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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。
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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。
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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 3.5 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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引用次数: 0
Sputum colour charts to guide antibiotic self-treatment of acute exacerbation of chronic obstructive pulmonary disease: the Colour-COPD RCT. 痰色图指导慢性阻塞性肺疾病急性加重期抗生素自我治疗：颜色- copd随机对照试验
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-05-01 DOI: 10.3310/KPFD5558
Eleni Gkini, Rachel L Adams, Daniella Spittle, Paul Ellis, Katherine Allsopp, Sanya Saleem, Matthew McKenna, Nick le Mesurier, Nicola Gale, Sarah Tearne, Peymane Adab, Rachel E Jordan, Nawar Diar Bakerly, Alice M Turner

Background: Chronic obstructive pulmonary disease exacerbations (acute exacerbation of chronic obstructive pulmonary disease) are characterised by increased sputum volume, purulence and breathlessness. Patients are encouraged to recognise and treat acute exacerbation of chronic obstructive pulmonary disease as part of a self-management plan. Only half of acute exacerbation of chronic obstructive pulmonary disease are caused by bacterial infection, but self-management plans generally advocate use of antibiotics and steroids for all events, hence antibiotics may be overused. Sputum colour relates closely to bacterial load; thus it could determine whether antibiotics are appropriate. This pragmatic randomised controlled trial tested whether use of a sputum colour chart is safe and effective in United Kingdom primary care.

Methods: Colour chronic obstructive pulmonary disease was a multicentre, randomised controlled trial in adults with chronic obstructive pulmonary disease who had ≥ 2 acute exacerbations of chronic obstructive pulmonary disease or ≥ 1 hospital admission for acute exacerbation of chronic obstructive pulmonary disease in the preceding year. The primary objective was to demonstrate that the Bronkotest® (London) sputum colour chart is non-inferior to usual care (safe). The primary outcome was rate of hospital admission for acute exacerbation of chronic obstructive pulmonary disease at 12 months; secondary outcomes included requirement for second courses of treatment and quality of life (chronic obstructive pulmonary disease assessment test score). Nested substudies examining daily symptoms via an e-diary and sputum culture assessed untreated acute exacerbation of chronic obstructive pulmonary disease rate and antibiotic resistance, respectively. A process evaluation examined trial fidelity and acceptability of the intervention, employing qualitative research methods incorporating patients as co-researchers.

Limitations: The study was terminated early due to low recruitment (115/2954 planned sample size).

Results: One hundred and fifteen patients were recruited and randomised 1 : 1 to colour chart use or usual care; they generally had severe Global Initiative for Chronic Obstructive Lung Disease D chronic obstructive pulmonary disease, with significant breathlessness (54% Medical Research Council score of 4 or 5) and poor quality of life (chronic obstructive pulmonary disease assessment test score at baseline 24). Comorbid respiratory and systemic disease was common. Self-management was delivered well in both arms, and the colour chart acceptable to patients and staff; no specific issues for patients with multiple long-term conditions were identified. Hospital admissions for acute exacerbation of chronic obstructive pulmonary disease tended to occur more in colour chart users [32 vs. 16%, relative risk 1.95 (0.92 to 4.18)], and antibiotic

背景：慢性阻塞性肺疾病急性加重（慢性阻塞性肺疾病急性加重）的特征是痰量增加、化脓和呼吸困难。作为自我管理计划的一部分，鼓励患者认识和治疗慢性阻塞性肺疾病的急性加重。慢性阻塞性肺疾病急性加重中只有一半是由细菌感染引起的，但自我管理计划通常主张对所有事件使用抗生素和类固醇，因此抗生素可能被过度使用。痰液颜色与细菌负荷密切相关；因此，它可以确定抗生素是否合适。这项实用的随机对照试验测试了在英国初级保健中使用痰液颜色表是否安全有效。方法：彩色慢性阻塞性肺疾病是一项多中心、随机对照试验，研究对象为成人慢性阻塞性肺疾病患者，这些患者在前一年有≥2次慢性阻塞性肺疾病急性加重或≥1次慢性阻塞性肺疾病急性加重住院。主要目的是证明Bronkotest®（London）痰液颜色图不逊于常规护理（安全）。主要终点为慢性阻塞性肺疾病急性加重12个月住院率；次要结局包括对第二疗程的要求和生活质量（慢性阻塞性肺疾病评估测试得分）。巢式亚研究通过电子日记和痰培养检查每日症状，分别评估未经治疗的慢性阻塞性肺疾病急性加重率和抗生素耐药性。过程评估检查了试验的保真度和干预的可接受性，采用定性研究方法，将患者作为共同研究人员。局限性：由于招募人数少（115/2954计划样本量），研究提前终止。结果：115例患者被招募并随机分为1∶1组，使用彩色图表或常规护理；他们通常患有严重的慢性阻塞性肺疾病全球倡议D慢性阻塞性肺疾病，伴有明显的呼吸困难（54%的医学研究委员会评分为4或5）和生活质量差（慢性阻塞性肺疾病评估测试基线评分为24）。呼吸道和全身性疾病合并症很常见。两组的自我管理都很好，患者和工作人员都能接受彩色图表；没有发现患有多种长期疾病的患者的具体问题。慢性阻塞性肺疾病急性加重住院在彩色图表使用者中更容易发生[32比16%，相对危险度1.95(0.92至4.18)]，慢性阻塞性肺疾病首次急性加重治疗后14天内的抗生素疗程也更常见[34比18%，调整相对危险度1.80(0.85至3.79)]。尽管如此，彩色图表使用者在12个月时的生活质量更好[慢性阻塞性肺疾病评估测试19.9 vs. -24.5，调整后平均差-2.95(-5.93至-0.04)]。38名患者同意痰液亚组研究，接受了57份样本（42份处于稳定状态，15份处于慢性阻塞性肺疾病急性加重期），其中30%含有潜在致病菌。支气管扩张受试者的痰更有可能是化脓的，与疾病状态（稳定还是恶化）或样本是否对潜在致病菌呈阳性无关，这表明仅凭颜色不能用于指导抗生素的使用。11名患者完成了电子日记研究，并捕获了42例症状定义的慢性阻塞性肺疾病急性加重事件，其中许多未经治疗，慢性肺病工具评分低于接受治疗的患者。未处理的事件解决得较慢。由于数量少，研究组之间的差异没有意义。结论和未来的工作：我们的结果表明Bronkotest痰色图不太可能成为初级保健中慢性阻塞性肺疾病患者自我管理的有用补充，但需要进一步的工作来证实这一点。资助：本摘要介绍了由国家卫生与保健研究所（NIHR）卫生技术评估项目资助的独立研究，奖励号为17/128/04。
{"title":"Sputum colour charts to guide antibiotic self-treatment of acute exacerbation of chronic obstructive pulmonary disease: the Colour-COPD RCT.","authors":"Eleni Gkini, Rachel L Adams, Daniella Spittle, Paul Ellis, Katherine Allsopp, Sanya Saleem, Matthew McKenna, Nick le Mesurier, Nicola Gale, Sarah Tearne, Peymane Adab, Rachel E Jordan, Nawar Diar Bakerly, Alice M Turner","doi":"10.3310/KPFD5558","DOIUrl":"10.3310/KPFD5558","url":null,"abstract":"<p><strong>Background: </strong>Chronic obstructive pulmonary disease exacerbations (acute exacerbation of chronic obstructive pulmonary disease) are characterised by increased sputum volume, purulence and breathlessness. Patients are encouraged to recognise and treat acute exacerbation of chronic obstructive pulmonary disease as part of a self-management plan. Only half of acute exacerbation of chronic obstructive pulmonary disease are caused by bacterial infection, but self-management plans generally advocate use of antibiotics and steroids for all events, hence antibiotics may be overused. Sputum colour relates closely to bacterial load; thus it could determine whether antibiotics are appropriate. This pragmatic randomised controlled trial tested whether use of a sputum colour chart is safe and effective in United Kingdom primary care.</p><p><strong>Methods: </strong>Colour chronic obstructive pulmonary disease was a multicentre, randomised controlled trial in adults with chronic obstructive pulmonary disease who had ≥ 2 acute exacerbations of chronic obstructive pulmonary disease or ≥ 1 hospital admission for acute exacerbation of chronic obstructive pulmonary disease in the preceding year. The primary objective was to demonstrate that the Bronkotest<sup>®</sup> (London) sputum colour chart is non-inferior to usual care (safe). The primary outcome was rate of hospital admission for acute exacerbation of chronic obstructive pulmonary disease at 12 months; secondary outcomes included requirement for second courses of treatment and quality of life (chronic obstructive pulmonary disease assessment test score). Nested substudies examining daily symptoms via an e-diary and sputum culture assessed untreated acute exacerbation of chronic obstructive pulmonary disease rate and antibiotic resistance, respectively. A process evaluation examined trial fidelity and acceptability of the intervention, employing qualitative research methods incorporating patients as co-researchers.</p><p><strong>Limitations: </strong>The study was terminated early due to low recruitment (115/2954 planned sample size).</p><p><strong>Results: </strong>One hundred and fifteen patients were recruited and randomised 1 : 1 to colour chart use or usual care; they generally had severe Global Initiative for Chronic Obstructive Lung Disease D chronic obstructive pulmonary disease, with significant breathlessness (54% Medical Research Council score of 4 or 5) and poor quality of life (chronic obstructive pulmonary disease assessment test score at baseline 24). Comorbid respiratory and systemic disease was common. Self-management was delivered well in both arms, and the colour chart acceptable to patients and staff; no specific issues for patients with multiple long-term conditions were identified. Hospital admissions for acute exacerbation of chronic obstructive pulmonary disease tended to occur more in colour chart users [32 vs. 16%, relative risk 1.95 (0.92 to 4.18)], and antibiotic ","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-42"},"PeriodicalIF":4.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12403665/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144142392","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
Bisoprolol for patients with chronic obstructive pulmonary disease at high risk of exacerbation: the BICS RCT. 比索洛尔用于慢性阻塞性肺疾病高危加重患者：BICS随机对照试验
IF 3.5 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-05-01 DOI: 10.3310/TNDG8641
Graham Devereux, Seonaidh Cotton, Mintu Nath, Nicola McMeekin, Karen Campbell, Rekha Chaudhuri, Gourab Choudhury, Anthony De Soyza, Shona Fielding, Simon Gompertz, John Haughney, Amanda Lee, Graeme MacLennan, Alyn Morice, John Norrie, David Price, Philip Short, Jorgen Vestbo, Paul Walker, Jadwiga Wedzicha, Andrew Wilson, Olivia Wu, Brian Lipworth

Background: Observational studies of people with chronic obstructive pulmonary disease using beta-blockers for cardiovascular disease indicate that beta-blocker use is associated with reduced risk of chronic obstructive pulmonary disease exacerbation. However, at the time this study was initiated, there had been no randomised controlled trials confirming or refuting this.

Objective(s): To determine the clinical and cost-effectiveness of adding bisoprolol (maximal dose 5 mg once daily) to usual chronic obstructive pulmonary disease therapies in patients with chronic obstructive pulmonary disease at high risk of exacerbation.

Design: A multicentre, pragmatic, double-blind, randomised, placebo-controlled clinical trial.

Setting: Seventy-six United Kingdom primary and secondary care sites.

Participants: People aged ≥ 40 years with a diagnosis of at least moderately severe chronic obstructive pulmonary disease with a history of at least two exacerbations in the previous year.

Interventions: Participants were randomised (1 : 1) to receive either bisoprolol or placebo for 1 year. During a 4- to 7-week titration period, the maximum tolerated dose was established (1.25 mg, 2.5 mg, 3.75 mg, 5 mg once daily).

Primary outcome: A number of participant-reported exacerbations during the 1-year treatment period.

Results: In total, 519 participants were recruited and randomised. Four post-randomisation exclusions left 259 in the bisoprolol group and 256 in the placebo group. Treatment groups were balanced at baseline: mean (standard deviation) age 68 (7.9) years; 53% men; mean (standard deviation) pack year smoking history 45 (25.2); mean (standard deviation) 3.5 (1.9) exacerbations in previous year. Primary outcome data were available for 99.8% of participants (bisoprolol 259, placebo 255). The mean (standard deviation) number of exacerbations was 2.03 (1.91) in the bisoprolol group and 2.01 (1.75) in the placebo group (adjusted incidence rate ratio 0.97, 95% confidence interval 0.84 to 1.13), p = 0.72. The number of participants with serious adverse events was similar between the two groups (bisoprolol 37, placebo 36). The total number of adverse reactions was also similar between the two groups. As expected, bisoprolol was associated with a higher proportion of vascular adverse reactions (e.g. hypotension, cold peripheries) than placebo, but was not associated with an excess of other adverse reactions, including those classified as respiratory. Adding bisoprolol resulted in a statistically insignificant trend towards higher costs (£636, 95% confidence interval £118 to £1391) and fewer quality-adjusted life-years (0.035, 95% confidence interval 0.059 to 0.010) compared to placebo.

Limitations: The study findings should be interpreted with caution as the

背景：对慢性阻塞性肺疾病患者使用β受体阻滞剂治疗心血管疾病的观察性研究表明，β受体阻滞剂的使用与慢性阻塞性肺疾病恶化的风险降低有关。然而，在本研究开始时，还没有随机对照试验证实或反驳这一点。目的：确定在慢性阻塞性肺疾病急性加重高风险患者的常规慢性阻塞性肺疾病治疗中添加比索洛尔（最大剂量5mg，每日一次）的临床和成本效益。设计：一项多中心、实用、双盲、随机、安慰剂对照的临床试验。环境：英国76个初级和二级医疗机构。参与者：年龄≥40岁，诊断为至少中重度慢性阻塞性肺疾病，前一年至少有两次发作史的患者。干预措施：参与者随机（1:1）接受比索洛尔或安慰剂治疗1年。在4- 7周的滴定期内，确定了最大耐受剂量（1.25 mg, 2.5 mg, 3.75 mg, 5mg，每日一次）。主要结局：在1年的治疗期间，一些参与者报告了病情恶化。结果：共有519名参与者被招募并随机分组。随机化后，比索洛尔组有259例，安慰剂组有256例。治疗组在基线时平衡：平均（标准差）年龄68（7.9）岁；男性53%;平均（标准差）吸烟史45 (25.2)；前一年平均（标准差）加重3.5次（1.9次）。99.8%的参与者（比索洛尔259，安慰剂255）可获得主要结局数据。比索洛尔组的平均（标准差）加重次数为2.03次（1.91次），安慰剂组的平均（标准差）加重次数为2.01次（1.75次）（校正发生率比0.97,95%可信区间0.84 ~ 1.13），p = 0.72。两组发生严重不良事件的参与者数量相似（比索洛尔37例，安慰剂36例）。两组的不良反应总数也相似。正如预期的那样，比索洛尔与血管不良反应（如低血压、外周冷）的比例高于安慰剂，但与其他不良反应（包括呼吸道不良反应）的过量无关。与安慰剂相比，添加比索洛尔导致更高的成本（636英镑，95%置信区间为118英镑至1391英镑）和更少的质量调整寿命年（0.035英镑，95%置信区间为0.059至0.010），这在统计学上没有显著的趋势。局限性：研究结果应谨慎解释，因为资助者认为该研究在COVID-19大流行临床研究环境中不可行，因此没有达到1574个目标样本量。虽然28%的参与者没有开始使用比索洛尔/安慰剂（1.6%）或在治疗期间停止使用（26.2%），但这与英国的类似试验一致。结论：在这项研究中，在常规的慢性阻塞性肺疾病治疗中添加比索洛尔并没有降低恶化的可能性，比索洛尔不能被推荐作为慢性阻塞性肺疾病的治疗方法。未来的工作：将比索洛尔用于慢性阻塞性肺疾病患者心血管适应症的安全性的明确声明纳入适当的临床指南。试验注册：该试验注册号为ISRCTN10497306。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：15/130/20）资助，全文发表在《卫生技术评估》杂志上；第29卷第17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
Pramipexole in addition to mood stabilisers for treatment-resistant bipolar depression: the PAX-BD randomised double-blind placebo-controlled trial. 抗药双相抑郁患者在服用情绪稳定剂的同时服用普拉克索：PAX-BD随机双盲安慰剂对照试验
IF 3.5 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-05-01 DOI: 10.3310/HBFC1953
Hamish McAllister-Williams, Nicola Goudie, Lumbini Azim, Victoria Bartle, Michael Berger, Chrissie Butcher, Thomas Chadwick, Emily Clare, Paul Courtney, Lyndsey Dixon, Nichola Duffelen, Tony Fouweather, William Gann, John Geddes, Sumeet Gupta, Beth Hall, Timea Helter, Paul Hindmarch, Eva-Maria Holstein, Ward Lawrence, Phil Mawson, Iain McKinnon, Adam Milne, Aisling Molloy, Abigail Moore, Richard Morriss, Anisha Nakulan, Judit Simon, Daniel Smith, Bryony Stokes-Crossley, Paul Stokes, Andrew Swain, Zoë Walmsley, Christopher Weetman, Allan H Young, Stuart Watson
<p><strong>Background: </strong>There are limited options currently recommended in National Institute for Health and Care Excellence guidelines for the treatment of bipolar depression. Pramipexole has been shown to improve mood symptoms in two small pilot studies in such patients.</p><p><strong>Objectives: </strong>Primary: to evaluate the clinical effectiveness of pramipexole versus placebo alongside routine mood-stabilising medications over 12 weeks in patients with treatment-resistant bipolar depression. Secondary: evaluate the impact of pramipexole on mood and anxiety, psychosocial function, cost-effectiveness, and safety and tolerability over 48 weeks.</p><p><strong>Design: </strong>Multicentre, randomised, placebo-controlled trial of pramipexole versus placebo in addition to standard-of-care mood stabilisers. Clinicians, researchers and participants were blinded throughout the duration of the study. Pre-randomisation stage (to adjust antipsychotics or commence mood stabilisers where required) before randomisation. Weekly online assessments of mood and anxiety from randomisation to week 52, with psychosocial function, quality of life and healthcare resource utilisation assessments conducted at regular intervals.</p><p><strong>Setting: </strong>Twenty-one National Health Service trusts and Health Boards across England and Scotland.</p><p><strong>Participants: </strong>Patients aged 18 years and over with a diagnosis of treatment-resistant bipolar depression currently under secondary care mental health services. Aim to randomise 290 participants.</p><p><strong>Interventions: </strong>Pramipexole or matched placebo orally once daily, titrated from 0.25 mg to maximum of 2.5 mg (salt weight) depending on efficacy and tolerability.</p><p><strong>Main outcome measures: </strong>Depression - Quick Inventory for Depressive Symptomology; anxiety - Generalised Anxiety Disorder-7-item scale; psychosocial functioning - Work and Social Adjustment Scale; hypomania/mania - Altman Self-rating Scale of Mania; tolerability - Treatment Satisfaction Questionnaire for Medication; well-being and quality of life - EuroQol-5 Dimensions, five-level version, ICEpop CAPability measure for Adults and Oxford CAPabilities questionnaire-Mental Health tools.</p><p><strong>Results: </strong>Thirty-nine participants randomised (18 to pramipexole and 21 to placebo) with 36 providing data for the primary analysis. Pramipexole led to greater reductions in depressive symptoms at 12 weeks compared to placebo [4.4 (4.8) vs. 2.1 (5.1)]: a medium-sized (<i>d</i> = -0.72) but not statistically significant difference (95% confidence interval -0.4 to 6.3; <i>p</i> = 0.087). There were some statistically significant positive effects of pramipexole on secondary outcomes (reduction in depressive symptoms at 36 weeks, response and remission rates at trial exit, psychosocial function). Pramipexole was associated with an increased rate of hypomania/manic symptoms, but this appeared to be redu
背景：目前，国家健康与护理研究所推荐的治疗双相抑郁症的方法有限。在两项针对此类患者的小型初步研究中，普拉克索已被证明可以改善情绪症状。目的：主要：在难治性双相抑郁症患者中，评估丙克索与安慰剂联合常规情绪稳定药物治疗12周的临床疗效。其次：评估普拉克索在48周内对情绪和焦虑、社会心理功能、成本效益、安全性和耐受性的影响。设计：多中心、随机、安慰剂对照试验，比较普拉克索与安慰剂以及标准护理情绪稳定剂。临床医生、研究人员和参与者在整个研究期间都是盲法的。随机化前阶段（调整抗精神病药物或在需要时开始使用情绪稳定剂）。从随机分组到第52周，每周进行情绪和焦虑在线评估，定期进行心理社会功能、生活质量和医疗资源利用评估。环境：横跨英格兰和苏格兰的21个国家卫生服务信托和卫生委员会。参与者：年龄在18岁及以上，诊断为难治性双相抑郁症的患者，目前正在接受二级保健精神卫生服务。目标是随机抽取290名参与者。干预措施：每天口服一次普拉克索或匹配的安慰剂，根据疗效和耐受性从0.25 mg滴定到最大2.5 mg（盐重）。主要观察指标：抑郁症-抑郁症状快速量表；焦虑-广泛性焦虑障碍-7项量表；社会心理功能-工作和社会适应量表；轻躁/狂躁-奥特曼狂躁自评量表；耐受性-用药治疗满意度问卷；幸福和生活质量- EuroQol-5维度，五级版本，ICEpop成人能力测量和牛津能力问卷-心理健康工具。结果：39名参与者随机分配（18名接受普拉克索治疗，21名接受安慰剂治疗），其中36名提供了初步分析的数据。与安慰剂相比，普拉克索在12周时导致抑郁症状的更大减轻[4.4（4.8）比2.1(5.1)]：中等（d = -0.72），但无统计学显著差异(95%置信区间-0.4至6.3；p = 0.087)。普拉克索对次要结局（36周时抑郁症状的减轻、试验结束时的反应和缓解率、社会心理功能）有一些统计学上显著的积极影响。普拉克索与轻度躁狂/躁狂症状的发生率增加有关，但与抗精神病药物共同使用似乎可以降低这种发生率。普拉克索的总体耐受性良好。在与健康有关的生活质量和能力-福祉以及降低健康和社会护理成本的趋势方面，每年都有显著的进步。局限性：由于在COVID-19大流行期间招募人员和试验提前结束，样本量小，随访时间可变。参与者仅限于那些在二级保健精神卫生服务的人。所有评估只提供英文版本。结论：由于普拉克索和安慰剂在主要疗效结局指标上没有显著差异，因此不建议在临床实践中进行改变。然而，有证据表明普拉克索对情绪、社会心理功能和生活质量有积极影响。未来的工作：在更大的人群中进行复制，并研究抗精神病药物与普拉克索共同使用的影响。试验注册：该试验注册号为ISRCTN72151939， EudraCT 2018-2869-18。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奖号：16/154/01）资助，全文发表在《卫生技术评估》杂志上；第29卷，第21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。
{"title":"Pramipexole in addition to mood stabilisers for treatment-resistant bipolar depression: the PAX-BD randomised double-blind placebo-controlled trial.","authors":"Hamish McAllister-Williams, Nicola Goudie, Lumbini Azim, Victoria Bartle, Michael Berger, Chrissie Butcher, Thomas Chadwick, Emily Clare, Paul Courtney, Lyndsey Dixon, Nichola Duffelen, Tony Fouweather, William Gann, John Geddes, Sumeet Gupta, Beth Hall, Timea Helter, Paul Hindmarch, Eva-Maria Holstein, Ward Lawrence, Phil Mawson, Iain McKinnon, Adam Milne, Aisling Molloy, Abigail Moore, Richard Morriss, Anisha Nakulan, Judit Simon, Daniel Smith, Bryony Stokes-Crossley, Paul Stokes, Andrew Swain, Zoë Walmsley, Christopher Weetman, Allan H Young, Stuart Watson","doi":"10.3310/HBFC1953","DOIUrl":"10.3310/HBFC1953","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;There are limited options currently recommended in National Institute for Health and Care Excellence guidelines for the treatment of bipolar depression. Pramipexole has been shown to improve mood symptoms in two small pilot studies in such patients.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;Primary: to evaluate the clinical effectiveness of pramipexole versus placebo alongside routine mood-stabilising medications over 12 weeks in patients with treatment-resistant bipolar depression. Secondary: evaluate the impact of pramipexole on mood and anxiety, psychosocial function, cost-effectiveness, and safety and tolerability over 48 weeks.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;Multicentre, randomised, placebo-controlled trial of pramipexole versus placebo in addition to standard-of-care mood stabilisers. Clinicians, researchers and participants were blinded throughout the duration of the study. Pre-randomisation stage (to adjust antipsychotics or commence mood stabilisers where required) before randomisation. Weekly online assessments of mood and anxiety from randomisation to week 52, with psychosocial function, quality of life and healthcare resource utilisation assessments conducted at regular intervals.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Twenty-one National Health Service trusts and Health Boards across England and Scotland.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Patients aged 18 years and over with a diagnosis of treatment-resistant bipolar depression currently under secondary care mental health services. Aim to randomise 290 participants.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Pramipexole or matched placebo orally once daily, titrated from 0.25 mg to maximum of 2.5 mg (salt weight) depending on efficacy and tolerability.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;Depression - Quick Inventory for Depressive Symptomology; anxiety - Generalised Anxiety Disorder-7-item scale; psychosocial functioning - Work and Social Adjustment Scale; hypomania/mania - Altman Self-rating Scale of Mania; tolerability - Treatment Satisfaction Questionnaire for Medication; well-being and quality of life - EuroQol-5 Dimensions, five-level version, ICEpop CAPability measure for Adults and Oxford CAPabilities questionnaire-Mental Health tools.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Thirty-nine participants randomised (18 to pramipexole and 21 to placebo) with 36 providing data for the primary analysis. Pramipexole led to greater reductions in depressive symptoms at 12 weeks compared to placebo [4.4 (4.8) vs. 2.1 (5.1)]: a medium-sized (&lt;i&gt;d&lt;/i&gt; = -0.72) but not statistically significant difference (95% confidence interval -0.4 to 6.3; &lt;i&gt;p&lt;/i&gt; = 0.087). There were some statistically significant positive effects of pramipexole on secondary outcomes (reduction in depressive symptoms at 36 weeks, response and remission rates at trial exit, psychosocial function). Pramipexole was associated with an increased rate of hypomania/manic symptoms, but this appeared to be redu","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 21","pages":"1-216"},"PeriodicalIF":3.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12146921/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144198956","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
Anti-VEGF drugs compared with laser photocoagulation for the treatment of diabetic retinopathy: a systematic review and economic analysis. 抗vegf药物与激光光凝治疗糖尿病视网膜病变的比较：系统综述和经济分析。
IF 3.5 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-05-01 DOI: 10.3310/KRWP1264
Mark Simmonds, Matthew Walton, Rob Hodgson, Alexis Llewellyn, Ruth Walker, Helen Fulbright, Laura Bojke, Lesley Stewart, Sofia Dias, Thomas Rush, John Lawrenson, Tunde Peto, David Steel
<p><strong>Background: </strong>Diabetic retinopathy is a major cause of sight loss in people with diabetes, with a high risk of macular oedema, vitreous haemorrhage or other complications. Panretinal photocoagulation is the primary treatment for proliferative retinopathy. Anti-vascular endothelial growth factor drugs are used to treat various eye conditions and may be beneficial for people with proliferative or non-proliferative retinopathy.</p><p><strong>Methods: </strong>The Anti-VEGF In Diabetes project sought to investigate the clinical and cost-effectiveness of using anti-vascular endothelial growth factor to prevent retinopathy progression when compared to panretinal photocoagulation or no treatment. A systematic review with network meta-analysis of randomised controlled trials of anti-vascular endothelial growth factor (alone or in combination with panretinal photocoagulation) to treat retinopathy was conducted. The database searches were updated in May 2023. Individual participant data from larger trials were sought. A systematic review of non-randomised studies was performed. Existing cost-effectiveness analyses were reviewed, and a new economic model was developed, informed by the individual participant data meta-analysis. The model also estimated the value of undertaking further research to resolve decision uncertainty.</p><p><strong>Results: </strong>The review found that anti-vascular endothelial growth factors produced a slight, and not clinically meaningful, benefit over panretinal photocoagulation in best corrected visual acuity, after 1 year of follow-up in people with proliferative retinopathy (mean difference of 4.5 ETDRS letters; 95% credible interval -0.7 to 8.2). There was no evidence of a difference in effectiveness among the different anti-vascular endothelial growth factors. The benefit of anti-vascular endothelial growth factor appears to decline over time. Anti-vascular endothelial growth factor therapy may be more effective in people with poorer initial visual acuity. Anti-vascular endothelial growth factor had no impact on vision in people with non-proliferative retinopathy. Anti-vascular endothelial growth factor reduces rates of macular oedema and vitreous haemorrhage and may slow down the progression of retinopathy. Anti-vascular endothelial growth factors were predicted to be more costly but similarly effective to panretinal photocoagulation, with a net health benefit of -0.214 quality-adjusted life-years at a £20,000 willingness-to-pay threshold. Only under very select conditions might anti-vascular endothelial growth factors have the potential for cost-effectiveness to treat proliferative retinopathy. There is potentially significant value in reducing uncertainty through further primary research.</p><p><strong>Conclusions: </strong>Anti-vascular endothelial growth factor has no clinically meaningful benefit over panretinal photocoagulation for preserving visual acuity, but it may delay or prevent progression to
背景：糖尿病视网膜病变是糖尿病患者视力丧失的主要原因，伴有黄斑水肿、玻璃体出血或其他并发症的高风险。全视网膜光凝是增殖性视网膜病变的主要治疗方法。抗血管内皮生长因子药物用于治疗各种眼病，可能对患有增殖性或非增殖性视网膜病变的人有益。方法：糖尿病抗血管内皮生长因子项目旨在研究与全视网膜光凝或不治疗相比，使用抗血管内皮生长因子预防视网膜病变进展的临床和成本效益。通过网络荟萃分析对抗血管内皮生长因子（单独或联合全视网膜光凝）治疗视网膜病变的随机对照试验进行了系统回顾。数据库搜索于2023年5月更新。从大型试验中寻找个体参与者的数据。对非随机研究进行系统回顾。对现有的成本效益分析进行了回顾，并在个体参与者数据荟萃分析的基础上建立了一个新的经济模型。该模型还估计了进一步研究解决决策不确定性的价值。结果：该综述发现，在对增生性视网膜病变患者进行1年随访后，抗血管内皮生长因子在最佳矫正视力方面比全视网膜光凝治疗产生轻微的、无临床意义的益处(ETDRS字母平均值为4.5；95%可信区间为-0.7 ~ 8.2)。没有证据表明不同的抗血管内皮生长因子在有效性上存在差异。抗血管内皮生长因子的益处似乎随着时间的推移而下降。抗血管内皮生长因子治疗可能对初始视力较差的人更有效。抗血管内皮生长因子对非增殖性视网膜病变患者的视力没有影响。抗血管内皮生长因子可降低黄斑水肿和玻璃体出血的发生率，并可减缓视网膜病变的进展。据预测，抗血管内皮生长因子的成本更高，但对全视网膜光凝治疗同样有效，以2万英镑的愿意支付门槛计算，其净健康效益为-0.214质量调整生命年。只有在非常特定的条件下，抗血管内皮生长因子才有可能具有治疗增殖性视网膜病变的成本效益。通过进一步的初步研究减少不确定性具有潜在的重要价值。结论：与全视网膜光凝相比，抗血管内皮生长因子在保持视力方面没有临床意义的益处，但它可能延缓或预防黄斑水肿和玻璃体出血的进展。抗血管内皮生长因子治疗的长期有效性和安全性尚不清楚，特别是随着时间的推移，需要额外的全视网膜光凝和抗血管内皮生长因子治疗。因此，与全视网膜光凝相比，抗血管内皮生长因子不太可能是早期增殖性视网膜病变的一种经济有效的治疗方法。在各种情况下，它们通常与较高的成本和类似的健康结果相关。由于缺乏长期临床证据，抗血管内皮生长因子的长期成本效益尚不确定。未来的工作：进一步，需要超过2年的随访研究来评估抗血管内皮生长因子使用的长期疗效和安全性，以及额外的抗血管内皮生长因子和全视网膜光凝治疗随时间的影响。临床试验或观察性研究关注在治疗时视力较差的人使用抗血管内皮生长因子也可能是有用的。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NIHR132948。
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