"abstract":"<p><strong>Background: </strong>Proliferative diabetic retinopathy is a major cause of sight loss in people with diabetes, with a high risk of vitreous haemorrhage, tractional retinal detachment and other complications. Panretinal photocoagulation is the primary established 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 proliferative diabetic retinopathy.</p><p><strong>Objective: </strong>To investigate the efficacy and safety of anti-vascular endothelial growth factor therapy for the treatment of proliferative diabetic retinopathy when compared to panretinal photocoagulation.</p><p><strong>Methods: </strong>A systematic review and network meta-analysis of randomised controlled trials comparing anti-vascular endothelial growth factor (alone or in combination) to panretinal photocoagulation in people with proliferative 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. Key outcomes were best corrected visual acuity, diabetic macular oedema and vitreous haemorrhage. Individual participant data were obtained and analysed for three large, high-quality trials in combination with published data from other trials. Network meta-analyses of best corrected visual acuity and meta-analyses of other outcomes combined individual participant data with published data from other trials; regression analyses against patient covariates used just the individual participant data.</p><p><strong>Results: </strong>Twelve trials were included: one of aflibercept, five of bevacizumab and six of ranibizumab. Individual participant data were available from 1 aflibercept and 2 ranibizumab trials, representing 624 patients (33% of the total). When considered together, anti-vascular endothelial growth factors produced a modest, but not clinically meaningful, benefit over panretinal photocoagulation in best corrected visual acuity, after 1 year of follow-up (mean difference in logarithm of the minimum angle of resolution -0.116, 95% credible interval -0.183 to -0.038). There was no clear evidence of a difference in effectiveness between the anti-vascular endothelial growth factors. The benefit of anti-vascular endothelial growth factor appears to decline over time. Analysis of the individual participant data trials suggested that anti-vascular endothelial growth factor therapy may be more effective in people with poorer visual acuity, in those who have vitreous haemorrhage and, possibly, in people with poorer vision generally. Anti-vascular endothelial growth factor was superior to panretinal photocoagulation at preventing macular oedema after 1 year (relative risk 0.48, 95% confidence interval 0.28 to 0.83) and possibly at preventing vitreous haemorrhage (relative risk 0.72, 95% confidence interval 0.47 to 1.10). Anti-vascular endoth","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-75"},"PeriodicalIF":4.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11995242/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143788268","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":"Stopping anticoagulation for isolated or incidental subsegmental pulmonary embolism: the challenges and lessons from the STOPAPE RCT.","authors":"Daniel Lasserson, Pooja Gaddu, Samir Mehta, Agnieszka Ignatowicz, Sheila Greenfield, Clare Prince, Carole Cummins, Graham Robinson, Jonathan Rodrigues, Simon Noble, Susan Jowett, Mark Toshner, Michael Newnham, Alice Turner","doi":"10.3310/UGHF6892","DOIUrl":"https://doi.org/10.3310/UGHF6892","url":null,"abstract":"<p><strong>Background: </strong>The increasing use of computed tomography pulmonary angiography to investigate patients with suspected pulmonary embolism has led to an increase in diagnosis of small subsegmental pulmonary embolism, which is rarely detectable with nuclear medicine-based imaging, the standard imaging modality prior to the development of computed tomography pulmonary angiography. The case fatality of pulmonary embolism has fallen in line with the increase in subsegmental pulmonary embolism diagnoses from computed tomography pulmonary angiography suggesting that we may be over-diagnosing pulmonary embolism (i.e. we may be diagnosing mild forms of pulmonary embolism which may not need any treatment). Given that full anticoagulation has significant side effects of bleeding and subsegmental pulmonary embolism was not commonly diagnosed previously with nuclear medicine imaging (and therefore left predominantly untreated prior to computed tomography pulmonary angiography scanning), there is growing equipoise about the value of full anticoagulation for patients with subsegmental pulmonary embolism.</p><p><strong>Methods: </strong>We tried to undertake an open randomised trial with blinded end-point adjudication that recruited patients diagnosed with subsegmental pulmonary embolism without evidence of thrombus in the leg veins, termed 'isolated subsegmental pulmonary embolism'. We allocated patients with isolated subsegmental pulmonary embolism to either continuing with at least 3 months of full-dose anticoagulation (standard care) or stopping anticoagulation completely, unless they had a temporary hospital admission where prophylactic (i.e. preventative doses) of anticoagulation is standard practice. In addition, we interviewed patients and clinicians about their views on stopping anticoagulation for isolated subsegmental pulmonary embolism which would be a substantial change from current practice. We planned to assess the accuracy of isolated subsegmental pulmonary embolism diagnoses from computed tomography pulmonary angiographies.</p><p><strong>Results: </strong>The trial was stopped prematurely due to low recruitment. This was due to a combination of insufficient trial sites, problems with identifying patients who were suitable to be recruited at the time of acute assessment in hospital, the impact of COVID-19 on research infrastructure and a lower prevalence than had been predicted based on published studies. Our interview study showed that the intervention (i.e. changing practice to stopping treatment) is feasible, although there were concerns raised about safety, which a trial would be needed to address. We did not have sufficient trial participants to determine accuracy of initial isolated subsegmental pulmonary embolism diagnoses.</p><p><strong>Conclusion: </strong>Although we were not able to answer the question of whether it is clinically effective and cost-effective to stop anticoagulating patients with isolated subsegmental pu","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 11","pages":"1-12"},"PeriodicalIF":3.5,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12010252/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144019691","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":"Supporting self-management with an internet intervention for low back pain in primary care: a RCT (SupportBack 2).","authors":"Adam W A Geraghty, Taeko Becque, Lisa C Roberts, Jonathan Hill, Nadine E Foster, Lucy Yardley, Beth Stuart, David A Turner, Gareth Griffiths, Frances Webley, Lorraine Durcan, Alannah Morgan, Stephanie Hughes, Sarah Bathers, Stephanie Butler-Walley, Simon Wathall, Gemma Mansell, Malcolm White, Firoza Davies, Paul Little","doi":"10.3310/GDPS2418","DOIUrl":"https://doi.org/10.3310/GDPS2418","url":null,"abstract":"<p><strong>Background: </strong>Low back pain is highly prevalent and a leading cause of disability. Internet-delivered interventions may provide rapid and scalable support for behavioural self-management. There is a need to determine the effectiveness of highly accessible, internet-delivered support for self-management of low back pain.</p><p><strong>Objective: </strong>To determine the clinical and cost-effectiveness of an accessible internet intervention, with and without physiotherapist telephone support, on low back pain-related disability.</p><p><strong>Design: </strong>A multicentre, pragmatic, three parallel-arm randomised controlled trial with parallel economic evaluation.</p><p><strong>Setting: </strong>Participants were recruited from 179 United Kingdom primary care practices.</p><p><strong>Participants: </strong>Participants had current low back pain without indicators of serious spinal pathology.</p><p><strong>Interventions: </strong>Participants were block randomised by a computer algorithm (stratified by severity and centre) to one of three trial arms: (1) usual care, (2) usual care + internet intervention and (3) usual care + internet intervention + telephone support. 'SupportBack' was an accessible internet intervention. A physiotherapist telephone support protocol was integrated with the internet programme, creating a combined intervention with three brief calls from a physiotherapist.</p><p><strong>Outcomes: </strong>The primary outcome was low back pain-related disability over 12 months using the Roland-Morris Disability Questionnaire with measures at 6 weeks, 3, 6 and 12 months. Analyses used repeated measures over 12 months, were by intention to treat and used 97.5% confidence intervals. The economic evaluation estimated costs and effects from the National Health Service perspective. A cost-utility study was conducted using quality-adjusted life-years estimated from the EuroQol-5 Dimensions, five-level version. A cost-effectiveness study estimated cost per point improvement in the Roland-Morris Disability Questionnaire. Costs were estimated using data from general practice patient records. Researchers involved in data collection and statistical analysis were blind to group allocation.</p><p><strong>Results: </strong>Eight hundred and twenty-five participants were randomised (274 to usual primary care, 275 to usual care + internet intervention and 276 to the physiotherapist-supported arm). Follow-up rates were 83% at 6 weeks, 72% at 3 months, 70% at 6 months and 79% at 12 months. For the primary analysis, 736 participants were analysed (249 usual care, 245 internet intervention, 242 telephone support). There was a small reduction in the Roland-Morris Disability Questionnaire over 12 months compared to usual care following the internet intervention without physiotherapist support (adjusted mean difference of -0.5, 97.5% confidence interval -1.2 to 0.2; <i>p</i> = 0.085) and the internet intervention with physiotherapist suppor","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 7","pages":"1-90"},"PeriodicalIF":3.5,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12010237/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143965512","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":"Recent research in myalgic encephalomyelitis/chronic fatigue syndrome: an evidence map.","authors":"Alex Todhunter-Brown, Pauline Campbell, Cathryn Broderick, Julie Cowie, Bridget Davis, Candida Fenton, Sarah Markham, Ceri Sellers, Katie Thomson","doi":"10.3310/BTBD8846","DOIUrl":"10.3310/BTBD8846","url":null,"abstract":"<p><strong>Background: </strong>Myalgic encephalomyelitis/chronic fatigue syndrome is a chronic condition, classified by the World Health Organization as a nervous system disease, impacting around 17 million people worldwide. Presentation involves persistent fatigue and postexertional malaise (a worsening of symptoms after minimal exertion) and a wide range of other symptoms. Case definitions have historically varied; postexertional malaise is a core diagnostic criterion in current definitions. In 2022, a James Lind Alliance Priority Setting Partnership established research priorities relating to myalgic encephalomyelitis/chronic fatigue syndrome.</p><p><strong>Objective(s): </strong>We created a map of myalgic encephalomyelitis/chronic fatigue syndrome evidence (2018-23), showing the volume and key characteristics of recent research in this field. We considered diagnostic criteria and how current research maps against the James Lind Alliance Priority Setting Partnership research priorities.</p><p><strong>Methods: </strong>Using a predefined protocol, we conducted a comprehensive search of Cochrane, MEDLINE, EMBASE and Cumulative Index to Nursing and Allied Health Literature. We included all English-language research studies published between January 2018 and May 2023. Two reviewers independently applied inclusion criteria with consensus involving additional reviewers. Studies including people diagnosed with myalgic encephalomyelitis/chronic fatigue syndrome using any criteria (including self-report), of any age and in any setting were eligible. Studies with < 10 myalgic encephalomyelitis/chronic fatigue syndrome participants were excluded. Data extraction, coding of topics (involving stakeholder consultation) and methodological quality assessment of systematic reviews (using A MeaSurement Tool to Assess systematic Reviews 2) was conducted independently by two reviewers, with disagreements resolved by a third reviewer. Studies were presented in an evidence map.</p><p><strong>Results: </strong>Of the 11,278 identified studies, 742 met the selection criteria, but only 639 provided sufficient data for inclusion in the evidence map. These reported data from approximately 610,000 people with myalgic encephalomyelitis/chronic fatigue syndrome. There were 81 systematic reviews, 72 experimental studies, 423 observational studies and 63 studies with other designs. Most studies (94%) were from high-income countries. Reporting of participant details was poor; 16% did not report gender, 74% did not report ethnicity and 81% did not report the severity of myalgic encephalomyelitis/chronic fatigue syndrome. Forty-four per cent of studies used multiple diagnostic criteria, 16% did not specify criteria, 24% used a single criterion not requiring postexertional malaise and 10% used a single criterion requiring postexertional malaise. Most (89%) systematic reviews had a low methodological quality. Five main topics (37 subtopics) were included in the evidence map. Of","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-78"},"PeriodicalIF":3.5,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11973615/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143752305","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":"Gait Rehabilitation for Early rheumatoid Arthritis Trial (GREAT): lessons learnt from a mixed-methods feasibility study and internal pilot trial.","authors":"Gordon J Hendry, Lindsay Bearne, Linda Fenocchi, Nadine E Foster, Sally Gates, Emma Godfrey, Samantha Hider, Lisa Jolly, Helen Mason, Alex McConnachie, Iain McInnes, Aimie Patience, Yeliz Prior, Catherine Sackley, Mandeep Sekhon, Bethany Stanley, Jason Vickers, Jim Woodburn, Martijn Pm Steultjens","doi":"10.3310/XBDJ8546","DOIUrl":"10.3310/XBDJ8546","url":null,"abstract":"<p><strong>Background: </strong>People with rheumatoid arthritis experience foot and lower limb pain due to active synovitis, resulting in impaired lower limb function. Earlier intervention may help with prevention of functional decline. The aims of this research were to develop and evaluate a new gait rehabilitation intervention for people with early rheumatoid arthritis, evaluate its feasibility, and to test whether or not gait rehabilitation plus usual care is more clinically and cost-effective than usual care alone.</p><p><strong>Design and methods: </strong>We undertook a single-arm, repeated-measures, pre- and post-intervention, mixed-methods feasibility study with embedded qualitative components. We planned to undertake a pragmatic, two-arm, multicentre, superiority randomised controlled trial, with health economic evaluation, process evaluation and internal pilot.</p><p><strong>Setting and participants: </strong>Participants with early rheumatoid arthritis (< 2 years post diagnosis) were identified from early arthritis and rheumatology outpatient clinics and referred for intervention in either podiatry or physiotherapy clinics.</p><p><strong>Intervention(s): </strong>Participants were randomised to a gait rehabilitation programme (Gait Rehabilitation Early Arthritis Trial Strides) involving a six-task gait circuit. Sessions were underpinned by motivational interviewing to facilitate behaviour change, supported by trained physiotherapists or podiatrists for a minimum of two sessions. Both groups received their normal usual care from the rheumatology multidisciplinary team.</p><p><strong>Main outcome measures: </strong>Outcome measures for the feasibility study were intervention acceptability, adherence using the Exercise Adherence Rating Scale and fidelity using the Motivational Interviewing Treatment Integrity Scale. The main outcome measure for the internal pilot/randomised controlled trial was the Foot Function Index disability subscale. Outcomes were measured at baseline, 3 months, 6 months and 12 months. Other outcomes: intervention acceptability questionnaire, Exercise Adherence Rating Scale, exercise treatment beliefs via the Theory of Planned Behaviour Questionnaire, intervention fidelity (Motivational Interviewing Treatment Integrity Scale), health-related quality of life (EuroQol-5 Dimensions, five-level score).</p><p><strong>Results: </strong>Thirty-five participants were recruited for feasibility and 23 (65.7%) completed 12-week follow-up. Intervention acceptability was excellent: 21/23 were confident that it could help and would recommend it and 22/23 indicated it made sense to them. Adherence was good, with a median (interquartile range) Exercise Adherence Rating Scale score of 17/24 (12.5-22.5). Twelve participants' and nine therapists' interviews confirmed intervention acceptability, identified perceptions of benefit, but highlighted some barriers to completion. Motivational Interviewing Treatment Integrity Scale scores dem","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-48"},"PeriodicalIF":3.5,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11973904/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143752298","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":"Experiences of support to return to work after stroke: longitudinal case studies from RETAKE trial.","authors":"Diane Trusson, Katie Powers, Kathryn Radford, Audrey Bowen, Kristelle Craven, Jain Holmes, Rebecca Lindley, Christopher McKevitt, Julie Phillips, Ellen Thompson, Caroline Watkins, David J Clarke","doi":"10.3310/WRKS9661","DOIUrl":"10.3310/WRKS9661","url":null,"abstract":"<p><strong>Background: </strong>Returning to work after stroke has physical, psychological and financial benefits for stroke survivors. However, global evidence estimates return-to-work rates 1 year post stroke at < 50%. Although its importance is acknowledged by policy-makers and healthcare providers, vocational rehabilitation is not always part of National Health Service usual care post stroke. Currently, there is limited evidence of the effectiveness of return-to-work support interventions. RETurn to work After stroKE was a multicentre individually randomised controlled pragmatic trial, with embedded process and health economic evaluations. RETurn to work After stroKE aimed to establish whether Early Stroke Specialist Vocational Rehabilitation plus usual care improves the likelihood of return to work at 12 months post stroke compared to usual care alone. As part of an embedded process evaluation, longitudinal case studies enabled exploration of participants' experiences of support to return to work in the trial.</p><p><strong>Objectives: </strong>This article aims to understand participants' experiences of being supported to return to work and explores the social and structural factors which support, or act as barriers to, implementation of the Early Stroke Specialist Vocational Rehabilitation intervention.</p><p><strong>Method: </strong>A longitudinal case-study approach was used to compare experiences of post-stroke return-to-work support received over 12 months by 15 participants who received the Early Stroke Specialist Vocational Rehabilitation intervention plus usual care, and 11 participants who received usual care only. Data were gathered at three time points using follow-up questionnaires, health records, intervention delivery records and semistructured interviews with participants and seven nominated informal carers. Interviews were also conducted with 1 employer and 11 occupational therapists delivering the intervention.</p><p><strong>Setting: </strong>Sixteen National Health Service sites across England and Wales.</p><p><strong>Findings: </strong>In the intervention arm, stroke survivors, carers and employers reported benefits from information and support from the treating occupational therapist to facilitate acceptance of, and adaptation to, post-stroke abilities. Participants also valued occupational therapists' provision of sustained and tailored vocational rehabilitation, co-ordinating their care and advocating for them in return-to-work discussions with their employers. Those unable to return to their previous employment were supported to consider alternative options. In contrast, participants who received usual care only reported feeling abandoned when community rehabilitation support ended, typically after 2-8 weeks. Usual care largely focused on restoring physical function, leaving these participants struggling to find return-to-work information, advice and support. Longitudinal case studies enabled psychosocial and environm","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-27"},"PeriodicalIF":3.5,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11973616/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143752287","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":"Management of diarrhoea in patients with stable ulcerative colitis with low FODMAP diet, amitriptyline, ondansetron or loperamide: the MODULATE RCT.","authors":"Lauren A Moreau, Alexander Charles Ford, Matthew James Brookes, Sandra Graca, Elspeth Guthrie, Suzanne Hartley, Lesley Houghton, Karen Kemp, Nicholas A Kennedy, Yvonne McKenzie, Delia Muir, Pei Loo Ow, Christopher Probert, Emma Pryde, Christopher Taylor, Thomas A Willis, Alexandra Wright-Hughes, Amanda J Farrin","doi":"10.3310/GHFE4871","DOIUrl":"10.3310/GHFE4871","url":null,"abstract":"<p><strong>Background: </strong>Many patients with ulcerative colitis report ongoing diarrhoea even when their disease is stable and in remission.</p><p><strong>Design: </strong>MODULATE was a pragmatic, multicentre, seamless, adaptive, phase 2/3 open-label, parallel-group, multiarm multistage randomised controlled trial.</p><p><strong>Setting and participants: </strong>People aged over 18 years with stable ulcerative colitis who had diarrhoea, recruited from secondary care sites in the United Kingdom.</p><p><strong>Interventions: </strong>The control arm consisted of modified first-line dietary advice given to all patients with irritable bowel syndrome; the first interventional arm was amitriptyline, a tricyclic antidepressant, which at low doses slows colonic transit; the second intervention was loperamide, an antidiarrhoeal drug also thought to slow colonic transit; the third was ondansetron, an antiemetic thought to slow colonic transit; and the fourth was a diet low in fermentable oligo-, di-, and mono-saccharides and polyols, which is thought to reduce bloating and gas within the small intestine. All patients randomised to an interventional arm were to receive treatment for 6 months.</p><p><strong>Main outcome measures: primary outcome measures: </strong>Phase 2: Improvement in diarrhoea measured using the Gastrointestinal Symptom Rating Scale-irritable bowel syndrome questionnaire at 8 weeks post randomisation: improvement defined as those reporting minor discomfort from diarrhoea or less (scoring ≤ 2 on the diarrhoea subscale).</p><p><strong>Secondary outcome measures: </strong>Phases 2 and 3: Measured at both 8 weeks and 6 months: Improvement in diarrhoea measured using the Gastrointestinal Symptom Rating Scale-irritable bowel syndrome. Blood for C-reactive protein, stool for faecal calprotectin at 6 months only, reviewing case notes for escalation of medical therapy for ulcerative colitis. Anxiety and depression, via the Hospital Anxiety and Depression Scale.</p><p><strong>Results: </strong>The MODULATE trial opened in December 2021 and closed in January 2023. Of the eight secondary care sites that completed contracting, only four opened to recruitment during this time, and one person was randomised. Trial timelines coincided with the start of the COVID-19 pandemic, causing substantial delays and, ultimately, its early closure. During this time, the trial underwent two major redesign phases, enabling a fully remote participant pathway incorporating electronic consent, remote data capture, posted blood and stool sample kits for eligibility screening, delivery of the dietary intervention via telephone or video call platform, postage of trial investigational medicinal products directly to participants' homes and all trial follow-up appointments conducted via telephone. The second phase of redesign pushed the trial towards a fully decentralised model. However, this stage was not implemented due to the decision to close the trial early.</p><","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-30"},"PeriodicalIF":3.5,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11931405/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143624415","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":"The acceptability of blood spot screening and genome sequencing in newborn screening: a systematic review examining evidence and frameworks.","authors":"Duncan Chambers, Susan Baxter, Anastasios Bastounis, Katherine Jones, Burak Kundakci, Anna Cantrell, Andrew Booth","doi":"10.3310/RTPQ2268","DOIUrl":"10.3310/RTPQ2268","url":null,"abstract":"<p><strong>Background: </strong>Population-wide newborn blood spot screening programmes are a successful public health intervention used to detect whether the baby is at risk of certain rare conditions, with the aim of earlier diagnosis and provision of optimal care and treatment. Evaluating candidate conditions to include in newborn blood spot and genetic sequencing raises questions regarding acceptability to parents/carers.</p><p><strong>Methods: </strong>In the context of the possible expansion of the newborn blood spot screening programme in the United Kingdom, this review aimed to systematically review research on the acceptability to parents of newborn blood spot screening and genetic sequencing. A protocol was developed prior to commencing the review and was registered on the PROSPERO database. A team of researchers carried out the review, with checking at all stages carried out by at least two individuals. We included research published after 2013 with participants who were pregnant or a recent parent of a newborn and were resident in a high-income country. We included quantitative and qualitative studies that investigated the acceptability to parents/carers of newborn blood spot screening or genetic sequencing. Quantitative studies were narratively synthesised, and theories/frameworks identified and evaluated. Qualitative studies were analysed for recurring themes, and a meta-synthesis was carried out to compare and contrast these two types of data. We quality appraised included articles using tools appropriate for their study design.</p><p><strong>Results: </strong>Searches were carried out in September to November 2023 and screening identified 25 relevant research articles. Just over half were from North America, with four existing reviews and nine qualitative studies. Domains of acceptability described in the literature were: support for screening; level of anxiety, information and knowledge; consent; views of the procedure; and support after screening. The research indicated consensus support for blood spot screening, and for expanding to some other conditions, although some parental anxiety was reported. Parents/carers mostly perceived that they had received sufficient information, but the timing of this could be improved. While parents indicated interest in genomic screening, studies highlighted the need for clearer consent procedures and greater support for parents following genomic screening than for blood spot screening. Only three included studies reported using any kind of theoretical framework.</p><p><strong>Discussion: </strong>Most parents/carers found newborn blood spot screening programmes to be acceptable and favoured their large-scale implementation. A minority of parents/carers expressed concerns regarding the acceptability of processes underpinning newborn blood spot screening, such as consent, the timing of receiving information and support available after testing. More research is needed regarding the acceptability ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-53"},"PeriodicalIF":3.5,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11931406/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143630329","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":"Benefits of aldosterone receptor antagonism in chronic kidney disease: the BARACK-D RCT.","authors":"F D Richard Hobbs, Richard McManus, Clare Taylor, Nicholas Jones, Joy Rahman, Jane Wolstenholme, Louise Jones, Jennifer Hirst, Sam Mort, Ly-Mee Yu","doi":"10.3310/PYFT6977","DOIUrl":"10.3310/PYFT6977","url":null,"abstract":"<p><strong>Background: </strong>Chronic kidney disease affects around 10% of the global population and is associated with significant risk of progression to end-stage renal disease and vascular events. Aldosterone receptor antagonists such as spironolactone have shown prognostic benefits in patients with heart failure, but effects on patients with chronic kidney disease are uncertain.</p><p><strong>Objectives: </strong>To determine the effect of low-dose spironolactone on mortality and cardiovascular outcomes in people with chronic kidney disease stage 3b.</p><p><strong>Design: </strong>Prospective randomised open blinded end-point trial.</p><p><strong>Settings: </strong>Three hundred and twenty-nine general practitioner practices throughout the United Kingdom.</p><p><strong>Participants: </strong>Patients meeting the criteria for chronic kidney disease stage 3b (estimated glomerular filtration rate 30-44 ml/minute/1.73 m<sup>2</sup>) according to National Institute for Health and Care Excellence guidelines were recruited. Due to the higher than anticipated measurement error/fluctuations, the eligible range was extended to 30-50 ml/minute/1.73 m<sup>2</sup> following the initial recruitment period.</p><p><strong>Intervention: </strong>Participants were randomised 1 : 1 to receive either spironolactone 25 mg once daily in addition to standard care, or standard care only.</p><p><strong>Outcome measures: </strong>Primary outcome was the first occurring of all-cause mortality, first hospitalisation for heart disease (coronary heart disease, arrhythmia, atrial fibrillation, sudden death, failed sudden death), stroke, heart failure, transient ischaemic attack or peripheral arterial disease, or first occurrence of any condition not listed at baseline. Secondary outcome measures included changes in blood pressure, renal function, B-type natriuretic peptide, incidence of hyperkalaemia and treatment costs and benefits.</p><p><strong>Results: </strong>One thousand four hundred and thirty-four participants were randomised of the 3022 planned. We found no evidence of differences between the intervention and control groups in terms of effectiveness with the primary combined vascular end points, nor with the secondary clinical outcomes, including progression in renal decline. These results were similar for the total treatment periods or a 3-year follow-up period as originally planned. More adverse events were experienced and more participants discontinued treatment in the intervention group. Two-thirds of participants randomised to spironolactone stopped treatment within six months because they met pre-specified safety stop criteria. The addition of low-dose spironolactone was estimated to have a cost per quality-adjusted life-year gained value above the National Institute for Health and Care Excellence's threshold of £30,000.</p><p><strong>Limitations: </strong>Main limitations were difficulties in recruiting eligible participants resulting in an underpowered ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 5","pages":"1-130"},"PeriodicalIF":3.5,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11931407/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143663338","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}