Health technology assessment最新文献

{"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":"373-402"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","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":"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":3.4,"publicationDate":"2026-08-01","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}

{"title":"Methods and mechanisms for measuring and monitoring outcomes from newborn bloodspot screening: a scoping review.","authors":"Katie Scandrett, Jacqueline Dinnes, Breanna Morrison, April Coombe, Ridhi Agarwal, Isaac Adu Asare, Phoebe Mead, Andy De Souza, David Elliman, Silvia Lombardo, John Marshall, Sian Taylor-Phillips, Yemisi Takwoingi","doi":"10.3310/GJJD1717","DOIUrl":"10.3310/GJJD1717","url":null,"abstract":"<p><strong>Background: </strong>Newborn bloodspot screening offers the potential to detect rare diseases early, enabling timely treatment that can reduce mortality and morbidity. Generating evidence for rare diseases often depends on observational data, making it challenging to formulate recommendations for new screening programmes and evaluate the effectiveness of existing ones.</p><p><strong>Objective(s): </strong>To identify the range of methods and mechanisms used to measure and monitor outcomes from newborn screening programmes using a scoping review.</p><p><strong>Methods: </strong>We included studies published between 2019 and 2024, which evaluated a current or candidate newborn screening programme, or which reported outcomes in screen-detected cases. Studies were categorised into four groups: group 1 reported a comparison and follow-up; group 2 reported a comparison but no follow-up; group 3 reported no comparison with follow-up; and group 4 reported no comparison or follow-up. Data were extracted from a random sample of studies within each group; studies in group 1 were prioritised. Results were reported narratively according to study group. The review was conducted and reported according to current guidance for scoping reviews.</p><p><strong>Data sources: </strong>EMBASE (Ovid), MEDLINE (Ovid) and Science Citation Index (Web of Science - Clarivate).</p><p><strong>Results: </strong>We included 574 primary studies and extracted data from 178. Of the 75 studies in group 1, most compared screen-detected cases with controls (74%). Studies in this group used newborn bloodspot programme databases, registries or record review to identify participants and outcomes; only six (8%) reported use of record linkage. Studies in group 2 (<i>n</i> = 31) mostly reported comparisons of screening tests (25, 81%). Over half of studies in group 3 (<i>n</i> = 34) used newborn bloodspot programme databases to identify participants (53%) and outcomes (65%). A similar pattern was seen in the group 4 (<i>n</i> = 38). Studies reporting follow-up typically relied on retrospective record review or were not well reported. Across all study groups, data on accuracy, epidemiology and genetic variants were common. Studies in group 1 also reported on the effectiveness of newborn bloodspot screening (32/75, 43%), treatment effectiveness (20%) or harms of newborn bloodspot screening (3%).</p><p><strong>Limitations: </strong>Restricting data extraction to a random sample of studies risks missing novel methods or mechanisms.</p><p><strong>Conclusions: </strong>Many studies reported test accuracy metrics and genetic variants in newborn screening. Some data on programme effectiveness were identified, but assessment of potential harms remains limited, and methods for follow-up were poorly reported. Assessment of harms, including overdiagnosis and psychological impact, is crucial to ensuring a net benefit at the population level.</p><p><strong>Future work: </strong>In a second pha","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"221-268"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12862532/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146046461","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical and cost-effectiveness of medical management versus surgery for deep infiltrating endometriosis: synopsis from the DIAMOND RCT.","authors":"Kevin Cooper, Lynda Constable, Thenmalar Vadiveloo, Ayodeji Matuluko, Christine Kennedy, Sharon McCann, Seonaidh Cotton, Katie Gillies, Rebecca Bruce, Paul Smith, Graeme MacLennan, T Justin Clark","doi":"10.3310/GJKC5715","DOIUrl":"10.3310/GJKC5715","url":null,"abstract":"<p><strong>Background: </strong>Deep endometriosis causes significant pain which adversely affects quality of life and utilises healthcare and wider societal resources. Laparoscopic excision of endometriosis has shown to improve pain symptoms in observational series but 1 in 14 patients experience serious surgical complications. Medical management centres around hormonal treatment, which is less risky and has been shown to be efficacious but can cause troublesome side effects and is incompatible with conception. There are no randomised controlled trials providing conclusive comparative evidence on clinical and cost-effectiveness of these treatments.</p><p><strong>Objective(s): </strong>To compare the clinical and cost-effectiveness of laparoscopic surgery versus optimised medical treatment for managing deep endometriosis.</p><p><strong>Design and methods: </strong>A multicentre randomised controlled trial, with an internal pilot phase, and economic evaluation, to compare early planned laparoscopic surgery (first attempt at definitive surgery) with or without adjuvant medical treatment versus optimised medical management alone in women with deep endometriosis.</p><p><strong>Setting and participants: </strong>Women presenting with pelvic pain associated with surgically or radiologically confirmed deep endometriosis, suitable for either surgical or medical management, recruited and managed at accredited British Society for Gynaecological Endoscopy Endometriosis Centres.</p><p><strong>Interventions: </strong>Early planned laparoscopic surgery to excise deep endometriosis (with or without medical treatment) or medical management alone.</p><p><strong>Main outcome measures: </strong>The primary outcome was condition-specific quality of life measured using the pain domain of the Endometriosis Health Profile-30 at 18 months post randomisation. The primary health economic outcome was to be incremental cost per quality-adjusted life-year gained at 18 months. Secondary outcomes included quality of life (Endometriosis Health Profile-30), pain, complications, occupational and reproductive outcomes.</p><p><strong>Results: </strong>Three hundred and seventy-seven patients were screened, 103 were eligible and 18 were randomised. Of the eight patients allocated surgery, only one had had their surgery by the time of trial closure and six participants (2/4, 50% allocated surgery and 4/8, 50% allocated medical treatment) had reached the first trial end point at 3 months. No participant reached the primary outcome at 18 months post randomisation.</p><p><strong>Limitations: </strong>The overriding limitation was failure to recruit participants at a satisfactory rate resulting in a final sample of only 18 patients with a target of 320 (inflated to 400 to account for a projected 20% attrition rate). Given the nature of the intervention, it was not possible to blind either the care providers, investigators or participants to their allocated group.</p><p><strong>Conclusion","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"437-464"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12884356/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146099599","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":"Decompressive craniectomy versus craniotomy for patients undergoing surgical evacuation of an acute subdural hematoma: RESCUE-ASDH RCT and cost effectiveness.","authors":"Peter Hutchinson, Harry Mee, Hadie Adams, Midhun Mohan, Bhagavatula I Devi, Christopher Uff, Shumaila Hasan, Mark Wilson, Deepak Gupta, Diederik Bulters, Ardalan H Zolnourian, Catherine McMahon, Matthew Stovell, Yahia Al-Tamimi, Manoj Tewari, Manjul Tripathi, Simon Thomson, Edoardo Viaroli, Karen Caldwell, Antonio Belli, Andrew King, Adel Helmy, Ivan Timofeev, Sarah Pyne, Dhaval Shukla, Dhananjaya I Bhat, Andrew Maas, Franco Servadei, Geoffrey Manley, Garry Barton, David Turner, Natalia Igosheva, Alicia Gore, Tapiwa Tungamirai, Kerstin Wolf, Carol Davis-Wilkie, Kamila Walker, Michelle Jillings, Christopher Bushell, Carole Turner, David Menon, Barbara Gregson, Angelos Kolias","doi":"10.3310/GJPH0512","DOIUrl":"10.3310/GJPH0512","url":null,"abstract":"<p><strong>Background: </strong>Traumatic acute subdural haematomas often require surgical evacuation via craniotomy or decompressive craniectomy. Decompressive craniectomy may prevent intracranial hypertension; however, it is unclear whether it is associated with better outcomes.</p><p><strong>Objective: </strong>Multicentre, pragmatic, parallel-group randomised trial to compare the clinical and cost-effectiveness of decompressive craniectomy versus craniotomy for evacuation of acute subdural haematomas.</p><p><strong>Design: </strong>International, multicentre, pragmatic, parallel-group randomised trial with additional observational arm.</p><p><strong>Setting: </strong>Hospitals with neurosurgical services in the UK and internationally.</p><p><strong>Participants: </strong>Patients aged ≥ 16 years, with a diagnosis of acute subdural haematomas on a computed tomography scan that required evacuation with a large bone flap either by craniotomy or decompressive craniectomy according to the opinion of the admitting neurosurgeon.</p><p><strong>Interventions: </strong>The enrolled patients underwent acute subdural haematoma evacuation in the operating room under general anaesthesia. A large bone flap ipsilateral to the haematoma was raised, the dura opened and the haematoma evacuated. Other haematomas, such as contusions, were evacuated at the discretion of the surgeon. If clinically appropriate randomisation occurred, the bone flap was either replaced (craniotomy) or not replaced (decompressive craniectomy). Patients who could not be randomised were followed up in the observational arm.</p><p><strong>Main outcome measures: </strong>Primary outcome measure was the extended Glasgow Outcome Scale assessed 12 months post injury. An economic evaluation (based on UK participants) was undertaken to estimate the cost-effectiveness of craniotomy compared to decompressive craniectomy.</p><p><strong>Results: </strong>Four hundred and fifty patients were randomised: 228 to craniotomy and 222 to craniectomy - with the common odds ratio for the differences across the Glasgow Outcome Scale scores of 0.85 [95% confidence interval (0.6 to 1.18; <i>p</i> = 0.324)]. The results were similar at 6 months. At 12 months, death occurred in 30.2% of the craniotomy group versus 32.2% of the decompressive craniectomy group, vegetative state occurred in 2.3% versus 2.8%, and good recovery occurred in 25.6% and 19.9%, respectively. In the observed cohort, those who had a decompressive craniectomy had significantly worse outcomes at 6 and 12 months, but their baseline characteristics were different.</p><p><strong>Limitations: </strong>Clinicians were not blinded to the trial groups. Decompressive craniectomy was performed in 8.8% of patients allocated to the craniotomy group, and 5.4% of patients allocated to the decompressive craniectomy group underwent craniotomy. Intraoperative non-adherence with allocation did not influence the primary analysis, which was based on the intenti","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 66","pages":"1-96"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13548103/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148840209","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":"Initial assessment and management of adults with suspected acute respiratory infection: a rapid evidence synthesis of reviews and cost-effectiveness studies.","authors":"Ros Wade, Nyanar Jasmine Deng, Chinyereugo Umemneku-Chikere, Melissa Harden, Helen Fulbright, Robert Hodgson, Alison Eastwood, Rachel Churchill","doi":"10.3310/GRPL6978","DOIUrl":"10.3310/GRPL6978","url":null,"abstract":"<p><strong>Background: </strong>This work was undertaken to inform a National Institute for Health and Care Excellence guideline on the initial assessment of adults with suspected acute respiratory infection.</p><p><strong>Objective: </strong>To undertake a rapid evidence synthesis of systematic reviews and cost-effectiveness studies of signs, symptoms and early warning scores for the initial assessment of adults with suspected acute respiratory infection.</p><p><strong>Methods: </strong>MEDLINE, EMBASE and Cochrane Database of Systematic Reviews were searched for systematic reviews and MEDLINE, EMBASE, EconLit and National Health Service Economic Evaluation Database were searched for cost-effectiveness studies in May 2023. References of relevant studies were checked. Clinical outcomes of interest included escalation of care, antibiotic/antiviral use, time to resolution of symptoms, mortality and health-related quality of life. Risk of bias was assessed using the Risk of Bias in Systematic Reviews tool or the National Institute for Health and Care Excellence economic evaluations checklist. Results were summarised using narrative synthesis.</p><p><strong>Results: </strong>Nine systematic reviews and one cost-effectiveness study met eligibility criteria. Seven reviews assessed several early warning scores for patients with community- acquired pneumonia, one assessed early warning scores for nursing home-acquired pneumonia and one assessed individual signs/symptoms and the Centor score for patients with sore throat symptoms; all in face-to-face settings. Two good-quality reviews concluded that further research is needed to validate the CRB-65 in primary care/community settings. One also concluded that further research is needed on the Pneumonia Severity Index in community settings; however, the Pneumonia Severity Index requires data from tests not routinely conducted in community settings. One good-quality review concluded that National Early Warning Score appears to be useful in an emergency department/acute medical setting. One review (unclear quality) concluded that the Pneumonia Severity Index and CURB-65 appear useful in an emergency department setting. Two poor-quality reviews concluded that early warning scores can support clinical judgement and one poor-quality review found numerous problems with using early warning scores in a nursing home setting. A good-quality review concluded that individual signs and symptoms have a modest ability to diagnose streptococcal pharyngitis, and that the Centor score can enhance appropriate prescribing of antibiotics. The cost-effectiveness study assessed clinical scores and rapid antigen detection tests for sore throat, compared to delayed antibiotic prescribing. The study concluded that the clinical score is a cost-effective approach when compared to delayed prescribing and rapid antigen testing.</p><p><strong>Conclusions: </strong>Several early warning scores have been evaluated in adults with suspected a","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"579-632"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13572989/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285838","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":"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":"617-642"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","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":"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":"375-428"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","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":"Ambulatory Oxygen for Pulmonary Fibrosis (OxyPuF): a randomised controlled trial and acceptability study.","authors":"Rachel L Adams, Alisha Maher, Nicola Gale, Anjali Crawshaw, David Thickett, Alice M Turner","doi":"10.3310/TWKS4194","DOIUrl":"10.3310/TWKS4194","url":null,"abstract":"<p><strong>Introduction: </strong>Idiopathic pulmonary fibrosis is a devastating condition of unknown cause that results in progressive, irreversible scarring of the lung, manifesting as breathlessness and dry cough. Idiopathic pulmonary fibrosis is thought to be responsible for as many as 1 in 100 deaths in the United Kingdom, killing 5300 people a year. Ambulatory oxygen therapy is commonly used in idiopathic pulmonary fibrosis to relieve exertional breathlessness, although evidence to support this strategy is lacking. This pragmatic randomised controlled trial was planned to test whether use of ambulatory oxygen therapy is beneficial in people with idiopathic pulmonary fibrosis.</p><p><strong>Methods: </strong>We planned a randomised controlled trial in 260 patients with idiopathic pulmonary fibrosis who are breathless on exertion and do not meet criteria for long-term oxygen therapy, randomising in a 1 : 1 ratio between ambulatory oxygen therapy and best supportive care. Primary outcome was a quality-of-life questionnaire validated in pulmonary fibrosis, the King's Brief Interstitial Lung Disease questionnaire, measured at 6 months. We calculated our sample size based on the minimum clinically important difference of four units and standard deviation equal to 8.85 in King's Brief Interstitial Lung Disease questionnaire; assuming power of 90% and 5% two-sided significance level, thus required 130 per arm, after accounting for 20% dropout. The trials unit's web-based randomisation algorithm minimises on factors potentially influencing response to ambulatory oxygen therapy, such as severity of idiopathic pulmonary fibrosis, desaturation to < 88% present on walking, current or recent (within 6 months) pulmonary rehabilitation, and recruitment centre. Secondary outcomes included symptoms, exercise capacity and cost-effectiveness. A process evaluation included assessment of trial fidelity and acceptability of the intervention with use of qualitative research methods and arts approaches with patients and staff. Qualitative interviews were conducted with patients from the Ambulatory Oxygen for Pulmonary Fibrosis trial and the idiopathic pulmonary fibrosis patient support group Action for Pulmonary Fibrosis, and stakeholders: healthcare professionals and policy-makers. Interviews were audio-recorded, transcribed clean verbatim. Photovoice methodology was conducted with patients. A workshop prior to data collection informed and guided data collection and analysis. Traditional qualitative analysis and arts-based coproduction analysis approaches were used to produce a short film. An economic model was planned but could not occur due to early termination.</p><p><strong>Results: </strong>The trial was stopped prematurely due to low recruitment. This was due to a combination of the impact of COVID-19 on research infrastructure, financial issues for sites with the payment structure for the trial and lack of equipoise which limited site recruitment. Seven out","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"403-436"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376201/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144560001","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Home-testing devices for diagnosing obstructive sleep apnoea hypopnoea syndrome - a systematic review and economic evaluation.","authors":"Jaime Peters, Jonathan Shepherd, Emma Maund, Bogdan Grigore, Lois Woods, Joanne Lord, David Alexander Scott, Christopher Hyde","doi":"10.3310/GJJS3014","DOIUrl":"10.3310/GJJS3014","url":null,"abstract":"<p><strong>Background: </strong>Obstructive sleep apnoea and hypopnea syndrome is a sleep-related breathing disorder. Diagnostic sleep studies for obstructive sleep apnoea and hypopnoea syndrome are conducted overnight in clinic using oximetry, respiratory polygraphy and polysomnography. Portable novel devices have been developed to facilitate home testing.</p><p><strong>Objectives: </strong>To conduct: a systematic review of clinical effectiveness of novel home-testing devices in people with suspected obstructive sleep apnoea and hypopnoea syndrome a health economic evaluation modelling the incremental cost-effectiveness of novel home-testing devices compared to home respiratory polygraphy or oximetry.</p><p><strong>Data sources and methods: </strong>A systematic review of clinical effectiveness was conducted based on a predefined protocol. Searches were conducted between 22 May 2023 and 24 May 2023 and were updated on 25 September 2023. Screening, data extraction and critical appraisal were conducted by two reviewers. To be included, studies (of any design) had to evaluate one or more of six novel home device products used in adults and/or children with suspected obstructive sleep apnoea and hypopnoea syndrome. A structured descriptive synthesis was done. We reviewed health economic studies and developed a model to assess cost-effectiveness.</p><p><strong>Results: </strong>In adults, novel devices were compared to home respiratory polygraphy in four studies, to hospital sleep laboratory polysomnography in five studies, and home-based polysomnography in one study. No studies compared novel devices to home-based pulse oximetry. Sensitivity and specificity estimates vary between, and within, studies at different severity cut-offs. Sensitivity was generally high (between 80% and 100%), falling below 80% in just two studies. Specificity was more variable (between 25% and 100%), with many estimates in the 70-80% range. Some estimates were uncertain, and we caution making inferences about relative differences between novel devices. The percentage of test failures ranged from 0% to 18%. Limited data were available on many other outcomes. Only three studies included children, and all of these were done in the clinic rather than the home setting. In the base-case economic analysis, all six novel devices are less costly than respiratory polygraphy and slightly less effective. For the AcuPebble and Sunrise (Sunrise) devices, the reduction in quality-adjusted life-years was cost-effective compared to the reduction in costs (i.e. incremental net monetary benefit > £0 at the £20,000 and £30,000 per quality-adjusted life-year thresholds). All six novel devices are more costly and more effective than oximetry, with incremental costs below £10,000 per quality-adjusted life-year gained. Additional analyses highlight the extent of uncertainty.</p><p><strong>Limitations: </strong>There is uncertainty in the diagnostic test accuracy data comparing the novel devices ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 65","pages":"1-242"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13548101/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148840196","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}