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Pediatric acute stroke alert in Nantes and Angers hospitals: description of patients included and comparison with published data 南特和昂热医院的儿科急性卒中预警:纳入患者的描述和与已发表数据的比较
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2026-01-01 DOI: 10.1016/j.arcped.2025.10.007
Floriane Le Bricquir , Sophie Gueden , Didier Loisel , Pauline de Cambourg , Patrick Van Bogaert

Objective

This study aimed to describe data of patients included in the in-hospital pediatric stroke alert and compare it to published data.

Methods

This bicentric retrospective study includes all children aged one month to 18 years who underwent an MRI (magnetic resonance imaging) for an acute neurological deficit according to the stroke alert protocol established at Nantes and Angers University Hospitals between January 2017 and July 2021. Demographic characteristics, time from stroke onset to imaging, diagnosis, treatments if any, and outcomes were collected.

Results

Seventy-eight children were included. The stroke alert procedure led to early stroke detection in 16 children (21%). Four of them received acute recanalization therapy. Other diagnoses needing specific management were identified in 12% of cases. Migraine was the most common diagnosis, affecting more than half of the patients.

Conclusions

This stroke alert protocol allowed children presenting with acute neurological deficit to have rapid access to brain MRI, which confirmed the diagnosis of stroke or other acute neurological disorder necessitating specific management in one-third of the cases.
目的:本研究旨在描述住院儿童脑卒中预警患者的数据,并将其与已发表的数据进行比较。方法:这项双中心回顾性研究纳入了所有1个月至18岁的儿童,这些儿童根据南特和昂热大学医院在2017年1月至2021年7月期间制定的中风警报方案接受了急性神经功能障碍MRI(磁共振成像)检查。收集了人口统计学特征、从中风发作到成像的时间、诊断、治疗(如果有的话)和结果。结果:共纳入78名儿童。卒中预警程序导致16名儿童(21%)早期卒中检测。其中4例接受急性再通治疗。在12%的病例中发现了需要特殊处理的其他诊断。偏头痛是最常见的诊断,影响了一半以上的患者。结论:该卒中预警方案使出现急性神经功能缺损的儿童能够快速获得脑MRI,在三分之一的病例中证实了卒中或其他急性神经系统疾病的诊断,需要进行特殊治疗。
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引用次数: 0
Impact of the francophone pediatric intensive care group bronchiolitis recommendations on diagnostic testing, antibiotic therapy and patient prognosis 法语儿童重症监护组细支气管炎建议对诊断检测、抗生素治疗和患者预后的影响
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2026-01-01 DOI: 10.1016/j.arcped.2025.10.008
Pauline Labe , Matthieu Bendavid , Julie Toubiana , Céline Anquetil , Mehdi Oualha , Charlotte Collignon

Background

Acute bronchiolitis is a common viral infection in infants, with severe cases requiring admission to pediatric intensive care units (PICU). While bacterial co-infections are rare, timely diagnosis and management are essential. Updated guidelines from the French-speaking Group of Pediatric Intensive and Emergency Care (GFRUP) aim to standardize the standard of care, reducing unnecessary interventions.

Objectives

This study evaluated the impact of updated bronchiolitis management guidelines on sepsis incidence, diagnostic test use, and antibiotic prescriptions in a single center.

Methods and settings

A retrospective study compared two epidemic periods (2022–2023 and 2023–2024) at the PICU, Necker-Enfants Malades Hospital. Clinical and microbiological data, including sepsis incidence, bacterial co-infections, diagnostic tests, and antibiotic use, were analyzed.

Results

Among 257 infants admitted for bronchiolitis, sepsis incidence remained low (8% vs. 7%). Implementation of the guidelines led to a significant reduction in additional diagnostic tests (65% vs. 49%, p = 0.013). Antibiotic prescription rates were unchanged, but a narrower spectrum of antibiotics was observed, with decreased amoxicillin/clavulanate use and increased amoxicillin prescriptions. Lung infections were the most frequent bacterial co-infections, with no rise in bacterial infection rates.

Conclusions

Implementation of updated guidelines reduced unnecessary diagnostic tests and promoted narrower-spectrum antibiotic use without increasing sepsis incidence. These findings highlight the potential of evidence-based guidelines to optimize care, support antibiotic stewardship, and minimize unnecessary interventions in pediatric critical care.
背景:急性细支气管炎是一种常见的婴儿病毒感染,严重者需要进入儿科重症监护病房(PICU)。虽然细菌合并感染很少见,但及时诊断和管理是必不可少的。法语儿科重症和急诊护理小组(GFRUP)的最新指南旨在使护理标准标准化,减少不必要的干预措施。目的:本研究评估了单中心更新的细支气管炎管理指南对脓毒症发生率、诊断测试使用和抗生素处方的影响。方法和背景:一项回顾性研究比较了内克-马拉德斯儿童医院PICU的两个流行时期(2022-2023和2023-2024)。临床和微生物学数据,包括败血症发生率、细菌合并感染、诊断测试和抗生素使用进行了分析。结果:257名因毛细支气管炎入院的婴儿中,败血症的发生率仍然很低(8%对7%)。指南的实施导致额外诊断测试的显著减少(65%对49%,p = 0.013)。抗生素处方率没有变化,但抗生素的使用范围较窄,阿莫西林/克拉维酸盐的使用减少,阿莫西林的处方增加。肺部感染是最常见的细菌合并感染,细菌感染率没有上升。结论:更新指南的实施减少了不必要的诊断测试,促进了窄谱抗生素的使用,而不增加败血症的发生率。这些发现强调了循证指南在优化护理、支持抗生素管理和减少儿科危重病护理不必要干预方面的潜力。
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引用次数: 0
Correlates of depression and anxiety among family caregivers of pediatric patients with cancer in Lebanon 黎巴嫩儿科癌症患者家庭照顾者抑郁和焦虑的相关性
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2026-01-01 DOI: 10.1016/j.arcped.2025.10.006
Jhony Baissary , Souheil Hallit , Melyssa Assaf , Feten Fekih-Romdhane , Mohamad Farhat , Peter Noun

Background

Cancer diagnosis in the child is stressful for the family caregiver and could have a major impact on his/her mental health, including depression and anxiety, especially during the harsh current Lebanese economic crisis. This study aims at evaluating the correlates of depression and anxiety among family caregivers of pediatric cancer patients in Lebanon.

Methods

It is a cross-sectional study, using a convenience sample of 193 caregivers of pediatric patients with cancer. Depression, anxiety, and work fatigue were assessed using the Patient Health Questionnaire-9, Lebanese Anxiety Scale, and 3D-Work Fatigue Inventory respectively.

Results

The results showed that 18.1 % of the participants have moderately severe depression, whereas 22.2 % show severe depression. Moreover, 79.8 % had anxiety. Having a shortage of medications (Beta = 2.38) and more mental work fatigue (Beta = 0.36) were significantly associated with more depression. Higher physical (Beta = 0.32) and mental (Beta = 0.37) work fatigue were significantly associated with more anxiety.

Conclusion

The study results should raise concerns and make physicians in Lebanon aware of the psychological status of the family caregivers and encourage them to provide adequate support. The results underscore the urgent need for advocacy to improve conditions for families and enhance access to care for children with cancer and their families.
背景:儿童的癌症诊断给家庭照顾者带来压力,并可能对他/她的心理健康产生重大影响,包括抑郁和焦虑,特别是在当前黎巴嫩严重的经济危机期间。本研究旨在评估黎巴嫩儿童癌症患者家庭照顾者抑郁与焦虑的相关关系。方法:这是一项横断面研究,使用193名儿科癌症患者护理人员的方便样本。分别使用患者健康问卷-9、黎巴嫩焦虑量表和3d工作疲劳量表评估抑郁、焦虑和工作疲劳。结果:18.1%的参与者表现为中度抑郁症,22.2%的参与者表现为重度抑郁症。此外,79.8%的人有焦虑。缺乏药物(Beta = 2.38)和更多的脑力工作疲劳(Beta = 0.36)与更多的抑郁显著相关。更高的身体(Beta = 0.32)和精神(Beta = 0.37)工作疲劳与更多的焦虑显著相关。结论:研究结果应引起关注,使黎巴嫩的医生了解家庭照顾者的心理状况,并鼓励他们提供足够的支持。研究结果强调,迫切需要进行宣传,以改善家庭条件,增加癌症儿童及其家庭获得护理的机会。
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引用次数: 0
Annular lesions in childhood: A narrative review 儿童环形病变:叙述回顾。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2026-01-01 DOI: 10.1016/j.arcped.2025.10.004
Sergio Castillo-Pinto , Margarita Gomez , Monica Novoa-Candia

Backgound

Annular skin lesions are ring-shaped manifestations characterized by an erythematous margin and a preserved or atrophic center. Their pathophysiology involves mechanisms such as peripheral inflammatory mediator expansion or immune tolerance at the center of the lesions.

Methods

This article reviews some of the most common annular lesions observed in pediatric populations within our setting.

Results

We included urticarial vasculitis, childhood cutaneous lupus erythematous, Still’s disease, annular granuloma, centriphugal annular erythema, pigmentary dermatoses, erythema migrans, multiform urticaria, acute hemorrhagic edema of the infancy, infectious dermatoses such as dermatophyte infections, and blistering conditions like linear IgA dermatosis, highlighting their clinical presentations, diagnostic approaches, and treatment options, emphasizing the importance of clinical correlation, biopsy findings, and appropriate therapeutic strategies.

Conclusion

This review will assist clinicians in diagnosing diseases associated with annular skin lesions.
背景:环状皮肤病变是一种环状病变,其特征是边缘红斑和中心保存或萎缩。其病理生理机制包括外周炎症介质扩张或病灶中心的免疫耐受。方法:这篇文章回顾了一些最常见的环性病变观察在儿科人群在我们的设置。结果:我们纳入了荨麻疹血管炎、儿童皮肤红斑狼疮、Still’s病、环状肉芽肿、离心性环状红斑、色素性皮肤病、迁移性红斑、多种形式的荨麻疹、婴幼儿急性出血性水肿、感染性皮肤病(如皮肤真菌感染)和水泡性疾病(如线性IgA皮肤病),强调了它们的临床表现、诊断方法和治疗方案,强调了临床相关性的重要性。活检结果,以及适当的治疗策略。结论:本综述将有助于临床医生诊断与环形皮肤病变相关的疾病。
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引用次数: 0
Seizure freedom in pediatric juvenile myoclonic epilepsy: The roles of cognition, chronodependency, and EEG 儿童青少年肌阵挛性癫痫的发作自由:认知、时间依赖性和脑电图的作用:儿童青少年肌阵挛性癫痫的发作结局。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2026-01-01 DOI: 10.1016/j.arcped.2025.10.003
Ünal Akça , Emre Sanrı , Gülfer Akça

Objective

To identify clinical, chronobiological, and electroencephalographic (EEG) predictors of seizure freedom in pediatric patients with juvenile myoclonic epilepsy (JME), and to evaluate the prognostic utility of both classic and novel markers in a real-world setting.

Methods

We conducted a retrospective cohort study of 71 pediatric JME patients (mean age at diagnosis 16.1 ± 1.05 years; 76.1 % female) diagnosed at a single tertiary center between January 2023 and January 2025. Data collected included demographic variables, seizure characteristics, academic performance, comorbidities, antiseizure medication use, and detailed EEG findings. Classic (e.g., seizure type, academic achievement) and novel predictors (e.g., chronodependency) were assessed for their association with seizure outcome. Chronodependency was defined as a pattern of seizures predominantly occurring after awakening or during specific circadian periods. Multivariate logistic regression and receiver operating characteristic (ROC) analyses were performed.

Results

At last follow-up, 63.4 % (n = 45) of patients achieved seizure freedom. High academic performance (OR=42.00, p < 0.001) and initial myoclonic seizure type (OR=6.96, p < 0.001) were strongly associated with seizure freedom, whereas high initial frequency of generalized tonic-clonic seizures (OR=0.05, p < 0.001) and failure of at least two appropriate antiseizure medications (OR=0.07, p < 0.001) predicted poor outcomes. Among novel predictors, chronodependency (OR=0.29, p = 0.025), major focal EEG abnormalities (OR=0.30, p = 0.038), and EEG hyperventilation response (OR=0.35, p = 0.025) were independent negative prognostic factors. The full multivariate model, integrating classic and novel predictors, demonstrated excellent discriminative performance (AUC=0.96). Levetiracetam was the most frequently prescribed monotherapy, reflecting the predominance of adolescent and female patients; multidrug regimens were linked to poor seizure control.

Conclusion

Seizure outcome in pediatric JME is influenced by a complex interplay of cognitive, chronobiological, and electrographic factors. Integrating academic performance, seizure type, chronodependency, and advanced EEG findings enhances prognostication and supports a personalized, multidimensional approach to management. Early identification of at-risk patients may inform tailored interventions and improve long-term outcomes.
目的:确定小儿幼年肌阵挛性癫痫(JME)患者癫痫发作自由的临床、时间生物学和脑电图(EEG)预测因素,并评估经典和新型标志物在现实世界中的预后效用。方法:我们对2023年1月至2025年1月在单一三级中心诊断的71例小儿JME患者(诊断时平均年龄16.1±1.05岁,76.1%为女性)进行了回顾性队列研究。收集的数据包括人口统计学变量、癫痫发作特征、学业表现、合并症、抗癫痫药物使用和详细的脑电图结果。经典的(例如,癫痫发作类型,学习成绩)和新颖的预测因子(例如,时间依赖性)被评估与癫痫发作结果的关系。时间依赖性被定义为一种癫痫发作模式,主要发生在醒来后或特定的昼夜节律期间。进行多因素logistic回归和受试者工作特征(ROC)分析。结果:最后一次随访,63.4% (n = 45)患者癫痫发作自由。高学习成绩(OR=42.00, p < 0.001)和初始肌阵挛性发作类型(OR=6.96, p < 0.001)与癫痫发作自由密切相关,而高初始全身性强直-阵挛性发作频率(OR=0.05, p < 0.001)和至少两种合适的抗癫痫药物失败(OR=0.07, p < 0.001)预测不良预后。在新的预测因素中,时间依赖性(OR=0.29, p = 0.025)、主要局灶性脑电图异常(OR=0.30, p = 0.038)和脑电图过度通气反应(OR=0.35, p = 0.025)是独立的负面预后因素。完整的多元模型,整合了经典和新颖的预测因子,表现出良好的判别性能(AUC=0.96)。左乙拉西坦是最常用的单药治疗,反映了青少年和女性患者的优势;多药治疗方案与癫痫控制不佳有关。结论:小儿JME发作结局受认知、时间生物学和电图因素复杂的相互作用影响。综合学习成绩、发作类型、时间依赖性和先进的脑电图结果可以增强预测,并支持个性化、多维的治疗方法。早期识别有风险的患者可以为量身定制的干预措施提供信息,并改善长期结果。
{"title":"Seizure freedom in pediatric juvenile myoclonic epilepsy: The roles of cognition, chronodependency, and EEG","authors":"Ünal Akça ,&nbsp;Emre Sanrı ,&nbsp;Gülfer Akça","doi":"10.1016/j.arcped.2025.10.003","DOIUrl":"10.1016/j.arcped.2025.10.003","url":null,"abstract":"<div><h3>Objective</h3><div>To identify clinical, chronobiological, and electroencephalographic (EEG) predictors of seizure freedom in pediatric patients with juvenile myoclonic epilepsy (JME), and to evaluate the prognostic utility of both classic and novel markers in a real-world setting.</div></div><div><h3>Methods</h3><div>We conducted a retrospective cohort study of 71 pediatric JME patients (mean age at diagnosis 16.1 ± 1.05 years; 76.1 % female) diagnosed at a single tertiary center between January 2023 and January 2025. Data collected included demographic variables, seizure characteristics, academic performance, comorbidities, antiseizure medication use, and detailed EEG findings. Classic (e.g., seizure type, academic achievement) and novel predictors (e.g., chronodependency) were assessed for their association with seizure outcome. Chronodependency was defined as a pattern of seizures predominantly occurring after awakening or during specific circadian periods. Multivariate logistic regression and receiver operating characteristic (ROC) analyses were performed.</div></div><div><h3>Results</h3><div>At last follow-up, 63.4 % (<em>n</em> = 45) of patients achieved seizure freedom. High academic performance (OR=42.00, <em>p</em> &lt; 0.001) and initial myoclonic seizure type (OR=6.96, <em>p</em> &lt; 0.001) were strongly associated with seizure freedom, whereas high initial frequency of generalized tonic-clonic seizures (OR=0.05, <em>p</em> &lt; 0.001) and failure of at least two appropriate antiseizure medications (OR=0.07, <em>p</em> &lt; 0.001) predicted poor outcomes. Among novel predictors, chronodependency (OR=0.29, <em>p</em> = 0.025), major focal EEG abnormalities (OR=0.30, <em>p</em> = 0.038), and EEG hyperventilation response (OR=0.35, <em>p</em> = 0.025) were independent negative prognostic factors. The full multivariate model, integrating classic and novel predictors, demonstrated excellent discriminative performance (AUC=0.96). Levetiracetam was the most frequently prescribed monotherapy, reflecting the predominance of adolescent and female patients; multidrug regimens were linked to poor seizure control.</div></div><div><h3>Conclusion</h3><div>Seizure outcome in pediatric JME is influenced by a complex interplay of cognitive, chronobiological, and electrographic factors. Integrating academic performance, seizure type, chronodependency, and advanced EEG findings enhances prognostication and supports a personalized, multidimensional approach to management. Early identification of at-risk patients may inform tailored interventions and improve long-term outcomes.</div></div>","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":"33 1","pages":"Pages 36-42"},"PeriodicalIF":1.3,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145607552","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
The role of primary care physicians in preventing and managing dangerous games among adolescents: A qualitative study 初级保健医生在预防和管理青少年危险游戏中的作用:一项定性研究。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2026-01-01 DOI: 10.1016/j.arcped.2025.10.009
Chantal Stheneur , Sirine Sandli , Solène Sirieys , Alexandra Loisel , Marie Rose Moro , Rahmethnissah Radjack , Anne Li

Background

Dangerous games are playful activities engaged in by children and adolescents, posing significant risks to their health. Various types exist, aggressive games, challenge games, and asphyxiation games, with the ‘Choking game’ (where individuals attempt strangulation) being the most well-known. The prevalence of dangerous games among adolescents is increasing, yet it remains a little-known and studied societal issue among healthcare professionals.

Objective

To assess physicians’ role in the prevention and management of dangerous games.

Methods and Settings

Qualitative semi-structured interviews with general practitioners, pediatricians, and psychiatrists were performed. All data were transcribed verbatim and analyzed using a content analysis approach.

Results

We interviewed 6 general practitioners, 6 psychiatrists, and 5 pediatricians practicing in the Paris region. Three main themes emerged from the data: 1) physicians' lack of knowledge about the topic due to insufficient training, 2) players’ profiles characterized by psychological vulnerability, influence of peer pressure and social media, with no clear suicidal intent, and 3) the need to primarily focus on screening, prevention, and management at the school level.

Conclusion

This study highlights a critical public health issue. Physicians' roles are essential for identifying psychological vulnerabilities and raising awareness among youth of the associated risks. However, prevention requires close collaboration between the educational sector and healthcare professionals, who could benefit from targeted training and regular interventions within educational institutions.
背景:危险游戏是儿童和青少年参与的好玩的活动,对他们的健康构成重大风险。有各种各样的游戏,攻击性游戏,挑战游戏和窒息游戏,其中“窒息游戏”(个人试图扼杀)是最著名的。青少年中危险游戏的流行程度正在增加,但在医疗保健专业人员中,它仍然是一个鲜为人知和研究的社会问题。目的:评价医生在危险游戏预防和管理中的作用。方法和背景:对全科医生、儿科医生和精神科医生进行定性半结构化访谈。所有数据逐字转录并使用内容分析方法进行分析。结果:我们采访了在巴黎地区执业的6名全科医生、6名精神科医生和5名儿科医生。数据中出现了三个主要主题:1)由于培训不足,医生对这一主题缺乏了解;2)球员的个人资料以心理脆弱性、同伴压力和社交媒体的影响为特征,没有明确的自杀意图;3)需要主要关注学校层面的筛查、预防和管理。结论:这项研究突出了一个重要的公共卫生问题。医生的角色对于识别心理脆弱性和提高青少年对相关风险的认识至关重要。然而,预防需要教育部门和保健专业人员之间的密切合作,他们可以从教育机构内的有针对性的培训和定期干预中受益。
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引用次数: 0
Implementation in the Ile-de-France region of French clinical guidelines for the management of newborns at risk of early-onset neonatal infection 在法兰西岛大区实施法国早发性新生儿感染风险管理临床指南。
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2026-01-01 DOI: 10.1016/j.arcped.2025.11.002
Mathilde Letouzey , Elisabeth Outtier , Tiphaine Biaggi , Anne Rousseau , Pascal Boileau , Laurence Foix-L’Hélias

Background

Accurately targeting newborns suspected of early-onset bacterial neonatal infection (EONI) and optimizing the administration of antibiotics is challenging. French clinical guidelines (HAS/SFN) on the management of newborns over 34 weeks at risk of EONI were deployed in September 2017.

Objective

Our objective was to assess their implementation 3 years later in the Paris area, representing approximately 180,000 births.

Methods and Settings

We conducted an observational study in all maternity units of the Ile-de-France region from July 2020 to January 2021. We sent a self-administered questionnaire on the application of the new guidelines by email addressed to a pediatrician in each maternity unit.

Results

Out of the 80 maternity units in the Ile-de-France region, 78 participated. Among the participating units, 40 (51 %) reported applying the new clinical guidelines fully, 28 (36 %) partially, while 10 (13 %) reported not applying them. Among participants, 45 (66 %) maternity units found the implementation of the clinical guidelines feasible, and nearly 80 % of them observed that applying them resulted in fewer additional tests, such as blood samples or peripheral bacteriological tests. Only 35 % of maternity units no longer collect gastric fluid sample, even though this practice is no longer recommended. Among the difficulties identified by the 10 centers that reported not implementing the guidelines were a lack of equipment, insufficient numbers of competent personnel, and insufficient time to train relevant professionals.

Conclusion

Three years after the publication of the French clinical guidelines (HAS/SFN) on the management of newborns over 34 weeks at risk of EONI, this study showed that over 85 % of responding maternity units in the Ile-de-France region reported have implemented the guidelines either fully or partially.
背景:准确定位疑似早发型新生儿细菌性新生儿感染(EONI)并优化抗生素给药具有挑战性。法国临床指南(HAS/SFN)关于34周以上EONI风险新生儿的管理于2017年9月发布。目标:我们的目标是评估3年后在巴黎地区的实施情况,该地区约有18万名新生儿。方法和环境:我们于2020年7月至2021年1月在法兰西岛地区的所有产科单位进行了一项观察性研究。我们通过电子邮件向每个产科病房的儿科医生发送了一份关于新指南应用情况的自我管理问卷。结果:在法兰西岛地区的80个产科单位中,有78个参加了该计划。在参与的单位中,40个(51%)报告完全应用新的临床指南,28个(36%)部分应用,而10个(13%)报告未应用。在参与者中,有45家(66%)产科单位认为实施临床指南是可行的,其中近80%的单位观察到,应用这些指南可以减少额外的检查,如血液样本或外周细菌学检查。只有35%的产科单位不再收集胃液样本,尽管这种做法不再被推荐。据报告,未执行指导方针的10个中心指出的困难包括缺乏设备、合格人员数量不足以及培训相关专业人员的时间不足。结论:在法国临床指南(HAS/SFN)关于34周以上EONI风险新生儿管理的出版三年后,这项研究表明,在法兰西岛地区,85%以上的响应产科单位完全或部分地实施了指南。
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引用次数: 0
Respiratory care in Duchenne muscular dystrophy 杜氏肌营养不良症的呼吸护理
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-12-01 DOI: 10.1016/S0929-693X(25)00250-7
Hélène Prigent
Respiratory complications are amajor cause of morbidity and mortality in Duchenne muscular dystrophy (DMD). Progressive weakness of inspiratory and expiratory muscles leads to ineffective cough, mucus retention, sleep-disordered breathing, nocturnal hypoventilation, and ultimately chronic respiratory failure. Before the advent of mechanical ventilation, respiratory failure was the leading cause of early death. Advances in multidisciplinary care, including noninvasive ventilation (NIV), airway clearance techniques, corticosteroid therapy, scoliosis management, and cardioprotective treatments, have markedly improved survival and quality of life. Respiratory decline typically follows loss of ambulation, reflecting the progressive loss of muscle strength. Hypoventilation first appears during REM sleep and evolves into daytime hypercapnia. Regular monitoring—annually before and biannually after loss of ambulation—is essential. Key respiratory monitoring parameters include decreased vital capacity, maximal inspiratory and expiratory pressures, and peak cough flow. Sleep studies are critical for early detection of nocturnal hypoventilation and sleep-disordered breathing. NIV remains the cornerstone of therapy, improving gas exchange, sleep quality, and life expectancy. As weakness increases, patients may become ventilator dependent; invasive ventilation is reserved for NIV failure. Adjunctive strategies such as lung volume recruitment and mechanical insufflation–exsufflation enhance cough efficacy and secretion clearance. Long-term corticosteroid therapy delays respiratory decline, while scoliosis correction and infection prevention further optimize outcomes. Nutritional management also supports respiratory health. Overall, early detection, individualized ventilatory management, and comprehensive multidisciplinary care are critical to improving survival and quality of life in patients with DMD.
呼吸系统并发症是杜氏肌营养不良症(DMD)发病和死亡的主要原因。吸气和呼气肌的进行性无力导致无效咳嗽、粘液潴留、睡眠呼吸障碍、夜间换气不足,最终导致慢性呼吸衰竭。在机械通气出现之前,呼吸衰竭是早期死亡的主要原因。多学科治疗的进步,包括无创通气(NIV)、气道清除技术、皮质类固醇治疗、脊柱侧凸管理和心脏保护治疗,显著提高了生存率和生活质量。呼吸衰退通常伴随着行走能力的丧失,反映了肌肉力量的逐渐丧失。低通气首先出现在快速眼动睡眠期间,并演变为白天高碳酸血症。定期监测——丧失行动能力之前每年一次,丧失行动能力之后每两年一次——是必要的。关键的呼吸监测参数包括肺活量下降、最大吸气和呼气压力以及咳嗽流量峰值。睡眠研究对于早期发现夜间低通气和睡眠呼吸障碍至关重要。NIV仍然是治疗的基石,改善气体交换,睡眠质量和预期寿命。随着虚弱程度的增加,患者可能会依赖呼吸机;有创通气用于无创通气失败。辅助策略,如肺容量增加和机械吸气-呼气,可提高咳嗽疗效和分泌物清除。长期皮质类固醇治疗可延缓呼吸衰退,而脊柱侧凸矫正和感染预防可进一步优化预后。营养管理也有助于呼吸系统健康。总之,早期发现、个体化通气管理和综合多学科护理对改善DMD患者的生存和生活质量至关重要。
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引用次数: 0
Corticosteroid treatment in Duchenne muscular dystrophy 皮质类固醇治疗杜氏肌营养不良症
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-12-01 DOI: 10.1016/S0929-693X(25)00253-2
Stéphanie Fontaine-Carbonnel
Duchenne muscular dystrophy (DMD) is a severe degenerative disease that remains incurable. Its prognosis has been transformed by multidisciplinary care, which has significantly improved the life expectancy of patients. Corticosteroids have been used for over 20 years and have modified the care of young boys with DMD: They delay the loss of ambulation, support the gradual introduction of technical mobility aids, and reduce the frequency of scoliosis surgery. Corticoids are started and maintained at the theoretical dose of 0.75 mg/kg/day of prednisone/prednisolone or 0.9 mg/kg/day of deflazacort, adjusting the dose to weight if the benefit–risk ratio favors treatment. If introduced early enough, when muscle degeneration is not too advanced, they prolong the ability to climb stairs, rise from the floor, walk, and bring the hands to the mouth. They have been suggested to contribute to delaying the onset of respiratory failure and cardiomyopathy. The well-known side effects of corticosteroids require specific management (to prevent obesity and osteoporosis) and/or adjustment of their dosage. The only reasons to discontinue treatment when the patient and family are adhering to it are the uncontrolled side effects. Thus, in many countries including France, as of late 2025, the only routinely prescribed symptomatic treatment for DMD remains conventional corticosteroids, which will be discussed in this article, followed by a description of vamorolone, a dissociative steroidal compound, and givinostat, a histone deacetylase inhibitor.
杜氏肌营养不良症(DMD)是一种严重的退行性疾病,目前仍无法治愈。多学科治疗改变了该病的预后,显著提高了患者的预期寿命。皮质类固醇已经使用了20多年,并改善了患有DMD的年轻男孩的护理:它们延迟了行动能力的丧失,支持逐渐引入技术活动辅助设备,并减少了脊柱侧凸手术的频率。皮质激素开始并维持在理论剂量0.75 mg/kg/天的强的松/泼尼松或0.9 mg/kg/天的地氮柯,如果获益-风险比有利于治疗,调整剂量与体重。如果尽早使用,在肌肉退化不太严重的时候,它们可以延长爬楼梯、从地板上站起来、走路和用手到嘴的能力。它们被认为有助于延缓呼吸衰竭和心肌病的发作。众所周知,皮质类固醇的副作用需要特殊的管理(防止肥胖和骨质疏松)和/或调整其剂量。当病人和家人坚持治疗时,停止治疗的唯一原因是无法控制的副作用。因此,在包括法国在内的许多国家,截至2025年底,DMD唯一的常规对症治疗仍然是常规的皮质类固醇,这将在本文中讨论,然后是对氨莫洛酮(一种解离性类固醇化合物)和吉维司他(一种组蛋白去乙酰化酶抑制剂)的描述。
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引用次数: 0
Duchenne muscular dystrophy in 2025 2025年的杜氏肌萎缩症
IF 1.3 4区 医学 Q3 PEDIATRICS Pub Date : 2025-12-01 DOI: 10.1016/S0929-693X(25)00245-3
Isabelle Desguerre
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引用次数: 0
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Archives De Pediatrie
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