T Justin Clark, Lina Antoun, Rebecca Woolley, Sheriden Bevan, Kamila Ziomek, William McKinnon, Paul Smith, Kevin Cooper, Ertan Saridogan, Bibi Zeyah Sairally, Jayne Fullard, Monique Morgan, Lynsay Matthews, Laura Jones, Tracy Roberts, Lee Middleton
<p><strong>Background: </strong>The comparative rates of major complications and recovery times between laparoscopic hysterectomy and abdominal hysterectomy for benign gynaecological conditions remain uncertain.</p><p><strong>Objective(s): </strong>To assess the clinical and cost-effectiveness of laparoscopic hysterectomy compared to abdominal hysterectomy in women with benign gynaecological conditions.</p><p><strong>Design and methods: </strong>A parallel, open, non-inferiority, multicentre, randomised controlled, expertise-based surgery trial with integrated health economic evaluation and an internal pilot with an embedded qualitative process evaluation, and a post-closure survey after recruitment ended.</p><p><strong>Setting and participants: </strong>Women in secondary care requiring hysterectomy and eligible for either surgical method.</p><p><strong>Interventions: </strong>Laparoscopic hysterectomy versus abdominal hysterectomy.</p><p><strong>Main outcome measures: </strong>The primary outcome was major complications (Clavien-Dindo ≥ level III) up to 6 completed weeks post surgery, and the key secondary outcome was time from surgery to resumption of usual activities using the personalised Patient-Reported Outcomes Measurement Information System Physical Function questionnaire. The principal outcome for the economic evaluation was to be cost per quality-adjusted life-year at 12 months post surgery and was feasibility and acceptability for the qualitative process evaluation.</p><p><strong>Results: </strong>Two hundred and fifty-two patients were screened from 13 open sites over 13 months, 156 (62%) were eligible and 75 (49%) randomised. Of the 53 women not randomised, 23 (43%) preferred laparoscopic hysterectomy and 6 (11%) abdominal hysterectomy. About 32/39 (82%) and 30/36 (83%) participants randomised to laparoscopic hysterectomy and abdominal hysterectomy, respectively, had their surgery, of which 31/32 (97%) and 25/30 (83%) received their allocated route of hysterectomy. Major complications occurred in 2/32 (6%) laparoscopic hysterectomy versus 4/30 (13%) abdominal hysterectomy groups. There was no difference in time to resumption of activities [median (interquartile range, <i>N</i>) 7.5 weeks (3.6-8.2, 25) laparoscopic hysterectomy vs. 7.5 weeks (5.5-10.6, 26) abdominal hysterectomy groups] or quality of recovery [mean (standard deviation, <i>N</i>) 81.1 (13.4, 27) vs. 72.3 (17.6, 22) respectively; adjusted mean difference 7.2, 95% confidence interval -3.2 to 17.6]. The qualitative evaluation found that the trial was viewed positively by women and healthcare professionals. The reasons for failure to recruit from 21 sites open or in set-up were lack of research/clinical capacity imposed by the COVID-19 pandemic (14, 67%) and lack of clinician equipoise (11, 52%).</p><p><strong>Limitations: </strong>The main limitation was failure to recruit, resulting in a final sample of 75 patients from a target of 3250. At the time of analysis, 13 (17%
{"title":"Laparoscopic hysterectomy versus open abdominal hysterectomy for women with a benign gynaecological condition: the LAVA RCT.","authors":"T Justin Clark, Lina Antoun, Rebecca Woolley, Sheriden Bevan, Kamila Ziomek, William McKinnon, Paul Smith, Kevin Cooper, Ertan Saridogan, Bibi Zeyah Sairally, Jayne Fullard, Monique Morgan, Lynsay Matthews, Laura Jones, Tracy Roberts, Lee Middleton","doi":"10.3310/GJTC1718","DOIUrl":"10.3310/GJTC1718","url":null,"abstract":"<p><strong>Background: </strong>The comparative rates of major complications and recovery times between laparoscopic hysterectomy and abdominal hysterectomy for benign gynaecological conditions remain uncertain.</p><p><strong>Objective(s): </strong>To assess the clinical and cost-effectiveness of laparoscopic hysterectomy compared to abdominal hysterectomy in women with benign gynaecological conditions.</p><p><strong>Design and methods: </strong>A parallel, open, non-inferiority, multicentre, randomised controlled, expertise-based surgery trial with integrated health economic evaluation and an internal pilot with an embedded qualitative process evaluation, and a post-closure survey after recruitment ended.</p><p><strong>Setting and participants: </strong>Women in secondary care requiring hysterectomy and eligible for either surgical method.</p><p><strong>Interventions: </strong>Laparoscopic hysterectomy versus abdominal hysterectomy.</p><p><strong>Main outcome measures: </strong>The primary outcome was major complications (Clavien-Dindo ≥ level III) up to 6 completed weeks post surgery, and the key secondary outcome was time from surgery to resumption of usual activities using the personalised Patient-Reported Outcomes Measurement Information System Physical Function questionnaire. The principal outcome for the economic evaluation was to be cost per quality-adjusted life-year at 12 months post surgery and was feasibility and acceptability for the qualitative process evaluation.</p><p><strong>Results: </strong>Two hundred and fifty-two patients were screened from 13 open sites over 13 months, 156 (62%) were eligible and 75 (49%) randomised. Of the 53 women not randomised, 23 (43%) preferred laparoscopic hysterectomy and 6 (11%) abdominal hysterectomy. About 32/39 (82%) and 30/36 (83%) participants randomised to laparoscopic hysterectomy and abdominal hysterectomy, respectively, had their surgery, of which 31/32 (97%) and 25/30 (83%) received their allocated route of hysterectomy. Major complications occurred in 2/32 (6%) laparoscopic hysterectomy versus 4/30 (13%) abdominal hysterectomy groups. There was no difference in time to resumption of activities [median (interquartile range, <i>N</i>) 7.5 weeks (3.6-8.2, 25) laparoscopic hysterectomy vs. 7.5 weeks (5.5-10.6, 26) abdominal hysterectomy groups] or quality of recovery [mean (standard deviation, <i>N</i>) 81.1 (13.4, 27) vs. 72.3 (17.6, 22) respectively; adjusted mean difference 7.2, 95% confidence interval -3.2 to 17.6]. The qualitative evaluation found that the trial was viewed positively by women and healthcare professionals. The reasons for failure to recruit from 21 sites open or in set-up were lack of research/clinical capacity imposed by the COVID-19 pandemic (14, 67%) and lack of clinician equipoise (11, 52%).</p><p><strong>Limitations: </strong>The main limitation was failure to recruit, resulting in a final sample of 75 patients from a target of 3250. At the time of analysis, 13 (17%","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 14","pages":"1-27"},"PeriodicalIF":4.0,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12914755/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146149682","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}
Hugh Paterson, Thenmalar Vadiveloo, Karen Innes, Angie Balfour, Marek Atter, Andrew Stoddart, Seonaidh Cotton, Robert Arnott, Lorna Aucott, Zoe Batham, Irwin Foo, Graeme MacLennan, Susan Nimmo, Doug Speake, John Norrie
<p><strong>Background: </strong>Delayed return of gut function after colonic resection is a common impediment to early postoperative recovery. Small clinical studies, combined into meta-analyses, have suggested that intravenous lidocaine can improve return of gut function after colorectal surgery.</p><p><strong>Objective(s): </strong>To determine the clinical effectiveness and cost-effectiveness of perioperative intravenous lidocaine infusion compared with placebo in return of gut function after elective minimally invasive colonic resection.</p><p><strong>Design and methods: </strong>Multicentre, pragmatic, placebo-controlled, randomised trial with cost-effectiveness analysis.</p><p><strong>Setting and participants: </strong>Twenty-seven hospital sites across the United Kingdom. Adult patients scheduled for elective minimally invasive colon resection were randomised in 1 : 1 ratio to treatment or control groups using a web-based portal, stratified by age, sex and trial site.</p><p><strong>Interventions: </strong>A sterile solution of 2% lidocaine (made isotonic with sodium chloride) and matching placebo (a sterile solution of 0.9% sodium chloride). Participants received an intravenous bolus of study drug/placebo at induction of anaesthesia (1.5 mg/kg ideal body weight) given over 20 minutes, followed by intravenous infusion of 1.5 mg/hour/kg ideal body weight with a maximum rate of 120 mg/hour (6 ml/hour) for a minimum of 6 hours up to a maximum of 12 hours. The planned duration of infusion was determined by the participating unit's availability of postoperative continuous cardiac monitoring.</p><p><strong>Main outcome measures: </strong>Primary outcome: return of gut function at 72 hours postoperatively measured by 'GI-3 recovery' (defined as tolerating diet and passage of flatus or stool). Other outcomes were GI-2 recovery, prolonged postoperative ileus, patient-reported measures of quality of life, recovery and pain, 30- and 90-day mortality, unplanned re-admissions, adverse events, serious adverse events and cost per quality-adjusted life-year at 30 days. Participants, care givers and those assessing the outcomes were blinded to group assignment.</p><p><strong>Results: </strong>The trial enrolled 590 patients (295 interventions, 295 control); after 33 post-randomisation exclusions, 557 patients were included (279 interventions, 278 control). There was no statistically significant or clinically meaningful difference in GI-3 recovery at 72 hours after surgery [160/279 patients (57.3%) for intravenous lidocaine versus 164/278 patients (59%) for placebo (absolute difference 1.9% (-8.0 to 4.2), odds ratio 0.97 (0.88 to 1.07), <i>p</i> = 0.54)]. There was no effect of intravenous lidocaine found in predetermined subgroup analyses (6- vs. 12-hour duration of infusion, right vs. non-right colectomy, sex, age band and enhanced recovery after surgery compliance). There was no evidence of a difference in other measures of gut function return, pain, qual
{"title":"Intravenous lidocaine for gastrointestinal recovery after colorectal surgery: the ALLEGRO placebo-controlled randomised trial and cost-effectiveness analysis.","authors":"Hugh Paterson, Thenmalar Vadiveloo, Karen Innes, Angie Balfour, Marek Atter, Andrew Stoddart, Seonaidh Cotton, Robert Arnott, Lorna Aucott, Zoe Batham, Irwin Foo, Graeme MacLennan, Susan Nimmo, Doug Speake, John Norrie","doi":"10.3310/GJHP2321","DOIUrl":"10.3310/GJHP2321","url":null,"abstract":"<p><strong>Background: </strong>Delayed return of gut function after colonic resection is a common impediment to early postoperative recovery. Small clinical studies, combined into meta-analyses, have suggested that intravenous lidocaine can improve return of gut function after colorectal surgery.</p><p><strong>Objective(s): </strong>To determine the clinical effectiveness and cost-effectiveness of perioperative intravenous lidocaine infusion compared with placebo in return of gut function after elective minimally invasive colonic resection.</p><p><strong>Design and methods: </strong>Multicentre, pragmatic, placebo-controlled, randomised trial with cost-effectiveness analysis.</p><p><strong>Setting and participants: </strong>Twenty-seven hospital sites across the United Kingdom. Adult patients scheduled for elective minimally invasive colon resection were randomised in 1 : 1 ratio to treatment or control groups using a web-based portal, stratified by age, sex and trial site.</p><p><strong>Interventions: </strong>A sterile solution of 2% lidocaine (made isotonic with sodium chloride) and matching placebo (a sterile solution of 0.9% sodium chloride). Participants received an intravenous bolus of study drug/placebo at induction of anaesthesia (1.5 mg/kg ideal body weight) given over 20 minutes, followed by intravenous infusion of 1.5 mg/hour/kg ideal body weight with a maximum rate of 120 mg/hour (6 ml/hour) for a minimum of 6 hours up to a maximum of 12 hours. The planned duration of infusion was determined by the participating unit's availability of postoperative continuous cardiac monitoring.</p><p><strong>Main outcome measures: </strong>Primary outcome: return of gut function at 72 hours postoperatively measured by 'GI-3 recovery' (defined as tolerating diet and passage of flatus or stool). Other outcomes were GI-2 recovery, prolonged postoperative ileus, patient-reported measures of quality of life, recovery and pain, 30- and 90-day mortality, unplanned re-admissions, adverse events, serious adverse events and cost per quality-adjusted life-year at 30 days. Participants, care givers and those assessing the outcomes were blinded to group assignment.</p><p><strong>Results: </strong>The trial enrolled 590 patients (295 interventions, 295 control); after 33 post-randomisation exclusions, 557 patients were included (279 interventions, 278 control). There was no statistically significant or clinically meaningful difference in GI-3 recovery at 72 hours after surgery [160/279 patients (57.3%) for intravenous lidocaine versus 164/278 patients (59%) for placebo (absolute difference 1.9% (-8.0 to 4.2), odds ratio 0.97 (0.88 to 1.07), <i>p</i> = 0.54)]. There was no effect of intravenous lidocaine found in predetermined subgroup analyses (6- vs. 12-hour duration of infusion, right vs. non-right colectomy, sex, age band and enhanced recovery after surgery compliance). There was no evidence of a difference in other measures of gut function return, pain, qual","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 19","pages":"1-51"},"PeriodicalIF":4.0,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12951268/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147276148","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}
Shankar Kumar, Andrew Plumb, Sue Mallett, Caroline Clarke, Tom Parry, Jing Yi Jessica Weng, Gauraang Bhatnagar, Stuart Bloom, John Hamlin, Ailsa Hart, Simon Travis, Roser Vega, Maira Hameed, Anisha Bhagwanani, Rebecca Greenhalgh, Emma Helbren, James A Stephenson, Ian Zealley, Vivienne Eze, James Franklin, Alison Corr, Arun Gupta, Elizabeth Isaac, Damian Tolan, William Hogg, Antony Higginson, Michela Cicchetti, Sunita Gupta, Miguel Serran, Tim Raine, Mohamed Ahmed, Biljana Brezina, Ilse Patterson, Louise Lee, Richard Pollok, Jaymin Patel, Abigail Seward, Samantha Baillie, Kashfia Chowdary, Sue Philpott, Anvi Wadke, Steve Halligan, Stuart A Taylor
<p><strong>Background: </strong>The ability to predict whether patients with a new diagnosis of Crohn's disease will develop disabling disease is an unmet clinical need. Magnetic resonance enterography is a first-line investigation for Crohn's disease, but its role in prognostication is unknown.</p><p><strong>Objective(s): </strong>To improve prediction of disabling Crohn's disease within 5 years of diagnosis by developing and internally evaluating a multivariable prediction model comprising clinical predictors and adding magnetic resonance enterography scores (Magnetic resonance Enterography Global Score, Simplified Magnetic Resonance Index of Activity and Lémann Index). To estimate the healthcare costs incurred within 5 years of Crohn's disease diagnosis and to explore factors driving costs.</p><p><strong>Design: </strong>A multicentre diagnostic inception cohort.</p><p><strong>Setting: </strong>Nine National Health Service hospitals.</p><p><strong>Participants: </strong>Aged ≥ 16 years with newly diagnosed Crohn's disease.</p><p><strong>Main outcome measures: </strong>Comparative predictive ability of prognostic models, including magnetic resonance enterography scores (Magnetic resonance Enterography Global Score, Simplified Magnetic Resonance Index of Activity and Lémann Index) versus a model based on clinical predictors alone for the development of modified Beaugerie disabling Crohn's disease within 5 years of diagnosis.</p><p><strong>Statistical analysis: </strong>We censored development of modified Beaugerie disabling disease ≤ 90 days from diagnosis, and utilised time-to-event models using Royston-Parmar flexible parametric models. Risk group definitions were prespecified; for risk group definition 1, the high-risk patients were the top 40% with the greatest predicted risk, and the high-risk patients had an absolute risk ≥ 10% for risk group definition 2. The absolute risk cut-off was calculated by sorting patients by predicted risk and using the risk of the eighth (10% of 81) patient who developed modified Beaugerie disabling disease.</p><p><strong>Results: </strong>We studied 194 patients, median age 29, interquartile range 22-44 years. Within 5 years from diagnosis, 42% (81/194) developed modified Beaugerie disabling disease. There was a univariable association between initial need for steroid therapy and developing modified Beaugerie disabling disease [hazard ratio 2.11 (95% confidence interval 1.36 to 3.26)]. Using risk group definition 1, the baseline clinical model had 49% (95% confidence interval 39 to 60) sensitivity and 66% (95% confidence interval 57 to 74) specificity for predicting the development of modified Beaugerie disabling disease. There was no difference in sensitivity and specificity between models incorporating Magnetic resonance Enterography Global Score, Simplified Magnetic Resonance Index of Activity and Lémann Index compared to the baseline clinical model. Using risk group definition 2, the model, including magne
{"title":"Magnetic resonance enterography to predict disabling disease in newly diagnosed Crohn's disease: the METRIC-EF multivariable prediction model, multicentre diagnostic inception cohort study.","authors":"Shankar Kumar, Andrew Plumb, Sue Mallett, Caroline Clarke, Tom Parry, Jing Yi Jessica Weng, Gauraang Bhatnagar, Stuart Bloom, John Hamlin, Ailsa Hart, Simon Travis, Roser Vega, Maira Hameed, Anisha Bhagwanani, Rebecca Greenhalgh, Emma Helbren, James A Stephenson, Ian Zealley, Vivienne Eze, James Franklin, Alison Corr, Arun Gupta, Elizabeth Isaac, Damian Tolan, William Hogg, Antony Higginson, Michela Cicchetti, Sunita Gupta, Miguel Serran, Tim Raine, Mohamed Ahmed, Biljana Brezina, Ilse Patterson, Louise Lee, Richard Pollok, Jaymin Patel, Abigail Seward, Samantha Baillie, Kashfia Chowdary, Sue Philpott, Anvi Wadke, Steve Halligan, Stuart A Taylor","doi":"10.3310/THSN9956","DOIUrl":"10.3310/THSN9956","url":null,"abstract":"<p><strong>Background: </strong>The ability to predict whether patients with a new diagnosis of Crohn's disease will develop disabling disease is an unmet clinical need. Magnetic resonance enterography is a first-line investigation for Crohn's disease, but its role in prognostication is unknown.</p><p><strong>Objective(s): </strong>To improve prediction of disabling Crohn's disease within 5 years of diagnosis by developing and internally evaluating a multivariable prediction model comprising clinical predictors and adding magnetic resonance enterography scores (Magnetic resonance Enterography Global Score, Simplified Magnetic Resonance Index of Activity and Lémann Index). To estimate the healthcare costs incurred within 5 years of Crohn's disease diagnosis and to explore factors driving costs.</p><p><strong>Design: </strong>A multicentre diagnostic inception cohort.</p><p><strong>Setting: </strong>Nine National Health Service hospitals.</p><p><strong>Participants: </strong>Aged ≥ 16 years with newly diagnosed Crohn's disease.</p><p><strong>Main outcome measures: </strong>Comparative predictive ability of prognostic models, including magnetic resonance enterography scores (Magnetic resonance Enterography Global Score, Simplified Magnetic Resonance Index of Activity and Lémann Index) versus a model based on clinical predictors alone for the development of modified Beaugerie disabling Crohn's disease within 5 years of diagnosis.</p><p><strong>Statistical analysis: </strong>We censored development of modified Beaugerie disabling disease ≤ 90 days from diagnosis, and utilised time-to-event models using Royston-Parmar flexible parametric models. Risk group definitions were prespecified; for risk group definition 1, the high-risk patients were the top 40% with the greatest predicted risk, and the high-risk patients had an absolute risk ≥ 10% for risk group definition 2. The absolute risk cut-off was calculated by sorting patients by predicted risk and using the risk of the eighth (10% of 81) patient who developed modified Beaugerie disabling disease.</p><p><strong>Results: </strong>We studied 194 patients, median age 29, interquartile range 22-44 years. Within 5 years from diagnosis, 42% (81/194) developed modified Beaugerie disabling disease. There was a univariable association between initial need for steroid therapy and developing modified Beaugerie disabling disease [hazard ratio 2.11 (95% confidence interval 1.36 to 3.26)]. Using risk group definition 1, the baseline clinical model had 49% (95% confidence interval 39 to 60) sensitivity and 66% (95% confidence interval 57 to 74) specificity for predicting the development of modified Beaugerie disabling disease. There was no difference in sensitivity and specificity between models incorporating Magnetic resonance Enterography Global Score, Simplified Magnetic Resonance Index of Activity and Lémann Index compared to the baseline clinical model. Using risk group definition 2, the model, including magne","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 18","pages":"1-72"},"PeriodicalIF":4.0,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12926852/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146206963","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}
Matthew Walton, Alexis Llewellyn, Eleonora Uphoff, Joseph Lord, Melissa Harden, Robert Hodgson, Mark Simmonds
<p><strong>Background: </strong>Skin cancers are some of the most common types of cancer. Dermatology services receive about 1.2 million referrals a year, but only a small minority are confirmed skin cancer. Artificial intelligence may be helpful in the diagnosis of skin cancer by identifying lesions that are or are not cancerous.</p><p><strong>Objectives: </strong>To investigate the clinical and cost-effectiveness of two artificial intelligence technologies: DERM (Deep Ensemble for Recognition of Malignancy, Skin Analytics) and Moleanalyzer Pro (FotoFinder), as decision aids following a primary care referral.</p><p><strong>Methods: </strong>A rapid systematic review of evidence on the two technologies was conducted. A narrative synthesis was performed, with a meta-analysis of diagnostic accuracy data. Published and unpublished cost-effectiveness evidence on the named technologies, as well as other diagnostic technologies were reviewed. A conceptual model was developed that could form the basis of a full economic evaluation.</p><p><strong>Results: </strong>Four studies of DERM and two of Moleanalyzer Pro were subject to full synthesis. DERM had a sensitivity of 96.1% to detect any malignant lesion (95% confidence interval 95.4 to 96.8); at a specificity of 65.4% (95% confidence interval 64.7 to 66.1). For detecting benign lesions, the sensitivity was 71.5% (95% confidence interval 70.7 to 72.3) for a specificity of 86.2% (95% confidence interval 85.4 to 87.0). Moleanalyzer Pro had lower sensitivity, but higher specificity for detecting melanoma than face-to-face dermatologists. DERM might lead to around half of all patients being discharged without assessment by a dermatologist, but a small number of malignant lesions would be missed. Patient and clinical opinions showed substantial resistance to using artificial intelligence without any assessment of lesions by a dermatologist. No published assessments of the cost-effectiveness of the technologies were identified; three assessments related to skin cancer more broadly in a National Health Service setting were identified. These studies employed similar model structures, but the mechanism by which diagnostic accuracy influenced costs and health outcomes differed. An unpublished cost-utility model was provided by Skin Analytics. Several issues with the modelling approach were identified, particularly the mechanisms by which value is driven and how diagnostic accuracy evidence was used. The conceptual model presents an alternative approach, which aligns more closely with the National Institute for Health and Care Excellence reference case and which more appropriately characterises the long-term consequences of basal cell carcinoma.</p><p><strong>Limitations: </strong>The rapid review approach meant that some relevant material may have been missed, and capacity for synthesis was limited. The proposed conceptual model does not capture non-cash benefits associated with demand on dermatologist time. An a
{"title":"Artificial Intelligence technologies for assessing skin lesions for referral on the urgent suspected cancer pathway to detect benign lesions and reduce secondary care specialist appointments: early value assessment.","authors":"Matthew Walton, Alexis Llewellyn, Eleonora Uphoff, Joseph Lord, Melissa Harden, Robert Hodgson, Mark Simmonds","doi":"10.3310/GJMS0317","DOIUrl":"10.3310/GJMS0317","url":null,"abstract":"<p><strong>Background: </strong>Skin cancers are some of the most common types of cancer. Dermatology services receive about 1.2 million referrals a year, but only a small minority are confirmed skin cancer. Artificial intelligence may be helpful in the diagnosis of skin cancer by identifying lesions that are or are not cancerous.</p><p><strong>Objectives: </strong>To investigate the clinical and cost-effectiveness of two artificial intelligence technologies: DERM (Deep Ensemble for Recognition of Malignancy, Skin Analytics) and Moleanalyzer Pro (FotoFinder), as decision aids following a primary care referral.</p><p><strong>Methods: </strong>A rapid systematic review of evidence on the two technologies was conducted. A narrative synthesis was performed, with a meta-analysis of diagnostic accuracy data. Published and unpublished cost-effectiveness evidence on the named technologies, as well as other diagnostic technologies were reviewed. A conceptual model was developed that could form the basis of a full economic evaluation.</p><p><strong>Results: </strong>Four studies of DERM and two of Moleanalyzer Pro were subject to full synthesis. DERM had a sensitivity of 96.1% to detect any malignant lesion (95% confidence interval 95.4 to 96.8); at a specificity of 65.4% (95% confidence interval 64.7 to 66.1). For detecting benign lesions, the sensitivity was 71.5% (95% confidence interval 70.7 to 72.3) for a specificity of 86.2% (95% confidence interval 85.4 to 87.0). Moleanalyzer Pro had lower sensitivity, but higher specificity for detecting melanoma than face-to-face dermatologists. DERM might lead to around half of all patients being discharged without assessment by a dermatologist, but a small number of malignant lesions would be missed. Patient and clinical opinions showed substantial resistance to using artificial intelligence without any assessment of lesions by a dermatologist. No published assessments of the cost-effectiveness of the technologies were identified; three assessments related to skin cancer more broadly in a National Health Service setting were identified. These studies employed similar model structures, but the mechanism by which diagnostic accuracy influenced costs and health outcomes differed. An unpublished cost-utility model was provided by Skin Analytics. Several issues with the modelling approach were identified, particularly the mechanisms by which value is driven and how diagnostic accuracy evidence was used. The conceptual model presents an alternative approach, which aligns more closely with the National Institute for Health and Care Excellence reference case and which more appropriately characterises the long-term consequences of basal cell carcinoma.</p><p><strong>Limitations: </strong>The rapid review approach meant that some relevant material may have been missed, and capacity for synthesis was limited. The proposed conceptual model does not capture non-cash benefits associated with demand on dermatologist time. An a","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 10","pages":"1-96"},"PeriodicalIF":4.0,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12951253/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146142299","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}
Andrew Bryant, Jan Lecouturier, Giovany Orozco-Leal, Vicky Brocklebank, Sonya Carnell, Thomas J Chadwick, Sarah Dunn, Sally A Johnson, David Kavanagh, Ciara A Kennedy, Michal Malina, Emma K Montgomery, Colin R Muirhead, Yemi Oluboyede, Luke Vale, Chris Weetman, Edwin Kwan Soon Wong, Len Woodward, Neil S Sheerin
<p><strong>Background: </strong>Atypical haemolytic uraemic syndrome is a rare disease (incidence: 0.4 cases per million per year) which, without treatment, is associated with high morbidity and mortality. Eculizumab, a monoclonal complement inhibitor, is an effective treatment but the optimal way to use this high-cost medication (£360,000 per year for an adult) has not been established.</p><p><strong>Objective: </strong>Establish the safety of eculizumab withdrawal and the effectiveness of a monitoring protocol to detect disease relapse and reintroduction of treatment if relapse occurs.</p><p><strong>Setting: </strong>Fifteen hospitals in the United Kingdom.</p><p><strong>Design: </strong>SETS aHUS is a multicentre, open label, prospective, single arm study of the safety and impact of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome using Bayes single arm analysis with a health economic analysis and qualitative study.</p><p><strong>Participants: </strong>Patients over 2 years of age with atypical haemolytic uraemic syndrome who were receiving eculizumab therapy for at least 6 months. Two study arms are described with 28 participants recruited to the withdrawal arm and 11 additional participants recruited to the standard of care arm of the study.</p><p><strong>Intervention: </strong>Withdrawal of eculizumab treatment and replacement with monitoring to assess disease activity with reintroduction of treatment if relapse occurs.</p><p><strong>Main outcome measures: </strong>The primary outcome measure was to determine the safety of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome during the 2-year study period. Patients met a primary outcome of 'safety event occurred' if there was a permanent reduction in estimated glomerular filtration rate or requirement for renal replacement therapy or significant extra-renal manifestation of disease. The health economic analysis compared the cost and health outcomes on and off eculizumab treatment. The qualitative study explored the experiences of patients on living with atypical haemolytic uraemic syndrome and eculizumab treatment, views on withdrawing from treatment and the proposed monitoring plan.</p><p><strong>Results: </strong>One of 28 patients (3.6%) who withdrew from treatment met a primary outcome. Based on the pre-study analysis plan, withdrawal from treatment is not associated with a greater risk to patients compared to remaining on treatment. Of 17 patients with an abnormality in complement regulation, 4 relapsed. Of 11 patients with no abnormality in complement regulation, 0 relapsed. It was possible, by monitoring and rapid patient access, to reintroduce eculizumab treatment when relapse was identified. Most patients welcomed the opportunity to withdraw from treatment but identified concerns about monitoring and the risk of relapse, informed by initial experience at presentation. Withdrawing a patient from treatment saves £4.2M in healthcare co
{"title":"Clinical and cost-effectiveness of eculizumab withdrawal in atypical haemolytic uraemic syndrome: the SETS aHUS multi-centre, open-label, prospective and single-arm study.","authors":"Andrew Bryant, Jan Lecouturier, Giovany Orozco-Leal, Vicky Brocklebank, Sonya Carnell, Thomas J Chadwick, Sarah Dunn, Sally A Johnson, David Kavanagh, Ciara A Kennedy, Michal Malina, Emma K Montgomery, Colin R Muirhead, Yemi Oluboyede, Luke Vale, Chris Weetman, Edwin Kwan Soon Wong, Len Woodward, Neil S Sheerin","doi":"10.3310/GJNS4701","DOIUrl":"10.3310/GJNS4701","url":null,"abstract":"<p><strong>Background: </strong>Atypical haemolytic uraemic syndrome is a rare disease (incidence: 0.4 cases per million per year) which, without treatment, is associated with high morbidity and mortality. Eculizumab, a monoclonal complement inhibitor, is an effective treatment but the optimal way to use this high-cost medication (£360,000 per year for an adult) has not been established.</p><p><strong>Objective: </strong>Establish the safety of eculizumab withdrawal and the effectiveness of a monitoring protocol to detect disease relapse and reintroduction of treatment if relapse occurs.</p><p><strong>Setting: </strong>Fifteen hospitals in the United Kingdom.</p><p><strong>Design: </strong>SETS aHUS is a multicentre, open label, prospective, single arm study of the safety and impact of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome using Bayes single arm analysis with a health economic analysis and qualitative study.</p><p><strong>Participants: </strong>Patients over 2 years of age with atypical haemolytic uraemic syndrome who were receiving eculizumab therapy for at least 6 months. Two study arms are described with 28 participants recruited to the withdrawal arm and 11 additional participants recruited to the standard of care arm of the study.</p><p><strong>Intervention: </strong>Withdrawal of eculizumab treatment and replacement with monitoring to assess disease activity with reintroduction of treatment if relapse occurs.</p><p><strong>Main outcome measures: </strong>The primary outcome measure was to determine the safety of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome during the 2-year study period. Patients met a primary outcome of 'safety event occurred' if there was a permanent reduction in estimated glomerular filtration rate or requirement for renal replacement therapy or significant extra-renal manifestation of disease. The health economic analysis compared the cost and health outcomes on and off eculizumab treatment. The qualitative study explored the experiences of patients on living with atypical haemolytic uraemic syndrome and eculizumab treatment, views on withdrawing from treatment and the proposed monitoring plan.</p><p><strong>Results: </strong>One of 28 patients (3.6%) who withdrew from treatment met a primary outcome. Based on the pre-study analysis plan, withdrawal from treatment is not associated with a greater risk to patients compared to remaining on treatment. Of 17 patients with an abnormality in complement regulation, 4 relapsed. Of 11 patients with no abnormality in complement regulation, 0 relapsed. It was possible, by monitoring and rapid patient access, to reintroduce eculizumab treatment when relapse was identified. Most patients welcomed the opportunity to withdraw from treatment but identified concerns about monitoring and the risk of relapse, informed by initial experience at presentation. Withdrawing a patient from treatment saves £4.2M in healthcare co","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 20","pages":"1-37"},"PeriodicalIF":4.0,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12969051/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147344059","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}
Sarah Baos, Terrie Walker-Smith, Mandy Lui, Elizabeth A Stokes, Jingjing Jiang, Maria Pufulete, Ben Gibbison, Chris A Rogers
<p><strong>Background: </strong>Gabapentin is an anticonvulsant medication with a United Kingdom licence to treat partial seizures and neuropathic pain. It is used off-licence for acute pain and is frequently added to multimodal analgesic regimens after surgery to try and reduce opioid use while controlling pain effectively.</p><p><strong>Objective: </strong>To test the hypothesis that gabapentin reduces opioid use after major surgery and speeds up recovery, thereby reducing postoperative hospital length of stay compared to standard multimodal analgesia.</p><p><strong>Design, setting and participants: </strong>The GAP study was a multicentre, blinded, randomised controlled trial in patients aged ≥ 18 years, undergoing cardiac, thoracic or abdominal surgery with an expected postoperative stay of ≥ 2 days in seven National Health Service hospitals. The trial was designed to provide 90% power to detect a difference of 12.5% in the proportion of participants discharged by the median length of stay in <i>each</i> specialty (500 participants/specialty), which was reduced to 80% (340 participants/specialty) due to COVID-19-related recruitment challenges.</p><p><strong>Interventions: </strong>Participants were randomised 1 : 1 (stratified by surgical specialty) to receive either gabapentin (600 mg before surgery, 300 mg twice daily for 2 days after surgery) or placebo as an adjunct to multimodal pain regimens.</p><p><strong>Main outcome measures: </strong>Primary outcome was length of stay. Secondary outcomes included acute and chronic (Brief Pain Inventory) pain, total opioid use, adverse health events, health-related quality of life (-EQ-5D-5L, Short Form questionnaire-12 items physical component score and mental component score), resource use; cost-effectiveness (outcome measure quality-adjusted life-years using EQ-5D, five-level version).</p><p><strong>Results: </strong>One thousand one hundred and ninety-six (cardiac 500, thoracic 346, abdominal 350) participants consented and were randomised. Baseline characteristics were well balanced across the two groups: median age: 68 years; male sex 796/1195 (66.4%). Of the participants, 223/1195 (18.7%) did not receive all prescribed medication or received medication out of window. There was no difference in length of stay; median placebo (<i>n</i> = 589): 6.15, gabapentin (<i>n</i> = 595): 5.94 days [hazard ratio for discharge 1.07, 95% confidence interval (0.95 to 1.20), <i>p</i> = 0.26]. Opioid use <i>in-hospital</i> differed between surgical specialties (<i>p</i> = 0.001); in the abdominal specialty, it was significantly lower in the gabapentin group in 4 of the first 5 postoperative days [range -26% (-46% to 0%) to -36% (-52% to -14%)], with no differences in the cardiac specialty nor in the thoracic specialty beyond day 2. <i>During follow-up</i>, opioid use was similar in the two groups across all specialties. Acute pain beyond 24 hours was similar (<i>p</i> ≥ 0.15). The incidence of one or more serio
{"title":"Gabapentin as an adjunct to multimodal pain regimens in surgical patients: the GAP placebo-controlled RCT and economic evaluation.","authors":"Sarah Baos, Terrie Walker-Smith, Mandy Lui, Elizabeth A Stokes, Jingjing Jiang, Maria Pufulete, Ben Gibbison, Chris A Rogers","doi":"10.3310/PLMH9787","DOIUrl":"10.3310/PLMH9787","url":null,"abstract":"<p><strong>Background: </strong>Gabapentin is an anticonvulsant medication with a United Kingdom licence to treat partial seizures and neuropathic pain. It is used off-licence for acute pain and is frequently added to multimodal analgesic regimens after surgery to try and reduce opioid use while controlling pain effectively.</p><p><strong>Objective: </strong>To test the hypothesis that gabapentin reduces opioid use after major surgery and speeds up recovery, thereby reducing postoperative hospital length of stay compared to standard multimodal analgesia.</p><p><strong>Design, setting and participants: </strong>The GAP study was a multicentre, blinded, randomised controlled trial in patients aged ≥ 18 years, undergoing cardiac, thoracic or abdominal surgery with an expected postoperative stay of ≥ 2 days in seven National Health Service hospitals. The trial was designed to provide 90% power to detect a difference of 12.5% in the proportion of participants discharged by the median length of stay in <i>each</i> specialty (500 participants/specialty), which was reduced to 80% (340 participants/specialty) due to COVID-19-related recruitment challenges.</p><p><strong>Interventions: </strong>Participants were randomised 1 : 1 (stratified by surgical specialty) to receive either gabapentin (600 mg before surgery, 300 mg twice daily for 2 days after surgery) or placebo as an adjunct to multimodal pain regimens.</p><p><strong>Main outcome measures: </strong>Primary outcome was length of stay. Secondary outcomes included acute and chronic (Brief Pain Inventory) pain, total opioid use, adverse health events, health-related quality of life (-EQ-5D-5L, Short Form questionnaire-12 items physical component score and mental component score), resource use; cost-effectiveness (outcome measure quality-adjusted life-years using EQ-5D, five-level version).</p><p><strong>Results: </strong>One thousand one hundred and ninety-six (cardiac 500, thoracic 346, abdominal 350) participants consented and were randomised. Baseline characteristics were well balanced across the two groups: median age: 68 years; male sex 796/1195 (66.4%). Of the participants, 223/1195 (18.7%) did not receive all prescribed medication or received medication out of window. There was no difference in length of stay; median placebo (<i>n</i> = 589): 6.15, gabapentin (<i>n</i> = 595): 5.94 days [hazard ratio for discharge 1.07, 95% confidence interval (0.95 to 1.20), <i>p</i> = 0.26]. Opioid use <i>in-hospital</i> differed between surgical specialties (<i>p</i> = 0.001); in the abdominal specialty, it was significantly lower in the gabapentin group in 4 of the first 5 postoperative days [range -26% (-46% to 0%) to -36% (-52% to -14%)], with no differences in the cardiac specialty nor in the thoracic specialty beyond day 2. <i>During follow-up</i>, opioid use was similar in the two groups across all specialties. Acute pain beyond 24 hours was similar (<i>p</i> ≥ 0.15). The incidence of one or more serio","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 9","pages":"1-144"},"PeriodicalIF":4.0,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12907993/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146142399","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}
Julia Sanders, Christian Barlow, Peter Brocklehurst, Rebecca Cannings-John, Susan Channon, Christopher Gale, Judith Cutter, Jacqueline Hughes, Billie Hunter, Fiona Lugg-Widger, Sarah Milosevic, Rebecca Milton, Leah Morantz, Mary Nolan, Rachel Plachcinski, Shantini Paranjothy, Michael Robling
<p><strong>Background: </strong>Intrapartum water immersion analgesia has been recommended by the National Institute for Health and Care Excellence since 2007, but high-quality evidence relating to the safety of waterbirth for mothers and their babies was lacking.</p><p><strong>Primary study objective: </strong>To establish whether, in the case of 'low-risk' women who use water immersion during labour, waterbirth, compared to birth out of water, is as safe for mothers and their babies.</p><p><strong>Methods: </strong>A cohort study with non-inferiority design.</p><p><strong>Setting: </strong>Twenty-six National Health Service organisations in England and Wales.</p><p><strong>Participants: </strong>The primary analysis included 60,402 births between January 2015 and June 2022. Primary analysis was restricted to births where the woman: (1) was without complicating medical conditions at the time of pool entry, (2) used water immersion during labour and (3) did not receive obstetric or anaesthetic interventions prior to birth. Comparisons were undertaken between women who gave birth in water and women who gave birth out of water.</p><p><strong>Main outcome measures: </strong>Maternal primary outcome: obstetric anal sphincter injury (with planned subgroup analysis by parity); neonatal composite primary outcome: fetal or neonatal death (after the commencement of intrapartum care and prior to discharge home), neonatal unit admission with respiratory support or the administration of intravenous antibiotics within 48 hours of birth. Separate a priori sample size calculations were undertaken for the maternal and neonatal primary outcomes.</p><p><strong>Results: </strong>After adjusting for differences in the characteristics of women who used intrapartum water immersion and gave birth in or out of water: (1) among nulliparous women, rates of recorded obstetric anal sphincter injury were no higher among women who gave birth in water than among women who left the pool before birth [730 of 15,176 women (4.8%) vs. 641 of 12,210 women (5.3%); adjusted odds ratio 0.97; one-sided 95% confidence interval, -∞ to 1.08]; (2) among parous women, rates of recorded obstetric anal sphincter injury were no higher among women who gave birth in water than among women who left the pool before birth [269 of 24,451 women (1.1%) vs. 144 of 8565 women (1.7%); adjusted odds ratio 0.64; -∞ to 0.78]. Among babies, rates of the primary outcome were no higher among babies born in water than among babies born out of water [263 of 9868 infants (2.7%) vs. 224 of 5078 infants (4.4%); adjusted odds ratio, 0.65; -∞ to 0.79]. All upper confidence intervals of the primary outcomes were lower than the prespecified margins of non-inferiority; therefore, we conclude that the rate of the primary outcomes for mothers and their babies were no higher among waterbirths than among births out of water. Rates of the individual components of the neonatal primary outcome were: Intrapartum or neonatal deat
背景：自2007年以来，国家健康与护理卓越研究所（National Institute for Health and Care Excellence）一直推荐分娩时用水浸泡镇痛，但缺乏与母亲及其婴儿水中分娩安全性相关的高质量证据。主要研究目的：确定在分娩过程中使用水浸泡的“低风险”妇女的情况下，与在水中分娩相比，水中分娩对母亲和婴儿是否同样安全。方法：采用非劣效性设计的队列研究。环境：英格兰和威尔士的26个国家卫生服务组织。参与者：主要分析包括2015年1月至2022年6月期间出生的60402人。初步分析仅限于以下情况的分娩：(1)入池时没有复杂的医疗条件，(2)分娩时使用浸泡水，(3)分娩前未接受产科或麻醉干预。对在水中分娩的妇女和不在水中分娩的妇女进行了比较。主要结局指标：产妇主要结局：产科肛门括约肌损伤（按胎次进行计划亚组分析）；新生儿复合主要结局：胎儿或新生儿死亡（在分娩时护理开始后和出院前），新生儿病房在出生后48小时内接受呼吸支持或静脉注射抗生素。对产妇和新生儿的主要结局进行单独的先验样本量计算。结果：在调整了产时用水浸泡和在水中或在水中分娩的妇女的特征差异后：(1)在未分娩妇女中，记录的产科肛门括约肌损伤率在水中分娩的妇女中并不高于出生前离开游泳池的妇女[15,176名妇女中有730名（4.8%）比在12,210名妇女中有641名（5.3%）]；调整优势比0.97；单侧95%置信区间，-∞至1.08]；(2)在分娩妇女中，在水中分娩的妇女的产科肛门括约肌损伤率不高于在出生前离开游泳池的妇女[24,451名妇女中有269名（1.1%）比在8565名妇女中有144名（1.7%）]；调整优势比0.64；-∞至0.78]。在婴儿中，水中出生的婴儿的主要转归率并不高于非水中出生的婴儿[9868例婴儿中有263例（2.7%）对5078例婴儿中有224例（4.4%）；调整后优势比为0.65；-∞到0.79]。所有主要结局的上置信区间均低于预定的非劣效性边际；因此，我们得出结论，水中分娩的母亲及其婴儿的主要结局率并不高于非水中分娩。新生儿主要结局的各个组成部分的比率为：产时或新生儿死亡，发生在水中出生的3名婴儿中(0.3。每1000名新生儿)，而非在水中出生的婴儿则为零。在新生儿病房为91名（0.9%）水中出生的婴儿和104名（2.0%）非水中出生的婴儿提供呼吸支持；（调整优势比0.44，单侧95%置信区间-∞至0.60）。263名（2.7%）水中出生的婴儿和224名（4.4%）非水中出生的婴儿在出生48小时内使用抗生素（调整后的优势比为0.65，-∞至0.79）。在线调查和访谈确定了影响联合王国生育池使用的各种因素，并强调需要解决与资源可用性（包括具有水中分娩经验的助产士）、单位文化和准则以及工作人员认可相关的问题。现场案例研究发现，与助产单位相比，产科单位在设备和资源、工作人员的态度和信心、高级工作人员的支持和妇女对水中分娩的认识方面更不便利。局限性：该研究的局限性包括无法可靠地识别医疗记录中记录的患有医学或产科并发症的妇女，以及不知道或无法调整的组间混淆的可能性，包括离开游泳池的原因。结论：对于没有怀孕和分娩复杂性的妇女，在分娩过程中使用水浸泡，在水中分娩对母亲和婴儿的安全性与在水中分娩一样。这项研究支持政策和实践，使使用产时水浸泡的无并发症妊娠和分娩妇女能够选择留在水中或离开水中分娩。
{"title":"Establishing the safety of waterbirth for mothers and their babies: the POOL cohort study with nested qualitative component.","authors":"Julia Sanders, Christian Barlow, Peter Brocklehurst, Rebecca Cannings-John, Susan Channon, Christopher Gale, Judith Cutter, Jacqueline Hughes, Billie Hunter, Fiona Lugg-Widger, Sarah Milosevic, Rebecca Milton, Leah Morantz, Mary Nolan, Rachel Plachcinski, Shantini Paranjothy, Michael Robling","doi":"10.3310/GGHD6684","DOIUrl":"10.3310/GGHD6684","url":null,"abstract":"<p><strong>Background: </strong>Intrapartum water immersion analgesia has been recommended by the National Institute for Health and Care Excellence since 2007, but high-quality evidence relating to the safety of waterbirth for mothers and their babies was lacking.</p><p><strong>Primary study objective: </strong>To establish whether, in the case of 'low-risk' women who use water immersion during labour, waterbirth, compared to birth out of water, is as safe for mothers and their babies.</p><p><strong>Methods: </strong>A cohort study with non-inferiority design.</p><p><strong>Setting: </strong>Twenty-six National Health Service organisations in England and Wales.</p><p><strong>Participants: </strong>The primary analysis included 60,402 births between January 2015 and June 2022. Primary analysis was restricted to births where the woman: (1) was without complicating medical conditions at the time of pool entry, (2) used water immersion during labour and (3) did not receive obstetric or anaesthetic interventions prior to birth. Comparisons were undertaken between women who gave birth in water and women who gave birth out of water.</p><p><strong>Main outcome measures: </strong>Maternal primary outcome: obstetric anal sphincter injury (with planned subgroup analysis by parity); neonatal composite primary outcome: fetal or neonatal death (after the commencement of intrapartum care and prior to discharge home), neonatal unit admission with respiratory support or the administration of intravenous antibiotics within 48 hours of birth. Separate a priori sample size calculations were undertaken for the maternal and neonatal primary outcomes.</p><p><strong>Results: </strong>After adjusting for differences in the characteristics of women who used intrapartum water immersion and gave birth in or out of water: (1) among nulliparous women, rates of recorded obstetric anal sphincter injury were no higher among women who gave birth in water than among women who left the pool before birth [730 of 15,176 women (4.8%) vs. 641 of 12,210 women (5.3%); adjusted odds ratio 0.97; one-sided 95% confidence interval, -∞ to 1.08]; (2) among parous women, rates of recorded obstetric anal sphincter injury were no higher among women who gave birth in water than among women who left the pool before birth [269 of 24,451 women (1.1%) vs. 144 of 8565 women (1.7%); adjusted odds ratio 0.64; -∞ to 0.78]. Among babies, rates of the primary outcome were no higher among babies born in water than among babies born out of water [263 of 9868 infants (2.7%) vs. 224 of 5078 infants (4.4%); adjusted odds ratio, 0.65; -∞ to 0.79]. All upper confidence intervals of the primary outcomes were lower than the prespecified margins of non-inferiority; therefore, we conclude that the rate of the primary outcomes for mothers and their babies were no higher among waterbirths than among births out of water. Rates of the individual components of the neonatal primary outcome were: Intrapartum or neonatal deat","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 15","pages":"1-128"},"PeriodicalIF":4.0,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12907988/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146142423","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}
Ben Shelley, Lee Middleton, Andreas Goebel, Stephen Grant, Louise Jackson, Mishal Javed, Marcus Jepson, Nandor Marczin, Rajnikant Mehta, Teresa Melody, Babu Naidu, Hannah Summers, Lajos Szentgyorgyi, Sarah Tearne, Ben Watkins, Matthew Wilson, Andrew Worrall, Joyce Yeung, Fang Gao Smith
<p><strong>Background: </strong>More than a third of patients undergoing thoracotomy suffer from debilitating chronic post-thoracotomy pain lasting months or years postoperatively. Aggressive management of acute pain during the perioperative period may mitigate this risk.</p><p><strong>Objective(s): </strong>To determine the clinical and cost-effectiveness of paravertebral blockade compared to thoracic epidural blockade, by testing the hypothesis that paravertebral blockade reduces the incidence of chronic post-thoracotomy pain.</p><p><strong>Design and methods: </strong>A parallel, open, multicentre, randomised controlled with integrated health-economic evaluation and an internal pilot that incorporated a qualitative recruitment intervention.</p><p><strong>Setting and participants: </strong>Adult patients undergoing thoracotomy in 15 United Kingdom centres.</p><p><strong>Interventions: </strong>Paravertebral blockade compared to thoracic epidural blockade.</p><p><strong>Main outcome measures: </strong>The primary outcome was the presence of chronic post-thoracotomy pain at 6 months post randomisation defined as 'worst chest pain over the last week' of at least moderate intensity, with a visual analogue scale score ≥ 40 mm. Secondary outcomes included visual analogue scale pain scores in the acute (days 1, 2, 3 and discharge) and chronic (3, 6 and 12 months) phases postoperatively; Brief Pain Inventory; Short Form McGill Pain Questionnaire 2; Hospital Anxiety and Depression Scale; patient satisfaction; analgesia use in the acute and chronic phases; complications (analgesic, surgical and pulmonary) and mortality. For the economic evaluation, the EuroQol-5 Dimensions, five-level version questionnaire was utilised.</p><p><strong>Results: </strong>Between 8 January 2019 and 29 September 2023, 770 patients underwent randomisation; 33 did not proceed to thoracotomy. At 6 months, 59 (22%) of 272 participants in the paravertebral blockade group and 47 (16%) of 292 in the thoracic epidural blockade group developed chronic pain [adjusted risk ratio = 1.32 (95% confidence interval 0.93 to 1.86); adjusted risk difference = 0.05 (95% confidence interval -0.01 to 0.11); <i>p</i> = 0.12]. During the acute phase, both worst and average pain was higher on day 1 with paravertebral blockade [adjusted mean difference 7.7 mm (95% confidence interval 2.8 to 12.5) and 7.0 mm (95% confidence interval 2.7 to 11.2), respectively] but not different on days 2 and 3. Hypotension was less common in the paravertebral blockade group [adjusted risk ratio = 0.66 (95% confidence interval 0.46 to 0.94)], and overall complications were comparable between groups. The health-economic analysis demonstrated that thoracic epidural blockade produced an additional 0.04 quality-adjusted life-years when compared to paravertebral blockade, and was associated with slightly lower costs, but these differences were not statistically significant.</p><p><strong>Limitations: </strong>The main limita
{"title":"The clinical and cost-effectiveness of paravertebral blockade versus thoracic epidural blockade in reducing chronic post-thoracotomy pain: TOPIC2 RCT synopsis.","authors":"Ben Shelley, Lee Middleton, Andreas Goebel, Stephen Grant, Louise Jackson, Mishal Javed, Marcus Jepson, Nandor Marczin, Rajnikant Mehta, Teresa Melody, Babu Naidu, Hannah Summers, Lajos Szentgyorgyi, Sarah Tearne, Ben Watkins, Matthew Wilson, Andrew Worrall, Joyce Yeung, Fang Gao Smith","doi":"10.3310/GJFG1715","DOIUrl":"10.3310/GJFG1715","url":null,"abstract":"<p><strong>Background: </strong>More than a third of patients undergoing thoracotomy suffer from debilitating chronic post-thoracotomy pain lasting months or years postoperatively. Aggressive management of acute pain during the perioperative period may mitigate this risk.</p><p><strong>Objective(s): </strong>To determine the clinical and cost-effectiveness of paravertebral blockade compared to thoracic epidural blockade, by testing the hypothesis that paravertebral blockade reduces the incidence of chronic post-thoracotomy pain.</p><p><strong>Design and methods: </strong>A parallel, open, multicentre, randomised controlled with integrated health-economic evaluation and an internal pilot that incorporated a qualitative recruitment intervention.</p><p><strong>Setting and participants: </strong>Adult patients undergoing thoracotomy in 15 United Kingdom centres.</p><p><strong>Interventions: </strong>Paravertebral blockade compared to thoracic epidural blockade.</p><p><strong>Main outcome measures: </strong>The primary outcome was the presence of chronic post-thoracotomy pain at 6 months post randomisation defined as 'worst chest pain over the last week' of at least moderate intensity, with a visual analogue scale score ≥ 40 mm. Secondary outcomes included visual analogue scale pain scores in the acute (days 1, 2, 3 and discharge) and chronic (3, 6 and 12 months) phases postoperatively; Brief Pain Inventory; Short Form McGill Pain Questionnaire 2; Hospital Anxiety and Depression Scale; patient satisfaction; analgesia use in the acute and chronic phases; complications (analgesic, surgical and pulmonary) and mortality. For the economic evaluation, the EuroQol-5 Dimensions, five-level version questionnaire was utilised.</p><p><strong>Results: </strong>Between 8 January 2019 and 29 September 2023, 770 patients underwent randomisation; 33 did not proceed to thoracotomy. At 6 months, 59 (22%) of 272 participants in the paravertebral blockade group and 47 (16%) of 292 in the thoracic epidural blockade group developed chronic pain [adjusted risk ratio = 1.32 (95% confidence interval 0.93 to 1.86); adjusted risk difference = 0.05 (95% confidence interval -0.01 to 0.11); <i>p</i> = 0.12]. During the acute phase, both worst and average pain was higher on day 1 with paravertebral blockade [adjusted mean difference 7.7 mm (95% confidence interval 2.8 to 12.5) and 7.0 mm (95% confidence interval 2.7 to 11.2), respectively] but not different on days 2 and 3. Hypotension was less common in the paravertebral blockade group [adjusted risk ratio = 0.66 (95% confidence interval 0.46 to 0.94)], and overall complications were comparable between groups. The health-economic analysis demonstrated that thoracic epidural blockade produced an additional 0.04 quality-adjusted life-years when compared to paravertebral blockade, and was associated with slightly lower costs, but these differences were not statistically significant.</p><p><strong>Limitations: </strong>The main limita","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":"30 16","pages":"1-21"},"PeriodicalIF":4.0,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12907990/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146149640","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}
Kevin Cooper, Lynda Constable, Thenmalar Vadiveloo, Ayodeji Matuluko, Christine Kennedy, Sharon McCann, Seonaidh Cotton, Katie Gillies, Rebecca Bruce, Paul Smith, Graeme MacLennan, T Justin Clark
<p><strong>Background: </strong>Deep endometriosis causes significant pain which adversely affects quality of life and utilises healthcare and wider societal resources. Laparoscopic excision of endometriosis has shown to improve pain symptoms in observational series but 1 in 14 patients experience serious surgical complications. Medical management centres around hormonal treatment, which is less risky and has been shown to be efficacious but can cause troublesome side effects and is incompatible with conception. There are no randomised controlled trials providing conclusive comparative evidence on clinical and cost-effectiveness of these treatments.</p><p><strong>Objective(s): </strong>To compare the clinical and cost-effectiveness of laparoscopic surgery versus optimised medical treatment for managing deep endometriosis.</p><p><strong>Design and methods: </strong>A multicentre randomised controlled trial, with an internal pilot phase, and economic evaluation, to compare early planned laparoscopic surgery (first attempt at definitive surgery) with or without adjuvant medical treatment versus optimised medical management alone in women with deep endometriosis.</p><p><strong>Setting and participants: </strong>Women presenting with pelvic pain associated with surgically or radiologically confirmed deep endometriosis, suitable for either surgical or medical management, recruited and managed at accredited British Society for Gynaecological Endoscopy Endometriosis Centres.</p><p><strong>Interventions: </strong>Early planned laparoscopic surgery to excise deep endometriosis (with or without medical treatment) or medical management alone.</p><p><strong>Main outcome measures: </strong>The primary outcome was condition-specific quality of life measured using the pain domain of the Endometriosis Health Profile-30 at 18 months post randomisation. The primary health economic outcome was to be incremental cost per quality-adjusted life-year gained at 18 months. Secondary outcomes included quality of life (Endometriosis Health Profile-30), pain, complications, occupational and reproductive outcomes.</p><p><strong>Results: </strong>Three hundred and seventy-seven patients were screened, 103 were eligible and 18 were randomised. Of the eight patients allocated surgery, only one had had their surgery by the time of trial closure and six participants (2/4, 50% allocated surgery and 4/8, 50% allocated medical treatment) had reached the first trial end point at 3 months. No participant reached the primary outcome at 18 months post randomisation.</p><p><strong>Limitations: </strong>The overriding limitation was failure to recruit participants at a satisfactory rate resulting in a final sample of only 18 patients with a target of 320 (inflated to 400 to account for a projected 20% attrition rate). Given the nature of the intervention, it was not possible to blind either the care providers, investigators or participants to their allocated group.</p><p><strong>Conclusion
{"title":"Clinical and cost-effectiveness of medical management versus surgery for deep infiltrating endometriosis: synopsis from the DIAMOND RCT.","authors":"Kevin Cooper, Lynda Constable, Thenmalar Vadiveloo, Ayodeji Matuluko, Christine Kennedy, Sharon McCann, Seonaidh Cotton, Katie Gillies, Rebecca Bruce, Paul Smith, Graeme MacLennan, T Justin Clark","doi":"10.3310/GJKC5715","DOIUrl":"10.3310/GJKC5715","url":null,"abstract":"<p><strong>Background: </strong>Deep endometriosis causes significant pain which adversely affects quality of life and utilises healthcare and wider societal resources. Laparoscopic excision of endometriosis has shown to improve pain symptoms in observational series but 1 in 14 patients experience serious surgical complications. Medical management centres around hormonal treatment, which is less risky and has been shown to be efficacious but can cause troublesome side effects and is incompatible with conception. There are no randomised controlled trials providing conclusive comparative evidence on clinical and cost-effectiveness of these treatments.</p><p><strong>Objective(s): </strong>To compare the clinical and cost-effectiveness of laparoscopic surgery versus optimised medical treatment for managing deep endometriosis.</p><p><strong>Design and methods: </strong>A multicentre randomised controlled trial, with an internal pilot phase, and economic evaluation, to compare early planned laparoscopic surgery (first attempt at definitive surgery) with or without adjuvant medical treatment versus optimised medical management alone in women with deep endometriosis.</p><p><strong>Setting and participants: </strong>Women presenting with pelvic pain associated with surgically or radiologically confirmed deep endometriosis, suitable for either surgical or medical management, recruited and managed at accredited British Society for Gynaecological Endoscopy Endometriosis Centres.</p><p><strong>Interventions: </strong>Early planned laparoscopic surgery to excise deep endometriosis (with or without medical treatment) or medical management alone.</p><p><strong>Main outcome measures: </strong>The primary outcome was condition-specific quality of life measured using the pain domain of the Endometriosis Health Profile-30 at 18 months post randomisation. The primary health economic outcome was to be incremental cost per quality-adjusted life-year gained at 18 months. Secondary outcomes included quality of life (Endometriosis Health Profile-30), pain, complications, occupational and reproductive outcomes.</p><p><strong>Results: </strong>Three hundred and seventy-seven patients were screened, 103 were eligible and 18 were randomised. Of the eight patients allocated surgery, only one had had their surgery by the time of trial closure and six participants (2/4, 50% allocated surgery and 4/8, 50% allocated medical treatment) had reached the first trial end point at 3 months. No participant reached the primary outcome at 18 months post randomisation.</p><p><strong>Limitations: </strong>The overriding limitation was failure to recruit participants at a satisfactory rate resulting in a final sample of only 18 patients with a target of 320 (inflated to 400 to account for a projected 20% attrition rate). Given the nature of the intervention, it was not possible to blind either the care providers, investigators or participants to their allocated group.</p><p><strong>Conclusion","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-27"},"PeriodicalIF":4.0,"publicationDate":"2026-01-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12884356/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146099599","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}
Katie Scandrett, Jacqueline Dinnes, Breanna Morrison, April Coombe, Ridhi Agarwal, Isaac Adu Asare, Phoebe Mead, Andy De Souza, David Elliman, Silvia Lombardo, John Marshall, Sian Taylor-Phillips, Yemisi Takwoingi
<p><strong>Background: </strong>Newborn bloodspot screening offers the potential to detect rare diseases early, enabling timely treatment that can reduce mortality and morbidity. Generating evidence for rare diseases often depends on observational data, making it challenging to formulate recommendations for new screening programmes and evaluate the effectiveness of existing ones.</p><p><strong>Objective(s): </strong>To identify the range of methods and mechanisms used to measure and monitor outcomes from newborn screening programmes using a scoping review.</p><p><strong>Methods: </strong>We included studies published between 2019 and 2024, which evaluated a current or candidate newborn screening programme, or which reported outcomes in screen-detected cases. Studies were categorised into four groups: group 1 reported a comparison and follow-up; group 2 reported a comparison but no follow-up; group 3 reported no comparison with follow-up; and group 4 reported no comparison or follow-up. Data were extracted from a random sample of studies within each group; studies in group 1 were prioritised. Results were reported narratively according to study group. The review was conducted and reported according to current guidance for scoping reviews.</p><p><strong>Data sources: </strong>EMBASE (Ovid), MEDLINE (Ovid) and Science Citation Index (Web of Science - Clarivate).</p><p><strong>Results: </strong>We included 574 primary studies and extracted data from 178. Of the 75 studies in group 1, most compared screen-detected cases with controls (74%). Studies in this group used newborn bloodspot programme databases, registries or record review to identify participants and outcomes; only six (8%) reported use of record linkage. Studies in group 2 (<i>n</i> = 31) mostly reported comparisons of screening tests (25, 81%). Over half of studies in group 3 (<i>n</i> = 34) used newborn bloodspot programme databases to identify participants (53%) and outcomes (65%). A similar pattern was seen in the group 4 (<i>n</i> = 38). Studies reporting follow-up typically relied on retrospective record review or were not well reported. Across all study groups, data on accuracy, epidemiology and genetic variants were common. Studies in group 1 also reported on the effectiveness of newborn bloodspot screening (32/75, 43%), treatment effectiveness (20%) or harms of newborn bloodspot screening (3%).</p><p><strong>Limitations: </strong>Restricting data extraction to a random sample of studies risks missing novel methods or mechanisms.</p><p><strong>Conclusions: </strong>Many studies reported test accuracy metrics and genetic variants in newborn screening. Some data on programme effectiveness were identified, but assessment of potential harms remains limited, and methods for follow-up were poorly reported. Assessment of harms, including overdiagnosis and psychological impact, is crucial to ensuring a net benefit at the population level.</p><p><strong>Future work: </strong>In a second pha
背景：新生儿血斑筛查提供了早期发现罕见疾病的潜力，使及时治疗能够降低死亡率和发病率。为罕见病提供证据往往依赖于观察数据，因此很难为新的筛查方案提出建议，也很难评估现有方案的有效性。目的：通过范围审查确定用于测量和监测新生儿筛查项目结果的方法和机制的范围。方法：我们纳入了2019年至2024年间发表的研究，这些研究评估了当前或候选的新生儿筛查项目，或报告了筛查检测病例的结果。研究分为四组：第一组报告了比较和随访；第二组报告了比较，但没有随访；第三组无随访比较；第4组没有进行比较或随访。数据从每组的随机研究样本中提取；第一组的研究被优先考虑。结果按研究组分组记叙。审查是根据当前范围审查指南进行和报告的。数据来源：EMBASE (Ovid), MEDLINE （Ovid）和Science Citation Index （Web of Science - Clarivate）。结果：我们纳入了574项初步研究，提取了178项数据。在第一组的75项研究中，大多数将筛查检测到的病例与对照组进行比较（74%）。该组研究使用新生儿血斑规划数据库、登记处或记录审查来确定参与者和结果；只有6个（8%）报告使用了记录链接。第2组（n = 31）的研究大多报告了筛查试验的比较（25.81%）。第3组中超过一半的研究（n = 34）使用新生儿血斑规划数据库来确定参与者（53%）和结果（65%）。第4组（n = 38）也出现了类似的情况。报告随访的研究通常依赖于回顾性记录审查或没有得到很好的报道。在所有研究组中，准确性、流行病学和遗传变异的数据都很常见。第1组的研究还报告了新生儿血斑筛查的有效性（32/ 75,43 %）、治疗有效性（20%）或新生儿血斑筛查的危害（3%）。局限性：将数据提取限制在随机的研究样本中，可能会错过新的方法或机制。结论：许多研究报告了新生儿筛查的测试准确性指标和遗传变异。确定了一些关于方案有效性的数据，但对潜在危害的评估仍然有限，后续行动的方法报告也很差。评估危害，包括过度诊断和心理影响，对于确保在人口层面上获得净效益至关重要。未来的工作：在第二阶段的工作中，将对使用不同方法和机制的研究进行深入评估，以确定它们可以提供结果数据的程度，从而为正在进行的和候选筛选计划的评估提供信息。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NIHR167910。
{"title":"Methods and mechanisms for measuring and monitoring outcomes from newborn bloodspot screening: a scoping review.","authors":"Katie Scandrett, Jacqueline Dinnes, Breanna Morrison, April Coombe, Ridhi Agarwal, Isaac Adu Asare, Phoebe Mead, Andy De Souza, David Elliman, Silvia Lombardo, John Marshall, Sian Taylor-Phillips, Yemisi Takwoingi","doi":"10.3310/GJJD1717","DOIUrl":"10.3310/GJJD1717","url":null,"abstract":"<p><strong>Background: </strong>Newborn bloodspot screening offers the potential to detect rare diseases early, enabling timely treatment that can reduce mortality and morbidity. Generating evidence for rare diseases often depends on observational data, making it challenging to formulate recommendations for new screening programmes and evaluate the effectiveness of existing ones.</p><p><strong>Objective(s): </strong>To identify the range of methods and mechanisms used to measure and monitor outcomes from newborn screening programmes using a scoping review.</p><p><strong>Methods: </strong>We included studies published between 2019 and 2024, which evaluated a current or candidate newborn screening programme, or which reported outcomes in screen-detected cases. Studies were categorised into four groups: group 1 reported a comparison and follow-up; group 2 reported a comparison but no follow-up; group 3 reported no comparison with follow-up; and group 4 reported no comparison or follow-up. Data were extracted from a random sample of studies within each group; studies in group 1 were prioritised. Results were reported narratively according to study group. The review was conducted and reported according to current guidance for scoping reviews.</p><p><strong>Data sources: </strong>EMBASE (Ovid), MEDLINE (Ovid) and Science Citation Index (Web of Science - Clarivate).</p><p><strong>Results: </strong>We included 574 primary studies and extracted data from 178. Of the 75 studies in group 1, most compared screen-detected cases with controls (74%). Studies in this group used newborn bloodspot programme databases, registries or record review to identify participants and outcomes; only six (8%) reported use of record linkage. Studies in group 2 (<i>n</i> = 31) mostly reported comparisons of screening tests (25, 81%). Over half of studies in group 3 (<i>n</i> = 34) used newborn bloodspot programme databases to identify participants (53%) and outcomes (65%). A similar pattern was seen in the group 4 (<i>n</i> = 38). Studies reporting follow-up typically relied on retrospective record review or were not well reported. Across all study groups, data on accuracy, epidemiology and genetic variants were common. Studies in group 1 also reported on the effectiveness of newborn bloodspot screening (32/75, 43%), treatment effectiveness (20%) or harms of newborn bloodspot screening (3%).</p><p><strong>Limitations: </strong>Restricting data extraction to a random sample of studies risks missing novel methods or mechanisms.</p><p><strong>Conclusions: </strong>Many studies reported test accuracy metrics and genetic variants in newborn screening. Some data on programme effectiveness were identified, but assessment of potential harms remains limited, and methods for follow-up were poorly reported. Assessment of harms, including overdiagnosis and psychological impact, is crucial to ensuring a net benefit at the population level.</p><p><strong>Future work: </strong>In a second pha","PeriodicalId":12898,"journal":{"name":"Health technology assessment","volume":" ","pages":"1-48"},"PeriodicalIF":4.0,"publicationDate":"2026-01-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12862532/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146046461","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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