Nicola Curry, Ross Davenport, Helen Thomas, Erin Fox, Joanne Lucas, Amy Evans, Efthalia Massou, Rupa Sharma, Shaminie Shanmugaranjan, Claire Rourke, Alice Newton, Alison Deary, Nikki Dallas, Chloe Fitzpatrick-Creamer, Jeanette M Podbielski, Charles E Wade, Antoinette Edwards, Jonathan Benger, Stephen Morris, Bryan A Cotton, James Piercy, Laura Green, Karim Brohi, Simon Stanworth
<p><strong>Background: </strong>Traumatic haemorrhage is common after severe injury, leading to disability and death. Cryoprecipitate, a source of fibrinogen, may improve outcomes for patients with traumatic haemorrhage.</p><p><strong>Objective: </strong>To investigate the effects of early fibrinogen supplementation in the form of 3 pools (15 units, approximately 6 g of fibrinogen) of cryoprecipitate on 28-day mortality.</p><p><strong>Design: </strong>A randomised, parallel-group, unblinded, multicentre, international trial and economic evaluation. Patients were randomised to either the intervention (early cryoprecipitate) or the comparator (standard major haemorrhage protocol) arm via opaque, sealed envelopes in the emergency department or the transfusion laboratory/blood bank. All analyses were performed on an intention-to-treat basis. A cost-effectiveness analysis was undertaken.</p><p><strong>Setting: </strong>Twenty-five major trauma centres in the UK and one level 1 trauma centre in the USA.</p><p><strong>Participants: </strong>Adults who had traumatic haemorrhage following severe injury requiring activation of the major haemorrhage protocol and had received a blood transfusion.</p><p><strong>Intervention: </strong>Early cryoprecipitate - 3 pools (equivalent to 15 single units of cryoprecipitate or 6 g of fibrinogen supplementation), infused as rapidly as possible, within 90 minutes of arrival at hospital in addition to standard major haemorrhage protocol or standard major haemorrhage protocol only.</p><p><strong>Main outcome measures: </strong>The primary outcome was all-cause mortality at 28 days. The secondary outcomes were all-cause mortality at 6 hours, 24 hours, 6 months and 12 months from admission; death from bleeding at 6 hours and 24 hours; transfusion requirements at 24 hours from admission; destination of participant at discharge; quality-of-life measurements (EuroQol-5 Dimensions, five-level version and Glasgow Outcome Scale) at discharge/day 28 and 6 months after injury; and hospital resource use up to discharge or day 28 (including ventilator-days, hours spent in critical care and inpatient stays).</p><p><strong>Results: </strong>Eight hundred and five patients were randomised to receive the standard major haemorrhage protocol (control arm). Seven hundred and ninety-nine patients were randomised to receive an additional three pools of cryoprecipitate in addition to standard care (intervention arm). Baseline characteristics appeared well matched. Patients had a median age of 39 (interquartile range 26-55) years, and the majority (79%) were male. All-cause 28-day mortality (<i>n</i> = 1531 patients; intention to treat) was 25.3% in the intervention arm compared with 26.1% in the control arm (odds ratio 0.96; <i>p</i> = 0.74).</p><p><strong>Limitations: </strong>There was variability in the timing of cryoprecipitate administration, with overlap between the treatment arms, limiting the degree of intervention separation.</p><p><st
{"title":"Early high-dose cryoprecipitate to reduce mortality in adult patients with traumatic haemorrhage: the CRYOSTAT-2 RCT with cost-effectiveness analysis.","authors":"Nicola Curry, Ross Davenport, Helen Thomas, Erin Fox, Joanne Lucas, Amy Evans, Efthalia Massou, Rupa Sharma, Shaminie Shanmugaranjan, Claire Rourke, Alice Newton, Alison Deary, Nikki Dallas, Chloe Fitzpatrick-Creamer, Jeanette M Podbielski, Charles E Wade, Antoinette Edwards, Jonathan Benger, Stephen Morris, Bryan A Cotton, James Piercy, Laura Green, Karim Brohi, Simon Stanworth","doi":"10.3310/JYTR6938","DOIUrl":"https://doi.org/10.3310/JYTR6938","url":null,"abstract":"<p><strong>Background: </strong>Traumatic haemorrhage is common after severe injury, leading to disability and death. Cryoprecipitate, a source of fibrinogen, may improve outcomes for patients with traumatic haemorrhage.</p><p><strong>Objective: </strong>To investigate the effects of early fibrinogen supplementation in the form of 3 pools (15 units, approximately 6 g of fibrinogen) of cryoprecipitate on 28-day mortality.</p><p><strong>Design: </strong>A randomised, parallel-group, unblinded, multicentre, international trial and economic evaluation. Patients were randomised to either the intervention (early cryoprecipitate) or the comparator (standard major haemorrhage protocol) arm via opaque, sealed envelopes in the emergency department or the transfusion laboratory/blood bank. All analyses were performed on an intention-to-treat basis. A cost-effectiveness analysis was undertaken.</p><p><strong>Setting: </strong>Twenty-five major trauma centres in the UK and one level 1 trauma centre in the USA.</p><p><strong>Participants: </strong>Adults who had traumatic haemorrhage following severe injury requiring activation of the major haemorrhage protocol and had received a blood transfusion.</p><p><strong>Intervention: </strong>Early cryoprecipitate - 3 pools (equivalent to 15 single units of cryoprecipitate or 6 g of fibrinogen supplementation), infused as rapidly as possible, within 90 minutes of arrival at hospital in addition to standard major haemorrhage protocol or standard major haemorrhage protocol only.</p><p><strong>Main outcome measures: </strong>The primary outcome was all-cause mortality at 28 days. The secondary outcomes were all-cause mortality at 6 hours, 24 hours, 6 months and 12 months from admission; death from bleeding at 6 hours and 24 hours; transfusion requirements at 24 hours from admission; destination of participant at discharge; quality-of-life measurements (EuroQol-5 Dimensions, five-level version and Glasgow Outcome Scale) at discharge/day 28 and 6 months after injury; and hospital resource use up to discharge or day 28 (including ventilator-days, hours spent in critical care and inpatient stays).</p><p><strong>Results: </strong>Eight hundred and five patients were randomised to receive the standard major haemorrhage protocol (control arm). Seven hundred and ninety-nine patients were randomised to receive an additional three pools of cryoprecipitate in addition to standard care (intervention arm). Baseline characteristics appeared well matched. Patients had a median age of 39 (interquartile range 26-55) years, and the majority (79%) were male. All-cause 28-day mortality (<i>n</i> = 1531 patients; intention to treat) was 25.3% in the intervention arm compared with 26.1% in the control arm (odds ratio 0.96; <i>p</i> = 0.74).</p><p><strong>Limitations: </strong>There was variability in the timing of cryoprecipitate administration, with overlap between the treatment arms, limiting the degree of intervention separation.</p><p><st","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 76","pages":"1-69"},"PeriodicalIF":3.5,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638748","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}
Katie E Webster, Tom Parkhouse, Sarah Dawson, Hayley E Jones, Emily L Brown, Alastair D Hay, Penny Whiting, Christie Cabral, Deborah M Caldwell, Julian Pt Higgins
<p><strong>Background: </strong>Acute respiratory infections are a common reason for consultation with primary and emergency healthcare services. Identifying individuals with a bacterial infection is crucial to ensure appropriate treatment. However, it is also important to avoid overprescription of antibiotics, to prevent unnecessary side effects and antimicrobial resistance. We conducted a systematic review to summarise evidence on the diagnostic accuracy of symptoms, signs and point-of-care tests to diagnose bacterial respiratory tract infection in adults, and to diagnose two common respiratory viruses, influenza and respiratory syncytial virus.</p><p><strong>Methods: </strong>The primary approach was an overview of existing systematic reviews. We conducted literature searches (22 May 2023) to identify systematic reviews of the diagnostic accuracy of point-of-care tests. Where multiple reviews were identified, we selected the most recent and comprehensive review, with the greatest overlap in scope with our review question. Methodological quality was assessed using the Risk of Bias in Systematic Reviews tool. Summary estimates of diagnostic accuracy (sensitivity, specificity or area under the curve) were extracted. Where no systematic review was identified, we searched for primary studies. We extracted sufficient data to construct a 2 × 2 table of diagnostic accuracy, to calculate sensitivity and specificity. Methodological quality was assessed using the Quality Assessment of Diagnostic Accuracy Studies version 2 tool. Where possible, meta-analyses were conducted. We used GRADE to assess the certainty of the evidence from existing reviews and new analyses.</p><p><strong>Results: </strong>We identified 23 reviews which addressed our review question; 6 were selected as the most comprehensive and similar in scope to our review protocol. These systematic reviews considered the following tests for bacterial respiratory infection: individual symptoms and signs; combinations of symptoms and signs (in clinical prediction models); clinical prediction models incorporating C-reactive protein; and biological markers related to infection (including C-reactive protein, procalcitonin and others). We also identified systematic reviews that reported the accuracy of specific tests for influenza and respiratory syncytial virus. No reviews were found that assessed the diagnostic accuracy of white cell count for bacterial respiratory infection, or multiplex tests for influenza and respiratory syncytial virus. We therefore conducted searches for primary studies, and carried out meta-analyses for these index tests. Overall, we found that symptoms and signs have poor diagnostic accuracy for bacterial respiratory infection (sensitivity ranging from 9.6% to 89.1%; specificity ranging from 13.4% to 95%). Accuracy of biomarkers was slightly better, particularly when combinations of biomarkers were used (sensitivity 80-90%, specificity 82-93%). The sensitivity and specifici
背景：急性呼吸道感染是初级和急诊医疗服务中常见的就诊原因。识别细菌感染患者对于确保适当治疗至关重要。然而，避免过度处方抗生素以防止不必要的副作用和抗菌药耐药性也很重要。我们进行了一项系统性综述，总结了诊断成人细菌性呼吸道感染以及诊断两种常见呼吸道病毒（流感和呼吸道合胞病毒）的症状、体征和护理点检测诊断准确性的证据：主要方法是概述现有的系统综述。我们进行了文献检索（2023 年 5 月 22 日），以确定有关护理点检测诊断准确性的系统综述。在发现多篇综述的情况下，我们选择了最新、最全面的综述，其范围与我们的综述问题重合度最高。我们使用 "系统综述偏倚风险"（Risk of Bias in Systematic Reviews）工具对方法学质量进行了评估。提取诊断准确性（灵敏度、特异性或曲线下面积）的简要估计值。在未发现系统综述的情况下，我们搜索了主要研究。我们提取了足够的数据来构建诊断准确性的 2 × 2 表，以计算灵敏度和特异性。方法学质量采用诊断准确性研究质量评估第 2 版工具进行评估。在可能的情况下，我们进行了荟萃分析。我们使用 GRADE 评估现有综述和新分析中证据的确定性：我们确定了 23 篇综述涉及我们的综述问题；其中 6 篇被选为最全面且与我们的综述方案范围相似的综述。这些系统性综述考虑了细菌性呼吸道感染的以下检测方法：单个症状和体征；症状和体征组合（在临床预测模型中）；包含 C 反应蛋白的临床预测模型；以及与感染相关的生物标记物（包括 C 反应蛋白、降钙素原等）。我们还发现了报告流感和呼吸道合胞病毒特定检测准确性的系统性综述。我们没有发现评估细菌性呼吸道感染白细胞计数诊断准确性的综述，也没有发现评估流感和呼吸道合胞病毒多重检测准确性的综述。因此，我们检索了主要研究，并对这些指标检测进行了荟萃分析。总体而言，我们发现症状和体征对细菌性呼吸道感染的诊断准确性较低（敏感性从 9.6% 到 89.1%；特异性从 13.4% 到 95%）。生物标志物的准确性稍好，尤其是在使用生物标志物组合时（灵敏度为 80-90%，特异性为 82-93%）。不同类型检测对流感或呼吸道合胞病毒的敏感性和特异性差异很大。涉及核酸扩增技术的检测（单一病原体或多重检测）对流感的诊断准确率最高（灵敏度 91-99.8%，特异性 96.8-99.4%）：在使用 GRADE 进行评估时，大多数证据被认为确定性较低或非常低，原因包括效果估计不精确、可能存在偏差以及纳入了本综述范围之外的参与者（儿童或住院患者）：目前的证据不足以支持在初级和急诊护理中常规使用护理点检测。进一步的工作必须确定引入护理点检测是增加了价值，还是仅仅增加了医疗成本：本文是由美国国家健康与护理研究所（NIHR）健康技术评估项目资助的独立研究，获奖编号为NIHR159948。
{"title":"Diagnostic accuracy of point-of-care tests for acute respiratory infection: a systematic review of reviews.","authors":"Katie E Webster, Tom Parkhouse, Sarah Dawson, Hayley E Jones, Emily L Brown, Alastair D Hay, Penny Whiting, Christie Cabral, Deborah M Caldwell, Julian Pt Higgins","doi":"10.3310/JLCP4570","DOIUrl":"https://doi.org/10.3310/JLCP4570","url":null,"abstract":"<p><strong>Background: </strong>Acute respiratory infections are a common reason for consultation with primary and emergency healthcare services. Identifying individuals with a bacterial infection is crucial to ensure appropriate treatment. However, it is also important to avoid overprescription of antibiotics, to prevent unnecessary side effects and antimicrobial resistance. We conducted a systematic review to summarise evidence on the diagnostic accuracy of symptoms, signs and point-of-care tests to diagnose bacterial respiratory tract infection in adults, and to diagnose two common respiratory viruses, influenza and respiratory syncytial virus.</p><p><strong>Methods: </strong>The primary approach was an overview of existing systematic reviews. We conducted literature searches (22 May 2023) to identify systematic reviews of the diagnostic accuracy of point-of-care tests. Where multiple reviews were identified, we selected the most recent and comprehensive review, with the greatest overlap in scope with our review question. Methodological quality was assessed using the Risk of Bias in Systematic Reviews tool. Summary estimates of diagnostic accuracy (sensitivity, specificity or area under the curve) were extracted. Where no systematic review was identified, we searched for primary studies. We extracted sufficient data to construct a 2 × 2 table of diagnostic accuracy, to calculate sensitivity and specificity. Methodological quality was assessed using the Quality Assessment of Diagnostic Accuracy Studies version 2 tool. Where possible, meta-analyses were conducted. We used GRADE to assess the certainty of the evidence from existing reviews and new analyses.</p><p><strong>Results: </strong>We identified 23 reviews which addressed our review question; 6 were selected as the most comprehensive and similar in scope to our review protocol. These systematic reviews considered the following tests for bacterial respiratory infection: individual symptoms and signs; combinations of symptoms and signs (in clinical prediction models); clinical prediction models incorporating C-reactive protein; and biological markers related to infection (including C-reactive protein, procalcitonin and others). We also identified systematic reviews that reported the accuracy of specific tests for influenza and respiratory syncytial virus. No reviews were found that assessed the diagnostic accuracy of white cell count for bacterial respiratory infection, or multiplex tests for influenza and respiratory syncytial virus. We therefore conducted searches for primary studies, and carried out meta-analyses for these index tests. Overall, we found that symptoms and signs have poor diagnostic accuracy for bacterial respiratory infection (sensitivity ranging from 9.6% to 89.1%; specificity ranging from 13.4% to 95%). Accuracy of biomarkers was slightly better, particularly when combinations of biomarkers were used (sensitivity 80-90%, specificity 82-93%). The sensitivity and specifici","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-75"},"PeriodicalIF":3.5,"publicationDate":"2024-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142365067","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}
Jacqui Prieto, Jennie Wilson, Alison Tingle, Emily Cooper, Melanie Handley, Jo Rycroft-Malone, Jennifer Bostock, Lynne Williams, Heather Loveday
<p><strong>Background: </strong>Urinary tract infection is the most diagnosed infection in older people. It accounts for more than 50% of antibiotic prescriptions in care homes and is a frequent reason for care home residents being hospitalised.</p><p><strong>Objective: </strong>This realist review developed and refined programme theories for preventing and recognising urinary tract infection, exploring what works, for whom and in what circumstances.</p><p><strong>Design: </strong>The review used realist synthesis to explore existing literature on the detection and prevention of urinary tract infection, complemented by stakeholder consultation. It applies to the UK context, although other healthcare systems may identify synergies in our findings.</p><p><strong>Data sources: </strong>Bibliographic databases searched included MEDLINE, CINAHL, EMBASE, Cochrane Library, Web of Science Core Collection (including the Social Sciences Citation Index), Sociological Abstracts, Bibliomap and National Institute for Health and Care Research Journals Library.</p><p><strong>Data selection and extraction: </strong>Title and abstract screening were undertaken by two researchers independently of each other. Selection and assessment were based on relevance and rigour and cross-checked by a second researcher. Data extracted from the included studies were explored for explanations about how the interventions were considered to work (or not). Evidence tables were constructed to enable identification of patterns across studies that offered insight about the features of successful interventions.</p><p><strong>Data analysis and synthesis: </strong>Programme theories were constructed through a four-stage process involving scoping workshops, examination of relevant extant theory, analysis and synthesis of primary research, teacher-learner interviews and a cross-system stakeholder event. A process of abductive and retroductive reasoning was used to construct context-mechanism-outcome configurations to inform programme theory.</p><p><strong>Results: </strong>The scoping review and stakeholder engagement identified three theory areas that address the prevention and recognition of urinary tract infection and show what is needed to implement best practice. Nine context-mechanism-outcome configurations provided an explanation of how interventions to prevent and recognise urinary tract infection might work in care homes. These were (1) recognition of urinary tract infection is informed by skills in clinical reasoning, (2) decision-support tools enable a whole care team approach to communication, (3) active monitoring is recognised as a legitimate care routine, (4) hydration is recognised as a care priority for all residents, (5) systems are in place to drive action that helps residents to drink more, (6) good infection prevention practice is applied to indwelling urinary catheters, (7) proactive strategies are in place to prevent recurrent urinary tract infection, (8) care home l
资助：该奖项由国家健康与护理研究所（NIHR）健康技术评估计划（NIHR奖项编号：NIHR130396）资助，全文发表于《健康技术评估》第28卷第68期。如需了解更多奖项信息，请访问 NIHR Funding and Awards 网站。
{"title":"Strategies for older people living in care homes to prevent urinary tract infection: the StOP UTI realist synthesis.","authors":"Jacqui Prieto, Jennie Wilson, Alison Tingle, Emily Cooper, Melanie Handley, Jo Rycroft-Malone, Jennifer Bostock, Lynne Williams, Heather Loveday","doi":"10.3310/DADT3410","DOIUrl":"10.3310/DADT3410","url":null,"abstract":"<p><strong>Background: </strong>Urinary tract infection is the most diagnosed infection in older people. It accounts for more than 50% of antibiotic prescriptions in care homes and is a frequent reason for care home residents being hospitalised.</p><p><strong>Objective: </strong>This realist review developed and refined programme theories for preventing and recognising urinary tract infection, exploring what works, for whom and in what circumstances.</p><p><strong>Design: </strong>The review used realist synthesis to explore existing literature on the detection and prevention of urinary tract infection, complemented by stakeholder consultation. It applies to the UK context, although other healthcare systems may identify synergies in our findings.</p><p><strong>Data sources: </strong>Bibliographic databases searched included MEDLINE, CINAHL, EMBASE, Cochrane Library, Web of Science Core Collection (including the Social Sciences Citation Index), Sociological Abstracts, Bibliomap and National Institute for Health and Care Research Journals Library.</p><p><strong>Data selection and extraction: </strong>Title and abstract screening were undertaken by two researchers independently of each other. Selection and assessment were based on relevance and rigour and cross-checked by a second researcher. Data extracted from the included studies were explored for explanations about how the interventions were considered to work (or not). Evidence tables were constructed to enable identification of patterns across studies that offered insight about the features of successful interventions.</p><p><strong>Data analysis and synthesis: </strong>Programme theories were constructed through a four-stage process involving scoping workshops, examination of relevant extant theory, analysis and synthesis of primary research, teacher-learner interviews and a cross-system stakeholder event. A process of abductive and retroductive reasoning was used to construct context-mechanism-outcome configurations to inform programme theory.</p><p><strong>Results: </strong>The scoping review and stakeholder engagement identified three theory areas that address the prevention and recognition of urinary tract infection and show what is needed to implement best practice. Nine context-mechanism-outcome configurations provided an explanation of how interventions to prevent and recognise urinary tract infection might work in care homes. These were (1) recognition of urinary tract infection is informed by skills in clinical reasoning, (2) decision-support tools enable a whole care team approach to communication, (3) active monitoring is recognised as a legitimate care routine, (4) hydration is recognised as a care priority for all residents, (5) systems are in place to drive action that helps residents to drink more, (6) good infection prevention practice is applied to indwelling urinary catheters, (7) proactive strategies are in place to prevent recurrent urinary tract infection, (8) care home l","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 68","pages":"1-139"},"PeriodicalIF":3.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11513742/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142463900","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}
Catherine A Moakes, Andrew W Bradbury, Zainab Abdali, Gareth R Bate, Jack Hall, Hugh Jarrett, Lisa Kelly, Jesse Kigozi, Suzanne Lockyer, Lewis Meecham, Smitaa Patel, Matthew Popplewell, Gemma Slinn, Jonathan J Deeks
<p><strong>Background: </strong>Chronic limb-threatening ischaemia with ischaemic pain and/or tissue loss.</p><p><strong>Objective: </strong>To examine the clinical and cost-effectiveness of a vein bypass-first compared to a best endovascular treatment-first revascularisation strategy in preventing major amputation or death.</p><p><strong>Design: </strong>Superiority, open, pragmatic, multicentre, phase III randomised trial.</p><p><strong>Setting: </strong>Thirty-nine vascular surgery units in the United Kingdom, and one each in Sweden and Denmark.</p><p><strong>Participants: </strong>Patients with chronic limb-threatening ischaemia due to atherosclerotic peripheral arterial disease who required an infra-popliteal revascularisation, with or without an additional more proximal infra-inguinal revascularisation procedure, to restore limb perfusion.</p><p><strong>Interventions: </strong>A vein bypass-first or a best endovascular treatment-first infra-popliteal, with or without an additional more proximal infra-inguinal revascularisation strategy.</p><p><strong>Main outcome measures: </strong>The primary outcome was amputation-free survival. Secondary outcomes included overall survival, major amputation, further revascularisation interventions, major adverse limb event, health-related quality of life and serious adverse events.</p><p><strong>Methods: </strong>Participants were randomised to a vein bypass-first or a best endovascular treatment-first revascularisation strategy. The original sample size of 600 participants (247 events) was based on a hazard ratio of 0.66 with amputation-free survival rates of 0.72, 0.62, 0.53, 0.47 and 0.35 in years 1-5 in the best endovascular treatment-first group with 90% power and alpha at <i>p</i> = 0.05. The sample size was revised to an event-based approach as a result of increased follow-up time due to slower than anticipated recruitment rates. Participants were followed up for a minimum of 2 years. A cost-effectiveness analysis was employed to estimate differences in total hospital costs and amputation-free survival between the groups. Additionally, a cost-utility analysis was carried out and the total cost and quality-adjusted life-years, 2 and 3 years after randomisation were used.</p><p><strong>Results: </strong>Between 22 July 2014 and 30 November 2020, 345 participants were randomised, 172 to vein bypass-first and 173 to best endovascular treatment-first. Non-amputation-free survival occurred in 108 (63%) of 172 patients in the vein bypass-first group and 92 (53%) of 173 patients in the best endovascular treatment-first group [adjusted hazard ratio 1.35 (95% confidence interval 1.02 to 1.80); <i>p</i> = 0.037]. Ninety-one (53%) of 172 patients in the vein bypass-first group and 77 (45%) of 173 patients in the best endovascular treatment-first group died [adjusted hazard ratio 1.37 (95% confidence interval 1.00 to 1.87)]. Over follow-up, the economic evaluation discounted results showed that best endovascula
{"title":"Vein bypass first vs. best endovascular treatment first revascularisation strategy for chronic limb-threatening ischaemia due to infra-popliteal disease: the BASIL-2 RCT.","authors":"Catherine A Moakes, Andrew W Bradbury, Zainab Abdali, Gareth R Bate, Jack Hall, Hugh Jarrett, Lisa Kelly, Jesse Kigozi, Suzanne Lockyer, Lewis Meecham, Smitaa Patel, Matthew Popplewell, Gemma Slinn, Jonathan J Deeks","doi":"10.3310/YTFV4524","DOIUrl":"10.3310/YTFV4524","url":null,"abstract":"<p><strong>Background: </strong>Chronic limb-threatening ischaemia with ischaemic pain and/or tissue loss.</p><p><strong>Objective: </strong>To examine the clinical and cost-effectiveness of a vein bypass-first compared to a best endovascular treatment-first revascularisation strategy in preventing major amputation or death.</p><p><strong>Design: </strong>Superiority, open, pragmatic, multicentre, phase III randomised trial.</p><p><strong>Setting: </strong>Thirty-nine vascular surgery units in the United Kingdom, and one each in Sweden and Denmark.</p><p><strong>Participants: </strong>Patients with chronic limb-threatening ischaemia due to atherosclerotic peripheral arterial disease who required an infra-popliteal revascularisation, with or without an additional more proximal infra-inguinal revascularisation procedure, to restore limb perfusion.</p><p><strong>Interventions: </strong>A vein bypass-first or a best endovascular treatment-first infra-popliteal, with or without an additional more proximal infra-inguinal revascularisation strategy.</p><p><strong>Main outcome measures: </strong>The primary outcome was amputation-free survival. Secondary outcomes included overall survival, major amputation, further revascularisation interventions, major adverse limb event, health-related quality of life and serious adverse events.</p><p><strong>Methods: </strong>Participants were randomised to a vein bypass-first or a best endovascular treatment-first revascularisation strategy. The original sample size of 600 participants (247 events) was based on a hazard ratio of 0.66 with amputation-free survival rates of 0.72, 0.62, 0.53, 0.47 and 0.35 in years 1-5 in the best endovascular treatment-first group with 90% power and alpha at <i>p</i> = 0.05. The sample size was revised to an event-based approach as a result of increased follow-up time due to slower than anticipated recruitment rates. Participants were followed up for a minimum of 2 years. A cost-effectiveness analysis was employed to estimate differences in total hospital costs and amputation-free survival between the groups. Additionally, a cost-utility analysis was carried out and the total cost and quality-adjusted life-years, 2 and 3 years after randomisation were used.</p><p><strong>Results: </strong>Between 22 July 2014 and 30 November 2020, 345 participants were randomised, 172 to vein bypass-first and 173 to best endovascular treatment-first. Non-amputation-free survival occurred in 108 (63%) of 172 patients in the vein bypass-first group and 92 (53%) of 173 patients in the best endovascular treatment-first group [adjusted hazard ratio 1.35 (95% confidence interval 1.02 to 1.80); <i>p</i> = 0.037]. Ninety-one (53%) of 172 patients in the vein bypass-first group and 77 (45%) of 173 patients in the best endovascular treatment-first group died [adjusted hazard ratio 1.37 (95% confidence interval 1.00 to 1.87)]. Over follow-up, the economic evaluation discounted results showed that best endovascula","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 65","pages":"1-72"},"PeriodicalIF":3.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11491987/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142463897","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}
Miriam Wittmann, Isabelle L Smith, Sarah Tess Brown, Anna Berekméri, Armando Vargas-Palacios, Lesley Sunderland, Amy Barker, Fiona Cowdell, Steven Ersser, Rachael Gilberts, Cathy Green, Philip Hampton, Catherine Smith, Jane Nixon
<p><strong>Background: </strong>Hand eczema is common and a cause of morbidity and occupational disability. When education, irritant/contact allergen avoidance, moisturisation and topical corticosteroids are insufficient to control chronic hand eczema, ultraviolet therapy or systemic immune-modifying drugs are used. There is no treatment pathway generally accepted by UK dermatologists.</p><p><strong>Primary objective: </strong>Compare alitretinoin and ultraviolet therapy as first-line therapy in terms of disease activity at 12 weeks post planned start of treatment.</p><p><strong>Design: </strong>Prospective, multicentre, open-label, two-arm parallel group, adaptive randomised controlled trial with one planned interim analysis, and an economic evaluation.</p><p><strong>Setting: </strong>UK secondary care dermatology outpatient clinics.</p><p><strong>Participants: </strong>Patients with severe chronic hand eczema unresponsive to at least 4 weeks of treatment with potent topical corticosteroids.</p><p><strong>Primary end point: </strong>Natural logarithm of the Hand Eczema Severity Index + 1, 12 weeks post planned start of treatment.</p><p><strong>Randomisation: </strong>Participants randomised 1 : 1 by minimisation to alitretinoin or ultraviolet therapy for 12 to 24 weeks.</p><p><strong>Blinding: </strong>Blinded primary end-point assessor.</p><p><strong>Results: </strong>Intention-to-treat population: 441 (100.0%) participants; 220 (49.9%) alitretinoin and 221 (50.1%) ultraviolet therapy. At least one dose was received by 212 (96.4%) alitretinoin and 196 (88.7%) ultraviolet therapy participants.</p><p><strong>Primary outcome: </strong>The unadjusted median (interquartile range) relative change in hand eczema severity index at 12 weeks was 30% (10-70%) of that at baseline for alitretinoin compared with 50% (20-100%) for ultraviolet therapy. There was a statistically significant benefit of alitretinoin compared with ultraviolet therapy at 12 weeks, with an estimated fold change or relative difference (95% confidence interval) = 0.66 (0.52 to 0.82), <i>p</i> = 0.0003 at 12 weeks. There was no evidence of a difference at 24 or 52 weeks, with the estimated fold change (95% confidence interval) equal to 0.92 (0.798 to 1.08) and 1.27 (0.97 to 1.67), respectively.</p><p><strong>Primary analysis results were consistent for secondary end points: </strong>Fifty-nine per cent allocated to alitretinoin and 61% allocated to ultraviolet therapy achieved a clear/almost clear assessment during the trial period. Differential treatment compliance observed: 145 (65.9%) alitretinoin and 53 (24.0%) ultraviolet therapy participants confirmed compliance (≥ 80% received, no treatment breaks > 7 days during first 12 weeks). High levels of missing data were observed.</p><p><strong>Safety: </strong>One hundred and thirty-five reportable adverse events across 79 participants, 55 (25.0%) alitretinoin and 24 (10.9%) ultraviolet therapy. Four serious adverse events (two alitreti
{"title":"Alitretinoin versus phototherapy as the first-line treatment in adults with severe chronic hand eczema: the ALPHA RCT.","authors":"Miriam Wittmann, Isabelle L Smith, Sarah Tess Brown, Anna Berekméri, Armando Vargas-Palacios, Lesley Sunderland, Amy Barker, Fiona Cowdell, Steven Ersser, Rachael Gilberts, Cathy Green, Philip Hampton, Catherine Smith, Jane Nixon","doi":"10.3310/TWQC0141","DOIUrl":"10.3310/TWQC0141","url":null,"abstract":"<p><strong>Background: </strong>Hand eczema is common and a cause of morbidity and occupational disability. When education, irritant/contact allergen avoidance, moisturisation and topical corticosteroids are insufficient to control chronic hand eczema, ultraviolet therapy or systemic immune-modifying drugs are used. There is no treatment pathway generally accepted by UK dermatologists.</p><p><strong>Primary objective: </strong>Compare alitretinoin and ultraviolet therapy as first-line therapy in terms of disease activity at 12 weeks post planned start of treatment.</p><p><strong>Design: </strong>Prospective, multicentre, open-label, two-arm parallel group, adaptive randomised controlled trial with one planned interim analysis, and an economic evaluation.</p><p><strong>Setting: </strong>UK secondary care dermatology outpatient clinics.</p><p><strong>Participants: </strong>Patients with severe chronic hand eczema unresponsive to at least 4 weeks of treatment with potent topical corticosteroids.</p><p><strong>Primary end point: </strong>Natural logarithm of the Hand Eczema Severity Index + 1, 12 weeks post planned start of treatment.</p><p><strong>Randomisation: </strong>Participants randomised 1 : 1 by minimisation to alitretinoin or ultraviolet therapy for 12 to 24 weeks.</p><p><strong>Blinding: </strong>Blinded primary end-point assessor.</p><p><strong>Results: </strong>Intention-to-treat population: 441 (100.0%) participants; 220 (49.9%) alitretinoin and 221 (50.1%) ultraviolet therapy. At least one dose was received by 212 (96.4%) alitretinoin and 196 (88.7%) ultraviolet therapy participants.</p><p><strong>Primary outcome: </strong>The unadjusted median (interquartile range) relative change in hand eczema severity index at 12 weeks was 30% (10-70%) of that at baseline for alitretinoin compared with 50% (20-100%) for ultraviolet therapy. There was a statistically significant benefit of alitretinoin compared with ultraviolet therapy at 12 weeks, with an estimated fold change or relative difference (95% confidence interval) = 0.66 (0.52 to 0.82), <i>p</i> = 0.0003 at 12 weeks. There was no evidence of a difference at 24 or 52 weeks, with the estimated fold change (95% confidence interval) equal to 0.92 (0.798 to 1.08) and 1.27 (0.97 to 1.67), respectively.</p><p><strong>Primary analysis results were consistent for secondary end points: </strong>Fifty-nine per cent allocated to alitretinoin and 61% allocated to ultraviolet therapy achieved a clear/almost clear assessment during the trial period. Differential treatment compliance observed: 145 (65.9%) alitretinoin and 53 (24.0%) ultraviolet therapy participants confirmed compliance (≥ 80% received, no treatment breaks > 7 days during first 12 weeks). High levels of missing data were observed.</p><p><strong>Safety: </strong>One hundred and thirty-five reportable adverse events across 79 participants, 55 (25.0%) alitretinoin and 24 (10.9%) ultraviolet therapy. Four serious adverse events (two alitreti","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 59","pages":"1-123"},"PeriodicalIF":3.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11472215/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142371696","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}
Elizabeth Hughes, Jane Harris, Tom Ainscough, Angela Bate, Alex Copello, Sonia Dalkin, Gail Gilchrist, Emma Griffith, Lisa Jones, Michelle Maden, Luke Mitcheson, Harry Sumnall, Charlotte Walker
<p><strong>Background: </strong>People with severe mental illness who experience co-occurring substance use experience poor outcome including suicide, violence, relapses and use of crisis services. They struggle to access care and treatment due to a lack of an integrated and co-ordinated approach which means that some people can fall between services. Despite these concerns, there is limited evidence as to what works for this population.</p><p><strong>Objectives: </strong>To undertake a realist evaluation of service models in order to identify and refine programme theories of what works under what contexts for this population.</p><p><strong>Design: </strong>Realist synthesis and evaluation using published literature and case study data.</p><p><strong>Setting: </strong>Mental health, substance use and related services that had some form of service provision in six locations in the United Kingdom (five in England and one in Northern Ireland).</p><p><strong>Participants: </strong>People with lived experience of severe mental illness and co-occurring substance use, carers and staff who work in the specialist roles as well as staff in mental health and substance use services.</p><p><strong>Results: </strong>Eleven initial programme theories were generated by the evidence synthesis and in conjunction with stakeholders. These theories were refined through focus groups and interviews with 58 staff, 25 service users and 12 carers across the 6 case study areas. We identified three forms of service provision (network, consultancy and lead and link worker); however, all offered broadly similar interventions. Evidence was identified to support most of the 11 programme theories. Theories clustered around effective leadership, workforce development and collaborative integrated care pathways. Outcomes that are meaningful for service users and staff were identified, including the importance of engagement.</p><p><strong>Limitations: </strong>The requirement for online data collection (due to the COVID-19 pandemic) worked well for staff data but worked less well for service users and carers. Consequently, this may have reduced the involvement of those without access to information technology equipment.</p><p><strong>Conclusion: </strong>The realist evaluation co-occurring study provides details on how and in what circumstances integrated care can work better for people with co-occurring severe mental health and alcohol/drug conditions. This requires joined-up policy at government level and local integration of services. We have also identified the value of expert clinicians who can support the workforce in sustaining this programme of work. People with co-occurring severe mental health and alcohol/drug conditions have complex and multifaceted needs which require a comprehensive and long-term integrated approach. The shift to integrated health and social care is promising but will require local support (local expert leaders, network opportunities and clarity of role
{"title":"Care models for coexisting serious mental health and alcohol/drug conditions: the RECO realist evidence synthesis and case study evaluation.","authors":"Elizabeth Hughes, Jane Harris, Tom Ainscough, Angela Bate, Alex Copello, Sonia Dalkin, Gail Gilchrist, Emma Griffith, Lisa Jones, Michelle Maden, Luke Mitcheson, Harry Sumnall, Charlotte Walker","doi":"10.3310/JTNT0476","DOIUrl":"10.3310/JTNT0476","url":null,"abstract":"<p><strong>Background: </strong>People with severe mental illness who experience co-occurring substance use experience poor outcome including suicide, violence, relapses and use of crisis services. They struggle to access care and treatment due to a lack of an integrated and co-ordinated approach which means that some people can fall between services. Despite these concerns, there is limited evidence as to what works for this population.</p><p><strong>Objectives: </strong>To undertake a realist evaluation of service models in order to identify and refine programme theories of what works under what contexts for this population.</p><p><strong>Design: </strong>Realist synthesis and evaluation using published literature and case study data.</p><p><strong>Setting: </strong>Mental health, substance use and related services that had some form of service provision in six locations in the United Kingdom (five in England and one in Northern Ireland).</p><p><strong>Participants: </strong>People with lived experience of severe mental illness and co-occurring substance use, carers and staff who work in the specialist roles as well as staff in mental health and substance use services.</p><p><strong>Results: </strong>Eleven initial programme theories were generated by the evidence synthesis and in conjunction with stakeholders. These theories were refined through focus groups and interviews with 58 staff, 25 service users and 12 carers across the 6 case study areas. We identified three forms of service provision (network, consultancy and lead and link worker); however, all offered broadly similar interventions. Evidence was identified to support most of the 11 programme theories. Theories clustered around effective leadership, workforce development and collaborative integrated care pathways. Outcomes that are meaningful for service users and staff were identified, including the importance of engagement.</p><p><strong>Limitations: </strong>The requirement for online data collection (due to the COVID-19 pandemic) worked well for staff data but worked less well for service users and carers. Consequently, this may have reduced the involvement of those without access to information technology equipment.</p><p><strong>Conclusion: </strong>The realist evaluation co-occurring study provides details on how and in what circumstances integrated care can work better for people with co-occurring severe mental health and alcohol/drug conditions. This requires joined-up policy at government level and local integration of services. We have also identified the value of expert clinicians who can support the workforce in sustaining this programme of work. People with co-occurring severe mental health and alcohol/drug conditions have complex and multifaceted needs which require a comprehensive and long-term integrated approach. The shift to integrated health and social care is promising but will require local support (local expert leaders, network opportunities and clarity of role","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 67","pages":"1-100"},"PeriodicalIF":3.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11491988/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142463899","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}
Helen Snooks, Jonathan Benger, Fiona Bell, Sarah Black, Simon Dixon, Helena Emery, Bridie Angela Evans, Gordon Fuller, Rebecca Hoskins, Jane Hughes, Jenna Jones, Matthew Jones, Sasha Johnston, Jaqui Long, Chris Moore, Rakshita Parab, Richard Pilbery, Fiona C Sampson, Alan Watkins
<p><strong>Background: </strong>Opioids kill more people than any other drug. Naloxone is an opioid antagonist which can be distributed in take-home 'kits' for peer administration (take-home naloxone).</p><p><strong>Aim: </strong>To determine the feasibility of carrying out a definitive randomised controlled trial of take-home naloxone in emergency settings.</p><p><strong>Design: </strong>We used Welsh routine data (2015-21) to test the feasibility of developing a discriminant function to identify people at high risk of fatal opioid overdose. We carried out a cluster randomised controlled trial and qualitative study to examine experiences of service users and providers. We assessed feasibility of intervention and trial methods against predetermined progression criteria related to: site sign-up, staff trained, identification of eligible patients, proportion given kits, identification of people who died of opioid poisoning, data linkage and retrieval of outcomes.</p><p><strong>Setting: </strong>This study was carried out in the emergency environment; sites comprised an emergency department and associated ambulance service catchment area.</p><p><strong>Participants: </strong>At intervention sites, we invited emergency department clinicians and paramedics to participate. We recruited adult patients who arrived at the emergency department or were attended to by ambulance paramedics for a problem related to opioid use with capacity to consent to receiving the take-home naloxone and related training.</p><p><strong>Interventions: </strong>Usual care comprised basic life support plus naloxone by paramedics or emergency department staff. The take-home naloxone intervention was offered in addition to usual care, with guidance for recipients on basic life support, the importance of calling the emergency services, duration of effect, safety and legality of naloxone administration.</p><p><strong>Discriminant function: </strong>With low numbers of opioid-related deaths (1105/3,227,396) and a high proportion having no contact with health services in the year before death, the predictive link between death and opioid-related healthcare events was weak. Logistic regression models indicated we would need to monitor one-third of the population to capture 75% of the decedents from opioid overdose in 1-year follow-up.</p><p><strong>Randomised controlled trial: </strong>Four sites participated in the trial and 299 of 687 (44%) eligible clinical staff were trained. Sixty take-home naloxone kits were supplied to patients during 1-year recruitment. Eligible patients were not offered take-home naloxone kits 164 times: 'forgot' (<i>n</i> = 136); 'too busy' (<i>n</i> = 15); suspected intentional overdose (<i>n</i> = 3).</p><p><strong>Qualitative interviews: </strong>Service users had high levels of knowledge about take-home naloxone. They were supportive of the intervention but noted concerns about opioid withdrawal and resistance to attending hospital for an overdose. Servi
{"title":"Take-home naloxone in multicentre emergency settings: the TIME feasibility cluster RCT.","authors":"Helen Snooks, Jonathan Benger, Fiona Bell, Sarah Black, Simon Dixon, Helena Emery, Bridie Angela Evans, Gordon Fuller, Rebecca Hoskins, Jane Hughes, Jenna Jones, Matthew Jones, Sasha Johnston, Jaqui Long, Chris Moore, Rakshita Parab, Richard Pilbery, Fiona C Sampson, Alan Watkins","doi":"10.3310/YNRC8249","DOIUrl":"https://doi.org/10.3310/YNRC8249","url":null,"abstract":"<p><strong>Background: </strong>Opioids kill more people than any other drug. Naloxone is an opioid antagonist which can be distributed in take-home 'kits' for peer administration (take-home naloxone).</p><p><strong>Aim: </strong>To determine the feasibility of carrying out a definitive randomised controlled trial of take-home naloxone in emergency settings.</p><p><strong>Design: </strong>We used Welsh routine data (2015-21) to test the feasibility of developing a discriminant function to identify people at high risk of fatal opioid overdose. We carried out a cluster randomised controlled trial and qualitative study to examine experiences of service users and providers. We assessed feasibility of intervention and trial methods against predetermined progression criteria related to: site sign-up, staff trained, identification of eligible patients, proportion given kits, identification of people who died of opioid poisoning, data linkage and retrieval of outcomes.</p><p><strong>Setting: </strong>This study was carried out in the emergency environment; sites comprised an emergency department and associated ambulance service catchment area.</p><p><strong>Participants: </strong>At intervention sites, we invited emergency department clinicians and paramedics to participate. We recruited adult patients who arrived at the emergency department or were attended to by ambulance paramedics for a problem related to opioid use with capacity to consent to receiving the take-home naloxone and related training.</p><p><strong>Interventions: </strong>Usual care comprised basic life support plus naloxone by paramedics or emergency department staff. The take-home naloxone intervention was offered in addition to usual care, with guidance for recipients on basic life support, the importance of calling the emergency services, duration of effect, safety and legality of naloxone administration.</p><p><strong>Discriminant function: </strong>With low numbers of opioid-related deaths (1105/3,227,396) and a high proportion having no contact with health services in the year before death, the predictive link between death and opioid-related healthcare events was weak. Logistic regression models indicated we would need to monitor one-third of the population to capture 75% of the decedents from opioid overdose in 1-year follow-up.</p><p><strong>Randomised controlled trial: </strong>Four sites participated in the trial and 299 of 687 (44%) eligible clinical staff were trained. Sixty take-home naloxone kits were supplied to patients during 1-year recruitment. Eligible patients were not offered take-home naloxone kits 164 times: 'forgot' (<i>n</i> = 136); 'too busy' (<i>n</i> = 15); suspected intentional overdose (<i>n</i> = 3).</p><p><strong>Qualitative interviews: </strong>Service users had high levels of knowledge about take-home naloxone. They were supportive of the intervention but noted concerns about opioid withdrawal and resistance to attending hospital for an overdose. Servi","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 74","pages":"1-69"},"PeriodicalIF":3.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142564365","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}
Christine Hazelton, Alex Todhunter-Brown, Pauline Campbell, Katie Thomson, Donald J Nicolson, Kris McGill, Charlie Sy Chung, Liam Dorris, David C Gillespie, Susan M Hunter, Linda J Williams, Marian C Brady
<p><strong>Background: </strong>Stroke often affects recognition and interpretation of information from our senses, resulting in perceptual disorders. Evidence to inform treatment is unclear.</p><p><strong>Objective: </strong>To determine the breadth and effectiveness of interventions for stroke-related perceptual disorders and identify priority research questions.</p><p><strong>Methods: </strong>We undertook a scoping review and then Cochrane systematic review. Definitions, outcome prioritisation, data interpretation and research prioritisation were coproduced with people who had perceptual disorders post stroke and healthcare professionals. We systematically searched electronic databases (including MEDLINE, EMBASE, inception to August 2021) and grey literature. We included studies (any design) of interventions for people with hearing, smell, somatosensation, taste, touch or visual perception disorders following stroke. Abstracts and full texts were independently dual reviewed. Data were tabulated, synthesised narratively and mapped by availability, sense and interventions. Research quality was not evaluated. Our Cochrane review synthesised the randomised controlled trial data, evaluated risk of bias (including randomisation, blinding, reporting) and meta-analysed intervention comparisons (vs. controls or no treatment) using RevMan 5.4. We judged certainty of evidence using grading of recommendations, assessment, development and evaluation. Activities of daily living after treatment was our primary outcome. Extended activities of daily living, quality of life, mental health and psychological well-being perceptual functional and adverse event data were also extracted.</p><p><strong>Results: </strong>We included 80 studies (<i>n</i> = 893): case studies (36/80) and randomised controlled trials (22/80). No stroke survivor or family stakeholder involvement was reported. Studies addressed visual (42.5%, 34/80), somatosensation (35%, 28/80), auditory (8.7%, 7/80) and tactile (7.5%, 6/80) perceptual disorders; some studies focused on 'mixed perceptual disorders' (6.2%, 5/80 such as taste-smell disorders). We identified 93 pharmacological, non-invasive brain stimulation or rehabilitation (restitution, substitution, compensation or mixed) interventions. Details were limited. Studies commonly measured perceptual (75%, 60/80), motor-sensorimotor (40%, 32/80) activities of daily living (22.5%, 18/80) or sensory function (15%, 12/80) outcomes.</p><p><strong>Cochrane systematic review: </strong>We included 18 randomised controlled trials (<i>n</i> = 541) addressing tactile (3 randomised controlled trials; <i>n</i> = 70), somatosensory (7 randomised controlled trials; <i>n</i> = 196), visual (7 randomised controlled trials; <i>n</i> = 225) and mixed tactile-somatosensory (1 randomised controlled trial; <i>n</i> = 50) disorders. None addressed hearing, taste or smell disorders. One non-invasive brain stimulation, one compensation, 25 restitution and 4 mixed int
{"title":"Interventions for people with perceptual disorders after stroke: the PIONEER scoping review, Cochrane systematic review and priority setting project.","authors":"Christine Hazelton, Alex Todhunter-Brown, Pauline Campbell, Katie Thomson, Donald J Nicolson, Kris McGill, Charlie Sy Chung, Liam Dorris, David C Gillespie, Susan M Hunter, Linda J Williams, Marian C Brady","doi":"10.3310/WGJT3471","DOIUrl":"https://doi.org/10.3310/WGJT3471","url":null,"abstract":"<p><strong>Background: </strong>Stroke often affects recognition and interpretation of information from our senses, resulting in perceptual disorders. Evidence to inform treatment is unclear.</p><p><strong>Objective: </strong>To determine the breadth and effectiveness of interventions for stroke-related perceptual disorders and identify priority research questions.</p><p><strong>Methods: </strong>We undertook a scoping review and then Cochrane systematic review. Definitions, outcome prioritisation, data interpretation and research prioritisation were coproduced with people who had perceptual disorders post stroke and healthcare professionals. We systematically searched electronic databases (including MEDLINE, EMBASE, inception to August 2021) and grey literature. We included studies (any design) of interventions for people with hearing, smell, somatosensation, taste, touch or visual perception disorders following stroke. Abstracts and full texts were independently dual reviewed. Data were tabulated, synthesised narratively and mapped by availability, sense and interventions. Research quality was not evaluated. Our Cochrane review synthesised the randomised controlled trial data, evaluated risk of bias (including randomisation, blinding, reporting) and meta-analysed intervention comparisons (vs. controls or no treatment) using RevMan 5.4. We judged certainty of evidence using grading of recommendations, assessment, development and evaluation. Activities of daily living after treatment was our primary outcome. Extended activities of daily living, quality of life, mental health and psychological well-being perceptual functional and adverse event data were also extracted.</p><p><strong>Results: </strong>We included 80 studies (<i>n</i> = 893): case studies (36/80) and randomised controlled trials (22/80). No stroke survivor or family stakeholder involvement was reported. Studies addressed visual (42.5%, 34/80), somatosensation (35%, 28/80), auditory (8.7%, 7/80) and tactile (7.5%, 6/80) perceptual disorders; some studies focused on 'mixed perceptual disorders' (6.2%, 5/80 such as taste-smell disorders). We identified 93 pharmacological, non-invasive brain stimulation or rehabilitation (restitution, substitution, compensation or mixed) interventions. Details were limited. Studies commonly measured perceptual (75%, 60/80), motor-sensorimotor (40%, 32/80) activities of daily living (22.5%, 18/80) or sensory function (15%, 12/80) outcomes.</p><p><strong>Cochrane systematic review: </strong>We included 18 randomised controlled trials (<i>n</i> = 541) addressing tactile (3 randomised controlled trials; <i>n</i> = 70), somatosensory (7 randomised controlled trials; <i>n</i> = 196), visual (7 randomised controlled trials; <i>n</i> = 225) and mixed tactile-somatosensory (1 randomised controlled trial; <i>n</i> = 50) disorders. None addressed hearing, taste or smell disorders. One non-invasive brain stimulation, one compensation, 25 restitution and 4 mixed int","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 69","pages":"1-141"},"PeriodicalIF":3.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142562719","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}
Catherine M Sackley, Caroline Rick, Marian C Brady, Christopher Burton, Sue Jowett, Smitaa Patel, Rebecca Woolley, Patricia Masterson-Algar, Avril Nicoll, Christina H Smith, Zainab Abdali, Natalie Ives, Gillian Beaton, Sylvia Dickson, Ryan Ottridge, Helen Nankervis, Carl E Clarke
<p><strong>Background: </strong>Speech impairments are common with Parkinson's disease (reported prevalence 68%), increasing conversational demands, reliance on family and social withdrawal.</p><p><strong>Objective(s): </strong>The PD COMM trial compared the clinical and cost-effectiveness of two speech and language therapy approaches: Lee Silverman Voice Treatment LOUD and National Health Service speech and language therapy for the treatment of speech or voice problems in people with Parkinson's disease to no speech and language therapy (control) and against each other.</p><p><strong>Design: </strong>PD COMM is a phase III, multicentre, three-arm, unblinded, randomised controlled trial. Participants were randomised in a 1 : 1 : 1 ratio to control, National Health Service speech and language therapy or Lee Silverman Voice Treatment LOUD via a central computer-generated programme, using a minimisation procedure with a random element, to ensure allocation concealment. Mixed-methods process and health economic evaluations were conducted.</p><p><strong>Setting: </strong>United Kingdom outpatient and home settings.</p><p><strong>Participants: </strong>People with idiopathic Parkinson's disease, with self-reported or carer-reported speech or voice problems. We excluded people with dementia, laryngeal pathology and those within 24 months of previous speech and language therapy.</p><p><strong>Interventions: </strong>The Lee Silverman Voice Treatment LOUD intervention included maximum effort drills and high-effort speech production tasks delivered over four 50-minute therapist-led personalised sessions per week, for 4 weeks with prescribed daily home practice. National Health Service speech and language therapy content and dosage reflected local non-Lee Silverman Voice Treatment speech and language therapy practices, usually 1 hour, once weekly, for 6 weeks. Trained, experienced speech and language therapists or assistants provided interventions. The control was no speech and language therapy until the trial was completed.</p><p><strong>Main outcome measures: </strong>Primary outcome: Voice Handicap Index total score at 3 months. Secondary outcomes: Voice Handicap Index subscales, Parkinson's Disease Questionnaire-39; Questionnaire on Acquired Speech Disorders; EuroQol-5D-5L; ICEpop Capabilities Measure for Older Adults; Parkinson's Disease Questionnaire - Carers; resource utilisation; and adverse events. Assessments were completed pre-randomisation and at 3, 6 and 12 months post randomisation.</p><p><strong>Results: </strong>Three hundred and eighty-eight participants were randomised to Lee Silverman Voice Treatment LOUD (<i>n</i> = 130), National Health Service speech and language therapy (<i>n</i> = 129) and control (<i>n</i> = 129). The impact of voice problems at 3 months after randomisation was lower for Lee Silverman Voice Treatment LOUD participants than control [-8.0 (99% confidence interval: -13.3, -2.6); <i>p</i> = 0.001]. There was no evidence
{"title":"The effect of two speech and language approaches on speech problems in people with Parkinson's disease: the PD COMM RCT.","authors":"Catherine M Sackley, Caroline Rick, Marian C Brady, Christopher Burton, Sue Jowett, Smitaa Patel, Rebecca Woolley, Patricia Masterson-Algar, Avril Nicoll, Christina H Smith, Zainab Abdali, Natalie Ives, Gillian Beaton, Sylvia Dickson, Ryan Ottridge, Helen Nankervis, Carl E Clarke","doi":"10.3310/ADWP8001","DOIUrl":"10.3310/ADWP8001","url":null,"abstract":"<p><strong>Background: </strong>Speech impairments are common with Parkinson's disease (reported prevalence 68%), increasing conversational demands, reliance on family and social withdrawal.</p><p><strong>Objective(s): </strong>The PD COMM trial compared the clinical and cost-effectiveness of two speech and language therapy approaches: Lee Silverman Voice Treatment LOUD and National Health Service speech and language therapy for the treatment of speech or voice problems in people with Parkinson's disease to no speech and language therapy (control) and against each other.</p><p><strong>Design: </strong>PD COMM is a phase III, multicentre, three-arm, unblinded, randomised controlled trial. Participants were randomised in a 1 : 1 : 1 ratio to control, National Health Service speech and language therapy or Lee Silverman Voice Treatment LOUD via a central computer-generated programme, using a minimisation procedure with a random element, to ensure allocation concealment. Mixed-methods process and health economic evaluations were conducted.</p><p><strong>Setting: </strong>United Kingdom outpatient and home settings.</p><p><strong>Participants: </strong>People with idiopathic Parkinson's disease, with self-reported or carer-reported speech or voice problems. We excluded people with dementia, laryngeal pathology and those within 24 months of previous speech and language therapy.</p><p><strong>Interventions: </strong>The Lee Silverman Voice Treatment LOUD intervention included maximum effort drills and high-effort speech production tasks delivered over four 50-minute therapist-led personalised sessions per week, for 4 weeks with prescribed daily home practice. National Health Service speech and language therapy content and dosage reflected local non-Lee Silverman Voice Treatment speech and language therapy practices, usually 1 hour, once weekly, for 6 weeks. Trained, experienced speech and language therapists or assistants provided interventions. The control was no speech and language therapy until the trial was completed.</p><p><strong>Main outcome measures: </strong>Primary outcome: Voice Handicap Index total score at 3 months. Secondary outcomes: Voice Handicap Index subscales, Parkinson's Disease Questionnaire-39; Questionnaire on Acquired Speech Disorders; EuroQol-5D-5L; ICEpop Capabilities Measure for Older Adults; Parkinson's Disease Questionnaire - Carers; resource utilisation; and adverse events. Assessments were completed pre-randomisation and at 3, 6 and 12 months post randomisation.</p><p><strong>Results: </strong>Three hundred and eighty-eight participants were randomised to Lee Silverman Voice Treatment LOUD (<i>n</i> = 130), National Health Service speech and language therapy (<i>n</i> = 129) and control (<i>n</i> = 129). The impact of voice problems at 3 months after randomisation was lower for Lee Silverman Voice Treatment LOUD participants than control [-8.0 (99% confidence interval: -13.3, -2.6); <i>p</i> = 0.001]. There was no evidence","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 58","pages":"1-141"},"PeriodicalIF":3.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11474952/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142371695","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}
Hema Mistry, Seyran Naghdi, Anna Brown, Sophie Rees, Jason Madan, Amy Grove, Saval Khanal, Callum Duncan, Manjit Matharu, Andrew Cooklin, Aiva Aksentyte, Natasha Davies, Martin Underwood
<p><strong>Background: </strong>Chronic migraine is a disabling condition, affecting 2-4% of adults globally. With the introduction of expensive calcitonin gene-related peptide monoclonal antibodies, it is timely to compare the clinical effectiveness and cost-effectiveness of preventive drugs for chronic migraine.</p><p><strong>Objective: </strong>To assess the clinical effectiveness and cost-effectiveness of medications used for chronic migraine through systematic reviews and economic modelling.</p><p><strong>Eligibility criteria: </strong>Randomised controlled trials of drug treatments for efficacy with > 100 participants with chronic migraine per arm; for adverse events > 100 participants with episodic or chronic migraine per arm. Previous economic analyses of preventive drugs for chronic migraine.</p><p><strong>Data sources: </strong>Eight databases.</p><p><strong>Reviews methods: </strong>Systematic reviews, network meta-analysis and economic modelling.</p><p><strong>Outcomes: </strong>Monthly headache days, monthly migraine days, headache-related quality of life, cost-effectiveness.</p><p><strong>Results: </strong>We found 51 individual articles, reporting 11 randomised controlled trials, testing 6 drugs (topiramate, Botox, eptinezumab, erenumab, fremanezumab, galcanezumab), versus placebo, on 7352 adults with chronic migraine. Calcitonin gene-related peptide monoclonal antibodies, Botox and topiramate reduced headache/migraine days by 2.0-2.5, just under two, or by less than 1.5 days per month, respectively. In the network meta-analysis, eptinezumab 300 mg and fremanezumab monthly ranked in first place in both monthly headache day and monthly migraine day analyses. The calcitonin gene-related peptide monoclonal antibodies were consistently the best choices for headache/migraine days and headache-related quality of life. Topiramate was very unlikely to be the best choice for headache/migraine days and headache-related quality of life when compared to calcitonin gene-related peptide monoclonal antibodies or Botox. We found no trials of the commonly used drugs, such as propranolol or amitriptyline, to include in the analysis. The adverse events review included 40 randomised controlled trials with 25,891 participants; 3 additional drugs, amitriptyline, atogepant and rimegepant, were included. There were very few serious adverse events - none of which were linked to the use of these medications. Adverse events were common. Most people using some calcitonin gene-related peptide monoclonal antibodies reported injection site issues; and people using topiramate or amitriptyline had nervous system or gastrointestinal issues. The cost-effectiveness review identified 16 studies evaluating chronic migraine medications in adults. The newer, injected drugs are more costly than the oral preventatives, but they were cost-effective. Our economic model showed that topiramate was the least costly option and had the fewest quality-adjusted life-year gains, whe
{"title":"Preventive drug treatments for adults with chronic migraine: a systematic review with economic modelling.","authors":"Hema Mistry, Seyran Naghdi, Anna Brown, Sophie Rees, Jason Madan, Amy Grove, Saval Khanal, Callum Duncan, Manjit Matharu, Andrew Cooklin, Aiva Aksentyte, Natasha Davies, Martin Underwood","doi":"10.3310/AYWA5297","DOIUrl":"10.3310/AYWA5297","url":null,"abstract":"<p><strong>Background: </strong>Chronic migraine is a disabling condition, affecting 2-4% of adults globally. With the introduction of expensive calcitonin gene-related peptide monoclonal antibodies, it is timely to compare the clinical effectiveness and cost-effectiveness of preventive drugs for chronic migraine.</p><p><strong>Objective: </strong>To assess the clinical effectiveness and cost-effectiveness of medications used for chronic migraine through systematic reviews and economic modelling.</p><p><strong>Eligibility criteria: </strong>Randomised controlled trials of drug treatments for efficacy with > 100 participants with chronic migraine per arm; for adverse events > 100 participants with episodic or chronic migraine per arm. Previous economic analyses of preventive drugs for chronic migraine.</p><p><strong>Data sources: </strong>Eight databases.</p><p><strong>Reviews methods: </strong>Systematic reviews, network meta-analysis and economic modelling.</p><p><strong>Outcomes: </strong>Monthly headache days, monthly migraine days, headache-related quality of life, cost-effectiveness.</p><p><strong>Results: </strong>We found 51 individual articles, reporting 11 randomised controlled trials, testing 6 drugs (topiramate, Botox, eptinezumab, erenumab, fremanezumab, galcanezumab), versus placebo, on 7352 adults with chronic migraine. Calcitonin gene-related peptide monoclonal antibodies, Botox and topiramate reduced headache/migraine days by 2.0-2.5, just under two, or by less than 1.5 days per month, respectively. In the network meta-analysis, eptinezumab 300 mg and fremanezumab monthly ranked in first place in both monthly headache day and monthly migraine day analyses. The calcitonin gene-related peptide monoclonal antibodies were consistently the best choices for headache/migraine days and headache-related quality of life. Topiramate was very unlikely to be the best choice for headache/migraine days and headache-related quality of life when compared to calcitonin gene-related peptide monoclonal antibodies or Botox. We found no trials of the commonly used drugs, such as propranolol or amitriptyline, to include in the analysis. The adverse events review included 40 randomised controlled trials with 25,891 participants; 3 additional drugs, amitriptyline, atogepant and rimegepant, were included. There were very few serious adverse events - none of which were linked to the use of these medications. Adverse events were common. Most people using some calcitonin gene-related peptide monoclonal antibodies reported injection site issues; and people using topiramate or amitriptyline had nervous system or gastrointestinal issues. The cost-effectiveness review identified 16 studies evaluating chronic migraine medications in adults. The newer, injected drugs are more costly than the oral preventatives, but they were cost-effective. Our economic model showed that topiramate was the least costly option and had the fewest quality-adjusted life-year gains, whe","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 63","pages":"1-329"},"PeriodicalIF":3.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11474956/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142371698","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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