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Cessation of smoking in people attending UK emergency departments: the COSTED RCT with economic and process evaluation. 在英国急诊科就诊的人戒烟：经济和过程评估的成本计算随机对照试验
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/JHFR0841
Ian Pope, Lucy V Clark, Allan Clark, Emma Ward, Pippa Belderson, Susan Stirling, Steve Parrott, Jinshuo Li, Timothy Coats, Linda Bauld, Richard Holland, Sarah Gentry, Sanjay Agrawal, Benjamin M Bloom, Adrian Boyle, Alasdair Gray, M Geraint Morris, Caitlin Notley
<p><strong>Background: </strong>The emergency department represents a potentially valuable opportunity to support smoking cessation. Evidence is lacking around the use of e-cigarettes in opportunistic settings like the emergency department.</p><p><strong>Objective: </strong>To undertake a randomised controlled trial in people who smoke attending United Kingdom emergency departments, testing a brief intervention which included provision of an e-cigarette versus signposting to smoking cessation services, assessing smoking abstinence.</p><p><strong>Design: </strong>A two-arm pragmatic, multicentre, parallel-group, individually randomised, controlled superiority trial with an internal pilot, economic evaluation and mixed-methods process evaluation.</p><p><strong>Setting: </strong>Six emergency departments across England and Scotland.</p><p><strong>Participants: </strong>Adults who smoked daily, who were attending the emergency department for medical treatment or accompanying someone attending for medical treatment, were invited to participate. People were excluded if they had an expired carbon monoxide of < 8 parts per million, required immediate medical treatment, were in police custody, had a known allergy to nicotine, were daily e-cigarette users, were considered not to have capacity to consent or had already taken part in the trial.</p><p><strong>Intervention: </strong>Brief stop smoking advice, e-cigarette starter kit and referral to stop smoking services.</p><p><strong>Main outcome measures: </strong>The primary outcome was biochemically validated sustained abstinence at 6 months. Those lost to follow-up, or not providing biochemical verification, were considered not to be abstinent. Secondary outcomes were: self-reported 7-day smoking abstinence, number of quit attempts, number of cigarettes per day, nicotine dependence and incidence of self-reported dry cough or mouth or throat irritation.</p><p><strong>Results: </strong>At 6 months, of 972 participants randomised, biochemically verified smoking abstinence was 7.2% in the intervention group and 4.1% in the control group (percentage difference = 3.3%) (95% confidence interval 0.3 to 6.3; <i>p</i> = 0.032) [relative risk 1.76 (95% confidence interval 1.03 to 3.01)]. Self-reported 7-day abstinence at 6 months was 23.3% in the intervention group and 12.9% in the control group (percentage difference = 10.6%) (95% confidence interval 5.86 to 15.41; <i>p</i> < 0.001) [relative risk 1.80 (95% confidence interval 1.36 to 2.38)]. Daily e-cigarette use was 39.4% in the intervention group and 17.5% in the control group at 6 months. No serious adverse events related to taking part in the trial were reported. The economic evaluation found the intervention was likely to be cost-effective, judged by the National Institute for Health and Care Excellence threshold. The process evaluation found the intervention to be acceptable to both staff delivering it and participants receiving it. The brief nature of the i
背景：急诊科是支持戒烟的潜在宝贵机会。在急诊等机会性环境中使用电子烟的证据不足。目的：对在英国急诊科就诊的吸烟者进行一项随机对照试验，测试一种简短的干预措施，包括提供电子烟与戒烟服务的指示牌，评估戒烟情况。设计：两臂实用、多中心、平行组、单独随机、控制优势试验，内部试点，经济评估和混合方法过程评估。环境：英格兰和苏格兰的六个急诊科。参与者：每天吸烟的成年人，在急诊室接受医疗治疗或陪同他人接受医疗治疗，被邀请参加。如果人们有过期的一氧化碳干预：简短的戒烟建议，电子烟入门工具包和转介戒烟服务，则被排除在外。主要结局指标：主要结局是6个月时经生化验证的持续禁欲。那些失去随访或没有提供生化验证的人被认为不是禁欲。次要结果是：自我报告的7天戒烟情况、戒烟尝试次数、每天吸烟次数、尼古丁依赖以及自我报告的干咳、口腔或喉咙发炎的发生率。结果：在6个月时，972名随机受试者中，经生化验证的戒烟率在干预组为7.2%，在对照组为4.1%（百分比差异= 3.3%）(95%置信区间0.3 ~ 6.3；P = 0.032)[相对危险度1.76(95%可信区间1.03 ~ 3.01)]。干预组6个月时自我报告的7天戒断率为23.3%，对照组为12.9%（百分比差异= 10.6%）(95%置信区间5.86 ~ 15.41；p限制：无法使参与者或研究人员失明，由于所招募人群的性质，生化验证水平相对较低，并且对照组的人没有接受常规护理。结论：机会性戒烟干预包括简短的建议、电子烟入门工具包和转诊戒烟服务，对持续戒烟有效，几乎没有不良事件报告。未来的工作：未来的工作将包括在急诊科测试其他行为改变干预措施，并将戒烟试验在急诊科的干预措施适应其他环境。资助：本摘要介绍了由国家卫生与保健研究所（NIHR）卫生技术评估计划资助的独立研究，奖励号为NIHR129438。
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引用次数: 0
Feasibility, acceptability and appropriateness of laparoscopic versus abdominal hysterectomy for women and healthcare professionals: the LAVA trial qualitative process evaluation. 妇女和医疗保健专业人员腹腔镜子宫切除术与腹式子宫切除术的可行性、可接受性和适宜性：LAVA试验定性过程评价
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/GJTC1325
Lynsay Matthews, T Justin Clark, Sheriden Bevan, Lee Middleton, Lina Antoun, Paul Smith, Ertan Saridogan, Rebecca Woolley, Monique Morgan, Laura L Jones
<p><strong>Background: </strong>Laparoscopic hysterectomies performed for benign gynaecological conditions are increasing. However, there is a lack of up-to-date evidence on their surgical outcomes when compared with abdominal hysterectomy. The LAparoscopic Versus Abdominal hysterectomy trial aimed to address this gap. A qualitative process evaluation was embedded within the pilot phase of the LAparoscopic Versus Abdominal hysterectomy trial.</p><p><strong>Objective: </strong>To explore the feasibility, acceptability and appropriateness of LAparoscopic Versus Abdominal hysterectomy for women and healthcare professionals.</p><p><strong>Design and methods: </strong>A qualitative process evaluation using semistructured interviews informed by the Medical Research Council/National Institute for Health and Care Research updated Framework for Developing and Evaluating Complex Interventions. Interviews were thematically analysed to inform the development of a LAparoscopic Versus Abdominal hysterectomy trial programme theory (used to demonstrate how an intervention is expected to lead to its effects, under what conditions and for which stakeholders).</p><p><strong>Setting and participants: </strong>Eligible women and healthcare professionals (gynaecologists, research nurses and research midwives) from participating clinical sites in National Health Service England.</p><p><strong>Main outcome measures: </strong>Insight on the feasibility,acceptability and appropriateness of LAparoscopic Versus Abdominal hysterectomy related to the: (1) environment, (2) patient and (3) the healthcare professionals.</p><p><strong>Results: </strong>Eleven women and 7 healthcare professionals (6 research nurses and 1 consultant gynaecologist) were interviewed. Four themes were interpreted. Theme 1 identified <b>decision-making processes</b> for LAparoscopic Versus Abdominal hysterectomy participation. <i>Conditional altruism</i> motivated women to participate, alongside the 'relief' of being offered a hysterectomy. The decision to decline participation was influenced by surgical preference and beliefs of laparoscopy having a faster recovery rate. Theme 2 highlighted <b>surgical preferences</b>, with women's preferences being influenced by their previous experiences of surgery or perceived recovery times and family/friends. All healthcare professionals demonstrated <i>community equipoise</i> but did observe that 'younger surgeons' may prefer laparoscopic surgery based on their contemporary training. Theme 3 identified <b>attitudes towards the LAparoscopic Versus Abdominal hysterectomy trial</b>, with women and healthcare professionals reporting positively about LAparoscopic Versus Abdominal hysterectomy's feasibility, acceptability and appropriateness in terms of burden, information and understanding of the study. Theme 4 identified the <b>facilitators and barriers</b> for LAparoscopic Versus Abdominal hysterectomy participation. Facilitators included the key role of the resear
背景：腹腔镜子宫切除术在妇科良性疾病中的应用越来越多。然而，与腹式子宫切除术相比，缺乏最新的手术结果证据。腹腔镜与腹部子宫切除术试验旨在解决这一差距。在腹腔镜子宫切除术与腹式子宫切除术试验的试点阶段进行了定性过程评估。目的：探讨腹腔镜子宫切除术与腹式子宫切除术的可行性、可接受性和适宜性。设计和方法：采用医学研究委员会/国家卫生和保健研究所更新的制定和评估复杂干预措施框架通知的半结构化访谈进行定性过程评估。对访谈进行主题分析，为腹腔镜与腹部子宫切除术试验计划理论的发展提供信息（用于证明干预措施如何预期产生效果，在什么条件下以及针对哪些利益相关者）。环境和参与者：符合条件的妇女和医疗保健专业人员（妇科医生、研究护士和研究助产士），来自英格兰国家卫生服务中心参与的临床站点。主要观察指标：腹腔镜子宫切除术与腹式子宫切除术的可行性、可接受性和适宜性与：(1)环境、(2)患者和(3)医护人员相关。结果：11名妇女和7名保健专业人员（6名研究护士和1名妇科咨询医生）接受了采访。阐释了四个主题。主题1确定参与腹腔镜子宫切除术与腹式子宫切除术的决策过程。有条件的利他主义激励着女性参与其中，同时还有被提供子宫切除术的“解脱”。拒绝参与的决定受手术偏好和相信腹腔镜有更快的恢复率的影响。主题2强调了手术偏好，女性的偏好受到她们以前的手术经历或预期的恢复时间以及家人/朋友的影响。所有医疗保健专业人员都表现出社区平等，但确实观察到“年轻外科医生”可能更喜欢基于他们当代培训的腹腔镜手术。主题3确定了对腹腔镜与腹式子宫切除术试验的态度，妇女和医疗保健专业人员在负担、信息和对研究的理解方面积极报告腹腔镜与腹式子宫切除术的可行性、可接受性和适宜性。主题4确定了腹腔镜子宫切除术与腹部子宫切除术的促进因素和障碍。促进者包括研究护士的关键作用，以及在康复期间获得个人社会支持的妇女。电话咨询可能是一个障碍，妇女和保健专业人员更喜欢面对面的讨论。这些发现完善了腹腔镜与腹部子宫切除术方案理论，确定了与环境、患者和医疗保健专业人员相关的因素的相互作用。局限性：大多数来自女性的见解来自一个站点（72.7%），大多数医疗保健专业人员的见解来自研究护士（85.7%）。只有说英语的参与者被招募到腹腔镜与腹部子宫切除术。结论：总的来说，腹腔镜子宫切除术和腹式子宫切除术对于女性和医疗保健专业人员来说是可以接受的。然而，由于COVID-19大流行的负面影响和缺乏医疗保健专业平衡（这些发现已在之前发表），该试验提前结束。定性过程评价强调了未来试验需要考虑的其他因素，包括对妇女决策的影响以及解决患者和保健专业人员平等问题的挑战。未来的工作：腹腔镜和腹式子宫切除术的结果的比较仍需要在大规模随机对照试验中进行探索。需要从拒绝参与的妇女和缺乏平等的保健专业人员那里获得进一步的定性见解。资助：本文介绍了由国家卫生与保健研究所（NIHR）卫生技术评估计划资助的独立研究，奖励号为NIHR128991。
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引用次数: 0
Extracorporeal carbon dioxide removal for the treatment of acute hypoxaemic respiratory failure: the REST RCT. 体外二氧化碳去除治疗急性低氧性呼吸衰竭：REST RCT。
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/GJDM0320
James J McNamee, Ashley Agus, Andrew J Boyle, Colette Jackson, Cliona McDowell, Jeanette Haglund, Danny F McAuley
<p><strong>Background: </strong>In patients who require mechanical ventilation for acute hypoxaemic respiratory failure, further reduction in tidal volumes, compared with conventional low tidal volume ventilation, may improve outcomes.</p><p><strong>Objective: </strong>To determine whether using extracorporeal carbon dioxide removal improves outcomes in patients with acute hypoxaemic respiratory failure and is cost-effective.</p><p><strong>Design: </strong>A multicentre, randomised, allocation-concealed, open-label, pragmatic clinical trial.</p><p><strong>Setting: </strong>Fifty-one intensive care units across the United Kingdom.</p><p><strong>Participants: </strong>Four hundred and twelve adult patients receiving mechanical ventilation for acute hypoxaemic respiratory failure, of a planned sample size of 1120.</p><p><strong>Interventions: </strong>Lower tidal volume ventilation facilitated by extracorporeal carbon dioxide removal for at least 48 hours (<i>n</i> = 202) or standard care with conventional low tidal volume ventilation (<i>n</i> = 210).</p><p><strong>Main outcome measures: </strong>All-cause mortality 90 days. Secondary outcomes included ventilator-free days; adverse events; extracorporeal membrane oxygenation use; long-term mortality; health-related quality of life; health service costs; long-term respiratory morbidity.</p><p><strong>Results: </strong>The trial was stopped early because of futility and feasibility. The 90-day mortality rate was 41.5% in the extracorporeal carbon dioxide removal group versus 39.5% in the standard care group (risk ratio 1.05, 95% confidence interval 0.83 to 1.33; difference 2.0%, 95% confidence interval  - 7.6% to 11.5%; <i>p</i> = 0.68). There were significantly fewer mean ventilator-free days in the extracorporeal carbon dioxide removal group compared with the standard care group (7.1, 95% confidence interval 5.9 to 8.3) versus (9.2, 95% confidence interval 7.9 to 10.4) days; mean difference, -2.1 (95% confidence interval -3.8 to -0.3; <i>p</i> = 0.02). Serious adverse events were reported for 62 patients (31%) in extracorporeal carbon dioxide removal group and 18 (9%) in the standard care group, including intracranial haemorrhage in 9 patients (4.5%) versus 0 (0%) and bleeding at other sites in 6 (3.0%) versus 1 (0.5%) in the extracorporeal carbon dioxide removal group versus the control group. Two-year mortality data were available for 95% of patients. There was no difference in the time to death between groups (hazard ratio 1.08, 95% confidence interval 0.81 to 1.44; log-rank test <i>p</i> = 0.61). There was no difference in long-term outcomes between groups. There was no difference in quality-adjusted life-years at 12 months (mean difference -0.01, 95% confidence interval -0.06 to 0.05). Total 12-month costs were statistically significantly higher in the extracorporeal carbon dioxide removal group (mean difference £7668.76, 95% confidence interval £159.75 to £15,177.77). Secondary analyses indic
背景：在急性低氧性呼吸衰竭需要机械通气的患者中，与传统的低潮气量通气相比，进一步降低潮气量可能改善预后。目的：确定体外二氧化碳清除术是否能改善急性低氧性呼吸衰竭患者的预后，是否具有成本效益。设计：一项多中心、随机、分配隐蔽、开放标签、实用的临床试验。环境：全英国共有51个重症监护病房。参与者：1200名因急性低氧性呼吸衰竭接受机械通气的成年患者，计划样本量为1120人。干预措施：低潮气量通气通过体外二氧化碳清除至少48小时（n = 202）或标准护理常规低潮气量通气（n = 210）。主要结局指标：90天全因死亡率。次要结局包括无呼吸机天数；不良事件;体外膜氧合应用；长期死亡率;与健康有关的生活质量；保健服务费用；长期呼吸道疾病。结果：试验因无效和可行性而提前终止。体外二氧化碳去除组90天死亡率为41.5%，标准护理组为39.5%(风险比1.05,95%可信区间0.83 ~ 1.33；差异2.0%，95%置信区间- 7.6%至11.5%；p = 0.68)。体外二氧化碳去除组的平均无呼吸机天数明显少于标准护理组（7.1,95%可信区间5.9至8.3）和（9.2,95%可信区间7.9至10.4）天；平均差值为-2.1(95%置信区间为-3.8 ~ -0.3；p = 0.02)。体外二氧化碳去除组报告了62例（31%）患者的严重不良事件，标准护理组报告了18例（9%）患者的严重不良事件，包括9例（4.5%）患者颅内出血，0例（0%），6例（3.0%）患者其他部位出血，1例（0.5%）患者体外二氧化碳去除组与对照组。95%的患者可获得两年死亡率数据。两组患者死亡时间差异无统计学意义(风险比1.08,95%可信区间0.81 ~ 1.44；Log-rank检验p = 0.61)。两组之间的长期结果没有差异。12个月时质量调整生命年无差异（平均差异为-0.01,95%可信区间为-0.06 ~ 0.05）。体外二氧化碳去除组12个月的总费用在统计学上显著高于对照组（平均差值为7668.76英镑，95%可信区间为159.75英镑至15177.77英镑）。二次分析表明，根据患者的生理特征，治疗效果可能存在异质性。一项系统综述支持了这些发现。局限性：只有6%的筛查患者被纳入研究；在研究开始之前，大多数地点对干预措施一无所知；由于这是一项实用的试验，护理的其他方面在每个组中都没有标准化；该试验可能没有足够的能力来发现临床上重要的差异，因为试验提前停止了；不可能对临床医生或患者进行盲测。结论：没有发现短期或长期的益处，并且该装置与较高的成本和潜在的显著并发症相关。在未来的临床试验之外，我们不建议在治疗低氧性呼吸衰竭患者的标准护理之外使用该设备。未来的工作：未来的研究可以进一步探索接受更大剂量体外二氧化碳去除的不同患者群体是否会受益，使用核心结果集并收集更广泛的长期结果，并考虑在恢复能力后尽快测量患者与健康相关的生活质量。资助：本摘要介绍了由国家卫生与保健研究所（NIHR）卫生技术评估方案资助的独立研究，奖励号为13/143/02。
{"title":"Extracorporeal carbon dioxide removal for the treatment of acute hypoxaemic respiratory failure: the REST RCT.","authors":"James J McNamee, Ashley Agus, Andrew J Boyle, Colette Jackson, Cliona McDowell, Jeanette Haglund, Danny F McAuley","doi":"10.3310/GJDM0320","DOIUrl":"10.3310/GJDM0320","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;In patients who require mechanical ventilation for acute hypoxaemic respiratory failure, further reduction in tidal volumes, compared with conventional low tidal volume ventilation, may improve outcomes.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To determine whether using extracorporeal carbon dioxide removal improves outcomes in patients with acute hypoxaemic respiratory failure and is cost-effective.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;A multicentre, randomised, allocation-concealed, open-label, pragmatic clinical trial.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Fifty-one intensive care units across the United Kingdom.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Four hundred and twelve adult patients receiving mechanical ventilation for acute hypoxaemic respiratory failure, of a planned sample size of 1120.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Lower tidal volume ventilation facilitated by extracorporeal carbon dioxide removal for at least 48 hours (&lt;i&gt;n&lt;/i&gt; = 202) or standard care with conventional low tidal volume ventilation (&lt;i&gt;n&lt;/i&gt; = 210).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;All-cause mortality 90 days. Secondary outcomes included ventilator-free days; adverse events; extracorporeal membrane oxygenation use; long-term mortality; health-related quality of life; health service costs; long-term respiratory morbidity.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The trial was stopped early because of futility and feasibility. The 90-day mortality rate was 41.5% in the extracorporeal carbon dioxide removal group versus 39.5% in the standard care group (risk ratio 1.05, 95% confidence interval 0.83 to 1.33; difference 2.0%, 95% confidence interval  - 7.6% to 11.5%; &lt;i&gt;p&lt;/i&gt; = 0.68). There were significantly fewer mean ventilator-free days in the extracorporeal carbon dioxide removal group compared with the standard care group (7.1, 95% confidence interval 5.9 to 8.3) versus (9.2, 95% confidence interval 7.9 to 10.4) days; mean difference, -2.1 (95% confidence interval -3.8 to -0.3; &lt;i&gt;p&lt;/i&gt; = 0.02). Serious adverse events were reported for 62 patients (31%) in extracorporeal carbon dioxide removal group and 18 (9%) in the standard care group, including intracranial haemorrhage in 9 patients (4.5%) versus 0 (0%) and bleeding at other sites in 6 (3.0%) versus 1 (0.5%) in the extracorporeal carbon dioxide removal group versus the control group. Two-year mortality data were available for 95% of patients. There was no difference in the time to death between groups (hazard ratio 1.08, 95% confidence interval 0.81 to 1.44; log-rank test &lt;i&gt;p&lt;/i&gt; = 0.61). There was no difference in long-term outcomes between groups. There was no difference in quality-adjusted life-years at 12 months (mean difference -0.01, 95% confidence interval -0.06 to 0.05). Total 12-month costs were statistically significantly higher in the extracorporeal carbon dioxide removal group (mean difference £7668.76, 95% confidence interval £159.75 to £15,177.77). Secondary analyses indic","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 33","pages":"1-16"},"PeriodicalIF":4.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376005/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144775303","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}
引用次数: 0
The risks, benefits, and resource implications of different diets in gastrostomy-fed children: The YourTube mixed method study. 对胃造口喂养儿童不同饮食的风险、益处和资源含义：YourTube混合方法研究。
IF 3.5 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/RRREF7741
Lorna Fraser, Andre Bedendo, Mark O'Neill, Johanna Taylor, Julia Hackett, Karen Horridge, Janet Cade, Gerry Richardson, Thai Han Phung, Bryony Beresford, Alison McCarter, Catherine Hewitt

Background: Many children receive some or all their nutritional intake via a gastrostomy. More parents are using home-blended meals to feed their children, reporting beneficial effects, such as improved gastro-oesophageal reflux and less distress.

Aim: To compare safety, outcomes and resource use of those on home-blended diets compared to formula diets.

Methods: A mixed-methods study of gastrostomy-fed children.

Workstream 1: Qualitative study involving semistructured interviews with parents (n ≈ 20) and young people (n ≈ 2) and focus groups with health professionals (n ≈ 41).

Workstream 2: Cohort study; data were collected on 180 children at months 0, 12 and 18 from parents and clinicians using standardised measures. Data included gastrointestinal symptoms, quality of life, sleep (child and parent), dietary intake, anthropometry, healthcare usage, safety outcomes and resource use. Outcomes were compared using propensity scored weighted multiple regression analyses.

Results: workstream 1: Participants believed the type of diet would most likely affect gastrointestinal symptoms, time spent on feeding, sleep and physical health.

Workstream 2: Baseline: Children receiving a home-blended diet and those receiving a formula diet were similar in terms of diagnoses and age, but those receiving a home-blended diet were more likely to live in areas of lower deprivation and their parents had higher levels of education. They also had a higher dietary fibre intake and demonstrated significantly better gastrointestinal symptom scores compared to those receiving a formula diet (beta 13.8, p < 0.001). The number of gut infections and tube blockages were similar between the two groups, but stoma site infections were lower in those receiving a home-blended diet. Follow-up: There were 134 (74%) and 105 (58%) children who provided follow-up data at 12 and 18 months. Gastrointestinal symptoms were lower at all time points in the home-blended diet group, but there was no difference in change over time within or between the groups. The nutritional intake of those on a home-blended diet had higher calories/kg and fibre, and both home-blended and formula-fed children have values above the Dietary Reference Values for most micronutrients. Safety outcomes were similar between groups and over time. Total costs to the statutory sector were higher among children who were formula fed, but costs of purchasing special equipment for home-blended food and the total time spent on child care were higher for families with home-blended diet.

Conclusion: Findings show that home-blended diets for children who are gastrostomy fed should be seen as a safe alternative to formula feeding for children unless there is a clinical contraindication.

Limitations:

背景：许多儿童通过胃造口术获得部分或全部营养摄入。越来越多的父母使用家庭混合餐来喂养他们的孩子，报告了有益的效果，例如改善胃食管反流和减轻痛苦。目的：比较家庭混合饮食与配方饮食的安全性、结果和资源利用。方法：采用混合方法对胃造口喂养儿童进行研究。工作流程1：定性研究，包括与父母（n≈20）和年轻人（n≈2）以及与卫生专业人员（n≈41）的焦点小组进行半结构化访谈。工作流程2：队列研究；使用标准化测量方法从父母和临床医生处收集了180名儿童0、12和18个月的数据。数据包括胃肠道症状、生活质量、睡眠（儿童和父母）、饮食摄入、人体测量、医疗保健使用、安全结果和资源使用。结果采用倾向评分加权多元回归分析进行比较。结果：工作流程1：参与者认为饮食类型最有可能影响胃肠道症状、进食时间、睡眠和身体健康。工作流程2：基线：接受家庭混合饮食的儿童和接受配方饮食的儿童在诊断和年龄方面相似，但接受家庭混合饮食的儿童更有可能生活在贫困程度较低的地区，其父母的教育水平较高。与接受配方饮食的儿童相比，他们也有更高的膳食纤维摄入量，并表现出明显更好的胃肠道症状评分（β值13.8,p）。随访：有134（74%）和105（58%）名儿童在12个月和18个月时提供了随访数据。家庭混合饮食组的胃肠道症状在所有时间点都较低，但在组内或组间没有随时间变化的差异。家庭混合饮食的儿童的营养摄入量每公斤热量和纤维含量较高，家庭混合饮食和配方奶粉喂养的儿童的大多数微量营养素含量都高于膳食参考值。各组之间和不同时间的安全性结果相似。使用配方奶粉喂养的儿童的法定部门总成本较高，但购买家庭混合食品专用设备的成本和用于儿童保育的总时间在使用家庭混合饮食的家庭中较高。结论：研究结果表明，除非有临床禁忌症，否则对胃造口喂养儿童的家庭混合饮食应被视为儿童配方喂养的安全替代品。局限性：工作流程1中孩子的目标样本没有实现。观察性研究设计意味着未测量的混杂可能仍然是一个问题。在这个队列中，孩子们在不同的时间里一直在吃他们的家庭混合饮食。缺乏关于残疾儿童营养和人体测量数据的良好参考数据确实阻碍了对营养充足性的进一步解释。未来的工作：未来的研究：家庭混合饮食对胃造口喂养儿童肠道微生物群的影响以及获得机会的平等。儿童的胃造口术生活经历、营养需求和生活质量也应优先考虑。资助：本摘要介绍了由国家卫生与保健研究所（NIHR）卫生技术评估方案资助的独立研究，奖励号为17/76/06。
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引用次数: 0
BioImpedance Spectroscopy to maintain Renal Output: the BISTRO randomised controlled trial. 生物阻抗谱维持肾输出量：BISTRO随机对照试验。
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/RHON2378
Simon J Davies, David Coyle, Elizabeth Lindley, David Keane, John Belcher, Fergus Caskey, Indranil Dasgupta, Andrew Davenport, Ken Farrington, Sandip Mitra, Paula Ormandy, Martin Wilkie, Jamie MacDonald, Mandana Zanganeh, Lazaros Andronis, Ivonne Solis-Trapala, Julius Sim

Background: Fluid removal is a key component of dialysis treatment but, if excessive, can result in a faster decline in residual kidney function. Prescribing the optimal removal of fluid on dialysis to avoid this is therefore important. Bioimpedance spectroscopy, a bedside device that estimates tissue hydration, might improve this prescription, so reducing the rate of decline in kidney function and improving patient outcomes. We wished to establish the efficacy and cost-effectiveness of bioimpedance in pursuing this treatment strategy.

Methods: We undertook a multicentre, open-label, parallel, individually randomised controlled trial in incident haemodialysis patients, with clinicians and patients blinded to bioimpedance readings in the control group. Eligible patients had a urine output of > 500 ml/day or a glomerular filtration rate > 3 ml/minute/1.73 m2. Randomisation was 1 : 1 using a concealed automated computer-generated allocation system stratified by centre. Clinical assessments were made monthly for 3 months and then every 3 months for up to 24 months using a standardised proforma in both groups, supplemented in the intervention group by the bioimpedance estimate of the normally hydrated weight. The primary outcome was time to anuria; secondary outcomes were rate in decline of residual kidney function, blood pressure, dialysis-related symptoms (Integrated Palliative Care Outcome Scale-Renal), quality of life (EuroQol) and incremental cost per additional quality-adjusted life-year gained.

Results: Four hundred and thirty-nine patients were recruited and analysed from 34 United Kingdom centres. There were no between-group differences in cause-specific hazard rates of anuria, 0.751 (95% confidence interval 0.459 to 1.229) or subdistribution hazard rates 0.742 (95% confidence interval 0.453 to 1.215). Kidney function decline was slower than anticipated, pooled linear rates in year 1: -0.178 (95% confidence interval -0.196 to -0.159) ml/minute/1.73 m2/month; year 2: -0.061 (95% confidence interval -0.086 to -0.036) ml/minute/1.73 m2/month. Longitudinal blood pressure, symptoms and patient-reported outcomes did not differ by group. The intervention was associated with £382 (95% confidence interval -£3319 to £2556) lower average cost per patient (price year 2020) and 0.043 (95% confidence interval -0.019 to -0.105) more quality-adjusted life-years and no harm compared to control. A post hoc 5-year analysis found better survival with more residual kidney function at enrolment and at any time over the next 2 years.

Conclusion: The use of a standardised clinical protocol for fluid assessment to avoid excessive fluid removal is associated with excellent preservation of residual kidney function and better medium-term survival in this cohort. Bioimpedance measurements are not necessary to achieve this. Probability of the intervent

背景：液体清除是透析治疗的关键组成部分，但如果过度，可导致残留肾功能更快下降。因此，在透析中规定最佳的液体清除方法以避免这种情况是重要的。生物阻抗光谱是一种评估组织水合作用的床边设备，它可能会改善这种处方，从而降低肾功能下降的速度，改善患者的治疗效果。我们希望在追求这种治疗策略时建立生物阻抗的功效和成本效益。方法：我们在血液透析患者中进行了一项多中心、开放标签、平行、单独随机对照试验，对照组的临床医生和患者对生物阻抗读数不了解。符合条件的患者尿量为> 500 ml/天或肾小球滤过率> 3 ml/分钟/1.73 m2。随机化是1:1，使用隐藏的自动计算机生成的分配系统按中心分层。临床评估每月进行一次，持续3个月，然后每3个月进行一次，持续24个月，在两组中使用标准化表格，在干预组中补充正常水合体重的生物阻抗估计。主要观察指标为无尿时间；次要结局是剩余肾功能、血压、透析相关症状（综合缓和治疗结局量表-肾脏）、生活质量（EuroQol）和每增加一个质量调整生命年的增量成本下降率。结果：从34个英国中心招募并分析了439名患者。无尿症的病因特异性危险率组间无差异，分别为0.751（95%可信区间0.459 ~ 1.229）和0.742（95%可信区间0.453 ~ 1.215）。肾功能下降比预期的要慢，第一年的合并线性率为-0.178(95%可信区间-0.196至-0.159)ml/min /1.73 m2/month；第二年：-0.061（95%置信区间-0.086至-0.036）毫升/分钟/1.73平方米/月。纵向血压、症状和患者报告的结果在组间没有差异。干预与对照组相比，每位患者的平均成本（2020年价格）降低了382英镑（95%可信区间- 3319至2556英镑），质量调整生命年增加了0.043英镑（95%可信区间-0.019至-0.105），且无危害。一项为期5年的事后分析发现，在入组时和未来2年的任何时候，患者的生存率更高，肾功能更残余。结论：在该队列中，使用标准化的临床方案进行液体评估，以避免过多的液体排出，可以很好地保存残余肾功能和更好的中期生存。生物阻抗测量并不需要实现这一点。在每个质量调整生命年增加2万英镑和3万英镑的支付意愿阈值下，干预具有成本效益的概率分别为76%和83%。局限性：该试验没有招募到目标（85%），主要结局的数量少于预期。该试验因2019年发现的冠状病毒疾病而中断，期间进行了193项（6.7%）液体评估和276项（8.1%）肾功能测量，但没有遗漏主要结果。未来工作：年龄、种族和残余肾功能下降之间的关系需要进一步研究。维持肾输出量的生物阻抗光谱确定了血液透析中与液体管理相关的中心水平变化，需要评估。资助：本摘要介绍了由国家卫生与保健研究所（NIHR）卫生技术评估方案资助的独立研究，奖励号为14/216/01。
{"title":"BioImpedance Spectroscopy to maintain Renal Output: the BISTRO randomised controlled trial.","authors":"Simon J Davies, David Coyle, Elizabeth Lindley, David Keane, John Belcher, Fergus Caskey, Indranil Dasgupta, Andrew Davenport, Ken Farrington, Sandip Mitra, Paula Ormandy, Martin Wilkie, Jamie MacDonald, Mandana Zanganeh, Lazaros Andronis, Ivonne Solis-Trapala, Julius Sim","doi":"10.3310/RHON2378","DOIUrl":"10.3310/RHON2378","url":null,"abstract":"<p><strong>Background: </strong>Fluid removal is a key component of dialysis treatment but, if excessive, can result in a faster decline in residual kidney function. Prescribing the optimal removal of fluid on dialysis to avoid this is therefore important. Bioimpedance spectroscopy, a bedside device that estimates tissue hydration, might improve this prescription, so reducing the rate of decline in kidney function and improving patient outcomes. We wished to establish the efficacy and cost-effectiveness of bioimpedance in pursuing this treatment strategy.</p><p><strong>Methods: </strong>We undertook a multicentre, open-label, parallel, individually randomised controlled trial in incident haemodialysis patients, with clinicians and patients blinded to bioimpedance readings in the control group. Eligible patients had a urine output of > 500 ml/day or a glomerular filtration rate > 3 ml/minute/1.73 m<sup>2</sup>. Randomisation was 1 : 1 using a concealed automated computer-generated allocation system stratified by centre. Clinical assessments were made monthly for 3 months and then every 3 months for up to 24 months using a standardised proforma in both groups, supplemented in the intervention group by the bioimpedance estimate of the normally hydrated weight. The primary outcome was time to anuria; secondary outcomes were rate in decline of residual kidney function, blood pressure, dialysis-related symptoms (Integrated Palliative Care Outcome Scale-Renal), quality of life (EuroQol) and incremental cost per additional quality-adjusted life-year gained.</p><p><strong>Results: </strong>Four hundred and thirty-nine patients were recruited and analysed from 34 United Kingdom centres. There were no between-group differences in cause-specific hazard rates of anuria, 0.751 (95% confidence interval 0.459 to 1.229) or subdistribution hazard rates 0.742 (95% confidence interval 0.453 to 1.215). Kidney function decline was slower than anticipated, pooled linear rates in year 1: -0.178 (95% confidence interval -0.196 to -0.159) ml/minute/1.73 m<sup>2</sup>/month; year 2: -0.061 (95% confidence interval -0.086 to -0.036) ml/minute/1.73 m<sup>2</sup>/month. Longitudinal blood pressure, symptoms and patient-reported outcomes did not differ by group. The intervention was associated with £382 (95% confidence interval -£3319 to £2556) lower average cost per patient (price year 2020) and 0.043 (95% confidence interval -0.019 to -0.105) more quality-adjusted life-years and no harm compared to control. A post hoc 5-year analysis found better survival with more residual kidney function at enrolment and at any time over the next 2 years.</p><p><strong>Conclusion: </strong>The use of a standardised clinical protocol for fluid assessment to avoid excessive fluid removal is associated with excellent preservation of residual kidney function and better medium-term survival in this cohort. Bioimpedance measurements are not necessary to achieve this. Probability of the intervent","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 32","pages":"1-23"},"PeriodicalIF":4.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12336963/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144775302","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}
引用次数: 0
Urodynamics tests for the diagnosis and management of male bladder outlet obstruction: long-term follow-up of the UPSTREAM non-inferiority RCT. 尿动力学测试对男性膀胱出口梗阻的诊断和治疗：UPSTREAM非劣效随机对照试验的长期随访。
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/SLPT4675
Madeleine Clout, Amanda L Lewis, Madeleine Cochrane, Grace J Young, Paul Abrams, Peter S Blair, Christopher Chapple, Gordon T Taylor, Sian Noble, Tom Steuart-Feilding, Jodi Taylor, J Athene Lane, Marcus J Drake
<p><strong>Background: </strong>Lower urinary tract symptoms are common in older men and can be bothersome, leading to treatment. The UPSTREAM randomised controlled trial (Phase I) investigated whether assessment of these symptoms with invasive urodynamic testing could improve symptoms when guiding treatment options.</p><p><strong>Objective: </strong>To assess the long-term lower urinary tract symptoms and the rates of surgery for bladder outlet obstruction in men participating in the UPSTREAM study (Phase I).</p><p><strong>Design: </strong>Pragmatic, multicentre, parallel-group, two-group open randomised controlled study, with outcome assessors blinded to aggregate data.</p><p><strong>Setting: </strong>Urology departments of 26 National Health Service hospitals in England.</p><p><strong>Participants: </strong>Men ≥ 18 years, seeking further treatment for their bothersome lower urinary tract symptoms, which may include surgery, who were existing participants of the UPSTREAM study (Phase I). Men were excluded if they were unable to pass urine without a catheter, had a relevant neurological disease, were currently undergoing treatment for prostate or bladder cancer, had previous prostate surgery or were unfit for surgery.</p><p><strong>Interventions: </strong>Routine care plus invasive urodynamics (intervention) or non-invasive routine care.</p><p><strong>Main outcome measures: </strong>The primary outcome was a patient-reported International Prostate Symptom Score 5 years post randomisation. Rates of surgery was the key secondary outcome. Patient-reported outcomes included measures of lower urinary tract symptoms, sexual function, overall quality of life and cost-effectiveness from a secondary care perspective.</p><p><strong>Data sources: </strong>Questionnaires to participants for patient-reported outcome measures, and National Health Service England Hospital Episode Statistics and mortality data.</p><p><strong>Results: </strong>Of 820 men randomised in UPSTREAM (Phase I) between October 2014 and December 2016, 211/427 men randomised to the intervention group completed Phase II questionnaires (49.4%) and 205/363 in the routine care group (56.5%). There was no difference found between International Prostate Symptom Scores in the two groups at 5 years (adjusted difference 0.41, 95% confidence interval -1.10 to 1.93). There was also no difference in other lower urinary tract symptoms, sexual function or quality of life. Routine data were received for 98% of men. Three hundred and forty-seven (43.3%) men with routine data available had received at least one related surgical procedure for the treatment of lower urinary tract symptoms. Over the 5-year time horizon, incremental mean costs were slightly higher (£176.63, 95% confidence interval -£464.06 to £817.32) in the intervention group and incremental mean QALYs were slightly lower (-0.039, 95% confidence interval -0.152 to 0.073) in the intervention group. This suggests that routine care is the cost
背景：下尿路症状在老年男性中很常见，可能很麻烦，需要治疗。UPSTREAM随机对照试验（I期）调查了用有创尿动力学测试评估这些症状是否可以在指导治疗方案时改善症状。目的：评估参与UPSTREAM研究（I期）的男性长期下尿路症状和膀胱出口梗阻的手术率。设计：实用、多中心、平行组、两组开放随机对照研究，结果评估者对汇总数据不知情。环境：英国26家国家卫生服务医院的泌尿科。参与者：≥18岁的男性，为其恼人的下尿路症状寻求进一步治疗，可能包括手术，他们是UPSTREAM研究（I期）的现有参与者。如果男性不能在没有导尿管的情况下排尿，患有相关的神经系统疾病，目前正在接受前列腺癌或膀胱癌治疗，以前做过前列腺手术或不适合手术，则排除在外。干预措施：常规护理加侵入性尿动力学（干预）或非侵入性常规护理。主要结局指标：主要结局是随机分组后5年患者报告的国际前列腺症状评分。手术率是主要的次要结果。患者报告的结果包括测量下尿路症状、性功能、总体生活质量和二级护理角度的成本效益。数据来源：对参与者进行问卷调查，以获得患者报告的结果测量，以及英国国家卫生服务医院事件统计和死亡率数据。结果：在2014年10月至2016年12月期间，在UPSTREAM （I期）中随机分配的820名男性中，随机分配到干预组的211/427名男性完成了II期问卷（49.4%），常规护理组的205/363名男性完成了II期问卷（56.5%）。两组在5年时的国际前列腺症状评分无差异（校正差为0.41,95%可信区间为-1.10 ~ 1.93）。在其他下尿路症状、性功能或生活质量方面也没有差异。98%的男性接受了常规数据。347名（43.3%）有常规资料的男性至少接受过一次相关手术治疗下尿路症状。在5年的时间范围内，干预组的增量平均成本略高（176.63英镑，95%可信区间- 464.06英镑至817.32英镑），干预组的增量平均QALYs略低（-0.039英镑，95%可信区间-0.152至0.073英镑）。这表明常规护理是具有成本效益的选择。局限性：第一阶段研究中约有一半的男性在5年后完成了问卷调查，尽管他们的特征与无反应者、退出参与者或死亡参与者相似。大多数男性是白人，所以结果可能不太适用于其他种族。结论：从临床或成本效益的角度来看，5年随访不支持引入侵入性尿动力学来减少下尿路症状或前列腺手术的发生率。未来工作：这应该确定是否有亚组患者可能受益于在常规护理中增加尿动力学来治疗恼人的下尿路症状。试验注册：该试验注册号为ISRCTN56164274。资助：该奖项由美国国家卫生与保健研究所（NIHR）卫生技术评估项目（NIHR奖励编号：12/140/01）资助，全文发表在《卫生技术评估》杂志上；第29卷，第26号有关进一步的奖励信息，请参阅美国国立卫生研究院资助和奖励网站。
{"title":"Urodynamics tests for the diagnosis and management of male bladder outlet obstruction: long-term follow-up of the UPSTREAM non-inferiority RCT.","authors":"Madeleine Clout, Amanda L Lewis, Madeleine Cochrane, Grace J Young, Paul Abrams, Peter S Blair, Christopher Chapple, Gordon T Taylor, Sian Noble, Tom Steuart-Feilding, Jodi Taylor, J Athene Lane, Marcus J Drake","doi":"10.3310/SLPT4675","DOIUrl":"10.3310/SLPT4675","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Lower urinary tract symptoms are common in older men and can be bothersome, leading to treatment. The UPSTREAM randomised controlled trial (Phase I) investigated whether assessment of these symptoms with invasive urodynamic testing could improve symptoms when guiding treatment options.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To assess the long-term lower urinary tract symptoms and the rates of surgery for bladder outlet obstruction in men participating in the UPSTREAM study (Phase I).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;Pragmatic, multicentre, parallel-group, two-group open randomised controlled study, with outcome assessors blinded to aggregate data.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Urology departments of 26 National Health Service hospitals in England.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Men ≥ 18 years, seeking further treatment for their bothersome lower urinary tract symptoms, which may include surgery, who were existing participants of the UPSTREAM study (Phase I). Men were excluded if they were unable to pass urine without a catheter, had a relevant neurological disease, were currently undergoing treatment for prostate or bladder cancer, had previous prostate surgery or were unfit for surgery.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Routine care plus invasive urodynamics (intervention) or non-invasive routine care.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;The primary outcome was a patient-reported International Prostate Symptom Score 5 years post randomisation. Rates of surgery was the key secondary outcome. Patient-reported outcomes included measures of lower urinary tract symptoms, sexual function, overall quality of life and cost-effectiveness from a secondary care perspective.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Data sources: &lt;/strong&gt;Questionnaires to participants for patient-reported outcome measures, and National Health Service England Hospital Episode Statistics and mortality data.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Of 820 men randomised in UPSTREAM (Phase I) between October 2014 and December 2016, 211/427 men randomised to the intervention group completed Phase II questionnaires (49.4%) and 205/363 in the routine care group (56.5%). There was no difference found between International Prostate Symptom Scores in the two groups at 5 years (adjusted difference 0.41, 95% confidence interval -1.10 to 1.93). There was also no difference in other lower urinary tract symptoms, sexual function or quality of life. Routine data were received for 98% of men. Three hundred and forty-seven (43.3%) men with routine data available had received at least one related surgical procedure for the treatment of lower urinary tract symptoms. Over the 5-year time horizon, incremental mean costs were slightly higher (£176.63, 95% confidence interval -£464.06 to £817.32) in the intervention group and incremental mean QALYs were slightly lower (-0.039, 95% confidence interval -0.152 to 0.073) in the intervention group. This suggests that routine care is the cost","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 26","pages":"1-57"},"PeriodicalIF":4.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12278039/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144575362","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}
引用次数: 0
The quantity, quality and findings of network meta-analyses evaluating the effectiveness of GLP-1 RAs for weight loss: a scoping review. 评估GLP-1 RAs减肥效果的网络荟萃分析的数量、质量和结果：范围综述。
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-06-25 DOI: 10.3310/SKHT8119
Michael Nunns, Samantha Febrey, Jill Buckland, Rebecca Abbott, Rebecca Whear, Alison Bethel, Kate Boddy, Liz Shaw, Jo Thompson Coon, G J Melendez-Torres

Background: Glucagon-like peptide 1 receptor agonists are a class of drug originally developed to treat type 2 diabetes but now increasingly used for weight loss, especially in people living with obesity. Despite an abundance of evidence about the effectiveness and safety of glucagon-like peptide 1 receptor agonists for weight loss, network meta-analyses are inconsistent in their quality and scope, and this is a fast-moving field.

Objectives: We sought to identify the most recent network meta-analyses evaluating the effectiveness of glucagon-like peptide 1 receptor agonists for weight loss; critically appraise included network meta-analyses; provide an overview of the quality and findings of existing network meta-analyses, and identify any pertinent gaps in the evidence; and consider the value of updating the most recent, comprehensive and high-quality network meta-analyses.

Methods: On 6 June 2023, we searched MEDLINE, EMBASE, the Cochrane Database of Systematic Reviews and Epistemonikos for systematic reviews with network meta-analyses published since 2020 in adults (18 or above) with body mass index ≥ 25 (or ≥ 23 for Asian populations), including at least one relevant glucagon-like peptide 1 receptor agonist and weight loss outcomes. We screened and selected reviews in duplicate and independently, and appraised reviews using a modified A MeaSurement Tool to Assess systematic Reviews 2 (AMSTAR-2) and a network meta-analysis reliability checklist. The highest-quality reviews were then extracted in depth, and the most relevant network meta-analysis models identified, focusing on weight loss and safety outcomes. A top-up search for trials published since October 2022 was also undertaken to identify relevant trials not included in published network meta-analyses. A further search for new network meta-analyses was conducted on 26 September 2024.

Results: Of 22 systematic reviews identified, 14 were prioritised for analysis as the remaining 8 reviews were rated as low or critically low quality. We focused on network meta-analyses of weight loss outcomes measured at 6 months, 12 months, longer than 12 months or over a mix of time points. At 6 months, subcutaneous tirzepatide was the most effective drug associated with 9 kg (at 5 mg) to 12 kg (at 15 mg) of weight loss. However, the largest effects were seen for subcutaneous semaglutide 2.4 mg, which was associated with between 11.5 and 12.5 kg of weight loss, though this came from two network meta-analyses, both informed by six trials, and both merging findings across multiple time points. The relative effectiveness among glucagon-like peptide 1 receptor agonists followed a pattern suggested by their performance against placebo, with tirzepatide and semaglutide standing out as the most effective drugs for weight loss. No network meta-analyses compared tirzepatide and semaglutide 2.4 mg. The drugs associated with th

背景：胰高血糖素样肽1受体激动剂是一类最初用于治疗2型糖尿病的药物，但现在越来越多地用于减肥，特别是肥胖人群。尽管有大量证据表明胰高血糖素样肽1受体激动剂对减肥的有效性和安全性，但网络meta分析的质量和范围并不一致，而且这是一个快速发展的领域。目的：我们试图确定最新的网络荟萃分析，评估胰高血糖素样肽1受体激动剂对减肥的有效性；批判性评价包括网络元分析；概述现有网络荟萃分析的质量和发现，并确定证据中的相关差距；并考虑更新最新的、全面的、高质量的网络元分析的价值。方法：2023年6月6日，我们检索MEDLINE、EMBASE、Cochrane系统评价数据库和Epistemonikos，检索自2020年以来发表的网络荟萃分析的系统评价，其中包括体重指数≥25（或亚洲人群≥23）的成人（18岁或以上），包括至少一种相关的胰高血糖素样肽1受体激动剂和体重减轻结果。我们筛选和选择了重复且独立的评价，并使用改进的评估系统评价的测量工具2 （AMSTAR-2）和网络元分析可靠性检查表对评价进行评价。然后深入提取最高质量的评论，并确定最相关的网络元分析模型，重点关注减肥和安全结果。还对2022年10月以来发表的试验进行了补充检索，以确定未包括在已发表的网络荟萃分析中的相关试验。在2024年9月26日进行了新的网络meta分析的进一步搜索。结果：在确定的22个系统评价中，14个优先进行分析，其余8个评价被评为低质量或极低质量。我们专注于6个月、12个月、超过12个月或多个时间点的减肥结果的网络荟萃分析。6个月时，皮下替西帕肽是最有效的药物，体重减轻9公斤（5毫克）至12公斤（15毫克）。然而，最大的效果是皮下注射2.4 mg的semaglutide，与11.5至12.5 kg的体重减轻有关，尽管这是来自两个网络荟萃分析，都是由六个试验提供的，并且都是在多个时间点合并的结果。胰高血糖素样肽1受体激动剂的相对有效性遵循了它们与安慰剂相比的表现模式，其中替西帕肽和西马鲁肽是最有效的减肥药物。没有网络荟萃分析比较替西帕肽和西马鲁肽2.4 mg。与安慰剂相比，体重减轻效果最好的药物替西帕肽和西马鲁肽（2.4 mg）通常与安全性问题的风险增加有关。更新的试验搜索确定了11个新的试验，尽管这些试验规模很小，但可以使新的网络元分析有用。对网络元分析的更新搜索产生了13个新词条。在其他新颖的比较中，替西帕肽间接比较了2.4 mg的西马鲁肽，优于15 mg的西马鲁肽，而不是5或10 mg。数据同样来自合并的时间点。讨论：据我们所知，这是对胰高血糖素样肽1受体激动剂的网络荟萃分析的首次回顾。关于减肥的证据与更广泛的文献基本一致，尽管替西帕肽的数据不像一些荟萃分析中报道的那样令人信服。局限性：目前关于胰高血糖素样肽1受体激动剂的网络荟萃分析与减肥结果通常缺乏网络荟萃分析方法的清晰度，例如哪些试验被纳入。联合使用多种剂量药物的趋势，以及合并来自多个时间点的发现，限制了我们对剂量和时间效应的理解。未来的工作：作为两种最有希望的减肥选择，需要对替西帕肽和西马鲁肽2.4 mg进行头对头试验来确定它们的相对有效性和安全性，同时还需要长期试验来确定胰高血糖素样肽1受体激动剂在服用bb0 - 72周时的有效性和安全性。资助：本文介绍了由国家卫生与保健研究所（NIHR）证据综合计划资助的独立研究，奖励号为NIHR159924。
{"title":"The quantity, quality and findings of network meta-analyses evaluating the effectiveness of GLP-1 RAs for weight loss: a scoping review.","authors":"Michael Nunns, Samantha Febrey, Jill Buckland, Rebecca Abbott, Rebecca Whear, Alison Bethel, Kate Boddy, Liz Shaw, Jo Thompson Coon, G J Melendez-Torres","doi":"10.3310/SKHT8119","DOIUrl":"10.3310/SKHT8119","url":null,"abstract":"<p><strong>Background: </strong>Glucagon-like peptide 1 receptor agonists are a class of drug originally developed to treat type 2 diabetes but now increasingly used for weight loss, especially in people living with obesity. Despite an abundance of evidence about the effectiveness and safety of glucagon-like peptide 1 receptor agonists for weight loss, network meta-analyses are inconsistent in their quality and scope, and this is a fast-moving field.</p><p><strong>Objectives: </strong>We sought to identify the most recent network meta-analyses evaluating the effectiveness of glucagon-like peptide 1 receptor agonists for weight loss; critically appraise included network meta-analyses; provide an overview of the quality and findings of existing network meta-analyses, and identify any pertinent gaps in the evidence; and consider the value of updating the most recent, comprehensive and high-quality network meta-analyses.</p><p><strong>Methods: </strong>On 6 June 2023, we searched MEDLINE, EMBASE, the Cochrane Database of Systematic Reviews and Epistemonikos for systematic reviews with network meta-analyses published since 2020 in adults (18 or above) with body mass index ≥ 25 (or ≥ 23 for Asian populations), including at least one relevant glucagon-like peptide 1 receptor agonist and weight loss outcomes. We screened and selected reviews in duplicate and independently, and appraised reviews using a modified A MeaSurement Tool to Assess systematic Reviews 2 (AMSTAR-2) and a network meta-analysis reliability checklist. The highest-quality reviews were then extracted in depth, and the most relevant network meta-analysis models identified, focusing on weight loss and safety outcomes. A top-up search for trials published since October 2022 was also undertaken to identify relevant trials not included in published network meta-analyses. A further search for new network meta-analyses was conducted on 26 September 2024.</p><p><strong>Results: </strong>Of 22 systematic reviews identified, 14 were prioritised for analysis as the remaining 8 reviews were rated as low or critically low quality. We focused on network meta-analyses of weight loss outcomes measured at 6 months, 12 months, longer than 12 months or over a mix of time points. At 6 months, subcutaneous tirzepatide was the most effective drug associated with 9 kg (at 5 mg) to 12 kg (at 15 mg) of weight loss. However, the largest effects were seen for subcutaneous semaglutide 2.4 mg, which was associated with between 11.5 and 12.5 kg of weight loss, though this came from two network meta-analyses, both informed by six trials, and both merging findings across multiple time points. The relative effectiveness among glucagon-like peptide 1 receptor agonists followed a pattern suggested by their performance against placebo, with tirzepatide and semaglutide standing out as the most effective drugs for weight loss. No network meta-analyses compared tirzepatide and semaglutide 2.4 mg. The drugs associated with th","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-73"},"PeriodicalIF":4.0,"publicationDate":"2025-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12336958/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144527680","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}
引用次数: 0
Establishing the best step-up treatments for children with uncontrolled asthma despite inhaled corticosteroids: the EINSTEIN systematic review, network meta-analysis and cost-effectiveness analysis using individual participant data. 为吸入皮质类固醇后仍无法控制哮喘的儿童建立最佳强化治疗：爱因斯坦系统评价、网络荟萃分析和使用个体参与者数据的成本效益分析。
IF 3.5 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-05-01 DOI: 10.3310/HGWT3617
Sofia Cividini, Ian Sinha, Giovanna Culeddu, Sarah Donegan, Michelle Maden, Katie Rose, Olivia Fulton, Dyfrig Hughes, Stephen Turner, Catrin Tudur Smith
<p><strong>Background: </strong>There is no clear preferential option for initial step-up of treatment for children with uncontrolled asthma on inhaled corticosteroid.</p><p><strong>Objectives: </strong>Evaluate the clinical effectiveness of pharmacological treatments to use in children with uncontrolled asthma on inhaled corticosteroid; identify and evaluate the potential for treatment effect modification to optimise treatment delivery; assess the cost-effectiveness of treatments.</p><p><strong>Methods: </strong>Systematic review and individual participant data network meta-analysis. Studies were eligible if they were parallel or crossover randomised controlled trials comparing at least one of the pharmacological treatments of interest in participants aged < 18 years with uncontrolled asthma on any dose inhaled corticosteroid alone. We searched MEDLINE<sup>®</sup>, Cochrane Library, Cochrane Central Register of Controlled Trials, EMBASE, National Institute for Health and Care Excellence Technology Appraisals, and the National Institute for Health and Care Research Health Technology Assessment series. Primary outcomes: exacerbation and asthma control. Secondary outcomes: health-related quality of life, mortality, forced expiratory volume in 1 second, adverse events, hospital admissions, symptoms (not analysed). We assessed the Risk Of Bias using the Cochrane Risk Of Bias tool and carried out Bayesian meta-analyses, network meta-analysis and network meta-regression, including treatment by covariate (age, sex, ethnicity, eczema, eosinophilia, asthma severity) interactions. A Markov decision-analytic model with a 12-month time horizon, which adopted the perspective of the National Health Service and Personal Social Services in the United Kingdom, was developed to compare alternative treatments. Cost-effectiveness was based on incremental costs per quality-adjusted life-years gained, with uncertainty considered in one-way, structural and probabilistic sensitivity analyses.</p><p><strong>Results: </strong>We identified and screened 4708 publications from the search and confirmed 144 randomised controlled trials as eligible. We obtained individual participant data from 29 trials (5381 participants) and extracted limited aggregate data from a further 19 trials. The majority of trials had low risk of bias. The network meta-analysis suggests that medium-dose inhaled corticosteroid + long-acting <i>β</i><sub>2</sub>-agonist is the preferred treatment for reducing odds of exacerbation [odds ratio 95% credibility interval: 0.43 (0.20 to 0.92) vs. low-dose inhaled corticosteroid; 40 studies, 8168 patients] and increasing forced expiratory volume in 1 second [mean difference 95% credibility interval: 0.71 (0.35 to 1.06) vs. low-dose inhaled corticosteroid; 23 studies, 2518 patients] while leukotriene receptor antagonist alone is the least preferred. No clear differences were found for asthma control (16 studies, 3027 patients). Limited pairwise analyses sugges
背景：对于吸入皮质类固醇治疗不受控制的哮喘儿童，没有明确的优先选择。目的：评价吸入皮质类固醇药物治疗不受控制哮喘患儿的临床疗效；识别和评估治疗效果改善的潜力，以优化治疗交付；评估治疗的成本效益。方法：系统评价和个体参与者数据网络荟萃分析。如果研究是平行或交叉随机对照试验，比较了至少一种感兴趣的药物治疗，受试者为老年人®、Cochrane图书馆、Cochrane中央对照试验登记册、EMBASE、国家卫生与保健卓越技术评估研究所和国家卫生与保健研究所卫生技术评估系列，则研究符合条件。主要结局：病情恶化和哮喘控制。次要结局：健康相关生活质量、死亡率、1秒用力呼气量、不良事件、住院情况、症状（未分析）。我们使用Cochrane偏倚风险工具评估偏倚风险，并进行贝叶斯荟萃分析、网络荟萃分析和网络荟萃回归，包括协变量（年龄、性别、种族、湿疹、嗜酸性粒细胞、哮喘严重程度）相互作用的治疗。采用联合王国国家卫生服务和个人社会服务的观点，开发了一个12个月时间范围的马尔可夫决策分析模型，以比较不同的治疗方法。成本效益基于每个质量调整寿命年的增量成本，在单向、结构和概率敏感性分析中考虑了不确定性。结果：我们从检索中确定并筛选了4708篇出版物，并确认144项随机对照试验符合条件。我们从29项试验（5381名受试者）中获得个体受试者数据，并从另外19项试验中提取有限的汇总数据。大多数试验偏倚风险较低。网络荟萃分析表明，中剂量吸入皮质类固醇+长效β2激动剂是降低恶化几率的首选治疗方法[比值比95%可信区间：0.43 (0.20 ~ 0.92)；40项研究，8168例患者]和在1秒内增加用力呼气量[95%可信区间：0.71（0.35 ~ 1.06）相比于低剂量吸入皮质类固醇；23项研究，2518例患者]而单独使用白三烯受体拮抗剂是最不受欢迎的。哮喘控制方面无明显差异（16项研究，3027例患者）。有限的两两分析表明，与吸入皮质类固醇+长效β2激动剂相比，中剂量吸入皮质类固醇可改善与健康相关的生活质量[两项研究，儿科哮喘生活质量问卷，平均差异95%可信区间：0.91(0.29至1.53)]。在5项试验中，因哮喘发作而住院的患者比例从0.5%到2.7%不等。与吸入皮质类固醇+长效β2受体拮抗剂相比，吸入皮质类固醇+白三烯受体拮抗剂报告的神经系统疾病（轻度/中度）患者略少[优势比95%置信区间：0.09(0.01至0.82)，一项研究]。没有死亡记录。我们没有找到令人信服的、一致的证据表明，年龄、性别、种族、湿疹、嗜酸性粒细胞、哮喘严重程度将被视为影响因素。经济分析表明，低剂量吸入皮质类固醇是最具成本效益的治疗选择，而中剂量吸入皮质类固醇（单独使用和+长效β2激动剂）与最高的质量调整生命年相关，但其增量成本效益超过了国家健康与护理卓越研究所的阈值。讨论：中剂量吸入皮质类固醇+长效β2激动剂推荐用于单独吸入皮质类固醇无法控制的哮喘儿童；应避免单独使用白三烯受体拮抗剂。我们不能纳入67%符合条件的试验的数据，因此结论应谨慎看待。研究注册：本研究注册号为PROSPERO CRD42019127599。资助：该奖项由美国国立卫生与保健研究所（NIHR）卫生技术评估项目（NIHR奖励编号：16/110/16）资助，全文发表在《卫生技术评估》杂志上；第29卷第15期有关进一步的奖励信息，请参阅美国国立卫生研究院资助和奖励网站。
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引用次数: 0
Ivacaftor-tezacaftor-elexacaftor, tezacaftor-ivacaftor and lumacaftor-ivacaftor for treating cystic fibrosis: a systematic review and economic evaluation. 治疗囊性纤维化的干扰素-干扰素-干扰素、干扰素-干扰素和干扰素-干扰素：系统综述和经济评价。
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-05-01 DOI: 10.3310/CPLD8546
Steven J Edwards, Benjamin G Farrar, Kate Ennis, Nicole Downes, Victoria Wakefield, Isaac Mackenzie, Archie Walters, Tracey Jhita
<p><strong>Background: </strong>Cystic fibrosis is a life-limiting genetic condition that affects over 9000 people in England. Cystic fibrosis is usually diagnosed through newborn screening and causes symptoms throughout the body, including the lungs and digestive system. Around 90% of individuals with cystic fibrosis have at least one copy of the <i>F508del</i> mutation on the cystic fibrosis transmembrane conductance regulator gene.</p><p><strong>Objectives: </strong>To appraise the clinical effectiveness and cost-effectiveness of elexacaftor-tezacaftor-ivacaftor, tezacaftor-ivacaftor and lumacaftor-ivacaftor within their expected marketing authorisations for treating people with cystic fibrosis and at least one <i>F508del</i> mutation, compared with each other and with established clinical management before these treatments.</p><p><strong>Methods: </strong>A de novo systematic literature review (search date February 2023) was conducted searching electronic databases (MEDLINE, EMBASE, Cochrane Central Register of Controlled Trials), bibliographies of relevant systematic literature reviews, clinical trial registers, recent conferences and evidence provided by Vertex Pharmaceuticals (Boston, MA, USA). Data on the following outcomes were summarised: acute change in per cent predicted forced expiratory volume in 1 second (change in weight-for-age <i>z</i>-score; and change in pulmonary exacerbation frequency requiring intravenous antibiotics. Network meta-analyses were conducted where head-to-head data were not available. Data from clinical trials and real-world evidence were examined to assess long-term effectiveness. A patient-level simulation model was developed to assess the cost-effectiveness of the three modulator treatments. The model employed a lifetime horizon and was developed from the perspective of the National Health Service.</p><p><strong>Results: </strong>Data from 19 primary studies and 7 open-label extension studies were prioritised in the systematic literature review. Elexacaftor/tezacaftor/ivacaftor was associated with a statistically significant increase in predicted forced expiratory volume in 1 second and weight-for-age <i>z</i>-score and a reduction in pulmonary exacerbations compared with established clinical management, lumacaftor/ivacaftor and tezacaftor/ivacaftor, and also led to a reduction in the rate of predicted forced expiratory volume in 1 second decline relative to established clinical management, although the magnitude of this decrease was uncertain. Lumacaftor/ivacaftor and tezacaftor/ivacaftor were also associated with a statistically significant increase in predicted forced expiratory volume in 1 second and reduction in pulmonary exacerbations relative to established clinical management, but with a smaller effect size than elexacaftor/tezacaftor/ivacaftor. There was some evidence that tezacaftor/ivacaftor reduced the rate of predicted forced expiratory volume in 1 second decline relative to established clinical
背景：囊性纤维化是一种限制生命的遗传疾病，在英国影响着9000多人。囊性纤维化通常是通过新生儿筛查诊断出来的，它会在全身引起症状，包括肺部和消化系统。大约90%的囊性纤维化患者在囊性纤维化跨膜传导调节基因上至少有一个F508del突变拷贝。目的：评估elexacaftor-tezacaftor-ivacaftor、tezacaftor-ivacaftor和lumacaftor-ivacaftor在其预期上市许可范围内治疗囊性纤维化和至少一种F508del突变患者的临床疗效和成本效益，并与这些治疗前已建立的临床管理进行比较。方法：检索电子数据库（MEDLINE、EMBASE、Cochrane中央对照试验注册库）、相关系统文献综述书目、临床试验注册库、近期会议和Vertex Pharmaceuticals （Boston， MA， USA）提供的证据，重新进行系统文献综述（检索日期为2023年2月）。总结了以下结果的数据：1秒内预测用力呼气量的急性变化(年龄体重z-score的变化；以及需要静脉注射抗生素的肺恶化频率的变化。网络荟萃分析是在没有面对面数据的情况下进行的。研究了临床试验数据和真实世界证据，以评估长期有效性。建立了患者水平的模拟模型来评估三种调节剂治疗的成本效益。该模型采用了一生的视角，是从国民保健服务的角度发展起来的。结果：系统文献综述优先考虑了19项初步研究和7项开放标签扩展研究的数据。与已建立的临床管理、lumacaftor/ivacaftor和tezacaftor/ivacaftor相比，Elexacaftor/tezacaftor/ivacaftor与1秒内预测用力呼气量和年龄体重z评分的统计学显著增加以及肺恶化的减少相关，并且还导致1秒内预测用力呼气量下降的比率与已建立的临床管理相关。虽然这种减少的幅度是不确定的。与已建立的临床管理相比，Lumacaftor/ivacaftor和tezacaftor/ivacaftor也与1秒内预测用力呼气量的增加和肺恶化的减少有统计学意义上的显著相关，但与elexaftor /tezacaftor/ivacaftor相比，其效应较小。有一些证据表明，相对于现有的临床管理，tezacaftor/ivacaftor降低了1秒内预测用力呼气量的下降率，但很少有证据表明，相对于现有的临床管理，lumacaftor/ivacaftor降低了1秒内预测用力呼气量的下降率。经济分析的增量成本效益比是保密的。然而，对于所研究的所有基因型，增量成本效益比高于国家健康和护理卓越研究所的门槛，即每个质量调整生命年获得2万至3万英镑，这将被视为具有成本效益。结论：尽管观察到改善的临床效益，但根据国家健康和护理卓越研究所每获得质量调整生命年20,000-30,000英镑的门槛，评估的囊性纤维化跨膜传导调节基因调节剂均不具有成本效益。这在很大程度上是由囊性纤维化跨膜传导调节基因调节剂治疗的高获取成本驱动的。研究注册：本研究注册号为PROSPERO CRD42023399583。资助：该奖项由美国国家卫生与保健研究所（NIHR）证据综合计划（NIHR奖励编号：NIHR135829）资助，全文发表在《卫生技术评估》上；第29卷，第19号有关进一步的奖励信息，请参阅美国国立卫生研究院资助和奖励网站。
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引用次数: 0
Behaviour change interventions to promote physical activity in people with intermittent claudication: the OPTIMA systematic review. 促进间歇性跛行患者身体活动的行为改变干预措施：OPTIMA系统综述
IF 3.5 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-05-01 DOI: 10.3310/ZBNG5240
Ukachukwu O Abaraogu, Philippa Dall, Chris Seenan, Sarah Rhodes, Trish Gorely, Joanna McParland, Julie Brittenden, Ebuka M Anieto, Lorna Booth, Cathy Gormal, Jeremy Dearling, Candida Fenton, Sarah Audsley, Kimberley Fairer, Lindsay Bearne, Dawn A Skelton

Background: People with intermittent claudication are significantly less active compared to their peers without intermittent claudication, worsening future health outcomes. Supervised exercise therapy is not commonly available, but behaviour change techniques in unsupervised interventions can improve physical activity. Specific behaviour change techniques, theoretical mechanisms and contextual features linked to effectiveness remain unclear.

Objectives: To conduct an integrative synthesis of: effectiveness of behaviour change technique-based interventions on daily physical activity and clinical-/patient-reported outcomes; behaviour change techniques and theoretical mechanisms within effective behaviour change technique-based interventions; feasibility and acceptability. Primary outcomes: short term (< 6 months) and maintenance (> 6 months) of daily physical activity. Secondary outcomes: clinical-/patient-reported outcomes.

Data sources: Seven primary studies databases; Cochrane Database of Systematic Reviews, the Database of Abstracts of Reviews of Effects, Health Technology Assessment Database and Trial Registers to 31 August 2023.

Review methods: Systematic review 1: interventions incorporating ≥ 1 behaviour change technique (coded using Behaviour Change Technique Taxonomy version 1, and Theoretical Domains Framework). Systematic review 2: quantitative, qualitative, mixed-methods research on patient/provider experiences. Study quality assessed using revised Cochrane risk-of-bias tool for randomised trials; Risk Of Bias In Non-randomised Studies - of Interventions and Mixed Methods Appraisal Tool.

Results: Fifty-three articles (41 studies) were included in systematic review 1, and 28 articles (28 studies) in systematic review 2. Eleven randomised controlled trials demonstrated that behaviour change technique-based interventions increased daily physical activity in the short term [increase of 0.20 standardised mean difference (95% confidence interval 0.07 to 0.33), ~ 473 steps/day] with high certainty. Evidence of maintenance of daily physical activity is unclear (increase of 0.12 standardised mean difference; ~ 288 steps/day). Behaviour change techniques aimed at improving patients' intentions to engage in physical activity were most effective. Network analysis suggests that behaviour change technique-based interventions improved daily physical activity and may be better than supervised exercise therapy in maintaining daily physical activity. behaviour change technique-based interventions were acceptable and had short-medium-term benefits to initial/absolute claudication distance/time, walking impairment scores and disease-specific quality of life.

Conclusions: The behaviour change technique-based interventions are effective, targeting intention to engage in physical activity, in improving daily physical activity

背景：与没有间歇性跛行的同龄人相比，间歇性跛行患者活动量明显减少，未来健康状况恶化。有监督的运动疗法并不常见，但在无监督的干预中，行为改变技术可以改善身体活动。具体的行为改变技术、理论机制和与有效性相关的背景特征仍不清楚。目的：对基于行为改变技术的干预措施对日常身体活动和临床/患者报告的结果的有效性进行综合综合；行为改变技术和有效的基于行为改变技术的干预措施中的理论机制；可行性和可接受性。主要结果：短期（6个月）的日常身体活动。次要结局：临床/患者报告的结局。数据来源：七个主要研究数据库；截至2023年8月31日，Cochrane系统评价数据库、效果评价摘要数据库、卫生技术评估数据库和试验登记册。综述方法：系统综述1：纳入≥1种行为改变技术的干预措施（使用行为改变技术分类法第1版和理论领域框架进行编码）。系统评价2：定量、定性、混合方法对患者/提供者经验的研究。使用改进的Cochrane随机试验风险偏倚工具评估研究质量；非随机研究中的偏倚风险-干预和混合方法评估工具。结果：系统评价1纳入53篇文章（41项研究），系统评价2纳入28篇文章（28项研究）。11项随机对照试验表明，基于行为改变技术的干预措施在短期内增加了日常身体活动[增加0.20标准化平均差（95%置信区间0.07至0.33），约473步/天]。维持日常体力活动的证据尚不清楚(标准化平均差增加0.12；~ 288步/天)。旨在提高患者参与体育活动意愿的行为改变技术是最有效的。网络分析表明，基于行为改变技术的干预措施改善了日常身体活动，在维持日常身体活动方面可能比监督运动疗法更好。基于行为改变技术的干预是可接受的，并且对初始/绝对跛行距离/时间、行走障碍评分和疾病特异性生活质量具有中短期益处。结论：基于行为改变技术的干预措施是有效的，针对参与身体活动的意图，在短期内改善日常身体活动和功能结果，尽管维持的证据有限。有必要进行更多的随机对照试验，检查日常身体活动和临床结果，包括长期随访，详细描述行为改变技术、成本和提供者的观点。研究注册：本研究注册号为PROSPERO CRD42020159869。资助：该奖项由美国国家卫生与保健研究所（NIHR）卫生技术评估计划（NIHR奖励编号：NIHR130664）资助，全文发表在《卫生技术评估》上；第29卷第18期有关进一步的奖励信息，请参阅美国国立卫生研究院资助和奖励网站。
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