{"title":"Echoes of healing: late effects of HCT for non-malignant disease in childhood","authors":"Hasan Hashem","doi":"10.1016/S2352-4642(24)00168-8","DOIUrl":"10.1016/S2352-4642(24)00168-8","url":null,"abstract":"","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"8 10","pages":"Pages 709-711"},"PeriodicalIF":19.9,"publicationDate":"2024-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142116704","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Antoine Brault PhD , Isabelle Pontais PhD , Vincent Enouf PhD , Christine Debeuret PharmD , Emma Bloch MSc , Juliette Paireau PhD , Prof Marie-Anne Rameix-Welti PhD , Michael White PhD , Gaëlle Baudemont MsC , Prof Bruno Lina PhD , Isabelle Parent du Châtelet MD , Jean-Sébastien Casalegno MD , Sophie Vaux PharmD , Prof Simon Cauchemez PhD
{"title":"Effect of nirsevimab on hospitalisations for respiratory syncytial virus bronchiolitis in France, 2023–24: a modelling study","authors":"Antoine Brault PhD , Isabelle Pontais PhD , Vincent Enouf PhD , Christine Debeuret PharmD , Emma Bloch MSc , Juliette Paireau PhD , Prof Marie-Anne Rameix-Welti PhD , Michael White PhD , Gaëlle Baudemont MsC , Prof Bruno Lina PhD , Isabelle Parent du Châtelet MD , Jean-Sébastien Casalegno MD , Sophie Vaux PharmD , Prof Simon Cauchemez PhD","doi":"10.1016/S2352-4642(24)00143-3","DOIUrl":"10.1016/S2352-4642(24)00143-3","url":null,"abstract":"<div><h3>Background</h3><p>Respiratory syncytial virus (RSV) is a major cause of hospitalisations and deaths among infants worldwide. France was one of the first countries to implement a national programme (beginning on Sept 15, 2023) for administration of nirsevimab, a single-dose long-acting monoclonal antibody treatment, to infants born on or after Feb 6, 2023, to prevent lower respiratory tract infection caused by RSV. We aimed to estimate the effectiveness of nirsevimab and the number of hospitalisations averted in children younger than 24 months in real-world settings.</p></div><div><h3>Methods</h3><p>In this modelling study, we developed an age-structured deterministic model characterising RSV transmission as well as plausible scenarios for the administration of nirsevimab doses based on maternity ward and community pharmacy supply data. We retrospectively estimated nirsevimab effectiveness in infants younger than 24 months during the 2023–24 RSV season in France (excluding overseas territories) and the number of averted hospitalisations for RSV bronchiolitis occurring after emergency department visits, by calibrating the model to hospital and virological surveillance data from Aug 21, 2017, to Feb 4, 2024, alongside serological data from a previous cross-sectional study. To assess the robustness of our estimates, we conducted sensitivity analyses in which we modified our assumptions about the number of doses administered, the reconstruction of the number of RSV-associated hospitalisations for bronchiolitis, the duration of maternal and post-infection immunity to RSV, and the number of contacts in children aged 0–2 months.</p></div><div><h3>Findings</h3><p>We estimated that nirsevimab administration prevented 5800 (95% credible interval 3700–7800) RSV-associated hospitalisations for bronchiolitis after emergency department visits among children younger than 24 months, including 4200 (2900–5600) hospitalisations among those aged 0–2 months, between Sept 15, 2023 (the date nirsevimab was introduced), and Feb 4, 2024—a 23% (16–30) reduction in the total number of hospitalisations and a 35% (25–44) reduction in the 0–2 months age group, compared with the scenario without administration. In our baseline scenario, in which we estimated that 215 000 doses of nirsevimab were administered by Jan 31, 2024, the estimated effectiveness against RSV-associated hospitalisations for bronchiolitis was 73% (61–84), corresponding to one hospitalisation averted for every 39 (26–54) doses administered. In sensitivity analyses, nirsevimab remained effective against RSV-associated hospitalisations for bronchiolitis after emergency department attendance.</p></div><div><h3>Interpretation</h3><p>Our findings show that nirsevimab administration campaigns could effectively reduce the RSV-related hospital burden of bronchiolitis in children younger than 24 months.</p></div><div><h3>Funding</h3><p>European Commission, Laboratoire d'Excellence Integrative Biology of Eme","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"8 10","pages":"Pages 721-729"},"PeriodicalIF":19.9,"publicationDate":"2024-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142116705","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Real-world effectiveness of nirsevimab immunisation against bronchiolitis in infants: a case–control study in Paris, France","authors":"Prof Ricardo Carbajal PhD , Prof Pierre-Yves Boelle PhD , Aurélie Pham PhD , Yoann Chazette , Mathilde Schellenberger MD , Clara Weil , Anne-Sophie Colas MD , Thibault Lecarpentier MD , Aurélie Schnuriger PhD , Romain Guedj PhD , Prof Mathie Lorrot PhD , Prof Harriet Corvol PhD , Maxime Enault MD","doi":"10.1016/S2352-4642(24)00171-8","DOIUrl":"10.1016/S2352-4642(24)00171-8","url":null,"abstract":"<div><h3>Background</h3><p>Respiratory syncytial virus (RSV) is the most common cause of bronchiolitis in infants. Nirsevimab, an RSV-neutralising monoclonal antibody, was approved for use in the EU in 2022, and a national immunisation campaign began in France in September, 2023. We aimed to assess the effectiveness of nirsevimab in reducing paediatric emergency department visits (and subsequent hospitalisations) for all-cause bronchiolitis and RSV-associated bronchiolitis.</p></div><div><h3>Methods</h3><p>In this case–control study in a paediatric emergency department in Paris, France, we included all infants aged 12 months or younger who attended the department between Oct 14, 2023, and Feb 29, 2024, and whose nirsevimab status was known. Infants were classed as cases if they had all-cause bronchiolitis; all other infants were classed as controls. The primary outcome was the effectiveness of nirsevimab against paediatric emergency department visits for all-cause bronchiolitis during the 2023–24 RSV season. Secondary outcomes were paediatric emergency department visits for RSV-associated bronchiolitis; hospitalisations for all-cause bronchiolitis, RSV-associated bronchiolitis, and severe RSV-associated bronchiolitis requiring supplemental oxygen or feeding by nasogastric tube; and severe RSV-associated bronchiolitis requiring admission to the paediatric intensive care unit. Effectiveness estimates were adjusted for age, week of paediatric emergency department visit, and sex.</p></div><div><h3>Findings</h3><p>Our study included 2786 infants, 864 with all-cause bronchiolitis (cases) and 1922 without bronchiolitis (controls). 178 (21%) of the 864 cases had received nirsevimab, and 305 (35%) cases were hospitalised for all-cause bronchiolitis. 200 (72%) of the 277 cases tested for RSV were positive, of whom 22 (11%) had received nirsevimab. 701 (36%) of 1922 infants in the control group had received nirsevimab. The effectiveness of nirsevimab against paediatric emergency department visits for all-cause bronchiolitis was 47% (95% CI 33–58). Nirsevimab effectiveness was 83% (71–90) against paediatric emergency department visits for RSV-associated bronchiolitis, 59% (42–71) against hospitalisations for all-cause bronchiolitis, 83% (72–90) against hospitalisations for RSV-associated bronchiolitis (91% [78−96] against those necessitating supplement oxygen and 88% [74−95] against those necessitating feeding via a nasogastric tube). Nirsevimab did not significantly reduce admissions to the paediatric intensive care unit (67% [95% CI –100 to 95]).</p></div><div><h3>Interpretation</h3><p>During the first French national immunisation campaign, a single dose of nirsevimab effectively reduced paediatric emergency department visits (both all-cause visits and visits related to RSV-associated bronchiolitis) and subsequent hospitalisations.</p></div><div><h3>Funding</h3><p>None.</p></div>","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"8 10","pages":"Pages 730-739"},"PeriodicalIF":19.9,"publicationDate":"2024-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142116708","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Fatima Elbasri Abuelgasim Mohammed, Saad Chaibi, Elaine Tan Su Yin, Daniel Wainstock
{"title":"The role of youth in fighting neglected tropical diseases","authors":"Fatima Elbasri Abuelgasim Mohammed, Saad Chaibi, Elaine Tan Su Yin, Daniel Wainstock","doi":"10.1016/S2352-4642(24)00230-X","DOIUrl":"10.1016/S2352-4642(24)00230-X","url":null,"abstract":"","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"8 11","pages":"Pages 786-787"},"PeriodicalIF":19.9,"publicationDate":"2024-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142006253","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jette J Bakhuizen MD , Freerk van Dijk MSc , Marco J Koudijs PhD , Reno S Bladergroen BSc , Sebastian B B Bon MD , Saskia M J Hopman PhD , Lennart A Kester PhD , Mariëtte E G Kranendonk PhD , Jan L C Loeffen PhD , Stephanie E Smetsers PhD , Edwin Sonneveld PhD , Melissa Tachdjian BA , Evelien de Vos-Kerkhof PhD , Catherine Goudie MD , Prof Johannes H M Merks PhD , Prof Roland P Kuiper PhD , Marjolijn C J Jongmans PhD
{"title":"Comparison of clinical selection-based genetic testing with phenotype-agnostic extensive germline sequencing to diagnose genetic predisposition in children with cancer: a prospective diagnostic study","authors":"Jette J Bakhuizen MD , Freerk van Dijk MSc , Marco J Koudijs PhD , Reno S Bladergroen BSc , Sebastian B B Bon MD , Saskia M J Hopman PhD , Lennart A Kester PhD , Mariëtte E G Kranendonk PhD , Jan L C Loeffen PhD , Stephanie E Smetsers PhD , Edwin Sonneveld PhD , Melissa Tachdjian BA , Evelien de Vos-Kerkhof PhD , Catherine Goudie MD , Prof Johannes H M Merks PhD , Prof Roland P Kuiper PhD , Marjolijn C J Jongmans PhD","doi":"10.1016/S2352-4642(24)00144-5","DOIUrl":"10.1016/S2352-4642(24)00144-5","url":null,"abstract":"<div><h3>Background</h3><p>Germline data have become widely available in paediatric oncology since the introduction of paired tumour-germline sequencing. To guide best practice in cancer predisposition syndrome (CPS) diagnostics, we aimed to assess the diagnostic yield of extensive germline analysis compared with clinical selection-based genetic testing among all children with cancer.</p></div><div><h3>Methods</h3><p>In this prospective diagnostic study, all children (aged 0–19 years) with newly diagnosed neoplasms treated in the Netherlands national centre, the Princess Máxima Center for Pediatric Oncology (Utrecht, Netherlands), between June 1, 2020, and July 31, 2022, were offered two approaches to identify CPSs. In a phenotype-driven approach, paediatric oncologists used the McGill Interactive Pediatric OncoGenetic Guidelines tool to select children for referral to a clinical geneticist, and for genetic testing. In a phenotype-agnostic approach, CPS gene panel sequencing (143 genes) was offered to all children. In children declining the research CPS gene panel, 49 CPS genes were still analysed as part of routine diagnostics by the pathologist. Children with a causative CPS identified before neoplasm diagnosis were excluded. The primary objective was to compare the number and type of patients diagnosed with a CPS between the two approaches.</p></div><div><h3>Findings</h3><p>1052 children were eligible for this study, of whom 733 (70%) completed both the phenotype-driven approach and received phenotype-agnostic CPS gene panel sequencing (143 genes n=600; 49 genes n=133). In 53 children, a CPS was identified: 14 (26%) were diagnosed by the phenotype-driven approach only, 22 (42%) by CPS gene sequencing only, and 17 (32%) by both approaches. In 27 (51%) of the 53 children, the identified CPS was considered causative for the child's neoplasm. Only one (4%) of the 27 causative CPSs was missed by the phenotype-driven approach and was identified solely by phenotype-agnostic CPS gene sequencing. In 26 (49%) children, a CPS with uncertain causality was identified, including 14 adult-onset CPSs. The CPSs with uncertain causality were mainly detected by the phenotype-agnostic approach (21 [81%] of 26).</p></div><div><h3>Interpretation</h3><p>Phenotype-driven genetic testing and phenotype-agnostic CPS gene panel sequencing were complementary. The phenotype-driven approach identified the most causative CPSs. CPS gene panel sequencing identified additional CPSs, many of those with uncertain causality, but some with clinical utility. We advise clinical evaluation for CPSs in all children with neoplasms. Phenotype-agnostic testing of all CPS genes is preferably conducted only in research settings and should be paired with counseling.</p></div><div><h3>Funding</h3><p>Stichting Kinderen Kankervrij.</p></div>","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"8 10","pages":"Pages 751-761"},"PeriodicalIF":19.9,"publicationDate":"2024-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142006252","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Michael J Carter MRCPCH DPhil , Enitan D Carrol MD MBChB , Suchitra Ranjit MD , Rebeca Mozun MD PhD , Niranjan Kissoon MBBS , R Scott Watson MD MPH , Prof Luregn J Schlapbach MD PhD
{"title":"Susceptibility to childhood sepsis, contemporary management, and future directions","authors":"Michael J Carter MRCPCH DPhil , Enitan D Carrol MD MBChB , Suchitra Ranjit MD , Rebeca Mozun MD PhD , Niranjan Kissoon MBBS , R Scott Watson MD MPH , Prof Luregn J Schlapbach MD PhD","doi":"10.1016/S2352-4642(24)00141-X","DOIUrl":"10.1016/S2352-4642(24)00141-X","url":null,"abstract":"<div><p>Sepsis disproportionally affects children across all health-care settings and is one of the leading causes of morbidity and mortality in neonatal and paediatric age groups. As shown in the first paper in this Series, the age-specific incidence of sepsis is highest during the first years of life, before approaching adult incidence rates during adolescence. In the second paper in this Series, we focus on the unique susceptibility of paediatric patients to sepsis and how the underlying dysregulated host response relates to developmental aspects of children's immune system, genetic, perinatal, and environmental factors, and comorbidities and socioeconomic determinants of health, which often differ between children and adults. State-of-the-art clinical management of paediatric sepsis is organised around three treatment pillars—diagnosis, early resuscitation, and titration of advanced care—and we examine available treatment guidelines and the limitations of their supporting evidence. Serious evidence gaps remain in key areas of paediatric sepsis care, especially surrounding recognition, common interventions, and survivor support, and to this end we offer a research roadmap for the next decade that could accelerate targeted diagnostics and personalised use of immunomodulation. However, improving outcomes for children with sepsis relies fundamentally on systematic quality improvement in both recognition and treatment, which is the theme of the third paper in this Series. Digital health, as shown in the fourth and final paper of this Series, holds promising potential in breaking down the barriers that hinder progress in paediatric sepsis care and, ultimately, global child health.</p></div>","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"8 9","pages":"Pages 682-694"},"PeriodicalIF":19.9,"publicationDate":"2024-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S235246422400141X/pdfft?md5=b4ad6b1a63e506179c07ee5b706be9e3&pid=1-s2.0-S235246422400141X-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141979676","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jiangwei Sun , Henrik Arnell , Jonas F Ludvigsson , Ola Olén
{"title":"Rare and severe adverse events in children with inflammatory bowel disease","authors":"Jiangwei Sun , Henrik Arnell , Jonas F Ludvigsson , Ola Olén","doi":"10.1016/S2352-4642(24)00176-7","DOIUrl":"10.1016/S2352-4642(24)00176-7","url":null,"abstract":"","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"8 9","pages":"Page e8"},"PeriodicalIF":19.9,"publicationDate":"2024-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141979870","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}