The Lancet. Child & adolescent health最新文献

{"title":"Model-informed precision dosing: from a niche for pharmacologists to daily business of paediatricians?","authors":"Saskia N de Wildt","doi":"10.1016/s2352-4642(26)00018-0","DOIUrl":"https://doi.org/10.1016/s2352-4642(26)00018-0","url":null,"abstract":"","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"21 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147666750","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":"Bedside model-informed precision dosing of vancomycin in severely ill neonates and children in Belgium (the BENEFICIAL trial): a multicentre, randomised controlled trial.","authors":"Pieter A De Cock,Roos Colman,Anne Smits,Fiammetta Piersigilli,Marleen Renard,An Van Damme,Alexander Clarysse,Evelyn Dhont,Sophie Vanhaesebrouck,Victoria Bordon,Lidvine Boland,Bram De Wilde,Anca Amza,Lieselot Vanlanduyt,Thi Van Anh Nguyen,Filip Cools,Jutte van der Werff Ten Bosch,Daphne Vens,Frank Derriks,Laurent Houtekie,Reiner Mauel,Dimitri Van der Linden,Petra Schelstraete, ","doi":"10.1016/s2352-4642(25)00385-2","DOIUrl":"https://doi.org/10.1016/s2352-4642(25)00385-2","url":null,"abstract":"BACKGROUNDVancomycin is widely used to treat serious Gram-positive infections but is difficult to dose given its narrow therapeutic index and risk of acute kidney injury at high doses. We aimed to study whether model-informed precision dosing (MIPD) of vancomycin, compared with standard-of-care therapeutic drug monitoring (TDM), increases pharmacokinetic and pharmacodynamic target attainment, is safe, and reduces vancomycin-associated acute kidney injury in children with severe illness.METHODSThe BENEFICIAL trial is a pragmatic, individually randomised, controlled superiority trial done in 14 paediatric or neonatal intensive care and haemato-oncology units in seven hospitals in Belgium. Critically ill patients younger than 18 years initiating intravenous vancomycin for suspected or confirmed Gram-positive infection were eligible. Key exclusion criteria were extracorporeal support, severe acute kidney injury, chronic kidney disease, and imminent death. The intervention combined the use of an MIPD dosing calculator for starting and follow-up doses, with extra sampling for TDM in the first hours of treatment compared with standard-of-care TDM. Patients were randomly assigned (1:1) to MIPD or standard-of-care TDM of vancomycin using stratified permuted blocks by ward type. Allocation occurred via a secure web interface; patients, families, and the biostatistician were masked, but treating physicians and pharmacist staff were not. The intervention used Bayesian software with early sampling to estimate AUC. The primary outcome was the proportion of patients with a 24-h AUC-to-MIC ratio of 400-600 mg·h/L, assuming a minimum inhibitory concentration of 1 mg/L, 24-48 h after treatment initiation. A key secondary outcome was the proportion of patients with new or worsening acute kidney injury or death. These outcomes were assessed in the intention-to-treat (ITT) population (all randomly assigned patients who provided informed consent). Safety was evaluated in all patients who received at least one dose of vancomycin. This trial is registered with ClinicalTrials.gov (NCT04666948) and the EU Clinical Trials register (EudraCT 2019-004538-40), and is closed to recruitment.FINDINGSBetween Dec 28, 2020, and Dec 14, 2023, 332 patients aged between 1 day and 18 years were randomly assigned, 165 to the standard-of-care group and 167 to the intervention group. 18 participants were excluded from the analysis when their deferred consent was not followed by informed consent; therefore 314 patients (179 male and 135 female) were included in the analysis. Target AUC-to-MIC ratio attainment at 24-48 h was found in 82 (53·9%) of 152 patients with available data in the standard-of-care group and 112 (71·8%) of 156 in the MIPD group (absolute difference 18·9% [1·7 to 34·7]). The proportion of patients with acute kidney injury or all-cause mortality was numerically but not significantly lower in the intervention group (26 [16·9%] of 154 vs 19 [12·4%] of 153; absolute differen","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"48 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147666749","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":"Bridging silos in child-inclusive One Health practice.","authors":"Andreas Schultz,Ludmila Lobkowicz,Madeleine Kalisch,David Mir-Salim,Andrea S Winkler","doi":"10.1016/s2352-4642(26)00038-6","DOIUrl":"https://doi.org/10.1016/s2352-4642(26)00038-6","url":null,"abstract":"","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"40 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147663732","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":"The war in Ukraine: mental health toll on children and adolescents","authors":"Ed Holt","doi":"10.1016/s2352-4642(26)00076-3","DOIUrl":"https://doi.org/10.1016/s2352-4642(26)00076-3","url":null,"abstract":"","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"18 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147597990","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":"Familial confounding and causal inference in child and adolescent neurodevelopment and mental health.","authors":"David Mataix-Cols,Josep Pol-Fuster,Lorena Fernández de la Cruz,Paul Lichtenstein,Brian M D'Onofrio,Gustaf Brander,Renee Gardner","doi":"10.1016/s2352-4642(26)00045-3","DOIUrl":"https://doi.org/10.1016/s2352-4642(26)00045-3","url":null,"abstract":"Recent announcements by the US Government linking paracetamol use during pregnancy to autism in offspring highlight the risks of misinterpreting observational research to inform policy; this is a clear example of the principle that association does not equal causation. Unmeasured familial confounding is a common bias in epidemiological studies, whereby shared genetic or environmental factors within families produce spurious associations between risk factors and outcomes. In this Viewpoint, aimed at clinicians from a range of disciplines working with children and young people with neurodevelopmental and mental health conditions, we discuss the concept of familial confounding and why it matters for causal inference. We illustrate the concept through several examples and outline study designs that can be used to minimise bias due to familial confounding, along with their strengths and limitations. We also highlight the potential consequences of ignoring familial confounding, both for causal inference and for policy making. Finally, we emphasise triangulation across complementary study designs as a key strategy for strengthening causal inference and informing policy decisions based on observational evidence.","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"22 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-03-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147524513","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":"Tuberculosis research principles and priorities in children and adolescents: an international consensus statement","authors":"Moorine P Sekadde, Silvia S Chiang, Annemieke Brands, Martina Casenghi, Chishala Chabala, Stephen M Graham, Tiziana Masini, James A Seddon, Sabine E Verkuijl, Kerri Viney, Ben J Marais, Shakil Ahmed, Pauline M. Amuge, Robindra Basu Roy, Maryline Bonnet, Grania Brigden, Charlotte Colvin, Claudia M. Denkinger, Peter J. Dodd, Leslie A. Enane, Molly F. Franke, Jennifer J. Furin, Betina Mendez Alcântra Gabardo, Anthony J. Garcia-Prats, Norma E. González, Graeme Hoddinott, Cleotilde H. How, Helena Huerga, Julie Huynh, Devan Jaganath, Alexander W. Kay, Kobto G. Koura, Katharina Kranzer, Marian Loveday, Elizabeth Maleche-Obimbo, Mariama Mahmoud, Olivier Marcy, Mansa Mbenga, Lindsay R. McKenna, Brittany K. Moore, Sharon Nachman, Nicole Salazar-Austin, Clemax C. Sant'Anna, Adong Shen, H. Simon Schaaf, Sangeeta Sharma, Alena Skrahina, Jeffrey R. Starke, Marc Tebruegge, Rina Triasih, Marieke M. van der Zalm, Courtney M. Yuen, Heather J. Zar","doi":"10.1016/s2352-4642(26)00046-5","DOIUrl":"https://doi.org/10.1016/s2352-4642(26)00046-5","url":null,"abstract":"","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"38 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-03-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147502055","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":"Listening to the room","authors":"Apurva Parikh","doi":"10.1016/s2352-4642(26)00068-4","DOIUrl":"https://doi.org/10.1016/s2352-4642(26)00068-4","url":null,"abstract":"","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"127 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147447393","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":"Autistic SPACE: clinician advocacy for improving school environments for autistic young people","authors":"Chun-Hao Liu, Mary Doherty, Meng-Chuan Lai","doi":"10.1016/s2352-4642(26)00043-x","DOIUrl":"https://doi.org/10.1016/s2352-4642(26)00043-x","url":null,"abstract":"","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"9 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147447408","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":"Support without identification: a regressive shift in neurodevelopmental care?","authors":"Jason Lang, Marion Rutherford, Ruchika Gajwani, Sebastian C K Shaw, Mary Doherty, Helen Minnis","doi":"10.1016/s2352-4642(26)00037-4","DOIUrl":"https://doi.org/10.1016/s2352-4642(26)00037-4","url":null,"abstract":"","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"53 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147447402","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":"Safety of in vivo gene therapy in children: mechanisms and management of liver injury","authors":"Rebecca Jeyaraj MRCPCH, Li-An K Brown FRCPath, Barath Jagadisan MD, Prof Francesco Muntoni MD, Julien Baruteau MD PhD, Girish Gupte MD, Prof Judith Breuer MD, Prof Paul Gissen PhD, Prof Ye H Oo PhD, Prof Anil Dhawan MD","doi":"10.1016/s2352-4642(25)00380-3","DOIUrl":"https://doi.org/10.1016/s2352-4642(25)00380-3","url":null,"abstract":"Gene therapies based on recombinant adeno-associated viruses (rAAVs) are radically changing the disease course of several inherited disorders with historically few treatment options. Increasing use of these therapies has revealed multiple immune-mediated adverse effects. Liver injury following systemic delivery of rAAV gene therapy is now well documented, ranging from asymptomatic liver enzyme elevations to rare cases of fatal liver failure. Most cases of severe or fatal liver failure have been reported in patients with neuromuscular disorders, who receive high systemic rAAV doses compared with patients receiving liver-targeted therapies. This Review summarises the principles of modern gene therapy and important adverse effects. We discuss mechanisms of liver injury and current uncertainties, focusing on the T cell-mediated immune response that is thought to underlie the development of severe hepatocellular injury. Finally, we describe the clinical management of liver injury in paediatric patients, recognising the scarce evidence to inform decisions about immunosuppression, and consider strategies to help address this risk.","PeriodicalId":94246,"journal":{"name":"The Lancet. Child & adolescent health","volume":"247 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146146648","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}