Lancet Child & Adolescent Health最新文献

{"title":"Correction to Lancet Child Adolesc Health 2025; 9: 89–99","authors":"","doi":"10.1016/S2352-4642(25)00096-3","DOIUrl":"10.1016/S2352-4642(25)00096-3","url":null,"abstract":"","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"9 5","pages":"Page e13"},"PeriodicalIF":19.9,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143833150","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":"Treatment for paediatric respiratory syncytial virus infection","authors":"Eric A F Simões","doi":"10.1016/S2352-4642(25)00097-5","DOIUrl":"10.1016/S2352-4642(25)00097-5","url":null,"abstract":"","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"9 5","pages":"Pages 285-286"},"PeriodicalIF":19.9,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143833148","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":"Accelerating access to paediatric medicines: lessons learned from the Global Accelerator for Paediatric Formulations, a WHO-hosted network","authors":"Martina Penazzato PhD ,&nbsp;Asma Hafiz MSc ,&nbsp;Sébastien Morin PhD ,&nbsp;Farihah Malik PhD ,&nbsp;Bernadette Cappello MPH ,&nbsp;Jennifer Cohn MD ,&nbsp;Tim R Cressey PhD ,&nbsp;Patricia Fassinou MD ,&nbsp;Anthony J Garcia-Prats MD ,&nbsp;Luann Hatane MPhil ,&nbsp;Janice Soo Fern Lee MPharm ,&nbsp;Tiziana Masini PhD ,&nbsp;Atieno Ojoo MPH ,&nbsp;Theodore Ruel MD ,&nbsp;David Ruiz Villafranca PhD ,&nbsp;Marie Valentin PharmD ,&nbsp;John C Reeder PhD","doi":"10.1016/S2352-4642(25)00040-9","DOIUrl":"10.1016/S2352-4642(25)00040-9","url":null,"abstract":"<div><div>Despite progress in reducing neonatal and child mortality, access to age-appropriate medicines remains inequitable, particularly in low-income and middle-income countries. The Global Accelerator for Paediatric Formulations (GAP-f), a WHO-hosted network established in 2020, addresses these gaps by uniting 33 partners to promote innovation and access to child-friendly formulations. Phase 2 (2022–24) of GAP-f's work focused on therapeutic areas where innovation and access efforts often did not have stakeholder alignment and coordination of designing and implementing innovative clinical trial methodology, engaging with regulators to address systemic barriers, identifying novel technologies for safe and effective delivery, and collaborating across stakeholders for product roll out. Learnings from GAP-f work include the need to adapt prioritisation processes to diverse therapeutic areas and focus on high-impact areas based on unmet needs. Platform trials emerged as a promising tool to accelerate evidence generation but require guidance from regulators, collaboration among pharmaceutical companies, and operational challenges and funding constraints to be overcome. Incentivising paediatric formulation development remains crucial for small volume paediatric markets, and efforts are also needed to more proactively enhance, accelerate, and effectively match innovations to deliver medicines to children more efficiently. Even when products are successfully developed, political will and community engagement are essential to creating demand, supporting roll out, and ensuring equitable access and appropriate use in children. Dedicated funding and targeted programmes are crucial to drive systemic and sustainable change. GAP-f implementation lessons discussed in this Personal View will inform the future of this needed initiative, accelerating progress towards improved medicines for children.</div></div>","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"9 5","pages":"Pages 337-348"},"PeriodicalIF":19.9,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143833144","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":"Early childhood height, weight, and BMI development in children with monogenic obesity: a European multicentre, retrospective, observational study","authors":"Stefanie Zorn PhD ,&nbsp;Cornelis Jan de Groot PhD ,&nbsp;Stephanie Brandt-Heunemann PhD ,&nbsp;Julia von Schnurbein PhD ,&nbsp;Ozair Abawi PhD ,&nbsp;Rebecca Bounds PhD ,&nbsp;Lisa Ruck MD ,&nbsp;Blanca Guijo MD ,&nbsp;Gabriel Á Martos-Moreno PhD ,&nbsp;Clarisse Nicaise MD ,&nbsp;Sophie Courbage MD ,&nbsp;Margit Klehr-Martinelli PhD ,&nbsp;Prof Reiner Siebert MD ,&nbsp;Prof Béatrice Dubern PhD ,&nbsp;Prof Christine Poitou PhD ,&nbsp;Prof Karine Clément PhD ,&nbsp;Prof Jesús Argente PhD ,&nbsp;Prof Peter Kühnen PhD ,&nbsp;Prof Ismaa Sadaf Farooqi PhD ,&nbsp;Prof Martin Wabitsch PhD ,&nbsp;Prof Erica van den Akker MD","doi":"10.1016/S2352-4642(25)00065-3","DOIUrl":"10.1016/S2352-4642(25)00065-3","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Background&lt;/h3&gt;&lt;div&gt;Monogenic defects in the leptin-melanocortin pathway are associated with hyperphagia and severe, early-onset obesity. Early childhood growth patterns in height, weight, and BMI, might serve as phenotypic markers for specific genetic disorders; however, reliable data are scarce. This study aimed to evaluate the natural history of height, weight, and BMI in early childhood in a large European group of individuals with monogenic obesity.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Methods&lt;/h3&gt;&lt;div&gt;This multicentre observational study analysed height, weight, and BMI from birth to age 5 years in individuals diagnosed with biallelic (likely) pathogenic LEP, LEPR, POMC, PCSK1, or MC4R variants or monoallelic (likely) pathogenic &lt;em&gt;MC4R&lt;/em&gt; variants from six European centres (Berlin and Ulm, Germany; Cambridge, UK; Madrid, Spain; Paris, France; Rotterdam, Netherlands). All patient data up to May 31, 2022 were included in this analysis. All individuals had at least two height or weight measurements between birth and age 5 years. Early childhood growth trajectories were compared with those of control children with obesity without a known genetic cause, following a negative next-generation sequencing panel. Diagnostic performance of BMI as a predictor test for monogenic obesity was also evaluated.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Findings&lt;/h3&gt;&lt;div&gt;We included 147 individuals with monogenic obesity. From the age of 6 months onwards, children with biallelic variants (n=88, 55% female &lt;em&gt;vs&lt;/em&gt; 45% male) had substantially higher BMIs than those with monoallelic &lt;em&gt;MC4R&lt;/em&gt; variants (n=59, 53% female &lt;em&gt;vs&lt;/em&gt; 47% male) and control children (n=113, 59% female &lt;em&gt;vs&lt;/em&gt; 41% male). Children with biallelic &lt;em&gt;LEP, LEPR&lt;/em&gt;, and &lt;em&gt;MC4R&lt;/em&gt; variants showed a steep BMI increase during the first year of life, followed by a plateau until age 5 years, whereas those with biallelic &lt;em&gt;POMC&lt;/em&gt; variants did not plateau. Accelerated linear growth was only observed in children with biallelic &lt;em&gt;MC4R&lt;/em&gt; variants starting from age 1 year. The optimal BMI cut-off for distinguishing individuals with biallelic variants from control individuals was identified at age 2 years, with a test positivity cutoff of 24·0 kg/m&lt;sup&gt;2&lt;/sup&gt; (sensitivity: 0·96 [95% CI 0·89–1·00], specificity: 0·83 [0·74–0·90], AUC: 0·96 [0·91–0·99], p&lt;0·0001). However, BMI had poor diagnostic performance for monoallelic &lt;em&gt;MC4R&lt;/em&gt; variants.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Interpretation&lt;/h3&gt;&lt;div&gt;This study identified characteristic early childhood BMI trajectories for different forms of monogenic obesity. From age 6 months onwards, individuals with biallelic variants can be distinguished from those with monoallelic variants and common obesity. A BMI ≥24 kg/m&lt;sup&gt;2&lt;/sup&gt; at age 2 years had good diagnostic performance for biallelic variants, informing future recommendations for genetic screening for monogenic obesity.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Funding&lt;/h3&gt;&lt;div&gt;Federal Ministry of Education and Research as part ","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"9 5","pages":"Pages 297-305"},"PeriodicalIF":19.9,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143833151","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":"Correction to Lancet Child Adolesc Health 2025; 9: 292–94","authors":"","doi":"10.1016/S2352-4642(25)00101-4","DOIUrl":"10.1016/S2352-4642(25)00101-4","url":null,"abstract":"","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"9 5","pages":"Page e13"},"PeriodicalIF":19.9,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143833937","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":"BMI profiling for monogenic obesity testing","authors":"Vidhu V Thaker ,&nbsp;Dympna Gallagher","doi":"10.1016/S2352-4642(25)00095-1","DOIUrl":"10.1016/S2352-4642(25)00095-1","url":null,"abstract":"","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"9 5","pages":"Pages 284-285"},"PeriodicalIF":19.9,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143833147","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":"Centring the child in complex decision making","authors":"Giuliana C Antolovich,&nbsp;Ingrid Sutherland,&nbsp;Zoe McCallum,&nbsp;Monica S Cooper","doi":"10.1016/S2352-4642(25)00104-X","DOIUrl":"10.1016/S2352-4642(25)00104-X","url":null,"abstract":"","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"9 5","pages":"Pages 295-296"},"PeriodicalIF":19.9,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143833149","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":"Efficacy of interventions for the treatment of irritable bowel syndrome, functional abdominal pain—not otherwise specified, and abdominal migraine in children: a systematic review and network meta-analysis","authors":"Vasiliki Sinopoulou RD ,&nbsp;Jip Groen MD ,&nbsp;Prof Morris Gordon PhD ,&nbsp;Ed Mougey PhD ,&nbsp;James P Franciosi MD ,&nbsp;Tim G J de Meij PhD ,&nbsp;Merit M Tabbers PhD ,&nbsp;Prof Marc A Benninga PhD","doi":"10.1016/S2352-4642(25)00058-6","DOIUrl":"10.1016/S2352-4642(25)00058-6","url":null,"abstract":"<div><h3>Background</h3><div>Many treatments for abdominal pain-related disorders of gut–brain interaction (AP-DGBI) in children have been studied. We aimed to assess the efficacy and safety of all known treatment options for paediatric AP-DGBI.</div></div><div><h3>Methods</h3><div>For this systematic review and network meta-analysis, we searched Embase, MEDLINE, and CENTRAL databases from inception to Jan 16, 2025, for published randomised controlled trials. We included trials of any treatment for AP-DGBIs (irritable bowel syndrome, functional abdominal pain—not otherwise specified, and abdominal migraine, excluding functional dyspepsia) in children aged 4–18 years. We excluded randomised controlled trials that solely included children with functional dyspepsia, but we included studies in which children with functional dyspepsia were included alongside children with the other AP-DGBI diagnoses and outcome data could not be separated. Data extraction and quality appraisal were performed in duplicate. The primary outcome for this network meta-analysis was author-defined treatment success. Network meta-analysis methodology was used within a frequentist framework using multivariate meta-analysis and outcomes were assessed using the Grading of Recommendations, Assessment, Development and Evaluation methodology. Clinical relevance of effect sizes was interpreted according to consensus definitions.</div></div><div><h3>Findings</h3><div>Of 19 337 records identified through the database search, 155 records representing 91 original randomised controlled trials were included in the network meta-analysis: these 91 trials comprised 7226 participants (4119 females and 2673 males). 12 studies assessed dietary treatments (n=730), 25 assessed pharmacological treatments (n=2140), 23 assessed probiotic treatments (n=1762), and 35 assessed psychosocial treatments (n=2952). Two treatments were probably more effective for treatment success than control treatments (moderate certainty): hypnotherapy (risk ratio [RR] 4·99 [95% CI 2·15 to 11·57]; large effect size) and cognitive behavioural therapy (CBT; RR 1·99 [95% CI 1·33 to 2·98]; moderate effect size). All other treatments evaluated for treatment success were either not effective or the data were of very low certainty and thus no conclusions could be made.</div></div><div><h3>Interpretation</h3><div>Hypnotherapy and CBT show moderate certainty for treatment efficacy with clinically relevant effect sizes. No conclusions can be made about the other therapies and treatment success due to very low evidence certainty. Future randomised controlled trials should focus on improving the evidence certainty for those other therapies with regard to core AP-DGBI outcomes.</div></div><div><h3>Funding</h3><div>None.</div></div>","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"9 5","pages":"Pages 315-324"},"PeriodicalIF":19.9,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143833143","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":"Efficacy and safety of ziresovir in hospitalised infants aged 6 months or younger with respiratory syncytial virus infection in China: findings from a phase 3 randomised trial with 24-month follow-up","authors":"Han Zhang MD ,&nbsp;Prof Wei Zhao PhD ,&nbsp;Xiaoyan Zhang MD ,&nbsp;Lei Zhang MD ,&nbsp;Run Guo MD ,&nbsp;Han Huang MD ,&nbsp;Li Lin MD ,&nbsp;Feng Liu MD ,&nbsp;Haidan Chen MD ,&nbsp;Fangfang Shen MD ,&nbsp;Jinzhun Wu MD ,&nbsp;Xiaowen Huang MD ,&nbsp;Xiaoping Zhu PhD ,&nbsp;Feng Li PhD ,&nbsp;Gang Zou PhD ,&nbsp;Jason Chien MD ,&nbsp;Michael Humphries MD ,&nbsp;Prof Quan Lu MD ,&nbsp;Jim Z Wu PhD ,&nbsp;Prof Shunying Zhao PhD ,&nbsp;Chuangli Hao","doi":"10.1016/S2352-4642(25)00067-7","DOIUrl":"10.1016/S2352-4642(25)00067-7","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Background&lt;/h3&gt;&lt;div&gt;Respiratory syncytial virus (RSV) is a particularly dangerous infection in some populations, including very young infants. This study examined the efficacy and safety of ziresovir in hospitalised infants aged 6 months or younger with RSV infection.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Methods&lt;/h3&gt;&lt;div&gt;In this double-blind, randomised, placebo-controlled trial conducted across 28 hospitals in China, patients aged 1–24 months hospitalised for virologically confirmed RSV infection were randomly allocated (2:1) to receive ziresovir (10–40 mg by weight twice daily) or placebo orally for 5 days, with 2 years of follow-up. Patients were included if they had a Wang bronchiolitis clinical score (WBCS) of at least 5 at first dosing and were administered their first dose of study drug within 7 days of the onset of symptoms of RSV infection. In this prespecified subanalysis of patients aged 6 months and younger at randomisation, we analysed the primary endpoint (change from baseline in WBCS on day 3 [48 h post-baseline]) in the intention-to-treat infected (ITT-i) population (comprising patients who received at least one dose of study drug and who had PCR-confirmed RSV infection). Safety endpoints were assessed in all patients who received at least one dose. This study is registered with &lt;span&gt;&lt;span&gt;ClinicalTrials.gov&lt;/span&gt;&lt;svg&gt;&lt;path&gt;&lt;/path&gt;&lt;/svg&gt;&lt;/span&gt; (&lt;span&gt;&lt;span&gt;NCT04231968&lt;/span&gt;&lt;svg&gt;&lt;path&gt;&lt;/path&gt;&lt;/svg&gt;&lt;/span&gt;) and is completed.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Findings&lt;/h3&gt;&lt;div&gt;Participants were recruited from Sept 22, 2020, to Jan 19, 2022, and followed up to Feb 4, 2024. Among patients aged 6 months or younger, 188 participants (125 in the ziresovir group and 63 in the placebo group) received at least one dose of study drug and were included in the safety analysis, while the ITT-i population included 150 patients (100 in the ziresovir group and 50 in the placebo group). In the ziresovir group, 33 (26%) of 125 patients were female, 92 (74%) were male, mean age was 3·4 months (SD 1·4), and mean baseline WBCS was 6·8 (SD 1·7). In the placebo group, 15 (24%) of 63 patients were female, 48 (76%) were male, mean age was 3·3 months (1·5), and mean baseline WBCS was 6·9 (1·8). The least-squares mean change in WBCS from baseline to day 3 was −3·5 points (95% CI −3·9 to −3·1) with ziresovir versus −2·2 points (−2·8 to −1·7) with placebo (difference −1·2 [95% CI −1·9 to −0·6], p=0·0004). Drug-related treatment-emergent adverse events occurred in 22 (18%) of 125 patients who received ziresovir and seven (11%) of 63 patients who received placebo. No drug-related serious adverse events were observed and no deaths occurred.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Interpretation&lt;/h3&gt;&lt;div&gt;Ziresovir had a favourable safety profile and was associated with a significant clinical benefit during the treatment period compared with placebo in patients aged 6 months or younger.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Funding&lt;/h3&gt;&lt;div&gt;Shanghai Ark Biopharmaceutical, National Clinical Research Center for Respiratory","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"9 5","pages":"Pages 325-336"},"PeriodicalIF":19.9,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143833145","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":"Navigating the complexities of adolescent health and social media","authors":"Megan A Moreno ,&nbsp;Jenny Radesky ,&nbsp;Nicole Owings-Fonner","doi":"10.1016/S2352-4642(25)00041-0","DOIUrl":"10.1016/S2352-4642(25)00041-0","url":null,"abstract":"","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"9 6","pages":"Pages 365-367"},"PeriodicalIF":19.9,"publicationDate":"2025-04-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143822478","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}