Journal of Pediatrics最新文献

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Therapeutic Hypothermia in Low- and Middle-Income Countries: A Systematic Review and Meta-Analysis 低收入和中等收入国家的治疗性低温:一项系统回顾和荟萃分析。
IF 3.5 2区 医学
Journal of Pediatrics Pub Date : 2025-08-22 DOI: 10.1016/j.jpeds.2025.114793
Henry C. Lee MD , Daniela T. Costa-Nobre MD, PhD , Anup C. Katheria MD , Richard Mausling MBChB , Firdose L. Nakwa MBBCh , Georg M. Schmölzer MD, PhD , Gary M. Weiner MD , Helen G. Liley MBChB
{"title":"Therapeutic Hypothermia in Low- and Middle-Income Countries: A Systematic Review and Meta-Analysis","authors":"Henry C. Lee MD ,&nbsp;Daniela T. Costa-Nobre MD, PhD ,&nbsp;Anup C. Katheria MD ,&nbsp;Richard Mausling MBChB ,&nbsp;Firdose L. Nakwa MBBCh ,&nbsp;Georg M. Schmölzer MD, PhD ,&nbsp;Gary M. Weiner MD ,&nbsp;Helen G. Liley MBChB","doi":"10.1016/j.jpeds.2025.114793","DOIUrl":"10.1016/j.jpeds.2025.114793","url":null,"abstract":"<div><h3>Objective</h3><div>To evaluate therapeutic hypothermia (TH) for moderate or severe hypoxic-ischemic encephalopathy in low- and middle-income countries.</div></div><div><h3>Study design</h3><div>Medline, Embase, and CENTRAL were searched until September 19, 2024. Screening, article selection, bias assessment using Cochrane RoB2, and data extraction were performed. Meta-analyses of randomized controlled trials were performed for the composite primary outcome of death or moderate to severe neurodevelopmental impairment (NDI) at 18-24 months, and secondary outcomes were followed by certainty of evidence evaluation using Grading of Recommendations, Assessment, Development and Evaluations.</div></div><div><h3>Results</h3><div>From 804 records retrieved, 18 randomized controlled trials were included. Twelve included only term infants. All used TH protocols and provided neonatal intensive care, but methods of TH varied. Comparing TH with no TH, for the primary outcome of death or NDI at 18-24 months, benefit or harm could not be excluded (relative risk [RR] 0.63; 95% CI 0.38-1.04; <em>P</em> = .07; I<sup>2</sup> 80%, 4 studies, 717 infants; moderate certainty). For death by 18 to 24 months, benefit or harm could not be excluded (RR 0.81, 95% CI 0.43-1.52; <em>P</em> = .51; I<sup>2</sup> 67%, 4 studies, 722 infants; moderate certainty). In TH-treated infants, NDI at 18-24 months was possibly lower (RR 0.51; 95% CI 0.35-0.76; <em>P</em> &lt; .0001; I<sup>2</sup> 0%, 4 studies, 511 infants, low certainty).</div></div><div><h3>Conclusions</h3><div>In low- and middle-income countries, in hospitals using defined protocols and having capacity for intensive care and follow-up, TH has possible benefit for infants ≥37 weeks gestational age for important secondary outcomes, including NDI.</div></div>","PeriodicalId":54774,"journal":{"name":"Journal of Pediatrics","volume":"288 ","pages":"Article 114793"},"PeriodicalIF":3.5,"publicationDate":"2025-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144979225","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Defining Child Health Research to Guide Equitable Federal Appropriations. 定义儿童健康研究以指导公平的联邦拨款。
IF 3.5 2区 医学
Journal of Pediatrics Pub Date : 2025-08-21 DOI: 10.1016/j.jpeds.2025.114794
Terence S Dermody, Susan L Furth, Karen F Murray, Brian Sims, Nicholas P Manetto
{"title":"Defining Child Health Research to Guide Equitable Federal Appropriations.","authors":"Terence S Dermody, Susan L Furth, Karen F Murray, Brian Sims, Nicholas P Manetto","doi":"10.1016/j.jpeds.2025.114794","DOIUrl":"10.1016/j.jpeds.2025.114794","url":null,"abstract":"","PeriodicalId":54774,"journal":{"name":"Journal of Pediatrics","volume":" ","pages":"114794"},"PeriodicalIF":3.5,"publicationDate":"2025-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144979118","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Self-Inflicted Suction Bruise: A Case in Which Child Protective Services Reporting Could Be Deferred 自己造成的吸挫伤:一个儿童保护服务报告可以推迟的案例。
IF 3.5 2区 医学
Journal of Pediatrics Pub Date : 2025-08-21 DOI: 10.1016/j.jpeds.2025.114785
Cheryl Antal MD, MPH, C. Rashaan Ford MD, Daniel M. Lindberg MD
{"title":"Self-Inflicted Suction Bruise: A Case in Which Child Protective Services Reporting Could Be Deferred","authors":"Cheryl Antal MD, MPH,&nbsp;C. Rashaan Ford MD,&nbsp;Daniel M. Lindberg MD","doi":"10.1016/j.jpeds.2025.114785","DOIUrl":"10.1016/j.jpeds.2025.114785","url":null,"abstract":"","PeriodicalId":54774,"journal":{"name":"Journal of Pediatrics","volume":"288 ","pages":"Article 114785"},"PeriodicalIF":3.5,"publicationDate":"2025-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144979051","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Missed Opportunities: Single-Center Insights into Prenatal Cardiac Palliative Care 错失的机会:单中心洞察产前心脏姑息治疗。
IF 3.5 2区 医学
Journal of Pediatrics Pub Date : 2025-08-21 DOI: 10.1016/j.jpeds.2025.114784
Hannah L. Tredway MD , Katie M. Moynihan MBBS , Hadley Bloomhardt MD , Jennifer M. Snaman MD, MS , Nicholas Purol LICSW , David M. Hoganson MD , Christina Ronai MD, MSEd
{"title":"Missed Opportunities: Single-Center Insights into Prenatal Cardiac Palliative Care","authors":"Hannah L. Tredway MD ,&nbsp;Katie M. Moynihan MBBS ,&nbsp;Hadley Bloomhardt MD ,&nbsp;Jennifer M. Snaman MD, MS ,&nbsp;Nicholas Purol LICSW ,&nbsp;David M. Hoganson MD ,&nbsp;Christina Ronai MD, MSEd","doi":"10.1016/j.jpeds.2025.114784","DOIUrl":"10.1016/j.jpeds.2025.114784","url":null,"abstract":"<div><h3>Objective</h3><div>To determine whether prenatal palliative care (PC) utilization for patients with critical congenital heart disease (CCHD) at our institution is less than 10%, in spite of improvements in shared decision-making, communication, and family stress afforded by subspecialty PC consultation.</div></div><div><h3>Study design</h3><div>A retrospective study of prenatal CCHD diagnoses at a tertiary center from January 2020 to December 2023. Initial hospitalization characteristics, presence and timing of PC, and survival or mechanism of death were collected. Early PC was defined as occurring prenatally or more than 30 days prior to death. Clinical characteristics were compared between patients with early, late, or no PC consultation.</div></div><div><h3>Results</h3><div>CCHD was diagnosed prenatally in 506 patients. Only 12% (n = 61) had a PC consultation, with 4 performed prenatally. Compared with those who received late or no PC, early PC consultation occurred more frequently for patients with genetic diagnoses (61% [n = 27] vs 35% [n = 6] vs 26% [n = 117], <em>P</em> &lt; .001) or noncardiac congenital anomalies (61% [n = 27] vs 29% [n = 5] vs 35% [n = 154], <em>P</em> &lt; .001). Of patients undergoing the highest complexity neonatal surgeries, 18% (n = 41) had subspecialty PC consultation. Overall, 12% (n = 60) of these patients died.</div></div><div><h3>Conclusions</h3><div>Patients with CCHD experience significant morbidity and mortality. Given the underutilization of subspecialty PC upon prenatal diagnosis of CCHD, implementation of routine PC consultation for high-risk patients could enhance PC involvement in this cohort.</div></div>","PeriodicalId":54774,"journal":{"name":"Journal of Pediatrics","volume":"287 ","pages":"Article 114784"},"PeriodicalIF":3.5,"publicationDate":"2025-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144979113","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Probiotic Receipt and Neurodevelopmental Outcomes of Infants <29 Weeks' Gestation: A Cohort Study <29周妊娠婴儿的益生菌摄入与神经发育结局:一项队列研究。
IF 3.5 2区 医学
Journal of Pediatrics Pub Date : 2025-08-20 DOI: 10.1016/j.jpeds.2025.114783
Prakesh S. Shah MD, MSc , Nicole Bando PhD , Seungwoo Lee MSc , Kamini Raghuram MD, MSc , Marc Beltempo MD, MSc , Carlos Fajardo MD , Valérie Bertelle MD , Linh G. Ly MD , Rudaina Banihani MD , Belal N. Alshaikh MD, MSc , Canadian Preterm Birth Network, Canadian Neonatal Network and Canadian Neonatal Follow-Up Network Investigators
{"title":"Probiotic Receipt and Neurodevelopmental Outcomes of Infants <29 Weeks' Gestation: A Cohort Study","authors":"Prakesh S. Shah MD, MSc ,&nbsp;Nicole Bando PhD ,&nbsp;Seungwoo Lee MSc ,&nbsp;Kamini Raghuram MD, MSc ,&nbsp;Marc Beltempo MD, MSc ,&nbsp;Carlos Fajardo MD ,&nbsp;Valérie Bertelle MD ,&nbsp;Linh G. Ly MD ,&nbsp;Rudaina Banihani MD ,&nbsp;Belal N. Alshaikh MD, MSc ,&nbsp;Canadian Preterm Birth Network, Canadian Neonatal Network and Canadian Neonatal Follow-Up Network Investigators","doi":"10.1016/j.jpeds.2025.114783","DOIUrl":"10.1016/j.jpeds.2025.114783","url":null,"abstract":"<div><h3>Objective</h3><div>To examine neurodevelopment and growth following probiotic receipt in neonatal intensive care units.</div></div><div><h3>Study design</h3><div>A national, population-representative, multicenter, retrospective cohort study was conducted on children born at &lt;29 weeks' gestation between January 1, 2014, and December 31, 2020, from 21 neonatal units in Canada who survived and then underwent follow-up between ages 18 and 30 months. Children who received vs did not receive probiotics were compared for the primary outcome of significant neurodevelopmental impairment, defined as any of the following: Bayley Scales of Infant and Toddler Development-III cognitive, language, or motor score &lt;70; cerebral palsy with Gross Motor Function Classification System III-V; hearing aid or cochlear implant; or bilateral visual impairment. Equivalence analyses utilizing a 4% margin for outcomes between groups were conducted using logistic regression and propensity score-matched analyses to calculate adjusted risk difference (RD) and CIs.</div></div><div><h3>Results</h3><div>A total of 2749 children in the probiotics group (median gestation 26 weeks and birth weight 920 grams) and 2442 in the no probiotics group (median gestation 26 weeks and birth weight 890 grams) were evaluated. Baseline characteristics were similar between groups. For the outcome of significant neurodevelopmental impairment, logistic regression analyses indicated that probiotics were equivalent to no probiotics (adjusted RD −0.23%, 95% CI: 2.61%, 2.15%), whereas propensity score-matched analyses (1474 pairs of children) indicated probiotics were nonequivalent and potentially superior (adjusted RD −4.01%, 95% CI: 6.92%, −1.01%). Results of the majority of secondary outcomes were similar, including growth outcomes.</div></div><div><h3>Conclusions</h3><div>Probiotics were not associated with harm in neurodevelopment and growth of surviving preterm neonates and may be potentially superior to no probiotics for neurodevelopment and growth among surviving preterm neonates &lt;29 weeks' gestation.</div></div>","PeriodicalId":54774,"journal":{"name":"Journal of Pediatrics","volume":"287 ","pages":"Article 114783"},"PeriodicalIF":3.5,"publicationDate":"2025-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144979100","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Chair's Report: A New Twist on an Old Tradition 主席报告:旧传统的新转折。
IF 3.5 2区 医学
Journal of Pediatrics Pub Date : 2025-08-19 DOI: 10.1016/j.jpeds.2025.114781
Aditi J. Reis MD, Yonairy A. Collado MD , Martha E. Stewart MD , Krishna A. Trivedi MD , Aaron Altman MD, PhD , Catherine Nettesheim MD , Maeve A. Reidy MB BCh BAO, MRCPI , David M. Roth MD, MSPH , Lindsey J. Narron BA , Terence S. Dermody MD
{"title":"Chair's Report: A New Twist on an Old Tradition","authors":"Aditi J. Reis MD,&nbsp;Yonairy A. Collado MD ,&nbsp;Martha E. Stewart MD ,&nbsp;Krishna A. Trivedi MD ,&nbsp;Aaron Altman MD, PhD ,&nbsp;Catherine Nettesheim MD ,&nbsp;Maeve A. Reidy MB BCh BAO, MRCPI ,&nbsp;David M. Roth MD, MSPH ,&nbsp;Lindsey J. Narron BA ,&nbsp;Terence S. Dermody MD","doi":"10.1016/j.jpeds.2025.114781","DOIUrl":"10.1016/j.jpeds.2025.114781","url":null,"abstract":"","PeriodicalId":54774,"journal":{"name":"Journal of Pediatrics","volume":"286 ","pages":"Article 114781"},"PeriodicalIF":3.5,"publicationDate":"2025-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144979077","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Glucagon-Like Peptide-1-Receptor Agonist Treatment of Obesity: Is It Cost-Effective and Equitable? glp1受体激动剂治疗肥胖:是否具有成本效益和公平性?
IF 3.5 2区 医学
Journal of Pediatrics Pub Date : 2025-08-18 DOI: 10.1016/j.jpeds.2025.114779
Lauren Hertzer BS , Robert M. Siegel MD, MS
{"title":"Glucagon-Like Peptide-1-Receptor Agonist Treatment of Obesity: Is It Cost-Effective and Equitable?","authors":"Lauren Hertzer BS ,&nbsp;Robert M. Siegel MD, MS","doi":"10.1016/j.jpeds.2025.114779","DOIUrl":"10.1016/j.jpeds.2025.114779","url":null,"abstract":"","PeriodicalId":54774,"journal":{"name":"Journal of Pediatrics","volume":"287 ","pages":"Article 114779"},"PeriodicalIF":3.5,"publicationDate":"2025-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144979086","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Corrigendum to Birth Weight and Weight Changes from Infancy to Early Childhood as Predictors of Body Mass Index in Adolescence [J Pediatr 222 (2020) 120-126.e3] 新生儿出生体重与青少年体重指数的相关性研究[J].中华儿科杂志,2012(5):379 - 379。
IF 3.5 2区 医学
Journal of Pediatrics Pub Date : 2025-08-16 DOI: 10.1016/j.jpeds.2025.114759
Katharina Stock , Rajini Nagrani , Nina Gande , Benoit Bernar , Anna Staudt , Peter Willeit , Ralf Geiger , Michael Knoflach , Ursula Kiechl-Kohlendorfer , Early Vascular Aging (EVA) Study Group, Bernhard Winder , Carmen Reiter , Christina Burger , Christoph Hochmayr , Julia Klingenschmid , Julia Marxer , Mandy Asare , Manuela Bock-Bartl , Martina Kothmayer , Maximilian Bohl , Stefan Kiechl
{"title":"Corrigendum to Birth Weight and Weight Changes from Infancy to Early Childhood as Predictors of Body Mass Index in Adolescence [J Pediatr 222 (2020) 120-126.e3]","authors":"Katharina Stock ,&nbsp;Rajini Nagrani ,&nbsp;Nina Gande ,&nbsp;Benoit Bernar ,&nbsp;Anna Staudt ,&nbsp;Peter Willeit ,&nbsp;Ralf Geiger ,&nbsp;Michael Knoflach ,&nbsp;Ursula Kiechl-Kohlendorfer ,&nbsp;Early Vascular Aging (EVA) Study Group,&nbsp;Bernhard Winder ,&nbsp;Carmen Reiter ,&nbsp;Christina Burger ,&nbsp;Christoph Hochmayr ,&nbsp;Julia Klingenschmid ,&nbsp;Julia Marxer ,&nbsp;Mandy Asare ,&nbsp;Manuela Bock-Bartl ,&nbsp;Martina Kothmayer ,&nbsp;Maximilian Bohl ,&nbsp;Stefan Kiechl","doi":"10.1016/j.jpeds.2025.114759","DOIUrl":"10.1016/j.jpeds.2025.114759","url":null,"abstract":"","PeriodicalId":54774,"journal":{"name":"Journal of Pediatrics","volume":"285 ","pages":"Article 114759"},"PeriodicalIF":3.5,"publicationDate":"2025-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144862709","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
BNT162b2 XBB.1.5-Adapted Single Dose Vaccine Uptake and Effectiveness in Children Aged 5-17 Years Using Linked Claims and Vaccine Registries in California and Louisiana xbb .1.5-加利福尼亚州和路易斯安那州5-17岁儿童适应单剂疫苗的摄取和有效性。
IF 3.5 2区 医学
Journal of Pediatrics Pub Date : 2025-08-14 DOI: 10.1016/j.jpeds.2025.114778
Kathleen M. Andersen PhD, MSc , Maria D. McColgan MD , Jazmine S. Mateus MPH , Tiange Yu MPH , Anan Zhou MPH , Laura Puzniak PhD , Santiago M.C. Lopez MD
{"title":"BNT162b2 XBB.1.5-Adapted Single Dose Vaccine Uptake and Effectiveness in Children Aged 5-17 Years Using Linked Claims and Vaccine Registries in California and Louisiana","authors":"Kathleen M. Andersen PhD, MSc ,&nbsp;Maria D. McColgan MD ,&nbsp;Jazmine S. Mateus MPH ,&nbsp;Tiange Yu MPH ,&nbsp;Anan Zhou MPH ,&nbsp;Laura Puzniak PhD ,&nbsp;Santiago M.C. Lopez MD","doi":"10.1016/j.jpeds.2025.114778","DOIUrl":"10.1016/j.jpeds.2025.114778","url":null,"abstract":"<div><h3>Objective</h3><div>To assess BNT162b2 XBB.1.5-adapted COVID-19 vaccine uptake and effectiveness in children aged 5-17 years against emergency department (ED) encounters, urgent care (UC) visits, and hospital admissions.</div></div><div><h3>Study design</h3><div>We conducted a retrospective cohort study using HealthVerity claims tokenized to California and Louisiana state vaccine registries. Children were followed using a time-varying exposure definition, with vaccinated time ≥14 days after vaccination, from September 25, 2023 through March 31, 2024. Incidence and incidence rates were calculated for vaccinated and unvaccinated time. Time-dependent Cox models were used to estimate adjusted hazard ratios, with percent vaccine effectiveness calculated as (1 – hazard ratio)∗100.</div></div><div><h3>Results</h3><div>Of 2 449 261 children aged 5-17 years who met inclusion criteria, 34 389 (1.4%) received an XBB.1.5-adapted BNT162b2 vaccine. Vaccination uptake was higher in California than Louisiana and increased with age. Half (51.2%) of children who received the XBB.1.5-adapted vaccine had received a 2022-2023 season BA.4/5 bivalent COVID-19 vaccine. In contrast, the majority (66.0%) of XBB.1.5 unvaccinated children had not received any prior COVID-19 vaccine. For all measured outcomes, the XBB vaccinated children had significantly lower incidence rates compared with those who did not receive any XBB vaccine. There were no COVID-19 associated hospitalizations in the vaccinated group. Adjusted vaccine effectiveness among children aged 5-17 years was 63% (95% CI: 39%–77%) against COVID-19-associated ED/UC encounters with similar results stratified by age and among varying outcomes.</div></div><div><h3>Conclusion</h3><div>BNT162b2 XBB.1.5-adapted formulations provided significant protection in children aged 5-17 years against COVID-19 associated hospitalization and ED or UC visits.</div></div><div><h3>Clinical Trial Registration</h3><div><span><span>NCT06199934</span><svg><path></path></svg></span> (<span><span>https://clinicaltrials.gov/study/NCT06199934</span><svg><path></path></svg></span>).</div></div>","PeriodicalId":54774,"journal":{"name":"Journal of Pediatrics","volume":"286 ","pages":"Article 114778"},"PeriodicalIF":3.5,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144862708","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Growth Patterns in Shwachman-Diamond Syndrome: Findings from the North American Shwachman-Diamond Syndrome Registry Shwachman-Diamond综合征的生长模式:来自北美Shwachman-Diamond综合征登记处的发现。
IF 3.5 2区 医学
Journal of Pediatrics Pub Date : 2025-08-14 DOI: 10.1016/j.jpeds.2025.114780
Lois Schwarz BA , Leah Cheng MA, CCRP , Sarah Steltz MPH , Diana Schwarz BA , Elizabeth Korn BA , Sara Loveless RN, BSN, BMTCN , Richard Cooper BS , Claire Dusa BS , Lindsey Hornung MS , Jonathan C. Howell MD, PhD , Akiko Shimamura MD, PhD , Kasiani C. Myers MD , Jane Koo MD
{"title":"Growth Patterns in Shwachman-Diamond Syndrome: Findings from the North American Shwachman-Diamond Syndrome Registry","authors":"Lois Schwarz BA ,&nbsp;Leah Cheng MA, CCRP ,&nbsp;Sarah Steltz MPH ,&nbsp;Diana Schwarz BA ,&nbsp;Elizabeth Korn BA ,&nbsp;Sara Loveless RN, BSN, BMTCN ,&nbsp;Richard Cooper BS ,&nbsp;Claire Dusa BS ,&nbsp;Lindsey Hornung MS ,&nbsp;Jonathan C. Howell MD, PhD ,&nbsp;Akiko Shimamura MD, PhD ,&nbsp;Kasiani C. Myers MD ,&nbsp;Jane Koo MD","doi":"10.1016/j.jpeds.2025.114780","DOIUrl":"10.1016/j.jpeds.2025.114780","url":null,"abstract":"<div><h3>Objective</h3><div>To characterize growth patterns in individuals with Shwachman-Diamond syndrome (SDS) in North America by generating SDS-specific growth curves and assessing the impact of hematopoietic stem cell transplant (HSCT) and growth hormone (GH) on growth.</div></div><div><h3>Study design</h3><div>We conducted a retrospective cohort study of 127 subjects with confirmed biallelic Shwachman-Bodian-Diamond syndrome mutations enrolled on the North American SDS Registry. Height-for-age, weight-for-age, and body mass index (BMI)-for-age Z-scores were analyzed and compared with Centers for Disease Control and Prevention and World Health Organization reference populations. The effects of HSCT and GH therapy on growth velocity and final height outcomes were assessed.</div></div><div><h3>Results</h3><div>Individuals with SDS demonstrated significantly lower height-for-age Z-scores, with median adult height being approximately 7 cm shorter in females and 11 cm shorter in males compared with Centers for Disease Control and Prevention standards. Although weight-for-age was diminished, BMI-for-age trajectories were preserved. HSCT significantly reduced height-for-age Z-scores slope, and in a small subsample, GH therapy showed limited efficacy in improving final height.</div></div><div><h3>Conclusions</h3><div>This study of SDS across a diverse North American sample extends available growth data for this rare condition, confirming persistent short stature but normal BMI for age in SDS. HSCT was associated with a reduction in growth. No significant change in height velocity was observed with GH therapy. These data will inform clinical growth evaluation in SDS patients.</div></div>","PeriodicalId":54774,"journal":{"name":"Journal of Pediatrics","volume":"287 ","pages":"Article 114780"},"PeriodicalIF":3.5,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144862710","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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