Health technology assessment最新文献

{"title":"Azithromycin therapy for prevention of chronic lung disease of prematurity (AZTEC): a randomised placebo-controlled trial.","authors":"John Lowe, David Gillespie, Ali Aboklaish, Tin Man Mandy Lau, Claudia Consoli, Malavika Babu, Mark Goddard, Kerenza Hood, Nigel Klein, Emma Thomas-Jones, Sinead Ahearn-Ford, Greg Young, Christopher Stewart, Mark Turner, Marie Hubbard, Julian Marchesi, Janet Berrington, Sailesh Kotecha","doi":"10.3310/GJSK0401","DOIUrl":"10.3310/GJSK0401","url":null,"abstract":"<p><strong>Background: </strong>Systematic reviews have reported conflicting evidence to confirm if macrolides reduce rates of chronic lung disease of prematurity in at-risk preterm-born infants, including in those colonised with pulmonary <i>Ureaplasma</i> spp. Since an adequately powered trial has been lacking, we conducted a double-blind, randomised, placebo-controlled trial to assess if the macrolide azithromycin improved survival without the development of physiologically defined moderate or severe chronic lung disease of prematurity in infants born at < 30 weeks' gestation.</p><p><strong>Methods: </strong>Infants recruited from 30 neonatal units (median gestational age 27.0 weeks, interquartile range 25.3-28.6) requiring respiratory support within 72 hours of birth were randomised to intravenous azithromycin 20 mg/kg/day for 3 days followed by 10 mg/kg for 7 days or to placebo. Primary outcome was survival without development of physiologically defined moderate/severe chronic lung disease of prematurity at 36 weeks' postmenstrual age. A total of 796 infants were required to detect 12% improvement in survival without development of moderate or severe chronic lung disease of prematurity, including 10% dropout, with two-sided <i>α</i>-level of 5% and 90% power. The primary outcome was analysed using three-level logistic regression to account for clustering of multiple births and participants within centres and was adjusted for gestational age as a fixed effect. Secondary outcomes included death, chronic lung disease of prematurity severity, treatment interaction with <i>Ureaplasma</i> spp. colonisation, days of invasive and days of non-invasive respiratory support, treatment for nosocomial infections, treated patent ductus arteriosus, severe intraventricular haemorrhage, necrotising enterocolitis, treated retinopathy of prematurity and emergence of azithromycin resistance in stool and respiratory samples. Quantitative polymerase chain reaction identified respiratory <i>Ureaplasma</i> spp. and antibiotic resistance genes. Safety was also monitored.</p><p><strong>Findings: </strong>After three withdrawals, 796 randomised infants were included in the final analyses. Survivors without physiologically defined moderate/severe chronic lung disease of prematurity were: 166/394 (42.1%) and 179/402 (44.5%) in the intervention and placebo groups, respectively (adjusted odds ratio 0.84; 95% confidence interval 0.55 to 1.29; <i>p</i> = 0.43). Secondary outcomes were not significantly different between the treatment groups, except for treated retinopathy of prematurity in survivors (3.5% vs. 7.4%, azithromycin vs. placebo; odds ratio: 0.42, 95% confidence interval 0.18 to 0.98). <i>Ureaplasma</i> spp. colonisation did not influence treatment effect. No significant serious adverse effects were reported. From 1108 (<i>n</i> = 541 azithromycin, <i>n</i> = 567 placebo) respiratory aspirates and 709 stool samples from 348 infants, <i>erm</i>(C) and <i>msr</i>(A)","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 12","pages":"1-17"},"PeriodicalIF":3.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12914749/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146142412","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":"Conservative versus liberal oxygenation targets in critically ill children: the Oxy-PICU RCT.","authors":"Doug Gould, Samiran Ray, Irene Chang, Elisa Giallongo, Marzena Orzol, Lauran O'Neill, Rachel Agbeko, Carly Au, Elizabeth Draper, Lee Elliot-Major, Gareth Jones, Lamprini Lampro, Jon Lillie, John Pappachan, Samiran Peters, Padmanabhan Ramnarayan, Zia Sadique, Karen Thomas, Silvia Moler-Zapata, Kathryn Rowan, David Harrison, Paul Mouncey, Mark Peters","doi":"10.3310/HHYY5898","DOIUrl":"10.3310/HHYY5898","url":null,"abstract":"<p><strong>Background: </strong>The optimal target for systemic oxygenation in critically ill children is unknown. Liberal oxygenation is widely practised but is associated with harm in observational studies.</p><p><strong>Objectives: </strong>To evaluate the clinical and cost-effectiveness of a conservative oxygenation target of peripheral oxygen saturation 88-92% compared with peripheral oxygen saturation > 94% in critically ill children admitted to paediatric intensive care unit as an emergency.</p><p><strong>Design and setting: </strong>A pragmatic, open, multicentre, parallel-group, randomised clinical trial conducted in 15 National Health Service paediatric intensive care units and associated emergency transport services across England and Scotland.</p><p><strong>Participants: </strong>Children aged > 38 weeks corrected gestational age and < 16 years, enrolled within 6 hours of being accepted for admission to paediatric intensive care unit as an emergency; receiving invasive mechanical ventilation with supplemental oxygen; and in face-to-face contact with paediatric intensive care unit or emergency transport services staff.</p><p><strong>Interventions: </strong>Adjustment of ventilator and inspired oxygen settings aiming to achieve peripheral oxygen saturation 88-92% (conservative oxygenation) or peripheral oxygen saturation > 94% (liberal oxygenation) during invasive mechanical ventilation.</p><p><strong>Main outcome measures: </strong>Primary outcomes: duration of organ support at 30 days, with death by day 30 ranked as the worst outcome (clinical effectiveness) and incremental costs, quality-adjusted life-years and net monetary benefit at 12 months (cost-effectiveness). Secondary outcomes: incremental costs at 30 days; mortality at paediatric intensive care unit discharge, 30 days, 90 days and 12 months; time to liberation from ventilation; duration of organ support; length of paediatric intensive care unit and hospital stay; functional status at paediatric intensive care unit discharge; and health-related quality of life at 12 months.</p><p><strong>Results: </strong>Two thousand and forty children were randomised between 1 September 2020 and 15 May 2022. Consent was obtained for 1872 (94%) - 939 to the conservative and 933 to the liberal oxygenation group - who were included in the primary analysis. Duration of organ support or death in the first 30 days was lower in the conservative oxygenation group [probabilistic index 0.53, 95% confidence interval 0.50 to 0.55; <i>p</i> = 0.04 Wilcoxon rank-sum test, adjusted odds ratio 0.84 (95% confidence interval 0.72 to 0.99)]. Both components of the composite primary outcome and secondary outcomes favoured conservative oxygenation. Average costs at 30 days strongly indicated lower costs with conservative oxygenation. Longer-term estimated incremental costs and quality-adjusted life-years were lower and net monetary benefit marginally favoured conservative oxygenation but with wide uncertainty [","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 13","pages":"1-20"},"PeriodicalIF":3.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12907991/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146149670","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 clinical and cost-effectiveness of improving sleep via carer delivered strategies in people with dementia: the DREAMS START parallel multi-centre RCT.","authors":"Penny Rapaport, Sarah Amador, Mariam Adeleke, Julie Barber, Sube Banerjee, Ankita Bhojwani, Georgina Charlesworth, Chris Clarke, Colin Espie, Lina Gonzalez, Rossana Horsley, Rachael Hunter, Simon Kyle, Monica Manela, Naaheed Mukadam, Malvika Muralidhar, Malgorzata Raczek, Zuzana Walker, Lucy Webster, Hang Yuan, Gill Livingston","doi":"10.3310/GJPR2620","DOIUrl":"10.3310/GJPR2620","url":null,"abstract":"<p><strong>Background: </strong>Sleep disturbances are common and distressing for people with dementia and their family carers and can lead to carers having interrupted sleep, low mood and care breakdown. Medication can have harmful side effects and is generally ineffective. Non-pharmacological interventions should be first-line treatments, yet until now there have not been effective treatments.</p><p><strong>Objectives: </strong>To establish whether Dementia RElAted Manual for Sleep; STrAtegies for RelaTives (DREAMS START), a multicomponent intervention, reduced sleep disturbance in people with dementia living at home at 8 months compared with National Health Service treatment (treatment as usual).</p><p><strong>Design and methods: </strong>We conducted a two-arm, multicentre, parallel-arm, superiority randomised controlled trial with masked outcome assessment. Participants were randomised (1 : 1 ratio) to DREAMS START intervention plus treatment as usual or treatment as usual alone. Analyses were intention to treat. We conducted a mixed-method process evaluation with additional substudies: one exploring how United Kingdom-based South Asians experience sleep disturbance and dementia, and one exploring the interaction of sleep, dementia and long-term conditions.</p><p><strong>Settings and participants: </strong>We recruited dyads of people with dementia and sleep disturbance living at home and family carers from 12 National Health Service trusts and the Join Dementia Research service in England.</p><p><strong>Interventions: </strong>DREAMS START is a six-session, multicomponent, manualised intervention delivered to family carers of people with dementia who implement strategies to improve their relatives' sleep. It is delivered face to face or remotely by non-clinically trained graduates weekly or fortnightly and incorporates information about sleep and dementia, promotes de-arousal at night, adaptive stimulus control (e.g. bedtime routine maintenance), daytime behavioural activation, increasing access to light, improving carer sleep and making a tailored action plan.</p><p><strong>Main outcome measures: </strong>The primary outcome was sleep disturbance measured using the Sleep Disorders Inventory at 8 months.</p><p><strong>Results: </strong>Between February 2021 and March 2023, 377 dyads were randomly assigned, 189 to treatment as usual and 188 to DREAMS START plus treatment as usual. Mean age of participants with dementia was 79.4 years (standard deviation 9.0), and 206 (55%) were women. Mean Sleep Disorders Inventory score at 8 months was lower in the intervention versus treatment-as-usual arm [15.16 (standard deviation 12.77), <i>n</i> = 159, vs. 20.34 (16.67), <i>n</i> = 163]; adjusted difference in means [-4.70 (95% confidence interval -7.65 to -1.74); <i>p</i> = 0.002]. Seventeen (9%) people with dementia in the intervention and 17 (9%) in the control arm died during the trial; deaths were unrelated to the intervention. The mean incrementa","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 17","pages":"1-26"},"PeriodicalIF":3.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12907995/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146149594","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":"Outcome after Selective early treatment for Closure of patent ductus ARteriosus in preterm babies, a multicentre, masked, randomised placebo-controlled parallel group trial (Baby-OSCAR trial).","authors":"Samir Gupta, Nimish V Subhedar, Jennifer L Bell, Ursula Bowler, Charlotte Clarke, Christina Cole, Kerrianne Dempster, Clare Edwards, David Field, Jane Greenaway, Elizabeth Hutchison, Nina Jamieson, Samantha Johnson, Wilf Kelsell, Ann Kennedy, Andy King, Marketa Laube, Louise Linsell, David Murray, Heather O'Connor, Chidubem Okeke Ogwulu, Justine Pepperell, Tracy Roberts, Charles Roehr, Sunil Sinha, Kayleigh Stanbury, Julia Sutton, Richard Welsh, Joy Wiles, Jonathan Wyllie, Edmund Juszczak, Pollyanna Hardy","doi":"10.3310/GJSG2422","DOIUrl":"10.3310/GJSG2422","url":null,"abstract":"<p><strong>Background: </strong>In extremely preterm babies, born before 28 weeks' gestation, a large (≥ 1.5 mm in diameter) patent ductus arteriosus present beyond 3 days of age is associated with higher mortality and morbidity than infants without a patent ductus arteriosus. The cyclooxygenase inhibitor ibuprofen may be used to treat patent ductus arteriosus. Whether selective early treatment of a large patent ductus arteriosus with ibuprofen improves health and developmental outcomes is not known.</p><p><strong>Methods: </strong>We conducted a multicentre, randomised, double-blind, placebo-controlled trial evaluating early treatment (≤ 72 hours after birth) with ibuprofen for a large patent ductus arteriosus in extremely preterm infants. The primary outcome was a composite of death or moderate or severe bronchopulmonary dysplasia at 36 weeks' of post menstrual age. The short-term secondary outcomes included complications of prematurity, patent ductus arteriosus closure and side effects of treatment. The main long-term outcome was survival without moderate or severe neurodevelopmental impairment, using parent report or classified by blinded end-point review committee at 24 months of corrected age. Other secondary outcomes included survival without respiratory morbidity and duration of oxygen supplementation. A health economic evaluation was undertaken.</p><p><strong>Results: </strong>A total of 326 infants were randomised to ibuprofen and 327 to placebo. The primary outcome occurred in 220/318 infants (69.2%) in the ibuprofen group and in 202/318 infants (63.5%) in the placebo group (adjusted risk ratio 1.09, 95% confidence interval 0.98 to 1.20; <i>p</i> = 0.10). A total of 44 of 323 infants (13.6%) in the ibuprofen group and 33 of 321 infants (10.3%) in the placebo group died by 36 weeks of gestation (adjusted risk ratio 1.32, 95% confidence interval 0.92 to 1.90). Two unforeseeable serious adverse events occurred that were possibly related to ibuprofen. At 24 months of corrected age, outcome data were available for 263 and 274 children in the ibuprofen and placebo groups, respectively. Survival without moderate to severe neurodevelopmental impairment in the ibuprofen and placebo groups was 131/248 (53.0%) and 134/259 (51.9%), respectively; adjusted risk ratio 1.01 (95% confidence interval 0.86 to 1.18); <i>p</i> = 0.901. Survival without respiratory morbidity was 66/210 (31.4%) and 74/220 (33.6%), respectively; adjusted risk ratio 0.92 (95% confidence interval 0.70 to 1.20); <i>p</i> = 0.536. Median duration of oxygen supplementation was 76.0 and 78.0 days, respectively.</p><p><strong>Conclusion: </strong>The risk of death or moderate or severe bronchopulmonary dysplasia at 36 weeks of post menstrual age was not statistically significantly lower for extremely preterm infants randomised to early treatment with ibuprofen compared to placebo. There was no evidence of an improvement in survival without moderate to severe neurodevelopmental impai","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 11","pages":"1-17"},"PeriodicalIF":3.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12914754/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146142401","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":"Mapping components of behavioural weight management interventions using electronic survey and component selection by expert consensus: the BE:COME Study.","authors":"Rebecca Gregg, Nishant Jaiswal, Sahar Sharif, Alison Avenell, Louisa Ells, Sandra Jayacodi, Ruth Mackenzie, Sharon Simpson, Olivia Wu, Jennifer Logue","doi":"10.3310/JLGJ1630","DOIUrl":"10.3310/JLGJ1630","url":null,"abstract":"<p><strong>Background: </strong>Behavioural weight management interventions are the main funded interventions for people living with obesity in the United Kingdom, but there is high intervention variability, including mode of delivery, dietary, physical activity and behavioural components.</p><p><strong>Objective: </strong>To map individual components of behavioural weight management interventions used in pragmatic clinical trials and those commissioned in the real world. To decide on the components which vary across the interventions and are hypothesised to be of importance to attendance, completion and weight loss.</p><p><strong>Design: </strong>Cross-sectional survey.</p><p><strong>Setting: </strong>Behavioural weight management interventions in two separate scenarios: randomised controlled trials and real-world services (local authority and commercial).</p><p><strong>Main outcome measure: </strong>Identification of components of interest that demonstrate variation in both settings.</p><p><strong>Methods: </strong>Mapping exercise of randomised controlled trials and real-world services using the standardized reporting  of adult behavioural weight management interventions to aid evaluation. Selection of components by expert group consensus derived from online survey and discussion.</p><p><strong>Data sources: </strong>Mapping performed by a local contact for real-world services and by one BECOME researcher for randomised controlled trials. Study expert group provided their opinions via online survey and discussion.</p><p><strong>Results: </strong>Real-world services providing data on 19 services and 6 randomised controlled trials were mapped using an intervention template. Survey and discussion led to expert group consensus of components for analysis within a meta-analysis. Summary descriptions are provided for each programme displaying variability in eligibility and exclusion criteria. Results provide a description of real-world services and randomised controlled trials, demonstrating variation between the programme components, for example, programme delivery (face-to-face group based was the most common answer for 28.6% randomised controlled trials and 63.2% real-world services), setting (community centre was the most common answer for 0% of randomised controlled trials and 69.2% real-world services) and total duration of the programme (12 weeks for 7.1% randomised controlled trials and 57.9% real-world services).</p><p><strong>Limitations: </strong>The standardised reporting template is lengthy and can take up to 1.5 hours to complete. The template for randomised controlled was not completed by the trials themselves. An expert group derived the components of interest, which could produce different results with a different group of people.</p><p><strong>Conclusions: </strong>Our work has provided an example of how standardized reporting of adult behavioural weight management interventions to aid evaluation can be implemented. Interventions we","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-27"},"PeriodicalIF":4.0,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12951269/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147276220","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":"Magnetic resonance enterography to predict disabling disease in newly diagnosed Crohn's disease: the METRIC-EF multivariable prediction model, multicentre diagnostic inception cohort study.","authors":"Shankar Kumar, Andrew Plumb, Sue Mallett, Caroline Clarke, Tom Parry, Jing Yi Jessica Weng, Gauraang Bhatnagar, Stuart Bloom, John Hamlin, Ailsa Hart, Simon Travis, Roser Vega, Maira Hameed, Anisha Bhagwanani, Rebecca Greenhalgh, Emma Helbren, James A Stephenson, Ian Zealley, Vivienne Eze, James Franklin, Alison Corr, Arun Gupta, Elizabeth Isaac, Damian Tolan, William Hogg, Antony Higginson, Michela Cicchetti, Sunita Gupta, Miguel Serran, Tim Raine, Mohamed Ahmed, Biljana Brezina, Ilse Patterson, Louise Lee, Richard Pollok, Jaymin Patel, Abigail Seward, Samantha Baillie, Kashfia Chowdary, Sue Philpott, Anvi Wadke, Steve Halligan, Stuart A Taylor","doi":"10.3310/THSN9956","DOIUrl":"10.3310/THSN9956","url":null,"abstract":"<p><strong>Background: </strong>The ability to predict whether patients with a new diagnosis of Crohn's disease will develop disabling disease is an unmet clinical need. Magnetic resonance enterography is a first-line investigation for Crohn's disease, but its role in prognostication is unknown.</p><p><strong>Objective(s): </strong>To improve prediction of disabling Crohn's disease within 5 years of diagnosis by developing and internally evaluating a multivariable prediction model comprising clinical predictors and adding magnetic resonance enterography scores (Magnetic resonance Enterography Global Score, Simplified Magnetic Resonance Index of Activity and Lémann Index). To estimate the healthcare costs incurred within 5 years of Crohn's disease diagnosis and to explore factors driving costs.</p><p><strong>Design: </strong>A multicentre diagnostic inception cohort.</p><p><strong>Setting: </strong>Nine National Health Service hospitals.</p><p><strong>Participants: </strong>Aged ≥ 16 years with newly diagnosed Crohn's disease.</p><p><strong>Main outcome measures: </strong>Comparative predictive ability of prognostic models, including magnetic resonance enterography scores (Magnetic resonance Enterography Global Score, Simplified Magnetic Resonance Index of Activity and Lémann Index) versus a model based on clinical predictors alone for the development of modified Beaugerie disabling Crohn's disease within 5 years of diagnosis.</p><p><strong>Statistical analysis: </strong>We censored development of modified Beaugerie disabling disease ≤ 90 days from diagnosis, and utilised time-to-event models using Royston-Parmar flexible parametric models. Risk group definitions were prespecified; for risk group definition 1, the high-risk patients were the top 40% with the greatest predicted risk, and the high-risk patients had an absolute risk ≥ 10% for risk group definition 2. The absolute risk cut-off was calculated by sorting patients by predicted risk and using the risk of the eighth (10% of 81) patient who developed modified Beaugerie disabling disease.</p><p><strong>Results: </strong>We studied 194 patients, median age 29, interquartile range 22-44 years. Within 5 years from diagnosis, 42% (81/194) developed modified Beaugerie disabling disease. There was a univariable association between initial need for steroid therapy and developing modified Beaugerie disabling disease [hazard ratio 2.11 (95% confidence interval 1.36 to 3.26)]. Using risk group definition 1, the baseline clinical model had 49% (95% confidence interval 39 to 60) sensitivity and 66% (95% confidence interval 57 to 74) specificity for predicting the development of modified Beaugerie disabling disease. There was no difference in sensitivity and specificity between models incorporating Magnetic resonance Enterography Global Score, Simplified Magnetic Resonance Index of Activity and Lémann Index compared to the baseline clinical model. Using risk group definition 2, the model, including magne","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 18","pages":"1-72"},"PeriodicalIF":3.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12926852/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146206963","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":"Intravenous lidocaine for gastrointestinal recovery after colorectal surgery: the ALLEGRO placebo-controlled randomised trial and cost-effectiveness analysis.","authors":"Hugh Paterson, Thenmalar Vadiveloo, Karen Innes, Angie Balfour, Marek Atter, Andrew Stoddart, Seonaidh Cotton, Robert Arnott, Lorna Aucott, Zoe Batham, Irwin Foo, Graeme MacLennan, Susan Nimmo, Doug Speake, John Norrie","doi":"10.3310/GJHP2321","DOIUrl":"10.3310/GJHP2321","url":null,"abstract":"<p><strong>Background: </strong>Delayed return of gut function after colonic resection is a common impediment to early postoperative recovery. Small clinical studies, combined into meta-analyses, have suggested that intravenous lidocaine can improve return of gut function after colorectal surgery.</p><p><strong>Objective(s): </strong>To determine the clinical effectiveness and cost-effectiveness of perioperative intravenous lidocaine infusion compared with placebo in return of gut function after elective minimally invasive colonic resection.</p><p><strong>Design and methods: </strong>Multicentre, pragmatic, placebo-controlled, randomised trial with cost-effectiveness analysis.</p><p><strong>Setting and participants: </strong>Twenty-seven hospital sites across the United Kingdom. Adult patients scheduled for elective minimally invasive colon resection were randomised in 1 : 1 ratio to treatment or control groups using a web-based portal, stratified by age, sex and trial site.</p><p><strong>Interventions: </strong>A sterile solution of 2% lidocaine (made isotonic with sodium chloride) and matching placebo (a sterile solution of 0.9% sodium chloride). Participants received an intravenous bolus of study drug/placebo at induction of anaesthesia (1.5 mg/kg ideal body weight) given over 20 minutes, followed by intravenous infusion of 1.5 mg/hour/kg ideal body weight with a maximum rate of 120 mg/hour (6 ml/hour) for a minimum of 6 hours up to a maximum of 12 hours. The planned duration of infusion was determined by the participating unit's availability of postoperative continuous cardiac monitoring.</p><p><strong>Main outcome measures: </strong>Primary outcome: return of gut function at 72 hours postoperatively measured by 'GI-3 recovery' (defined as tolerating diet and passage of flatus or stool). Other outcomes were GI-2 recovery, prolonged postoperative ileus, patient-reported measures of quality of life, recovery and pain, 30- and 90-day mortality, unplanned re-admissions, adverse events, serious adverse events and cost per quality-adjusted life-year at 30 days. Participants, care givers and those assessing the outcomes were blinded to group assignment.</p><p><strong>Results: </strong>The trial enrolled 590 patients (295 interventions, 295 control); after 33 post-randomisation exclusions, 557 patients were included (279 interventions, 278 control). There was no statistically significant or clinically meaningful difference in GI-3 recovery at 72 hours after surgery [160/279 patients (57.3%) for intravenous lidocaine versus 164/278 patients (59%) for placebo (absolute difference 1.9% (-8.0 to 4.2), odds ratio 0.97 (0.88 to 1.07), <i>p</i> = 0.54)]. There was no effect of intravenous lidocaine found in predetermined subgroup analyses (6- vs. 12-hour duration of infusion, right vs. non-right colectomy, sex, age band and enhanced recovery after surgery compliance). There was no evidence of a difference in other measures of gut function return, pain, qual","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 19","pages":"1-51"},"PeriodicalIF":3.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12951268/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147276148","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":"Laparoscopic hysterectomy versus open abdominal hysterectomy for women with a benign gynaecological condition: the LAVA RCT.","authors":"T Justin Clark, Lina Antoun, Rebecca Woolley, Sheriden Bevan, Kamila Ziomek, William McKinnon, Paul Smith, Kevin Cooper, Ertan Saridogan, Bibi Zeyah Sairally, Jayne Fullard, Monique Morgan, Lynsay Matthews, Laura Jones, Tracy Roberts, Lee Middleton","doi":"10.3310/GJTC1718","DOIUrl":"10.3310/GJTC1718","url":null,"abstract":"<p><strong>Background: </strong>The comparative rates of major complications and recovery times between laparoscopic hysterectomy and abdominal hysterectomy for benign gynaecological conditions remain uncertain.</p><p><strong>Objective(s): </strong>To assess the clinical and cost-effectiveness of laparoscopic hysterectomy compared to abdominal hysterectomy in women with benign gynaecological conditions.</p><p><strong>Design and methods: </strong>A parallel, open, non-inferiority, multicentre, randomised controlled, expertise-based surgery trial with integrated health economic evaluation and an internal pilot with an embedded qualitative process evaluation, and a post-closure survey after recruitment ended.</p><p><strong>Setting and participants: </strong>Women in secondary care requiring hysterectomy and eligible for either surgical method.</p><p><strong>Interventions: </strong>Laparoscopic hysterectomy versus abdominal hysterectomy.</p><p><strong>Main outcome measures: </strong>The primary outcome was major complications (Clavien-Dindo ≥ level III) up to 6 completed weeks post surgery, and the key secondary outcome was time from surgery to resumption of usual activities using the personalised Patient-Reported Outcomes Measurement Information System Physical Function questionnaire. The principal outcome for the economic evaluation was to be cost per quality-adjusted life-year at 12 months post surgery and was feasibility and acceptability for the qualitative process evaluation.</p><p><strong>Results: </strong>Two hundred and fifty-two patients were screened from 13 open sites over 13 months, 156 (62%) were eligible and 75 (49%) randomised. Of the 53 women not randomised, 23 (43%) preferred laparoscopic hysterectomy and 6 (11%) abdominal hysterectomy. About 32/39 (82%) and 30/36 (83%) participants randomised to laparoscopic hysterectomy and abdominal hysterectomy, respectively, had their surgery, of which 31/32 (97%) and 25/30 (83%) received their allocated route of hysterectomy. Major complications occurred in 2/32 (6%) laparoscopic hysterectomy versus 4/30 (13%) abdominal hysterectomy groups. There was no difference in time to resumption of activities [median (interquartile range, <i>N</i>) 7.5 weeks (3.6-8.2, 25) laparoscopic hysterectomy vs. 7.5 weeks (5.5-10.6, 26) abdominal hysterectomy groups] or quality of recovery [mean (standard deviation, <i>N</i>) 81.1 (13.4, 27) vs. 72.3 (17.6, 22) respectively; adjusted mean difference 7.2, 95% confidence interval -3.2 to 17.6]. The qualitative evaluation found that the trial was viewed positively by women and healthcare professionals. The reasons for failure to recruit from 21 sites open or in set-up were lack of research/clinical capacity imposed by the COVID-19 pandemic (14, 67%) and lack of clinician equipoise (11, 52%).</p><p><strong>Limitations: </strong>The main limitation was failure to recruit, resulting in a final sample of 75 patients from a target of 3250. At the time of analysis, 13 (17%","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 14","pages":"1-28"},"PeriodicalIF":3.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12914755/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146149682","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 eculizumab withdrawal in atypical haemolytic uraemic syndrome: the SETS aHUS multi-centre, open-label, prospective and single-arm study.","authors":"Andrew Bryant, Jan Lecouturier, Giovany Orozco-Leal, Vicky Brocklebank, Sonya Carnell, Thomas J Chadwick, Sarah Dunn, Sally A Johnson, David Kavanagh, Ciara A Kennedy, Michal Malina, Emma K Montgomery, Colin R Muirhead, Yemi Oluboyede, Luke Vale, Chris Weetman, Edwin Kwan Soon Wong, Len Woodward, Neil S Sheerin","doi":"10.3310/GJNS4701","DOIUrl":"10.3310/GJNS4701","url":null,"abstract":"<p><strong>Background: </strong>Atypical haemolytic uraemic syndrome is a rare disease (incidence: 0.4 cases per million per year) which, without treatment, is associated with high morbidity and mortality. Eculizumab, a monoclonal complement inhibitor, is an effective treatment but the optimal way to use this high-cost medication (£360,000 per year for an adult) has not been established.</p><p><strong>Objective: </strong>Establish the safety of eculizumab withdrawal and the effectiveness of a monitoring protocol to detect disease relapse and reintroduction of treatment if relapse occurs.</p><p><strong>Setting: </strong>Fifteen hospitals in the United Kingdom.</p><p><strong>Design: </strong>SETS aHUS is a multicentre, open label, prospective, single arm study of the safety and impact of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome using Bayes single arm analysis with a health economic analysis and qualitative study.</p><p><strong>Participants: </strong>Patients over 2 years of age with atypical haemolytic uraemic syndrome who were receiving eculizumab therapy for at least 6 months. Two study arms are described with 28 participants recruited to the withdrawal arm and 11 additional participants recruited to the standard of care arm of the study.</p><p><strong>Intervention: </strong>Withdrawal of eculizumab treatment and replacement with monitoring to assess disease activity with reintroduction of treatment if relapse occurs.</p><p><strong>Main outcome measures: </strong>The primary outcome measure was to determine the safety of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome during the 2-year study period. Patients met a primary outcome of 'safety event occurred' if there was a permanent reduction in estimated glomerular filtration rate or requirement for renal replacement therapy or significant extra-renal manifestation of disease. The health economic analysis compared the cost and health outcomes on and off eculizumab treatment. The qualitative study explored the experiences of patients on living with atypical haemolytic uraemic syndrome and eculizumab treatment, views on withdrawing from treatment and the proposed monitoring plan.</p><p><strong>Results: </strong>One of 28 patients (3.6%) who withdrew from treatment met a primary outcome. Based on the pre-study analysis plan, withdrawal from treatment is not associated with a greater risk to patients compared to remaining on treatment. Of 17 patients with an abnormality in complement regulation, 4 relapsed. Of 11 patients with no abnormality in complement regulation, 0 relapsed. It was possible, by monitoring and rapid patient access, to reintroduce eculizumab treatment when relapse was identified. Most patients welcomed the opportunity to withdraw from treatment but identified concerns about monitoring and the risk of relapse, informed by initial experience at presentation. Withdrawing a patient from treatment saves £4.2M in healthcare co","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 20","pages":"1-37"},"PeriodicalIF":3.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12969051/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147344059","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":"Artificial Intelligence technologies for assessing skin lesions for referral on the urgent suspected cancer pathway to detect benign lesions and reduce secondary care specialist appointments: early value assessment.","authors":"Matthew Walton, Alexis Llewellyn, Eleonora Uphoff, Joseph Lord, Melissa Harden, Robert Hodgson, Mark Simmonds","doi":"10.3310/GJMS0317","DOIUrl":"10.3310/GJMS0317","url":null,"abstract":"<p><strong>Background: </strong>Skin cancers are some of the most common types of cancer. Dermatology services receive about 1.2 million referrals a year, but only a small minority are confirmed skin cancer. Artificial intelligence may be helpful in the diagnosis of skin cancer by identifying lesions that are or are not cancerous.</p><p><strong>Objectives: </strong>To investigate the clinical and cost-effectiveness of two artificial intelligence technologies: DERM (Deep Ensemble for Recognition of Malignancy, Skin Analytics) and Moleanalyzer Pro (FotoFinder), as decision aids following a primary care referral.</p><p><strong>Methods: </strong>A rapid systematic review of evidence on the two technologies was conducted. A narrative synthesis was performed, with a meta-analysis of diagnostic accuracy data. Published and unpublished cost-effectiveness evidence on the named technologies, as well as other diagnostic technologies were reviewed. A conceptual model was developed that could form the basis of a full economic evaluation.</p><p><strong>Results: </strong>Four studies of DERM and two of Moleanalyzer Pro were subject to full synthesis. DERM had a sensitivity of 96.1% to detect any malignant lesion (95% confidence interval 95.4 to 96.8); at a specificity of 65.4% (95% confidence interval 64.7 to 66.1). For detecting benign lesions, the sensitivity was 71.5% (95% confidence interval 70.7 to 72.3) for a specificity of 86.2% (95% confidence interval 85.4 to 87.0). Moleanalyzer Pro had lower sensitivity, but higher specificity for detecting melanoma than face-to-face dermatologists. DERM might lead to around half of all patients being discharged without assessment by a dermatologist, but a small number of malignant lesions would be missed. Patient and clinical opinions showed substantial resistance to using artificial intelligence without any assessment of lesions by a dermatologist. No published assessments of the cost-effectiveness of the technologies were identified; three assessments related to skin cancer more broadly in a National Health Service setting were identified. These studies employed similar model structures, but the mechanism by which diagnostic accuracy influenced costs and health outcomes differed. An unpublished cost-utility model was provided by Skin Analytics. Several issues with the modelling approach were identified, particularly the mechanisms by which value is driven and how diagnostic accuracy evidence was used. The conceptual model presents an alternative approach, which aligns more closely with the National Institute for Health and Care Excellence reference case and which more appropriately characterises the long-term consequences of basal cell carcinoma.</p><p><strong>Limitations: </strong>The rapid review approach meant that some relevant material may have been missed, and capacity for synthesis was limited. The proposed conceptual model does not capture non-cash benefits associated with demand on dermatologist time. An a","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 10","pages":"1-96"},"PeriodicalIF":3.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12951253/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146142299","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}