Health technology assessment最新文献

{"title":"Artificial intelligence software to help detect fractures on X-rays in urgent care: An Early Value Assessment.","authors":"Caroline Farmer, Helen Coelho, Madhusubramanian Muthukumar, Sophie Robinson, Robert Meertens, Obioha C Ukoumunne, Val Santo, Niamh Gale, Jonathan T Evans, Jonathan P Evans, Jenny Lowe, G J Melendez-Torres, Edward Cf Wilson","doi":"10.3310/GJCF0822","DOIUrl":"10.3310/GJCF0822","url":null,"abstract":"<p><strong>Background: </strong>Artificial intelligence algorithms have been developed to support clinicians in diagnosing fractures, with the intention to improve the diagnostic accuracy of clinicians reviewing X-rays. The purpose of this rapid early value assessment was to identify the existing evidence base for the technology and to assess whether there was a prima facie case for the technology to represent positive outcomes for patients and a value-for-money investment for people in the National Health Service.</p><p><strong>Methods: </strong>This early value assessment assessed the potential value of the use of artificial intelligence to aid clinician diagnosis of fractures in emergency care settings as compared to clinician-diagnosis alone. A rapid evidence review was conducted followed by 'light touch' early economic modelling to explore whether a plausible case could be made for cost-effectiveness at the prices charged by the companies. Evidence searches were conducted in June and July 2024 to identify clinical, diagnostic and service outcomes associated with the technology. A simple decision model incorporating prevalence, sensitivity, specificity and cost per scan for each of the technologies was developed to evaluate plausible cost-effectiveness for detecting ankle and foot, wrist and hand, and hip fractures, selected based on the availability of evidence and their downstream costs and consequences.</p><p><strong>Results: </strong>Sixteen studies identified evaluated the diagnostic accuracy of the technology. None of the included studies were conducted in the United Kingdom and all were associated with limitations. While the studies were not considered to be able to provide reliable estimates of diagnostic accuracy, there was a trend for the technology to improve sensitivity for detecting fractures. The technology had no discernible impact on the rate of false-positive diagnoses. Overall, most of the evaluated technologies were associated with a positive incremental net health benefit at willingness-to-pay thresholds of £20,000 and £30,000 per quality-adjusted life-year gained. Due to data limitations, it was not possible to compare technologies against each other. The results were mostly robust to scenario analyses.</p><p><strong>Discussion: </strong>The evidence base for the technology is currently limited to studies evaluating diagnostic accuracy and it is unclear whether increases in fracture detection would translate into meaningful benefits for patients and services. While there are some fractures that, if missed, can result in significant harm to patients, it is plausible that the technology would improve diagnosis of more subtle fractures that may not require a change in management. Use of the technology would not eradicate the risk of missed fractures, meaning that health services would need to continue to take precautions to avoid the risk of a missed fracture in clinical practice. A simple decision tree analysis suggested th","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 33","pages":"1-97"},"PeriodicalIF":3.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13181505/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147863817","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":"Outpatient paracentesis for Ovarian Hyperstimulation Syndrome: STOP-OHSS feasibility study and RCT Synopsis.","authors":"Mostafa Metwally, Katie Ridsdale, Clare Pye, Munya Dimairo, Amy Barr, Ying Cheong, Lauren Desoysa, Sarah Drabble, Andrew Drakeley, Isaac Evbuomwan, Laura Flight, Jennifer Hannington, Jane Hughes, Anju Keetharuth, Amanda Loban, Elizabeth Lumley, Raj Mathur, Kirsty McKendrick, Cara Mooney, Alicia O'Cathain, Eloise Pyper, Rich Simmonds, Liz Taylor, Jessica Wright, Siqi Wu, Tracey Young, David White","doi":"10.3310/GJMM1724","DOIUrl":"10.3310/GJMM1724","url":null,"abstract":"<p><strong>Background: </strong>Ovarian hyperstimulation syndrome is a potentially serious complication of fertility treatments. Standard care often involves monitoring, then hospitalisation for severe cases. Some evidence suggests early outpatient paracentesis may prevent hospitalisation, but adequately powered randomised trials are lacking.</p><p><strong>Objectives: </strong>To establish the clinical and cost-effectiveness, safety and acceptability of early active outpatient management for moderate or severe ovarian hyperstimulation syndrome.</p><p><strong>Design and methods: </strong>A pragmatic, parallel, open-label, multicentre, superiority, adaptive, group sequential randomised controlled trial with an internal pilot was planned. The study included preliminary qualitative work and a post-trial survey. The main trial recruited participants with moderate or severe, early or late ovarian hyperstimulation syndrome from UK fertility clinics. Participants were randomised 1 : 1 to receive either outpatient paracentesis or usual care (conservative management). The primary outcome was ovarian hyperstimulation syndrome-related hospital admission within 28 days.</p><p><strong>Results: </strong>The trial was terminated early due to poor recruitment. Only 8 participants were randomised (5 to conservative management, 3 to outpatient paracentesis) across 3 of 9 opened sites, compared to the planned 224 participants. All eight had moderate ovarian hyperstimulation syndrome at baseline. Two participants were hospitalised for ovarian hyperstimulation syndrome-related reasons (one from each group). Six participants' symptoms resolved during follow-up, while two had unknown outcomes. There were no reported serious adverse events caused by the intervention. The post-trial survey of 16 fertility centres found increased use of preventive measures like freeze-all cycles, antagonist protocols and gonadotropin-releasing hormone trigger, during and after the SARS-CoV-2 (COVID-19) pandemic.</p><p><strong>Limitations: </strong>The small sample size precludes drawing any definitive conclusions about effectiveness, safety or cost-effectiveness. The study was severely impacted by the COVID-19 pandemic, affecting site set-up and recruitment, and changes in clinical practice during the pandemic may have reduced ovarian hyperstimulation syndrome cases.</p><p><strong>Conclusions: </strong>No definitive clinical conclusions can be drawn from this study about the effectiveness of outpatient management compared to conservative management. The pandemic prompted lasting modifications in ovarian hyperstimulation syndrome prevention strategies at many fertility clinics.</p><p><strong>Future work: </strong>Given the observed low incidence rates of ovarian hyperstimulation syndrome and challenges in recruitment, future research may need to consider alternative study designs in observational settings rather than randomised trials that reflect the changing reality of clinical management","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 42","pages":"1-34"},"PeriodicalIF":3.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13202624/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147972182","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":"Comparing the effectiveness, cost-effectiveness and implementation of low-dose oral modified release morphine in people with chronic breathlessness: a synopsis of a RCT and process evaluation.","authors":"Miriam J Johnson, Bronwen Williams, Catriona Keerie, Sharon Tuck, Simon P Hart, Sabrina Bajwah, Nazia Chaudhuri, Mark Pearson, Judith Cohen, Rachael A Evans, David C Currow, Irene J Higginson, Peter S Hall, Marek Atter, John Norrie, Charlotte Thompson, Chao Huang, Lukas Pitel, Annie Jones, Gareth Stewart, Marie T Fallon","doi":"10.3310/GJMF3819","DOIUrl":"10.3310/GJMF3819","url":null,"abstract":"<p><strong>Background: </strong>Chronic breathlessness is common and disabling in chronic medical conditions. There is good rationale to use opioids from neuroscience and exercise laboratory studies.</p><p><strong>Objectives: </strong>We assessed the 56-day effectiveness, cost-consequences and safety of long-acting oral morphine for breathlessness in people with cardio-respiratory diseases or cancer.</p><p><strong>Methods: </strong>We conducted a parallel-group, randomised, placebo-controlled trial in 11 UK outpatient services, comparing 10-20 mg daily oral morphine with placebo (and blinded laxative) in people with moderate to severe breathlessness, with embedded health economic evaluation. The primary outcome was <i>worst breathlessness</i>/24 hours (0-10 numerical rating scale) at day 28. Secondary outcomes included: physical activity levels; worst cough numerical rating scale/24 hours; Short Form questionnaire-12 items; EuroQol-5 Dimensions, five-level version; morphine-related toxicities; caregiver burden. Analyses were by modified intention-to-treat (modified intention-to-treat; ≥ 1 dose of study drug). Primary analysis used repeated measures of covariance adjusted for baseline <i>worst breathlessness</i> and stratification variables. The planned sample size (90% power, 20% withdrawal) was 158. We undertook two exploratory substudies, a process theory-informed evaluation and an open-label follow-on study.</p><p><strong>Results: </strong>Between 18 March 2021 and 26 October 2023, 143 were randomised (75 morphine, 68 placebo); 140 formed the modified intention-to-treat population [90% power; males 66%; mean age 70.5 (standard deviation 9.4)]. By day 28, 60/73 (82%) morphine <i>versus</i> 56/67 (84%) placebo participants had ≥ 90% adherence. At day 28, we found no evidence of a primary outcome difference [adjusted mean difference; 0.09 (95% confidence interval -0.57 to 0.75), <i>p</i> = 0.78], or any secondary measure except improved cough seen at day 56 [adjusted mean difference; -1.41 (-2.18 to -0.64)]. Increased moderate/vigorous physical activity was seen at day 28 [adjusted mean difference; 9.51 minutes/day (0.54 to 18.48)], but this was not significant after multiple measures correction. There were 413 adverse events: 251 <i>versus</i> 162 events in 63/73 (86%) morphine <i>versus</i> 51/67 (76%) placebo participants; 18 serious adverse events in 15 participants (morphine, 3/15 related; placebo, 0/3 related); no treatment-related deaths. Morphine participants used more inpatient and fewer outpatient services. In the 56-day base-case analysis, morphine was associated with similar mean per-patient costs and quality-adjusted life-years: an increase of £24 (95% confidence interval -£395 to £552) and 0.002 (95% confidence interval -0.004 to 0.008) quality-adjusted life-years.</p><p><strong>Limitations: </strong>Interpretation is cautious, but findings are plausible and consistent with other evidence. We infer improved exercise endurance. Most ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 37","pages":"1-30"},"PeriodicalIF":3.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13181778/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147863866","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":"Mammographic surveillance in breast cancer patients aged 50 years or older: a synopsis of the Mammo-50 RCT.","authors":"Janet Dunn, Peter Donnelly, Andrea Marshall, Nada Elbeltagi, Amy Hopkins, Maria Ramirez, Alastair Thompson, Riccardo Audisio, Sarah Pinder, David Cameron, Sue Hartup, Maggie Wilcox, Lesley Turner, Annie Young, Helen Higgins, Eila Watson, Sophie Gasson, Peter Barrett-Lee, Claire Hulme, Bethany Shinkins, Peter Hall, Andrew Evans","doi":"10.3310/GJJD0722","DOIUrl":"10.3310/GJJD0722","url":null,"abstract":"<p><strong>Background: </strong>Annual surveillance mammograms for an unspecified period, after treatment for early breast cancer, are widely practised in the United States of America and Europe. Current UK guidelines recommend annual mammograms for 5 years, then reverts to 3-yearly screening. The aim of this trial was to evaluate whether less than annual mammography was non-inferior in terms of breast cancer-specific survival and cost-effectiveness in women aged 50 years or older at diagnosis and 3 years post curative surgery.</p><p><strong>Methods: </strong>We conducted a multicentre, randomised phase III trial of annual mammography versus less-frequent mammography (2-yearly after conservation surgery or 3-yearly after mastectomy). Women were eligible if aged ≥ 50 years at initial diagnosis of breast cancer (invasive or ductal carcinoma in situ) and recurrence-free 3 years post curative surgery. The trial was conducted at 114 NHS hospitals in the UK. Participants were randomly assigned (1 : 1) to annual or less-frequent mammograms; followed up for 6 years. Coprimary outcomes were breast cancer-specific-survival and cost-effectiveness; secondary outcomes included recurrence-free interval and overall survival. Analyses were by intention to treat, with a pre-planned per-protocol analysis. Planned sample size was 5000. Clinical results are now reported.</p><p><strong>Results: </strong>Five thousand two hundred and thirty-five women were randomised between April 2014 and September 2018. With a median of 5.7-year follow-up, 343 women have died, of whom 116 died of breast cancer (61 on annual arm; 55 on less-frequent arm). Breast cancer-specific-survival at 5 years was 98% on both arms with a hazard ratio of 0.92 (95% confidence interval 0.64 to 1.32), which demonstrated non-inferiority of less-frequent mammograms at the 3% margin (non-inferiority <i>p</i> < 0.0001) and the 1% margin (non-inferiority <i>p</i> = 0.003). Non-inferiority was demonstrated at the 2% level for both recurrence-free interval [hazard ratio 1.00 (95% confidence interval 0.83 to 1.28); non-inferiority <i>p</i> = 0.0024] and overall survival [hazard ratio 1.07 (95% confidence interval 0.87 to 1.33); non-inferiority <i>p</i> = 0.008]. Less-frequent mammograms were associated with a significant cost saving (mean difference £544, 95% confidence interval -£1116 to £26), heavily driven by mammogram costs. Incorporating societal costs resulted in a larger cost-saving (£1543 per person, 95% confidence interval -£2416 to -£669), increasing cost-effectiveness. There was no impact of less-frequent mammograms on patients' quality of life.</p><p><strong>Conclusion: </strong>For patients aged ≥ 50 years and 3 years post diagnosis, less-frequent mammograms were non-inferior and cost-effective compared with annual mammograms, with no detriment to patients' quality of life. Mammo-50 provides evidence to inform guideline development.</p><p><strong>Limitations: </strong>Adherence to the mammograph","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 38","pages":"1-15"},"PeriodicalIF":3.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13200029/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147873849","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":"Prevention and treatment of incontinence-associated dermatitis through optimising care: the PREVENT-IAD Synopsis.","authors":"Sue Woodward, Tanya Graham, Nicholas Beckley-Hoelscher, Dimitri Beeckman, Christopher Chatterton, Mandy Fader, Francesca Fiorentino, Joanne M Fitzpatrick, Ruth Harris, Jan Kottner, Christine Norton, Sangeeta Sooriah, Peter R Worsley","doi":"10.3310/LMAS8546","DOIUrl":"10.3310/LMAS8546","url":null,"abstract":"<p><strong>Background: </strong>Incontinence-associated dermatitis is prevalent in long-term care facilities and homecare settings among adults who are incontinent of urine and/or faeces. Strategies to protect skin integrity are needed. This study aimed to co-design and test the feasibility of a training manual and care guidance (Incontinence-Associated Dermatitis Manual) to prevent and treat incontinence-associated dermatitis in long-term care facilities and homecare settings.</p><p><strong>Methods: </strong>This was a three-phase study: (1) evidence synthesis and intervention development, (2) definitive study design (a cluster-randomised controlled trial with an embedded process evaluation) and (3) a 3-month feasibility study. Our Cochrane review was updated following a similar methodology to the original review. The intervention (underpinned by findings from the Cochrane review) was developed with 16 stakeholders. The feasibility study targeted four long-term care facilities and two homecare providers, randomising them (each as a cluster) to intervention or control. Recruitment targeted 288 participants (48 per cluster). Feasibility outcomes included recruitment and retention rates, completeness of data, acceptability of intervention and methods and intervention fidelity. Outcome measures included: Ghent Global incontinence-associated dermatitis categorisation tool, minimum data set and incontinence-associated dermatitis intervention tool. Process evaluation interviews with two care recipients, 11 family carers and 13 care staff implementing the Incontinence-Associated Dermatitis Manual and their managers were conducted. Observations of 22 episodes of care assessed intervention fidelity. Qualitative data were analysed using thematic analysis. Summary feasibility outcome measures using means or proportions, together with 95% confidence intervals, were reported.</p><p><strong>Results: </strong>The reviews of interventions for prevention and treatment of incontinence-associated dermatitis included 15 trials with 1089 participants and 19 trials with 1164 participants, respectively. All participants were incontinent of urine, stool or both. Little evidence, of very low to low quality, was found on the effects of interventions for preventing and treating incontinence-associated dermatitis in adults. The proposed cluster-randomised controlled trial was agreed, with one additional outcome measure added, and the co-designed intervention (incontinence-associated dermatitis manual) included training (3 hours face-to-face and online), an online manual and a poster/flow chart to support care decision-making. After a COVID-19-related pause of 7 months, obtaining governance and ethical approvals for the feasibility study was delayed by 27 months. Five sites were recruited from 49 approached. All randomised sites were retained. Seventy-six (16%) of the 477 participants approached were randomised, of which 58 (76%) completed the study. Ghent Global incontinenc","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 44","pages":"1-48"},"PeriodicalIF":3.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13244240/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148137841","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":"Effects of physical activity and diet in pregnancy to prevent gestational diabetes: an individual participant data (IPD) meta-analysis on the differential effects of interventions with economic evaluation.","authors":"John Allotey, Dyuti Coomar, Joie Ensor, Chidubem Okeke Ogwulu, Gabriel Ruiz Calvo, Mark Monahan, Valencia Kabeya, Rachel McNeill, Anna Boath, Ghadir Mahmoud, Cheryce Harrison, Mahnaz Bahri Khomami, Helena Teede, Nicola Heslehurst, Graham A Hitman, Sharon Anne Simpson, Krish Nirantharakumar, Julie Dodds, Kelly C Allison, Garry Shen, Elisabetta Petrella, Fabio Facchinetti, Christina Vinter, Mireia Pelaez, Dorte Møller Jensen, Narges Sadat Motahari-Tabari, Tarja I Kinnunen, Jonatan R Ruiz, Annick Bogaerts, Kristina Martha Renault, Alka Kothari, Jose Guilherme Cecatti, Fionnuala M McAuliffe, Suzanne Phelan, Lucilla Poston, Ana Pilar Betrán, Ngawai Moss, Stamatina Iliodromiti, Frances Austin, Nuria García de la Torre, Alfonso Luis Calle Pascual, Javier Zamora, Tracy Roberts, Richard D Riley, Shakila Thangaratinam","doi":"10.3310/GJST1327","DOIUrl":"10.3310/GJST1327","url":null,"abstract":"<p><strong>Background: </strong>Physical inactivity and suboptimal diet in pregnancy are important modifiable risk factors for gestational diabetes, a major contributor to pregnancy complications.</p><p><strong>Objectives: </strong>We aimed to assess the effects of physical activity and/or diet-based lifestyle interventions during pregnancy on gestational diabetes and if these vary by maternal (body mass index, age, parity, ethnicity, education) and intervention characteristics using individual participant data meta-analysis of randomised trials, and a cost-effectiveness analysis.</p><p><strong>Data sources: </strong>International Weight Management in Pregnancy Collaborative Network database was updated by searching major databases from February 2017 to March 2022.</p><p><strong>Review methods: </strong>The main outcomes were gestational diabetes by any criteria and by the National Institute for Health and Care Excellence. Other outcomes were gestational diabetes as per International Association of Diabetes in Pregnancy Study Group and maternal and perinatal outcomes. We performed a two-stage random-effects individual participant data meta-analysis to obtain summary estimates (odds ratio) with 95% confidence intervals. Study quality of included trials was assessed, and heterogeneity summarised using τ<sup>2</sup>. Where possible, we added the aggregate data from non-individual participant data trials to the meta-analysis. We ranked interventions by effectiveness using network meta-analysis and undertook model-based economic evaluation to assess cost-effectiveness. The cost-effectiveness analysis took an NHS cost perspective compared an overall lifestyle intervention versus usual care with a time horizon covering the beginning of pregnancy until the discharge of the mother and infant from the hospital following delivery.</p><p><strong>Results: </strong>Ninety-two trials (32,284 women) were included; 54 (23,698 women) provided individual participant data. Lifestyle interventions reduced the odds of gestational diabetes (any criteria) by 10% in individual participant data trials (odds ratio 0.90, 95% confidence interval 0.80 to 1.02, 54 studies, 23,361 women), and the findings reached statistical significance when non-individual participant data were included (odds ratio 0.81, 95% confidence interval 0.73 to 0.89, 92 studies, 31,947 women). Physical activity significantly reduced the odds of gestational diabetes by 36% (odds ratio 0.64; 95% confidence interval 0.48 to 0.84), and diet by 19% (odds ratio 0.81; 0.69 to 0.96), but not mixed interventions. Women with middle (odds ratio 0.68, 95% confidence interval 0.51 to 0.90) and high educational level (odds ratio 0.71, 95% confidence interval 0.54 to 0.93) benefited more than those with low educational status, and no differences by maternal body mass index, age, parity or ethnicity. There was no significant reduction in gestational diabetes defined by National Institute for Health and Care Excellence","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 39","pages":"1-252"},"PeriodicalIF":3.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13181776/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147873868","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":"Olanzapine for young people with anorexia nervosa: a synopsis of the main results from the OPEN open-label feasibility study.","authors":"Ece Sengun Filiz, Dominic Stringer, Vanessa Kellermann, Rachel Rowan Olive, Olena Said, Hiba Mutwalli, Sevgi Bektas, Melahat Nur Akkese, Katie Ireland, Demelza Beishon-Murley, Joel W T Khor, Lee Allman, Nicus Kotze, Ben Carter, Elizabeth Tyrrell-Bunge, Katie Rowlands, Johanna Louise Keeler, Glynis Ivin, Mima Simic, Dilveer Singh Sually, Jessica Bentley, Allan H Young, Sloane Madden, Sarah Byford, Sabine Landau, Janet Treasure, Ulrike Schmidt, Dasha Nicholls, Vanessa Lawrence, Hubertus Himmerich","doi":"10.3310/GJHH0812","DOIUrl":"10.3310/GJHH0812","url":null,"abstract":"<p><strong>Background: </strong>In clinical practice, olanzapine is commonly used in young people with anorexia nervosa, although the underpinning evidence-base is limited. However, its efficacy, tolerability, acceptability and adherence rate, and the patients', carers' and clinicians' views of olanzapine treatment are unclear. This synopsis article summarises the methods and results of the OPEN feasibility study, overarching the findings and drawing comprehensive conclusions from a quantitative feasibility outcome paper and two qualitative research papers.</p><p><strong>Methods: </strong>The OPEN study assessed the feasibility of a future randomised controlled trial on olanzapine in young people with anorexia nervosa in an open-label, one-armed feasibility study. In this study, we aimed to include 55 patients with anorexia nervosa or atypical anorexia nervosa aged 12-24 who gained < 2 kg within at least 1 month of treatment as usual. Time points for assessments were at baseline, 8 weeks, 16 weeks, and 6 or 12 months. We estimated recruitment, adherence and attrition rates and mean changes in body weight, body mass index and eating disorder psychopathology. In addition, we explored the views on and experiences with olanzapine treatment within a clinical trial setting using qualitative interviews with young people with anorexia nervosa, their families and clinicians.</p><p><strong>Results: </strong>Fifty-two people were pre-screened, 35 were eligible and 20 participants were recruited and started olanzapine. Of these, 15 continued olanzapine for ≥ 16 weeks. Participants experienced, on average, a decrease in their eating disorder psychopathology, and body weight and body mass index increased during treatment with olanzapine as an adjunct to treatment as usual. Important themes derived from semi-structured qualitative interviews with young people and their parents were: moving away from the illness towards recovery, evaluating information on olanzapine, consent and trust in shared decision-making and the ambivalence around recovery. The main themes expressed by clinicians included: acknowledging the concerns of young people with anorexia nervosa and their families, prioritising person-centred care, the limited service capacity and strict study eligibility criteria.</p><p><strong>Limitations: </strong>The study failed to meet the recruitment target and showed low adherence rates for treatment with olanzapine. Possible reasons for the recruitment difficulties and the low adherence rate include the high clinical workload of eating disorder services during and after the COVID-19 pandemic, the heterogeneity of eating disorder service setup across the country, and the reluctance of patients to agree to take olanzapine under the relatively restricted conditions of a clinical study.</p><p><strong>Conclusions: </strong>A realistic time schedule for site-preparation, recruitment, treatment and follow-up, realistic recruitment targets and easy access to medic","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 36","pages":"1-17"},"PeriodicalIF":3.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13181506/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147873855","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":"Manualised package intervention to achieve treatment adherence in people with tuberculosis: the IMPACT pilot cluster-RCT.","authors":"Marc Lipman, Ibrahim Abubakar, Colin Nj Campbell, Caroline Clarke, Andrew J Copas, Patricia Costello, Marcia Darvell, Robert Horne, Rachael M Hunter, Annie Sk Jones, Aaron S Karat, Karina Kielmann, Ayşenur Kılıç, Heinke Kunst, Mike Mandelbaum, Zoe Moon, Al Story, Jing Yi Weng, Helen R Stagg","doi":"10.3310/GJML0604","DOIUrl":"10.3310/GJML0604","url":null,"abstract":"<p><strong>Background and objectives: </strong>Tuberculosis remains a United Kingdom health concern. It occurs predominantly in people who have lived in tuberculosis endemic countries or have links there. Adherence to anti-tuberculosis treatment can be challenging, especially for people who experience severe side effects or social marginalisation. Poor adherence can lead to treatment failure. Current adherence support interventions make little difference to outcome. We identified the need for a 'manualised' approach to (1) improve case-managers' ability to detect people likely to non-adhere and (2) guide targeted adherence support.</p><p><strong>Objectives: </strong>Synthesise knowledge on drivers and interventions to support anti-tuberculosis treatment adherence Apply the Perceptions and Practicalities framework to understand poor adherence Develop a manualised intervention to identify adherence-related risks, modifiable barriers and support mechanisms Pilot the intervention and assess feasibility of data collection Evaluate implementation through fidelity and reach, and assess impact on adherence Assess intervention delivery costs to guide a full trial plus economic evaluation.</p><p><strong>Methods: </strong>The study ran April 2018-September 2022. Formative work included scoping reviews of adherence literature; National Health Service patients, caregivers, and health worker interviews; and clinic observations. A multidisciplinary group, including people with lived experience of tuberculosis, healthcare professionals, and researchers, coproduced the intervention package. We performed a (1 : 1) pilot cluster-randomised trial (<i>N</i> = 79 participants evaluated), randomising four London tuberculosis clinics, in preparation for a definitive cluster-randomised trial. Participants in control clinics received standard care. The primary outcome was adherence, doses taken of a possible 168 measured using evriMED boxes and other sources. We recorded treatment outcomes and changes in participants' needs, health-related beliefs and perceptions, costs, and health status. We conducted a mixed-methods process evaluation, using questionnaires, interviews, case-report forms, checklists and clinic observations.</p><p><strong>At intervention sites, additional resources were: </strong>Electronic tuberculosis needs assessment completed at all visits. Two animated videos to increase motivation and ability to take treatment. Interactive treatment guide designed around the Perceptions and Practicalities framework. Detailed manual for case managers.</p><p><strong>Results: </strong>We developed a tuberculosis needs assessment for tuberculosis services. This appeared better than standard care at identifying people requiring adherence support [e.g. at baseline 21/36 (58.3%) intervention vs. 4/43 (9.3%) control] and social support (over 24 weeks, on 29 vs. 6 occasions respectively). Cumulative dose-taking was high across the study population at 24 weeks [84% (95% confi","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 28","pages":"1-138"},"PeriodicalIF":3.4,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13112134/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147721945","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":"A novel methodology using direct patient contact and UK national registries to collect long-term data from randomised trials: TARGIT-X - an extended follow-up study of the TARGIT-A trial of targeted intraoperative radiotherapy for breast cancer.","authors":"Jayant S Vaidya, Norman R Williams, Max Bulsara, Chris Brew-Graves, Ingrid Potyka, Nicholas Roberts, Julie Lindsay, Siobhan Laws, Sanjay Raj, Michael Douek, Mary Falzon, Gloria Petralia, Sarah Needleman, Anu Malhotra, Marcelle Bernstein, Jeffrey S Tobias","doi":"10.3310/GJJV2820","DOIUrl":"10.3310/GJJV2820","url":null,"abstract":"<p><strong>Background: </strong>Many diseases, including breast cancer, have a long natural history; therefore, longer-term effects of treatments are important for patients and for their full evaluation. However, trial follow-up data are collected by specific staff and are funded for a relatively short duration.</p><p><strong>Objective: </strong>We evaluated whether we could collect follow-up information for patients in a breast cancer randomised clinical trial by direct patient contact and data from national registries.</p><p><strong>Setting: </strong>The TARGIT-A randomised clinical trials of targeted intraoperative radiotherapy during lumpectomy versus whole-breast external beam radiotherapy (n=2298), and delayed TARGIT-IORT vs. external beam radiotherapy (<i>n</i> = 1153), recruited women with early breast cancer diagnosed in 33 centres in 12 countries, between March 2000 and June 2012. We planned to recruit all United Kingdom patients from the TARGIT-A trials for extended follow-up. These were the first randomised trials of intraoperative radiotherapy for breast cancer.</p><p><strong>Methods: </strong>We assessed the feasibility of recording whether patients are alive and their current health status, including events related to breast cancer, and effects of radiotherapy such as lung cancer diagnoses, by direct patient contact and data from NHS Digital (health episodes, diagnoses and death). Patients were consented in collaboration with the recruiting site and were then contacted annually, if appropriate, directly by the trial centre. We calculated the proportion of eligible patients whose status could be ascertained, contacted, consented and provided follow-up information via direct patient contact and/or NHS Digital data. We estimated the additional years of follow-up and its cost.</p><p><strong>Results: </strong>Six hundred and seven of 714 United Kingdom patients originally recruited in the TARGIT-A trials were initially eligible. We ascertained the current status or reason for non-participation of 574 (94.5%); 87% (502/574) of these patients' health status could be determined. Of these, 73% (366/502) or 60.3% of the total (366/607) were found to be in good health, provided valid consent for TARGIT-X and their health status. One hundred and thirty-six patients did not participate in TARGIT-X because: 105/136 (77%) were too unwell or had died, and for 6 patients, the consent was either incomplete or the physical form could not be traced. Less than 5% (25/502) of patients were unwilling to participate: 23 declined and 2 withdrew. We recorded an additional 103 deaths, more than doubling the initial number to 203. The quality of data returned by patients was very good [e.g. mismatch rate for recording date < 0.1% (1/1470 forms)]. Patients who participated increased their follow-up by a median 6 years [to 14 years (interquartile range 13-16)]. We found a much lower incidence of lung cancer diagnoses with TARGIT-IORT compared with EBRT (16-year","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 29","pages":"1-32"},"PeriodicalIF":3.4,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13112128/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147728880","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":"Development and description of Early Stroke Specialist Vocational Rehabilitation delivered in the RETAKE trial.","authors":"Kathryn A Radford, Mary I Grant, Jain A Holmes, Julie Phillips, Kathryn Powers, Rachel L Chambers, Kristelle Craven, Brian Bell, Christopher McKevitt, David Clarke, Amanda Farrin, Diane Trusson, Caroline Watkins, Audrey Bowen, Ellen Thompson, Alexandra Wright-Hughes","doi":"10.3310/GJKR0715","DOIUrl":"10.3310/GJKR0715","url":null,"abstract":"<p><strong>Objective: </strong>This paper describes the development of an Early Stroke Specialist Vocational Rehabilitation intervention to support return to work following stroke and its delivery in the RETAKE trial.</p><p><strong>Methods: </strong>Iterative three stage, target population approach to intervention development and evaluation informed by the Medical Research Council Framework. Stage 1 (Initial codevelopment): interviews with key stakeholder service providers and users' and mapping of services supporting return to work after stroke to identify and explore barriers to and unmet needs for support; intervention codevelopment with experts and patient and public involvement (PPI). Stage 2 (Refinement): expert panel codevelopment workshops and systematic review to identify vocational rehabilitation intervention mechanisms of change in supporting return to work after stroke. Stage 3 (Testing): intervention piloting in two case studies, feasibility testing in a randomised controlled trial, acceptability interviews with stroke and employer participants. Further intervention refinement following delivery in the RETAKE randomised controlled trial.</p><p><strong>Results: </strong>Stage 1: service mapping and 25 stakeholder interviews identified service gaps and unmet needs relating to early identification of employed stroke survivors, mild stroke, and hidden disabilities. Access to timely support relied on geographical proximity to a specialist hub and tacit knowledge of complex health, education and employment services and provider roles. Return to work issues reported by stroke survivors informed Early Stroke Specialist Vocational Rehabilitation prototype design objectives. Iterative developments following piloting included fatigue management, involvement of general practitioners, work simulation and liaison with other healthcare services. Interviews with 12 recipient stroke survivors and 6 employers identified additional features including occupational therapist negotiation skills, ability to respond to changing needs over time and patient empowerment to self-re-refer. The review corroborated intervention components and mechanisms and identified additional mechanisms, for example, peer support, supported self-management. Intervention mechanisms identified across the three stages were early intervention, understanding the impact of stroke on the person, their job and work environment, vocational goal setting, implementing workplace accommodations, individual tailoring, work preparation, colocation, case co-ordination, Multidisciplinary Team (MDT) working, employer engagement and education, and responsiveness, which involved monitoring work stability, providing feedback, and responding to changing needs over time and participant self-re-referral. In RETAKE, Early Stroke Specialist Vocational Rehabilitation was successfully delivered to 95.4% of allocated participants with 75.3% compliance. Intervention commenced a median 38 days (interquartile","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-64"},"PeriodicalIF":3.4,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13071780/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147608798","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}