Mandana Zanganeh, John Belcher, James Fotheringham, David Coyle, Elizabeth J Lindley, David F Keane, Fergus J Caskey, Indranil Dasgupta, Andrew Davenport, Ken Farrington, Sandip Mitra, Paula Ormandy, Martin Wilkie, Jamie H Macdonald, Ivonne Solis-Trapala, Julius Sim, Simon J Davies, Lazaros Andronis
<p><strong>Background: </strong>The BioImpedance Spectroscopy to maintain Renal Output randomised controlled trial investigated the effect of bioimpedance spectroscopy added to a standardised fluid management protocol on the risk of anuria and preservation of residual kidney function (primary trial outcomes) in incident haemodialysis patients. Despite the economic burden of kidney disease, the cost-effectiveness of using bioimpedance measurements to guide fluid management in haemodialysis is not known.</p><p><strong>Objectives: </strong>To assess the cost-effectiveness of bioimpedance-guided fluid management against current fluid management without bioimpedance.</p><p><strong>Design: </strong>Within-trial economic evaluation (cost-utility analysis) carried out alongside the open-label, multicentre BioImpedance Spectroscopy to maintain Renal Output randomised controlled trial.</p><p><strong>Setting: </strong>Thirty-four United Kingdom outpatient haemodialysis centres, both main and satellite units, and their associated inpatient hospitals.</p><p><strong>Participants: </strong>Four hundred and thirty-nine adult haemodialysis patients with > 500 ml urine/day or residual glomerular filtration rate > 3 ml/minute/1.73 m<sup>2</sup>.</p><p><strong>Intervention: </strong>The study intervention was the incorporation of bioimpedance technology-derived information about body composition into the clinical assessment of fluid status in patients with residual kidney function undergoing haemodialysis. Bioimpedance measurements were used in conjunction with usual clinical judgement to set a target weight that would avoid excessive fluid depletion at the end of a dialysis session.</p><p><strong>Main outcome measures: </strong>The primary outcome measure of the BioImpedance Spectroscopy to maintain Renal Output economic evaluation was incremental cost per additional quality-adjusted life-year gained over 24 months following randomisation. In the main (base-case) analysis, this was calculated from the perspective of the National Health Service and Personal Social Services. Sensitivity analyses explored the impact of different scenarios, sources of resource use data and value sets.</p><p><strong>Results: </strong>The bioimpedance-guided fluid management group was associated with £382 lower average cost per patient (95% CI -£3319 to £2556) and 0.043 more quality-adjusted life-years (95% CI -0.019 to 0.105) compared with the current fluid management group, with neither values being statistically significant. The probability of bioimpedance-guided fluid management being cost-effective was 76% and 83% at commonly cited willingness-to-pay threshold of £20,000 and £30,000 per quality-adjusted life-year gained, respectively. The results remained robust to a series of sensitivity analyses.</p><p><strong>Limitations: </strong>The missing data level was high for some resource use categories collected through case report forms, due to COVID-19 disruptions and a significant dro
{"title":"Cost-effectiveness of bioimpedance-guided fluid management in patients undergoing haemodialysis: the BISTRO RCT.","authors":"Mandana Zanganeh, John Belcher, James Fotheringham, David Coyle, Elizabeth J Lindley, David F Keane, Fergus J Caskey, Indranil Dasgupta, Andrew Davenport, Ken Farrington, Sandip Mitra, Paula Ormandy, Martin Wilkie, Jamie H Macdonald, Ivonne Solis-Trapala, Julius Sim, Simon J Davies, Lazaros Andronis","doi":"10.3310/JYPR4287","DOIUrl":"https://doi.org/10.3310/JYPR4287","url":null,"abstract":"<p><strong>Background: </strong>The BioImpedance Spectroscopy to maintain Renal Output randomised controlled trial investigated the effect of bioimpedance spectroscopy added to a standardised fluid management protocol on the risk of anuria and preservation of residual kidney function (primary trial outcomes) in incident haemodialysis patients. Despite the economic burden of kidney disease, the cost-effectiveness of using bioimpedance measurements to guide fluid management in haemodialysis is not known.</p><p><strong>Objectives: </strong>To assess the cost-effectiveness of bioimpedance-guided fluid management against current fluid management without bioimpedance.</p><p><strong>Design: </strong>Within-trial economic evaluation (cost-utility analysis) carried out alongside the open-label, multicentre BioImpedance Spectroscopy to maintain Renal Output randomised controlled trial.</p><p><strong>Setting: </strong>Thirty-four United Kingdom outpatient haemodialysis centres, both main and satellite units, and their associated inpatient hospitals.</p><p><strong>Participants: </strong>Four hundred and thirty-nine adult haemodialysis patients with > 500 ml urine/day or residual glomerular filtration rate > 3 ml/minute/1.73 m<sup>2</sup>.</p><p><strong>Intervention: </strong>The study intervention was the incorporation of bioimpedance technology-derived information about body composition into the clinical assessment of fluid status in patients with residual kidney function undergoing haemodialysis. Bioimpedance measurements were used in conjunction with usual clinical judgement to set a target weight that would avoid excessive fluid depletion at the end of a dialysis session.</p><p><strong>Main outcome measures: </strong>The primary outcome measure of the BioImpedance Spectroscopy to maintain Renal Output economic evaluation was incremental cost per additional quality-adjusted life-year gained over 24 months following randomisation. In the main (base-case) analysis, this was calculated from the perspective of the National Health Service and Personal Social Services. Sensitivity analyses explored the impact of different scenarios, sources of resource use data and value sets.</p><p><strong>Results: </strong>The bioimpedance-guided fluid management group was associated with £382 lower average cost per patient (95% CI -£3319 to £2556) and 0.043 more quality-adjusted life-years (95% CI -0.019 to 0.105) compared with the current fluid management group, with neither values being statistically significant. The probability of bioimpedance-guided fluid management being cost-effective was 76% and 83% at commonly cited willingness-to-pay threshold of £20,000 and £30,000 per quality-adjusted life-year gained, respectively. The results remained robust to a series of sensitivity analyses.</p><p><strong>Limitations: </strong>The missing data level was high for some resource use categories collected through case report forms, due to COVID-19 disruptions and a significant dro","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-45"},"PeriodicalIF":3.5,"publicationDate":"2024-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142345521","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ros Wade, Nyanar Jasmine Deng, Chinyereugo Umemneku-Chikere, Melissa Harden, Helen Fulbright, Robert Hodgson, Alison Eastwood, Rachel Churchill
<p><strong>Background: </strong>This work was undertaken to inform a National Institute for Health and Care Excellence guideline on the initial assessment of adults with suspected acute respiratory infection.</p><p><strong>Objective: </strong>To undertake a rapid evidence synthesis of systematic reviews and cost-effectiveness studies of signs, symptoms and early warning scores for the initial assessment of adults with suspected acute respiratory infection.</p><p><strong>Methods: </strong>MEDLINE, EMBASE and Cochrane Database of Systematic Reviews were searched for systematic reviews and MEDLINE, EMBASE, EconLit and National Health Service Economic Evaluation Database were searched for cost-effectiveness studies in May 2023. References of relevant studies were checked. Clinical outcomes of interest included escalation of care, antibiotic/antiviral use, time to resolution of symptoms, mortality and health-related quality of life. Risk of bias was assessed using the Risk of Bias in Systematic Reviews tool or the National Institute for Health and Care Excellence economic evaluations checklist. Results were summarised using narrative synthesis.</p><p><strong>Results: </strong>Nine systematic reviews and one cost-effectiveness study met eligibility criteria. Seven reviews assessed several early warning scores for patients with community- acquired pneumonia, one assessed early warning scores for nursing home-acquired pneumonia and one assessed individual signs/symptoms and the Centor score for patients with sore throat symptoms; all in face-to-face settings. Two good-quality reviews concluded that further research is needed to validate the CRB-65 in primary care/community settings. One also concluded that further research is needed on the Pneumonia Severity Index in community settings; however, the Pneumonia Severity Index requires data from tests not routinely conducted in community settings. One good-quality review concluded that National Early Warning Score appears to be useful in an emergency department/acute medical setting. One review (unclear quality) concluded that the Pneumonia Severity Index and CURB-65 appear useful in an emergency department setting. Two poor-quality reviews concluded that early warning scores can support clinical judgement and one poor-quality review found numerous problems with using early warning scores in a nursing home setting. A good-quality review concluded that individual signs and symptoms have a modest ability to diagnose streptococcal pharyngitis, and that the Centor score can enhance appropriate prescribing of antibiotics. The cost-effectiveness study assessed clinical scores and rapid antigen detection tests for sore throat, compared to delayed antibiotic prescribing. The study concluded that the clinical score is a cost-effective approach when compared to delayed prescribing and rapid antigen testing.</p><p><strong>Conclusions: </strong>Several early warning scores have been evaluated in adults with suspected a
背景：这项工作的目的是为美国国家健康与护理卓越研究所（National Institute of Health and Care Excellence）关于成人疑似急性呼吸道感染初步评估指南提供信息：这项工作的目的是为美国国家健康与护理卓越研究所（National Institute for Health and Care Excellence）关于对疑似急性呼吸道感染成人患者进行初步评估的指南提供信息：对用于初步评估疑似急性呼吸道感染成人的体征、症状和预警评分的系统综述和成本效益研究进行快速证据综合：方法：检索 MEDLINE、EMBASE 和 Cochrane 系统综述数据库中的系统综述，并检索 MEDLINE、EMBASE、EconLit 和国民健康服务经济评估数据库中 2023 年 5 月的成本效益研究。对相关研究的参考文献进行了核对。关注的临床结果包括护理升级、抗生素/抗病毒药物使用、症状缓解时间、死亡率和健康相关生活质量。偏倚风险采用系统性综述偏倚风险工具或美国国家健康与护理卓越研究所经济评估清单进行评估。结果采用叙事综合法进行总结：九篇系统综述和一项成本效益研究符合资格标准。七篇综述评估了社区获得性肺炎患者的几种早期预警评分，一篇评估了护理院获得性肺炎的早期预警评分，一篇评估了咽喉痛症状患者的个体体征/症状和 Centor 评分；所有综述都是在面对面的环境中进行的。两篇质量较高的综述认为，需要进一步研究以验证 CRB-65 在初级保健/社区环境中的有效性。其中一篇综述还认为，需要进一步研究社区环境中的肺炎严重程度指数；然而，肺炎严重程度指数需要来自社区环境中并非常规进行的检测的数据。一篇质量良好的综述认为，国家预警评分似乎在急诊科/急性医疗环境中很有用。一篇综述（质量不明确）认为，肺炎严重程度指数和 CURB-65 似乎在急诊科环境中很有用。两篇质量较差的综述认为，预警评分可支持临床判断，一篇质量较差的综述发现，在养老院环境中使用预警评分存在许多问题。一篇质量较高的综述认为，单个体征和症状诊断链球菌性咽炎的能力一般，Centor 评分可提高抗生素处方的合理性。成本效益研究评估了咽喉炎临床评分和快速抗原检测试验与延迟抗生素处方的比较。研究认为，与延迟处方和快速抗原检测相比，临床评分是一种具有成本效益的方法：在成人疑似急性呼吸道感染患者中评估了几种预警评分，主要是 CRB-65、CURB-65 和社区获得性肺炎患者肺炎严重程度指数。目前尚无足够证据来确定何种分流策略可避免严重疾病。一些早期预警评分（CURB-65、肺炎严重程度指数和国家早期预警评分）在急诊科/急性医疗环境中似乎很有用；但是，还需要进一步研究，以验证 CRB-65 和肺炎严重程度指数在初级医疗/社区环境中的有效性。经济学证据表明，与延迟处方相比，临床评分可能是一种具有成本效益的患者分流方法：只有系统综述符合纳入临床证据综述的条件。纳入综述的主要研究有很多重叠之处，其中许多研究有很大的局限性。没有研究是在偏远地区进行的（如 NHS 111）。仅发现了一项成本效益研究，对综述问题的适用性有限：本文是由美国国家健康与护理研究所（NIHR）健康技术评估项目资助的独立研究，获奖编号为 NIHR159945。
{"title":"Initial assessment and management of adults with suspected acute respiratory infection: a rapid evidence synthesis of reviews and cost-effectiveness studies.","authors":"Ros Wade, Nyanar Jasmine Deng, Chinyereugo Umemneku-Chikere, Melissa Harden, Helen Fulbright, Robert Hodgson, Alison Eastwood, Rachel Churchill","doi":"10.3310/GRPL6978","DOIUrl":"https://doi.org/10.3310/GRPL6978","url":null,"abstract":"<p><strong>Background: </strong>This work was undertaken to inform a National Institute for Health and Care Excellence guideline on the initial assessment of adults with suspected acute respiratory infection.</p><p><strong>Objective: </strong>To undertake a rapid evidence synthesis of systematic reviews and cost-effectiveness studies of signs, symptoms and early warning scores for the initial assessment of adults with suspected acute respiratory infection.</p><p><strong>Methods: </strong>MEDLINE, EMBASE and Cochrane Database of Systematic Reviews were searched for systematic reviews and MEDLINE, EMBASE, EconLit and National Health Service Economic Evaluation Database were searched for cost-effectiveness studies in May 2023. References of relevant studies were checked. Clinical outcomes of interest included escalation of care, antibiotic/antiviral use, time to resolution of symptoms, mortality and health-related quality of life. Risk of bias was assessed using the Risk of Bias in Systematic Reviews tool or the National Institute for Health and Care Excellence economic evaluations checklist. Results were summarised using narrative synthesis.</p><p><strong>Results: </strong>Nine systematic reviews and one cost-effectiveness study met eligibility criteria. Seven reviews assessed several early warning scores for patients with community- acquired pneumonia, one assessed early warning scores for nursing home-acquired pneumonia and one assessed individual signs/symptoms and the Centor score for patients with sore throat symptoms; all in face-to-face settings. Two good-quality reviews concluded that further research is needed to validate the CRB-65 in primary care/community settings. One also concluded that further research is needed on the Pneumonia Severity Index in community settings; however, the Pneumonia Severity Index requires data from tests not routinely conducted in community settings. One good-quality review concluded that National Early Warning Score appears to be useful in an emergency department/acute medical setting. One review (unclear quality) concluded that the Pneumonia Severity Index and CURB-65 appear useful in an emergency department setting. Two poor-quality reviews concluded that early warning scores can support clinical judgement and one poor-quality review found numerous problems with using early warning scores in a nursing home setting. A good-quality review concluded that individual signs and symptoms have a modest ability to diagnose streptococcal pharyngitis, and that the Centor score can enhance appropriate prescribing of antibiotics. The cost-effectiveness study assessed clinical scores and rapid antigen detection tests for sore throat, compared to delayed antibiotic prescribing. The study concluded that the clinical score is a cost-effective approach when compared to delayed prescribing and rapid antigen testing.</p><p><strong>Conclusions: </strong>Several early warning scores have been evaluated in adults with suspected a","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-53"},"PeriodicalIF":3.5,"publicationDate":"2024-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285838","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kevin G Cooper, Siladitya Bhattacharya, Jane P Daniels, Versha Cheed, Laura Gennard, Lisa Leighton, Danielle Pirie, Melyda Melyda, Mark Monahan, Annalise Weckesser, Tracy Roberts, Elaine Denny, Laura Ocansey, Clive Stubbs, Emma Cox, Georgina Jones, T Justin Clark, Ertan Saridogan, Janesh K Gupta, Hilary Om Critchley, Andrew Horne, Lee J Middleton
<p><strong>Background: </strong>Endometriosis affects 1 in 10 women, many of whom have surgery for persistent pain. Recurrence of symptoms following an operation is common. Although hormonal treatment can reduce this risk, there is uncertainty about the best option.</p><p><strong>Objectives: </strong>To evaluate the clinical and cost-effectiveness of long-acting progestogen therapy compared with the combined oral contraceptive pill in preventing recurrence of endometriosis-related pain and quality of life.</p><p><strong>Design: </strong>A multicentre, open, randomised trial with parallel economic evaluation. The final design was informed by a pilot study, qualitative exploration of women's lived experience of endometriosis and a pretrial economic model.</p><p><strong>Setting: </strong>Thirty-four United Kingdom hospitals.</p><p><strong>Participants: </strong>Women of reproductive age undergoing conservative surgery for endometriosis.</p><p><strong>Interventions: </strong>Long-acting progestogen reversible contraceptive (either 150 mg depot medroxyprogesterone acetate or 52 mg levonorgestrel-releasing intrauterine system) or combined oral contraceptive pill (30 µg ethinylestradiol, 150 µg levonorgestrel).</p><p><strong>Main outcome measures: </strong>The primary outcome was the pain domain of the Endometriosis Health Profile-30 questionnaire at 36 months post randomisation. The economic evaluation estimated the cost per quality-adjusted life-years gained.</p><p><strong>Results: </strong>Four hundred and five women were randomised to receive either long-acting reversible contraceptive (<i>N</i> = 205) or combined oral contraceptive pill (<i>N</i> = 200). Pain scores improved in both groups (24 and 23 points on average) compared with preoperative values but there was no difference between the two (adjusted mean difference: -0.8, 95% confidence interval -5.7 to 4.2; <i>p</i> = 0.76). The long-acting reversible contraceptive group underwent fewer surgical procedures or second-line treatments compared with the combined oral contraceptive group (73 vs. 97; hazard ratio 0.67, 95% confidence interval 0.44 to 1.00). The mean adjusted quality-adjusted life-year difference between two arms was 0.043 (95% confidence interval -0.069 to 0.152) in favour of the combined oral contraceptive pill, although this cost an additional £533 (95% confidence interval 52 to 983) per woman.</p><p><strong>Limitations: </strong>Limitations include the absence of a no-treatment group and the fact that many women changed treatments over the 3 years of follow-up. Use of telephone follow-up to collect primary outcome data in those who failed to return questionnaires resulted in missing data for secondary outcomes. The COVID pandemic may have affected rates of further surgical treatment.</p><p><strong>Conclusions: </strong>At 36 months, women allocated to either intervention had comparable levels of pain, with both groups showing around a 40% improvement from presurgical levels. Al
{"title":"Preventing recurrence of endometriosis-related pain by means of long-acting progestogen therapy: the PRE-EMPT RCT.","authors":"Kevin G Cooper, Siladitya Bhattacharya, Jane P Daniels, Versha Cheed, Laura Gennard, Lisa Leighton, Danielle Pirie, Melyda Melyda, Mark Monahan, Annalise Weckesser, Tracy Roberts, Elaine Denny, Laura Ocansey, Clive Stubbs, Emma Cox, Georgina Jones, T Justin Clark, Ertan Saridogan, Janesh K Gupta, Hilary Om Critchley, Andrew Horne, Lee J Middleton","doi":"10.3310/SQWY6998","DOIUrl":"10.3310/SQWY6998","url":null,"abstract":"<p><strong>Background: </strong>Endometriosis affects 1 in 10 women, many of whom have surgery for persistent pain. Recurrence of symptoms following an operation is common. Although hormonal treatment can reduce this risk, there is uncertainty about the best option.</p><p><strong>Objectives: </strong>To evaluate the clinical and cost-effectiveness of long-acting progestogen therapy compared with the combined oral contraceptive pill in preventing recurrence of endometriosis-related pain and quality of life.</p><p><strong>Design: </strong>A multicentre, open, randomised trial with parallel economic evaluation. The final design was informed by a pilot study, qualitative exploration of women's lived experience of endometriosis and a pretrial economic model.</p><p><strong>Setting: </strong>Thirty-four United Kingdom hospitals.</p><p><strong>Participants: </strong>Women of reproductive age undergoing conservative surgery for endometriosis.</p><p><strong>Interventions: </strong>Long-acting progestogen reversible contraceptive (either 150 mg depot medroxyprogesterone acetate or 52 mg levonorgestrel-releasing intrauterine system) or combined oral contraceptive pill (30 µg ethinylestradiol, 150 µg levonorgestrel).</p><p><strong>Main outcome measures: </strong>The primary outcome was the pain domain of the Endometriosis Health Profile-30 questionnaire at 36 months post randomisation. The economic evaluation estimated the cost per quality-adjusted life-years gained.</p><p><strong>Results: </strong>Four hundred and five women were randomised to receive either long-acting reversible contraceptive (<i>N</i> = 205) or combined oral contraceptive pill (<i>N</i> = 200). Pain scores improved in both groups (24 and 23 points on average) compared with preoperative values but there was no difference between the two (adjusted mean difference: -0.8, 95% confidence interval -5.7 to 4.2; <i>p</i> = 0.76). The long-acting reversible contraceptive group underwent fewer surgical procedures or second-line treatments compared with the combined oral contraceptive group (73 vs. 97; hazard ratio 0.67, 95% confidence interval 0.44 to 1.00). The mean adjusted quality-adjusted life-year difference between two arms was 0.043 (95% confidence interval -0.069 to 0.152) in favour of the combined oral contraceptive pill, although this cost an additional £533 (95% confidence interval 52 to 983) per woman.</p><p><strong>Limitations: </strong>Limitations include the absence of a no-treatment group and the fact that many women changed treatments over the 3 years of follow-up. Use of telephone follow-up to collect primary outcome data in those who failed to return questionnaires resulted in missing data for secondary outcomes. The COVID pandemic may have affected rates of further surgical treatment.</p><p><strong>Conclusions: </strong>At 36 months, women allocated to either intervention had comparable levels of pain, with both groups showing around a 40% improvement from presurgical levels. Al","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 55","pages":"1-77"},"PeriodicalIF":3.5,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11417646/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285849","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Miriam Santer, Megan Lawrence, Sarah Pyne, Susanne Renz, Beth L Stuart, Tracey Sach, Matthew Ridd, Kim S Thomas, Jacqueline Nuttall, Natalia Permyakova, Zina Eminton, Nick Francis, Paul Little, Ingrid Muller, Irene Soulsby, Karen Thomas, Gareth Griffiths, Alison M Layton
<p><strong>Background: </strong>Acne is common, can cause significant impact on quality of life and is a frequent reason for long-term antibiotic use. Spironolactone has been prescribed for acne in women for many years, but robust evidence is lacking.</p><p><strong>Objective: </strong>To evaluate whether spironolactone is clinically effective and cost-effective in treating acne in women.</p><p><strong>Design: </strong>Pragmatic, parallel, double-blind, randomised superiority trial.</p><p><strong>Setting: </strong>Primary and secondary healthcare and community settings (community and social media advertising).</p><p><strong>Participants: </strong>Women aged 18 years and older with facial acne persisting for at least 6 months, judged to potentially warrant oral antibiotic treatment.</p><p><strong>Interventions: </strong>Participants were randomised 1 : 1, using an independent web-based procedure, to either 50 mg/day spironolactone or matched placebo until week 6, increasing to 100 mg/day spironolactone or matched placebo until week 24. Participants continued usual topical treatment.</p><p><strong>Main outcome measures: </strong>Primary outcome was the adjusted mean difference in Acne-Specific Quality of Life symptom subscale score at 12 weeks. Secondary outcomes included Acne-Specific Quality of Life total and subscales; participant self-assessed improvement; Investigator's Global Assessment; Participant's Global Assessment; satisfaction; adverse effects and cost-effectiveness.</p><p><strong>Results: </strong>Of 1267 women assessed for eligibility, 410 were randomised (201 intervention, 209 control), 342 in the primary analysis (176 intervention, 166 control). Mean age was 29.2 years (standard deviation 7.2) and 7.9% (28/356) were from non-white backgrounds. At baseline, Investigator's Global Assessment classified acne as mild in 46%, moderate in 40% and severe in 13%. At baseline, 82.9% were using topical treatments. Over 95% of participants in both groups tolerated the treatment and increased their dose. Mean baseline Acne-Specific Quality of Life symptom subscale was 13.0 (standard deviation 4.7) across both groups. Mean scores at week 12 were 19.2 (standard deviation 6.1) for spironolactone and 17.8 (standard deviation 5.6) for placebo [difference favouring spironolactone 1.27 (95% confidence interval 0.07 to 2.46) adjusting for baseline variables]. Mean scores at week 24 were 21.2 (standard deviation 5.9) in spironolactone group and 17.4 (standard deviation 5.8) in placebo group [adjusted difference 3.77 (95% confidence interval 2.50 to 5.03) adjusted]. Secondary outcomes also favoured spironolactone at 12 weeks with greater differences at 24 weeks. Participants taking spironolactone were more likely than those taking placebo to report overall acne improvement at 12 weeks {72.2% vs. 67.9% [adjusted odds ratio 1.16 (95% confidence interval 0.70 to 1.91)]} and at 24 weeks {81.9% vs. 63.3% [adjusted odds ratio 2.72 (95% confidence interval 1.50 t
{"title":"Clinical and cost-effectiveness of spironolactone in treating persistent facial acne in women: SAFA double-blinded RCT.","authors":"Miriam Santer, Megan Lawrence, Sarah Pyne, Susanne Renz, Beth L Stuart, Tracey Sach, Matthew Ridd, Kim S Thomas, Jacqueline Nuttall, Natalia Permyakova, Zina Eminton, Nick Francis, Paul Little, Ingrid Muller, Irene Soulsby, Karen Thomas, Gareth Griffiths, Alison M Layton","doi":"10.3310/MYJT6804","DOIUrl":"10.3310/MYJT6804","url":null,"abstract":"<p><strong>Background: </strong>Acne is common, can cause significant impact on quality of life and is a frequent reason for long-term antibiotic use. Spironolactone has been prescribed for acne in women for many years, but robust evidence is lacking.</p><p><strong>Objective: </strong>To evaluate whether spironolactone is clinically effective and cost-effective in treating acne in women.</p><p><strong>Design: </strong>Pragmatic, parallel, double-blind, randomised superiority trial.</p><p><strong>Setting: </strong>Primary and secondary healthcare and community settings (community and social media advertising).</p><p><strong>Participants: </strong>Women aged 18 years and older with facial acne persisting for at least 6 months, judged to potentially warrant oral antibiotic treatment.</p><p><strong>Interventions: </strong>Participants were randomised 1 : 1, using an independent web-based procedure, to either 50 mg/day spironolactone or matched placebo until week 6, increasing to 100 mg/day spironolactone or matched placebo until week 24. Participants continued usual topical treatment.</p><p><strong>Main outcome measures: </strong>Primary outcome was the adjusted mean difference in Acne-Specific Quality of Life symptom subscale score at 12 weeks. Secondary outcomes included Acne-Specific Quality of Life total and subscales; participant self-assessed improvement; Investigator's Global Assessment; Participant's Global Assessment; satisfaction; adverse effects and cost-effectiveness.</p><p><strong>Results: </strong>Of 1267 women assessed for eligibility, 410 were randomised (201 intervention, 209 control), 342 in the primary analysis (176 intervention, 166 control). Mean age was 29.2 years (standard deviation 7.2) and 7.9% (28/356) were from non-white backgrounds. At baseline, Investigator's Global Assessment classified acne as mild in 46%, moderate in 40% and severe in 13%. At baseline, 82.9% were using topical treatments. Over 95% of participants in both groups tolerated the treatment and increased their dose. Mean baseline Acne-Specific Quality of Life symptom subscale was 13.0 (standard deviation 4.7) across both groups. Mean scores at week 12 were 19.2 (standard deviation 6.1) for spironolactone and 17.8 (standard deviation 5.6) for placebo [difference favouring spironolactone 1.27 (95% confidence interval 0.07 to 2.46) adjusting for baseline variables]. Mean scores at week 24 were 21.2 (standard deviation 5.9) in spironolactone group and 17.4 (standard deviation 5.8) in placebo group [adjusted difference 3.77 (95% confidence interval 2.50 to 5.03) adjusted]. Secondary outcomes also favoured spironolactone at 12 weeks with greater differences at 24 weeks. Participants taking spironolactone were more likely than those taking placebo to report overall acne improvement at 12 weeks {72.2% vs. 67.9% [adjusted odds ratio 1.16 (95% confidence interval 0.70 to 1.91)]} and at 24 weeks {81.9% vs. 63.3% [adjusted odds ratio 2.72 (95% confidence interval 1.50 t","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 56","pages":"1-86"},"PeriodicalIF":3.5,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11418016/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285850","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}
Kurinchi Gurusamy, Jeffrey Leung, Claire Vale, Danielle Roberts, Audrey Linden, Xiao Wei Tan, Priyal Taribagil, Sonam Patel, Elena Pizzo, Brian Davidson, Tim Mould, Mark Saunders, Omer Aziz, Sarah O'Dwyer
<p><strong>Background: </strong>We compared the relative benefits, harms and cost-effectiveness of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery ± systemic chemotherapy versus cytoreductive surgery ± systemic chemotherapy or systemic chemotherapy alone in people with peritoneal metastases from colorectal, gastric or ovarian cancers by a systematic review, meta-analysis and model-based cost-utility analysis.</p><p><strong>Methods: </strong>We searched MEDLINE, EMBASE, Cochrane Library and the Science Citation Index, ClinicalTrials.gov and WHO ICTRP trial registers until 14 April 2022. We included only randomised controlled trials addressing the research objectives. We used the Cochrane risk of bias tool version 2 to assess the risk of bias in randomised controlled trials. We used the random-effects model for data synthesis when applicable. For the cost-effectiveness analysis, we performed a model-based cost-utility analysis using methods recommended by The National Institute for Health and Care Excellence.</p><p><strong>Results: </strong>The systematic review included a total of eight randomised controlled trials (seven randomised controlled trials, 955 participants included in the quantitative analysis). All comparisons other than those for stage III or greater epithelial ovarian cancer contained only one trial, indicating the paucity of randomised controlled trials that provided data. For colorectal cancer, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably results in little to no difference in all-cause mortality (60.6% vs. 60.6%; hazard ratio 1.00, 95% confidence interval 0.63 to 1.58) and may increase the serious adverse event proportions compared to cytoreductive surgery ± systemic chemotherapy (25.6% vs. 15.2%; risk ratio 1.69, 95% confidence interval 1.03 to 2.77). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to fluorouracil-based systemic chemotherapy alone (40.8% vs. 60.8%; hazard ratio 0.55, 95% confidence interval 0.32 to 0.95). For gastric cancer, there is high uncertainty about the effects of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy versus cytoreductive surgery + systemic chemotherapy or systemic chemotherapy alone on all-cause mortality. For stage III or greater epithelial ovarian cancer undergoing interval cytoreductive surgery, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to cytoreductive surgery + systemic chemotherapy (46.3% vs. 57.4%; hazard ratio 0.73, 95% confidence interval 0.57 to 0.93). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy may not be cost-effective versus cytoreductive surgery + systemic chemotherapy for colorectal
背景：我们通过系统综述、荟萃分析和基于模型的成本效用分析，比较了在结直肠癌、胃癌或卵巢癌腹膜转移患者中，热疗术中腹膜化疗+细胞减灭术±全身化疗与细胞减灭术±全身化疗或单纯全身化疗的相对益处、危害和成本效用：截至 2022 年 4 月 14 日，我们检索了 MEDLINE、EMBASE、Cochrane 图书馆和科学引文索引、ClinicalTrials.gov 和 WHO ICTRP 试验登记。我们只纳入了针对研究目标的随机对照试验。我们使用 Cochrane 第 2 版偏倚风险工具来评估随机对照试验的偏倚风险。在适用的情况下，我们使用随机效应模型进行数据综合。在成本效益分析中，我们采用美国国家健康与护理卓越研究所推荐的方法进行了基于模型的成本效益分析：系统综述共包括 8 项随机对照试验（7 项随机对照试验，955 名参与者参与了定量分析）。除了针对 III 期或以上上皮性卵巢癌的比较外，其他所有比较都只包含一项试验，这表明提供数据的随机对照试验很少。对于结直肠癌，术中热腹膜化疗+细胞减灭术+全身化疗可能导致的全因死亡率几乎没有差异（60.6% vs. 60.6%；危险比 1.00，95% 置信区间 0.63 至 1.58），但与细胞减灭术+全身化疗相比，可能会增加严重不良事件的比例（25.6% vs. 15.2%；危险比 1.69，95% 置信区间 1.03 至 2.77）。与单纯氟尿嘧啶类全身化疗相比，术中腹膜热化疗+细胞切除手术+全身化疗可能会降低全因死亡率（40.8% 对 60.8%；危险比 0.55，95% 置信区间 0.32 至 0.95）。对于胃癌，术中腹膜热化疗+细胞切除手术+全身化疗与细胞切除手术+全身化疗或单纯全身化疗对全因死亡率的影响存在很大的不确定性。对于接受间歇性细胞减灭术的 III 期或以上上皮性卵巢癌患者，与细胞减灭术+全身化疗相比，术中腹腔热化疗+细胞减灭术+全身化疗可能会降低全因死亡率（46.3% 对 57.4%；危险比 0.73，95% 置信区间 0.57 至 0.93）。在结直肠癌方面，术中腹膜热化疗+细胞切除手术+全身化疗与细胞切除手术+全身化疗相比可能不具成本效益，但在其余比较中可能具有成本效益：我们无法按计划获得个体参与者的数据。每项比较的随机对照试验数量有限，健康相关生活质量方面的数据较少，这意味着随着新证据（来自偏倚风险较低的试验）的出现，建议可能会发生变化：结论：对于腹膜转移有限且可能承受大手术的结直肠癌腹膜转移患者，常规临床实践中不应采用腹腔内热化疗+细胞减灭术+全身化疗（强烈建议）。对于胃癌腹膜转移患者是否应进行术中腹腔热化疗+细胞减灭术+全身化疗或细胞减灭术+全身化疗，还存在很大的不确定性（不推荐）。对于Ⅲ期或Ⅲ期以上上皮性卵巢癌、转移灶局限于腹部、化疗后需要并可能耐受间歇性细胞减灭术的女性患者，应常规进行术中腹膜热化疗+细胞减灭术+全身化疗（强烈建议）：研究注册：研究注册：本研究注册为PROSPERO CRD42019130504：该奖项由美国国家健康与护理研究所（NIHR）健康技术评估项目资助（NIHR奖项编号：17/135/02），全文发表于《健康技术评估》第28卷第51期。如需了解更多奖项信息，请参阅 NIHR Funding and Awards 网站。
{"title":"Hyperthermic intraoperative peritoneal chemotherapy and cytoreductive surgery for people with peritoneal metastases: a systematic review and cost-effectiveness analysis.","authors":"Kurinchi Gurusamy, Jeffrey Leung, Claire Vale, Danielle Roberts, Audrey Linden, Xiao Wei Tan, Priyal Taribagil, Sonam Patel, Elena Pizzo, Brian Davidson, Tim Mould, Mark Saunders, Omer Aziz, Sarah O'Dwyer","doi":"10.3310/KWDG6338","DOIUrl":"10.3310/KWDG6338","url":null,"abstract":"<p><strong>Background: </strong>We compared the relative benefits, harms and cost-effectiveness of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery ± systemic chemotherapy versus cytoreductive surgery ± systemic chemotherapy or systemic chemotherapy alone in people with peritoneal metastases from colorectal, gastric or ovarian cancers by a systematic review, meta-analysis and model-based cost-utility analysis.</p><p><strong>Methods: </strong>We searched MEDLINE, EMBASE, Cochrane Library and the Science Citation Index, ClinicalTrials.gov and WHO ICTRP trial registers until 14 April 2022. We included only randomised controlled trials addressing the research objectives. We used the Cochrane risk of bias tool version 2 to assess the risk of bias in randomised controlled trials. We used the random-effects model for data synthesis when applicable. For the cost-effectiveness analysis, we performed a model-based cost-utility analysis using methods recommended by The National Institute for Health and Care Excellence.</p><p><strong>Results: </strong>The systematic review included a total of eight randomised controlled trials (seven randomised controlled trials, 955 participants included in the quantitative analysis). All comparisons other than those for stage III or greater epithelial ovarian cancer contained only one trial, indicating the paucity of randomised controlled trials that provided data. For colorectal cancer, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably results in little to no difference in all-cause mortality (60.6% vs. 60.6%; hazard ratio 1.00, 95% confidence interval 0.63 to 1.58) and may increase the serious adverse event proportions compared to cytoreductive surgery ± systemic chemotherapy (25.6% vs. 15.2%; risk ratio 1.69, 95% confidence interval 1.03 to 2.77). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to fluorouracil-based systemic chemotherapy alone (40.8% vs. 60.8%; hazard ratio 0.55, 95% confidence interval 0.32 to 0.95). For gastric cancer, there is high uncertainty about the effects of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy versus cytoreductive surgery + systemic chemotherapy or systemic chemotherapy alone on all-cause mortality. For stage III or greater epithelial ovarian cancer undergoing interval cytoreductive surgery, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to cytoreductive surgery + systemic chemotherapy (46.3% vs. 57.4%; hazard ratio 0.73, 95% confidence interval 0.57 to 0.93). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy may not be cost-effective versus cytoreductive surgery + systemic chemotherapy for colorectal","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 51","pages":"1-139"},"PeriodicalIF":3.5,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11417642/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285845","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}
Zoe Marshman, Hannah Ainsworth, Caroline Fairhurst, Katie Whiteside, Debbie Sykes, Anju Keetharuth, Sarab El Yousfi, Emma Turner, Peter F Day, Ivor G Chestnutt, Simon Dixon, Ian Kellar, Fiona Gilchrist, Mark Robertson, Sue Pavitt, Catherine Hewitt, Donna Dey, David Torgerson, Lesley Pollard, Emma Manser, Nassar Seifo, Mariana Araujo, Waraf Al-Yaseen, Claire Jones, Kate Hicks, Kathryn Rowles, Nicola Innes
<p><strong>Background: </strong>The presence of dental caries impacts on children's daily lives, particularly among those living in deprived areas. There are successful interventions across the United Kingdom for young children based on toothbrushing with fluoride toothpaste. However, evidence is lacking for oral health improvement programmes in secondary-school pupils to reduce dental caries and its sequelae.</p><p><strong>Objectives: </strong>To determine the clinical and cost effectiveness of a behaviour change intervention promoting toothbrushing for preventing dental caries in secondary-school pupils.</p><p><strong>Design: </strong>A multicentre, school-based, assessor-blinded, two-arm cluster randomised controlled trial with an internal pilot and embedded health economic and process evaluations.</p><p><strong>Setting: </strong>Secondary schools in Scotland, England and Wales with above-average proportion of pupils eligible for free school meals. Randomisation occurred within schools (year-group level), using block randomisation stratified by school.</p><p><strong>Participants: </strong>Pupils aged 11-13 years at recruitment, who have their own mobile telephone.</p><p><strong>Interventions: </strong>Two-component intervention based on behaviour change theory: (1) 50-minute lesson delivered by teachers, and (2) twice-daily text messages to pupils' mobile phones about toothbrushing, compared with routine education.</p><p><strong>Main outcome measures: </strong>Primary outcome: presence of at least one treated or untreated carious lesion using D<sub>ICDAS4-6</sub>MFT (Decayed, Missing and Filled Teeth) in any permanent tooth, measured at pupil level at 2.5 years. Secondary outcomes included: number of D<sub>ICDAS4-6</sub>MFT; presence and number of D<sub>ICDAS1-6</sub>MFT; plaque; bleeding; twice-daily toothbrushing; health-related quality of life (Child Health Utility 9D); and oral health-related quality of life (Caries Impacts and Experiences Questionnaire for Children).</p><p><strong>Results: </strong>Four thousand six hundred and eighty pupils (intervention, <i>n</i> = 2262; control, <i>n</i> = 2418) from 42 schools were randomised. The primary analysis on 2383 pupils (50.9%; intervention 1153, 51.0%; control 1230, 50.9%) with valid data at baseline and 2.5 years found 44.6% in the intervention group and 43.0% in control had obvious decay experience in at least one permanent tooth. There was no evidence of a difference (odds ratio 1.04, 95% confidence interval 0.85 to 1.26, <i>p</i> = 0.72) and no statistically significant differences in secondary outcomes except for twice-daily toothbrushing at 6 months (odds ratio 1.30, 95% confidence interval 1.03 to 1.63, <i>p</i> = 0.03) and gingival bleeding score (borderline) at 2.5 years (geometric mean difference 0.92, 95% confidence interval 0.85 to 1.00, <i>p</i> = 0.05). The intervention had higher incremental mean costs (£1.02, 95% confidence interval -1.29 to 3.23) and lower incremental mean q
{"title":"Behaviour change intervention (education and text) to prevent dental caries in secondary school pupils: BRIGHT RCT, process and economic evaluation.","authors":"Zoe Marshman, Hannah Ainsworth, Caroline Fairhurst, Katie Whiteside, Debbie Sykes, Anju Keetharuth, Sarab El Yousfi, Emma Turner, Peter F Day, Ivor G Chestnutt, Simon Dixon, Ian Kellar, Fiona Gilchrist, Mark Robertson, Sue Pavitt, Catherine Hewitt, Donna Dey, David Torgerson, Lesley Pollard, Emma Manser, Nassar Seifo, Mariana Araujo, Waraf Al-Yaseen, Claire Jones, Kate Hicks, Kathryn Rowles, Nicola Innes","doi":"10.3310/JQTA2103","DOIUrl":"10.3310/JQTA2103","url":null,"abstract":"<p><strong>Background: </strong>The presence of dental caries impacts on children's daily lives, particularly among those living in deprived areas. There are successful interventions across the United Kingdom for young children based on toothbrushing with fluoride toothpaste. However, evidence is lacking for oral health improvement programmes in secondary-school pupils to reduce dental caries and its sequelae.</p><p><strong>Objectives: </strong>To determine the clinical and cost effectiveness of a behaviour change intervention promoting toothbrushing for preventing dental caries in secondary-school pupils.</p><p><strong>Design: </strong>A multicentre, school-based, assessor-blinded, two-arm cluster randomised controlled trial with an internal pilot and embedded health economic and process evaluations.</p><p><strong>Setting: </strong>Secondary schools in Scotland, England and Wales with above-average proportion of pupils eligible for free school meals. Randomisation occurred within schools (year-group level), using block randomisation stratified by school.</p><p><strong>Participants: </strong>Pupils aged 11-13 years at recruitment, who have their own mobile telephone.</p><p><strong>Interventions: </strong>Two-component intervention based on behaviour change theory: (1) 50-minute lesson delivered by teachers, and (2) twice-daily text messages to pupils' mobile phones about toothbrushing, compared with routine education.</p><p><strong>Main outcome measures: </strong>Primary outcome: presence of at least one treated or untreated carious lesion using D<sub>ICDAS4-6</sub>MFT (Decayed, Missing and Filled Teeth) in any permanent tooth, measured at pupil level at 2.5 years. Secondary outcomes included: number of D<sub>ICDAS4-6</sub>MFT; presence and number of D<sub>ICDAS1-6</sub>MFT; plaque; bleeding; twice-daily toothbrushing; health-related quality of life (Child Health Utility 9D); and oral health-related quality of life (Caries Impacts and Experiences Questionnaire for Children).</p><p><strong>Results: </strong>Four thousand six hundred and eighty pupils (intervention, <i>n</i> = 2262; control, <i>n</i> = 2418) from 42 schools were randomised. The primary analysis on 2383 pupils (50.9%; intervention 1153, 51.0%; control 1230, 50.9%) with valid data at baseline and 2.5 years found 44.6% in the intervention group and 43.0% in control had obvious decay experience in at least one permanent tooth. There was no evidence of a difference (odds ratio 1.04, 95% confidence interval 0.85 to 1.26, <i>p</i> = 0.72) and no statistically significant differences in secondary outcomes except for twice-daily toothbrushing at 6 months (odds ratio 1.30, 95% confidence interval 1.03 to 1.63, <i>p</i> = 0.03) and gingival bleeding score (borderline) at 2.5 years (geometric mean difference 0.92, 95% confidence interval 0.85 to 1.00, <i>p</i> = 0.05). The intervention had higher incremental mean costs (£1.02, 95% confidence interval -1.29 to 3.23) and lower incremental mean q","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 52","pages":"1-142"},"PeriodicalIF":3.5,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11417644/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285846","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}
Garry Alan Tew, Laura Wiley, Lesley Ward, Jessica Grace Hugill-Jones, Camila Sofia Maturana, Caroline Marie Fairhurst, Kerry Jane Bell, Laura Bissell, Alison Booth, Jenny Howsam, Valerie Mount, Tim Rapley, Sarah Jane Ronaldson, Fiona Rose, David John Torgerson, David Yates, Catherine Elizabeth Hewitt
Joe Carroll, Catalina Lopez Manzano, Eve Tomlinson, Ayman Sadek, Chris Cooper, Hayley E Jones, Lorraine Rowsell, John Knight, Andrew Mumford, Rachel Palmer, William Hollingworth, Nicky J Welton, Penny Whiting
<p><strong>Background: </strong>Stroke or transient ischaemic attack patients are at increased risk of secondary vascular events. Antiplatelet medications, most commonly clopidogrel, are prescribed to reduce this risk. Factors including <i>CYP2C19</i> genetic variants can hinder clopidogrel metabolism. Laboratory-based or point-of-care tests can detect these variants, enabling targeted treatment.</p><p><strong>Objective: </strong>To assess the effectiveness of genetic testing to identify clopidogrel resistance in people with ischaemic stroke or transient ischaemic attack. Specific objectives: Do people tested for clopidogrel resistance, and treated accordingly, have a reduced risk of secondary vascular events? Do people with loss-of-function alleles associated with clopidogrel resistance have a reduced risk of secondary vascular events if treated with alternative interventions compared to clopidogrel? Do people with loss-of-function alleles associated with clopidogrel resistance have an increased risk of secondary vascular events when treated with clopidogrel? What is the accuracy of point-of-care tests for detecting variants associated with clopidogrel resistance? What is the technical performance and cost of <i>CYP2C19</i> genetic tests? Is genetic testing for clopidogrel resistance cost-effective compared with no testing?</p><p><strong>Design: </strong>Systematic review and economic model.</p><p><strong>Results: </strong>Objective 1: Two studies assessed secondary vascular events in patients tested for loss-of-function alleles and treated accordingly. They found a reduced risk, but confidence intervals were wide (hazard ratio 0.50, 95% confidence interval 0.09 to 2.74 and hazard ratio 0.53, 95% confidence interval 0.24 to 1.18). Objective 2: Seven randomised controlled trials compared clopidogrel with alternative treatment in people with genetic variants. Ticagrelor was associated with a lower risk of secondary vascular events than clopidogrel (summary hazard ratio 0.76, 95% confidence interval 0.65 to 0.90; two studies). Objective 3: Twenty-five studies compared outcomes in people with and without genetic variants treated with clopidogrel. People with genetic variants were at an increased risk of secondary vascular events (hazard ratio 1.72, 95% confidence interval 1.43 to 2.08; 18 studies). There was no difference in bleeding risk (hazard ratio 0.98, 95% confidence interval 0.68 to 1.40; five studies). Objective 4: Eleven studies evaluated Genomadix Cube accuracy; no studies evaluated Genedrive. Summary sensitivity and specificity against laboratory reference standards were both 100% (95% confidence interval 94% to 100% and 99% to 100%). Objective 5: Seventeen studies evaluated technical performance of point-of-care tests. Test failure rate ranged from 0.4% to 19% for Genomadix Cube. A survey of 8/10 genomic laboratory hubs revealed variation in preferred technologies for testing, and cost per test ranging from £15 to £250. Most laboratories
{"title":"Clinical and cost-effectiveness of clopidogrel resistance genotype testing after ischaemic stroke or transient ischaemic attack: a systematic review and economic model.","authors":"Joe Carroll, Catalina Lopez Manzano, Eve Tomlinson, Ayman Sadek, Chris Cooper, Hayley E Jones, Lorraine Rowsell, John Knight, Andrew Mumford, Rachel Palmer, William Hollingworth, Nicky J Welton, Penny Whiting","doi":"10.3310/PWCB4016","DOIUrl":"10.3310/PWCB4016","url":null,"abstract":"<p><strong>Background: </strong>Stroke or transient ischaemic attack patients are at increased risk of secondary vascular events. Antiplatelet medications, most commonly clopidogrel, are prescribed to reduce this risk. Factors including <i>CYP2C19</i> genetic variants can hinder clopidogrel metabolism. Laboratory-based or point-of-care tests can detect these variants, enabling targeted treatment.</p><p><strong>Objective: </strong>To assess the effectiveness of genetic testing to identify clopidogrel resistance in people with ischaemic stroke or transient ischaemic attack. Specific objectives: Do people tested for clopidogrel resistance, and treated accordingly, have a reduced risk of secondary vascular events? Do people with loss-of-function alleles associated with clopidogrel resistance have a reduced risk of secondary vascular events if treated with alternative interventions compared to clopidogrel? Do people with loss-of-function alleles associated with clopidogrel resistance have an increased risk of secondary vascular events when treated with clopidogrel? What is the accuracy of point-of-care tests for detecting variants associated with clopidogrel resistance? What is the technical performance and cost of <i>CYP2C19</i> genetic tests? Is genetic testing for clopidogrel resistance cost-effective compared with no testing?</p><p><strong>Design: </strong>Systematic review and economic model.</p><p><strong>Results: </strong>Objective 1: Two studies assessed secondary vascular events in patients tested for loss-of-function alleles and treated accordingly. They found a reduced risk, but confidence intervals were wide (hazard ratio 0.50, 95% confidence interval 0.09 to 2.74 and hazard ratio 0.53, 95% confidence interval 0.24 to 1.18). Objective 2: Seven randomised controlled trials compared clopidogrel with alternative treatment in people with genetic variants. Ticagrelor was associated with a lower risk of secondary vascular events than clopidogrel (summary hazard ratio 0.76, 95% confidence interval 0.65 to 0.90; two studies). Objective 3: Twenty-five studies compared outcomes in people with and without genetic variants treated with clopidogrel. People with genetic variants were at an increased risk of secondary vascular events (hazard ratio 1.72, 95% confidence interval 1.43 to 2.08; 18 studies). There was no difference in bleeding risk (hazard ratio 0.98, 95% confidence interval 0.68 to 1.40; five studies). Objective 4: Eleven studies evaluated Genomadix Cube accuracy; no studies evaluated Genedrive. Summary sensitivity and specificity against laboratory reference standards were both 100% (95% confidence interval 94% to 100% and 99% to 100%). Objective 5: Seventeen studies evaluated technical performance of point-of-care tests. Test failure rate ranged from 0.4% to 19% for Genomadix Cube. A survey of 8/10 genomic laboratory hubs revealed variation in preferred technologies for testing, and cost per test ranging from £15 to £250. Most laboratories","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 57","pages":"1-194"},"PeriodicalIF":3.5,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11417645/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285851","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}
Jan O Jansen, Jemma Hudson, Charlotte Kennedy, Claire Cochran, Graeme MacLennan, Katie Gillies, Robbie Lendrum, Samy Sadek, Dwayne Boyers, Gillian Ferry, Louisa Lawrie, Mintu Nath, Seonaidh Cotton, Samantha Wileman, Mark Forrest, Karim Brohi, Tim Harris, Fiona Lecky, Chris Moran, Jonathan J Morrison, John Norrie, Alan Paterson, Nigel Tai, Nick Welch, Marion K Campbell
<p><strong>Background: </strong>The most common cause of preventable death after injury is haemorrhage. Resuscitative endovascular balloon occlusion of the aorta is intended to provide earlier, temporary haemorrhage control, to facilitate transfer to an operating theatre or interventional radiology suite for definitive haemostasis.</p><p><strong>Objective: </strong>To compare standard care plus resuscitative endovascular balloon occlusion of the aorta versus standard care in patients with exsanguinating haemorrhage in the emergency department.</p><p><strong>Design: </strong>Pragmatic, multicentre, Bayesian, group-sequential, registry-enabled, open-label, parallel-group randomised controlled trial to determine the clinical and cost-effectiveness of standard care plus resuscitative endovascular balloon occlusion of the aorta, compared to standard care alone.</p><p><strong>Setting: </strong>United Kingdom Major Trauma Centres.</p><p><strong>Participants: </strong>Trauma patients aged 16 years or older with confirmed or suspected life-threatening torso haemorrhage deemed amenable to adjunctive treatment with resuscitative endovascular balloon occlusion of the aorta.</p><p><strong>Interventions: </strong>Participants were randomly assigned 1 : 1 to: standard care, as expected in a major trauma centre standard care plus resuscitative endovascular balloon occlusion of the aorta.</p><p><strong>Main outcome measures: </strong><i>Primary:</i> Mortality at 90 days. <i>Secondary:</i> Mortality at 6 months, while in hospital, and within 24, 6 and 3 hours; need for haemorrhage control procedures, time to commencement of haemorrhage procedure, complications, length of stay (hospital and intensive care unit-free days), blood product use. <i>Health economic:</i> Expected United Kingdom National Health Service perspective costs, life-years and quality-adjusted life-years, modelled over a lifetime horizon.</p><p><strong>Data sources: </strong>Case report forms, Trauma Audit and Research Network registry, NHS Digital (Hospital Episode Statistics and Office of National Statistics data).</p><p><strong>Results: </strong>Ninety patients were enrolled: 46 were randomised to standard care plus resuscitative endovascular balloon occlusion of the aorta and 44 to standard care. Mortality at 90 days was higher in the standard care plus resuscitative endovascular balloon occlusion of the aorta group (54%) compared to the standard care group (42%). The odds ratio was 1.58 (95% credible interval 0.72 to 3.52). The posterior probability of an odds ratio > 1 (indicating increased odds of death with resuscitative endovascular balloon occlusion of the aorta) was 86.9%. The overall effect did not change when an enthusiastic prior was used or when the estimate was adjusted for baseline characteristics. For the secondary outcomes (3, 6 and 24 hours mortality), the posterior probability that standard care plus resuscitative endovascular balloon occlusion of the aorta was harmful was hig
{"title":"The UK resuscitative endovascular balloon occlusion of the aorta in trauma patients with life-threatening torso haemorrhage: the (UK-REBOA) multicentre RCT.","authors":"Jan O Jansen, Jemma Hudson, Charlotte Kennedy, Claire Cochran, Graeme MacLennan, Katie Gillies, Robbie Lendrum, Samy Sadek, Dwayne Boyers, Gillian Ferry, Louisa Lawrie, Mintu Nath, Seonaidh Cotton, Samantha Wileman, Mark Forrest, Karim Brohi, Tim Harris, Fiona Lecky, Chris Moran, Jonathan J Morrison, John Norrie, Alan Paterson, Nigel Tai, Nick Welch, Marion K Campbell","doi":"10.3310/LTYV4082","DOIUrl":"10.3310/LTYV4082","url":null,"abstract":"<p><strong>Background: </strong>The most common cause of preventable death after injury is haemorrhage. Resuscitative endovascular balloon occlusion of the aorta is intended to provide earlier, temporary haemorrhage control, to facilitate transfer to an operating theatre or interventional radiology suite for definitive haemostasis.</p><p><strong>Objective: </strong>To compare standard care plus resuscitative endovascular balloon occlusion of the aorta versus standard care in patients with exsanguinating haemorrhage in the emergency department.</p><p><strong>Design: </strong>Pragmatic, multicentre, Bayesian, group-sequential, registry-enabled, open-label, parallel-group randomised controlled trial to determine the clinical and cost-effectiveness of standard care plus resuscitative endovascular balloon occlusion of the aorta, compared to standard care alone.</p><p><strong>Setting: </strong>United Kingdom Major Trauma Centres.</p><p><strong>Participants: </strong>Trauma patients aged 16 years or older with confirmed or suspected life-threatening torso haemorrhage deemed amenable to adjunctive treatment with resuscitative endovascular balloon occlusion of the aorta.</p><p><strong>Interventions: </strong>Participants were randomly assigned 1 : 1 to: standard care, as expected in a major trauma centre standard care plus resuscitative endovascular balloon occlusion of the aorta.</p><p><strong>Main outcome measures: </strong><i>Primary:</i> Mortality at 90 days. <i>Secondary:</i> Mortality at 6 months, while in hospital, and within 24, 6 and 3 hours; need for haemorrhage control procedures, time to commencement of haemorrhage procedure, complications, length of stay (hospital and intensive care unit-free days), blood product use. <i>Health economic:</i> Expected United Kingdom National Health Service perspective costs, life-years and quality-adjusted life-years, modelled over a lifetime horizon.</p><p><strong>Data sources: </strong>Case report forms, Trauma Audit and Research Network registry, NHS Digital (Hospital Episode Statistics and Office of National Statistics data).</p><p><strong>Results: </strong>Ninety patients were enrolled: 46 were randomised to standard care plus resuscitative endovascular balloon occlusion of the aorta and 44 to standard care. Mortality at 90 days was higher in the standard care plus resuscitative endovascular balloon occlusion of the aorta group (54%) compared to the standard care group (42%). The odds ratio was 1.58 (95% credible interval 0.72 to 3.52). The posterior probability of an odds ratio > 1 (indicating increased odds of death with resuscitative endovascular balloon occlusion of the aorta) was 86.9%. The overall effect did not change when an enthusiastic prior was used or when the estimate was adjusted for baseline characteristics. For the secondary outcomes (3, 6 and 24 hours mortality), the posterior probability that standard care plus resuscitative endovascular balloon occlusion of the aorta was harmful was hig","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 54","pages":"1-122"},"PeriodicalIF":3.5,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11418015/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285848","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}
Tristan M Snowsill, Helen Coelho, Nia G Morrish, Simon Briscoe, Kate Boddy, Tracy Smith, Emma J Crosbie, Neil Aj Ryan, Fiona Lalloo, Claire T Hulme
<p><strong>Background: </strong>Lynch syndrome is an inherited condition which leads to an increased risk of colorectal, endometrial and ovarian cancer. Risk-reducing surgery is generally recommended to manage the risk of gynaecological cancer once childbearing is completed. The value of gynaecological colonoscopic surveillance as an interim measure or instead of risk-reducing surgery is uncertain. We aimed to determine whether gynaecological surveillance was effective and cost-effective in Lynch syndrome.</p><p><strong>Methods: </strong>We conducted systematic reviews of the effectiveness and cost-effectiveness of gynaecological cancer surveillance in Lynch syndrome, as well as a systematic review of health utility values relating to cancer and gynaecological risk reduction. Study identification included bibliographic database searching and citation chasing (searches updated 3 August 2021). Screening and assessment of eligibility for inclusion were conducted by independent researchers. Outcomes were prespecified and were informed by clinical experts and patient involvement. Data extraction and quality appraisal were conducted and results were synthesised narratively. We also developed a whole-disease economic model for Lynch syndrome using discrete event simulation methodology, including natural history components for colorectal, endometrial and ovarian cancer, and we used this model to conduct a cost-utility analysis of gynaecological risk management strategies, including surveillance, risk-reducing surgery and doing nothing.</p><p><strong>Results: </strong>We found 30 studies in the review of clinical effectiveness, of which 20 were non-comparative (single-arm) studies. There were no high-quality studies providing precise outcome estimates at low risk of bias. There is some evidence that mortality rate is higher for surveillance than for risk-reducing surgery but mortality is also higher for no surveillance than for surveillance. Some asymptomatic cancers were detected through surveillance but some cancers were also missed. There was a wide range of pain experiences, including some individuals feeling no pain and some feeling severe pain. The use of pain relief (e.g. ibuprofen) was common, and some women underwent general anaesthetic for surveillance. Existing economic evaluations clearly found that risk-reducing surgery leads to the best lifetime health (measured using quality-adjusted life-years) and is cost-effective, while surveillance is not cost-effective in comparison. Our economic evaluation found that a strategy of surveillance alone or offering surveillance and risk-reducing surgery was cost-effective, except for <i>path_PMS2</i> Lynch syndrome. Offering only risk-reducing surgery was less effective than offering surveillance with or without surgery.</p><p><strong>Limitations: </strong>Firm conclusions about clinical effectiveness could not be reached because of the lack of high-quality research. We did not assume that women would im
{"title":"Gynaecological cancer surveillance for women with Lynch syndrome: systematic review and cost-effectiveness evaluation.","authors":"Tristan M Snowsill, Helen Coelho, Nia G Morrish, Simon Briscoe, Kate Boddy, Tracy Smith, Emma J Crosbie, Neil Aj Ryan, Fiona Lalloo, Claire T Hulme","doi":"10.3310/VBXX6307","DOIUrl":"10.3310/VBXX6307","url":null,"abstract":"<p><strong>Background: </strong>Lynch syndrome is an inherited condition which leads to an increased risk of colorectal, endometrial and ovarian cancer. Risk-reducing surgery is generally recommended to manage the risk of gynaecological cancer once childbearing is completed. The value of gynaecological colonoscopic surveillance as an interim measure or instead of risk-reducing surgery is uncertain. We aimed to determine whether gynaecological surveillance was effective and cost-effective in Lynch syndrome.</p><p><strong>Methods: </strong>We conducted systematic reviews of the effectiveness and cost-effectiveness of gynaecological cancer surveillance in Lynch syndrome, as well as a systematic review of health utility values relating to cancer and gynaecological risk reduction. Study identification included bibliographic database searching and citation chasing (searches updated 3 August 2021). Screening and assessment of eligibility for inclusion were conducted by independent researchers. Outcomes were prespecified and were informed by clinical experts and patient involvement. Data extraction and quality appraisal were conducted and results were synthesised narratively. We also developed a whole-disease economic model for Lynch syndrome using discrete event simulation methodology, including natural history components for colorectal, endometrial and ovarian cancer, and we used this model to conduct a cost-utility analysis of gynaecological risk management strategies, including surveillance, risk-reducing surgery and doing nothing.</p><p><strong>Results: </strong>We found 30 studies in the review of clinical effectiveness, of which 20 were non-comparative (single-arm) studies. There were no high-quality studies providing precise outcome estimates at low risk of bias. There is some evidence that mortality rate is higher for surveillance than for risk-reducing surgery but mortality is also higher for no surveillance than for surveillance. Some asymptomatic cancers were detected through surveillance but some cancers were also missed. There was a wide range of pain experiences, including some individuals feeling no pain and some feeling severe pain. The use of pain relief (e.g. ibuprofen) was common, and some women underwent general anaesthetic for surveillance. Existing economic evaluations clearly found that risk-reducing surgery leads to the best lifetime health (measured using quality-adjusted life-years) and is cost-effective, while surveillance is not cost-effective in comparison. Our economic evaluation found that a strategy of surveillance alone or offering surveillance and risk-reducing surgery was cost-effective, except for <i>path_PMS2</i> Lynch syndrome. Offering only risk-reducing surgery was less effective than offering surveillance with or without surgery.</p><p><strong>Limitations: </strong>Firm conclusions about clinical effectiveness could not be reached because of the lack of high-quality research. We did not assume that women would im","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"28 41","pages":"1-228"},"PeriodicalIF":3.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11403379/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142153872","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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