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Tacrolimus in pediatric patients with Henoch-Schönlein purpura nephritis: Target concentration range. 他克莫司在小儿紫癜性肾炎Henoch-Schönlein患者中的作用:目标浓度范围。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-11-12 DOI: 10.1016/j.arcped.2025.09.002
Fashuang Li, Huiying Li, Linbo Li, Lilin Zhang

Objective: This study seeks to establish the therapeutic reference range for tacrolimus blood concentrations in pediatric patients diagnosed with Henoch-Schönlein purpura nephritis (HSPN) and to evaluate the factors influencing tacrolimus pharmacokinetics in this population.

Methods: Receiver operating characteristic (ROC) curve analysis was utilized to determine the optimal therapeutic threshold for tacrolimus concentration. Comparative analyses were conducted to assess clinical efficacy and adverse event profiles across four groups of tacrolimus concentrations [Group A(<3 ng·mL-1), Group B (3-<5 ng·mL-1), Group C (5-<10 ng·mL-1), and Group D (≥10 ng·mL-1)], thereby establishing a reference therapeutic range. Kaplan-Meier survival analysis was employed to evaluate differences in continuous remission survival rate and remission duration among four distinct concentration groups, validating the precision of the target trough concentration range. Additionally, multivariate linear regression analysis was performed to identify significant factors influencing tacrolimus concentrations.

Results: A cohort of 105 pediatric patients diagnosed with HSPN were enrolled in this study. After six months of tacrolimus therapy, the ROC curve analysis revealed that the tacrolimus trough concentration had significant diagnostic value for clinical efficacy, with an area under the curve (AUC) of 0.816 (P < 0.01), and an optimal cutoff value of 3.02 ng·mL-1. Comparative analysis of clinical efficacy across four groups demonstrated that Group A exhibited significantly lower efficacy compared to the other groups. After 12 months of tacrolimus treatment, the diagnostic utility of the ROC curve was further confirmed, with an AUC of 0.798 (P > 0.05), indicating limited predictive performance. Although no significant differences in adverse event rates were observed among the four groups, Group D displayed a notably higher incidence of nephrotoxicity relative to the other groups. Multivariate linear regression analysis identified serum creatinine level as a significant determinant of tacrolimus concentration (P < 0.05).

Conclusion: In pediatric patients with HSPN in China, maintaining tacrolimus trough concentrations within the therapeutic range of 3-5 ng·mL-1 has been demonstrated to achieve optimal therapeutic efficacy.

目的:本研究旨在建立Henoch-Schönlein紫癜性肾炎(HSPN)患儿他克莫司血药浓度的治疗参考范围,并评价该人群中影响他克莫司药代动力学的因素。方法:采用受试者工作特征(ROC)曲线分析确定他克莫司的最佳治疗阈值。通过比较分析评估他克莫司浓度[A组(-1)、B组(3—1)、C组(5—1)和D组(≥10 ng·mL-1)]四组的临床疗效和不良事件概况,从而建立一个参考治疗范围。采用Kaplan-Meier生存分析评估4个不同浓度组持续缓解生存率和缓解持续时间的差异,验证目标谷浓度范围的准确性。此外,进行多元线性回归分析以确定影响他克莫司浓度的重要因素。结果:105名诊断为HSPN的儿科患者被纳入本研究。他克莫司治疗6个月后,ROC曲线分析显示他克莫司谷浓度对临床疗效具有显著的诊断价值,曲线下面积(AUC)为0.816 (P < 0.01),最佳截断值为3.02 ng·mL-1。四组临床疗效对比分析显示,A组疗效明显低于其他组。他克莫司治疗12个月后,ROC曲线的诊断效用进一步得到证实,AUC为0.798 (P < 0.05),表明预测效果有限。虽然四组间不良事件发生率无显著差异,但D组肾毒性发生率明显高于其他组。多因素线性回归分析发现血清肌酐水平是他克莫司浓度的重要决定因素(P < 0.05)。结论:在中国儿童HSPN患者中,维持他克莫司谷浓度在3-5 ng·mL-1治疗范围内可获得最佳治疗效果。
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引用次数: 0
The potential hidden dangers in household products for infant's immune system: Consequences for childcare. 家用产品对婴儿免疫系统的潜在隐患:对儿童保育的影响。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-11-05 DOI: 10.1016/j.arcped.2025.09.006
Christophe Dupont, Sandra Brancato, Rosan Meyer, Martin Chalumeau, Dominique Darmaun, Marie Laure Frelut, Dominique Guimbert, Alexandre Lapillonne, Arnaud de Luca, Sophie Nicklaus, Noel Peretti, Jean-Pierre Chouraqui

Background: The increase in allergic, autoimmune, and metabolic diseases observed in recent decades correlates with increased exposure to new chemicals that can damage the epithelial barrier, regardless of its location, the skin, respiratory, and digestive tract. Infants are often exposed to detergents and disinfectants from birth.

Objectives, methods, and setting: Published data indicate a potentially harmful impact of these chemicals on the epithelial barrier, which could promote allergies. The aim of this article is to review the available data on how these chemicals affect the epithelial barrier.

Results: We depict the likely harmful role of common household products in the development of allergies in children.

Conclusion: We suggest simple changes in everyday life aimed at reducing contact with these products in infants and young children.

背景:近几十年来观察到的过敏性、自身免疫性和代谢性疾病的增加与暴露于新的化学物质的增加有关,这些化学物质可以破坏上皮屏障,无论其位置、皮肤、呼吸和消化道如何。婴儿从出生起就经常接触清洁剂和消毒剂。目的、方法和环境:已发表的数据表明,这些化学物质对上皮屏障有潜在的有害影响,可能会促进过敏。本文的目的是回顾这些化学物质如何影响上皮屏障的现有数据。结果:我们描述了常见家用产品在儿童过敏发展中的可能有害作用。结论:我们建议在日常生活中进行简单的改变,以减少婴幼儿与这些产品的接触。
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引用次数: 0
Surgical management of 117 patients with halo-gravity traction: corrections, complications, and neurological risks 117例halo-gravity牵引患者的手术处理:矫正、并发症和神经系统风险:117例halo-gravity牵引患者的手术处理。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-11-01 DOI: 10.1016/j.arcped.2025.07.006
Lou Richard, Elie Saghbini, Gauthier Eloy, Reda Kabbaj, Clélia Thouement, Raphael Vialle, Mathilde Gaume

Background

Severe scoliosis, defined by a major curve exceeding 80°, requires surgical correction and is particularly prone to post-operative mechanical and neurological complications.

Objective

This study aimed to evaluate the corrections and complications obtained by halo-gravity traction (HGT) followed by single posterior approach surgery for severe scoliosis. The secondary objective was to identify any risk factors associated with neurological complications.

Methods

A single-center retrospective review of all patients operated on with severe scoliosis or kyphosis of any cause, by a single posterior approach after a period of HGT. Demographic data including age at surgery, sex, and body mass index were collected. Pre-traction, post-traction, postoperative major curve, and kyphosis angles were reported. Perioperative complications both during the traction period and postoperatively were recorded.

Results

117 patients with a mean age at surgery of 14.0 ± 2.0 years were included. The mean preoperative major curve was 90.7°±22.1° (from 80.1 to 147.2°) and the mean kyphosis was 60.8°± 31.70 (from 3.3 to 150.2°). The mean major curve after traction was 66.1°±15.2° (from 30.2 to 101.5°) and 33.7°±17.0° (from 0.2° to 104.4°) postoperatively, with a total correction of 69 %. During the HGT period, 8 (6.8 %) complications occurred: 3 superficial infections, 4 pin displacement, and 1 case of encopresis. Postoperatively, eight deficits were observed, with two being permanent. No risk factors for postoperative neurological complications were identified.

Conclusions

A 6-week period of HGT is associated with a low complication rate, and is effective to prepare severe scoliosis surgery, even for curvatures exceeding 120°
背景:重度脊柱侧凸,定义为主弯超过80°,需要手术矫正,尤其容易发生术后机械和神经系统并发症。目的:评价单纯后路手术后halo-gravity牵引治疗重度脊柱侧凸的矫正效果及并发症。次要目的是确定与神经系统并发症相关的任何危险因素。方法:单中心回顾性分析所有在HGT一段时间后采用单一后路手术治疗任何原因的严重脊柱侧凸或后凸的患者。统计数据包括手术年龄、性别和体重指数。报告牵引前、牵引后、术后主要曲线和后凸角。记录牵引期及术后围手术期并发症。结果:117例患者入组,平均手术年龄14.0±2.0岁。术前平均大弯为90.7°±22.1°(从80.1°到147.2°),平均后凸为60.8°±31.70°(从3.3°到150.2°)。牵引后平均主弯为66.1°±15.2°(从30.2°到101.5°),术后平均主弯为33.7°±17.0°(从0.2°到104.4°),总矫正率为69%。HGT期间共发生并发症8例(6.8%),其中浅表感染3例,针移位4例,穿刺1例。术后观察到8例缺损,其中2例为永久性缺损。未发现术后神经系统并发症的危险因素。结论:为期6周的HGT与低并发症发生率相关,并且可以有效地为重度脊柱侧凸手术做准备,即使曲率超过120°。
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引用次数: 0
Spectrum of DMD gene mutations in 507 patients: a retrospective genotype-phenotype study using next-generation sequencing 507例患者的DMD基因突变谱:使用新一代测序的回顾性基因型-表型研究
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-11-01 DOI: 10.1016/j.arcped.2025.07.002
Siyi Gan, Li Xu, Hongmei Liao, Liwen Wu

Background

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene, but comprehensive analyses of mutational patterns and clinical correlations remain limited.

Objective

To characterize the DMD mutational spectrum and its clinical implications in a large Asian cohort.

Methods & Settings

A retrospective genetic analysis of 507 unrelated male DMD/Becker muscular dystrophy patients was conducted at Hunan Children’s Hospital (2018–2021). Multiplex ligation-dependent probe amplification (MLPA) and next-generation sequencing (NGS) were employed for comprehensive variant detection. Variants were classified per ACMG/AMP guidelines.

Results

Exon deletions predominated (64.9%), followed by small mutations (26.0%) and duplications (9.1%). Nonsense mutations were the most frequent small variant (16.0%). Domain analysis revealed mutations clustered in the Central Rod Domain (CRD; exons 45–55). The Carboxy-Terminal Domain (CTD) was associated with the most severe phenotype (earliest loss of ambulation, P < 0.05 vs. Actin-Binding Domain [ABD] or CRD). Exon 53 skipping was applicable in 39.39% of eligible patients. De novo mutations accounted for 7.9% (40/507) of cases. Epilepsy comorbidity occurred in 1.34% (6/448) of DMD patients.

Conclusion

This study delineates the DMD mutational landscape in a Chinese cohort, highlighting domain-specific phenotypic severity (CTD > ABD > CRD) and identifying exon 53 as the primary therapeutic target for exon skipping. These findings enhance prognostic precision and guide targeted therapeutic strategies.
背景:杜氏肌营养不良症(DMD)是由DMD基因突变引起的,但对突变模式和临床相关性的综合分析仍然有限。目的:在一个大型亚洲队列中描述DMD突变谱及其临床意义。方法与背景:对湖南省儿童医院2018-2021年507例无亲缘关系的男性DMD/Becker肌营养不良患者进行回顾性遗传分析。多重连接依赖探针扩增(MLPA)和下一代测序(NGS)用于全面的变异检测。根据ACMG/AMP指南对变异进行分类。结果:外显子缺失居多(64.9%),其次为小突变(26.0%)和重复(9.1%)。无义突变是最常见的小变异(16.0%)。结构域分析显示突变聚集在中央杆结构域(CRD,外显子45-55)。羧基末端结构域(CTD)与最严重的表型相关(与肌动蛋白结合结构域[ABD]或CRD相比,最早丧失活动能力,P < 0.05)。外显子53省略适用于39.39%的符合条件的患者。新生突变占7.9%(40/507)。癫痫合并症发生率为1.34%(6/448)。结论:本研究描绘了中国队列中的DMD突变景观,突出了区域特异性表型严重程度(CTD > ABD > CRD),并确定外显子53是外显子跳变的主要治疗靶点。这些发现提高了预后的准确性,并指导了有针对性的治疗策略。
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引用次数: 0
Hepatitis in the time of pandemics: A comparative bibliometric analysis of early publications on MIS-C, acute hepatitis of unknown aetiology and Mpox 大流行时期的肝炎:misc、不明原因急性肝炎和Mpox早期出版物的比较文献计量学分析。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-11-01 DOI: 10.1016/j.arcped.2025.09.003
Alexandre Fabre MD, PhD , Paul Guerry PhD , Aurélie Morand MD, PhD

Background

The emergence of acute hepatitis of unknown etiology in the spring of 2022 was strikingly reminiscent of how multisystem inflammatory syndrome in children (MIS-C) had appeared two years earlier.

Objective

To carry out a bibliometric analysis of the early literature on acute hepatitis of unknown aetiology, and another coincident but non-pediatric disease, Mpox, in comparison with our previous findings on the early coverage of MIS-C.

Methods and Settings

A review of the literature was conducted in the MEDLINE database, on preprint servers, and among Reuters news articles. Articles were included if they had been published in 2022 and were specifically about acute hepatitis of unknown aetiology or Mpox.

Results

The first newspaper/non-academic journal article on acute hepatitis of unknown aetiology was published in week 14 and the weekly number rapidly peaked in week 16 before decreasing gradually down to zero in week 22. The first peer-reviewed article was published in week 15 and remained between 3 and 7 from weeks 17 to 23. Just under a quarter (23.5 %) of references in these articles were to health agency publications on acute hepatitis of unknown aetiology. Over 7-week periods beginning just before the corresponding health agency alerts, 25 articles were published on MIS-C (21 peer-reviewed and 4 preprints), 20 articles were published on acute hepatitis of unknown aetiology (18 peer-reviewed and 4 preprints), and 74 on Mpox (48 peer-reviewed and 26 preprints). The proportion of preprints was significantly associated with disease type (p = 0.03), as was the proportion of clinical studies, with values of 52 %, 10 %, and 18 %, respectively (p = 0.002). Many more non-academic articles were published on Mpox (N = 180) than on acute hepatitis of unknown aetiology (17) or MIS-C (24).

Conclusion

Although Mpox attracted more overall attention, the medical community responded similarly rapidly to acute hepatitis of unknown aetiology and to MIS-C. The fact that a major source of early information on these diseases was health agency reports suggests that these should be included in bibliographic databases for ease of access.
背景:2022年春季不明原因急性肝炎的出现令人想起两年前儿童多系统炎症综合征(MIS-C)的出现。目的:对病因不明的急性肝炎和另一种巧合但非儿科疾病Mpox的早期文献进行文献计量学分析,并与我们之前关于MIS-C早期覆盖率的研究结果进行比较。方法和设置:在MEDLINE数据库、预印本服务器和路透社新闻文章中进行文献综述。如果文章是在2022年发表的,并且是专门关于不明原因的急性肝炎或Mpox,则纳入其中。结果:关于不明原因急性肝炎的第一篇报纸/非学术期刊文章于第14周发表,周数在第16周迅速达到高峰,在第22周逐渐减少至零。第一篇同行评议的文章在第15周发表,从第17周到第23周一直保持在第3到7周之间。这些文章中近四分之一(23.5%)的参考文献是卫生机构关于病因不明的急性肝炎的出版物。在相应卫生机构发出警报之前的7周期间内,发表了25篇关于misc的文章(21篇经同行评议,4篇预印本),发表了20篇关于病因不明的急性肝炎的文章(18篇经同行评议,4篇预印本),74篇关于Mpox的文章(48篇经同行评议,26篇预印本)。预印本的比例与疾病类型显著相关(p = 0.03),临床研究的比例也是如此,分别为52%、10%和18% (p = 0.002)。发表在Mpox (N = 180)上的非学术文章比发表在病因不明的急性肝炎(17)或misc(24)上的文章要多。结论:虽然Mpox引起了更多的关注,但医学界对不明原因的急性肝炎和misc的反应同样迅速。关于这些疾病的早期信息的主要来源是卫生机构的报告,这一事实表明,这些报告应列入书目数据库,以方便查阅。
{"title":"Hepatitis in the time of pandemics: A comparative bibliometric analysis of early publications on MIS-C, acute hepatitis of unknown aetiology and Mpox","authors":"Alexandre Fabre MD, PhD ,&nbsp;Paul Guerry PhD ,&nbsp;Aurélie Morand MD, PhD","doi":"10.1016/j.arcped.2025.09.003","DOIUrl":"10.1016/j.arcped.2025.09.003","url":null,"abstract":"<div><h3>Background</h3><div>The emergence of acute hepatitis of unknown etiology in the spring of 2022 was strikingly reminiscent of how multisystem inflammatory syndrome in children (MIS-C) had appeared two years earlier.</div></div><div><h3>Objective</h3><div>To carry out a bibliometric analysis of the early literature on acute hepatitis of unknown aetiology, and another coincident but non-pediatric disease, Mpox, in comparison with our previous findings on the early coverage of MIS-C.</div></div><div><h3>Methods and Settings</h3><div>A review of the literature was conducted in the MEDLINE database, on preprint servers, and among Reuters news articles. Articles were included if they had been published in 2022 and were specifically about acute hepatitis of unknown aetiology or Mpox.</div></div><div><h3>Results</h3><div>The first newspaper/non-academic journal article on acute hepatitis of unknown aetiology was published in week 14 and the weekly number rapidly peaked in week 16 before decreasing gradually down to zero in week 22. The first peer-reviewed article was published in week 15 and remained between 3 and 7 from weeks 17 to 23. Just under a quarter (23.5 %) of references in these articles were to health agency publications on acute hepatitis of unknown aetiology. Over 7-week periods beginning just before the corresponding health agency alerts, 25 articles were published on MIS-C (21 peer-reviewed and 4 preprints), 20 articles were published on acute hepatitis of unknown aetiology (18 peer-reviewed and 4 preprints), and 74 on Mpox (48 peer-reviewed and 26 preprints). The proportion of preprints was significantly associated with disease type (<em>p</em> = 0.03), as was the proportion of clinical studies, with values of 52 %, 10 %, and 18 %, respectively (<em>p</em> = 0.002). Many more non-academic articles were published on Mpox (<em>N</em> = 180) than on acute hepatitis of unknown aetiology (17) or MIS-C (24).</div></div><div><h3>Conclusion</h3><div>Although Mpox attracted more overall attention, the medical community responded similarly rapidly to acute hepatitis of unknown aetiology and to MIS-C. The fact that a major source of early information on these diseases was health agency reports suggests that these should be included in bibliographic databases for ease of access.</div></div>","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":"32 8","pages":"Pages 527-531"},"PeriodicalIF":1.3,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145454221","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Enhancing maternal and infant health: A systematic review of breastfeeding interventions among refugee, migrant, and asylum seeker populations 加强孕产妇和婴儿健康:对难民、移民和寻求庇护者群体中母乳喂养干预措施的系统回顾。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-11-01 DOI: 10.1016/j.arcped.2025.09.001
Ana Claudia Lopes , Tatiana Pereira , Marisa Lousada

Background

Breastfeeding interventions among refugee, migrant, and asylum seeker populations hold significant promise for improving maternal, infant, and community health.

Objectives

This systematic review aims to comprehensively synthesize existing interventions and assess their effectiveness within these vulnerable demographics.

Methods and Setting

Following the PRISMA framework, a thorough literature search was conducted across four electronic databases—PubMed, Cochrane Library, Web of Science, and Scopus—from February 2014 to February 2024, focusing on studies involving refugees, migrants, and asylum seekers who received breastfeeding interventions. Methodological quality appraisal was executed utilizing the Effective Public Health Practice Project Quality Assessment Tool for Quantitative Studies.

Results

Of the scrutinized studies, nine met the stringent eligibility criteria and were subsequently included in this review. Two independent researchers meticulously performed data extraction and analysis. The synthesized findings revealed a spectrum of breastfeeding interventions tailored to migrant populations, comprising both individualized and group-based approaches. Significant outcomes included elevated breastfeeding initiation rates and positive indicators, such as sustained exclusive breastfeeding for up to six months, increased maternal self-efficacy, enhanced dietary diversity, and the adoption of healthier dietary practices.

Conclusion

The collated evidence underscores the potential of breastfeeding interventions to profoundly influence maternal and child health outcomes within migrant communities. Nonetheless, the interpretation of results warrants caution due to the scarcity of high-quality studies and inherent methodological limitations. This review underscores the imperative for further rigorous research to elucidate the nuanced impacts of breastfeeding interventions in migrant populations and to inform evidence-based practices for optimizing maternal and infant health outcomes globally.
背景:在难民、移民和寻求庇护者人群中进行母乳喂养干预,对改善孕产妇、婴儿和社区健康具有重大希望。目的:本系统综述旨在全面综合现有干预措施并评估其在这些脆弱人口统计中的有效性。方法和背景:遵循PRISMA框架,从2014年2月到2024年2月,在pubmed、Cochrane图书馆、Web of Science和scopus四个电子数据库中进行了全面的文献检索,重点研究涉及接受母乳喂养干预的难民、移民和寻求庇护者的研究。采用定量研究的有效公共卫生实践项目质量评估工具进行方法学质量评估。结果:在审查的研究中,有9项符合严格的资格标准,随后纳入本综述。两名独立研究人员细致地进行了数据提取和分析。综合研究结果揭示了针对流动人口量身定制的一系列母乳喂养干预措施,包括个性化和基于群体的方法。显著的结果包括提高母乳喂养开始率和积极指标,如持续纯母乳喂养长达6个月,提高产妇自我效能,增强饮食多样性,以及采用更健康的饮食习惯。结论:整理的证据强调了母乳喂养干预措施对移民社区孕产妇和儿童健康结果产生深远影响的潜力。尽管如此,由于缺乏高质量的研究和固有的方法局限性,对结果的解释需要谨慎。这篇综述强调了进一步严格研究的必要性,以阐明母乳喂养干预措施对移民人群的细微影响,并为全球优化母婴健康结果的循证实践提供信息。
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引用次数: 0
French-Belgian consensus statement to managing spinal deformities in children with spinal muscular atrophy treated with SMN restoring therapies 法国-比利时共识声明,以SMN恢复疗法治疗脊髓性肌萎缩症儿童脊柱畸形。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-11-01 DOI: 10.1016/j.arcped.2025.09.004
Mathilde Gaume , Lisa Viallard , Pascal Rippert , Martin Bail , Raphael Vialle , Isabelle Desguerres , Capucine de Lattre , Vincent Cunin , Carole Vuillerot

Background

Spinal muscular atrophy (SMA) affects the motor neurons of the anterior spinal cord, causing progressive muscle weakness and atrophy. SMN restoring therapies have led to the emergence of new phenotypes in spinal muscular atrophy (SMA), including early-onset scoliosis. Guidelines regarding the optimal treatment for scoliosis and its consequences are lacking.

Objective

To provide guidelines to help clinical decision-making and to standardise the management of neuromuscular scoliosis (NMS) in children with SMA.

Methods

We conducted a Delphi study with 31 experts between July 2023 and February 2024. Three rounds were conducted using anonymous electronic questionnaires to determine consensus on items grouped into 5 main domains: Respiratory monitoring (7 items), Trunk orthoses (4 items), Surgical approaches (13 items), Preoperative care (17), post-operative care (10 items). Experts were asked to rate their agreement with each item on a scale from 1 (strongly disagree) to 9 (strongly agree). Consensus was considered to have been achieved if the median value of the responses was ≥ 7. Items that did not reach consensus in one round were reformulated in the following round using comments provided by the experts.

Results

The experts included 12 orthopaedic surgeons, 9 physical medicine and rehabilitation physicians and 10 paediatricians/child neurologists. Fourty-seven items achieved consensus. In the first round, consensus was achieved for 33 of the 51 items. The second round included 15 items, and a consensus was achieved for 11 items. In the third round, 3 of the 4 items included achieved consensus and the fourth item was dropped due to a lack of agreement.

Conclusions

The recommendations generated from the questionnaire provide a relevant consensus-based guidance for the multidisciplinary management of spinal deformities in children with SMA.
背景:脊髓性肌萎缩症(SMA)影响脊髓前部的运动神经元,导致进行性肌肉无力和萎缩。SMN恢复疗法导致脊髓性肌萎缩症(SMA)出现新的表型,包括早发性脊柱侧凸。关于脊柱侧凸及其后果的最佳治疗指南缺乏。目的:为SMA患儿神经肌肉侧凸(NMS)的临床决策和规范化治疗提供指导。方法:于2023年7月至2024年2月对31名专家进行德尔菲调查。采用匿名电子问卷进行了三轮调查,以确定对5个主要领域的共识:呼吸监测(7项)、躯干矫形器(4项)、手术入路(13项)、术前护理(17项)、术后护理(10项)。专家们被要求在1(非常不同意)到9(非常同意)的范围内对他们对每个项目的同意程度进行评分。如果应答的中位数≥7,则认为达成了共识。在一轮中未达成协商一致意见的项目在下一轮中根据专家提供的意见重新拟订。结果:专家包括12名骨科医生,9名物理医学和康复医师,10名儿科/儿童神经科医师。达成共识的项目有47个。在第一轮谈判中,各方就51个议题中的33个达成共识。第二轮磋商包括15个议题,达成11个共识。在第三轮谈判中,4个议题中有3个达成了共识,第4个议题因未能达成一致而被取消。结论:从问卷中产生的建议为SMA儿童脊柱畸形的多学科管理提供了相关的基于共识的指导。
{"title":"French-Belgian consensus statement to managing spinal deformities in children with spinal muscular atrophy treated with SMN restoring therapies","authors":"Mathilde Gaume ,&nbsp;Lisa Viallard ,&nbsp;Pascal Rippert ,&nbsp;Martin Bail ,&nbsp;Raphael Vialle ,&nbsp;Isabelle Desguerres ,&nbsp;Capucine de Lattre ,&nbsp;Vincent Cunin ,&nbsp;Carole Vuillerot","doi":"10.1016/j.arcped.2025.09.004","DOIUrl":"10.1016/j.arcped.2025.09.004","url":null,"abstract":"<div><h3>Background</h3><div>Spinal muscular atrophy (SMA) affects the motor neurons of the anterior spinal cord, causing progressive muscle weakness and atrophy. SMN restoring therapies have led to the emergence of new phenotypes in spinal muscular atrophy (SMA), including early-onset scoliosis. Guidelines regarding the optimal treatment for scoliosis and its consequences are lacking.</div></div><div><h3>Objective</h3><div>To provide guidelines to help clinical decision-making and to standardise the management of neuromuscular scoliosis (NMS) in children with SMA.</div></div><div><h3>Methods</h3><div>We conducted a Delphi study with 31 experts between July 2023 and February 2024. Three rounds were conducted using anonymous electronic questionnaires to determine consensus on items grouped into 5 main domains: Respiratory monitoring (7 items), Trunk orthoses (4 items), Surgical approaches (13 items), Preoperative care (17), post-operative care (10 items). Experts were asked to rate their agreement with each item on a scale from 1 (strongly disagree) to 9 (strongly agree). Consensus was considered to have been achieved if the median value of the responses was ≥ 7. Items that did not reach consensus in one round were reformulated in the following round using comments provided by the experts.</div></div><div><h3>Results</h3><div>The experts included 12 orthopaedic surgeons, 9 physical medicine and rehabilitation physicians and 10 paediatricians/child neurologists. Fourty-seven items achieved consensus. In the first round, consensus was achieved for 33 of the 51 items. The second round included 15 items, and a consensus was achieved for 11 items. In the third round, 3 of the 4 items included achieved consensus and the fourth item was dropped due to a lack of agreement.</div></div><div><h3>Conclusions</h3><div>The recommendations generated from the questionnaire provide a relevant consensus-based guidance for the multidisciplinary management of spinal deformities in children with SMA.</div></div>","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":"32 8","pages":"Pages 538-546"},"PeriodicalIF":1.3,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145460661","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Occurrence and prediction of clinical interventions during transfer of near-term and term infants with respiratory distress on CPAP. An observational study 短期和足月婴儿呼吸窘迫在CPAP转移中的临床干预的发生和预测。一项观察性研究。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-11-01 DOI: 10.1016/j.arcped.2025.07.001
Alexia Morel , Julien Baleine , Christophe Milesi , Maliha Badr , Arthur Gaudaire , Alexandra Deveze , Sylvain Paulhac , Charline Andrieu , Marion Palpacuer , Gilles Cambonie , Arthur Gavotto

Background

Neonatal respiratory distress (NRD) requiring continuous positive airway pressure (CPAP) is a common indication for inter-hospital transfer of late preterm and term neonates. The optimal composition of the inter-hospital team transfer - involving an advanced neonatal nurse and an ambulance driver or a complete team, which also includes a paediatrician - remains uncertain. Particularly when clinical interventions are required.

Objectives

To assess the occurrence of clinical interventions necessitating a complete transport team during the inter-hospital transfer of neonates with NRD on CPAP. A secondary objective was to evaluate whether data provided to the transfer regulation centre predicted such interventions.

Methods and Setting

This retrospective observational study was conducted at the Paediatric Emergency Transport Service (PETS) of a level 3 maternity hospital between 2021 and 2023. A total of 110 infants ≥34 weeks’ gestational age were included, all transported for NRD with CPAP (mean gestational age 37.6 ± 2.2 weeks, mean birth weight 3042±615 g). Clinical interventions recorded included intubation, surfactant administration, inhaled nitric oxide (iNO) administration, needle aspiration for pneumothorax, and prostaglandin E1 infusion. Complete team transport involved a paediatrician, an advanced neonatal nurse, and an ambulance driver.

Results

Clinical interventions occurred in 11 cases (10%). Factors associated with the need for intervention included higher FiO₂ (71.4 ± 18.5% vs 28.3 ± 8.0%, p < 0.01), presence of pneumothorax (p < 0.01), and transfer from higher-level maternity hospitals (p < 0.01). The ROC curve for FiO₂ predicting intervention had an area of 0.99 (95% CI 0.97–1.01, p = 0.001), with a cut-off of >40% yielding 91% sensitivity, 98% specificity, 83% positive predictive value, and 99% negative predictive value.

Conclusions

High oxygen dependency and the presence of pneumothorax are key indicators for mobilizing a complete transport team during the transfer of late preterm and term neonates with NRD on CPAP. Early identification of these factors could enhance team allocation and resource utilization.
背景:需要持续气道正压通气(CPAP)的新生儿呼吸窘迫(NRD)是晚期早产儿和足月新生儿院间转院的常见指征。医院间转诊团队的最佳组成——包括一名高级新生儿护士和一名救护车司机,还是包括一名儿科医生的完整团队——仍不确定。特别是当需要临床干预时。目的:评估在医院间转移NRD新生儿进行CPAP时需要一个完整的转运小组的临床干预的发生情况。第二个目标是评估提供给转移监管中心的数据是否可以预测这些干预措施。方法和环境:这项回顾性观察性研究于2021年至2023年在一家三级妇产医院的儿科急诊转运服务(PETS)进行。共纳入110例≥34周胎龄的新生儿,均采用CPAP进行NRD转运(平均胎龄37.6±2.2周,平均出生体重3042±615 g)。记录的临床干预措施包括插管、表面活性剂给药、吸入性一氧化氮(iNO)给药、气胸针吸、前列腺素E1输注。完整的团队运输包括一名儿科医生,一名高级新生儿护士和一名救护车司机。结果:临床干预11例(10%)。与干预需求相关的因素包括较高的FiO₂(71.4±18.5% vs 28.3±8.0%,p < 0.01)、是否存在气胸(p < 0.01)、是否从上级妇产医院转院(p < 0.01)。FiO 2预测干预的ROC曲线面积为0.99 (95% CI 0.97-1.01, p = 0.001),截断值为bb0 40%,敏感性为91%,特异性为98%,阳性预测值为83%,阴性预测值为99%。结论:高氧依赖和气胸的存在是CPAP转移NRD晚期早产儿和足月新生儿时动员完整转运团队的关键指标。尽早识别这些因素可以提高团队分配和资源利用。
{"title":"Occurrence and prediction of clinical interventions during transfer of near-term and term infants with respiratory distress on CPAP. An observational study","authors":"Alexia Morel ,&nbsp;Julien Baleine ,&nbsp;Christophe Milesi ,&nbsp;Maliha Badr ,&nbsp;Arthur Gaudaire ,&nbsp;Alexandra Deveze ,&nbsp;Sylvain Paulhac ,&nbsp;Charline Andrieu ,&nbsp;Marion Palpacuer ,&nbsp;Gilles Cambonie ,&nbsp;Arthur Gavotto","doi":"10.1016/j.arcped.2025.07.001","DOIUrl":"10.1016/j.arcped.2025.07.001","url":null,"abstract":"<div><h3>Background</h3><div>Neonatal respiratory distress (NRD) requiring continuous positive airway pressure (CPAP) is a common indication for inter-hospital transfer of late preterm and term neonates. The optimal composition of the inter-hospital team transfer - involving an advanced neonatal nurse and an ambulance driver or a complete team, which also includes a paediatrician - remains uncertain. Particularly when clinical interventions are required.</div></div><div><h3>Objectives</h3><div>To assess the occurrence of clinical interventions necessitating a complete transport team during the inter-hospital transfer of neonates with NRD on CPAP. A secondary objective was to evaluate whether data provided to the transfer regulation centre predicted such interventions.</div></div><div><h3>Methods and Setting</h3><div>This retrospective observational study was conducted at the Paediatric Emergency Transport Service (PETS) of a level 3 maternity hospital between 2021 and 2023. A total of 110 infants ≥34 weeks’ gestational age were included, all transported for NRD with CPAP (mean gestational age 37.6 ± 2.2 weeks, mean birth weight 3042±615 g). Clinical interventions recorded included intubation, surfactant administration, inhaled nitric oxide (iNO) administration, needle aspiration for pneumothorax, and prostaglandin E1 infusion. Complete team transport involved a paediatrician, an advanced neonatal nurse, and an ambulance driver.</div></div><div><h3>Results</h3><div>Clinical interventions occurred in 11 cases (10%). Factors associated with the need for intervention included higher FiO₂ (71.4 ± 18.5% vs 28.3 ± 8.0%, <em>p</em> &lt; 0.01), presence of pneumothorax (<em>p</em> &lt; 0.01), and transfer from higher-level maternity hospitals (<em>p</em> &lt; 0.01). The ROC curve for FiO₂ predicting intervention had an area of 0.99 (95% CI 0.97–1.01, <em>p</em> = 0.001), with a cut-off of &gt;40% yielding 91% sensitivity, 98% specificity, 83% positive predictive value, and 99% negative predictive value.</div></div><div><h3>Conclusions</h3><div>High oxygen dependency and the presence of pneumothorax are key indicators for mobilizing a complete transport team during the transfer of late preterm and term neonates with NRD on CPAP. Early identification of these factors could enhance team allocation and resource utilization.</div></div>","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":"32 8","pages":"Pages 575-579"},"PeriodicalIF":1.3,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145245882","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Optimizing vitamin D supplementation: the role of physician awareness and patient adherence 优化维生素D补充:医生意识和患者依从性的作用。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-11-01 DOI: 10.1016/j.arcped.2025.08.003
Berkay Yalçınkaya, Ahmet Furkan Çolak, Murat Kara
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引用次数: 0
Franco-African paediatric teledermatology: a store-and-forward network 法非儿科远程皮肤病学:储存和转发网络。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-11-01 DOI: 10.1016/j.arcped.2025.09.007
Asmaa Lahrougui , Dalila Benlahcene , Saïd Afif , Emmanuel Mahé

Background

Given the frequency of dermatological diseases in children and the difficulties in accessing specialist care in Africa, teledermatology services, such as store-and-forward (SAF), could be used an alternative approach for improving patient care. The dermatology department of the Argenteuil hospital in France developed a SAF network with paediatricians from seven French-speaking African countries. This study assessed the value of this Franco-African SAF collaboration by identifying the advantages and challenges associated with the first 100 uses of the SAF system.

Methods

Paediatricians belonging to medical societies in French-speaking African countries were provided with information on how to register for the SAF service. The first 100 SAF requests were evaluated. Information about the paediatricians who used the service, and patient demographic, clinical and management data were systematically collected.

Results

Twenty-five (10.2%) paediatricians, out of 244 who registered, submitted cases to the SAF network. In 95 cases, a response was sent within 24 h of submission. In 24 cases, additional information was needed (clinical data, n = 17; photos, n = 6; a histological report, n = 1; identity details, n = 1). The mean age of the children was 5.8 ± 5.0 years, with a predominance of girls (n = 61). Most patients had chronic pathologies (≥ 1 month) (n = 56) belonging to three main groups: chronic inflammatory diseases (n = 42), infectious diseases (n = 23), and genetic diseases (n = 11). As a result of the SAF requests, further examinations were proposed in 39 cases, treatment in 82 cases, and hospitalisation in 11 cases.

Conclusion

The SAF approach is an effective solution for improving access to dermatological care in Africa, providing an alternative method for expert consultation when resources are limited, without diminishing the role of local dermatologists. For dermatologists, this solution provides access to expertise on unusual pathologies and allows solutions to be offered that are tailored to the resources available in the field.
背景:考虑到非洲儿童皮肤病的发病率和获得专科护理的困难,远程皮肤科服务,如储存转发(SAF),可作为改善患者护理的另一种方法。法国阿让特伊医院的皮肤科与七个讲法语的非洲国家的儿科医生建立了一个SAF网络。本研究通过确定与SAF系统的前100次使用相关的优势和挑战,评估了法国-非洲SAF合作的价值。方法:向非洲法语国家医学协会的儿科医生提供如何注册SAF服务的信息。评估了前100个SAF请求。系统地收集了使用该服务的儿科医生的信息以及患者人口统计、临床和管理数据。结果:244名注册的儿科医生中,有25名(10.2%)向SAF网络提交了病例。在95个案件中,在提交后24小时内发出了答复。24例需要补充资料(临床资料17例;照片6例;组织学报告1例;身份信息1例)。患儿平均年龄5.8±5.0岁,以女童为主(n = 61)。大多数患者有慢性病变(≥1个月)(n = 56),主要分为三大类:慢性炎症性疾病(n = 42)、感染性疾病(n = 23)和遗传性疾病(n = 11)。根据安全部队的要求,对39例进行了进一步检查,82例进行了治疗,11例住院治疗。结论:SAF方法是改善非洲皮肤科护理可及性的有效解决方案,在资源有限的情况下为专家咨询提供了另一种方法,而不会削弱当地皮肤科医生的作用。对于皮肤科医生来说,该解决方案提供了对异常病理的专业知识的访问,并允许根据该领域的可用资源提供量身定制的解决方案。
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引用次数: 0
期刊
Archives De Pediatrie
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