Health technology assessment最新文献

{"title":"Blood-test monitoring strategies for patients established on immune-suppressing drugs: a mixed-methods study.","authors":"Abhishek Abhishek, Christopher Carroll, Munira Essat, Jo Leaviss, Georgina Nakafero, Amy Fuller, Matthew J Grainge, Jennie Hancox, Hywel C Williams, Timothy Card, Maarten W Taal, Guruprasad P Aithal, Christopher P Fox, Christian D Mallen, Kavita Vedhara, Danielle A van der Windt, Matthew D Stevenson, Richard D Riley","doi":"10.3310/PLRF8845","DOIUrl":"10.3310/PLRF8845","url":null,"abstract":"<p><strong>Background: </strong>The optimal monitoring strategy for adults with inflammatory conditions established on immune-suppressing treatment is unknown.</p><p><strong>Objectives: </strong>To ascertain the optimum strategy of monitoring blood tests for patients established on immune-suppressing drugs. The objectives were to: investigate the association between different strategies of monitoring and detection of pre-specified haematological, hepatic or renal side effects identify prognostic factors associated with pre-specified haematological, hepatic or renal side effects develop prediction models for pre-specified haematological, hepatic or renal side effects evaluate cost-effectiveness of testing strategies, and explore patients' and clinicians' experiences of current testing strategies, and acceptability of recommended strategies.</p><p><strong>Design: </strong>The study undertook systematic review, prognostic modelling, health economic modelling of alternate monitoring intervals, and qualitative study.</p><p><strong>Setting: </strong>Primary and secondary care.</p><p><strong>Participants: </strong>Data from the UK's Clinical Practice Research Datalink and British Association of Dermatologists Biologics Register were used for prediction modelling. Patients with inflammatory conditions treated with immune-suppressing drugs and health professionals were interviewed.</p><p><strong>Results: </strong>There were differences in testing strategies used, outcome definitions and follow-up precluding evidence synthesis. However, clinically significant pre-specified side effects were found to be uncommon or very uncommon during long-term treatment. There was high-quality evidence for elevated liver enzymes and folate non-supplementation being prognostic for methotrexate hepatotoxicity, mercaptopurine being prognostic for thiopurine hepatotoxicity and myelotoxicity; poor thiopurine metabolizer being prognostic for cytopenia; and elevated liver enzymes being prognostic for hepatotoxicity due to anti-tumour necrosis factor-alpha. Five prediction models were developed and validated. They had excellent performance characteristics. The calibration slope (95% confidence interval) in validation cohort was 0.94 (0.85 to 1.02) for methotrexate, 1.10 (0.84 to 1.36) for thiopurines, 0.91 (0.74 to 1.07) for leflunomide, 1.19 (0.96 to 1.43) for sulfasalazine, and 0.90 (0.61 to 1.19) for 5-aminosalicylates. Prediction modelling could not be undertaken for anti-tumour necrosis factor-alpha and mycophenolate due to few outcomes. Health economic modelling found it was cost-effective to increase the interval between monitoring blood tests except in very high-risk scenarios for some drugs (e.g. biennial testing in leflunomide). Eighteen patients from a range of risk profiles, and 13 health professionals were interviewed remotely. They found it acceptable to increase the interval between monitoring blood tests from 3-monthly to 6-monthly or annually depending on their r","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 64","pages":"1-382"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13548102/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148840169","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":"Challenges to overcome in a randomised trial for Proper Understanding of Recurrent Stress Urinary Incontinence Treatment in women: the PURSUIT RCT.","authors":"Caroline Pope, Madeleine Cochrane, Clare Clement, Yumeng Liu, Sangeetha Paramasivan, Sian Noble, Stephanie J MacNeill, Amanda L Lewis, Jodi Taylor, Bethanie Fitzgerald, Nikki Cotterill, Tamsin Greenwell, Hashim Hashim, Swati Jha, Nikesh Thiruchelvam, Philip Toozs-Hobson, Alison White, Wael Agur, J Athene Lane, Marcus Drake","doi":"10.3310/AKAK8992","DOIUrl":"10.3310/AKAK8992","url":null,"abstract":"<p><strong>Background: </strong>Recurrence or persistence of symptoms after interventions to treat stress urinary incontinence in women is common, but without robust evidence to base treatment recommendations.</p><p><strong>Objectives: </strong>To investigate whether endoscopic or surgical treatments for stress urinary incontinence in women are effective and cost-effective.</p><p><strong>Design: </strong>A multicentre, unblinded, parallel-group randomised controlled trial.</p><p><strong>Setting: </strong>Fifteen centres across the United Kingdom.</p><p><strong>Participants: </strong>Adult women with recurrent or persistent stress urinary incontinence.</p><p><strong>Intervention: </strong>Individual randomisation to endoscopic (urethral bulking) or surgical (autologous sling, colposuspension, artificial urinary sphincter) interventions. Women randomised to surgery chose their operative intervention.</p><p><strong>Main outcomes: </strong>Primary outcome self-reported International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form at 1 year post randomisation. Secondary outcomes included International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form, Patient Global Impression of Improvement and Pelvic Organ Prolapse/Urinary Incontinence Sexual questionnaires up to 3 years post randomisation, operative assessment measures and adverse events, cost-effectiveness from National Health Service and societal perspectives (quality-adjusted life-years and International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form) at 1 year, and a secondary care perspective (quality-adjusted life-years) at 3 years. Semistructured qualitative interviews at baseline (post randomisation), follow-up (3-6 months) and longer-term (12 and 36 months), to explore stress urinary incontinence generally, the acceptability and attitudes to treatments and to improve understanding of outcomes. Qualitative interviews with clinicians at baseline were focused on potential difficulties of recruitment and optimising patient-facing information and training materials for clinicians.</p><p><strong>Results: </strong>Fifty-five women were deemed eligible after screening (<i>n</i> = 328 screened) from October 2019 to June 2022. Twenty-four eligible women consented, and 23 were randomised (between January 2020 and July 2022) from 8 sites with the average age of 57 years (standard deviation: 10.7) and all self-reported 'white' ethnicity. Participants reported a median International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form score at baseline of 16 (interquartile range: 13-19) and mean post-void residual volume of 4.64 ml (standard deviation: 8.45). Eleven participants received their allocated intervention, 2 participants withdrew prior to receiving their intervention and 10 were waiting for their intervention when the study closed. The most common reason for declining participation was a trea","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"329-372"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376003/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144560002","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":"Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis.","authors":"Mark Corbett, Chinyereugo Umemneku-Chikere, Sarah Nevitt, Nyanar Jasmine Deng, Matthew Walton, Helen Fulbright, Chong Yew Tan, Robin Lachmann, Rachel Churchill, Robert Hodgson","doi":"10.3310/GJRH0730","DOIUrl":"10.3310/GJRH0730","url":null,"abstract":"<p><strong>Background: </strong>Late-onset Pompe disease is a rare inherited genetic condition that causes progressive muscle dysfunction and damage. As the disease advances, the progressive weakening of respiratory muscles significantly increases the risk of respiratory failure, which is a major contributor to premature mortality. Enzyme replacement therapy is the primary treatment for Pompe disease.</p><p><strong>Objective: </strong>To investigate the clinical impact of enzyme replacement therapies for the treatment and management of late-onset Pompe disease and establish the relative effectiveness of enzyme replacement therapy compared to best supportive care (in the absence of enzyme replacement therapy).</p><p><strong>Methods: </strong>A systematic review and network meta-analysis of published evidence on the clinical effectiveness of enzyme replacement therapy and best supportive care was undertaken. Comprehensive bibliographic database searches were conducted up to May 2024 to identify randomised controlled trials or any other prospective enzyme replacement therapy studies in patients with Pompe disease. Network meta-analyses of randomised controlled trials were undertaken to estimate indirect treatment effects for forced vital capacity % predicted and the 6-minute walk test. Other studies were summarised using narrative synthesis.</p><p><strong>Results: </strong>The review included 60 studies: 38 on enzyme replacement therapy and 22 on best supportive care. Enzyme replacement therapy studies comprised 3 randomised controlled trials, 3 randomised controlled trial extensions, 7 registry studies and 25 single-group prospective studies. Two randomised controlled trials had a high risk of bias. Best supportive care studies included 14 longitudinal and 8 cross-sectional studies. In the network meta-analyses, after approximately 1 year, enzyme replacement therapy-naive patients showed significant 6-minute walk test improvements versus placebo: ~25 m with alglucosidase alfa and ~54 m with avalglucosidase alfa. No significant differences were found for forced vital capacity % predicted or comparisons with cipaglucosidase alfa, although very few enzyme replacement therapy-naive patients taking cipaglucosidase alfa were available for inclusion in the analyses. Intra-enzyme replacement therapy comparisons showed a significant 6-minute walk test advantage for avalglucosidase alfa. However, a sensitivity analysis adjusting for skewed data revealed no significant differences. Long-term enzyme replacement therapy effectiveness was assessed in single-group studies, showing initial gains maintained for 1-3 years, followed by gradual 10- to 15-year declines in 6-minute walk test and forced vital capacity % predicted. However, small sample sizes and missing data introduce uncertainty. Long-term evidence on best supportive care is limited, with most of the evidence focused on characterising basic demographic information and support needs. A small number of st","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"163-220"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12926851/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146201492","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":"Establishing a research partnership to investigate functional loss and rehabilitation towards the end of life.","authors":"Matthew Maddocks, Lisa Jane Brighton, Louise Connell, Alison Cowley, Barry Laird, Guy Peryer, Carmine Petrasso, Lucy Ziegler, Rowan Harwood","doi":"10.3310/PTHC7598","DOIUrl":"10.3310/PTHC7598","url":null,"abstract":"<p><strong>Background: </strong>Functional loss, the inability to perform necessary or desired tasks, is a common consequence of life-limiting illnesses and associated symptoms (pain, fatigue, breathlessness, etc.) and causes suffering for patients and families. Rehabilitation, a set of interventions designed to address functional loss, is recognised as essential within palliative care, as it can improve quality of life and reduce care costs. However, not everyone has equal access to rehabilitation. Despite limited life expectancy or uncertain ability to benefit from interventions, palliative rehabilitation services are often absent. This is partly due to a lack of high-quality research around optimal models of rehabilitation. Research in this area is methodologically challenging and requires multidisciplinary and cross-speciality collaboration.</p><p><strong>Aim and objectives: </strong>We aimed to establish and grow a United Kingdom research partnership across diverse areas, commencing with partners from Edinburgh, East Anglia, Lancashire, Leeds, London and Nottingham, around the topic area of functional loss and rehabilitation in palliative and end-of-life care. The objectives were to (1) develop a multidisciplinary, cross-speciality research partnership, (2) generate high-priority unanswered research questions with stakeholders, (3) co-design and submit high-quality competitive research proposals, including (4) sharing topic and methodological expertise, and (5) to build capacity and capability to deliver nationally generalisable studies.</p><p><strong>Activities: </strong>The partnership was established with professionals from across England and Scotland with complementary areas of expertise including complex palliative and geriatric research, physiotherapy, nursing, palliative medicine and psychology. Research questions were generated through a modified version of the Child Health and Nutrition Research Initiative, which allowed for the collation and refinement of research questions relating to functional loss and rehabilitation towards the end of life. Partnership members were supported through a series of workshops to transform research ideas into proposals for submission to stage one calls by the National Institute for Health and Care Research. The partnership not only supported students, clinicians and public members with training opportunities but also supported clinicians in securing protected time from clinical duties to allow them to focus on developing local research initiatives.</p><p><strong>Reflections: </strong>Through our partnership we established a network that offered researchers, clinicians, students and public members the chance to develop novel skills and explore opportunities for personal and professional development around the topic area of functional loss and rehabilitation in palliative and end-of-life care. The partnership was crucial to foster collaboration and facilitate exchange of ideas, knowledge and experience","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"685-696"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11070894/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140854536","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":"High or low dose oxytocin for nulliparous women delayed in the first stage of labour: the HOLDS RCT.","authors":"Sara Kenyon, Tracey Johnston, Jason Waugh, Kim Hinshaw, Julia Sanders, Andrew Ewer, Lee Middleton, Clive Stubbs, Versha Cheed, Hannah Summers, Ruth Hewston, Adrian Wilcockson, Kate Siddall, Dee Wherton, Peter Brocklehurst","doi":"10.3310/MALP6685","DOIUrl":"10.3310/MALP6685","url":null,"abstract":"<p><strong>Background: </strong>Delay in the first stage of labour occurs in approximately 20% of nulliparous women. Recommended treatment is intravenous oxytocin, which shortens labour but does not affect the mode of birth. There is some evidence that a higher dose regimen may decrease the need for caesarean section.</p><p><strong>Objective: </strong>The primary objective was to establish if a high-dose regimen of oxytocin compared to the current standard-dose regimen reduced the need for caesarean section for nulliparous women with confirmed delay in the first stage of labour.</p><p><strong>Design: </strong>Multicentre, randomised double-blind controlled trial.</p><p><strong>Setting: </strong>Twenty-one maternity units in the United Kingdom.</p><p><strong>Participants: </strong>Consenting nulliparous women who had a singleton cephalic pregnancy, gestation of 37-41 weeks inclusive, confirmed delay in labour in first stage, ruptured membranes and for whom the clinical decision has been made to prescribe oxytocin.</p><p><strong>Interventions: </strong>Standard-dose regimen of oxytocin (2 mU/min increasing every 30 minutes to a maximum of 32 mU/min) compared with high-dose regimen (4 mU/min increasing every 30 minutes to a maximum of 64 mU/min).</p><p><strong>Main outcome measures: </strong>The primary outcome was the rate of caesarean section. Secondary outcomes included maternal and neonatal birth outcomes and safety.</p><p><strong>Results: </strong>One hundred and eighteen women were successfully randomised via third-party minimisation from an intended sample size of 1500 between 30 June 2017 and 14 November 2022. The caesarean section rate in the standard-dose group was 34% (20/58) and 27% (16/60) in the high-dose group. The intervention was provided as intended in 96% (113/118) of participants. There was no obvious suggestion that the high-dose regime was unsafe (all neonates were discharged home with mother), but this size of sample prohibited any definitive conclusions.</p><p><strong>Limitations: </strong>The trial did not meet its intended sample size due to a number of challenges. It was difficult for busy clinical staff to recruit women in labour in this acute, but not emergency, situation. The legislative requirements of undertaking research using interventional medicinal products imposed further constraints, and we encountered challenges in the production, blinding and monitoring required. Changes in clinical practice since trial design and commencement 10 years ago have resulted in fewer women going into spontaneous labour (reduced from 66% to 47%), and therefore potentially becoming eligible, due to more women having labour induced (22%-33%) or elective caesarean sections (12%-20%). These challenges were further compounded by a falling birth rate and the impact of the COVID-19 pandemic.</p><p><strong>Conclusions: </strong>The question of the optimum dose of oxytocin for nulliparous women delayed in the first stage of spontaneous labou","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"643-666"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12683681/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145523410","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":"Microfracture with or without collagen scaffold insertion for adults with chondral or osteochondral defects of the knee: the SISMIC RCT and its challenges during and after the COVID-19 pandemic.","authors":"Michael R Whitehouse, Nicholas Howells, Lucy Dabner, Russell Thirard, Lucy Culliford, Elsa Marques, Petra Baji, Andrew Judge, Ashley W Blom, Amanda Burston, Catherine Jameson, Chris A Rogers","doi":"10.3310/BRTS2415","DOIUrl":"10.3310/BRTS2415","url":null,"abstract":"<p><strong>Background: </strong>Around 10,000 symptomatic knee articular cartilage injuries requiring repair occur annually in the United Kingdom, mostly in people under 35 years of age. Microfracture surgery aims to restore cartilage. Adding microstructural scaffolds made of collagen may further improve outcomes.</p><p><strong>Objectives: </strong>To evaluate the clinical and cost-effectiveness of microstructural scaffold in patients undergoing microfracture for a symptomatic chondral or osteochondral defect of the knee.</p><p><strong>Design: </strong>Multicentre, parallel two-group, superiority randomised controlled trial with blinding of participants, research staff and clinical care teams not involved in the surgery.</p><p><strong>Setting: </strong>National Health Service hospitals offering arthroscopic chondral surgery.</p><p><strong>Participants: </strong>Adults aged 18 years or older with symptomatic chondral or osteochondral defects of the knee on the medial or lateral femoral condyles, trochlea or patella and a chondral or osteochondral lesion measuring no more than 4 cm<sup>2</sup>. Exclusions were: unstable ligamentous injuries or meniscal tears that would not be treated; a knee with defects on the tibial chondral surface, < 50% native meniscal volume or requiring realignment surgery/osteotomy; and a lesion previously treated with microfracture.</p><p><strong>Interventions: </strong>Lesions were debrided, and microfracture was performed on the exposed subchondral bone. Intervention: microfracture of the chondral or osteochondral lesion with insertion of a bilayer collagen matrix microstructural scaffold, fixed with stiches or fibrin glue. Comparator: microfracture alone. Postoperative physiotherapy was standardised. Participants were randomised 1 : 1 between intervention and control.</p><p><strong>Main outcome measures: </strong>Primary outcome was the Knee Injury and Osteoarthritis Outcome Score at 24 months post randomisation. Secondary outcomes included International Knee Documentation Committee knee evaluation score; Tegner-Lysholm activity grading scale; EuroQol-5 Dimensions, five-level version; Work Productivity and Activity Impairment; complications and resource use measured at 3, 6, 12 and 24 months.</p><p><strong>Results: </strong>Twenty-two patients were screened across 8 sites, 20 of whom were eligible on screening. Of the 20 patients considered initially eligible, 2 patients were not interested and 1 opted for chondroplasty; the remaining 17 all consented to participate. Between November 2021 and October 2022, 10 participants were randomised, 5 to microfracture and 5 to microfracture with scaffold. Three patients failed the final in-surgery eligibility check (lesions had healed), one decided not to have surgery and three were still waiting when the study was closed. The median age was 38 years, and four participants were female. Most participants (seven) had damage to the lateral femoral condyle, and six had a medial and/or","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"229-254"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12666602/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145431262","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":"Brief psychodynamic-interpersonal therapy for adults with a history of self-harm: the SafePIT RCT.","authors":"Elspeth Guthrie, Bethan Copsey, Alexandra Wright-Hughes, Aaron Dowse, Chris Bojke, Richard Mattock, Florence Day, Judith Horrocks, Gina Bianco, Cathy Brennan, Marsha McAdam, Michael Crawford, Navneet Kapur, Catherine Fernandez, Petra Bijsterveld, Amanda Farrin","doi":"10.3310/TNGF8545","DOIUrl":"10.3310/TNGF8545","url":null,"abstract":"<p><strong>Background: </strong>There are over 200,000 hospital attendances for self-harm per annum in the United Kingdom at an estimated annual cost of £133-162M. Systematic reviews prior to commencing the study suggested that brief psychological interventions are effective in reducing psychological distress after self-harm and reduce repetition of self-harm.</p><p><strong>Objective: </strong>The SafePIT trial was designed to evaluate the effectiveness and cost-effectiveness of self-harm-focused psychological therapy plus standard care versus standard care alone.</p><p><strong>Design: </strong>Pragmatic, multicentre individually randomised controlled trial of brief psychodynamic-interpersonal therapy compared with standard care with internal pilot, cost-effectiveness and process evaluation.</p><p><strong>Setting and participants: </strong>People aged over 18 years who attend hospital after intentional self-harm with a history of ≤ 3 episodes in the last 12 months.</p><p><strong>Intervention: </strong>Individual psychodynamic-interpersonal therapy, delivered face to face or by video conferencing by liaison mental health nurses, over four (or fewer by mutual agreement) 50-minute weekly sessions with two optional boosters.</p><p><strong>Main outcome measures: </strong>The primary outcome was time from randomisation to first repetition of self-harm leading to hospital attendance. Secondary outcomes (at 6 and 12 months) included rate of repetition of self-harm leading to hospital attendance; self-reported self-harm using questionnaires and Short Message Service; psychological distress and clinically significant improvement (Clinical Outcomes in Routine Evaluation - Outcome Measure); anxiety (Generalised Anxiety Disorder-7); hopelessness (Beck Hopelessness Scale); interpersonal function (Inventory of Interpersonal Problems-32) and quality of life (EuroQol-5 Dimensions, five-level version; Recovering Quality of Life; Clinical Outcomes in Routine Evaluation-6D).</p><p><strong>Results: </strong>The planned sample size was 770 participants. The trial closed to recruitment early in January 2023 at the end of the 12-month internal pilot, with 22 randomised participants, 12 allocated to psychodynamic-interpersonal therapy and 10 to standard care. Due to the early trial closure, trial follow-up was curtailed to 6 months, and analyses are restricted to descriptive statistics. Seven of 12 participants allocated to psychodynamic-interpersonal therapy started therapy, and four completed therapy. Participant-reported secondary outcomes were completed for nine (40.9%) participants at 6 months. Repetition of self-harm leading to hospital presentation could be assessed for 18 participants and occurred in two participants in the psychodynamic-interpersonal therapy arm (18.2%) and no participants in the standard care arm within 6 months of randomisation. Economic findings indicated no substantive changes in health-related quality of life, or primary and secondary care ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-42"},"PeriodicalIF":3.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12683462/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145632593","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":"PillCam COLON 2 for investigation of the colon through direct visualisation: systematic review and economic evaluation.","authors":"Paul Tappenden, Sue Harnan, Sa Ren, Mon Mon-Yee, Aline Navega Biz, Gamze Nalbant, Abdullah Pandor, Mark Clowes, Sophie Whyte, Chloe Thomas, Laura Heathcote, Matthew Kurien, Kevin Monahan, Janine Tappenden","doi":"10.3310/GJPT1820","DOIUrl":"10.3310/GJPT1820","url":null,"abstract":"<p><strong>Background: </strong>Colorectal cancer is the fourth most common cancer and the second most common cause of cancer deaths in England. Most cases of colorectal cancer arise from a prior adenomatous polyp in the bowel lining. Colonoscopy is the gold standard investigation for people with symptoms suggestive of colorectal cancer. During a colonoscopy, polyps can be removed or a biopsy can be taken. However, waiting times for colonoscopy can be long and the procedure can be unpleasant. Colon capsule endoscopy may provide an alternative diagnostic procedure to rule out polyps or colorectal cancer.</p><p><strong>Objectives: </strong>To evaluate the clinical effectiveness, acceptability and cost-effectiveness of colon capsule endoscopy using PillCam COLON 2 for detecting colorectal polyps and colorectal cancer.</p><p><strong>Methods and data sources: </strong>A systematic review searched six bibliographic databases and eight conference proceedings in August 2024. Studies of PillCam COLON 2 in symptomatic or polyp surveillance patients were included if they were randomised controlled trials, or if they reported data on diagnostic test accuracy, yield or patient preference. Bayesian pooling of sensitivity and specificity was performed. The economic analysis included a review of existing models and development of an independent model to assess the cost-effectiveness of colon capsule endoscopy versus colonoscopy and computed tomography colonography in three main populations (symptomatic patients with a faecal immunochemical test score of 10-100 μg/g, symptomatic faecal immunochemical test < 10 μg/g and surveillance patients). Subgroup analyses were conducted in patients who are able and willing to undergo colonoscopy and those who are not (denoted 'COL-eligible' and 'COL-ineligible').</p><p><strong>Results: </strong>Among the diagnostic test accuracy studies (11-64% patients 'in-scope'), for polyps of any size (two studies), ≥ 6 mm (four studies) and ≥ 10 mm (four studies), pooled sensitivities were 0.78 (95% credible interval 0.51 to 0.90), 0.83 (95% credible interval 0.70 to 0.91) and 0.85 (95% credible interval 0.70 to 0.94), respectively. Specificities were 0.60 (95% credible interval 0.27 to 0.88), 0.69 (95% credible interval 0.52 to 0.81) and 0.90 (95% credible interval 0.82 to 0.95), respectively. Among yield studies, colonoscopy was spared in 37-50% of symptomatic patients (three studies). Data on colonoscopy spared in surveillance patients were available from one study; however, these data are confidential and cannot be reported here. In patients unwilling/unable to undergo colonoscopy, colon capsule endoscopy completed 70-98% of incomplete colonoscopies. Patient preference studies indicated general satisfaction with PillCam COLON 2, but some conflicting information on patient preference for colon capsule endoscopy compared to colonoscopy and computed tomography colonography. For colonoscopy-eligible patients, within all three main analy","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 58","pages":"1-164"},"PeriodicalIF":3.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13454905/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148602368","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":"Surgery for early structural osteoarthritis of the hip and knee: the SCORE evidence synthesis and economic evaluation.","authors":"Andrew Price, Emma Searle, Maggie Westby, Gianluca Fabiano, Pamela Royle, Jill Colquitt, Emma Loveman, Karen Barker, Sion Glyn-Jones, Antony Palmer, Hema Mistry, Toby Smith, Andy Metcalfe, Rafael Pinedo- Villanueva, Sally Hopewell","doi":"10.3310/TNFD7352","DOIUrl":"10.3310/TNFD7352","url":null,"abstract":"<p><strong>Research question: </strong>Is surgery for early hip or knee osteoarthritis clinically effective and cost-effective?</p><p><strong>Background: </strong>Osteoarthritis is the most common joint disease worldwide costing between 1% and 2.5% of gross domestic product in developed countries. Once a hip or knee develops end-stage disease, arthroplasty surgery is highly successful in most patients. Treatment options are limited for early disease, and patients may fall into a 'treatment gap', despite having severe symptoms. Many surgical procedures are used to treat early osteoarthritis of the hip and knee, but the clinical and cost-effectiveness of these techniques remains unclear.</p><p><strong>Overall aim: </strong>To investigate the role of surgery in the management of early hip or knee osteoarthritis?</p><p><strong>Objectives: </strong>Use systematic review and evidence synthesis to determine the clinical effectiveness of current surgical interventions for early hip and knee osteoarthritis. Estimate the cost-effectiveness of these procedures using decision model analytic modelling. Create clinical treatment guidance and identify areas where new clinical research is required.</p><p><strong>Methods: </strong>The programme of work consisted of systematic review, evidence synthesis and health economic modelling for surgical treatments used for early hip and knee osteoarthritis. We divided treatments into four groups: (1) non-regenerative procedures, (2) regenerative procedures, (3) load-modifying procedures and (4) arthroplasty procedures. They were compared with non-surgical treatment following National Institute for Health and Care Excellence guidance. We used outcome measures that satisfied the Outcome Measures in Rheumatoid Arthritis Clinical Trials and Osteoarthritis Research Society International core outcome set for hip and knee osteoarthritis. Decision-analytic models were developed to assess the cost-effectiveness of treatment alternatives expressed as cost per quality-adjusted life-year gained.</p><p><strong>Results: </strong>The systematic review identified 61,098 studies, and our evidence synthesis identified a number of surgical techniques that have been used to treat early hip and knee osteoarthritis. However, we found very little meaningful evidence of the clinical efficacy for the majority of the surgical procedures. We only found seven randomised controlled trials and three non-randomised studies that compared a surgical technique to non-operative treatment. There were two surgical treatments that had limited but supportive evidence. Femoro-acetabular impingement is an early stage of hip osteoarthritis, and there is some evidence that arthroscopic chondroplasty provides patients with clinical relief of symptoms, above that seen with non-operative measures. In the knee, weaker evidence suggests arthroscopic debridement of osteoarthritic articular cartilage fragments may be of benefit, above non-operative measures. Health econo","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 56","pages":"1-150"},"PeriodicalIF":3.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13403056/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148430326","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":"Paramedic analgesia comparing ketamine and morphine in trauma. A synopsis of the PACKMaN double-blind RCT.","authors":"Michael A Smyth, Hannah Noordali, Ranjit Lall, Felix Michelet, Stavros Petrou, Kamran Khan, Joyce Yeung, Gordon Fuller, Alison Walker, Rebecca McLaren, Duncan Buckley, Kath Starr, Gavin D Perkins","doi":"10.3310/KGTO6391","DOIUrl":"10.3310/KGTO6391","url":null,"abstract":"<p><strong>Background: </strong>Paramedics frequently administer analgesic medications for pain following trauma. Morphine is the most commonly administered strong analgesic. However, it may not be the best option as it may lower blood pressure, depress respiration and there is a risk of dependency. Ketamine might be a better option. We sought to compare clinical and cost-effectiveness of paramedic administered ketamine and morphine in patients with severe pain following trauma.</p><p><strong>Methods: </strong>PACKMaN was a double-blinded, randomised controlled, superiority trial. Eligible patients were 16 years of age or over, had an acute injury, and articulated a pain score of 7 or greater on a 0-10 numeric rating score. We excluded pregnant patients, prisoners, those unable to articulate a pain score and anyone lacking capacity. The maximum dose of morphine was 20 mg while the maximum dose of ketamine was 30 mg. The trial drug was titrated to effect. The primary outcome was the Sum of Pain Intensity Difference score.</p><p><strong>Results: </strong>We randomised 449 participants: 219 (49%) received ketamine and 230 (51%) received morphine. The Sum of Pain Intensity Difference score was 3.5 (standard deviation 2.8) for ketamine and 3.4 (standard deviation 3.0) for morphine. We found no significant difference in efficacy between drugs (adjusted mean difference 0.1, 95% confidence interval -0.4 to 0.6; <i>p</i> = 0.7). Ketamine was more likely to achieve 'very much improvement' (odds ratio 1.58, 95% confidence interval 1.08 to 2.31; <i>p</i> = 0.019) and to do so more rapidly than morphine (hazard ratio 1.42, 95% confidence interval 1.09 to 1.84; <i>p</i> = 0.009). However, morphine was likely to last longer than ketamine (hazard ratio 1.28, 95% confidence interval 1.05 to 1.56; <i>p</i> = 0.013). There was no evidence of a significant difference in serious adverse events.</p><p><strong>Conclusion: </strong>Ketamine does not provide superior analgesia than morphine when treating acute severe trauma pain. Ketamine is a suitable alternative that is safe for use by paramedics.</p><p><strong>Limitations: </strong>Patients were required to provide verbal assent to participate, consequently we were only able to recruit patients who had capacity to understand what was being asked of them. It is also therefore probable that we were unable to recruit the most severely injured patients. These factors may limit generalisability of our results. Furthermore, we were unable to complete a planned sensitivity analysis to determine if there was a difference in treatment response between patients with more minor injuries and those who were more severely injured. Finally, we experienced a loss to follow-up at 3 and 6 months, so our findings for long term outcomes may have been underpowered.</p><p><strong>Future work: </strong>Future research should focus on identification of the optimal drug (or combination of drugs), dosing and drug route to achieve rapid control","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 57","pages":"1-32"},"PeriodicalIF":3.4,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13403055/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148519858","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}