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Invasive urodynamic investigations in the management of women with refractory overactive bladder symptoms: FUTURE, a superiority RCT and economic evaluation. 有创尿动力学研究在治疗难治性膀胱过度活动症状的女性中的应用：FUTURE，一项优势随机对照试验和经济评估。
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/UKYW4923
Mohamed Abdel-Fattah, Christopher Chapple, Suzanne Breeman, David Cooper, Helen Bell-Gorrod, Preksha Kuppanda, Karen Guerrero, Simon Dixon, Nikki Cotterill, Karen Ward, Hashim Hashim, Ash Monga, Karen Brown, Marcus Drake, Andrew Gammie, Alyaa Mostafa, Rebecca Bruce, Victoria Bell, Christine Kennedy, Suzanne Evans, Graeme MacLennan, John Norrie
<p><strong>Background: </strong>Overactive bladder is a common problem affecting the United Kingdom adult female population. Symptoms include urinary urgency, with or without urgency incontinence, increased daytime urinary frequency and nocturia. Initial conservative treatments for overactive bladder are unsuccessful in 25-40% of women (refractory overactive bladder). Before considering invasive treatments, such as botulinum toxin injection-A or sacral neuromodulation, guidelines recommend urodynamics to confirm diagnosis of detrusor overactivity. However, the clinical and cost effectiveness of urodynamics has never been robustly assessed.</p><p><strong>Objectives: </strong>To compare the clinical and cost effectiveness of urodynamics plus comprehensive clinical assessment versus comprehensive clinical assessment only in the management of refractory overactive bladder in women.</p><p><strong>Design: </strong>Parallel-group, multicentre, superiority, open-label, randomised controlled trial. Allocation by remote web-based randomisation (1 : 1 ratio). The cost-effectiveness analysis took the National Health Service perspective with a model-based lifetime time horizon, as informed by a within-trial analysis.</p><p><strong>Setting: </strong>Sixty-three United Kingdom secondary and tertiary hospitals.</p><p><strong>Participants: </strong>Women aged ≥ 18 years with refractory overactive bladder or urgency-predominant mixed urinary incontinence who had failed conservative management and pharmacological treatment and were being considered for invasive treatment. Women were excluded if any of the following criteria were met: predominant stress urinary incontinence; previous urodynamics in last 12 months; current pelvic malignancy or clinically significant pelvic mass; bladder pain syndrome; neurogenic bladder; urogenital fistulae; previous treatment with botulinum toxin injection-A or sacral neuromodulation for urinary incontinence; previous pelvic radiotherapy; prolapse beyond introitus; pregnant or planning pregnancy; recurrent urinary tract infection where a significant pathology has not been excluded; and inability to give an informed consent.</p><p><strong>Interventions: </strong>Urodynamics plus comprehensive clinical assessment (urodynamics arm) versus comprehensive clinical assessment only.</p><p><strong>Main outcome measures: </strong>Participant-reported success at the last follow-up time point as measured by the Patient Global Impression of Improvement. Primary economic outcome was incremental cost per quality-adjusted life-year gained as modelled over the lifetime of participants.</p><p><strong>Results: </strong>A total of 1099 participants were included: 550 randomised to the urodynamics arm and 549 to the comprehensive clinical assessment only arm. At the final follow-up time point, participant-reported success rates of 'very much improved' and 'much improved' were not superior in the urodynamics arm (117 participants; 23.6%) compared to the
背景：膀胱过度活动是影响英国成年女性人群的常见问题。症状包括尿急，伴或不伴尿急尿失禁，日间尿频增加和夜尿。25-40%的女性（难治性膀胱过度活跃）最初的保守治疗不成功。在考虑有创性治疗前，如注射a型肉毒杆菌毒素或骶骨神经调节，指南建议尿动力学检查以确认逼尿肌过度活动的诊断。然而，尿动力学的临床和成本效益从未得到过有力的评估。目的：比较尿动力学加综合临床评估与单纯综合临床评估治疗难治性膀胱过动症的临床和成本效益。设计：平行组、多中心、优势、开放标签、随机对照试验。远程网络随机分配（1:1比例）。成本效益分析采用了基于模型的终身时间范围的国家卫生服务视角，根据试验内分析提供信息。环境：63家联合王国二级和三级医院。参与者：年龄≥18岁的女性，患有难治性膀胱过动症或急性病混合性尿失禁，保守治疗和药物治疗失败，正在考虑进行侵入性治疗。如果符合以下任何标准，则排除女性：主要压力性尿失禁；过去12个月的尿动力学记录；当前盆腔恶性肿瘤或有临床意义的盆腔肿块；膀胱疼痛综合征；神经性膀胱功能障碍;泌尿生殖管状器官;既往有a型肉毒毒素注射或骶神经调节治疗尿失禁；既往盆腔放疗；脱垂超过内向质；怀孕的或计划怀孕的；复发性尿路感染，未排除明显病理；以及无法给予知情同意。干预措施：尿动力学加综合临床评估（尿动力学组）vs仅综合临床评估。主要结果测量：参与者报告的最后一个随访时间点的成功，以患者总体改善印象来衡量。主要的经济结果是参与者一生中每个质量调整生命年的增量成本。结果：共纳入1099名参与者：550名随机分配到尿动力学组，549名随机分配到综合临床评估组。在最后的随访时间点，参与者报告的“非常改善”和“非常改善”的成功率在尿动力学组中并不优越(117名参与者；23.6%)与仅综合临床评估组(114名参与者；22.7%)[校正优势比1.12(95%可信区间0.73 ~ 1.74)；p = 0.601]。组间严重不良事件发生率低且相似。根据估计的尿动力学增量成本和质量调整生命年（分别为463英镑和0.011英镑），每个质量调整生命年的增量成本-效果比为42,643英镑。成本效益可接受度曲线显示，在每个质量调整生命年获得20,000英镑的支付意愿阈值下，尿动力学具有成本效益的概率为34%。当结果外推到病人的一生时，这种可能性进一步降低。局限性包括：只有短期结果，并且由于大多数参与者接受了肉毒杆菌毒素注射a治疗，因此不可能对某些结果（如骶神经调节）进行预先计划的二次分析。结论：随访15个月后，参与者报告的尿动力学组的成功率并不优于仅综合临床评估组。如果每个质量调整生命年的门槛为20,000英镑，Urodynamics就不具有成本效益。需要进行长期随访，以探讨进一步干预和治疗的必要性及其对临床和成本效益分析的影响。试验注册：该试验注册号为ISRCTN63268739。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：15/150/05）资助，全文发表在《卫生技术评估》第29卷第27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
Melatonin versus midazolam in the premedication of anxious children attending for elective surgery under general anaesthesia: the MAGIC non-inferiority RCT. 褪黑素与咪达唑仑在全麻下参加择期手术的焦虑儿童的预用药：MAGIC非劣效性随机对照试验。
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/CWKF1987
Christopher Deery, Robert Bolt, Diana Papaioannou, Matthew Wilson, Marie Hyslop, Esther Herbert, Nikki Totton, Zoe Marshman, Tracey Young, Jennifer Kettle, Sondos Albadri, Simon Atkins, Katie Biggs, Janet Clarkson, Chris Evans, Laura Flight, Jacqui Gath, Fiona Gilchrist, Kate Hutchence, Nicholas Ireland, Amanda Loban, Amy Norrington, Hamish Paton, Jaydip Ray, Helen Rodd, Elena Sheldon, Richard Simmonds, Christopher Vernazza
<p><strong>Background: </strong>Anxiety in children prior to general anaesthesia is common, with up to half displaying distress. Anxiety and distress may lead to unsuccessful anaesthesia, together with greater postoperative pain, agitation and behavioural changes after surgery including sleep disturbances. Midazolam is the current standard premedication; however, it has adverse effects such as the potential for respiratory suppression and unpredictable effects which may result in agitation rather than anxiolysis. Melatonin is an alternative preoperative anxiolytic; however, previous trials have delivered conflicting results. The aim of this non-inferiority trial was to evaluate the effectiveness of melatonin compared to midazolam in reducing anxiety in children undergoing general anaesthesia.</p><p><strong>Methods: </strong>We undertook a randomised-controlled, parallel-group, double-blind, non-inferiority trial in 20 United Kingdom National Health Service trusts, with an embedded qualitative study and health economic evaluation. Anxious children having day case elective surgery under general anaesthesia were randomly assigned to either control (standard of care) group: midazolam; or intervention group: melatonin. The primary outcome was preoperative distress (non-inferiority hypothesis) as assessed by modified Yale Preoperative Anxiety Scale Short Form. Secondary outcomes included safety and efficacy objectives. Analyses were by intention to treat, with an additional per-protocol analysis. The sample size of the trial was 624 children.</p><p><strong>Results: </strong>The trial was stopped early due to recruitment futility. Between 30 July 2019 and 9 November 2022, 110 children were recruited; 55 allocated to midazolam and 55 allocated to melatonin. Pre-planned analyses showed an adjusted mean difference of 13.1 (95% confidence interval 3.7 to 22.4) for the intention-to-treat population and 12.9 (95% confidence interval 3.1 to 22.6) for the per-protocol population, in favour of midazolam. In both analyses, the upper limit of the 95% confidence interval exceeds the predefined margin of 4.3; therefore, melatonin is not non-inferior to midazolam. The lower limit of the 95% confidence intervals excludes zero and thus melatonin is inferior to midazolam; the difference found is considered to be clinically meaningful. Adverse events in the midazolam arm (26%) were slightly higher than melatonin (18%); there were no serious adverse events in either arm. Challenges to recruitment included study-related factors (eligibility criteria and trial design), participant factors (caregiver stress on the day of treatment) and practitioner factors (valuing predictability). In terms of acceptability, preferences of the anaesthetist, patient and caregiver factors and medication side effects profile were influential and suggest the choice of preoperative anxiolytic is more complex than previously described. On average, costs over the 14 days post surgery were lower for
背景：全麻前儿童焦虑是常见的，多达一半表现出痛苦。焦虑和痛苦可能导致麻醉失败，以及术后更大的疼痛、躁动和手术后的行为改变，包括睡眠障碍。咪达唑仑是目前标准的前用药；然而，它有副作用，如潜在的呼吸抑制和不可预测的影响，可能导致躁动而不是焦虑。褪黑素是术前抗焦虑药的替代方案；然而，之前的试验得出了相互矛盾的结果。这项非劣效性试验的目的是评估褪黑素与咪达唑仑在减轻全身麻醉儿童焦虑方面的有效性。方法：我们在20个英国国家卫生服务信托机构进行了随机对照、平行组、双盲、非劣效性试验，并进行了嵌入式定性研究和卫生经济学评价。在全麻下进行日间择期手术的焦虑儿童被随机分配到对照组（标准护理组）：咪达唑仑；干预组：褪黑素。主要结局为术前焦虑（非劣效假设），采用改良的耶鲁术前焦虑量表短表评估。次要结局包括安全性和有效性目标。分析是根据治疗意向进行的，并进行了额外的协议分析。该试验的样本量为624名儿童。结果：由于招募无效，试验提前终止。2019年7月30日至2022年11月9日期间，招募了110名儿童；55人服用咪达唑仑，55人服用褪黑素。预先计划的分析显示，意向治疗人群的调整平均差异为13.1(95%可信区间3.7至22.4)，按方案人群的调整平均差异为12.9(95%可信区间3.1至22.6)，有利于咪达唑仑。在这两个分析中，95%置信区间的上限都超过了预先设定的4.3；因此，褪黑素并非不逊于咪达唑仑。95%置信区间的下限不包括零，因此褪黑素不如咪达唑仑；发现的差异被认为具有临床意义。咪达唑仑组的不良事件发生率（26%）略高于褪黑素组（18%）；两组均无严重不良事件发生。招募面临的挑战包括研究相关因素（资格标准和试验设计）、参与者因素（治疗当天照顾者的压力）和从业者因素（重视可预测性）。在可接受性方面，麻醉师的偏好、患者和护理人员的因素以及药物的副作用都是有影响的，这表明术前抗焦虑药的选择比之前描述的更复杂。平均而言，接受褪黑激素治疗的患者术后14天的费用较低（- 46.20英镑，95%可信区间- 166.14英镑至66.74英镑），手术成功率的平均增量差异为-0.02英镑（95%可信区间-0.08至0.004），尽管结果存在不确定性。结论：在术前焦虑的儿童中，咪达唑仑比褪黑素更有效地减少全身麻醉前的术前焦虑，尽管试验的早期终止增加了偏倚的可能性。局限性：由于招募无效，试验过早终止。尽管如此，在主要结局方面仍观察到具有临床意义和统计学意义的发现。未来的工作：仍然需要开发或重新使用另一种副作用比咪达唑仑更有利的药物。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16/80/08。
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引用次数: 0
Methylphenidate versus placebo for fatigue in patients with advanced cancer: the MePFAC randomised controlled trial. 哌醋甲酯与安慰剂治疗晚期癌症患者疲劳：MePFAC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/GJPS6321
Patrick Stone, Ollie Minton, Alison Richardson, Peter Buckle, Zinat E Enayat, Louise Marston, Nick Freemantle

Background: Previous meta-analyses suggested methylphenidate may be effective for cancer-related fatigue.

Trial design: Phase III, parallel-group, randomised, double-blind, placebo-controlled trial.

Methods: Participants were adults with advanced cancer with cancer-related fatigue receiving palliative care at 17 palliative care services in England between June 2018 and April 2023.

Principal exclusions: Pregnancy; glaucoma; pheochromocytoma; planned general anaesthesia; hyperthyroidism; severe psychiatric disorders; hypertension; severe cardiovascular disorders; cerebrovascular disorders; anaemia; thrombocytopenia; leucopenia; infection; renal or liver impairment; concomitant clonidine, warfarin, monoamine oxidase inhibitors or modafinil; alcohol or drug dependency; epilepsy.

Interventions: Methylphenidate 5 mg tablets or matching placebo. Starting at 1 tablet twice daily, titrated over 6 weeks to a maximum of 12 tablets/day.

Objective: To estimate clinical effectiveness of methylphenidate versus placebo for cancer-related fatigue in patients receiving palliative care.

Primary outcome: Fatigue at 6 (± 2) weeks measured using the Functional Assessment of Chronic Illness Therapy - Fatigue Scale score. Secondary outcomes were fatigue at other time points; quality of life, adverse events, activities of daily living; appetite; anxiety; depression; patient satisfaction; survival and need for other medication.

Randomisation: Computer-generated 1 : 1 randomisation, stratified by centre, concomitant treatment, depression and initial fatigue score.

Blinding: Participants and outcome assessors were blinded to group assignment.

Results: Eighty-four were allocated to methylphenidate and 78 to placebo.

Recruitment: : Study completed.

Numbers analysed: Seventy-five in methylphenidate group and 72 in placebo group were included in analysis of primary outcome.

Outcome: There was no statistically or clinically significant difference in primary outcome between groups. Functional Assessment of Chronic Illness Therapy - Fatigue Scale scores were 1.97 points (95% confidence interval -0.95 to 4.90; p = 0.186) higher (better) on methylphenidate than placebo. Functional Assessment of Chronic Illness Therapy - Fatigue Scale score was nominally statistically significantly higher (better) in methylphenidate group across duration of study [Diff 2.20 (95% confidence interval 0.39 to 4.01)] but did not reach the minimal clinically important difference (5 points). At 6 weeks, there were no statistically significant differences in quality-of-life or symptom domains except for depression scores [nominally statistically significantly reduced in methylphenidate group: Diff -1.35 (95% c

背景：先前的荟萃分析表明，哌醋甲酯可能对癌症相关疲劳有效。试验设计：III期，平行组，随机，双盲，安慰剂对照试验。方法：参与者是2018年6月至2023年4月期间在英国17家姑息治疗服务机构接受姑息治疗的晚期癌症伴癌症相关疲劳的成年人。主要除外：怀孕；青光眼;嗜铬细胞瘤;计划全身麻醉；甲状腺机能亢进;严重精神疾病；高血压;严重心血管疾病；脑血管疾病;贫血;血小板减少症;白细胞减少;感染;肾或肝损害；同时使用可乐定、华法林、单胺氧化酶抑制剂或莫达非尼；酒精或药物依赖；癫痫。干预措施：哌甲酯5mg片或配套安慰剂。起始剂量为1片，每日2次，6周后逐渐递增至最多12片/天。目的：评估哌醋甲酯与安慰剂对接受姑息治疗的癌症相关疲劳患者的临床疗效。主要结局：疲劳在6（±2）周测量使用慢性疾病治疗功能评估-疲劳量表评分。次要结局是其他时间点的疲劳；生活质量、不良事件、日常生活活动；食欲;焦虑;抑郁症;病人满意度;生存和其他药物的需要。随机化：计算机生成1:1随机化，按中心、伴随治疗、抑郁和初始疲劳评分分层。盲法：参与者和结果评估者对小组分配不知情。结果：84人被分配到哌甲酯组，78人被分配到安慰剂组。招聘：完成学习。数据分析：主要结局分析包括哌甲酯组75例和安慰剂组72例。结果：两组间的主要结果无统计学或临床显著差异。慢性疾病治疗功能评估-疲劳量表得分为1.97分(95%置信区间-0.95至4.90；P = 0.186)，哌醋甲酯组比安慰剂组更高（更好）。在整个研究期间，哌甲酯组的慢性疾病治疗功能评估-疲劳量表得分名义上有统计学意义上更高（更好）[Diff 2.20(95%可信区间0.39至4.01)]，但未达到最小的临床重要差异（5分）。6周时，除抑郁评分外，两组在生活质量或症状领域方面无统计学显著差异[哌甲酯组名义上统计学显著降低：Diff -1.35(95%置信区间-2.41至-0.30)]。危害：20名服用哌甲酯的参与者中有25例严重不良事件，16名服用安慰剂的参与者中有25例严重不良事件。未发现意料之外的严重不良反应。随机分组后75天内发生的死亡没有统计学上的显著差异(安慰剂组2名，哌醋甲酯组6名；费雪精确p值0.278)。两组的不良事件相似，没有模式表明哌醋甲酯会增加危害。局限性：由于多重排除标准，参与者被高度选择。选择慢性疾病治疗功能评估-疲劳量表评分的5分差作为具有临床意义的主要结局可能存在争议。结论：哌醋甲酯并没有减轻晚期癌症患者6（±2）周的疲劳严重程度，但安全且耐受性良好。未来的工作：不推荐对接受姑息治疗的晚期癌症患者进行哌醋甲酯治疗疲劳的进一步试验。在不同人群或不同适应症中可能有进一步研究的余地。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15/46/02。
{"title":"Methylphenidate versus placebo for fatigue in patients with advanced cancer: the MePFAC randomised controlled trial.","authors":"Patrick Stone, Ollie Minton, Alison Richardson, Peter Buckle, Zinat E Enayat, Louise Marston, Nick Freemantle","doi":"10.3310/GJPS6321","DOIUrl":"10.3310/GJPS6321","url":null,"abstract":"<p><strong>Background: </strong>Previous meta-analyses suggested methylphenidate may be effective for cancer-related fatigue.</p><p><strong>Trial design: </strong>Phase III, parallel-group, randomised, double-blind, placebo-controlled trial.</p><p><strong>Methods: </strong>Participants were adults with advanced cancer with cancer-related fatigue receiving palliative care at 17 palliative care services in England between June 2018 and April 2023.</p><p><strong>Principal exclusions: </strong>Pregnancy; glaucoma; pheochromocytoma; planned general anaesthesia; hyperthyroidism; severe psychiatric disorders; hypertension; severe cardiovascular disorders; cerebrovascular disorders; anaemia; thrombocytopenia; leucopenia; infection; renal or liver impairment; concomitant clonidine, warfarin, monoamine oxidase inhibitors or modafinil; alcohol or drug dependency; epilepsy.</p><p><strong>Interventions: </strong>Methylphenidate 5 mg tablets or matching placebo. Starting at 1 tablet twice daily, titrated over 6 weeks to a maximum of 12 tablets/day.</p><p><strong>Objective: </strong>To estimate clinical effectiveness of methylphenidate versus placebo for cancer-related fatigue in patients receiving palliative care.</p><p><strong>Primary outcome: </strong>Fatigue at 6 (± 2) weeks measured using the Functional Assessment of Chronic Illness Therapy - Fatigue Scale score. Secondary outcomes were fatigue at other time points; quality of life, adverse events, activities of daily living; appetite; anxiety; depression; patient satisfaction; survival and need for other medication.</p><p><strong>Randomisation: </strong>Computer-generated 1 : 1 randomisation, stratified by centre, concomitant treatment, depression and initial fatigue score.</p><p><strong>Blinding: </strong>Participants and outcome assessors were blinded to group assignment.</p><p><strong>Results: </strong>Eighty-four were allocated to methylphenidate and 78 to placebo.</p><p><strong>Recruitment: </strong>: Study completed.</p><p><strong>Numbers analysed: </strong>Seventy-five in methylphenidate group and 72 in placebo group were included in analysis of primary outcome.</p><p><strong>Outcome: </strong>There was no statistically or clinically significant difference in primary outcome between groups. Functional Assessment of Chronic Illness Therapy - Fatigue Scale scores were 1.97 points (95% confidence interval -0.95 to 4.90; <i>p</i> = 0.186) higher (better) on methylphenidate than placebo. Functional Assessment of Chronic Illness Therapy - Fatigue Scale score was nominally statistically significantly higher (better) in methylphenidate group across duration of study [Diff 2.20 (95% confidence interval 0.39 to 4.01)] but did not reach the minimal clinically important difference (5 points). At 6 weeks, there were no statistically significant differences in quality-of-life or symptom domains except for depression scores [nominally statistically significantly reduced in methylphenidate group: Diff -1.35 (95% c","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 36","pages":"1-47"},"PeriodicalIF":4.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376206/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144775305","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
Variation within and between digital pathology and light microscopy for the diagnosis of histopathology slides: blinded crossover comparison study. 组织病理学切片诊断中数字病理与光学显微镜之间的差异：盲法交叉比较研究。
IF 3.5 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/SPLK4325
David Rj Snead, Ayesha S Azam, Jenny Thirlwall, Peter Kimani, Louise Hiller, Adam Bickers, Clinton Boyd, David Boyle, David Clark, Ian Ellis, Kishore Gopalakrishnan, Mohammad Ilyas, Paul Kelly, Maurice Loughrey, Desley Neil, Emad Rakha, Ian Sd Roberts, Shatrughan Sah, Maria Soares, YeeWah Tsang, Manuel Salto-Tellez, Helen Higgins, Donna Howe, Abigail Takyi, Yan Chen, Agnieszka Ignatowicz, Jason Madan, Henry Nwankwo, George Partridge, Janet Dunn
<p><strong>Background: </strong>Digital pathology refers to the conversion of histopathology slides to digital image files for examination on computer workstations as opposed to conventional microscopes. Prior to adoption, it is important to demonstrate pathologists provide equivalent reports when using digital pathology in comparison to bright-field and immunofluorescent light microscopy, the current standard of care.</p><p><strong>Objective: </strong>A multicentre comparison of digital pathology with light microscopy for reporting of histopathology slides, measuring variation within and between pathologists on both modalities.</p><p><strong>Design: </strong>A blinded crossover 2000-case study estimating clinical management concordance (identical diagnoses plus differences not affecting patient management). Each sample was assessed twice by four pathologists (once using light microscopy, once using digital pathology, the order randomly assigned and a 6-week gap between viewings). Random-effects logistic regression models, including crossed random-effects terms for case and pathologist, estimated percentage clinical management concordance. Findings were interpreted with reference to 98.3% concordance (Azam AS, Miligy IM, Kimani PKU, Maqbool H, Hewitt K, Rajpoot NM, Snead DRJ. Diagnostic concordance and discordance in digital pathology: a systematic review and meta-analysis. <i>J Clin Pathol</i> 2021;<b>74</b>:448-55. https://doi.org/10.1136/jclinpath-2020-206764).</p><p><strong>Setting: </strong>Sixteen consultant pathologists, four for each specialty, from six National Health Service laboratories. Experience ranged from 3 to 35 years. Some were early adopters of digital pathology, but the majority were new to digital pathology.</p><p><strong>Interventions: </strong>Eight viewings per sample (four pathologists with light microscopy and with digital pathology), culminating in a consensus ground truth, enabling measurement of agreement within and between readers. Samples enrolled reflected routine practice, included cancer screening biopsies, and were enriched for areas of difficulty [e.g. dysplasia (7, 10, 11)]. State-of-the-art digital pathology equipment designed for diagnosis, and holding either Conformité Européene or Food and Drug Administration approval, was used.</p><p><strong>Main outcome: </strong>Intra-pathologist variation between reports issued on digital pathology and light microscopy, inter-pathologist variation against ground-truth diagnosis using light microscopy and digital pathology.</p><p><strong>Secondary outcomes: </strong>Pathologist-recorded reporting times, along with their confidence in diagnosis, analysis of eye-tracking evaluating examination techniques, and a qualitative study examining attitudes of pathologists and laboratory staff to digital pathology adoption.</p><p><strong>Results: </strong>Two thousand and twenty-four cases (608 breast, 607 gastrointestinal, 609 skin, 200 renal) were recruited, with breast and gast
背景：数字病理学是指将组织病理切片转换为数字图像文件，以便在计算机工作站进行检查，而不是传统的显微镜。在采用之前，重要的是要证明病理学家在使用数字病理学时提供与明场和免疫荧光光显微镜（目前的护理标准）相同的报告。目的：多中心比较数字病理与光学显微镜报告组织病理切片，测量病理学家内部和之间的差异。设计：一项2000例的盲法交叉研究，估计临床管理一致性（相同的诊断加上不影响患者管理的差异）。每个样本由四名病理学家评估两次（一次使用光学显微镜，一次使用数字病理学，顺序随机分配，检查间隔6周）。随机效应逻辑回归模型，包括病例和病理学家的交叉随机效应术语，估计临床管理一致性百分比。结果参照98.3%的一致性进行解释（Azam AS, mily IM, Kimani PKU, Maqbool H, Hewitt K, Rajpoot NM, Snead DRJ）。数字病理学诊断一致性和不一致性：系统回顾和荟萃分析。中华临床医学杂志（英文版）；2009;44(4):444 - 444。https://doi.org/10.1136/jclinpath-2020-206764).Setting: 16名咨询病理学家，每个专业4名，来自6个国家卫生服务实验室。经验从3年到35年不等。有些是数字病理学的早期采用者，但大多数是数字病理学的新手。干预措施：每个样本8次查看（4名病理学家使用光学显微镜和数字病理学），最终达成共识的基本事实，从而能够测量读者内部和读者之间的一致性。纳入的样本反映了常规做法，包括癌症筛查活检，并对困难的领域进行了充实[例如，发育不良（7,10,11）]。用于诊断的最先进的数字病理设备，并持有conformit europ或食品和药物管理局的批准。主要结果：病理学家内部对数字病理学和光学显微镜报告的差异，病理学家之间对使用光学显微镜和数字病理学的真实诊断的差异。次要结果：病理学家记录的报告时间，以及他们对诊断的信心，眼动追踪评估检查技术的分析，以及一项关于病理学家和实验室工作人员对数字病理学采用态度的定性研究。结果：共纳入2424例（乳腺608例，胃肠道607例，皮肤609例，肾脏200例），其中乳腺和胃肠道包括筛查样本[乳腺207例（34%），胃肠道250例（41%）]。总体而言，在光学显微镜与数字病理学比较中，临床管理一致性水平为99.95%（95%置信区间为99.91至99.97）。在各专科也观察到类似的结果[乳腺：99.40%(95%可信区间为99.06 ~ 99.62)；胃肠道99.96%(95%置信区间为99.89 ~ 99.99)；皮肤99.99%(95%置信区间99.92 ~ 100.0)；肾脏99.99%(95%可信区间99.57 ~ 100.0)，在筛查病例中[98.96%(95%可信区间98.42 ~ 99.32)，乳腺96.27%(94.63 ~ 97.43)，胃肠道99.93%(95%可信区间99.68 ~ 99.98)]。数字病理和光学显微镜之间的报告时间相似，但病理学家对数字病理的熟悉程度提高了。病理学家记录了高水平的信心，他们的诊断与光学显微镜，明显高于数字病理学。局限性：细胞学病例和专业组之外的测试没有检查。该研究使用了两种数字病理扫描系统。市场上可用的其他系统没有经过测试。结论：在乳腺、胃肠道、皮肤和肾脏专科以及合并乳腺癌和大肠癌筛查病例中，两种方式的临床管理一致性水平超过参考值98.3%。临床显著差异的亚组分析揭示了一系列差异，包括已知观察者间变异性较高的区域，这些差异分布在使用两种平台进行的读取之间，并且没有明显的趋势。未来工作：对细胞学样本使用数字病理学仍是一个有待进一步研究的领域。研究注册：本研究注册号为ISRCTN14513591。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：17/84/07）资助，全文发表在《卫生技术评估》杂志上；第29卷，第30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
Cessation of smoking in people attending UK emergency departments: the COSTED RCT with economic and process evaluation. 在英国急诊科就诊的人戒烟：经济和过程评估的成本计算随机对照试验
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/JHFR0841
Ian Pope, Lucy V Clark, Allan Clark, Emma Ward, Pippa Belderson, Susan Stirling, Steve Parrott, Jinshuo Li, Timothy Coats, Linda Bauld, Richard Holland, Sarah Gentry, Sanjay Agrawal, Benjamin M Bloom, Adrian Boyle, Alasdair Gray, M Geraint Morris, Caitlin Notley
<p><strong>Background: </strong>The emergency department represents a potentially valuable opportunity to support smoking cessation. Evidence is lacking around the use of e-cigarettes in opportunistic settings like the emergency department.</p><p><strong>Objective: </strong>To undertake a randomised controlled trial in people who smoke attending United Kingdom emergency departments, testing a brief intervention which included provision of an e-cigarette versus signposting to smoking cessation services, assessing smoking abstinence.</p><p><strong>Design: </strong>A two-arm pragmatic, multicentre, parallel-group, individually randomised, controlled superiority trial with an internal pilot, economic evaluation and mixed-methods process evaluation.</p><p><strong>Setting: </strong>Six emergency departments across England and Scotland.</p><p><strong>Participants: </strong>Adults who smoked daily, who were attending the emergency department for medical treatment or accompanying someone attending for medical treatment, were invited to participate. People were excluded if they had an expired carbon monoxide of < 8 parts per million, required immediate medical treatment, were in police custody, had a known allergy to nicotine, were daily e-cigarette users, were considered not to have capacity to consent or had already taken part in the trial.</p><p><strong>Intervention: </strong>Brief stop smoking advice, e-cigarette starter kit and referral to stop smoking services.</p><p><strong>Main outcome measures: </strong>The primary outcome was biochemically validated sustained abstinence at 6 months. Those lost to follow-up, or not providing biochemical verification, were considered not to be abstinent. Secondary outcomes were: self-reported 7-day smoking abstinence, number of quit attempts, number of cigarettes per day, nicotine dependence and incidence of self-reported dry cough or mouth or throat irritation.</p><p><strong>Results: </strong>At 6 months, of 972 participants randomised, biochemically verified smoking abstinence was 7.2% in the intervention group and 4.1% in the control group (percentage difference = 3.3%) (95% confidence interval 0.3 to 6.3; <i>p</i> = 0.032) [relative risk 1.76 (95% confidence interval 1.03 to 3.01)]. Self-reported 7-day abstinence at 6 months was 23.3% in the intervention group and 12.9% in the control group (percentage difference = 10.6%) (95% confidence interval 5.86 to 15.41; <i>p</i> < 0.001) [relative risk 1.80 (95% confidence interval 1.36 to 2.38)]. Daily e-cigarette use was 39.4% in the intervention group and 17.5% in the control group at 6 months. No serious adverse events related to taking part in the trial were reported. The economic evaluation found the intervention was likely to be cost-effective, judged by the National Institute for Health and Care Excellence threshold. The process evaluation found the intervention to be acceptable to both staff delivering it and participants receiving it. The brief nature of the i
背景：急诊科是支持戒烟的潜在宝贵机会。在急诊等机会性环境中使用电子烟的证据不足。目的：对在英国急诊科就诊的吸烟者进行一项随机对照试验，测试一种简短的干预措施，包括提供电子烟与戒烟服务的指示牌，评估戒烟情况。设计：两臂实用、多中心、平行组、单独随机、控制优势试验，内部试点，经济评估和混合方法过程评估。环境：英格兰和苏格兰的六个急诊科。参与者：每天吸烟的成年人，在急诊室接受医疗治疗或陪同他人接受医疗治疗，被邀请参加。如果人们有过期的一氧化碳干预：简短的戒烟建议，电子烟入门工具包和转介戒烟服务，则被排除在外。主要结局指标：主要结局是6个月时经生化验证的持续禁欲。那些失去随访或没有提供生化验证的人被认为不是禁欲。次要结果是：自我报告的7天戒烟情况、戒烟尝试次数、每天吸烟次数、尼古丁依赖以及自我报告的干咳、口腔或喉咙发炎的发生率。结果：在6个月时，972名随机受试者中，经生化验证的戒烟率在干预组为7.2%，在对照组为4.1%（百分比差异= 3.3%）(95%置信区间0.3 ~ 6.3；P = 0.032)[相对危险度1.76(95%可信区间1.03 ~ 3.01)]。干预组6个月时自我报告的7天戒断率为23.3%，对照组为12.9%（百分比差异= 10.6%）(95%置信区间5.86 ~ 15.41；p限制：无法使参与者或研究人员失明，由于所招募人群的性质，生化验证水平相对较低，并且对照组的人没有接受常规护理。结论：机会性戒烟干预包括简短的建议、电子烟入门工具包和转诊戒烟服务，对持续戒烟有效，几乎没有不良事件报告。未来的工作：未来的工作将包括在急诊科测试其他行为改变干预措施，并将戒烟试验在急诊科的干预措施适应其他环境。资助：本摘要介绍了由国家卫生与保健研究所（NIHR）卫生技术评估计划资助的独立研究，奖励号为NIHR129438。
{"title":"Cessation of smoking in people attending UK emergency departments: the COSTED RCT with economic and process evaluation.","authors":"Ian Pope, Lucy V Clark, Allan Clark, Emma Ward, Pippa Belderson, Susan Stirling, Steve Parrott, Jinshuo Li, Timothy Coats, Linda Bauld, Richard Holland, Sarah Gentry, Sanjay Agrawal, Benjamin M Bloom, Adrian Boyle, Alasdair Gray, M Geraint Morris, Caitlin Notley","doi":"10.3310/JHFR0841","DOIUrl":"10.3310/JHFR0841","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;The emergency department represents a potentially valuable opportunity to support smoking cessation. Evidence is lacking around the use of e-cigarettes in opportunistic settings like the emergency department.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To undertake a randomised controlled trial in people who smoke attending United Kingdom emergency departments, testing a brief intervention which included provision of an e-cigarette versus signposting to smoking cessation services, assessing smoking abstinence.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;A two-arm pragmatic, multicentre, parallel-group, individually randomised, controlled superiority trial with an internal pilot, economic evaluation and mixed-methods process evaluation.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Six emergency departments across England and Scotland.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Adults who smoked daily, who were attending the emergency department for medical treatment or accompanying someone attending for medical treatment, were invited to participate. People were excluded if they had an expired carbon monoxide of &lt; 8 parts per million, required immediate medical treatment, were in police custody, had a known allergy to nicotine, were daily e-cigarette users, were considered not to have capacity to consent or had already taken part in the trial.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Intervention: &lt;/strong&gt;Brief stop smoking advice, e-cigarette starter kit and referral to stop smoking services.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;The primary outcome was biochemically validated sustained abstinence at 6 months. Those lost to follow-up, or not providing biochemical verification, were considered not to be abstinent. Secondary outcomes were: self-reported 7-day smoking abstinence, number of quit attempts, number of cigarettes per day, nicotine dependence and incidence of self-reported dry cough or mouth or throat irritation.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;At 6 months, of 972 participants randomised, biochemically verified smoking abstinence was 7.2% in the intervention group and 4.1% in the control group (percentage difference = 3.3%) (95% confidence interval 0.3 to 6.3; &lt;i&gt;p&lt;/i&gt; = 0.032) [relative risk 1.76 (95% confidence interval 1.03 to 3.01)]. Self-reported 7-day abstinence at 6 months was 23.3% in the intervention group and 12.9% in the control group (percentage difference = 10.6%) (95% confidence interval 5.86 to 15.41; &lt;i&gt;p&lt;/i&gt; &lt; 0.001) [relative risk 1.80 (95% confidence interval 1.36 to 2.38)]. Daily e-cigarette use was 39.4% in the intervention group and 17.5% in the control group at 6 months. No serious adverse events related to taking part in the trial were reported. The economic evaluation found the intervention was likely to be cost-effective, judged by the National Institute for Health and Care Excellence threshold. The process evaluation found the intervention to be acceptable to both staff delivering it and participants receiving it. The brief nature of the i","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 35","pages":"1-36"},"PeriodicalIF":4.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376004/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144759961","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The risks, benefits, and resource implications of different diets in gastrostomy-fed children: The YourTube mixed method study. 对胃造口喂养儿童不同饮食的风险、益处和资源含义：YourTube混合方法研究。
IF 3.5 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/RRREF7741
Lorna Fraser, Andre Bedendo, Mark O'Neill, Johanna Taylor, Julia Hackett, Karen Horridge, Janet Cade, Gerry Richardson, Thai Han Phung, Bryony Beresford, Alison McCarter, Catherine Hewitt

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

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

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

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

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

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

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

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

Limitations:

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

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

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

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

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

背景：液体清除是透析治疗的关键组成部分，但如果过度，可导致残留肾功能更快下降。因此，在透析中规定最佳的液体清除方法以避免这种情况是重要的。生物阻抗光谱是一种评估组织水合作用的床边设备，它可能会改善这种处方，从而降低肾功能下降的速度，改善患者的治疗效果。我们希望在追求这种治疗策略时建立生物阻抗的功效和成本效益。方法：我们在血液透析患者中进行了一项多中心、开放标签、平行、单独随机对照试验，对照组的临床医生和患者对生物阻抗读数不了解。符合条件的患者尿量为> 500 ml/天或肾小球滤过率> 3 ml/分钟/1.73 m2。随机化是1:1，使用隐藏的自动计算机生成的分配系统按中心分层。临床评估每月进行一次，持续3个月，然后每3个月进行一次，持续24个月，在两组中使用标准化表格，在干预组中补充正常水合体重的生物阻抗估计。主要观察指标为无尿时间；次要结局是剩余肾功能、血压、透析相关症状（综合缓和治疗结局量表-肾脏）、生活质量（EuroQol）和每增加一个质量调整生命年的增量成本下降率。结果：从34个英国中心招募并分析了439名患者。无尿症的病因特异性危险率组间无差异，分别为0.751（95%可信区间0.459 ~ 1.229）和0.742（95%可信区间0.453 ~ 1.215）。肾功能下降比预期的要慢，第一年的合并线性率为-0.178(95%可信区间-0.196至-0.159)ml/min /1.73 m2/month；第二年：-0.061（95%置信区间-0.086至-0.036）毫升/分钟/1.73平方米/月。纵向血压、症状和患者报告的结果在组间没有差异。干预与对照组相比，每位患者的平均成本（2020年价格）降低了382英镑（95%可信区间- 3319至2556英镑），质量调整生命年增加了0.043英镑（95%可信区间-0.019至-0.105），且无危害。一项为期5年的事后分析发现，在入组时和未来2年的任何时候，患者的生存率更高，肾功能更残余。结论：在该队列中，使用标准化的临床方案进行液体评估，以避免过多的液体排出，可以很好地保存残余肾功能和更好的中期生存。生物阻抗测量并不需要实现这一点。在每个质量调整生命年增加2万英镑和3万英镑的支付意愿阈值下，干预具有成本效益的概率分别为76%和83%。局限性：该试验没有招募到目标（85%），主要结局的数量少于预期。该试验因2019年发现的冠状病毒疾病而中断，期间进行了193项（6.7%）液体评估和276项（8.1%）肾功能测量，但没有遗漏主要结果。未来工作：年龄、种族和残余肾功能下降之间的关系需要进一步研究。维持肾输出量的生物阻抗光谱确定了血液透析中与液体管理相关的中心水平变化，需要评估。资助：本摘要介绍了由国家卫生与保健研究所（NIHR）卫生技术评估方案资助的独立研究，奖励号为14/216/01。
{"title":"BioImpedance Spectroscopy to maintain Renal Output: the BISTRO randomised controlled trial.","authors":"Simon J Davies, David Coyle, Elizabeth Lindley, David Keane, John Belcher, Fergus Caskey, Indranil Dasgupta, Andrew Davenport, Ken Farrington, Sandip Mitra, Paula Ormandy, Martin Wilkie, Jamie MacDonald, Mandana Zanganeh, Lazaros Andronis, Ivonne Solis-Trapala, Julius Sim","doi":"10.3310/RHON2378","DOIUrl":"10.3310/RHON2378","url":null,"abstract":"<p><strong>Background: </strong>Fluid removal is a key component of dialysis treatment but, if excessive, can result in a faster decline in residual kidney function. Prescribing the optimal removal of fluid on dialysis to avoid this is therefore important. Bioimpedance spectroscopy, a bedside device that estimates tissue hydration, might improve this prescription, so reducing the rate of decline in kidney function and improving patient outcomes. We wished to establish the efficacy and cost-effectiveness of bioimpedance in pursuing this treatment strategy.</p><p><strong>Methods: </strong>We undertook a multicentre, open-label, parallel, individually randomised controlled trial in incident haemodialysis patients, with clinicians and patients blinded to bioimpedance readings in the control group. Eligible patients had a urine output of > 500 ml/day or a glomerular filtration rate > 3 ml/minute/1.73 m<sup>2</sup>. Randomisation was 1 : 1 using a concealed automated computer-generated allocation system stratified by centre. Clinical assessments were made monthly for 3 months and then every 3 months for up to 24 months using a standardised proforma in both groups, supplemented in the intervention group by the bioimpedance estimate of the normally hydrated weight. The primary outcome was time to anuria; secondary outcomes were rate in decline of residual kidney function, blood pressure, dialysis-related symptoms (Integrated Palliative Care Outcome Scale-Renal), quality of life (EuroQol) and incremental cost per additional quality-adjusted life-year gained.</p><p><strong>Results: </strong>Four hundred and thirty-nine patients were recruited and analysed from 34 United Kingdom centres. There were no between-group differences in cause-specific hazard rates of anuria, 0.751 (95% confidence interval 0.459 to 1.229) or subdistribution hazard rates 0.742 (95% confidence interval 0.453 to 1.215). Kidney function decline was slower than anticipated, pooled linear rates in year 1: -0.178 (95% confidence interval -0.196 to -0.159) ml/minute/1.73 m<sup>2</sup>/month; year 2: -0.061 (95% confidence interval -0.086 to -0.036) ml/minute/1.73 m<sup>2</sup>/month. Longitudinal blood pressure, symptoms and patient-reported outcomes did not differ by group. The intervention was associated with £382 (95% confidence interval -£3319 to £2556) lower average cost per patient (price year 2020) and 0.043 (95% confidence interval -0.019 to -0.105) more quality-adjusted life-years and no harm compared to control. A post hoc 5-year analysis found better survival with more residual kidney function at enrolment and at any time over the next 2 years.</p><p><strong>Conclusion: </strong>The use of a standardised clinical protocol for fluid assessment to avoid excessive fluid removal is associated with excellent preservation of residual kidney function and better medium-term survival in this cohort. Bioimpedance measurements are not necessary to achieve this. Probability of the intervent","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 32","pages":"1-23"},"PeriodicalIF":4.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12336963/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144775302","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Extracorporeal carbon dioxide removal for the treatment of acute hypoxaemic respiratory failure: the REST RCT. 体外二氧化碳去除治疗急性低氧性呼吸衰竭：REST RCT。
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/GJDM0320
James J McNamee, Ashley Agus, Andrew J Boyle, Colette Jackson, Cliona McDowell, Jeanette Haglund, Danny F McAuley
<p><strong>Background: </strong>In patients who require mechanical ventilation for acute hypoxaemic respiratory failure, further reduction in tidal volumes, compared with conventional low tidal volume ventilation, may improve outcomes.</p><p><strong>Objective: </strong>To determine whether using extracorporeal carbon dioxide removal improves outcomes in patients with acute hypoxaemic respiratory failure and is cost-effective.</p><p><strong>Design: </strong>A multicentre, randomised, allocation-concealed, open-label, pragmatic clinical trial.</p><p><strong>Setting: </strong>Fifty-one intensive care units across the United Kingdom.</p><p><strong>Participants: </strong>Four hundred and twelve adult patients receiving mechanical ventilation for acute hypoxaemic respiratory failure, of a planned sample size of 1120.</p><p><strong>Interventions: </strong>Lower tidal volume ventilation facilitated by extracorporeal carbon dioxide removal for at least 48 hours (<i>n</i> = 202) or standard care with conventional low tidal volume ventilation (<i>n</i> = 210).</p><p><strong>Main outcome measures: </strong>All-cause mortality 90 days. Secondary outcomes included ventilator-free days; adverse events; extracorporeal membrane oxygenation use; long-term mortality; health-related quality of life; health service costs; long-term respiratory morbidity.</p><p><strong>Results: </strong>The trial was stopped early because of futility and feasibility. The 90-day mortality rate was 41.5% in the extracorporeal carbon dioxide removal group versus 39.5% in the standard care group (risk ratio 1.05, 95% confidence interval 0.83 to 1.33; difference 2.0%, 95% confidence interval  - 7.6% to 11.5%; <i>p</i> = 0.68). There were significantly fewer mean ventilator-free days in the extracorporeal carbon dioxide removal group compared with the standard care group (7.1, 95% confidence interval 5.9 to 8.3) versus (9.2, 95% confidence interval 7.9 to 10.4) days; mean difference, -2.1 (95% confidence interval -3.8 to -0.3; <i>p</i> = 0.02). Serious adverse events were reported for 62 patients (31%) in extracorporeal carbon dioxide removal group and 18 (9%) in the standard care group, including intracranial haemorrhage in 9 patients (4.5%) versus 0 (0%) and bleeding at other sites in 6 (3.0%) versus 1 (0.5%) in the extracorporeal carbon dioxide removal group versus the control group. Two-year mortality data were available for 95% of patients. There was no difference in the time to death between groups (hazard ratio 1.08, 95% confidence interval 0.81 to 1.44; log-rank test <i>p</i> = 0.61). There was no difference in long-term outcomes between groups. There was no difference in quality-adjusted life-years at 12 months (mean difference -0.01, 95% confidence interval -0.06 to 0.05). Total 12-month costs were statistically significantly higher in the extracorporeal carbon dioxide removal group (mean difference £7668.76, 95% confidence interval £159.75 to £15,177.77). Secondary analyses indic
背景：在急性低氧性呼吸衰竭需要机械通气的患者中，与传统的低潮气量通气相比，进一步降低潮气量可能改善预后。目的：确定体外二氧化碳清除术是否能改善急性低氧性呼吸衰竭患者的预后，是否具有成本效益。设计：一项多中心、随机、分配隐蔽、开放标签、实用的临床试验。环境：全英国共有51个重症监护病房。参与者：1200名因急性低氧性呼吸衰竭接受机械通气的成年患者，计划样本量为1120人。干预措施：低潮气量通气通过体外二氧化碳清除至少48小时（n = 202）或标准护理常规低潮气量通气（n = 210）。主要结局指标：90天全因死亡率。次要结局包括无呼吸机天数；不良事件;体外膜氧合应用；长期死亡率;与健康有关的生活质量；保健服务费用；长期呼吸道疾病。结果：试验因无效和可行性而提前终止。体外二氧化碳去除组90天死亡率为41.5%，标准护理组为39.5%(风险比1.05,95%可信区间0.83 ~ 1.33；差异2.0%，95%置信区间- 7.6%至11.5%；p = 0.68)。体外二氧化碳去除组的平均无呼吸机天数明显少于标准护理组（7.1,95%可信区间5.9至8.3）和（9.2,95%可信区间7.9至10.4）天；平均差值为-2.1(95%置信区间为-3.8 ~ -0.3；p = 0.02)。体外二氧化碳去除组报告了62例（31%）患者的严重不良事件，标准护理组报告了18例（9%）患者的严重不良事件，包括9例（4.5%）患者颅内出血，0例（0%），6例（3.0%）患者其他部位出血，1例（0.5%）患者体外二氧化碳去除组与对照组。95%的患者可获得两年死亡率数据。两组患者死亡时间差异无统计学意义(风险比1.08,95%可信区间0.81 ~ 1.44；Log-rank检验p = 0.61)。两组之间的长期结果没有差异。12个月时质量调整生命年无差异（平均差异为-0.01,95%可信区间为-0.06 ~ 0.05）。体外二氧化碳去除组12个月的总费用在统计学上显著高于对照组（平均差值为7668.76英镑，95%可信区间为159.75英镑至15177.77英镑）。二次分析表明，根据患者的生理特征，治疗效果可能存在异质性。一项系统综述支持了这些发现。局限性：只有6%的筛查患者被纳入研究；在研究开始之前，大多数地点对干预措施一无所知；由于这是一项实用的试验，护理的其他方面在每个组中都没有标准化；该试验可能没有足够的能力来发现临床上重要的差异，因为试验提前停止了；不可能对临床医生或患者进行盲测。结论：没有发现短期或长期的益处，并且该装置与较高的成本和潜在的显著并发症相关。在未来的临床试验之外，我们不建议在治疗低氧性呼吸衰竭患者的标准护理之外使用该设备。未来的工作：未来的研究可以进一步探索接受更大剂量体外二氧化碳去除的不同患者群体是否会受益，使用核心结果集并收集更广泛的长期结果，并考虑在恢复能力后尽快测量患者与健康相关的生活质量。资助：本摘要介绍了由国家卫生与保健研究所（NIHR）卫生技术评估方案资助的独立研究，奖励号为13/143/02。
{"title":"Extracorporeal carbon dioxide removal for the treatment of acute hypoxaemic respiratory failure: the REST RCT.","authors":"James J McNamee, Ashley Agus, Andrew J Boyle, Colette Jackson, Cliona McDowell, Jeanette Haglund, Danny F McAuley","doi":"10.3310/GJDM0320","DOIUrl":"10.3310/GJDM0320","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;In patients who require mechanical ventilation for acute hypoxaemic respiratory failure, further reduction in tidal volumes, compared with conventional low tidal volume ventilation, may improve outcomes.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To determine whether using extracorporeal carbon dioxide removal improves outcomes in patients with acute hypoxaemic respiratory failure and is cost-effective.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;A multicentre, randomised, allocation-concealed, open-label, pragmatic clinical trial.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Setting: &lt;/strong&gt;Fifty-one intensive care units across the United Kingdom.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Participants: &lt;/strong&gt;Four hundred and twelve adult patients receiving mechanical ventilation for acute hypoxaemic respiratory failure, of a planned sample size of 1120.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interventions: &lt;/strong&gt;Lower tidal volume ventilation facilitated by extracorporeal carbon dioxide removal for at least 48 hours (&lt;i&gt;n&lt;/i&gt; = 202) or standard care with conventional low tidal volume ventilation (&lt;i&gt;n&lt;/i&gt; = 210).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Main outcome measures: &lt;/strong&gt;All-cause mortality 90 days. Secondary outcomes included ventilator-free days; adverse events; extracorporeal membrane oxygenation use; long-term mortality; health-related quality of life; health service costs; long-term respiratory morbidity.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The trial was stopped early because of futility and feasibility. The 90-day mortality rate was 41.5% in the extracorporeal carbon dioxide removal group versus 39.5% in the standard care group (risk ratio 1.05, 95% confidence interval 0.83 to 1.33; difference 2.0%, 95% confidence interval  - 7.6% to 11.5%; &lt;i&gt;p&lt;/i&gt; = 0.68). There were significantly fewer mean ventilator-free days in the extracorporeal carbon dioxide removal group compared with the standard care group (7.1, 95% confidence interval 5.9 to 8.3) versus (9.2, 95% confidence interval 7.9 to 10.4) days; mean difference, -2.1 (95% confidence interval -3.8 to -0.3; &lt;i&gt;p&lt;/i&gt; = 0.02). Serious adverse events were reported for 62 patients (31%) in extracorporeal carbon dioxide removal group and 18 (9%) in the standard care group, including intracranial haemorrhage in 9 patients (4.5%) versus 0 (0%) and bleeding at other sites in 6 (3.0%) versus 1 (0.5%) in the extracorporeal carbon dioxide removal group versus the control group. Two-year mortality data were available for 95% of patients. There was no difference in the time to death between groups (hazard ratio 1.08, 95% confidence interval 0.81 to 1.44; log-rank test &lt;i&gt;p&lt;/i&gt; = 0.61). There was no difference in long-term outcomes between groups. There was no difference in quality-adjusted life-years at 12 months (mean difference -0.01, 95% confidence interval -0.06 to 0.05). Total 12-month costs were statistically significantly higher in the extracorporeal carbon dioxide removal group (mean difference £7668.76, 95% confidence interval £159.75 to £15,177.77). Secondary analyses indic","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"29 33","pages":"1-16"},"PeriodicalIF":4.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12376005/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144775303","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Feasibility, acceptability and appropriateness of laparoscopic versus abdominal hysterectomy for women and healthcare professionals: the LAVA trial qualitative process evaluation. 妇女和医疗保健专业人员腹腔镜子宫切除术与腹式子宫切除术的可行性、可接受性和适宜性：LAVA试验定性过程评价
IF 4 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Health technology assessment
Pub Date : 2025-07-01 DOI: 10.3310/GJTC1325
Lynsay Matthews, T Justin Clark, Sheriden Bevan, Lee Middleton, Lina Antoun, Paul Smith, Ertan Saridogan, Rebecca Woolley, Monique Morgan, Laura L Jones
<p><strong>Background: </strong>Laparoscopic hysterectomies performed for benign gynaecological conditions are increasing. However, there is a lack of up-to-date evidence on their surgical outcomes when compared with abdominal hysterectomy. The LAparoscopic Versus Abdominal hysterectomy trial aimed to address this gap. A qualitative process evaluation was embedded within the pilot phase of the LAparoscopic Versus Abdominal hysterectomy trial.</p><p><strong>Objective: </strong>To explore the feasibility, acceptability and appropriateness of LAparoscopic Versus Abdominal hysterectomy for women and healthcare professionals.</p><p><strong>Design and methods: </strong>A qualitative process evaluation using semistructured interviews informed by the Medical Research Council/National Institute for Health and Care Research updated Framework for Developing and Evaluating Complex Interventions. Interviews were thematically analysed to inform the development of a LAparoscopic Versus Abdominal hysterectomy trial programme theory (used to demonstrate how an intervention is expected to lead to its effects, under what conditions and for which stakeholders).</p><p><strong>Setting and participants: </strong>Eligible women and healthcare professionals (gynaecologists, research nurses and research midwives) from participating clinical sites in National Health Service England.</p><p><strong>Main outcome measures: </strong>Insight on the feasibility,acceptability and appropriateness of LAparoscopic Versus Abdominal hysterectomy related to the: (1) environment, (2) patient and (3) the healthcare professionals.</p><p><strong>Results: </strong>Eleven women and 7 healthcare professionals (6 research nurses and 1 consultant gynaecologist) were interviewed. Four themes were interpreted. Theme 1 identified <b>decision-making processes</b> for LAparoscopic Versus Abdominal hysterectomy participation. <i>Conditional altruism</i> motivated women to participate, alongside the 'relief' of being offered a hysterectomy. The decision to decline participation was influenced by surgical preference and beliefs of laparoscopy having a faster recovery rate. Theme 2 highlighted <b>surgical preferences</b>, with women's preferences being influenced by their previous experiences of surgery or perceived recovery times and family/friends. All healthcare professionals demonstrated <i>community equipoise</i> but did observe that 'younger surgeons' may prefer laparoscopic surgery based on their contemporary training. Theme 3 identified <b>attitudes towards the LAparoscopic Versus Abdominal hysterectomy trial</b>, with women and healthcare professionals reporting positively about LAparoscopic Versus Abdominal hysterectomy's feasibility, acceptability and appropriateness in terms of burden, information and understanding of the study. Theme 4 identified the <b>facilitators and barriers</b> for LAparoscopic Versus Abdominal hysterectomy participation. Facilitators included the key role of the resear
背景：腹腔镜子宫切除术在妇科良性疾病中的应用越来越多。然而，与腹式子宫切除术相比，缺乏最新的手术结果证据。腹腔镜与腹部子宫切除术试验旨在解决这一差距。在腹腔镜子宫切除术与腹式子宫切除术试验的试点阶段进行了定性过程评估。目的：探讨腹腔镜子宫切除术与腹式子宫切除术的可行性、可接受性和适宜性。设计和方法：采用医学研究委员会/国家卫生和保健研究所更新的制定和评估复杂干预措施框架通知的半结构化访谈进行定性过程评估。对访谈进行主题分析，为腹腔镜与腹部子宫切除术试验计划理论的发展提供信息（用于证明干预措施如何预期产生效果，在什么条件下以及针对哪些利益相关者）。环境和参与者：符合条件的妇女和医疗保健专业人员（妇科医生、研究护士和研究助产士），来自英格兰国家卫生服务中心参与的临床站点。主要观察指标：腹腔镜子宫切除术与腹式子宫切除术的可行性、可接受性和适宜性与：(1)环境、(2)患者和(3)医护人员相关。结果：11名妇女和7名保健专业人员（6名研究护士和1名妇科咨询医生）接受了采访。阐释了四个主题。主题1确定参与腹腔镜子宫切除术与腹式子宫切除术的决策过程。有条件的利他主义激励着女性参与其中，同时还有被提供子宫切除术的“解脱”。拒绝参与的决定受手术偏好和相信腹腔镜有更快的恢复率的影响。主题2强调了手术偏好，女性的偏好受到她们以前的手术经历或预期的恢复时间以及家人/朋友的影响。所有医疗保健专业人员都表现出社区平等，但确实观察到“年轻外科医生”可能更喜欢基于他们当代培训的腹腔镜手术。主题3确定了对腹腔镜与腹式子宫切除术试验的态度，妇女和医疗保健专业人员在负担、信息和对研究的理解方面积极报告腹腔镜与腹式子宫切除术的可行性、可接受性和适宜性。主题4确定了腹腔镜子宫切除术与腹部子宫切除术的促进因素和障碍。促进者包括研究护士的关键作用，以及在康复期间获得个人社会支持的妇女。电话咨询可能是一个障碍，妇女和保健专业人员更喜欢面对面的讨论。这些发现完善了腹腔镜与腹部子宫切除术方案理论，确定了与环境、患者和医疗保健专业人员相关的因素的相互作用。局限性：大多数来自女性的见解来自一个站点（72.7%），大多数医疗保健专业人员的见解来自研究护士（85.7%）。只有说英语的参与者被招募到腹腔镜与腹部子宫切除术。结论：总的来说，腹腔镜子宫切除术和腹式子宫切除术对于女性和医疗保健专业人员来说是可以接受的。然而，由于COVID-19大流行的负面影响和缺乏医疗保健专业平衡（这些发现已在之前发表），该试验提前结束。定性过程评价强调了未来试验需要考虑的其他因素，包括对妇女决策的影响以及解决患者和保健专业人员平等问题的挑战。未来的工作：腹腔镜和腹式子宫切除术的结果的比较仍需要在大规模随机对照试验中进行探索。需要从拒绝参与的妇女和缺乏平等的保健专业人员那里获得进一步的定性见解。资助：本文介绍了由国家卫生与保健研究所（NIHR）卫生技术评估计划资助的独立研究，奖励号为NIHR128991。
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引用次数: 0
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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