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Clinical and cost-effectiveness of percutaneous nephrolithotomy, flexible ureterorenoscopy and extracorporeal shockwave lithotripsy for lower pole stones: the PUrE RCTs. 经皮肾镜、柔性输尿管镜和体外冲击波碎石术治疗下极结石的临床和成本效益：PUrE随机对照试验
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-08-01 DOI: 10.3310/WFRE6844
Oliver Wiseman, Daron Smith, Kath Starr, Lorna Aucott, Rodolfo Hernández, Ruth Thomas, Steven MacLennan, Charles Terry Clark, Graeme MacLennan, Dawn McRae, Victoria Bell, Seonaidh Cotton, Zara Gall, Ben Turney, Samuel McClinton
<p><strong>Background: </strong>Renal tract stone disease is common. The three intervention options are shockwave lithotripsy, flexible ureteroscopic stone treatment and keyhole surgery.</p><p><strong>Objectives: </strong>To determine which of shockwave lithotripsy, flexible ureteroscopic stone treatment and keyhole surgery offer the best outcomes in terms of health and quality of life, clinical effectiveness and cost-effectiveness for people with lower pole kidney stones.</p><p><strong>Design: </strong>The PUrE study comprised two pragmatic multicentre, open-label, superiority randomised controlled trials: RCT1 for lower pole stones ≤ 10 mm and RCT2 for lower pole stones > 10 and ≤ 25 mm.</p><p><strong>Setting: </strong>National Health Service Urology departments.</p><p><strong>Participants: </strong>Adults presenting with lower pole renal stones, able to undergo any of the treatments and complete trial procedures.</p><p><strong>Intervention: </strong>Eligible participants were randomised in RCT1 to flexible ureteroscopic stone treatment or shockwave lithotripsy; and in RCT2 to flexible ureteroscopic stone treatment or keyhole surgery.</p><p><strong>Main outcome measures: </strong>The primary outcome measure was health status 'area under the curve', measured weekly to 12 weeks post intervention with the EuroQol-5 Dimensions, five-level version. The primary economic outcome was the incremental cost per quality-adjusted life-year gained at 12 months from randomisation.</p><p><strong>Results: </strong><i>RCT1:</i> A total of 461 participants were randomised: 231 to flexible ureteroscopic stone treatment; and 230 to shockwave lithotripsy. <i>RCT2:</i> A total of 159 participants were randomised: 73 to flexible ureteroscopic stone treatment; and 86 to keyhole surgery.</p><p><strong>Primary outcome: </strong><i>RCT1:</i> The mean health status area under the curve was 0.807 (standard deviation 0.205) in the flexible ureteroscopic stone treatment group (<i>n</i> = 164) and 0.826 (standard deviation 0.207) in the shockwave lithotripsy group (<i>n</i> = 188). The between-group difference, 0.024 (95% confidence interval -0.004 to 0.053), was a small difference in favour of flexible ureteroscopic stone treatment after correcting for a baseline imbalance. Complete stone clearance was higher with flexible ureteroscopic stone treatment (72%) than shockwave lithotripsy (36%). <i>RCT2</i>: The mean health status area under the curve was 0.794 (standard deviation 0.198) in the flexible ureteroscopic stone treatment group (<i>n</i> = 57) and 0.818 (standard deviation 0.217) in the keyhole surgery group (<i>n</i> = 63). The between-group difference, -0.07 (95% confidence interval -0.11 to -0.02), was a borderline meaningful difference favouring keyhole surgery. Complete stone clearance was higher with keyhole surgery (71%) than flexible ureteroscopic stone treatment (48%).</p><p><strong>Economic evaluation: </strong><i>RCT1</i>: Flexible ureteroscopic stone treatm
背景：肾结石是一种常见的疾病。三种干预方法分别是冲击波碎石术、输尿管软镜结石治疗和锁孔手术。目的：探讨冲击波碎石术、输尿管软镜下结石治疗和锁孔手术中哪一种对下极肾结石患者的健康和生活质量、临床效果和成本效益最好。设计：PUrE研究包括两个实用的多中心、开放标签、优势随机对照试验：RCT1用于治疗≤10毫米的下极结石，RCT2用于治疗≤10毫米的下极结石。参与者：患有下极肾结石的成年人，能够接受任何治疗并完成试验程序。干预：符合条件的参与者在RCT1中随机分为输尿管软镜结石治疗或冲击波碎石术；而在RCT2中以输尿管软镜治疗结石或锁眼手术。主要结果测量：主要结果测量是健康状况“曲线下面积”，在干预后每周至12周用EuroQol-5维度，五级版本测量。主要的经济结果是随机化后12个月每个质量调整生命年的增量成本。结果：RCT1：共有461名参与者被随机分配：231名接受输尿管镜治疗；230是冲击波碎石术。RCT2：共有159名参与者被随机分配：73名接受输尿管镜治疗；86到钥匙孔手术。主要结局：RCT1：输尿管软镜结石治疗组（n = 164）的平均健康状况曲线下面积为0.807（标准差0.205），冲击波碎石组（n = 188）的平均健康状况曲线下面积为0.826（标准差0.207）。组间差异为0.024(95%可信区间为-0.004至0.053)，在纠正基线不平衡后，支持输尿管软性镜结石治疗的差异很小。输尿管软镜治疗结石的完全清除率（72%）高于冲击波碎石术（36%）。RCT2：输尿管软镜结石治疗组（n = 57）和锁眼手术组（n = 63）的平均曲线下健康状况面积分别为0.794（标准差0.198）和0.818（标准差0.217）。组间差异为-0.07(95%可信区间为-0.11至-0.02)，是支持锁孔手术的临界有意义差异。锁眼手术的结石完全清除率（71%）高于输尿管软镜结石治疗（48%）。经济评价：RCT1：输尿管软镜结石治疗成本更高（1138英镑，95%置信区间646 - 1631英镑），可额外增加0.017（95%置信区间-0.008 - 0.043）质量调整生命年；每个质量调整生命年的增量成本效益比为65 163英镑。冲击波碎石术在2万英镑的门槛下有99.9%的成本效益。RCT2：输尿管软腔镜结石治疗费用较高（733英镑；95%置信区间- 508英镑至1973英镑），质量调整生命年较少（-0.001；95%置信区间-0.044至0.042）。钥匙孔手术在2万英镑的门槛下有87%的成本效益。局限性：不可能对参与者和医疗保健提供者进行盲法。在RCT1中，干预措施之间的等待时间存在差异；然而，对此进行调整后得出了类似的治疗效果估计。结论：PUrE研究在RCT1中发现，冲击波碎石比输尿管软镜下的结石治疗更具成本效益，尽管输尿管软镜下的结石完全无结石率更高，但患者的健康状况没有显著差异。在RCT2中，在微观成本基础上，锁眼手术比输尿管软镜结石治疗更具成本效益，这更好地反映了治疗成本与NHS的差异。锁眼手术对健康状况有轻微益处，结石完全清除率较高。未来的工作：吸引装置、激光技术的改进和术中压力监测对输尿管软镜结石治疗的术后疼痛、生活质量、结石清除率、并发症和费用有什么影响？锁眼手术的小型化对术后疼痛、住院时间、并发症、无结石率和费用有什么影响？试验注册：该试验注册号为ISRCTN98970319。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：13/152/02）资助，全文发表在《卫生技术评估》杂志上；第29卷，第40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
Glycaemic control in labour with diabetes: GILD, a scoping study. 糖尿病分娩中的血糖控制：GILD，一项范围研究。
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-08-01 DOI: 10.3310/KHGD2761
Nia Wyn Jones, Eleanor J Mitchell, Kate F Walker, Susan Ayers, Lucy Bradshaw, Georgina Constantinou, Tasso Gazis, Shalini Ojha, Phoebe Pallotti, Stavros Petrou, Rachel Plachcinski, Michael Rimmer, Liz Schroeder, Jim G Thornton, Natalie Wakefield
<p><strong>Background: </strong>Diabetes in pregnancy is common, affecting 5-10% of pregnant women. Poor glycaemic control in labour is associated with neonatal hypoglycaemia and other adverse outcomes for mother and baby, but tight glucose control is burdensome, intrusive and may not always be necessary. The ideal intrapartum glucose target level is unknown, traditionally 'tight' control (target 4-7 mmol/l) has been recommended; however, this increases the risk of maternal hypoglycaemia.</p><p><strong>Objective: </strong>To determine the feasibility of a randomised clinical trial to compare clinical and cost-effectiveness of permissive versus intensive intrapartum glycaemic control in labour in pregnancies complicated by diabetes.</p><p><strong>Design: </strong>A mixed-methods study including audit of clinical guidelines from United Kingdom maternity units, online surveys of women with diabetes and healthcare professionals, service evaluation of intrapartum glycaemic care, Delphi survey and consensus meeting. Data from these work packages led to the design of a clinical trial, and qualitative interviews were held to understand acceptability of the trial.</p><p><strong>Setting: </strong>National Health Service maternity services and online input from service users.</p><p><strong>Participants: </strong>Healthcare professionals and women with type 1 or type 2 diabetes mellitus or gestational diabetes (currently pregnant or having birthed after active labour in past 3 years).</p><p><strong>Results: </strong>There is significant variation in the recommended frequency of testing for gestational diabetes in labour, technologies used to test glucose levels in labour and administer insulin in type 1 diabetes mellitus, and in how neonatal hypoglycaemia is defined. Of surveyed women, 66% would be willing to participate in a future trial, with 23% unsure without further information. The service evaluation showed that once glucose testing had commenced, it was repeated after 1 hour in 18%, 2 hours in 38% and 4 hours in 45% of women. Neonatal hypoglycaemia was considered the most important neonatal outcome for a future trial, with maternal satisfaction the most important maternal outcome. The incidence of neonatal hypoglycaemia (defined as glucose < 2.6 mmol/l) was 47% in type 1 diabetes mellitus, 45% in type 2 diabetes mellitus and 16% in gestational diabetes mellitus. A non-inferiority trial to compare permissive versus intensive glucose control was designed to include all types of diabetes in an umbrella trial (conduct more than one trial simultaneously). Women and healthcare professionals considered the trial design acceptable and feasible, though noted important considerations in the design and conduct.</p><p><strong>Limitations: </strong>Glucose levels may be poorly recorded in maternity notes and in practice tested more frequently than the study suggests. Sample sizes in some of the work packages were smaller than our pre-specified target, attrition in
背景：妊娠期糖尿病很常见，影响5-10%的孕妇。产程血糖控制不良与新生儿低血糖和母婴其他不良结局有关，但严格的血糖控制是繁重的，侵入性的，可能并不总是必要的。理想的产时血糖目标水平是未知的，传统上建议“严格”控制（目标4-7毫摩尔/升）；然而，这增加了产妇低血糖的风险。目的：确定一项随机临床试验的可行性，以比较糖尿病合并妊娠分娩时放任与强化产时血糖控制的临床和成本效益。设计：一项混合方法研究，包括对英国妇产单位临床指南的审核，对糖尿病妇女和医疗保健专业人员的在线调查，对分娩时血糖护理的服务评估，德尔菲调查和共识会议。来自这些工作包的数据导致了临床试验的设计，并进行了定性访谈以了解试验的可接受性。背景：国民保健服务的生育服务和服务用户的在线输入。参与者：医疗保健专业人员和患有1型或2型糖尿病或妊娠期糖尿病的妇女（目前怀孕或在过去3年内产程后分娩）。结果：在产程妊娠糖尿病的推荐检测频率、用于产程血糖水平检测和1型糖尿病患者胰岛素使用的技术以及如何定义新生儿低血糖方面存在显著差异。在接受调查的女性中，66%的人愿意参加未来的试验，23%的人在没有进一步信息的情况下不确定。服务评估显示，一旦开始葡萄糖检测，18%的女性在1小时后重复检测，38%的女性在2小时后重复检测，45%的女性在4小时后重复检测。新生儿低血糖被认为是未来试验中最重要的新生儿结局，而产妇满意度是最重要的产妇结局。新生儿低血糖的发生率（定义为葡萄糖限制）：产褥单上的血糖水平可能记录得很差，而且实际检测的频率比研究表明的要高。一些工作包的样本量小于我们预先指定的目标，德尔菲调查的损耗率大于预期，并且该研究是在COVID-19大流行影响结果期间进行的。愿意参加一个假设的试验可能不会转化为招募到一个真正的试验。结论：一项使用主方案的伞形试验旨在比较所有类型糖尿病的严格血糖控制（标准护理）和更宽松控制。对于怀孕期间有糖尿病经历的妇女以及参加定性访谈的妇女和保健专业人员来说，这样的试验是可行和可接受的。未来的工作：我们建议未来的随机试验应该包括一个内部试点阶段，以测试试验行为的关键方面和明确的进展标准，考虑到我们在这个范围研究中发现的挑战。研究注册：本研究注册号为researchregistry6832。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：NIHR130175）资助，全文发表在《卫生技术评估》上；第29卷，第41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
Medical management and intervention (using neurosurgical resection or stereotactic radiosurgery) versus medical management alone for symptomatic brain cavernoma: the CARE pilot RCT. 医疗管理和干预（使用神经外科切除或立体定向放射外科）与单独医疗管理对症脑海绵瘤：CARE先导RCT
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-08-01 DOI: 10.3310/GJRS5321
Rustam Al-Shahi Salman, Neil Kitchen, Laura Forsyth, Vijeya Ganesan, Peter S Hall, Kirsty Harkness, Peter Ja Hutchinson, Steff C Lewis, Matthias Wr Radatz, Carole Turner, Julia Wade, David Cs White, Philip M White
<p><strong>Background: </strong>The top priority for research into symptomatic cerebral cavernous malformation (also known as brain cavernoma) is whether to have medical management and intervention (using neurosurgical resection or stereotactic radiosurgery) or medical management alone.</p><p><strong>Objectives: </strong>The primary objective was to assess the feasibility of performing a definitive randomised trial addressing this top priority. The secondary objectives were to set up a collaboration involving patient advocacy organisations and clinicians in the United Kingdom and Ireland; perform a QuinteT Recruitment Intervention to identify facilitators and address barriers to recruitment; and conduct a pilot randomised trial with ≈60 participants.</p><p><strong>Design: </strong>Prospective, randomised, open-label, assessor-blinded, parallel-group trial. A mixed-methods QuinteT Recruitment Intervention analysed sites' screening logs and qualitative data from audio-recorded recruitment discussions, interviews with healthcare professionals and patients, investigator workshops and observation of meetings.</p><p><strong>Setting: </strong>Neuroscience hospitals in the United Kingdom and Ireland.</p><p><strong>Participants: </strong>We aimed to recruit ≈60 people of any age, gender and ethnicity who had mental capacity, resided in the United Kingdom/Ireland, and had a brain cavernoma that had caused symptoms due to intracranial haemorrhage, non-haemorrhagic progressive/persistent focal neurological deficit or epileptic seizure(s).</p><p><strong>Interventions: </strong>We identified and addressed barriers and facilitators to optimise informed consent and recruitment. Computerised, web-based randomisation assigned participants (1 : 1) to treatment of their symptomatic brain cavernoma with medical management and intervention (using neurosurgical resection or stereotactic radiosurgery) or medical management alone. Assignment was open to investigators, participants and carers but not clinical outcome event adjudicators.</p><p><strong>Main outcome measures: </strong>Feasibility outcomes included site engagement, recruitment, choice of surgical management, retention, adherence, data quality, clinical outcome event rate and protocol implementation. The primary clinical outcome was symptomatic intracranial haemorrhage or new persistent/progressive non-haemorrhagic focal neurological deficit due to brain cavernoma or intervention during ≥ 6 months of follow-up.</p><p><strong>Results: </strong>Investigators screened 511 patients at 28/40 (70%) sites in the United Kingdom: 322 (63%) eligible, 202 (63%) approached, 96 (48%) uncertain about whether to have intervention and 72 participants [median age was 51 years (interquartile range 39-59), 41 (57%) female, 66 (92%) white, 56 (78%) with prior intracranial haemorrhage and 28 (39%) with prior epileptic seizure] were randomly assigned to medical management and intervention (<i>n</i> = 36; 12 to neurosurgical resecti
背景：对症状性脑海绵状畸形（也称脑海绵状瘤）的研究，最重要的是是否进行药物治疗和干预（采用神经外科切除或立体定向放射外科）或单纯药物治疗。目的：主要目的是评估进行明确的随机试验解决这一首要问题的可行性。次要目标是在联合王国和爱尔兰建立一个涉及患者倡导组织和临床医生的合作；实施“招聘五要素干预”，以确定促进因素并解决招聘障碍；并进行一项有大约60名参与者的先导随机试验。设计：前瞻性、随机、开放标签、评估盲、平行组试验。混合方法的QuinteT招聘干预分析了网站的筛选日志和定性数据，这些数据来自招聘讨论的录音、对医疗保健专业人员和患者的访谈、调查员研讨会和会议观察。地点：英国和爱尔兰的神经科学医院。受试者：我们的目标是招募约60名任何年龄、性别和种族的人，这些人有智力，居住在英国/爱尔兰，患有脑海绵瘤，并因颅内出血、非出血性进行性/持续性局灶性神经功能缺损或癫痫发作而引起症状。干预措施：我们确定并解决了障碍和促进因素，以优化知情同意和招募。计算机化、基于网络的随机化将参与者（1:1）分配到医疗管理和干预（使用神经外科切除或立体定向放射外科）或单独医疗管理的症状性脑海绵瘤治疗。分配对研究者、参与者和护理人员开放，但不包括临床结果事件评审人员。主要结局指标：可行性结局包括手术地点参与、招募、手术方式选择、保留、依从性、数据质量、临床结局事件发生率和方案执行情况。在≥6个月的随访期间，主要临床结果为症状性颅内出血或脑海绵瘤或干预引起的新的持续/进行性非出血性局灶性神经功能缺损。结果：研究人员在英国28/40（70%）个地点筛选了511名患者：322名（63%）符合条件，202名（63%）接近，96名（48%）不确定是否进行干预，72名参与者[中位年龄51岁（四分位数范围39-59），41名（57%）女性，66名（92%）白人，56名（78%）既往颅内出血和28名（39%）既往癫痫发作]被随机分配到医疗管理和干预组(n = 36；12例行神经外科切除，24例行立体定向放射外科)或单纯内科治疗（n = 36）。67名参与者完成了随访（保留率93%），依从性为91%。招募的障碍包括常规护理实践和立体定向放射手术的后勤问题，而促进因素是神经外科医生准备为比常规护理更多的人提供干预，多学科团队平衡，并将研究作为平衡的解决方案。主要临床结果发生在2/33的医疗管理和干预组和2/34的单独医疗管理组。没有死亡或严重的不良事件。限制：我们无法激活爱尔兰的网站。我们的研究结果在英国以外的普遍性尚不清楚。结论：该试点随机试验确定了招聘的促进因素和障碍，超出了招聘目标，并达到了一些可行性指标。未来的工作：一项明确的随机试验将需要国际资助者和临床医生、研究人员和患者群体网络的广泛参与，以招募590-1900名参与者。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NIHR128694。
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引用次数: 0
Developing evidence-based guidelines for describing potential benefits and harms within patient information leaflets/sheets (PILs) that inform and do not cause harm (PrinciPILs). 制定以证据为基础的指南，在告知且不造成伤害的患者信息单张（pill）中描述潜在的益处和危害（原则）。
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-08-01 DOI: 10.3310/GJJH2402
Jeremy Howick, Martina Svobodova, Shaun Treweek, Katie Gillies, Adrian Edwards, Peter Bower, Jennifer Bostock, Nina Jacob, Kerenza Hood

Background: Variation in the way information about potential trial intervention benefits and harms is conveyed within patient information leaflets can cause avoidable information-induced ('nocebo') harm, research waste, and may be unethical.

Objectives: 1. To develop stakeholder-informed principles to guide how to describe information about potential trial intervention benefits and harms within patient information leaflets. 2. To test whether using these principles are feasible for testing in trials that measure whether they improve recruitment and adverse event rates. 3. To develop and disseminate guidance on how to implement the principles.

Methods: We used a mixed methodology consisting of three work packages. Work package 1 involved a modified Delphi survey and consensus meeting to develop the principles for harmonising the way information regarding potential benefits and harms are shared. Work package 2 involved testing whether the principles could be used to transform existing patient information leaflets by recruiting host trials to compare standard patient information leaflets with patient information leaflets developed using the principles 'principled patient information leaflets'. We also set up an infrastructure to test whether they could reduce variation, impact trial recruitment and reduce reported adverse events. Work package 3 involved developing and disseminating guidance for using the principles.

Results: For work package 1, 250 participants completed the Delphi survey and 7 principles were agreed upon: (1) all potential intervention harms should be listed, (2) potential harms should be separated into 'serious' and 'less serious', (3) if not all potential harms are known, this needs to be explicitly stated, (4) all potential benefits should be listed, (5) potential benefits and harms associated with trial participation need to be compared with those associated with non-participation, (6) suitable visual representations should be added where appropriate, and (7) information about potential benefits and harms should not be separated by more than one page. For work package 2, we developed principled patient information leaflets for five host trials and interviewed two members of each host trial team. Two host trials agreed to compare the patient information leaflets with principled patient information leaflets using Studies Within a Trial, and we published a protocol for a meta-analysis that will synthesise the results. For work package 3, 25 participants attended a hybrid workshop and recommended that researchers and Research Ethics Committee members should use the principles to design and evaluate patient information leaflets. We produced a guidance booklet and website, which are currently being used by some Health Research Authority Research Ethics Committees.

Conclusions: A strong consensus was reached regarding seven

背景：在患者信息单张中传达潜在试验干预益处和危害信息的方式变化可能导致可避免的信息诱导（“反安慰剂”）伤害、研究浪费，并且可能是不道德的。目的:1。制定利益相关者知情原则，以指导如何在患者信息单张中描述有关潜在试验干预益处和危害的信息。2. 为了检验在试验中使用这些原则是否可行，以衡量它们是否能提高招募率和不良事件发生率。3. 制定和传播关于如何实施这些原则的指导意见。方法：我们采用了由三个工作包组成的混合方法。工作包1涉及修改后的德尔菲调查和共识会议，以制定协调有关潜在利益和危害的信息共享方式的原则。工作包2涉及通过招募宿主试验来比较标准患者信息传单与使用“原则性患者信息传单”原则开发的患者信息传单，以测试这些原则是否可用于改造现有的患者信息传单。我们还建立了一个基础设施来测试它们是否可以减少变异、影响试验招募和减少报告的不良事件。工作包3涉及制定和传播使用这些原则的指南。结果：对于工作包，1,250名参与者完成了德尔菲调查，并达成了7项原则：(1)应列出所有潜在的干预危害；(2)应将潜在危害分为“严重”和“不太严重”；(3)如果不知道所有潜在危害，则需要明确说明；(4)应列出所有潜在益处；(5)需要将参与试验的潜在益处和危害与不参与试验的潜在益处和危害进行比较。(6)应在适当的地方添加适当的视觉表现，(7)有关潜在利益和危害的信息不应分开超过一页。对于工作包2，我们为五个宿主试验制定了原则性的患者信息传单，并采访了每个宿主试验小组的两名成员。两个宿主试验同意使用试验内研究将患者信息传单与原则性患者信息传单进行比较，我们发表了一项将综合结果的荟萃分析方案。对于工作包3,25名参与者参加了一个混合研讨会，并建议研究人员和研究伦理委员会成员应使用这些原则来设计和评估患者信息传单。我们制作了指导手册和网站，目前正在一些卫生研究机构的研究伦理委员会使用。结论：就七项原则达成了强烈的共识，这些原则可以协调有关试验干预措施潜在益处和危害的信息共享方式。这些原则可能会减少研究浪费和可避免的信息引起的伤害，并可能加强临床试验伦理。限制：由于COVID-19，美国国家卫生与保健研究所对正在进行的试验进行审查，导致若干试验的资金终止，以及试验工作人员压力很大，能力有限，无法将试验中的研究添加到他们的试验中，我们不得不修改第二个目标。虽然我们最初打算实际进行试验中的研究，但我们取而代之的是：一项试验中研究的荟萃分析方案，关于减少变异需要的额外研究，额外的传播工作，以及一篇关于在患者信息传单中提及试验干预的潜在利弊的伦理要求的论文。未来的工作：未来的工作可以应用这些结果来探索如何协调在知情同意过程中研究人员和患者之间的口头对话中分享潜在利益和危害的方式。资助：该奖项由医学研究委员会和国家卫生与保健研究所（NIHR）更好的方法，更好的研究计划（MRC奖励参考：MR/V020706/1）资助，全文发表在《卫生技术评估》上；第29卷第43期
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引用次数: 0
Eradication of Helicobacter pylori for prevention of aspirin-associated peptic ulcer bleeding in adults over 65 years: the HEAT RCT. 根除幽门螺杆菌预防65岁以上成人阿司匹林相关消化性溃疡出血：HEAT随机对照试验
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-08-01 DOI: 10.3310/LLKF7871
C J Hawkey, Anthony J Avery, Carol Ac Coupland, Colin J Crooks, Jennifer S Dumbleton, Fd Richard Hobbs, Denise Kendrick, Michael Moore, Clive Morris, Gregory Rubin, Murray Smith, Diane Stevenson

Background: Peptic ulcers in patients on aspirin are associated with Helicobacter pylori infection. We investigated whether H. pylori eradication would protect against aspirin-associated ulcer bleeding.

Methods: The Helicobacter Eradication Aspirin Trial was a randomised placebo-controlled trial (European Union Drug Regulating Authorities Clinical Trials 2011-003425-96), conducted in United Kingdom primary care using routinely collected clinical data. Consenting participants aged ≥ 60 years prescribed aspirin ≤ 325 mg but not ulcerogenic or gastroprotective medication underwent C13 urea breath testing for H. pylori. Those with a positive test were randomised to receive either a combination of clarithromycin 500 mg, metronidazole 400 mg and lansoprazole 30 mg, or placebos twice daily for 7 days. The primary outcome, time to death or hospitalisation due to peptic ulcer bleeding, was analysed using a Cox proportional hazards model.

Findings: Between 14 September 2012 and 22 November 2017, 30,166 participants underwent H. pylori breath testing, 5367 had a positive result, 5352 were randomised to an intention-to-treat population of 2677 (eradication) and 2675 (placebo) and followed up for a median of 5.0 years (interquartile range 3.9-6.4). Statistical analysis of the primary outcome showed an overall hazard ratio of 0.69 [95% confidence interval 0.38 to 1.25; p = 0.22], but there was a significant departure from the proportional hazards assumption (p = 0.0068), requiring analysis split at the median time to event: 2.5 years. There was a significant reduction in the primary outcome in the eradication treatment group in the first 2.5 years (hazard ratio 0.35, 95% confidence interval 0.14 to 0.89; p = 0.028) but not the second period (hazard ratio 1.31, 95% confidence interval 0.55 to 3.11). The number needed to treat (first period) was 238 (95% confidence interval 184 to 1661). Results in the first 2.5 years remained significant when accounting for the competing risk of death (p = 0.028). During the study period, 657 participants died (306 in the eradication group and 351 in the controls group; hazard ratio 0.86, 95% confidence interval 0.74 to 1.01; p = 0.058). Malignancy was the most common cause of death and largely accounted for the numerical difference between the treatment groups. A health economic analysis found proactive screening not cost-effective, since the monetised benefits of the intervention in preventing a peptic ulcer bleed failed to outweigh the costs.

Interpretation: Helicobacter pylori eradication protects against aspirin-associated peptic ulcer bleeding, but this may not be sustained or cost-effective when applied non-selectively to our study population. The possibility that H. pylori eradication, on a background of aspirin use, might affect death from malignanc

背景：阿司匹林患者的消化性溃疡与幽门螺杆菌感染有关。我们研究了根除幽门螺杆菌是否能预防阿司匹林相关性溃疡出血。方法：根除幽门螺杆菌阿司匹林试验是一项随机安慰剂对照试验（European Union Drug regulation Authorities Clinical Trials 2011-003425-96），在英国初级保健机构使用常规收集的临床数据进行。年龄≥60岁的同意参与者服用阿司匹林≤325 mg，但不服用溃疡性或胃保护药物，并进行C13尿素呼气幽门螺杆菌检测。试验结果呈阳性的患者被随机分组，接受克拉霉素500毫克、甲硝唑400毫克和兰索拉唑30毫克的联合治疗，或安慰剂，每天两次，持续7天。使用Cox比例风险模型分析主要结局，死亡时间或消化性溃疡出血住院时间。研究结果：在2012年9月14日至2017年11月22日期间，30,166名参与者接受了幽门螺杆菌呼气测试，5367名结果呈阳性，5352名随机分配到意向治疗人群中，其中2677人（根除）和2675人（安慰剂），随访中位数为5.0年（四分位数间距为3.9-6.4）。主要结局的统计分析显示，总风险比为0.69[95%可信区间0.38 ~ 1.25；P = 0.22]，但与比例风险假设有显著差异（P = 0.0068），需要在事件发生的中位数时间（2.5年）进行分析。根除治疗组的主要转归在前2.5年显著降低（风险比0.35,95%可信区间0.14 ~ 0.89;p = 0.028），但在第二期无显著降低（风险比1.31,95%可信区间0.55 ~ 3.11）。需要治疗的人数（第一期）为238人（95%置信区间184 - 1661）。当考虑到竞争死亡风险时，前2.5年的结果仍然显著（p = 0.028）。在研究期间，657名参与者死亡（根除组306名，对照组351名；风险比0.86,95%可信区间0.74 ~ 1.01;p = 0.058）。恶性肿瘤是最常见的死亡原因，在很大程度上解释了治疗组之间的数字差异。一项健康经济分析发现，主动筛查不具有成本效益，因为干预预防消化性溃疡出血的货币化收益未能超过成本。解释：根除幽门螺杆菌可以防止阿司匹林相关的消化性溃疡出血，但当非选择性地应用于我们的研究人群时，这可能不持久或成本效益不高。在使用阿司匹林的背景下，根除幽门螺杆菌可能影响恶性肿瘤死亡的可能性值得进一步评估。局限性和未来工作：研究对象已经建立阿司匹林可能有助于低事件发生率。未来的研究应该调查在事件发生率较高时开始服用阿司匹林的受试者。试验注册：本试验注册号为ISRCTN10134725；ClinicalTrials.gov编号NCT01506986。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：09/55/52）资助，全文发表在《卫生技术评估》杂志上；第29卷，第42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":"Eradication of <i>Helicobacter pylori</i> for prevention of aspirin-associated peptic ulcer bleeding in adults over 65 years: the HEAT RCT.","authors":"C J Hawkey, Anthony J Avery, Carol Ac Coupland, Colin J Crooks, Jennifer S Dumbleton, Fd Richard Hobbs, Denise Kendrick, Michael Moore, Clive Morris, Gregory Rubin, Murray Smith, Diane Stevenson","doi":"10.3310/LLKF7871","DOIUrl":"10.3310/LLKF7871","url":null,"abstract":"<p><strong>Background: </strong>Peptic ulcers in patients on aspirin are associated with <i>Helicobacter pylori</i> infection. We investigated whether <i>H. pylori</i> eradication would protect against aspirin-associated ulcer bleeding.</p><p><strong>Methods: </strong>The <i>Helicobacter</i> Eradication Aspirin Trial was a randomised placebo-controlled trial (European Union Drug Regulating Authorities Clinical Trials 2011-003425-96), conducted in United Kingdom primary care using routinely collected clinical data. Consenting participants aged ≥ 60 years prescribed aspirin ≤ 325 mg but not ulcerogenic or gastroprotective medication underwent C13 urea breath testing for <i>H. pylori</i>. Those with a positive test were randomised to receive either a combination of clarithromycin 500 mg, metronidazole 400 mg and lansoprazole 30 mg, or placebos twice daily for 7 days. The primary outcome, time to death or hospitalisation due to peptic ulcer bleeding, was analysed using a Cox proportional hazards model.</p><p><strong>Findings: </strong>Between 14 September 2012 and 22 November 2017, 30,166 participants underwent <i>H. pylori</i> breath testing, 5367 had a positive result, 5352 were randomised to an intention-to-treat population of 2677 (eradication) and 2675 (placebo) and followed up for a median of 5.0 years (interquartile range 3.9-6.4). Statistical analysis of the primary outcome showed an overall hazard ratio of 0.69 [95% confidence interval 0.38 to 1.25; <i>p</i> = 0.22], but there was a significant departure from the proportional hazards assumption (<i>p</i> = 0.0068), requiring analysis split at the median time to event: 2.5 years. There was a significant reduction in the primary outcome in the eradication treatment group in the first 2.5 years (hazard ratio 0.35, 95% confidence interval 0.14 to 0.89; <i>p</i> = 0.028) but not the second period (hazard ratio 1.31, 95% confidence interval 0.55 to 3.11). The number needed to treat (first period) was 238 (95% confidence interval 184 to 1661). Results in the first 2.5 years remained significant when accounting for the competing risk of death (<i>p</i> = 0.028). During the study period, 657 participants died (306 in the eradication group and 351 in the controls group; hazard ratio 0.86, 95% confidence interval 0.74 to 1.01; <i>p</i> = 0.058). Malignancy was the most common cause of death and largely accounted for the numerical difference between the treatment groups. A health economic analysis found proactive screening not cost-effective, since the monetised benefits of the intervention in preventing a peptic ulcer bleed failed to outweigh the costs.</p><p><strong>Interpretation: </strong><i>Helicobacter pylori</i> eradication protects against aspirin-associated peptic ulcer bleeding, but this may not be sustained or cost-effective when applied non-selectively to our study population. The possibility that <i>H. pylori</i> eradication, on a background of aspirin use, might affect death from malignanc","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 42","pages":"1-62"},"PeriodicalIF":4.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12434569/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144951691","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
Ambulatory Oxygen for Pulmonary Fibrosis (OxyPuF): a randomised controlled trial and acceptability study. 动态供氧治疗肺纤维化（OxyPuF）：一项随机对照试验和可接受性研究。
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-02 DOI: 10.3310/TWKS4194
Rachel L Adams, Alisha Maher, Nicola Gale, Anjali Crawshaw, David Thickett, Alice M Turner
<p><strong>Introduction: </strong>Idiopathic pulmonary fibrosis is a devastating condition of unknown cause that results in progressive, irreversible scarring of the lung, manifesting as breathlessness and dry cough. Idiopathic pulmonary fibrosis is thought to be responsible for as many as 1 in 100 deaths in the United Kingdom, killing 5300 people a year. Ambulatory oxygen therapy is commonly used in idiopathic pulmonary fibrosis to relieve exertional breathlessness, although evidence to support this strategy is lacking. This pragmatic randomised controlled trial was planned to test whether use of ambulatory oxygen therapy is beneficial in people with idiopathic pulmonary fibrosis.</p><p><strong>Methods: </strong>We planned a randomised controlled trial in 260 patients with idiopathic pulmonary fibrosis who are breathless on exertion and do not meet criteria for long-term oxygen therapy, randomising in a 1 : 1 ratio between ambulatory oxygen therapy and best supportive care. Primary outcome was a quality-of-life questionnaire validated in pulmonary fibrosis, the King's Brief Interstitial Lung Disease questionnaire, measured at 6 months. We calculated our sample size based on the minimum clinically important difference of four units and standard deviation equal to 8.85 in King's Brief Interstitial Lung Disease questionnaire; assuming power of 90% and 5% two-sided significance level, thus required 130 per arm, after accounting for 20% dropout. The trials unit's web-based randomisation algorithm minimises on factors potentially influencing response to ambulatory oxygen therapy, such as severity of idiopathic pulmonary fibrosis, desaturation to < 88% present on walking, current or recent (within 6 months) pulmonary rehabilitation, and recruitment centre. Secondary outcomes included symptoms, exercise capacity and cost-effectiveness. A process evaluation included assessment of trial fidelity and acceptability of the intervention with use of qualitative research methods and arts approaches with patients and staff. Qualitative interviews were conducted with patients from the Ambulatory Oxygen for Pulmonary Fibrosis trial and the idiopathic pulmonary fibrosis patient support group Action for Pulmonary Fibrosis, and stakeholders: healthcare professionals and policy-makers. Interviews were audio-recorded, transcribed clean verbatim. Photovoice methodology was conducted with patients. A workshop prior to data collection informed and guided data collection and analysis. Traditional qualitative analysis and arts-based coproduction analysis approaches were used to produce a short film. An economic model was planned but could not occur due to early termination.</p><p><strong>Results: </strong>The trial was stopped prematurely due to low recruitment. This was due to a combination of the impact of COVID-19 on research infrastructure, financial issues for sites with the payment structure for the trial and lack of equipoise which limited site recruitment. Seven out
特发性肺纤维化是一种病因不明的破坏性疾病，可导致进行性、不可逆的肺部瘢痕形成，表现为呼吸困难和干咳。在英国，特发性肺纤维化被认为是导致高达百分之一死亡的原因，每年造成5300人死亡。动态氧疗通常用于特发性肺纤维化，以缓解运动性呼吸困难，尽管缺乏支持该策略的证据。这项实用的随机对照试验旨在测试动态氧疗对特发性肺纤维化患者是否有益。方法：我们计划了一项随机对照试验，纳入260例特发性肺纤维化患者，这些患者在运动时呼吸困难，不符合长期氧疗标准，在门诊氧疗和最佳支持治疗之间按1:1的比例随机分组。主要结果是在肺纤维化中验证的生活质量问卷，即6个月时测量的King’s Brief间质性肺病问卷。我们根据King's Brief间质性肺病问卷的最小临床重要差异4个单位和标准偏差等于8.85计算样本量；假设功率为90%，双侧显著性水平为5%，则在考虑20%的退组后，每组需要130个。试验单元基于网络的随机化算法最大限度地减少了可能影响动态氧疗反应的因素，如特发性肺纤维化的严重程度、去饱和。结果：由于招募人数少，试验提前停止。这是由于COVID-19对研究基础设施的影响，试验支付结构的站点的财务问题以及缺乏平衡限制了站点招聘的综合原因。25名符合条件且感兴趣的患者中有7名在预筛选后被随机分配，这意味着患者对该研究缺乏兴趣。基线特征表明患者年龄较大（平均81岁），以男性为主。对11名患者和23名其他利益相关者进行的定性研究得出结论，动态氧疗在英国是可取的、可接受的和广泛委托的，因此进一步的试验不太可能是可行的。结论：虽然我们无法正式确定评估特发性肺纤维化动态氧疗的疗效和成本效益的目标，但由于缺乏平衡，进行随机对照试验是不太可能可行的。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NIHR131149。
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引用次数: 0
Challenges to overcome in a randomised trial for Proper Understanding of Recurrent Stress Urinary Incontinence Treatment in women: the PURSUIT RCT. 正确理解女性复发性应激性尿失禁治疗的随机试验中需要克服的挑战：PURSUIT随机对照试验
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-02 DOI: 10.3310/AKAK8992
Caroline Pope, Madeleine Cochrane, Clare Clement, Yumeng Liu, Sangeetha Paramasivan, Sian Noble, Stephanie J MacNeill, Amanda L Lewis, Jodi Taylor, Bethanie Fitzgerald, Nikki Cotterill, Tamsin Greenwell, Hashim Hashim, Swati Jha, Nikesh Thiruchelvam, Philip Toozs-Hobson, Alison White, Wael Agur, J Athene Lane, Marcus Drake

Background: Recurrence or persistence of symptoms after interventions to treat stress urinary incontinence in women is common, but without robust evidence to base treatment recommendations.

Objectives: To investigate whether endoscopic or surgical treatments for stress urinary incontinence in women are effective and cost-effective.

Design: A multicentre, unblinded, parallel-group randomised controlled trial.

Setting: Fifteen centres across the United Kingdom.

Participants: Adult women with recurrent or persistent stress urinary incontinence.

Intervention: Individual randomisation to endoscopic (urethral bulking) or surgical (autologous sling, colposuspension, artificial urinary sphincter) interventions. Women randomised to surgery chose their operative intervention.

Main outcomes: Primary outcome self-reported International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form at 1 year post randomisation. Secondary outcomes included International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form, Patient Global Impression of Improvement and Pelvic Organ Prolapse/Urinary Incontinence Sexual questionnaires up to 3 years post randomisation, operative assessment measures and adverse events, cost-effectiveness from National Health Service and societal perspectives (quality-adjusted life-years and International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form) at 1 year, and a secondary care perspective (quality-adjusted life-years) at 3 years. Semistructured qualitative interviews at baseline (post randomisation), follow-up (3-6 months) and longer-term (12 and 36 months), to explore stress urinary incontinence generally, the acceptability and attitudes to treatments and to improve understanding of outcomes. Qualitative interviews with clinicians at baseline were focused on potential difficulties of recruitment and optimising patient-facing information and training materials for clinicians.

Results: Fifty-five women were deemed eligible after screening (n = 328 screened) from October 2019 to June 2022. Twenty-four eligible women consented, and 23 were randomised (between January 2020 and July 2022) from 8 sites with the average age of 57 years (standard deviation: 10.7) and all self-reported 'white' ethnicity. Participants reported a median International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form score at baseline of 16 (interquartile range: 13-19) and mean post-void residual volume of 4.64 ml (standard deviation: 8.45). Eleven participants received their allocated intervention, 2 participants withdrew prior to receiving their intervention and 10 were waiting for their intervention when the study closed. The most common reason for declining participation was a trea

背景：干预治疗压力性尿失禁后症状复发或持续是常见的，但没有强有力的证据来支持治疗建议。目的：探讨内窥镜或手术治疗女性压力性尿失禁是否有效和经济。设计：多中心、非盲、平行组随机对照试验。环境：在英国有15个中心。参与者：复发性或持续性压力性尿失禁的成年女性。干预：个体随机分为内窥镜（尿道膨胀）或手术（自体吊带、阴道悬吊、人工尿道括约肌）干预。随机接受手术的妇女选择手术干预。主要结局：主要结局：自我报告的国际失禁咨询问卷-尿失禁-短表在随机化后1年。次要结局包括国际失禁咨询问卷-尿失禁-简短形式，患者总体改善印象和盆腔器官脱垂/尿失禁随机分组后3年的性问卷，手术评估措施和不良事件，从国民健康服务和社会角度（质量调整生命年和国际咨询失禁问卷-尿失禁-简表）1年的成本效益，以及从二级保健角度（质量调整生命年）3年的成本效益。在基线（随机化后）、随访（3-6个月）和更长期（12和36个月）进行半结构化定性访谈，以探讨压力性尿失禁的一般情况、对治疗的可接受性和态度，并提高对结果的理解。在基线与临床医生的定性访谈集中在招募和优化面向患者的信息和临床医生培训材料的潜在困难。结果：从2019年10月到2022年6月，55名女性在筛查后被认为符合条件（n = 328）。24名符合条件的女性表示同意，其中23名是随机抽取的（在2020年1月至2022年7月之间），来自8个站点，平均年龄为57岁（标准差：10.7），所有女性都自称为“白人”种族。参与者报告了基线时尿失禁问卷-尿失禁-简短形式国际咨询得分的中位数为16（四分位数范围：13-19），平均尿后残余容量为4.64 ml（标准差：8.45）。11名参与者接受了分配给他们的干预，2名参与者在接受干预之前退出，10名参与者在研究结束时等待干预。减少参与的最常见原因是治疗偏好（n = 14）。制定和实施了招聘培训课程和招聘提示文件，以解决以患者治疗偏好和临床医生平衡为中心的挑战。然而，最重要的招募挑战是符合条件的患者数量少，这主要是由于COVID-19大流行预防转诊和手术，以及国家卫生服务体系中相关的更广泛问题，导致研究于2023年1月关闭。结论：在早期阶段，最初的招募率达到了目标（在招募的前3个月随机分配了4名参与者），但一旦大流行开始，该研究就无法招募，因此提前结束。主要的限制是在征聘开始后不久就发生了全球大流行病，严重影响了服务的提供和病人的就诊情况。在正常的医疗服务条件下，该研究可能是可交付的。局限性：未能在大流行条件下招募人员使研究不可行。未来研究：研究和开发面向患者和员工培训材料的实践经验将有助于在患者转诊和医疗保健服务完全恢复正常后交付研究。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17/95/03。
{"title":"Challenges to overcome in a randomised trial for Proper Understanding of Recurrent Stress Urinary Incontinence Treatment in women: the PURSUIT RCT.","authors":"Caroline Pope, Madeleine Cochrane, Clare Clement, Yumeng Liu, Sangeetha Paramasivan, Sian Noble, Stephanie J MacNeill, Amanda L Lewis, Jodi Taylor, Bethanie Fitzgerald, Nikki Cotterill, Tamsin Greenwell, Hashim Hashim, Swati Jha, Nikesh Thiruchelvam, Philip Toozs-Hobson, Alison White, Wael Agur, J Athene Lane, Marcus Drake","doi":"10.3310/AKAK8992","DOIUrl":"10.3310/AKAK8992","url":null,"abstract":"<p><strong>Background: </strong>Recurrence or persistence of symptoms after interventions to treat stress urinary incontinence in women is common, but without robust evidence to base treatment recommendations.</p><p><strong>Objectives: </strong>To investigate whether endoscopic or surgical treatments for stress urinary incontinence in women are effective and cost-effective.</p><p><strong>Design: </strong>A multicentre, unblinded, parallel-group randomised controlled trial.</p><p><strong>Setting: </strong>Fifteen centres across the United Kingdom.</p><p><strong>Participants: </strong>Adult women with recurrent or persistent stress urinary incontinence.</p><p><strong>Intervention: </strong>Individual randomisation to endoscopic (urethral bulking) or surgical (autologous sling, colposuspension, artificial urinary sphincter) interventions. Women randomised to surgery chose their operative intervention.</p><p><strong>Main outcomes: </strong>Primary outcome self-reported International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form at 1 year post randomisation. Secondary outcomes included International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form, Patient Global Impression of Improvement and Pelvic Organ Prolapse/Urinary Incontinence Sexual questionnaires up to 3 years post randomisation, operative assessment measures and adverse events, cost-effectiveness from National Health Service and societal perspectives (quality-adjusted life-years and International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form) at 1 year, and a secondary care perspective (quality-adjusted life-years) at 3 years. Semistructured qualitative interviews at baseline (post randomisation), follow-up (3-6 months) and longer-term (12 and 36 months), to explore stress urinary incontinence generally, the acceptability and attitudes to treatments and to improve understanding of outcomes. Qualitative interviews with clinicians at baseline were focused on potential difficulties of recruitment and optimising patient-facing information and training materials for clinicians.</p><p><strong>Results: </strong>Fifty-five women were deemed eligible after screening (<i>n</i> = 328 screened) from October 2019 to June 2022. Twenty-four eligible women consented, and 23 were randomised (between January 2020 and July 2022) from 8 sites with the average age of 57 years (standard deviation: 10.7) and all self-reported 'white' ethnicity. Participants reported a median International Consultation on Incontinence Questionnaire-Urinary Incontinence-Short Form score at baseline of 16 (interquartile range: 13-19) and mean post-void residual volume of 4.64 ml (standard deviation: 8.45). Eleven participants received their allocated intervention, 2 participants withdrew prior to receiving their intervention and 10 were waiting for their intervention when the study closed. The most common reason for declining participation was a trea","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-43"},"PeriodicalIF":4.0,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376003/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144560002","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
Outcomes of specialist physiotherapy for functional motor disorder: the Physio4FMD RCT. 功能性运动障碍专科物理治疗的结果：Physio4FMD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/MKAC9495
Glenn Nielsen, Louise Marston, Rachael Maree Hunter, Alan Carson, Laura H Goldstein, Kate Holt, Teresa C Lee, Marie Le Novere, Jonathan Marsden, Irwin Nazareth, Hayley Noble, Markus Reuber, Jon Stone, Ann-Marie Strudwick, Beatriz Santana Suarez, Mark J Edwards
<p><strong>Background: </strong>Functional motor disorder often causes persistent disabling symptoms that are associated with high healthcare costs. In recent years, specialist physiotherapy, informed by an understanding of functional motor disorder, has emerged as a promising treatment, but there is an absence of evidence of its effectiveness from large randomised controlled trials.</p><p><strong>Methods: </strong>We conducted a pragmatic, multicentre, randomised controlled trial, comparing specialist physiotherapy for functional motor disorder to treatment as usual, which was defined as community neurological physiotherapy. The primary outcome was the Short Form questionnaire-36 items Physical Functioning domain at 12 months (scale range 0-100, with 100 indicating optimum health). The trial was powered to detect a 9-point difference in the primary outcome with 90% power at the 5% level of significance. Secondary domains of measurement included a patient perception of improvement, health-related quality of life, mobility, anxiety, depression and illness perception. We also completed a health economic analysis with the primary aim of calculating the mean incremental cost per quality-adjusted life-year over 12 months. In prespecified analysis plans, we excluded participants from the primary analysis if they were unable to receive their trial-allocated treatment due to COVID-19 lockdown restrictions. Sensitivity analysis explored the impact of this decision.</p><p><strong>Results: </strong>Between 19 October 2018 and 31 January 2022, 355 adults with functional motor disorder were randomised (1 : 1) to specialist physiotherapy (<i>n</i> = 179) and treatment as usual (<i>n</i> = 176). Eighty-nine participants were excluded due to COVID-19 disruptions. Retention for the primary analysis was 90% for both groups, leaving 241 participants in the primary analysis. At 12 months, there was no between-group difference in the primary outcome (adjusted mean difference 3.5, 95% confidence interval -2.3 to 9.3). However, several secondary outcomes favoured specialist physiotherapy, including the participant perception of improvement, Short Form questionnaire-36 items Mental Health domain, confidence in the diagnosis and two subscales (Personal Control and Illness Coherence) of the Revised Illness Perception Questionnaire. There were no differences in the remaining outcomes. At 6 months, the following outcome measures were significantly different, in favour of specialist physiotherapy: participant perception of improvement, the Short Form questionnaire-36 items Physical Role Limitations, Short Form questionnaire-36 items Social Functioning, Short Form questionnaire-36 items Mental Health, EuroQol-5 Dimensions five-level version utility score, confidence in the diagnosis and three subscales (Timeline Cyclical, Personal Control and Treatment Control) of the Revised Illness Perception Questionnaire. No outcomes significantly favoured treatment as usual. In the healt
背景：功能性运动障碍通常会导致持续的残疾症状，这些症状与高昂的医疗费用相关。近年来，基于对功能性运动障碍的了解，专业物理疗法已成为一种有希望的治疗方法，但缺乏大型随机对照试验证明其有效性的证据。方法：我们进行了一项实用的、多中心的、随机对照试验，比较功能性运动障碍的专科物理治疗和常规治疗，常规治疗被定义为社区神经物理治疗。主要结果是简短形式问卷-36项12个月的身体功能领域（量表范围0-100,100表示最佳健康）。在5%的显著性水平上，该试验检测到主要结局有9个点的差异，有90%的显著性。次要测量领域包括患者对改善的感知、与健康相关的生活质量、活动能力、焦虑、抑郁和疾病感知。我们还完成了一项健康经济分析，其主要目的是计算12个月内每个质量调整生命年的平均增量成本。在预先指定的分析计划中，如果参与者由于COVID-19封锁限制而无法接受试验分配的治疗，我们将他们排除在初级分析之外。敏感性分析探讨了这一决定的影响。结果：在2018年10月19日至2022年1月31日期间，355名功能性运动障碍成年人被随机（1:1）分配到专科物理治疗组（n = 179）和常规治疗组（n = 176）。89名参与者因COVID-19中断而被排除在外。两组的初步分析保留率均为90%，剩下241名参与者参与了初步分析。在12个月时，主要转归无组间差异（校正平均差为3.5,95%可信区间为-2.3至9.3）。然而，一些次要结果倾向于专科物理治疗，包括参与者对改善的感知，简短问卷-36项心理健康领域，对诊断的信心和修订疾病感知问卷的两个子量表（个人控制和疾病一致性）。其余结果没有差异。在6个月时，以下结果测量有显著差异，有利于专科物理治疗：被试的改善知觉、简式问卷-36项身体角色限制、简式问卷-36项社会功能、简式问卷-36项心理健康、EuroQol-5维度五水平版本效用评分、诊断信心和修订疾病知觉问卷的三个子量表（时间周期、个人控制和治疗控制）。没有结果明显支持常规治疗。在健康经济分析中，从健康和社会护理成本的角度来看，每个质量调整生命年的增量成本为4133英镑，与常规治疗相比，专科物理治疗具有成本效益的可能性为86%，成本效益阈值为每个质量调整生命年获得20,000英镑。没有与物理治疗相关的不良事件。结论：在12个月的36项简短问卷调查中，专家物理治疗并不优于常规治疗。然而，在6个月和12个月时，一些次要结果指标倾向于专科物理治疗。专科物理治疗很可能具有成本效益。局限性：像往常一样接受治疗的参与者等待更长的时间开始物理治疗，这导致结束治疗和完成主要结局之间的时间更短。大多数结果测量，包括主要结果，都是参与者报告的，这可能因随机治疗分配的看法而有偏差。未来的工作：未来的工作应该为功能性运动障碍研究确定或开发更合适的结果测量，探索谁最有可能从专业物理治疗中受益，并为那些不太可能从这种治疗中受益的人确定替代干预措施。需要做更多的工作来调整治疗，以满足少数群体和年轻人的需要。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16/31/63。
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引用次数: 0
Identifying optimal primary prevention interventions for major cardiovascular disease events and all-cause mortality: a systematic review and hierarchical network meta-analysis of RCTs. 确定主要心血管疾病事件和全因死亡率的最佳一级预防干预措施：随机对照试验的系统回顾和分层网络荟萃分析
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/RLDH7432
Olalekan A Uthman, Rachel Court, Jodie Enderby, Chidozie Nduka, Lena Al-Khudairy, Seun Anjorin, Hema Mistry, G J Melendez-Torres, Sian Taylor-Phillips, Aileen Clarke

Background: Cardiovascular disease accounts for substantial mortality and healthcare costs worldwide. Numerous interventions exist for primary prevention but lack head-to-head comparisons on long-term impacts.

Objective: To determine the comparative effectiveness of interventions for primary cardiovascular disease prevention through network meta-analysis of randomised trials.

Data sources: MEDLINE, EMBASE, Cochrane Central Register of Controlled Trials, conference abstracts and trial registries from inception to March 2021.

Review methods: Randomised controlled trials of pharmacologic therapies, nutritional supplements, lifestyle changes, behavioural approaches and health policies with at least 6 months' follow-up were included. Pairwise and network meta-analyses were conducted for all-cause mortality, cardiovascular disease events, coronary heart disease and cardiovascular disease mortality.

Results: Data from 139 randomised trials, including 1,053,772 participants, proved suitable for quantitative synthesis. Blood pressure-lowering medications (risk ratio 0.82, 95% confidence interval 0.71 to 0.94), tight blood pressure control (risk ratio 0.66, 95% confidence interval 0.46 to 0.96), statins (risk ratio 0.81, 95% confidence interval 0.71 to 0.91) and multifactorial lifestyle interventions (risk ratio 0.75, 95% confidence interval 0.61 to 0.92) reduced composite cardiovascular events and mortality.

Limitations: Residual confounding may exist. Few direct head-to-head comparisons limited differentiation between some specific modalities.

Conclusions: We found evidence that blood pressure treatments, intense blood pressure targets, statins when appropriate and multifactorial lifestyle changes are the most effective strategies for primary prevention of cardiovascular disease, with unclear effects from other interventions. These findings can inform clinical guidelines and health policies prioritising interventions.

Funding: This research article presents independent research funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme as award number 17/148/05.

背景：心血管疾病在世界范围内造成了大量的死亡率和医疗费用。存在许多初级预防干预措施，但缺乏对长期影响的正面比较。目的：通过随机试验的网络荟萃分析，确定初级心血管疾病预防干预措施的比较有效性。数据来源：MEDLINE， EMBASE, Cochrane Central Register of Controlled Trials，会议摘要和从成立到2021年3月的试验注册。回顾方法：纳入药物治疗、营养补充剂、生活方式改变、行为方法和健康政策的随机对照试验，随访至少6个月。对全因死亡率、心血管疾病事件、冠心病和心血管疾病死亡率进行两两和网络荟萃分析。结果：139项随机试验的数据，包括1,053,772名参与者，证明适合定量合成。降压药（风险比0.82,95%置信区间0.71 ~ 0.94）、严格血压控制（风险比0.66,95%置信区间0.46 ~ 0.96）、他汀类药物（风险比0.81,95%置信区间0.71 ~ 0.91）和多因素生活方式干预（风险比0.75,95%置信区间0.61 ~ 0.92）降低了复合心血管事件和死亡率。局限性：可能存在残留混淆。很少有直接的正面比较限制了某些特定模式之间的区别。结论：我们发现有证据表明，血压治疗、高强度血压目标、适当时使用他汀类药物和多因素生活方式改变是心血管疾病一级预防最有效的策略，其他干预措施的效果尚不清楚。这些发现可以为临床指南和优先考虑干预措施的卫生政策提供信息。资助：这篇研究文章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17/148/05。
{"title":"Identifying optimal primary prevention interventions for major cardiovascular disease events and all-cause mortality: a systematic review and hierarchical network meta-analysis of RCTs.","authors":"Olalekan A Uthman, Rachel Court, Jodie Enderby, Chidozie Nduka, Lena Al-Khudairy, Seun Anjorin, Hema Mistry, G J Melendez-Torres, Sian Taylor-Phillips, Aileen Clarke","doi":"10.3310/RLDH7432","DOIUrl":"10.3310/RLDH7432","url":null,"abstract":"<p><strong>Background: </strong>Cardiovascular disease accounts for substantial mortality and healthcare costs worldwide. Numerous interventions exist for primary prevention but lack head-to-head comparisons on long-term impacts.</p><p><strong>Objective: </strong>To determine the comparative effectiveness of interventions for primary cardiovascular disease prevention through network meta-analysis of randomised trials.</p><p><strong>Data sources: </strong>MEDLINE, EMBASE, Cochrane Central Register of Controlled Trials, conference abstracts and trial registries from inception to March 2021.</p><p><strong>Review methods: </strong>Randomised controlled trials of pharmacologic therapies, nutritional supplements, lifestyle changes, behavioural approaches and health policies with at least 6 months' follow-up were included. Pairwise and network meta-analyses were conducted for all-cause mortality, cardiovascular disease events, coronary heart disease and cardiovascular disease mortality.</p><p><strong>Results: </strong>Data from 139 randomised trials, including 1,053,772 participants, proved suitable for quantitative synthesis. Blood pressure-lowering medications (risk ratio 0.82, 95% confidence interval 0.71 to 0.94), tight blood pressure control (risk ratio 0.66, 95% confidence interval 0.46 to 0.96), statins (risk ratio 0.81, 95% confidence interval 0.71 to 0.91) and multifactorial lifestyle interventions (risk ratio 0.75, 95% confidence interval 0.61 to 0.92) reduced composite cardiovascular events and mortality.</p><p><strong>Limitations: </strong>Residual confounding may exist. Few direct head-to-head comparisons limited differentiation between some specific modalities.</p><p><strong>Conclusions: </strong>We found evidence that blood pressure treatments, intense blood pressure targets, statins when appropriate and multifactorial lifestyle changes are the most effective strategies for primary prevention of cardiovascular disease, with unclear effects from other interventions. These findings can inform clinical guidelines and health policies prioritising interventions.</p><p><strong>Funding: </strong>This research article presents independent research funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme as award number 17/148/05.</p>","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-65"},"PeriodicalIF":4.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376007/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144560003","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
Digital augmentation of aftercare for patients with anorexia nervosa: the TRIANGLE RCT and economic evaluation. 神经性厌食症患者术后护理的数字增强：三角随机对照试验和经济评价。
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/ADLS3672
Janet Treasure, Katie Rowlands, Valentina Cardi, Suman Ambwani, David McDaid, Jodie Lord, Danielle Clark Bryan, Pamela Macdonald, Eva Bonin, Ulrike Schmidt, Jon Arcelus, Amy Harrison, Sabine Landau
<p><strong>Background: </strong>High-risk patients with complex anorexia nervosa are managed in inpatient/day patient care, but re-admission rates are high, and new treatments are needed.</p><p><strong>Objective(s): </strong>To examine the effectiveness of a digital augmentation of aftercare (ECHOMANTRA).</p><p><strong>Design: </strong>Transition Care In Anorexia Nervosa through Guidance Online from Peer and Carer Expertise was a multicentre, parallel-group, superiority randomised controlled trial. ECHOMANTRA augmented treatment as usual was compared with treatment as usual. Patient-carer dyads were randomised using minimisation on a 1 : 1 ratio into ECHOMANTRA + treatment as usual (ECHOMANTRA) or treatment as usual alone.</p><p><strong>Setting: </strong>Specialised United Kingdom inpatient/day patient sites (<i>n</i> = 31) participated.</p><p><strong>Participants: </strong>Patient-carer dyads were randomised (<i>n</i> = 185 in ECHOMANTRA and <i>n</i> = 186 in treatment as usual).</p><p><strong>Interventions: </strong>The digital ECHOMANTRA intervention included self-management tools (recovery tips videos) for patients and task-sharing materials for carers (skill-sharing video), supplemented with guided group chat sessions. All participants randomised to ECHOMANTRA + treatment as usual had access to the psychoeducational materials and joint patient/carer chat sessions were also offered.</p><p><strong>Main outcome measures: </strong>The primary outcome was patient distress at 12 months. Other outcomes included patient distress at 18 months, and eating disorder symptoms, social and work adjustment, and carer distress and skills at 12 and 18 months.</p><p><strong>Results: </strong>There was no evidence of an intervention effect on the Depression Anxiety Stress Scale-21 outcome for patients (<i>n</i> = 370) at 12 months, estimated effect 0.48, 95% confidence interval -0.20 to 0.23, standardised estimate (0.02, <i>p</i> = 0.87). In the economic analysis, the intervention was dominated by treatment as usual from both a health system and wider societal perspective, as ECHOMANTRA cost more and resulted in fewer quality-adjusted life-years gained. However, the uptake of the interactive component of the intervention (i.e. the facilitated and moderated online groups) was limited, with only 20% of the dyad members attending the pre-set minimal adherence level (i.e. both the patient and carer attending at least four online forum group sessions). The feedback about the intervention was predominantly positive. For example, the group facilitators were rated highly. However, some feedback was that the intervention offered too little, too late, and that a more personalised intervention would be more helpful.</p><p><strong>Limitations: </strong>Participants were diverse (e.g. 20% were being treated under the Mental Health Act), and a large proportion had a range of comorbidities (depression, anxiety, obsessive-compulsive disorder and autistic spectrum disorders), a
背景：复杂神经性厌食症的高危患者在住院/日间护理中得到管理，但再入院率高，需要新的治疗方法。目的：探讨数字化增强术后护理（ECHOMANTRA）的有效性。设计：通过同伴在线指导和护理人员专业知识对神经性厌食症进行过渡护理是一项多中心、平行组、优势随机对照试验。常规ECHOMANTRA强化治疗与常规治疗比较。按照1:1的比例将患者-护理人员随机分为ECHOMANTRA +常规治疗（ECHOMANTRA）或单独常规治疗。环境：参与了英国专门的住院/日间病人中心（n = 31）。参与者：患者-护理者二人组随机分组（ECHOMANTRA组n = 185，常规治疗组n = 186）。干预措施：数字ECHOMANTRA干预措施包括患者的自我管理工具（康复提示视频）和护理人员的任务分享材料（技能分享视频），并辅以指导小组聊天会议。所有随机接受ECHOMANTRA +治疗的参与者都可以获得心理教育材料，并提供患者/护理人员联合聊天会议。主要观察指标：主要观察指标为患者12个月时的痛苦程度。其他结果包括患者在18个月时的痛苦，饮食失调症状，社会和工作适应，以及护理人员在12个月和18个月时的痛苦和技能。结果：没有证据表明干预对患者（n = 370） 12个月时的抑郁焦虑应激量表-21结局有影响，估计效果为0.48,95%置信区间为-0.20 ~ 0.23，标准化估计（0.02,p = 0.87）。在经济分析中，从卫生系统和更广泛的社会角度来看，干预措施主要是常规治疗，因为ECHOMANTRA成本更高，结果获得的质量调整生命年更少。然而，干预的互动部分（即促进和缓和的在线小组）的吸收是有限的，只有20%的双成员参加预先设定的最低遵守水平（即患者和护理人员参加至少四个在线论坛小组会议）。对干预的反馈主要是积极的。例如，小组引导者的评价很高。然而，一些反馈是，干预提供太少，太迟，更个性化的干预将更有帮助。局限性：参与者是多种多样的（例如，20%的参与者根据《精神卫生法》接受治疗），很大一部分患者有一系列合并症（抑郁、焦虑、强迫症和自闭症谱系障碍），所有这些因素都会影响预后。虽然为加强包容性作出了努力，但性别、性向和种族方面的多样性是有限的，技术障碍和/或缺乏照顾者可能导致排斥。对团体支持的高度不依从性（80%对）可能是导致非显著结果的原因。结论：这种指导性自我管理和任务分担干预得到了一些患者和支持者的积极评价；然而，没有证据表明干预比常规的术后护理改善了结果。未来的工作：确定增加参与的机制，如更个性化的护理方法，以满足这一患者群体的不同需求。在提供支持的同行工作者的指导下，强化服务和逐步服务之间的更大整合可能会优化护理。试验注册：该试验注册号为ISRCTN14644379。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：14/68/09）资助，全文发表在《卫生技术评估》杂志上；第29卷，第31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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