Sue Harnan, Aline Navega Biz, Jean Hamilton, Sophie Whyte, Emma Simpson, Shijie Ren, Katy Cooper, Mark Clowes, Muti Abulafi, Alex Ball, Sally C Benton, Richard Booth, Rachel Carten, Stephanie Edgar, Willie Hamilton, Matt Kurien, Louise Merriman, Kevin Monahan, Laura Heathcote, Matt Stevenson
<p><strong>Background: </strong>Faecal immunochemical tests may be better than symptoms alone at identifying which patients who present to primary care with symptoms are at high risk of colorectal cancer and should have a colonoscopy. This could reduce waiting lists and patient anxiety/discomfort and enable earlier treatment of colorectal cancer. The threshold used will affect how well faecal immunochemical tests work, with a higher threshold resulting in fewer referrals but a greater chance of missing disease.</p><p><strong>Objective: </strong>What is the most clinically effective and cost-effective way to use faecal immunochemical tests to reduce the number of people without significant bowel pathology who are referred to the suspected cancer pathway for colorectal cancer, taking into consideration potential colonoscopy capacity constraints for urgent and non-urgent referrals? Tests were HM-JACKarc, OC-Sensor, FOB Gold, NS-Prime, QuikRead go, IDK TurbiFIT, IDK Hb, IDK Hb/Hp complex and IDKHb+Hb/Hp ELISAs.</p><p><strong>Design: </strong>Systematic review, meta-analysis and cost-effectiveness analyses were conducted.</p><p><strong>Review methods: </strong>Searches across four databases and six registries were conducted (December 2022). Diagnostic accuracy studies conducted in patients presenting to or referred from primary care with symptoms suggestive of colorectal cancer using any reference standard were included. Risk of bias was assessed with quality assessment of diagnostic test accuracy studies version 2. For each test, sensitivity and specificity were pooled at all reported thresholds and summary estimates were provided at all possible thresholds within the observed range. Comparative accuracy between tests was considered. Other outcomes, for example test uptake, failure and patient acceptability, were also extracted.</p><p><strong>Cost-effectiveness analysis methods: </strong>A mathematical model was developed to compare three different diagnostic strategies that used quantitative faecal immunochemical tests in primary care patients with symptoms of colorectal cancer to determine subsequent management pathways. The model assessed the health outcomes and costs associated with each strategy over a lifetime horizon from the perspective of the United Kingdom National Health Service and Personal Social Services, using evidence from published literature and other sources.</p><p><strong>Results: </strong>Syntheses of sensitivity and specificity were conducted for HM-JACKarc (<i>n</i> = 16 studies), OC-Sensor (<i>n</i> = 11 studies) and FOB Gold (<i>n</i> = 3 studies). No synthesis was conducted for QuikRead go, NS-Prime IDK Hb or IDK Hb/Hp as there was only one study for each. No eligible studies were found for IDK Hb+Hb/Hp or for IDK TurbiFIT. Other outcomes (e.g. patient acceptability) were also synthesised. Model results suggest that faecal immunochemical tests generate a positive incremental net monetary benefit compared with current care, t
{"title":"Quantitative faecal immunochemical tests to guide colorectal cancer pathway referral in primary care. A systematic review, meta-analysis and cost-effectiveness analysis.","authors":"Sue Harnan, Aline Navega Biz, Jean Hamilton, Sophie Whyte, Emma Simpson, Shijie Ren, Katy Cooper, Mark Clowes, Muti Abulafi, Alex Ball, Sally C Benton, Richard Booth, Rachel Carten, Stephanie Edgar, Willie Hamilton, Matt Kurien, Louise Merriman, Kevin Monahan, Laura Heathcote, Matt Stevenson","doi":"10.3310/AHPE4211","DOIUrl":"10.3310/AHPE4211","url":null,"abstract":"<p><strong>Background: </strong>Faecal immunochemical tests may be better than symptoms alone at identifying which patients who present to primary care with symptoms are at high risk of colorectal cancer and should have a colonoscopy. This could reduce waiting lists and patient anxiety/discomfort and enable earlier treatment of colorectal cancer. The threshold used will affect how well faecal immunochemical tests work, with a higher threshold resulting in fewer referrals but a greater chance of missing disease.</p><p><strong>Objective: </strong>What is the most clinically effective and cost-effective way to use faecal immunochemical tests to reduce the number of people without significant bowel pathology who are referred to the suspected cancer pathway for colorectal cancer, taking into consideration potential colonoscopy capacity constraints for urgent and non-urgent referrals? Tests were HM-JACKarc, OC-Sensor, FOB Gold, NS-Prime, QuikRead go, IDK TurbiFIT, IDK Hb, IDK Hb/Hp complex and IDKHb+Hb/Hp ELISAs.</p><p><strong>Design: </strong>Systematic review, meta-analysis and cost-effectiveness analyses were conducted.</p><p><strong>Review methods: </strong>Searches across four databases and six registries were conducted (December 2022). Diagnostic accuracy studies conducted in patients presenting to or referred from primary care with symptoms suggestive of colorectal cancer using any reference standard were included. Risk of bias was assessed with quality assessment of diagnostic test accuracy studies version 2. For each test, sensitivity and specificity were pooled at all reported thresholds and summary estimates were provided at all possible thresholds within the observed range. Comparative accuracy between tests was considered. Other outcomes, for example test uptake, failure and patient acceptability, were also extracted.</p><p><strong>Cost-effectiveness analysis methods: </strong>A mathematical model was developed to compare three different diagnostic strategies that used quantitative faecal immunochemical tests in primary care patients with symptoms of colorectal cancer to determine subsequent management pathways. The model assessed the health outcomes and costs associated with each strategy over a lifetime horizon from the perspective of the United Kingdom National Health Service and Personal Social Services, using evidence from published literature and other sources.</p><p><strong>Results: </strong>Syntheses of sensitivity and specificity were conducted for HM-JACKarc (<i>n</i> = 16 studies), OC-Sensor (<i>n</i> = 11 studies) and FOB Gold (<i>n</i> = 3 studies). No synthesis was conducted for QuikRead go, NS-Prime IDK Hb or IDK Hb/Hp as there was only one study for each. No eligible studies were found for IDK Hb+Hb/Hp or for IDK TurbiFIT. Other outcomes (e.g. patient acceptability) were also synthesised. Model results suggest that faecal immunochemical tests generate a positive incremental net monetary benefit compared with current care, t","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 46","pages":"1-210"},"PeriodicalIF":4.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12666607/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145174835","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}
Barnaby Reeves, Maria Pufulete, Jessica Harris, Jo Dumville, Una Adderley, Ashley Burton, Michael Burton, Ross Atkinson, Madeleine Clout, Nicky Cullum, Abby O'Connell, Louise O'Connor, Stephen Palmer, Matthew Ridd, Jeremy Rodrigues, Jason Wong
<p><strong>Background: </strong>Surgical reconstruction to close a severe pressure ulcer has not been evaluated.</p><p><strong>Aim and objectives: </strong>We aimed to investigate the feasibility of research to evaluate surgical reconstruction for severe pressure ulcers by: systematically reviewing evidence about: the effectiveness of surgical reconstruction for severe pressure ulcers; the impact of pressure ulceration on health-related quality-of-life (review 2) surveying primary and secondary care healthcare professionals about surgical referrals of patients with severe pressure ulcers and severe pressure ulcer management, including surgical reconstruction describing patients with incident pressure ulcers and with severe pressure ulcers having surgical reconstruction comparing outcomes in patients with severe pressure ulcers having/not having surgical reconstruction seeking consensus about treatments and management strategies for severe pressure ulcers.</p><p><strong>Design: </strong>Systematic reviews; surveys; binary choice experiment; retrospective cohort studies using routine data; consensus meeting.</p><p><strong>Participants: </strong>General practitioners; nurses; and surgeons managing pressure ulcers; people with incident pressure ulcers and hospitalised with severe pressure ulcers.</p><p><strong>Intervention: </strong>Surgical reconstruction.</p><p><strong>Comparator: </strong>No surgical reconstruction.</p><p><strong>Outcomes: </strong>Surgical reconstruction, time to next admission with a severe pressure ulcer time to next admission, hospital stay, all-cause mortality, surgical reconstruction after discharge.</p><p><strong>Results: </strong>Review 1 included three studies comparing different surgical reconstruction techniques. None reported wound-free time. Recurrence occurred in ≈ 20%. Review 2 included three randomised controlled trials measuring health-related quality of life, but none observed benefits of interventions evaluated. Among primary care survey respondents, 54% did not know surgical reconstruction can treat severe pressure ulcers; > 50% had never referred a patient to a surgeon. Among nurses, 72% had considered surgical reconstruction for a severe pressure ulcer; 54% believed surgical reconstruction should be more available. Among surgeons, 39% had never offered surgical reconstruction and 52% offered surgical reconstruction to < 50%; 68% believed surgical reconstruction should be more available. Routine data recorded 367,884 admissions with severe pressure ulcer diagnoses in England over 7.5 years; surgical reconstructions were performed in at least 404 and at most 1018 admissions. Twenty English hospitals performed > 70% of the surgical reconstructions. Comparing surgical reconstruction (<i>n</i> = 325) versus no surgical reconstruction (<i>n</i> = 1474) patients, time to next admission with a severe pressure ulcer was longer in patients having surgical reconstruction (hazard ratio = 0.79, 95% confidence interval 0.6
{"title":"Effectiveness of surgical interventions in patients with severe pressure ulcers: the SIPS mixed-methods exploratory study.","authors":"Barnaby Reeves, Maria Pufulete, Jessica Harris, Jo Dumville, Una Adderley, Ashley Burton, Michael Burton, Ross Atkinson, Madeleine Clout, Nicky Cullum, Abby O'Connell, Louise O'Connor, Stephen Palmer, Matthew Ridd, Jeremy Rodrigues, Jason Wong","doi":"10.3310/DWKT1327","DOIUrl":"10.3310/DWKT1327","url":null,"abstract":"<p><strong>Background: </strong>Surgical reconstruction to close a severe pressure ulcer has not been evaluated.</p><p><strong>Aim and objectives: </strong>We aimed to investigate the feasibility of research to evaluate surgical reconstruction for severe pressure ulcers by: systematically reviewing evidence about: the effectiveness of surgical reconstruction for severe pressure ulcers; the impact of pressure ulceration on health-related quality-of-life (review 2) surveying primary and secondary care healthcare professionals about surgical referrals of patients with severe pressure ulcers and severe pressure ulcer management, including surgical reconstruction describing patients with incident pressure ulcers and with severe pressure ulcers having surgical reconstruction comparing outcomes in patients with severe pressure ulcers having/not having surgical reconstruction seeking consensus about treatments and management strategies for severe pressure ulcers.</p><p><strong>Design: </strong>Systematic reviews; surveys; binary choice experiment; retrospective cohort studies using routine data; consensus meeting.</p><p><strong>Participants: </strong>General practitioners; nurses; and surgeons managing pressure ulcers; people with incident pressure ulcers and hospitalised with severe pressure ulcers.</p><p><strong>Intervention: </strong>Surgical reconstruction.</p><p><strong>Comparator: </strong>No surgical reconstruction.</p><p><strong>Outcomes: </strong>Surgical reconstruction, time to next admission with a severe pressure ulcer time to next admission, hospital stay, all-cause mortality, surgical reconstruction after discharge.</p><p><strong>Results: </strong>Review 1 included three studies comparing different surgical reconstruction techniques. None reported wound-free time. Recurrence occurred in ≈ 20%. Review 2 included three randomised controlled trials measuring health-related quality of life, but none observed benefits of interventions evaluated. Among primary care survey respondents, 54% did not know surgical reconstruction can treat severe pressure ulcers; > 50% had never referred a patient to a surgeon. Among nurses, 72% had considered surgical reconstruction for a severe pressure ulcer; 54% believed surgical reconstruction should be more available. Among surgeons, 39% had never offered surgical reconstruction and 52% offered surgical reconstruction to < 50%; 68% believed surgical reconstruction should be more available. Routine data recorded 367,884 admissions with severe pressure ulcer diagnoses in England over 7.5 years; surgical reconstructions were performed in at least 404 and at most 1018 admissions. Twenty English hospitals performed > 70% of the surgical reconstructions. Comparing surgical reconstruction (<i>n</i> = 325) versus no surgical reconstruction (<i>n</i> = 1474) patients, time to next admission with a severe pressure ulcer was longer in patients having surgical reconstruction (hazard ratio = 0.79, 95% confidence interval 0.6","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 47","pages":"1-150"},"PeriodicalIF":4.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12668256/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145182057","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}
Adel Elfeky, Yen-Fu Chen, Amy Grove, Keith Couper, Rachel Court, Sara Tomassini, Anna Wilson, Amy Hooper, Alexandra Buckle, Sharvari Vadeyar, Marion Thompson, Olalekan Uthman, Joyce Yeung
<p><strong>Background: </strong>Perioperative oxygen administration has been proposed as a strategy to reduce postoperative complications. However, uncertainty exists as to which strategies are the most clinically effective.</p><p><strong>Objectives: </strong>To provide an overview on the effectiveness of perioperative oxygen therapy and formulate recommendations to inform clinical decision-making and research.</p><p><strong>Methods: </strong>We followed the Preferred Reporting Items for Overviews of Reviews guidelines. We searched key databases for systematic reviews (from inception to September 2021) and randomised controlled trials (from April 2018 to March 2022) comparing perioperative oxygen strategies. Reviews with the most comprehensive coverage of literature were chosen as anchoring reviews. We assessed risk of bias for each anchoring review using the Risk of Bias in Systematic Reviews tool. We updated meta-analyses from anchoring reviews with data from recent randomised controlled trials and conducted subgroup analyses and meta-regression. We assessed the certainty of evidence using grading of recommendations assessment, development and evaluation framework and conducted trial sequential analysis. We used grading of recommendations assessment, development and evaluation informative statements to communicate our findings. Our advisory panel reviewed mapping of studies and interpretation of evidence.</p><p><strong>Results: </strong>We identified 59 systematic reviews and selected 5 anchoring reviews. A high fraction of inspired oxygen may result in a slight reduction in surgical site infection compared with a low fraction of inspired oxygen (risk ratio 0.91, 95% confidence interval 0.78 to 1.05; risk difference 1.2% lower, 2.9% lower to 0.7% higher, low-certainty evidence). This effect may be modified by type of surgery, oxygen delivery method or study quality. The evidence suggests that a high fraction of inspired oxygen results in a large increase in the incidence of atelectasis (risk ratio 1.47, 95% confidence interval 1.20 to 1.79; risk difference 6.5% higher, 2.8% higher to 10.9% higher, low-certainty evidence) and may increase postoperative pulmonary complications slightly (risk ratio 1.06, 0.77 to 1.46; risk difference 1.1% higher, 4.1% lower to 8.2% higher) but the evidence is very uncertain. A high fraction of inspired oxygen may result in little to no difference in mortality, nausea and vomiting, and length of hospital stay. Postoperative high-flow nasal oxygen may reduce the need to escalate respiratory support compared with conventional oxygen therapy (risk ratio 0.61, 0.41 to 0.91; risk difference 7.8% lower, 11.7% lower to 1.8% lower) but the evidence is very uncertain. High-flow nasal oxygen may result in little to no difference in mortality and reintubation rate. Compared with conventional oxygen therapy, postoperative non-invasive ventilation may decrease postoperative pulmonary complications (risk ratio 0.62, 0.44 to 0.87
{"title":"Perioperative oxygen therapy in patients undergoing surgical procedures: an overview of systematic reviews and meta-analyses.","authors":"Adel Elfeky, Yen-Fu Chen, Amy Grove, Keith Couper, Rachel Court, Sara Tomassini, Anna Wilson, Amy Hooper, Alexandra Buckle, Sharvari Vadeyar, Marion Thompson, Olalekan Uthman, Joyce Yeung","doi":"10.3310/TNTC4360","DOIUrl":"10.3310/TNTC4360","url":null,"abstract":"<p><strong>Background: </strong>Perioperative oxygen administration has been proposed as a strategy to reduce postoperative complications. However, uncertainty exists as to which strategies are the most clinically effective.</p><p><strong>Objectives: </strong>To provide an overview on the effectiveness of perioperative oxygen therapy and formulate recommendations to inform clinical decision-making and research.</p><p><strong>Methods: </strong>We followed the Preferred Reporting Items for Overviews of Reviews guidelines. We searched key databases for systematic reviews (from inception to September 2021) and randomised controlled trials (from April 2018 to March 2022) comparing perioperative oxygen strategies. Reviews with the most comprehensive coverage of literature were chosen as anchoring reviews. We assessed risk of bias for each anchoring review using the Risk of Bias in Systematic Reviews tool. We updated meta-analyses from anchoring reviews with data from recent randomised controlled trials and conducted subgroup analyses and meta-regression. We assessed the certainty of evidence using grading of recommendations assessment, development and evaluation framework and conducted trial sequential analysis. We used grading of recommendations assessment, development and evaluation informative statements to communicate our findings. Our advisory panel reviewed mapping of studies and interpretation of evidence.</p><p><strong>Results: </strong>We identified 59 systematic reviews and selected 5 anchoring reviews. A high fraction of inspired oxygen may result in a slight reduction in surgical site infection compared with a low fraction of inspired oxygen (risk ratio 0.91, 95% confidence interval 0.78 to 1.05; risk difference 1.2% lower, 2.9% lower to 0.7% higher, low-certainty evidence). This effect may be modified by type of surgery, oxygen delivery method or study quality. The evidence suggests that a high fraction of inspired oxygen results in a large increase in the incidence of atelectasis (risk ratio 1.47, 95% confidence interval 1.20 to 1.79; risk difference 6.5% higher, 2.8% higher to 10.9% higher, low-certainty evidence) and may increase postoperative pulmonary complications slightly (risk ratio 1.06, 0.77 to 1.46; risk difference 1.1% higher, 4.1% lower to 8.2% higher) but the evidence is very uncertain. A high fraction of inspired oxygen may result in little to no difference in mortality, nausea and vomiting, and length of hospital stay. Postoperative high-flow nasal oxygen may reduce the need to escalate respiratory support compared with conventional oxygen therapy (risk ratio 0.61, 0.41 to 0.91; risk difference 7.8% lower, 11.7% lower to 1.8% lower) but the evidence is very uncertain. High-flow nasal oxygen may result in little to no difference in mortality and reintubation rate. Compared with conventional oxygen therapy, postoperative non-invasive ventilation may decrease postoperative pulmonary complications (risk ratio 0.62, 0.44 to 0.87","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 44","pages":"1-139"},"PeriodicalIF":4.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12451503/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145064715","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}
Sarah Cockayne, Kalpita Baird, Sally Gates, Caroline Fairhurst, Joy Adamson, Rachel M Bottomley-Wise, Amie Woodward, Michael R Backhouse, Rachel Bye, Nina Davies, Catherine Hewitt, Colin Holton, Peter Knapp, Anne-Maree Keenan, Stewart Morrison, Daniel Parker, Daniel C Perry, Sarah Ronaldson, Mark Smith, Tim Theologis, Victoria Exley, Jane McAdam, David J Torgerson
Background: Children and young people with symptomatic pes planus (flat feet) often seek treatment from healthcare professionals. There are various treatment options, but there is a lack of high-quality evidence about which is most effective.
Objectives: To assess the clinical and cost-effectiveness of prefabricated orthoses, plus exercise and advice, compared with exercise and advice alone on physical function, measured using the physical domain of the Oxford Ankle Foot Questionnaire for Children, among children with symptomatic pes planus.
Design and methods: A pragmatic, multicentre, two-armed individually randomised controlled trial with an internal pilot, economic evaluation and qualitative study.
Setting and participants: Children and young people aged 6-14 years with symptomatic flat feet were recruited from hospital or community healthcare facilities in England and Wales. Participants were randomised 1 : 1 using a secure web-based randomisation system and followed up for up to 12 months.
Interventions: We planned to provide all participants with advice and exercises, with the intervention group also receiving a prefabricated orthosis. Due to the nature of the study treatments, blinding of participants or the research team was not possible.
Main outcome measures: The primary outcome was the physical domain subscale of the Oxford Ankle Foot Questionnaire for Children over the 12-month follow-up. Secondary outcomes included the physical domain subscale at 3, 6 and 12 months, and the 'School and Play' and 'Emotional' domains of the Oxford Ankle Foot Questionnaire, pain scores, healthcare resource use, EQ-5D-Y and Child Health Utility 9D at all time points. The qualitative study drew on health literacy and health belief perspectives and examined fidelity and explored the experiences of being in the trial for those receiving and delivering the study treatments.
Results: COVID-19 severely delayed trial set-up and recruitment and the study closed before meeting its recruitment target. Of 549 participants assessed for eligibility, 134 were randomised (intervention n = 70, control n = 64). The mean age of participants was 10.6 years (range 6.3-14.8) and 55.2% were male. No adverse events were reported. The planned statistical and health economic analyses could not be fully conducted due to the limited data. The qualitative study identified pain, posture and gait as the most common concerns by participants with pain relief as the primary motivator for seeking health care. Participants generally reported little understanding of their condition with barriers including misattribution (e.g. growing pains). Misinformation was common emphasising a need for accessible accurate education materials and structured follow-up care. There was a common belief that orthoses were superior to exer
{"title":"OrthoticS for TReatment of symptomatic flat feet In CHildren (OSTRICH): a randomised controlled trial.","authors":"Sarah Cockayne, Kalpita Baird, Sally Gates, Caroline Fairhurst, Joy Adamson, Rachel M Bottomley-Wise, Amie Woodward, Michael R Backhouse, Rachel Bye, Nina Davies, Catherine Hewitt, Colin Holton, Peter Knapp, Anne-Maree Keenan, Stewart Morrison, Daniel Parker, Daniel C Perry, Sarah Ronaldson, Mark Smith, Tim Theologis, Victoria Exley, Jane McAdam, David J Torgerson","doi":"10.3310/PLKJ4541","DOIUrl":"10.3310/PLKJ4541","url":null,"abstract":"<p><strong>Background: </strong>Children and young people with symptomatic pes planus (flat feet) often seek treatment from healthcare professionals. There are various treatment options, but there is a lack of high-quality evidence about which is most effective.</p><p><strong>Objectives: </strong>To assess the clinical and cost-effectiveness of prefabricated orthoses, plus exercise and advice, compared with exercise and advice alone on physical function, measured using the physical domain of the Oxford Ankle Foot Questionnaire for Children, among children with symptomatic pes planus.</p><p><strong>Design and methods: </strong>A pragmatic, multicentre, two-armed individually randomised controlled trial with an internal pilot, economic evaluation and qualitative study.</p><p><strong>Setting and participants: </strong>Children and young people aged 6-14 years with symptomatic flat feet were recruited from hospital or community healthcare facilities in England and Wales. Participants were randomised 1 : 1 using a secure web-based randomisation system and followed up for up to 12 months.</p><p><strong>Interventions: </strong>We planned to provide all participants with advice and exercises, with the intervention group also receiving a prefabricated orthosis. Due to the nature of the study treatments, blinding of participants or the research team was not possible.</p><p><strong>Main outcome measures: </strong>The primary outcome was the physical domain subscale of the Oxford Ankle Foot Questionnaire for Children over the 12-month follow-up. Secondary outcomes included the physical domain subscale at 3, 6 and 12 months, and the 'School and Play' and 'Emotional' domains of the Oxford Ankle Foot Questionnaire, pain scores, healthcare resource use, EQ-5D-Y and Child Health Utility 9D at all time points. The qualitative study drew on health literacy and health belief perspectives and examined fidelity and explored the experiences of being in the trial for those receiving and delivering the study treatments.</p><p><strong>Results: </strong>COVID-19 severely delayed trial set-up and recruitment and the study closed before meeting its recruitment target. Of 549 participants assessed for eligibility, 134 were randomised (intervention <i>n</i> = 70, control <i>n</i> = 64). The mean age of participants was 10.6 years (range 6.3-14.8) and 55.2% were male. No adverse events were reported. The planned statistical and health economic analyses could not be fully conducted due to the limited data. The qualitative study identified pain, posture and gait as the most common concerns by participants with pain relief as the primary motivator for seeking health care. Participants generally reported little understanding of their condition with barriers including misattribution (e.g. growing pains). Misinformation was common emphasising a need for accessible accurate education materials and structured follow-up care. There was a common belief that orthoses were superior to exer","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-49"},"PeriodicalIF":4.0,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376204/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144834938","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}
Olalekan A Uthman, Lena Al-Khudairy, Chidozie Nduka, Rachel Court, Jodie Enderby, Seun Anjorin, Hema Mistry, G J Melendez-Torres, Sian Taylor-Phillips, Aileen Clarke
<p><strong>Background: </strong>Cardiovascular disease remains a leading cause of morbidity and mortality worldwide. This series of systematic reviews and meta-analyses synthesised evidence on the effectiveness, comparative effectiveness and cost-effectiveness of pharmacological and non-pharmacological interventions for primary cardiovascular disease prevention.</p><p><strong>Methods: </strong>Five systematic reviews and meta-analyses were conducted using rigorous methods, including comprehensive searches, duplicate screening, risk-of-bias assessments and adherence to reporting guidelines. An umbrella review summarised evidence from 95 systematic reviews. A machine learning study developed a parallel Convolutional Neural Network algorithm with 96.4% recall and 99.1% precision for study screening. A network meta-analysis compared preventive strategies across 139 trials (1,053,772 participants). Simulation modelling projected the population impact of policy interventions, and a cost-effectiveness review appraised eight United Kingdom-based economic evaluations.</p><p><strong>Results: </strong>The umbrella review found that antiplatelets reduced major cardiovascular disease events in 8/17 meta-analyses (relative risks 0.85-0.97), while statins reduced cardiovascular disease mortality (relative risks 0.71-0.89), all-cause mortality (relative risks 0.66-0.93) and major cardiovascular disease events (relative risks 0.59-0.90). sodium-glucose transport protein 2 inhibitors reduced major cardiovascular disease events by 8% (relative risk 0.92, 95% confidence interval 0.89 to 0.95) and all-cause mortality by 6% (relative risk 0.94, 95% confidence interval 0.90 to 0.98). Non-pharmacological interventions showed limited evidence, though vitamin D (relative risks 0.93-0.94) and dietary changes (relative risk 0.91, 95% confidence interval 0.85 to 0.97) had some benefits. The network meta-analysis found that antihypertensives (relative risk 0.76, 95% confidence interval 0.64 to 0.90), intensive blood pressure control (relative risk 0.66, 95% confidence interval 0.46 to 0.96), statins (relative risk 0.81, 95% confidence interval 0.71 to 0.91) and multifactorial lifestyle interventions (relative risk 0.75, 95% confidence interval 0.61 to 0.92) significantly reduced composite cardiovascular disease events and mortality. Blood pressure lowering also reduced all-cause mortality (relative risk 0.82, 95% confidence interval 0.71 to 0.94). Simulation modelling projected substantial population-level health gains. National salt reduction programmes could prevent 1900-48,000 cardiovascular disease deaths annually, while tobacco control initiatives could avert 15,500 deaths yearly. In the United Kingdom, salt reduction could prevent 4450 deaths annually, and transfat elimination could prevent 1700-3500 deaths yearly. Cost-effectiveness analyses found most interventions had incremental cost-effectiveness ratio below £20,000-30,000 per quality-adjusted life-year. However, i
{"title":"Determining optimal strategies for primary prevention of cardiovascular disease: a synopsis of an evidence synthesis study.","authors":"Olalekan A Uthman, Lena Al-Khudairy, Chidozie Nduka, Rachel Court, Jodie Enderby, Seun Anjorin, Hema Mistry, G J Melendez-Torres, Sian Taylor-Phillips, Aileen Clarke","doi":"10.3310/KGFA8471","DOIUrl":"10.3310/KGFA8471","url":null,"abstract":"<p><strong>Background: </strong>Cardiovascular disease remains a leading cause of morbidity and mortality worldwide. This series of systematic reviews and meta-analyses synthesised evidence on the effectiveness, comparative effectiveness and cost-effectiveness of pharmacological and non-pharmacological interventions for primary cardiovascular disease prevention.</p><p><strong>Methods: </strong>Five systematic reviews and meta-analyses were conducted using rigorous methods, including comprehensive searches, duplicate screening, risk-of-bias assessments and adherence to reporting guidelines. An umbrella review summarised evidence from 95 systematic reviews. A machine learning study developed a parallel Convolutional Neural Network algorithm with 96.4% recall and 99.1% precision for study screening. A network meta-analysis compared preventive strategies across 139 trials (1,053,772 participants). Simulation modelling projected the population impact of policy interventions, and a cost-effectiveness review appraised eight United Kingdom-based economic evaluations.</p><p><strong>Results: </strong>The umbrella review found that antiplatelets reduced major cardiovascular disease events in 8/17 meta-analyses (relative risks 0.85-0.97), while statins reduced cardiovascular disease mortality (relative risks 0.71-0.89), all-cause mortality (relative risks 0.66-0.93) and major cardiovascular disease events (relative risks 0.59-0.90). sodium-glucose transport protein 2 inhibitors reduced major cardiovascular disease events by 8% (relative risk 0.92, 95% confidence interval 0.89 to 0.95) and all-cause mortality by 6% (relative risk 0.94, 95% confidence interval 0.90 to 0.98). Non-pharmacological interventions showed limited evidence, though vitamin D (relative risks 0.93-0.94) and dietary changes (relative risk 0.91, 95% confidence interval 0.85 to 0.97) had some benefits. The network meta-analysis found that antihypertensives (relative risk 0.76, 95% confidence interval 0.64 to 0.90), intensive blood pressure control (relative risk 0.66, 95% confidence interval 0.46 to 0.96), statins (relative risk 0.81, 95% confidence interval 0.71 to 0.91) and multifactorial lifestyle interventions (relative risk 0.75, 95% confidence interval 0.61 to 0.92) significantly reduced composite cardiovascular disease events and mortality. Blood pressure lowering also reduced all-cause mortality (relative risk 0.82, 95% confidence interval 0.71 to 0.94). Simulation modelling projected substantial population-level health gains. National salt reduction programmes could prevent 1900-48,000 cardiovascular disease deaths annually, while tobacco control initiatives could avert 15,500 deaths yearly. In the United Kingdom, salt reduction could prevent 4450 deaths annually, and transfat elimination could prevent 1700-3500 deaths yearly. Cost-effectiveness analyses found most interventions had incremental cost-effectiveness ratio below £20,000-30,000 per quality-adjusted life-year. However, i","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 37","pages":"1-18"},"PeriodicalIF":4.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12434577/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144872833","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}
Richard Ig Holt, Katharine Barnard-Kelly, Mayank Patel, Philip Newland-Jones, Suvitesh Luthra, Jo Picot, Helen Partridge, Andrew Cook
<p><strong>Background: </strong>Surgical outcomes are worse in people with diabetes, in part, because of the effects of hyperglycaemia, obesity and other comorbidities. Two important uncertainties in the management of people with diabetes undergoing major surgery exist: (1) how to improve diabetes management prior to an elective procedure and (2) whether that improved management leads to better post-operative outcomes.</p><p><strong>Objective: </strong>The Optimising Cardiac Surgery ouTcOmes in People with diabeteS project aimed to assess whether a pre-operative outpatient intervention delivered by a multidisciplinary specialist diabetes team could improve diabetes management and cardiac surgical outcomes for people with diabetes. Although the intervention could be applied to any surgical discipline, cardiothoracic surgery was chosen because 30-40% of those undergoing elective cardiac revascularisation have diabetes.</p><p><strong>Methods: </strong>The project had three phases: (1) designing the intervention, (2) a pilot study of the intervention and (3) a multicentre randomised controlled study in United Kingdom cardiothoracic centres to assess whether the intervention could improve surgical outcomes. The first two phases were completed, but the COVID-19 pandemic and its subsequent effects on cardiothoracic services and research capacity in the United Kingdom meant that the randomised controlled study could not be undertaken.</p><p><strong>Intervention development: </strong>Two rapid literature reviews were undertaken to understand what factors influence surgical outcomes in people with diabetes and what interventions have previously been tested. The Optimising Cardiac Surgery ouTcOmes in People with diabeteS intervention was based on an existing nurse-led outpatient intervention, delivered in the 3 months before elective orthopaedic surgery. This intervention reduced pre-operative glycated haemoglobin and reduced length of stay. We undertook a survey of United Kingdom cardiothoracic surgeons, which found limited and inconsistent pre-operative management of people with diabetes awaiting cardiothoracic surgery. A prototype intervention was developed following discussions with relevant stakeholders.</p><p><strong>Pilot study: </strong>The pilot feasibility study recruited 17 people with diabetes and was undertaken by the diabetes and cardiothoracic surgery departments at University Hospital Southampton NHS Foundation Trust. Biomedical data were collected at baseline and prior to surgery. We assessed how the intervention was used. In-depth qualitative interviews with participants and healthcare professionals explored perceptions and experiences of the intervention and how it might be improved. Thirteen people completed the study and underwent cardiothoracic surgery. All components of the Optimising Cardiac Surgery ouTcOmes in People with diabeteS intervention were used, but not all parts were used for all participants. Minor changes were made to th
{"title":"Optimising cardiac surgery outcomes in people with diabetes: the OCTOPuS pilot feasibility study.","authors":"Richard Ig Holt, Katharine Barnard-Kelly, Mayank Patel, Philip Newland-Jones, Suvitesh Luthra, Jo Picot, Helen Partridge, Andrew Cook","doi":"10.3310/POYW3311","DOIUrl":"10.3310/POYW3311","url":null,"abstract":"<p><strong>Background: </strong>Surgical outcomes are worse in people with diabetes, in part, because of the effects of hyperglycaemia, obesity and other comorbidities. Two important uncertainties in the management of people with diabetes undergoing major surgery exist: (1) how to improve diabetes management prior to an elective procedure and (2) whether that improved management leads to better post-operative outcomes.</p><p><strong>Objective: </strong>The Optimising Cardiac Surgery ouTcOmes in People with diabeteS project aimed to assess whether a pre-operative outpatient intervention delivered by a multidisciplinary specialist diabetes team could improve diabetes management and cardiac surgical outcomes for people with diabetes. Although the intervention could be applied to any surgical discipline, cardiothoracic surgery was chosen because 30-40% of those undergoing elective cardiac revascularisation have diabetes.</p><p><strong>Methods: </strong>The project had three phases: (1) designing the intervention, (2) a pilot study of the intervention and (3) a multicentre randomised controlled study in United Kingdom cardiothoracic centres to assess whether the intervention could improve surgical outcomes. The first two phases were completed, but the COVID-19 pandemic and its subsequent effects on cardiothoracic services and research capacity in the United Kingdom meant that the randomised controlled study could not be undertaken.</p><p><strong>Intervention development: </strong>Two rapid literature reviews were undertaken to understand what factors influence surgical outcomes in people with diabetes and what interventions have previously been tested. The Optimising Cardiac Surgery ouTcOmes in People with diabeteS intervention was based on an existing nurse-led outpatient intervention, delivered in the 3 months before elective orthopaedic surgery. This intervention reduced pre-operative glycated haemoglobin and reduced length of stay. We undertook a survey of United Kingdom cardiothoracic surgeons, which found limited and inconsistent pre-operative management of people with diabetes awaiting cardiothoracic surgery. A prototype intervention was developed following discussions with relevant stakeholders.</p><p><strong>Pilot study: </strong>The pilot feasibility study recruited 17 people with diabetes and was undertaken by the diabetes and cardiothoracic surgery departments at University Hospital Southampton NHS Foundation Trust. Biomedical data were collected at baseline and prior to surgery. We assessed how the intervention was used. In-depth qualitative interviews with participants and healthcare professionals explored perceptions and experiences of the intervention and how it might be improved. Thirteen people completed the study and underwent cardiothoracic surgery. All components of the Optimising Cardiac Surgery ouTcOmes in People with diabeteS intervention were used, but not all parts were used for all participants. Minor changes were made to th","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 39","pages":"1-31"},"PeriodicalIF":4.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376008/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144872835","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}
Oliver Wiseman, Daron Smith, Kath Starr, Lorna Aucott, Rodolfo Hernández, Ruth Thomas, Steven MacLennan, Charles Terry Clark, Graeme MacLennan, Dawn McRae, Victoria Bell, Seonaidh Cotton, Zara Gall, Ben Turney, Samuel McClinton
<p><strong>Background: </strong>Renal tract stone disease is common. The three intervention options are shockwave lithotripsy, flexible ureteroscopic stone treatment and keyhole surgery.</p><p><strong>Objectives: </strong>To determine which of shockwave lithotripsy, flexible ureteroscopic stone treatment and keyhole surgery offer the best outcomes in terms of health and quality of life, clinical effectiveness and cost-effectiveness for people with lower pole kidney stones.</p><p><strong>Design: </strong>The PUrE study comprised two pragmatic multicentre, open-label, superiority randomised controlled trials: RCT1 for lower pole stones ≤ 10 mm and RCT2 for lower pole stones > 10 and ≤ 25 mm.</p><p><strong>Setting: </strong>National Health Service Urology departments.</p><p><strong>Participants: </strong>Adults presenting with lower pole renal stones, able to undergo any of the treatments and complete trial procedures.</p><p><strong>Intervention: </strong>Eligible participants were randomised in RCT1 to flexible ureteroscopic stone treatment or shockwave lithotripsy; and in RCT2 to flexible ureteroscopic stone treatment or keyhole surgery.</p><p><strong>Main outcome measures: </strong>The primary outcome measure was health status 'area under the curve', measured weekly to 12 weeks post intervention with the EuroQol-5 Dimensions, five-level version. The primary economic outcome was the incremental cost per quality-adjusted life-year gained at 12 months from randomisation.</p><p><strong>Results: </strong><i>RCT1:</i> A total of 461 participants were randomised: 231 to flexible ureteroscopic stone treatment; and 230 to shockwave lithotripsy. <i>RCT2:</i> A total of 159 participants were randomised: 73 to flexible ureteroscopic stone treatment; and 86 to keyhole surgery.</p><p><strong>Primary outcome: </strong><i>RCT1:</i> The mean health status area under the curve was 0.807 (standard deviation 0.205) in the flexible ureteroscopic stone treatment group (<i>n</i> = 164) and 0.826 (standard deviation 0.207) in the shockwave lithotripsy group (<i>n</i> = 188). The between-group difference, 0.024 (95% confidence interval -0.004 to 0.053), was a small difference in favour of flexible ureteroscopic stone treatment after correcting for a baseline imbalance. Complete stone clearance was higher with flexible ureteroscopic stone treatment (72%) than shockwave lithotripsy (36%). <i>RCT2</i>: The mean health status area under the curve was 0.794 (standard deviation 0.198) in the flexible ureteroscopic stone treatment group (<i>n</i> = 57) and 0.818 (standard deviation 0.217) in the keyhole surgery group (<i>n</i> = 63). The between-group difference, -0.07 (95% confidence interval -0.11 to -0.02), was a borderline meaningful difference favouring keyhole surgery. Complete stone clearance was higher with keyhole surgery (71%) than flexible ureteroscopic stone treatment (48%).</p><p><strong>Economic evaluation: </strong><i>RCT1</i>: Flexible ureteroscopic stone treatm
{"title":"Clinical and cost-effectiveness of percutaneous nephrolithotomy, flexible ureterorenoscopy and extracorporeal shockwave lithotripsy for lower pole stones: the PUrE RCTs.","authors":"Oliver Wiseman, Daron Smith, Kath Starr, Lorna Aucott, Rodolfo Hernández, Ruth Thomas, Steven MacLennan, Charles Terry Clark, Graeme MacLennan, Dawn McRae, Victoria Bell, Seonaidh Cotton, Zara Gall, Ben Turney, Samuel McClinton","doi":"10.3310/WFRE6844","DOIUrl":"10.3310/WFRE6844","url":null,"abstract":"<p><strong>Background: </strong>Renal tract stone disease is common. The three intervention options are shockwave lithotripsy, flexible ureteroscopic stone treatment and keyhole surgery.</p><p><strong>Objectives: </strong>To determine which of shockwave lithotripsy, flexible ureteroscopic stone treatment and keyhole surgery offer the best outcomes in terms of health and quality of life, clinical effectiveness and cost-effectiveness for people with lower pole kidney stones.</p><p><strong>Design: </strong>The PUrE study comprised two pragmatic multicentre, open-label, superiority randomised controlled trials: RCT1 for lower pole stones ≤ 10 mm and RCT2 for lower pole stones > 10 and ≤ 25 mm.</p><p><strong>Setting: </strong>National Health Service Urology departments.</p><p><strong>Participants: </strong>Adults presenting with lower pole renal stones, able to undergo any of the treatments and complete trial procedures.</p><p><strong>Intervention: </strong>Eligible participants were randomised in RCT1 to flexible ureteroscopic stone treatment or shockwave lithotripsy; and in RCT2 to flexible ureteroscopic stone treatment or keyhole surgery.</p><p><strong>Main outcome measures: </strong>The primary outcome measure was health status 'area under the curve', measured weekly to 12 weeks post intervention with the EuroQol-5 Dimensions, five-level version. The primary economic outcome was the incremental cost per quality-adjusted life-year gained at 12 months from randomisation.</p><p><strong>Results: </strong><i>RCT1:</i> A total of 461 participants were randomised: 231 to flexible ureteroscopic stone treatment; and 230 to shockwave lithotripsy. <i>RCT2:</i> A total of 159 participants were randomised: 73 to flexible ureteroscopic stone treatment; and 86 to keyhole surgery.</p><p><strong>Primary outcome: </strong><i>RCT1:</i> The mean health status area under the curve was 0.807 (standard deviation 0.205) in the flexible ureteroscopic stone treatment group (<i>n</i> = 164) and 0.826 (standard deviation 0.207) in the shockwave lithotripsy group (<i>n</i> = 188). The between-group difference, 0.024 (95% confidence interval -0.004 to 0.053), was a small difference in favour of flexible ureteroscopic stone treatment after correcting for a baseline imbalance. Complete stone clearance was higher with flexible ureteroscopic stone treatment (72%) than shockwave lithotripsy (36%). <i>RCT2</i>: The mean health status area under the curve was 0.794 (standard deviation 0.198) in the flexible ureteroscopic stone treatment group (<i>n</i> = 57) and 0.818 (standard deviation 0.217) in the keyhole surgery group (<i>n</i> = 63). The between-group difference, -0.07 (95% confidence interval -0.11 to -0.02), was a borderline meaningful difference favouring keyhole surgery. Complete stone clearance was higher with keyhole surgery (71%) than flexible ureteroscopic stone treatment (48%).</p><p><strong>Economic evaluation: </strong><i>RCT1</i>: Flexible ureteroscopic stone treatm","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 40","pages":"1-186"},"PeriodicalIF":4.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376203/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144872875","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}
Nia Wyn Jones, Eleanor J Mitchell, Kate F Walker, Susan Ayers, Lucy Bradshaw, Georgina Constantinou, Tasso Gazis, Shalini Ojha, Phoebe Pallotti, Stavros Petrou, Rachel Plachcinski, Michael Rimmer, Liz Schroeder, Jim G Thornton, Natalie Wakefield
<p><strong>Background: </strong>Diabetes in pregnancy is common, affecting 5-10% of pregnant women. Poor glycaemic control in labour is associated with neonatal hypoglycaemia and other adverse outcomes for mother and baby, but tight glucose control is burdensome, intrusive and may not always be necessary. The ideal intrapartum glucose target level is unknown, traditionally 'tight' control (target 4-7 mmol/l) has been recommended; however, this increases the risk of maternal hypoglycaemia.</p><p><strong>Objective: </strong>To determine the feasibility of a randomised clinical trial to compare clinical and cost-effectiveness of permissive versus intensive intrapartum glycaemic control in labour in pregnancies complicated by diabetes.</p><p><strong>Design: </strong>A mixed-methods study including audit of clinical guidelines from United Kingdom maternity units, online surveys of women with diabetes and healthcare professionals, service evaluation of intrapartum glycaemic care, Delphi survey and consensus meeting. Data from these work packages led to the design of a clinical trial, and qualitative interviews were held to understand acceptability of the trial.</p><p><strong>Setting: </strong>National Health Service maternity services and online input from service users.</p><p><strong>Participants: </strong>Healthcare professionals and women with type 1 or type 2 diabetes mellitus or gestational diabetes (currently pregnant or having birthed after active labour in past 3 years).</p><p><strong>Results: </strong>There is significant variation in the recommended frequency of testing for gestational diabetes in labour, technologies used to test glucose levels in labour and administer insulin in type 1 diabetes mellitus, and in how neonatal hypoglycaemia is defined. Of surveyed women, 66% would be willing to participate in a future trial, with 23% unsure without further information. The service evaluation showed that once glucose testing had commenced, it was repeated after 1 hour in 18%, 2 hours in 38% and 4 hours in 45% of women. Neonatal hypoglycaemia was considered the most important neonatal outcome for a future trial, with maternal satisfaction the most important maternal outcome. The incidence of neonatal hypoglycaemia (defined as glucose < 2.6 mmol/l) was 47% in type 1 diabetes mellitus, 45% in type 2 diabetes mellitus and 16% in gestational diabetes mellitus. A non-inferiority trial to compare permissive versus intensive glucose control was designed to include all types of diabetes in an umbrella trial (conduct more than one trial simultaneously). Women and healthcare professionals considered the trial design acceptable and feasible, though noted important considerations in the design and conduct.</p><p><strong>Limitations: </strong>Glucose levels may be poorly recorded in maternity notes and in practice tested more frequently than the study suggests. Sample sizes in some of the work packages were smaller than our pre-specified target, attrition in
{"title":"Glycaemic control in labour with diabetes: GILD, a scoping study.","authors":"Nia Wyn Jones, Eleanor J Mitchell, Kate F Walker, Susan Ayers, Lucy Bradshaw, Georgina Constantinou, Tasso Gazis, Shalini Ojha, Phoebe Pallotti, Stavros Petrou, Rachel Plachcinski, Michael Rimmer, Liz Schroeder, Jim G Thornton, Natalie Wakefield","doi":"10.3310/KHGD2761","DOIUrl":"10.3310/KHGD2761","url":null,"abstract":"<p><strong>Background: </strong>Diabetes in pregnancy is common, affecting 5-10% of pregnant women. Poor glycaemic control in labour is associated with neonatal hypoglycaemia and other adverse outcomes for mother and baby, but tight glucose control is burdensome, intrusive and may not always be necessary. The ideal intrapartum glucose target level is unknown, traditionally 'tight' control (target 4-7 mmol/l) has been recommended; however, this increases the risk of maternal hypoglycaemia.</p><p><strong>Objective: </strong>To determine the feasibility of a randomised clinical trial to compare clinical and cost-effectiveness of permissive versus intensive intrapartum glycaemic control in labour in pregnancies complicated by diabetes.</p><p><strong>Design: </strong>A mixed-methods study including audit of clinical guidelines from United Kingdom maternity units, online surveys of women with diabetes and healthcare professionals, service evaluation of intrapartum glycaemic care, Delphi survey and consensus meeting. Data from these work packages led to the design of a clinical trial, and qualitative interviews were held to understand acceptability of the trial.</p><p><strong>Setting: </strong>National Health Service maternity services and online input from service users.</p><p><strong>Participants: </strong>Healthcare professionals and women with type 1 or type 2 diabetes mellitus or gestational diabetes (currently pregnant or having birthed after active labour in past 3 years).</p><p><strong>Results: </strong>There is significant variation in the recommended frequency of testing for gestational diabetes in labour, technologies used to test glucose levels in labour and administer insulin in type 1 diabetes mellitus, and in how neonatal hypoglycaemia is defined. Of surveyed women, 66% would be willing to participate in a future trial, with 23% unsure without further information. The service evaluation showed that once glucose testing had commenced, it was repeated after 1 hour in 18%, 2 hours in 38% and 4 hours in 45% of women. Neonatal hypoglycaemia was considered the most important neonatal outcome for a future trial, with maternal satisfaction the most important maternal outcome. The incidence of neonatal hypoglycaemia (defined as glucose < 2.6 mmol/l) was 47% in type 1 diabetes mellitus, 45% in type 2 diabetes mellitus and 16% in gestational diabetes mellitus. A non-inferiority trial to compare permissive versus intensive glucose control was designed to include all types of diabetes in an umbrella trial (conduct more than one trial simultaneously). Women and healthcare professionals considered the trial design acceptable and feasible, though noted important considerations in the design and conduct.</p><p><strong>Limitations: </strong>Glucose levels may be poorly recorded in maternity notes and in practice tested more frequently than the study suggests. Sample sizes in some of the work packages were smaller than our pre-specified target, attrition in","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 41","pages":"1-150"},"PeriodicalIF":4.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376010/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144872876","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}
Rustam Al-Shahi Salman, Neil Kitchen, Laura Forsyth, Vijeya Ganesan, Peter S Hall, Kirsty Harkness, Peter Ja Hutchinson, Steff C Lewis, Matthias Wr Radatz, Carole Turner, Julia Wade, David Cs White, Philip M White
<p><strong>Background: </strong>The top priority for research into symptomatic cerebral cavernous malformation (also known as brain cavernoma) is whether to have medical management and intervention (using neurosurgical resection or stereotactic radiosurgery) or medical management alone.</p><p><strong>Objectives: </strong>The primary objective was to assess the feasibility of performing a definitive randomised trial addressing this top priority. The secondary objectives were to set up a collaboration involving patient advocacy organisations and clinicians in the United Kingdom and Ireland; perform a QuinteT Recruitment Intervention to identify facilitators and address barriers to recruitment; and conduct a pilot randomised trial with ≈60 participants.</p><p><strong>Design: </strong>Prospective, randomised, open-label, assessor-blinded, parallel-group trial. A mixed-methods QuinteT Recruitment Intervention analysed sites' screening logs and qualitative data from audio-recorded recruitment discussions, interviews with healthcare professionals and patients, investigator workshops and observation of meetings.</p><p><strong>Setting: </strong>Neuroscience hospitals in the United Kingdom and Ireland.</p><p><strong>Participants: </strong>We aimed to recruit ≈60 people of any age, gender and ethnicity who had mental capacity, resided in the United Kingdom/Ireland, and had a brain cavernoma that had caused symptoms due to intracranial haemorrhage, non-haemorrhagic progressive/persistent focal neurological deficit or epileptic seizure(s).</p><p><strong>Interventions: </strong>We identified and addressed barriers and facilitators to optimise informed consent and recruitment. Computerised, web-based randomisation assigned participants (1 : 1) to treatment of their symptomatic brain cavernoma with medical management and intervention (using neurosurgical resection or stereotactic radiosurgery) or medical management alone. Assignment was open to investigators, participants and carers but not clinical outcome event adjudicators.</p><p><strong>Main outcome measures: </strong>Feasibility outcomes included site engagement, recruitment, choice of surgical management, retention, adherence, data quality, clinical outcome event rate and protocol implementation. The primary clinical outcome was symptomatic intracranial haemorrhage or new persistent/progressive non-haemorrhagic focal neurological deficit due to brain cavernoma or intervention during ≥ 6 months of follow-up.</p><p><strong>Results: </strong>Investigators screened 511 patients at 28/40 (70%) sites in the United Kingdom: 322 (63%) eligible, 202 (63%) approached, 96 (48%) uncertain about whether to have intervention and 72 participants [median age was 51 years (interquartile range 39-59), 41 (57%) female, 66 (92%) white, 56 (78%) with prior intracranial haemorrhage and 28 (39%) with prior epileptic seizure] were randomly assigned to medical management and intervention (<i>n</i> = 36; 12 to neurosurgical resecti
{"title":"Medical management and intervention (using neurosurgical resection or stereotactic radiosurgery) versus medical management alone for symptomatic brain cavernoma: the CARE pilot RCT.","authors":"Rustam Al-Shahi Salman, Neil Kitchen, Laura Forsyth, Vijeya Ganesan, Peter S Hall, Kirsty Harkness, Peter Ja Hutchinson, Steff C Lewis, Matthias Wr Radatz, Carole Turner, Julia Wade, David Cs White, Philip M White","doi":"10.3310/GJRS5321","DOIUrl":"10.3310/GJRS5321","url":null,"abstract":"<p><strong>Background: </strong>The top priority for research into symptomatic cerebral cavernous malformation (also known as brain cavernoma) is whether to have medical management and intervention (using neurosurgical resection or stereotactic radiosurgery) or medical management alone.</p><p><strong>Objectives: </strong>The primary objective was to assess the feasibility of performing a definitive randomised trial addressing this top priority. The secondary objectives were to set up a collaboration involving patient advocacy organisations and clinicians in the United Kingdom and Ireland; perform a QuinteT Recruitment Intervention to identify facilitators and address barriers to recruitment; and conduct a pilot randomised trial with ≈60 participants.</p><p><strong>Design: </strong>Prospective, randomised, open-label, assessor-blinded, parallel-group trial. A mixed-methods QuinteT Recruitment Intervention analysed sites' screening logs and qualitative data from audio-recorded recruitment discussions, interviews with healthcare professionals and patients, investigator workshops and observation of meetings.</p><p><strong>Setting: </strong>Neuroscience hospitals in the United Kingdom and Ireland.</p><p><strong>Participants: </strong>We aimed to recruit ≈60 people of any age, gender and ethnicity who had mental capacity, resided in the United Kingdom/Ireland, and had a brain cavernoma that had caused symptoms due to intracranial haemorrhage, non-haemorrhagic progressive/persistent focal neurological deficit or epileptic seizure(s).</p><p><strong>Interventions: </strong>We identified and addressed barriers and facilitators to optimise informed consent and recruitment. Computerised, web-based randomisation assigned participants (1 : 1) to treatment of their symptomatic brain cavernoma with medical management and intervention (using neurosurgical resection or stereotactic radiosurgery) or medical management alone. Assignment was open to investigators, participants and carers but not clinical outcome event adjudicators.</p><p><strong>Main outcome measures: </strong>Feasibility outcomes included site engagement, recruitment, choice of surgical management, retention, adherence, data quality, clinical outcome event rate and protocol implementation. The primary clinical outcome was symptomatic intracranial haemorrhage or new persistent/progressive non-haemorrhagic focal neurological deficit due to brain cavernoma or intervention during ≥ 6 months of follow-up.</p><p><strong>Results: </strong>Investigators screened 511 patients at 28/40 (70%) sites in the United Kingdom: 322 (63%) eligible, 202 (63%) approached, 96 (48%) uncertain about whether to have intervention and 72 participants [median age was 51 years (interquartile range 39-59), 41 (57%) female, 66 (92%) white, 56 (78%) with prior intracranial haemorrhage and 28 (39%) with prior epileptic seizure] were randomly assigned to medical management and intervention (<i>n</i> = 36; 12 to neurosurgical resecti","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 38","pages":"1-24"},"PeriodicalIF":4.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376205/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144872834","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}
Jeremy Howick, Martina Svobodova, Shaun Treweek, Katie Gillies, Adrian Edwards, Peter Bower, Jennifer Bostock, Nina Jacob, Kerenza Hood
Background: Variation in the way information about potential trial intervention benefits and harms is conveyed within patient information leaflets can cause avoidable information-induced ('nocebo') harm, research waste, and may be unethical.
Objectives: 1. To develop stakeholder-informed principles to guide how to describe information about potential trial intervention benefits and harms within patient information leaflets. 2. To test whether using these principles are feasible for testing in trials that measure whether they improve recruitment and adverse event rates. 3. To develop and disseminate guidance on how to implement the principles.
Methods: We used a mixed methodology consisting of three work packages. Work package 1 involved a modified Delphi survey and consensus meeting to develop the principles for harmonising the way information regarding potential benefits and harms are shared. Work package 2 involved testing whether the principles could be used to transform existing patient information leaflets by recruiting host trials to compare standard patient information leaflets with patient information leaflets developed using the principles 'principled patient information leaflets'. We also set up an infrastructure to test whether they could reduce variation, impact trial recruitment and reduce reported adverse events. Work package 3 involved developing and disseminating guidance for using the principles.
Results: For work package 1, 250 participants completed the Delphi survey and 7 principles were agreed upon: (1) all potential intervention harms should be listed, (2) potential harms should be separated into 'serious' and 'less serious', (3) if not all potential harms are known, this needs to be explicitly stated, (4) all potential benefits should be listed, (5) potential benefits and harms associated with trial participation need to be compared with those associated with non-participation, (6) suitable visual representations should be added where appropriate, and (7) information about potential benefits and harms should not be separated by more than one page. For work package 2, we developed principled patient information leaflets for five host trials and interviewed two members of each host trial team. Two host trials agreed to compare the patient information leaflets with principled patient information leaflets using Studies Within a Trial, and we published a protocol for a meta-analysis that will synthesise the results. For work package 3, 25 participants attended a hybrid workshop and recommended that researchers and Research Ethics Committee members should use the principles to design and evaluate patient information leaflets. We produced a guidance booklet and website, which are currently being used by some Health Research Authority Research Ethics Committees.
Conclusions: A strong consensus was reached regarding seven
{"title":"Developing evidence-based guidelines for describing potential benefits and harms within patient information leaflets/sheets (PILs) that inform and do not cause harm (PrinciPILs).","authors":"Jeremy Howick, Martina Svobodova, Shaun Treweek, Katie Gillies, Adrian Edwards, Peter Bower, Jennifer Bostock, Nina Jacob, Kerenza Hood","doi":"10.3310/GJJH2402","DOIUrl":"10.3310/GJJH2402","url":null,"abstract":"<p><strong>Background: </strong>Variation in the way information about potential trial intervention benefits and harms is conveyed within patient information leaflets can cause avoidable information-induced ('nocebo') harm, research waste, and may be unethical.</p><p><strong>Objectives: </strong>1. To develop stakeholder-informed principles to guide how to describe information about potential trial intervention benefits and harms within patient information leaflets. 2. To test whether using these principles are feasible for testing in trials that measure whether they improve recruitment and adverse event rates. 3. To develop and disseminate guidance on how to implement the principles.</p><p><strong>Methods: </strong>We used a mixed methodology consisting of three work packages. Work package 1 involved a modified Delphi survey and consensus meeting to develop the principles for harmonising the way information regarding potential benefits and harms are shared. Work package 2 involved testing whether the principles could be used to transform existing patient information leaflets by recruiting host trials to compare standard patient information leaflets with patient information leaflets developed using the principles 'principled patient information leaflets'. We also set up an infrastructure to test whether they could reduce variation, impact trial recruitment and reduce reported adverse events. Work package 3 involved developing and disseminating guidance for using the principles.</p><p><strong>Results: </strong>For work package 1, 250 participants completed the Delphi survey and 7 principles were agreed upon: (1) all potential intervention harms should be listed, (2) potential harms should be separated into 'serious' and 'less serious', (3) if not all potential harms are known, this needs to be explicitly stated, (4) all potential benefits should be listed, (5) potential benefits and harms associated with trial participation need to be compared with those associated with non-participation, (6) suitable visual representations should be added where appropriate, and (7) information about potential benefits and harms should not be separated by more than one page. For work package 2, we developed principled patient information leaflets for five host trials and interviewed two members of each host trial team. Two host trials agreed to compare the patient information leaflets with principled patient information leaflets using Studies Within a Trial, and we published a protocol for a meta-analysis that will synthesise the results. For work package 3, 25 participants attended a hybrid workshop and recommended that researchers and Research Ethics Committee members should use the principles to design and evaluate patient information leaflets. We produced a guidance booklet and website, which are currently being used by some Health Research Authority Research Ethics Committees.</p><p><strong>Conclusions: </strong>A strong consensus was reached regarding seven ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 43","pages":"1-20"},"PeriodicalIF":4.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12434871/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144951732","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}
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