Steven Brown, Daniel Hind, Emily Strong, Mike Bradburn, Farhat Vanessa Nasim Din, Ellen Lee, Matthew J Lee, Jonathan Lund, Christine Moffatt, Jonathan Morton, Asha Senapati, Philip Shackley, Peter Vaughan-Shaw, Arkadiusz Peter Wysocki, Tia Callaghan, Helen Jones, Nyantara Wickramasekera
<p><strong>Background: </strong>There is no consensus on optimal management of pilonidal disease. Surgical practice is varied, and existing literature is mainly single-centre cohort studies of varied disease severity, interventions and outcome assessments.</p><p><strong>Objectives: </strong>A prospective cohort study to determine: • disease severity and intervention relationship • most valued outcomes and treatment preference by patients • recommendations for policy and future research.</p><p><strong>Design: </strong>Observational cohort study with nested mixed-methods case study. Discrete choice experiment. Clinician survey. Three-stage Delphi survey for patients and clinicians. Inter-rater reliability of classification system.</p><p><strong>Setting: </strong>Thirty-one National Health Service trusts.</p><p><strong>Participants: </strong>Patients aged > 16 years referred for elective surgical treatment of pilonidal disease.</p><p><strong>Interventions: </strong>Surgery.</p><p><strong>Main outcome measures: </strong>Pain postoperative days 1 and 7, time to healing and return to normal activities, complications, recurrence. Outcomes compared between major and minor procedures using regression modelling, propensity score-based approaches and augmented inverse probability weighting to account for measured potential confounding features.</p><p><strong>Results: </strong><i>Clinician survey</i>: There was significant heterogeneity in surgeon practice preference. Limited training opportunities may impede efforts to improve practice. <i>Cohort study</i>: Over half of patients (60%; <i>N</i> = 667) had a major procedure. For these procedures, pain was greater on day 1 and day 7 (mean difference day 1 pain 1.58 points, 95% confidence interval 1.14 to 2.01 points, <i>n</i> = 536; mean difference day 7 pain 1.53 points, 95% confidence interval 1.12 to 1.95 points, <i>n</i> = 512). There were higher complication rates (adjusted risk difference 17.5%, 95% confidence interval 9.1 to 25.9%, <i>n</i> = 579), lower recurrence (adjusted risk difference -10.1%, 95% confidence interval -18.1 to -2.1%, <i>n</i> = 575), and longer time to healing (>34 days estimated difference) and time to return to normal activities (difference 25.9 days, 95% confidence interval 18.4 to 33.4 days). <i>Mixed-methods analysis</i>: Patient decision-making was influenced by prior experience of disease and anticipated recovery time. The burden involved in wound care and the gap between expected and actual time for recovery were the principal reasons given for decision regret. <i>Discrete choice experiment</i>: The strongest predictors of patient treatment choice were risk of infection/persistence (attribute importance 70%), and shorter recovery time (attribute importance 30%). Patients were willing to trade off these attributes. Those aged over 30 years had a higher risk tolerance (22.35-34.67%) for treatment failure if they could experience rapid recovery. There was no strong evidence tha
{"title":"Treatment options for patients with pilonidal sinus disease: PITSTOP, a mixed-methods evaluation.","authors":"Steven Brown, Daniel Hind, Emily Strong, Mike Bradburn, Farhat Vanessa Nasim Din, Ellen Lee, Matthew J Lee, Jonathan Lund, Christine Moffatt, Jonathan Morton, Asha Senapati, Philip Shackley, Peter Vaughan-Shaw, Arkadiusz Peter Wysocki, Tia Callaghan, Helen Jones, Nyantara Wickramasekera","doi":"10.3310/KFDQ2017","DOIUrl":"10.3310/KFDQ2017","url":null,"abstract":"<p><strong>Background: </strong>There is no consensus on optimal management of pilonidal disease. Surgical practice is varied, and existing literature is mainly single-centre cohort studies of varied disease severity, interventions and outcome assessments.</p><p><strong>Objectives: </strong>A prospective cohort study to determine: • disease severity and intervention relationship • most valued outcomes and treatment preference by patients • recommendations for policy and future research.</p><p><strong>Design: </strong>Observational cohort study with nested mixed-methods case study. Discrete choice experiment. Clinician survey. Three-stage Delphi survey for patients and clinicians. Inter-rater reliability of classification system.</p><p><strong>Setting: </strong>Thirty-one National Health Service trusts.</p><p><strong>Participants: </strong>Patients aged > 16 years referred for elective surgical treatment of pilonidal disease.</p><p><strong>Interventions: </strong>Surgery.</p><p><strong>Main outcome measures: </strong>Pain postoperative days 1 and 7, time to healing and return to normal activities, complications, recurrence. Outcomes compared between major and minor procedures using regression modelling, propensity score-based approaches and augmented inverse probability weighting to account for measured potential confounding features.</p><p><strong>Results: </strong><i>Clinician survey</i>: There was significant heterogeneity in surgeon practice preference. Limited training opportunities may impede efforts to improve practice. <i>Cohort study</i>: Over half of patients (60%; <i>N</i> = 667) had a major procedure. For these procedures, pain was greater on day 1 and day 7 (mean difference day 1 pain 1.58 points, 95% confidence interval 1.14 to 2.01 points, <i>n</i> = 536; mean difference day 7 pain 1.53 points, 95% confidence interval 1.12 to 1.95 points, <i>n</i> = 512). There were higher complication rates (adjusted risk difference 17.5%, 95% confidence interval 9.1 to 25.9%, <i>n</i> = 579), lower recurrence (adjusted risk difference -10.1%, 95% confidence interval -18.1 to -2.1%, <i>n</i> = 575), and longer time to healing (>34 days estimated difference) and time to return to normal activities (difference 25.9 days, 95% confidence interval 18.4 to 33.4 days). <i>Mixed-methods analysis</i>: Patient decision-making was influenced by prior experience of disease and anticipated recovery time. The burden involved in wound care and the gap between expected and actual time for recovery were the principal reasons given for decision regret. <i>Discrete choice experiment</i>: The strongest predictors of patient treatment choice were risk of infection/persistence (attribute importance 70%), and shorter recovery time (attribute importance 30%). Patients were willing to trade off these attributes. Those aged over 30 years had a higher risk tolerance (22.35-34.67%) for treatment failure if they could experience rapid recovery. There was no strong evidence tha","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 33","pages":"1-113"},"PeriodicalIF":3.5,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11284621/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141751569","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}
Ruth E Hogg, Robin Wickens, Sean O'Connor, Eleanor Gidman, Elizabeth Ward, Charlene Treanor, Tunde Peto, Ben Burton, Paul Knox, Andrew J Lotery, Sobha Sivaprasad, Michael Donnelly, Chris A Rogers, Barnaby C Reeves
<p><strong>Background: </strong>Most neovascular age-related macular degeneration treatments involve long-term follow-up of disease activity. Home monitoring would reduce the burden on patients and those they depend on for transport, and release clinic appointments for other patients. The study aimed to evaluate three home-monitoring tests for patients to use to detect active neovascular age-related macular degeneration compared with diagnosing active neovascular age-related macular degeneration by hospital follow-up.</p><p><strong>Objectives: </strong>There were five objectives: Estimate the accuracy of three home-monitoring tests to detect active neovascular age-related macular degeneration. Determine the acceptability of home monitoring to patients and carers and adherence to home monitoring. Explore whether inequalities exist in recruitment, participants' ability to self-test and their adherence to weekly testing during follow-up. Provide pilot data about the accuracy of home monitoring to detect conversion to neovascular age-related macular degeneration in fellow eyes of patients with unilateral neovascular age-related macular degeneration. Describe challenges experienced when implementing home-monitoring tests.</p><p><strong>Design: </strong>Diagnostic test accuracy cohort study, stratified by time since starting treatment.</p><p><strong>Setting: </strong>Six United Kingdom Hospital Eye Service macular clinics (Belfast, Liverpool, Moorfields, James Paget, Southampton, Gloucester).</p><p><strong>Participants: </strong>Patients with at least one study eye being monitored by hospital follow-up.</p><p><strong>Reference standard: </strong>Detection of active neovascular age-related macular degeneration by an ophthalmologist at hospital follow-up.</p><p><strong>Index tests: </strong>KeepSight Journal: paper-based near-vision tests presented as word puzzles. MyVisionTrack®: electronic test, viewed on a tablet device. MultiBit: electronic test, viewed on a tablet device. Participants provided test scores weekly. Raw scores between hospital follow-ups were summarised as averages.</p><p><strong>Results: </strong>Two hundred and ninety-seven patients (mean age 74.9 years) took part. At least one hospital follow-up was available for 317 study eyes, including 9 second eyes that became eligible during follow-up, in 261 participants (1549 complete visits). Median testing frequency was three times/month. Estimated areas under receiver operating curves were < 0.6 for all index tests, and only KeepSight Journal summary score was significantly associated with the lesion activity (odds ratio = 3.48, 95% confidence interval 1.09 to 11.13, <i>p</i> = 0.036). Older age and worse deprivation for home address were associated with lower participation (χ<sup>2</sup> = 50.5 and 24.3, respectively, <i>p</i> < 0.001) but not ability or adherence to self-testing. Areas under receiver operating curves appeared higher for conversion of fellow eyes to neovascular age-relate
{"title":"Home-monitoring for neovascular age-related macular degeneration in older adults within the UK: the MONARCH diagnostic accuracy study.","authors":"Ruth E Hogg, Robin Wickens, Sean O'Connor, Eleanor Gidman, Elizabeth Ward, Charlene Treanor, Tunde Peto, Ben Burton, Paul Knox, Andrew J Lotery, Sobha Sivaprasad, Michael Donnelly, Chris A Rogers, Barnaby C Reeves","doi":"10.3310/CYRA9912","DOIUrl":"10.3310/CYRA9912","url":null,"abstract":"<p><strong>Background: </strong>Most neovascular age-related macular degeneration treatments involve long-term follow-up of disease activity. Home monitoring would reduce the burden on patients and those they depend on for transport, and release clinic appointments for other patients. The study aimed to evaluate three home-monitoring tests for patients to use to detect active neovascular age-related macular degeneration compared with diagnosing active neovascular age-related macular degeneration by hospital follow-up.</p><p><strong>Objectives: </strong>There were five objectives: Estimate the accuracy of three home-monitoring tests to detect active neovascular age-related macular degeneration. Determine the acceptability of home monitoring to patients and carers and adherence to home monitoring. Explore whether inequalities exist in recruitment, participants' ability to self-test and their adherence to weekly testing during follow-up. Provide pilot data about the accuracy of home monitoring to detect conversion to neovascular age-related macular degeneration in fellow eyes of patients with unilateral neovascular age-related macular degeneration. Describe challenges experienced when implementing home-monitoring tests.</p><p><strong>Design: </strong>Diagnostic test accuracy cohort study, stratified by time since starting treatment.</p><p><strong>Setting: </strong>Six United Kingdom Hospital Eye Service macular clinics (Belfast, Liverpool, Moorfields, James Paget, Southampton, Gloucester).</p><p><strong>Participants: </strong>Patients with at least one study eye being monitored by hospital follow-up.</p><p><strong>Reference standard: </strong>Detection of active neovascular age-related macular degeneration by an ophthalmologist at hospital follow-up.</p><p><strong>Index tests: </strong>KeepSight Journal: paper-based near-vision tests presented as word puzzles. MyVisionTrack®: electronic test, viewed on a tablet device. MultiBit: electronic test, viewed on a tablet device. Participants provided test scores weekly. Raw scores between hospital follow-ups were summarised as averages.</p><p><strong>Results: </strong>Two hundred and ninety-seven patients (mean age 74.9 years) took part. At least one hospital follow-up was available for 317 study eyes, including 9 second eyes that became eligible during follow-up, in 261 participants (1549 complete visits). Median testing frequency was three times/month. Estimated areas under receiver operating curves were < 0.6 for all index tests, and only KeepSight Journal summary score was significantly associated with the lesion activity (odds ratio = 3.48, 95% confidence interval 1.09 to 11.13, <i>p</i> = 0.036). Older age and worse deprivation for home address were associated with lower participation (χ<sup>2</sup> = 50.5 and 24.3, respectively, <i>p</i> < 0.001) but not ability or adherence to self-testing. Areas under receiver operating curves appeared higher for conversion of fellow eyes to neovascular age-relate","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 32","pages":"1-136"},"PeriodicalIF":3.5,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11261425/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141633283","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 diseases are the leading cause of death globally. The aim of this overview of systematic reviews was to compare the effectiveness of different pharmacological and non-pharmacological interventions for the primary prevention of cardiovascular disease.</p><p><strong>Methods: </strong>A structured search of the Cochrane Database of Systematic Reviews, MEDLINE, EMBASE and the Database of Abstracts of Reviews of Effects archive was conducted to find systematic reviews that reported the effect of various pharmacological and non-pharmacological interventions for the primary prevention of cardiovascular disease from inception to March 2021. References of included studies were also checked. The included systematic reviews' methodological quality was assessed using the Assessment of Multiple Systematic Reviews 2 instrument (range, 0-16). The outcomes of each included review's meta-analysis were extracted and described narratively.</p><p><strong>Results: </strong>This study analysed 95 systematic reviews, including 41 on non-pharmacological interventions and 54 on pharmacological interventions for cardiovascular health. The majority of the reviews focused on lipid-lowering interventions (<i>n</i> = 25) and antiplatelet medications (<i>n</i> = 21), followed by nutritional supplements, dietary interventions, physical activity, health promotion and other interventions. Only 1 of the 10 reviews addressing cardiovascular mortality showed a potential benefit, while the others found no effect. Antiplatelets were found to have a beneficial effect on all-cause mortality in 2 out of 12 meta-analyses and on major cardiovascular disease events in 8 out of 17 reviews. Lipid-lowering interventions showed beneficial effects on cardiovascular disease mortality, all-cause mortality and major cardiovascular disease events in varying numbers of the reviews. Glucose-lowering medications demonstrated significant benefits for major cardiovascular events, coronary heart disease events and mortality. However, the combination of dietary interventions, physical activities, nutritional supplements and polypills showed little or no significant benefit for major cardiovascular outcomes or mortality.</p><p><strong>Future work and limitations: </strong>More research is needed to determine whether the effect of treatment varies depending on population characteristics. The findings of this review should be interpreted with caution because the majority of studies of non-pharmacological interventions compare primary prevention with usual care, which may include recommended pharmacological treatment in higher-risk patients (e.g. statins and/or antihypertensive medications, etc.). In addition, randomised controlled trial evidence may be better suited to the study of pharmacological interventions than dietary and lifestyle interventions.</p><p><strong>Conclusions: </strong>This umbrella review captured the variability in different interventions on
{"title":"Interventions for primary prevention of cardiovascular disease: umbrella review of systematic reviews.","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/GJTR5006","DOIUrl":"https://doi.org/10.3310/GJTR5006","url":null,"abstract":"<p><strong>Background: </strong>Cardiovascular diseases are the leading cause of death globally. The aim of this overview of systematic reviews was to compare the effectiveness of different pharmacological and non-pharmacological interventions for the primary prevention of cardiovascular disease.</p><p><strong>Methods: </strong>A structured search of the Cochrane Database of Systematic Reviews, MEDLINE, EMBASE and the Database of Abstracts of Reviews of Effects archive was conducted to find systematic reviews that reported the effect of various pharmacological and non-pharmacological interventions for the primary prevention of cardiovascular disease from inception to March 2021. References of included studies were also checked. The included systematic reviews' methodological quality was assessed using the Assessment of Multiple Systematic Reviews 2 instrument (range, 0-16). The outcomes of each included review's meta-analysis were extracted and described narratively.</p><p><strong>Results: </strong>This study analysed 95 systematic reviews, including 41 on non-pharmacological interventions and 54 on pharmacological interventions for cardiovascular health. The majority of the reviews focused on lipid-lowering interventions (<i>n</i> = 25) and antiplatelet medications (<i>n</i> = 21), followed by nutritional supplements, dietary interventions, physical activity, health promotion and other interventions. Only 1 of the 10 reviews addressing cardiovascular mortality showed a potential benefit, while the others found no effect. Antiplatelets were found to have a beneficial effect on all-cause mortality in 2 out of 12 meta-analyses and on major cardiovascular disease events in 8 out of 17 reviews. Lipid-lowering interventions showed beneficial effects on cardiovascular disease mortality, all-cause mortality and major cardiovascular disease events in varying numbers of the reviews. Glucose-lowering medications demonstrated significant benefits for major cardiovascular events, coronary heart disease events and mortality. However, the combination of dietary interventions, physical activities, nutritional supplements and polypills showed little or no significant benefit for major cardiovascular outcomes or mortality.</p><p><strong>Future work and limitations: </strong>More research is needed to determine whether the effect of treatment varies depending on population characteristics. The findings of this review should be interpreted with caution because the majority of studies of non-pharmacological interventions compare primary prevention with usual care, which may include recommended pharmacological treatment in higher-risk patients (e.g. statins and/or antihypertensive medications, etc.). In addition, randomised controlled trial evidence may be better suited to the study of pharmacological interventions than dietary and lifestyle interventions.</p><p><strong>Conclusions: </strong>This umbrella review captured the variability in different interventions on ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-26"},"PeriodicalIF":3.5,"publicationDate":"2024-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141544768","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Daniel Lasserson, Pooja Gaddu, Samir Mehta, Agnieszka Ignatowicz, Sheila Greenfield, Clare Prince, Carole Cummins, Graham Robinson, Jonathan Rodrigues, Simon Noble, Sue Jowett, Mark Toshner, Michael Newnham, Alice Turner
<p><strong>Research question: </strong>Is withholding anticoagulation for patients with isolated or incidental subsegmental pulmonary embolism clinically and cost-effective compared with full anticoagulation for 3 months?</p><p><strong>Background: </strong>There has been an increase in the diagnosis of subsegmental pulmonary embolism since the advent of computed tomography pulmonary angiogram to investigate patients with suspected pulmonary embolism. Subsegmental pulmonary embolism is not often detectable with older nuclear medicine-based diagnostic imaging for ventilation/perfusion mismatch. The case fatality of pulmonary embolism has reduced as subsegmental pulmonary embolism diagnoses from computed tomography pulmonary angiogram have increased. There is growing equipoise about the optimal treatment for patients with subsegmental pulmonary embolism, given that full anticoagulation has significant risks of bleeding and subsegmental pulmonary embolism was not often diagnosed previously with ventilation/perfusion scanning and therefore most likely left predominantly untreated prior to the introduction of computed tomography pulmonary angiogram scanning.</p><p><strong>Objectives: </strong>Determine whether withholding anticoagulation for isolated or incidental subsegmental pulmonary embolism (i.e. subsegmental pulmonary embolism with no coexisting deep-vein thrombosis) reduces the harms of recurrent thromboembolism and major bleeding compared with 3 months of full anticoagulation at 3, 6 and 12 months. Determine the rate of complications of anticoagulation therapy (predominantly bleeding) in patients with isolated subsegmental pulmonary embolism. Determine whether not treating isolated subsegmental pulmonary embolism is acceptable to clinicians and patients. Determine the reclassification rate of subsegmental pulmonary embolism diagnoses made by general reporting radiologists when reviewed by specialist respiratory radiologists and develop a set of rules to improve general radiologists' diagnoses of subsegmental pulmonary embolism. Assess cost-effectiveness of not treating patients with isolated subsegmental pulmonary embolism with anticoagulation, taking a health service perspective.</p><p><strong>Methods: </strong>Prospective individually randomised open controlled trial with blinded end-point committee assessment for outcomes, powered for non-inferiority for recurrent venous thromboembolism and for superiority for bleeding events. An internal pilot phase is included for feasibility and acceptability of no anticoagulation. We planned to recruit 1466 patients from at least 50 acute hospital sites. Allowing for a dropout rate of 15%, this would have given us 90% power to detect a reduction in major and clinically relevant non-major bleeding from 7.3% in the anticoagulation arm to 3% in the intervention arm. We were powered to determine that a strategy of no anticoagulation was non-inferior to anticoagulation with an upper margin of a 2.3% increase
{"title":"Stopping anticoagulation for isolated or incidental pulmonary embolism: the STOPAPE RCT protocol.","authors":"Daniel Lasserson, Pooja Gaddu, Samir Mehta, Agnieszka Ignatowicz, Sheila Greenfield, Clare Prince, Carole Cummins, Graham Robinson, Jonathan Rodrigues, Simon Noble, Sue Jowett, Mark Toshner, Michael Newnham, Alice Turner","doi":"10.3310/HRCW7937","DOIUrl":"https://doi.org/10.3310/HRCW7937","url":null,"abstract":"<p><strong>Research question: </strong>Is withholding anticoagulation for patients with isolated or incidental subsegmental pulmonary embolism clinically and cost-effective compared with full anticoagulation for 3 months?</p><p><strong>Background: </strong>There has been an increase in the diagnosis of subsegmental pulmonary embolism since the advent of computed tomography pulmonary angiogram to investigate patients with suspected pulmonary embolism. Subsegmental pulmonary embolism is not often detectable with older nuclear medicine-based diagnostic imaging for ventilation/perfusion mismatch. The case fatality of pulmonary embolism has reduced as subsegmental pulmonary embolism diagnoses from computed tomography pulmonary angiogram have increased. There is growing equipoise about the optimal treatment for patients with subsegmental pulmonary embolism, given that full anticoagulation has significant risks of bleeding and subsegmental pulmonary embolism was not often diagnosed previously with ventilation/perfusion scanning and therefore most likely left predominantly untreated prior to the introduction of computed tomography pulmonary angiogram scanning.</p><p><strong>Objectives: </strong>Determine whether withholding anticoagulation for isolated or incidental subsegmental pulmonary embolism (i.e. subsegmental pulmonary embolism with no coexisting deep-vein thrombosis) reduces the harms of recurrent thromboembolism and major bleeding compared with 3 months of full anticoagulation at 3, 6 and 12 months. Determine the rate of complications of anticoagulation therapy (predominantly bleeding) in patients with isolated subsegmental pulmonary embolism. Determine whether not treating isolated subsegmental pulmonary embolism is acceptable to clinicians and patients. Determine the reclassification rate of subsegmental pulmonary embolism diagnoses made by general reporting radiologists when reviewed by specialist respiratory radiologists and develop a set of rules to improve general radiologists' diagnoses of subsegmental pulmonary embolism. Assess cost-effectiveness of not treating patients with isolated subsegmental pulmonary embolism with anticoagulation, taking a health service perspective.</p><p><strong>Methods: </strong>Prospective individually randomised open controlled trial with blinded end-point committee assessment for outcomes, powered for non-inferiority for recurrent venous thromboembolism and for superiority for bleeding events. An internal pilot phase is included for feasibility and acceptability of no anticoagulation. We planned to recruit 1466 patients from at least 50 acute hospital sites. Allowing for a dropout rate of 15%, this would have given us 90% power to detect a reduction in major and clinically relevant non-major bleeding from 7.3% in the anticoagulation arm to 3% in the intervention arm. We were powered to determine that a strategy of no anticoagulation was non-inferior to anticoagulation with an upper margin of a 2.3% increase ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-17"},"PeriodicalIF":3.5,"publicationDate":"2024-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141544769","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Karen Innes, Irfan Ahmed, Jemma Hudson, Rodolfo Hernández, Katie Gillies, Rebecca Bruce, Victoria Bell, Alison Avenell, Jane Blazeby, Miriam Brazzelli, Seonaidh Cotton, Bernard Croal, Mark Forrest, Graeme MacLennan, Peter Murchie, Samantha Wileman, Craig Ramsay
<p><strong>Background: </strong>Gallstone disease is a common gastrointestinal disorder in industrialised societies. The prevalence of gallstones in the adult population is estimated to be approximately 10-15%, and around 80% remain asymptomatic. At present, cholecystectomy is the default option for people with symptomatic gallstone disease.</p><p><strong>Objectives: </strong>To assess the clinical and cost-effectiveness of observation/conservative management compared with laparoscopic cholecystectomy for preventing recurrent symptoms and complications in adults presenting with uncomplicated symptomatic gallstones in secondary care.</p><p><strong>Design: </strong>Parallel group, multicentre patient randomised superiority pragmatic trial with up to 24 months follow-up and embedded qualitative research. Within-trial cost-utility and 10-year Markov model analyses. Development of a core outcome set for uncomplicated symptomatic gallstone disease.</p><p><strong>Setting: </strong>Secondary care elective settings.</p><p><strong>Participants: </strong>Adults with symptomatic uncomplicated gallstone disease referred to a secondary care setting were considered for inclusion.</p><p><strong>Interventions: </strong>Participants were randomised 1: 1 at clinic to receive either laparoscopic cholecystectomy or observation/conservative management.</p><p><strong>Main outcome measures: </strong>The primary outcome was quality of life measured by area under the curve over 18 months using the Short Form-36 bodily pain domain. Secondary outcomes included the Otago gallstones' condition-specific questionnaire, Short Form-36 domains (excluding bodily pain), area under the curve over 24 months for Short Form-36 bodily pain domain, persistent symptoms, complications and need for further treatment. No outcomes were blinded to allocation.</p><p><strong>Results: </strong>Between August 2016 and November 2019, 434 participants were randomised (217 in each group) from 20 United Kingdom centres. By 24 months, 64 (29.5%) in the observation/conservative management group and 153 (70.5%) in the laparoscopic cholecystectomy group had received surgery, median time to surgery of 9.0 months (interquartile range, 5.6-15.0) and 4.7 months (interquartile range 2.6-7.9), respectively. At 18 months, the mean Short Form-36 norm-based bodily pain score was 49.4 (standard deviation 11.7) in the observation/conservative management group and 50.4 (standard deviation 11.6) in the laparoscopic cholecystectomy group. The mean area under the curve over 18 months was 46.8 for both groups with no difference: mean difference -0.0, 95% confidence interval (-1.7 to 1.7); <i>p</i>-value 0.996; <i>n</i> = 203 observation/conservative, <i>n</i> = 205 cholecystectomy. There was no evidence of differences in quality of life, complications or need for further treatment at up to 24 months follow-up. Condition-specific quality of life at 24 months favoured cholecystectomy: mean difference 9.0, 95% confidence int
{"title":"Laparoscopic cholecystectomy versus conservative management for adults with uncomplicated symptomatic gallstones: the C-GALL RCT.","authors":"Karen Innes, Irfan Ahmed, Jemma Hudson, Rodolfo Hernández, Katie Gillies, Rebecca Bruce, Victoria Bell, Alison Avenell, Jane Blazeby, Miriam Brazzelli, Seonaidh Cotton, Bernard Croal, Mark Forrest, Graeme MacLennan, Peter Murchie, Samantha Wileman, Craig Ramsay","doi":"10.3310/MNBY3104","DOIUrl":"10.3310/MNBY3104","url":null,"abstract":"<p><strong>Background: </strong>Gallstone disease is a common gastrointestinal disorder in industrialised societies. The prevalence of gallstones in the adult population is estimated to be approximately 10-15%, and around 80% remain asymptomatic. At present, cholecystectomy is the default option for people with symptomatic gallstone disease.</p><p><strong>Objectives: </strong>To assess the clinical and cost-effectiveness of observation/conservative management compared with laparoscopic cholecystectomy for preventing recurrent symptoms and complications in adults presenting with uncomplicated symptomatic gallstones in secondary care.</p><p><strong>Design: </strong>Parallel group, multicentre patient randomised superiority pragmatic trial with up to 24 months follow-up and embedded qualitative research. Within-trial cost-utility and 10-year Markov model analyses. Development of a core outcome set for uncomplicated symptomatic gallstone disease.</p><p><strong>Setting: </strong>Secondary care elective settings.</p><p><strong>Participants: </strong>Adults with symptomatic uncomplicated gallstone disease referred to a secondary care setting were considered for inclusion.</p><p><strong>Interventions: </strong>Participants were randomised 1: 1 at clinic to receive either laparoscopic cholecystectomy or observation/conservative management.</p><p><strong>Main outcome measures: </strong>The primary outcome was quality of life measured by area under the curve over 18 months using the Short Form-36 bodily pain domain. Secondary outcomes included the Otago gallstones' condition-specific questionnaire, Short Form-36 domains (excluding bodily pain), area under the curve over 24 months for Short Form-36 bodily pain domain, persistent symptoms, complications and need for further treatment. No outcomes were blinded to allocation.</p><p><strong>Results: </strong>Between August 2016 and November 2019, 434 participants were randomised (217 in each group) from 20 United Kingdom centres. By 24 months, 64 (29.5%) in the observation/conservative management group and 153 (70.5%) in the laparoscopic cholecystectomy group had received surgery, median time to surgery of 9.0 months (interquartile range, 5.6-15.0) and 4.7 months (interquartile range 2.6-7.9), respectively. At 18 months, the mean Short Form-36 norm-based bodily pain score was 49.4 (standard deviation 11.7) in the observation/conservative management group and 50.4 (standard deviation 11.6) in the laparoscopic cholecystectomy group. The mean area under the curve over 18 months was 46.8 for both groups with no difference: mean difference -0.0, 95% confidence interval (-1.7 to 1.7); <i>p</i>-value 0.996; <i>n</i> = 203 observation/conservative, <i>n</i> = 205 cholecystectomy. There was no evidence of differences in quality of life, complications or need for further treatment at up to 24 months follow-up. Condition-specific quality of life at 24 months favoured cholecystectomy: mean difference 9.0, 95% confidence int","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 26","pages":"1-151"},"PeriodicalIF":3.5,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11228691/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141467521","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}
Beth Woods, Laetitia Schmitt, Dina Jankovic, Benjamin Kearns, Alison Scope, Shijie Ren, Tushar Srivastava, Chu Chang Ku, Jean Hamilton, Claire Rothery, Laura Bojke, Mark Sculpher, Sue Harnan
<p><strong>Background: </strong>To limit the use of antimicrobials without disincentivising the development of novel antimicrobials, there is interest in establishing innovative models that fund antimicrobials based on an evaluation of their value as opposed to the volumes used. The aim of this project was to evaluate the population-level health benefit of cefiderocol in the NHS in England, for the treatment of severe aerobic Gram-negative bacterial infections when used within its licensed indications. The results were used to inform the National Institute for Health and Care Excellence guidance in support of commercial discussions regarding contract value between the manufacturer and NHS England.</p><p><strong>Methods: </strong>The health benefit of cefiderocol was first derived for a series of high-value clinical scenarios. These represented uses that were expected to have a significant impact on patients' mortality risks and health-related quality of life. The clinical effectiveness of cefiderocol relative to its comparators was estimated by synthesising evidence on susceptibility of the pathogens of interest to the antimicrobials in a network meta-analysis. Patient-level costs and health outcomes of cefiderocol under various usage scenarios compared with alternative management strategies were quantified using decision modelling. Results were reported as incremental net health effects expressed in quality-adjusted life-years, which were scaled to 20-year population values using infection number forecasts based on data from Public Health England. The outcomes estimated for the high-value clinical scenarios were extrapolated to other expected uses for cefiderocol.</p><p><strong>Results: </strong>Among <i>Enterobacterales</i> isolates with the metallo-beta-lactamase resistance mechanism, the base-case network meta-analysis found that cefiderocol was associated with a lower susceptibility relative to colistin (odds ratio 0.32, 95% credible intervals 0.04 to 2.47), but the result was not statistically significant. The other treatments were also associated with lower susceptibility than colistin, but the results were not statistically significant. In the metallo-beta-lactamase <i>Pseudomonas aeruginosa</i> base-case network meta-analysis, cefiderocol was associated with a lower susceptibility relative to colistin (odds ratio 0.44, 95% credible intervals 0.03 to 3.94), but the result was not statistically significant. The other treatments were associated with no susceptibility. In the base case, patient-level benefit of cefiderocol was between 0.02 and 0.15 quality-adjusted life-years, depending on the site of infection, the pathogen and the usage scenario. There was a high degree of uncertainty surrounding the benefits of cefiderocol across all subgroups. There was substantial uncertainty in the number of infections that are suitable for treatment with cefiderocol, so population-level results are presented for a range of scenarios for the current in
{"title":"Cefiderocol for treating severe aerobic Gram-negative bacterial infections: technology evaluation to inform a novel subscription-style payment model.","authors":"Beth Woods, Laetitia Schmitt, Dina Jankovic, Benjamin Kearns, Alison Scope, Shijie Ren, Tushar Srivastava, Chu Chang Ku, Jean Hamilton, Claire Rothery, Laura Bojke, Mark Sculpher, Sue Harnan","doi":"10.3310/YGWR4511","DOIUrl":"10.3310/YGWR4511","url":null,"abstract":"<p><strong>Background: </strong>To limit the use of antimicrobials without disincentivising the development of novel antimicrobials, there is interest in establishing innovative models that fund antimicrobials based on an evaluation of their value as opposed to the volumes used. The aim of this project was to evaluate the population-level health benefit of cefiderocol in the NHS in England, for the treatment of severe aerobic Gram-negative bacterial infections when used within its licensed indications. The results were used to inform the National Institute for Health and Care Excellence guidance in support of commercial discussions regarding contract value between the manufacturer and NHS England.</p><p><strong>Methods: </strong>The health benefit of cefiderocol was first derived for a series of high-value clinical scenarios. These represented uses that were expected to have a significant impact on patients' mortality risks and health-related quality of life. The clinical effectiveness of cefiderocol relative to its comparators was estimated by synthesising evidence on susceptibility of the pathogens of interest to the antimicrobials in a network meta-analysis. Patient-level costs and health outcomes of cefiderocol under various usage scenarios compared with alternative management strategies were quantified using decision modelling. Results were reported as incremental net health effects expressed in quality-adjusted life-years, which were scaled to 20-year population values using infection number forecasts based on data from Public Health England. The outcomes estimated for the high-value clinical scenarios were extrapolated to other expected uses for cefiderocol.</p><p><strong>Results: </strong>Among <i>Enterobacterales</i> isolates with the metallo-beta-lactamase resistance mechanism, the base-case network meta-analysis found that cefiderocol was associated with a lower susceptibility relative to colistin (odds ratio 0.32, 95% credible intervals 0.04 to 2.47), but the result was not statistically significant. The other treatments were also associated with lower susceptibility than colistin, but the results were not statistically significant. In the metallo-beta-lactamase <i>Pseudomonas aeruginosa</i> base-case network meta-analysis, cefiderocol was associated with a lower susceptibility relative to colistin (odds ratio 0.44, 95% credible intervals 0.03 to 3.94), but the result was not statistically significant. The other treatments were associated with no susceptibility. In the base case, patient-level benefit of cefiderocol was between 0.02 and 0.15 quality-adjusted life-years, depending on the site of infection, the pathogen and the usage scenario. There was a high degree of uncertainty surrounding the benefits of cefiderocol across all subgroups. There was substantial uncertainty in the number of infections that are suitable for treatment with cefiderocol, so population-level results are presented for a range of scenarios for the current in","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 28","pages":"1-238"},"PeriodicalIF":3.5,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11229178/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141467523","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 Rivero-Arias, May Ee Png, Ashley White, Miaoqing Yang, Sian Taylor-Phillips, Lisa Hinton, Felicity Boardman, Abigail McNiven, Jane Fisher, Baskaran Thilaganathan, Sam Oddie, Anne-Marie Slowther, Svetlana Ratushnyak, Nia Roberts, Jenny Shilton Osborne, Stavros Petrou
<p><strong>Background: </strong>Health economic assessments are used to determine whether the resources needed to generate net benefit from an antenatal or newborn screening programme, driven by multiple benefits and harms, are justifiable. It is not known what benefits and harms have been adopted by economic evaluations assessing these programmes and whether they omit benefits and harms considered important to relevant stakeholders.</p><p><strong>Objectives: </strong>(1) To identify the benefits and harms adopted by health economic assessments in this area, and to assess how they have been measured and valued; (2) to identify attributes or relevance to stakeholders that ought to be considered in future economic assessments; and (3) to make recommendations about the benefits and harms that should be considered by these studies.</p><p><strong>Design: </strong>Mixed methods combining systematic review and qualitative work.</p><p><strong>Systematic review methods: </strong>We searched the published and grey literature from January 2000 to January 2021 using all major electronic databases. Economic evaluations of an antenatal or newborn screening programme in one or more Organisation for Economic Co-operation and Development countries were considered eligible. Reporting quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards checklist. We identified benefits and harms using an integrative descriptive analysis and constructed a thematic framework.</p><p><strong>Qualitative methods: </strong>We conducted a meta-ethnography of the existing literature on newborn screening experiences, a secondary analysis of existing individual interviews related to antenatal or newborn screening or living with screened-for conditions, and a thematic analysis of primary data collected with stakeholders about their experiences with screening.</p><p><strong>Results: </strong>The literature searches identified 52,244 articles and reports, and 336 unique studies were included. Thematic framework resulted in seven themes: (1) diagnosis of screened for condition, (2) life-years and health status adjustments, (3) treatment, (4) long-term costs, (5) overdiagnosis, (6) pregnancy loss and (7) spillover effects on family members. Diagnosis of screened-for condition (115, 47.5%), life-years and health status adjustments (90, 37.2%) and treatment (88, 36.4%) accounted for most of the benefits and harms evaluating antenatal screening. The same themes accounted for most of the benefits and harms included in studies assessing newborn screening. Long-term costs, overdiagnosis and spillover effects tended to be ignored. The wide-reaching family implications of screening were considered important to stakeholders. We observed good overlap between the thematic framework and the qualitative evidence.</p><p><strong>Limitations: </strong>Dual data extraction within the systematic literature review was not feasible due to the large number of studies included. I
{"title":"Benefits and harms of antenatal and newborn screening programmes in health economic assessments: the VALENTIA systematic review and qualitative investigation.","authors":"Oliver Rivero-Arias, May Ee Png, Ashley White, Miaoqing Yang, Sian Taylor-Phillips, Lisa Hinton, Felicity Boardman, Abigail McNiven, Jane Fisher, Baskaran Thilaganathan, Sam Oddie, Anne-Marie Slowther, Svetlana Ratushnyak, Nia Roberts, Jenny Shilton Osborne, Stavros Petrou","doi":"10.3310/PYTK6591","DOIUrl":"10.3310/PYTK6591","url":null,"abstract":"<p><strong>Background: </strong>Health economic assessments are used to determine whether the resources needed to generate net benefit from an antenatal or newborn screening programme, driven by multiple benefits and harms, are justifiable. It is not known what benefits and harms have been adopted by economic evaluations assessing these programmes and whether they omit benefits and harms considered important to relevant stakeholders.</p><p><strong>Objectives: </strong>(1) To identify the benefits and harms adopted by health economic assessments in this area, and to assess how they have been measured and valued; (2) to identify attributes or relevance to stakeholders that ought to be considered in future economic assessments; and (3) to make recommendations about the benefits and harms that should be considered by these studies.</p><p><strong>Design: </strong>Mixed methods combining systematic review and qualitative work.</p><p><strong>Systematic review methods: </strong>We searched the published and grey literature from January 2000 to January 2021 using all major electronic databases. Economic evaluations of an antenatal or newborn screening programme in one or more Organisation for Economic Co-operation and Development countries were considered eligible. Reporting quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards checklist. We identified benefits and harms using an integrative descriptive analysis and constructed a thematic framework.</p><p><strong>Qualitative methods: </strong>We conducted a meta-ethnography of the existing literature on newborn screening experiences, a secondary analysis of existing individual interviews related to antenatal or newborn screening or living with screened-for conditions, and a thematic analysis of primary data collected with stakeholders about their experiences with screening.</p><p><strong>Results: </strong>The literature searches identified 52,244 articles and reports, and 336 unique studies were included. Thematic framework resulted in seven themes: (1) diagnosis of screened for condition, (2) life-years and health status adjustments, (3) treatment, (4) long-term costs, (5) overdiagnosis, (6) pregnancy loss and (7) spillover effects on family members. Diagnosis of screened-for condition (115, 47.5%), life-years and health status adjustments (90, 37.2%) and treatment (88, 36.4%) accounted for most of the benefits and harms evaluating antenatal screening. The same themes accounted for most of the benefits and harms included in studies assessing newborn screening. Long-term costs, overdiagnosis and spillover effects tended to be ignored. The wide-reaching family implications of screening were considered important to stakeholders. We observed good overlap between the thematic framework and the qualitative evidence.</p><p><strong>Limitations: </strong>Dual data extraction within the systematic literature review was not feasible due to the large number of studies included. I","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 25","pages":"1-180"},"PeriodicalIF":3.5,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11228689/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141467520","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}
David J Beard, Loretta Davies, Jonathan A Cook, Jamie Stokes, Jose Leal, Heidi Fletcher, Simon Abram, Katie Chegwin, Akiko Greshon, William Jackson, Nicholas Bottomley, Matthew Dodd, Henry Bourke, Beverly A Shirkey, Arsenio Paez, Sarah E Lamb, Karen L Barker, Michael Phillips, Mark Brown, Vanessa Lythe, Burhan Mirza, Andrew Carr, Paul Monk, Carlos Morgado Areia, Sean O'Leary, Fares Haddad, Chris Wilson, Andrew Price
<p><strong>Background: </strong>Anterior cruciate ligament injury of the knee is common and leads to decreased activity and risk of secondary osteoarthritis of the knee. Management of patients with a non-acute anterior cruciate ligament injury can be non-surgical (rehabilitation) or surgical (reconstruction). However, insufficient evidence exists to guide treatment.</p><p><strong>Objective(s): </strong>To determine in patients with non-acute anterior cruciate ligament injury and symptoms of instability whether a strategy of surgical management (reconstruction) without prior rehabilitation was more clinically and cost-effective than non-surgical management (rehabilitation).</p><p><strong>Design: </strong>A pragmatic, multicentre, superiority, randomised controlled trial with two-arm parallel groups and 1:1 allocation. Due to the nature of the interventions, no blinding could be carried out.</p><p><strong>Setting: </strong>Twenty-nine NHS orthopaedic units in the United Kingdom.</p><p><strong>Participants: </strong>Participants with a symptomatic (instability) non-acute anterior cruciate ligament-injured knee.</p><p><strong>Interventions: </strong>Patients in the surgical management arm underwent surgical anterior cruciate ligament reconstruction as soon as possible and without any further rehabilitation. Patients in the rehabilitation arm attended physiotherapy sessions and only were listed for reconstructive surgery on continued instability following rehabilitation. Surgery following initial rehabilitation was an expected outcome for many patients and within protocol.</p><p><strong>Main outcome measures: </strong>The primary outcome was the Knee Injury and Osteoarthritis Outcome Score 4 at 18 months post randomisation. Secondary outcomes included return to sport/activity, intervention-related complications, patient satisfaction, expectations of activity, generic health quality of life, knee-specific quality of life and resource usage.</p><p><strong>Results: </strong>Three hundred and sixteen participants were recruited between February 2017 and April 2020 with 156 randomised to surgical management and 160 to rehabilitation. Forty-one per cent (<i>n</i> = 65) of those allocated to rehabilitation underwent subsequent reconstruction within 18 months with 38% (<i>n</i> = 61) completing rehabilitation and not undergoing surgery. Seventy-two per cent (<i>n</i> = 113) of those allocated to surgery underwent reconstruction within 18 months. Follow-up at the primary outcome time point was 78% (<i>n</i> = 248; surgical, <i>n</i> = 128; rehabilitation, <i>n</i> = 120). Both groups improved over time. Adjusted mean Knee Injury and Osteoarthritis Outcome Score 4 scores at 18 months had increased to 73.0 in the surgical arm and to 64.6 in the rehabilitation arm. The adjusted mean difference was 7.9 (95% confidence interval 2.5 to 13.2; <i>p</i> = 0.005) in favour of surgical management. The per-protocol analyses supported the intention-to-treat results, with a
{"title":"Comparison of surgical or non-surgical management for non-acute anterior cruciate ligament injury: the ACL SNNAP RCT.","authors":"David J Beard, Loretta Davies, Jonathan A Cook, Jamie Stokes, Jose Leal, Heidi Fletcher, Simon Abram, Katie Chegwin, Akiko Greshon, William Jackson, Nicholas Bottomley, Matthew Dodd, Henry Bourke, Beverly A Shirkey, Arsenio Paez, Sarah E Lamb, Karen L Barker, Michael Phillips, Mark Brown, Vanessa Lythe, Burhan Mirza, Andrew Carr, Paul Monk, Carlos Morgado Areia, Sean O'Leary, Fares Haddad, Chris Wilson, Andrew Price","doi":"10.3310/VDKB6009","DOIUrl":"10.3310/VDKB6009","url":null,"abstract":"<p><strong>Background: </strong>Anterior cruciate ligament injury of the knee is common and leads to decreased activity and risk of secondary osteoarthritis of the knee. Management of patients with a non-acute anterior cruciate ligament injury can be non-surgical (rehabilitation) or surgical (reconstruction). However, insufficient evidence exists to guide treatment.</p><p><strong>Objective(s): </strong>To determine in patients with non-acute anterior cruciate ligament injury and symptoms of instability whether a strategy of surgical management (reconstruction) without prior rehabilitation was more clinically and cost-effective than non-surgical management (rehabilitation).</p><p><strong>Design: </strong>A pragmatic, multicentre, superiority, randomised controlled trial with two-arm parallel groups and 1:1 allocation. Due to the nature of the interventions, no blinding could be carried out.</p><p><strong>Setting: </strong>Twenty-nine NHS orthopaedic units in the United Kingdom.</p><p><strong>Participants: </strong>Participants with a symptomatic (instability) non-acute anterior cruciate ligament-injured knee.</p><p><strong>Interventions: </strong>Patients in the surgical management arm underwent surgical anterior cruciate ligament reconstruction as soon as possible and without any further rehabilitation. Patients in the rehabilitation arm attended physiotherapy sessions and only were listed for reconstructive surgery on continued instability following rehabilitation. Surgery following initial rehabilitation was an expected outcome for many patients and within protocol.</p><p><strong>Main outcome measures: </strong>The primary outcome was the Knee Injury and Osteoarthritis Outcome Score 4 at 18 months post randomisation. Secondary outcomes included return to sport/activity, intervention-related complications, patient satisfaction, expectations of activity, generic health quality of life, knee-specific quality of life and resource usage.</p><p><strong>Results: </strong>Three hundred and sixteen participants were recruited between February 2017 and April 2020 with 156 randomised to surgical management and 160 to rehabilitation. Forty-one per cent (<i>n</i> = 65) of those allocated to rehabilitation underwent subsequent reconstruction within 18 months with 38% (<i>n</i> = 61) completing rehabilitation and not undergoing surgery. Seventy-two per cent (<i>n</i> = 113) of those allocated to surgery underwent reconstruction within 18 months. Follow-up at the primary outcome time point was 78% (<i>n</i> = 248; surgical, <i>n</i> = 128; rehabilitation, <i>n</i> = 120). Both groups improved over time. Adjusted mean Knee Injury and Osteoarthritis Outcome Score 4 scores at 18 months had increased to 73.0 in the surgical arm and to 64.6 in the rehabilitation arm. The adjusted mean difference was 7.9 (95% confidence interval 2.5 to 13.2; <i>p</i> = 0.005) in favour of surgical management. The per-protocol analyses supported the intention-to-treat results, with a","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 27","pages":"1-97"},"PeriodicalIF":3.5,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11228690/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141467522","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}
Edward Jd Webb, Natalie King, Daniel Howdon, Enitan D Carrol, Joanne Euden, Philip Howard, Philip Pallmann, Martin J Llewelyn, Emma Thomas-Jones, Bethany Shinkins, Jonathan Sandoe
Background: Information on the quality of life of people hospitalised with COVID-19 is important, both in assessing the burden of disease and the cost-effectiveness of treatments. However, there were potential barriers to collecting such evidence.
Objective: To review the existing evidence on quality of life for people hospitalised with COVID-19, with a focus on the amount of evidence available and methods used.
Design: A scoping review with systematic searches.
Results: A total of 35 papers were selected for data extraction. The most common study type was economic evaluation (N = 13), followed by cross-sectional (N = 10). All economic evaluations used published utility values for other conditions to represent COVID-19 inpatients' quality of life. The most popular quality-of-life survey measure was the Pittsburgh Sleep Quality Index (N = 8). There were 12 studies that used a mental health-related survey and 12 that used a sleep-related survey. Five studies used EQ-5D, but only one collected responses from people in the acute phase of COVID-19. Studies reported a negative impact on quality of life for people hospitalised with COVID-19, although many studies did not include a formal comparison group.
Limitations: Although it used systematic searches, this was not a full systematic review.
Conclusion: Quality-of-life data were collected from people hospitalised with COVID-19 from relatively early in the pandemic. However, there was a lack of consensus as to what survey measures to use, and few studies used generic health measures. Economic evaluations for COVID-19 treatments did not use utilities collected from people with COVID-19. In future health crises, researchers should be vigilant for opportunities to collect quality-of-life data from hospitalised patients but should try to co-ordinate as well as ensuring generic health measures are used more.
Funding: This article presents independent research funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme as award number NIHR132254.
{"title":"Evidence of quality of life for hospitalised patients with COVID-19: a scoping review.","authors":"Edward Jd Webb, Natalie King, Daniel Howdon, Enitan D Carrol, Joanne Euden, Philip Howard, Philip Pallmann, Martin J Llewelyn, Emma Thomas-Jones, Bethany Shinkins, Jonathan Sandoe","doi":"10.3310/ATPR4281","DOIUrl":"https://doi.org/10.3310/ATPR4281","url":null,"abstract":"<p><strong>Background: </strong>Information on the quality of life of people hospitalised with COVID-19 is important, both in assessing the burden of disease and the cost-effectiveness of treatments. However, there were potential barriers to collecting such evidence.</p><p><strong>Objective: </strong>To review the existing evidence on quality of life for people hospitalised with COVID-19, with a focus on the amount of evidence available and methods used.</p><p><strong>Design: </strong>A scoping review with systematic searches.</p><p><strong>Results: </strong>A total of 35 papers were selected for data extraction. The most common study type was economic evaluation (<i>N</i> = 13), followed by cross-sectional (<i>N</i> = 10). All economic evaluations used published utility values for other conditions to represent COVID-19 inpatients' quality of life. The most popular quality-of-life survey measure was the Pittsburgh Sleep Quality Index (<i>N</i> = 8). There were 12 studies that used a mental health-related survey and 12 that used a sleep-related survey. Five studies used EQ-5D, but only one collected responses from people in the acute phase of COVID-19. Studies reported a negative impact on quality of life for people hospitalised with COVID-19, although many studies did not include a formal comparison group.</p><p><strong>Limitations: </strong>Although it used systematic searches, this was not a full systematic review.</p><p><strong>Conclusion: </strong>Quality-of-life data were collected from people hospitalised with COVID-19 from relatively early in the pandemic. However, there was a lack of consensus as to what survey measures to use, and few studies used generic health measures. Economic evaluations for COVID-19 treatments did not use utilities collected from people with COVID-19. In future health crises, researchers should be vigilant for opportunities to collect quality-of-life data from hospitalised patients but should try to co-ordinate as well as ensuring generic health measures are used more.</p><p><strong>Funding: </strong>This article presents independent research funded by the National Institute for Health and Care Research (NIHR) <i>Health Technology Assessment</i> programme as award number NIHR132254.</p>","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-23"},"PeriodicalIF":3.6,"publicationDate":"2024-05-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141155181","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Carol Bugge, Suzanne Hagen, Andrew Elders, Helen Mason, Kirsteen Goodman, Melanie Dembinsky, Lynn Melone, Catherine Best, Sarkis Manoukian, Lucy Dwyer, Aethele Khunda, Margaret Graham, Wael Agur, Suzanne Breeman, Jane Culverhouse, Angela Forrest, Mark Forrest, Karen Guerrero, Christine Hemming, Doreen McClurg, John Norrie, Ranee Thakar, Rohna Kearney
<p><strong>Background: </strong>Pelvic organ prolapse is common, causes unpleasant symptoms and negatively affects women's quality of life. In the UK, most women with pelvic organ prolapse attend clinics for pessary care.</p><p><strong>Objectives: </strong>To determine the clinical effectiveness and cost-effectiveness of vaginal pessary self-management on prolapse-specific quality of life for women with prolapse compared with clinic-based care; and to assess intervention acceptability and contextual influences on effectiveness, adherence and fidelity.</p><p><strong>Design: </strong>A multicentre, parallel-group, superiority randomised controlled trial with a mixed-methods process evaluation.</p><p><strong>Participants: </strong>Women attending UK NHS outpatient pessary services, aged ≥ 18 years, using a pessary of any type/material (except shelf, Gellhorn or Cube) for at least 2 weeks. Exclusions: women with limited manual dexterity, with cognitive deficit (prohibiting consent or self-management), pregnant or non-English-speaking.</p><p><strong>Intervention: </strong>The self-management intervention involved a 30-minute teaching appointment, an information leaflet, a 2-week follow-up telephone call and a local clinic telephone helpline number. Clinic-based care involved routine appointments determined by centres' usual practice.</p><p><strong>Allocation: </strong>Remote web-based application; minimisation was by age, pessary user type and centre.</p><p><strong>Blinding: </strong>Participants, those delivering the intervention and researchers were not blinded to group allocation.</p><p><strong>Outcomes: </strong>The patient-reported primary outcome (measured using the Pelvic Floor Impact Questionnaire-7) was prolapse-specific quality of life, and the cost-effectiveness outcome was incremental cost per quality-adjusted life-year (a specifically developed health Resource Use Questionnaire was used) at 18 months post randomisation. Secondary outcome measures included self-efficacy and complications. Process evaluation data were collected by interview, audio-recording and checklist. Analysis was by intention to treat.</p><p><strong>Results: </strong>Three hundred and forty women were randomised (self-management, <i>n</i> = 169; clinic-based care, <i>n</i> = 171). At 18 months post randomisation, 291 questionnaires with valid primary outcome data were available (self-management, <i>n</i> = 139; clinic-based care, <i>n</i> = 152). Baseline economic analysis was based on 264 participants (self-management, <i>n</i> = 125; clinic-based care, <i>n</i> = 139) with valid quality of life and resource use data. Self-management was an acceptable intervention. There was no group difference in prolapse-specific quality of life at 18 months (adjusted mean difference -0.03, 95% confidence interval -9.32 to 9.25). There was fidelity to intervention delivery. Self-management was cost-effective at a willingness-to-pay threshold of £20,000 per quality-adjusted life-year
{"title":"Clinical and cost-effectiveness of pessary self-management versus clinic-based care for pelvic organ prolapse in women: the TOPSY RCT with process evaluation.","authors":"Carol Bugge, Suzanne Hagen, Andrew Elders, Helen Mason, Kirsteen Goodman, Melanie Dembinsky, Lynn Melone, Catherine Best, Sarkis Manoukian, Lucy Dwyer, Aethele Khunda, Margaret Graham, Wael Agur, Suzanne Breeman, Jane Culverhouse, Angela Forrest, Mark Forrest, Karen Guerrero, Christine Hemming, Doreen McClurg, John Norrie, Ranee Thakar, Rohna Kearney","doi":"10.3310/NWTB5403","DOIUrl":"10.3310/NWTB5403","url":null,"abstract":"<p><strong>Background: </strong>Pelvic organ prolapse is common, causes unpleasant symptoms and negatively affects women's quality of life. In the UK, most women with pelvic organ prolapse attend clinics for pessary care.</p><p><strong>Objectives: </strong>To determine the clinical effectiveness and cost-effectiveness of vaginal pessary self-management on prolapse-specific quality of life for women with prolapse compared with clinic-based care; and to assess intervention acceptability and contextual influences on effectiveness, adherence and fidelity.</p><p><strong>Design: </strong>A multicentre, parallel-group, superiority randomised controlled trial with a mixed-methods process evaluation.</p><p><strong>Participants: </strong>Women attending UK NHS outpatient pessary services, aged ≥ 18 years, using a pessary of any type/material (except shelf, Gellhorn or Cube) for at least 2 weeks. Exclusions: women with limited manual dexterity, with cognitive deficit (prohibiting consent or self-management), pregnant or non-English-speaking.</p><p><strong>Intervention: </strong>The self-management intervention involved a 30-minute teaching appointment, an information leaflet, a 2-week follow-up telephone call and a local clinic telephone helpline number. Clinic-based care involved routine appointments determined by centres' usual practice.</p><p><strong>Allocation: </strong>Remote web-based application; minimisation was by age, pessary user type and centre.</p><p><strong>Blinding: </strong>Participants, those delivering the intervention and researchers were not blinded to group allocation.</p><p><strong>Outcomes: </strong>The patient-reported primary outcome (measured using the Pelvic Floor Impact Questionnaire-7) was prolapse-specific quality of life, and the cost-effectiveness outcome was incremental cost per quality-adjusted life-year (a specifically developed health Resource Use Questionnaire was used) at 18 months post randomisation. Secondary outcome measures included self-efficacy and complications. Process evaluation data were collected by interview, audio-recording and checklist. Analysis was by intention to treat.</p><p><strong>Results: </strong>Three hundred and forty women were randomised (self-management, <i>n</i> = 169; clinic-based care, <i>n</i> = 171). At 18 months post randomisation, 291 questionnaires with valid primary outcome data were available (self-management, <i>n</i> = 139; clinic-based care, <i>n</i> = 152). Baseline economic analysis was based on 264 participants (self-management, <i>n</i> = 125; clinic-based care, <i>n</i> = 139) with valid quality of life and resource use data. Self-management was an acceptable intervention. There was no group difference in prolapse-specific quality of life at 18 months (adjusted mean difference -0.03, 95% confidence interval -9.32 to 9.25). There was fidelity to intervention delivery. Self-management was cost-effective at a willingness-to-pay threshold of £20,000 per quality-adjusted life-year","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 23","pages":"1-121"},"PeriodicalIF":3.5,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11145464/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141070849","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}
京公网安备 11010802042870号
京ICP备2023020795号-1
扫码关注我们
求助内容：
应助结果提醒方式：