Thomas C Williams PhD, Robin Marlow PhD, Prof Steve Cunningham PhD, Simon B Drysdale PhD, Helen E Groves MB BCh BAO PhD, Samantha Hunt BSc, Dalia Iskander PhD, Xinxue Liu PhD, Mark D Lyttle MBChB, Chengetai D Mpamhanga MSc, Shaun O'Hagan MB BCh BAO, Thomas Waterfield PhD, Damian Roland PhD, PERUKI & BronchStart Collaboration, Catriona M Middleton, Maggie Connon, Jill M Paxton, Nicola Thomson, James Thomas Baker, Susie R Hardwick, Karena Jan Fraser, Kathryn Frances Kimber, Asa JE French, Meriel FJ Tolhurst-Cleaver, Sarah Siner, Stephen Fletcher, Karen Geoghegan, Laura Ann Tremarco, Kelly Curran, Cynthia Dzokoto Diaba, Chidinma Joanne Iheanetu-Oguejiofor, Lobsong Dolma, Stephen Waithaka Ruo, Richard Benjamin Stuart Berg, Sara Mohammed Osman Farah, Elena Kehtarnavaz, Abigail Louise Whitehouse, Grace E Wild, Sobia Siraj, Ioannis Kyrgios, Amutha Anpananthar, Lee James Tubman, Sunil S Bhopal, Jessica Reynolds, Gabrielle Larkin, Elizabeth Ledger, Ruth E Bonsor, Dodd Hopgood, Robert Eggen, Katy Lillie, Mat Stuart, Alison O'Kelly, Chiara De Leonardis, Stephen P Brearey, Caroline A Burchett, Sarah E de-Beger, Claire Bell, Babu G Benjamin, Sarah E Farmer, Mudiyur Gopi, Natalie Keenan, Jen G Browning, Caroline Blackstock, Rachel O'Brien, Sylvester S Gomes, Gracita M Woods, Dilini Vasanthakumar, Kayleigh Kanhai, Heather J Jarman, Zee R Karame, Steve J Foster, Susanne Cathcart, Louise E Edwards, James R Poole-Cowley, Samantha E Hunt, Joanne L Tomlinson, Jo William Tillett, Simon R Dowson, Surendra T Singham, Perpetual S Palmer, Eilidh E Grant, Pauline Miller, Kelum K Perera, Jasmine L Stares, Manuela E Brazil, Sharryn Gardner, Kerri Bowness, Moira Morrison, Nicholas Richens, Heather Deall, Jemma Parratt, Stephanie - Grigsby, Nina Vanner, Francesca Diaz, Graham Johnson, Jonathan Riley, Alison Fletcher, Lianne Hufton, Alison Rockey, Alison Matthews, Claire Fearn, Elisha Cousins, Katie Louise Bub, Cynthia Dzokoto Diaba, Chidinma Joanne Iheanetu-Oguejiofor, Lobsong Dolma, Stephen Waithaka Ruo, Elena Kehtarnavaz, George R Hamson, Hannah Randall, Jessica J Evans, Virginia J Griffiths, Sarah L Diment, Sebastian J Gray, Nikki Leanne Furness, Stuart Gormley, Rebecca Scott, Victoria Bamber, Kathryn Jones, Megan Wilcock, Louise A Fairlie, Rophy Ganancial Nunes, Niall G Mullen, Clare O'Leary, Kerri Bowness, Richard George Burridge, Katrina S Hall, Leanne M Taylor, Lily J Kirkpatrick, Erum Jamall, Grace Wild, David Hartin, Rebecca Francis, Genevieve Nguya, Deborah Beeby
{"title":"Bivalent prefusion F vaccination in pregnancy and respiratory syncytial virus hospitalisation in infants in the UK: results of a multicentre, test-negative, case-control study","authors":"Thomas C Williams PhD, Robin Marlow PhD, Prof Steve Cunningham PhD, Simon B Drysdale PhD, Helen E Groves MB BCh BAO PhD, Samantha Hunt BSc, Dalia Iskander PhD, Xinxue Liu PhD, Mark D Lyttle MBChB, Chengetai D Mpamhanga MSc, Shaun O'Hagan MB BCh BAO, Thomas Waterfield PhD, Damian Roland PhD, PERUKI & BronchStart Collaboration, Catriona M Middleton, Maggie Connon, Jill M Paxton, Nicola Thomson, James Thomas Baker, Susie R Hardwick, Karena Jan Fraser, Kathryn Frances Kimber, Asa JE French, Meriel FJ Tolhurst-Cleaver, Sarah Siner, Stephen Fletcher, Karen Geoghegan, Laura Ann Tremarco, Kelly Curran, Cynthia Dzokoto Diaba, Chidinma Joanne Iheanetu-Oguejiofor, Lobsong Dolma, Stephen Waithaka Ruo, Richard Benjamin Stuart Berg, Sara Mohammed Osman Farah, Elena Kehtarnavaz, Abigail Louise Whitehouse, Grace E Wild, Sobia Siraj, Ioannis Kyrgios, Amutha Anpananthar, Lee James Tubman, Sunil S Bhopal, Jessica Reynolds, Gabrielle Larkin, Elizabeth Ledger, Ruth E Bonsor, Dodd Hopgood, Robert Eggen, Katy Lillie, Mat Stuart, Alison O'Kelly, Chiara De Leonardis, Stephen P Brearey, Caroline A Burchett, Sarah E de-Beger, Claire Bell, Babu G Benjamin, Sarah E Farmer, Mudiyur Gopi, Natalie Keenan, Jen G Browning, Caroline Blackstock, Rachel O'Brien, Sylvester S Gomes, Gracita M Woods, Dilini Vasanthakumar, Kayleigh Kanhai, Heather J Jarman, Zee R Karame, Steve J Foster, Susanne Cathcart, Louise E Edwards, James R Poole-Cowley, Samantha E Hunt, Joanne L Tomlinson, Jo William Tillett, Simon R Dowson, Surendra T Singham, Perpetual S Palmer, Eilidh E Grant, Pauline Miller, Kelum K Perera, Jasmine L Stares, Manuela E Brazil, Sharryn Gardner, Kerri Bowness, Moira Morrison, Nicholas Richens, Heather Deall, Jemma Parratt, Stephanie - Grigsby, Nina Vanner, Francesca Diaz, Graham Johnson, Jonathan Riley, Alison Fletcher, Lianne Hufton, Alison Rockey, Alison Matthews, Claire Fearn, Elisha Cousins, Katie Louise Bub, Cynthia Dzokoto Diaba, Chidinma Joanne Iheanetu-Oguejiofor, Lobsong Dolma, Stephen Waithaka Ruo, Elena Kehtarnavaz, George R Hamson, Hannah Randall, Jessica J Evans, Virginia J Griffiths, Sarah L Diment, Sebastian J Gray, Nikki Leanne Furness, Stuart Gormley, Rebecca Scott, Victoria Bamber, Kathryn Jones, Megan Wilcock, Louise A Fairlie, Rophy Ganancial Nunes, Niall G Mullen, Clare O'Leary, Kerri Bowness, Richard George Burridge, Katrina S Hall, Leanne M Taylor, Lily J Kirkpatrick, Erum Jamall, Grace Wild, David Hartin, Rebecca Francis, Genevieve Nguya, Deborah Beeby","doi":"10.1016/s2352-4642(25)00155-5","DOIUrl":"https://doi.org/10.1016/s2352-4642(25)00155-5","url":null,"abstract":"Respiratory syncytial virus (RSV) is the leading cause of acute lower respiratory infections (ALRI) in infants younger than 6 months globally. A maternal bivalent RSV prefusion F (RSVpreF) vaccine was introduced to the UK in late summer in 2024 (August 12 in Scotland and September 1 in England), with all pregnant women at 28 weeks or more of gestation eligible for vaccination. We aimed to understand RSVpreF vaccine effectiveness in a real-world setting.","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"96 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144665002","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Cal H Robinson MD, Eddy Fan MD PhD, Sonia M Grandi PhD, Martin Urner MD PhD, Prof Rulan S Parekh MD MS
{"title":"Target trial emulation in paediatric research: how can causal effects be estimated from observational data?","authors":"Cal H Robinson MD, Eddy Fan MD PhD, Sonia M Grandi PhD, Martin Urner MD PhD, Prof Rulan S Parekh MD MS","doi":"10.1016/s2352-4642(25)00131-2","DOIUrl":"https://doi.org/10.1016/s2352-4642(25)00131-2","url":null,"abstract":"Conducting randomised controlled trials (RCTs) in rare paediatric diseases is often impractical or prohibitively expensive. Observational data from longitudinal cohort studies, disease registries, and population-based databases exist for children and adolescents, but standard observational analyses are typically limited by bias. Target trial emulation methods can improve the quality of observational analysis, address common sources of bias, and help fill evidence gaps in paediatric clinical practice. Applying target trial emulation methods in paediatric research creates unique opportunities, but also poses specific challenges. Tailored approaches are needed to address issues with small sample size, treatment-effect heterogeneity, longitudinal follow-up, and missing data. This Review aims to outline key concepts, such as what causal inference and target trial emulation are, justify the use of target trial emulation methods in paediatric observational research, discuss approaches to emulating key elements of an RCT protocol, and highlight unique paediatric applications of target trial emulation.","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"109 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-07-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144621809","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Hannah K Mitchell BMBS MSc, Sarah E Seaton PhD, Khurram Mustafa MBBS, Gareth A L Jones MBChB, Hannah Buckley MSc, Peter Davis MBChB, Christopher Leahy PhD, Prof Richard G Feltbower PhD, Prof Padmanabhan Ramnarayan MBBS MD
{"title":"Contribution of ethnicity and deprivation to paediatric critical care outcomes in the UK, 2008–21: a national retrospective cohort study","authors":"Hannah K Mitchell BMBS MSc, Sarah E Seaton PhD, Khurram Mustafa MBBS, Gareth A L Jones MBChB, Hannah Buckley MSc, Peter Davis MBChB, Christopher Leahy PhD, Prof Richard G Feltbower PhD, Prof Padmanabhan Ramnarayan MBBS MD","doi":"10.1016/s2352-4642(25)00156-7","DOIUrl":"https://doi.org/10.1016/s2352-4642(25)00156-7","url":null,"abstract":"Evidence from UK paediatric intensive care units (PICUs) demonstrates increased incidence of admission among children of Asian and Black ethnicity and children residing in more deprived areas. We aimed to investigate whether mortality in PICU is associated with ethnicity and child poverty.","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"14 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144621810","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Marian J Bakermans-Kranenburg, Marinus H van IJzendoorn
{"title":"Monitoring change from residential housing care to family-based care for children.","authors":"Marian J Bakermans-Kranenburg, Marinus H van IJzendoorn","doi":"10.1016/S2352-4642(24)00102-0","DOIUrl":"10.1016/S2352-4642(24)00102-0","url":null,"abstract":"","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":" ","pages":"549-550"},"PeriodicalIF":0.0,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140961265","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Lipoprotein apheresis and long-term cardiovascular health: a real answer for children with HoFH?","authors":"Ari Horton","doi":"10.1016/S2352-4642(24)00105-6","DOIUrl":"10.1016/S2352-4642(24)00105-6","url":null,"abstract":"","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":" ","pages":"468-469"},"PeriodicalIF":0.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140961262","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
M Doortje Reijman, Tycho R Tromp, Barbara A Hutten, G Kees Hovingh, Dirk J Blom, Alberico L Catapano, Marina Cuchel, Eldad J Dann, Antonio Gallo, Lisa C Hudgins, Frederick J Raal, Kausik K Ray, Fouzia Sadiq, Handrean Soran, Jaap W Groothoff, Albert Wiegman, D Meeike Kusters
{"title":"Cardiovascular outcomes in patients with homozygous familial hypercholesterolaemia on lipoprotein apheresis initiated during childhood: long-term follow-up of an international cohort from two registries.","authors":"M Doortje Reijman, Tycho R Tromp, Barbara A Hutten, G Kees Hovingh, Dirk J Blom, Alberico L Catapano, Marina Cuchel, Eldad J Dann, Antonio Gallo, Lisa C Hudgins, Frederick J Raal, Kausik K Ray, Fouzia Sadiq, Handrean Soran, Jaap W Groothoff, Albert Wiegman, D Meeike Kusters","doi":"10.1016/S2352-4642(24)00073-7","DOIUrl":"10.1016/S2352-4642(24)00073-7","url":null,"abstract":"<p><strong>Background: </strong>Homozygous familial hypercholesterolaemia (HoFH) is a rare genetic disease characterised by extremely high plasma LDL cholesterol from birth, causing atherosclerotic cardiovascular disease at a young age. Lipoprotein apheresis in combination with lipid-lowering drugs effectively reduce LDL cholesterol, but long-term health outcomes of such treatment are unknown. We aimed to investigate the long-term cardiovascular outcomes associated with lipoprotein apheresis initiated in childhood or adolescence.</p><p><strong>Methods: </strong>In this cohort study, data were drawn from the HoFH International Clinical Collaboration (HICC) and the international registry for Children with Homozygous Hypercholesterolemia on Lipoprotein Apheresis (CHAIN). An overall cohort included patients diagnosed with HoFH aged 0-18 years who were alive and in follow-up between Jan 1, 2010, and Nov 8, 2021, and whose high plasma LDL cholesterol concentrations made them eligible for lipoprotein apheresis. To compare cardiovascular outcomes, patients who initiated lipoprotein apheresis in childhood (lipoprotein apheresis group) and patients who only received lipid-lowering drugs (pharmacotherapy-only group) were matched by sex and untreated plasma LDL cholesterol concentrations. The primary outcome was a composite of cardiovascular death, myocardial infarction, ischaemic stroke, percutaneous coronary intervention, coronary artery bypass grafting, aortic valve replacement, peripheral artery disease, carotid endarterectomy, angina pectoris, and supra-aortic or aortic stenosis (collectively referred to as atherosclerotic cardiovascular disease), for which survival analyses were performed in the matched cohort. Cox regression analyses were used to compare disease-free survival between cohorts and to calculate hazard ratio (HR) and 95% CI adjusted for sex, age at diagnosis, untreated plasma LDL cholesterol concentration, and number of lipid-lowering therapies other than lipoprotein apheresis.</p><p><strong>Findings: </strong>The overall cohort included 404 patients with a median age at diagnosis of 6·0 years (IQR 3·0-9·5) and median untreated plasma LDL cholesterol of 17·8 mmol/L (14·7-20·8). The matched cohorts included 250 patients (125 patients per group), with a median untreated LDL cholesterol of 17·2 mmol/L (14·8-19·7). Mean reduction in plasma LDL cholesterol concentrations between baseline and final follow-up was greater in the lipoprotein apheresis group (-55% [95% CI -60 to -51] vs -31% [-36 to -25]; p<0·0001). Patients in the lipoprotein apheresis group had longer atherosclerotic cardiovascular disease-free survival (adjusted HR 0·52 [95% CI 0·32-0·85]) and longer cardiovascular death-free survival (0·0301 [0·0021-0·4295]). Cardiovascular death was more common in the pharmacotherapy-only group than in the lipoprotein apheresis group (ten [8%] vs one [1%]; p=0·010), whereas median age at coronary artery bypass grafting was lower in the lipopro","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":" ","pages":"491-499"},"PeriodicalIF":0.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963317/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140961259","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}