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Learning-Based Models for Predicting IVIG Resistance and Coronary Artery Lesions in Kawasaki Disease: A Review of Technical Aspects and Study Features. 基于学习的川崎病 IVIG 抗药性和冠状动脉病变预测模型:技术方面和研究特点综述。
IF 3.4 3区 医学 Q1 PEDIATRICS Pub Date : 2025-04-03 DOI: 10.1007/s40272-025-00693-7
Danilo Mirata, Anna Chiara Tiezzi, Lorenzo Buffoni, Ilaria Pagnini, Ilaria Maccora, Edoardo Marrani, Maria Vincenza Mastrolia, Gabriele Simonini, Teresa Giani

Kawasaki disease (KD) is a common pediatric vasculitis, with coronary artery lesions (CALs) representing its most severe complication. Early identification of high-risk patients, including those with disease resistant to first-line treatments, is essential to guide personalized therapeutic approaches. Given the limited reliability of current scoring systems, there has been growing interest in the development of new prognostic models based on machine learning algorithms and artificial intelligence (AI). AI has the potential to revolutionize the management of KD by improving patient stratification and supporting more targeted treatment strategies. This narrative review examines recent applications of AI in stratifying patients with KD, with a particular focus on the ability of models to predict intravenous immunoglobulin resistance and the risk of CALs. We analyzed studies published between January 2019 and April 2024 that incorporated AI-based predictive models. In total, 21 papers met the inclusion criteria and were subject to technical and statistical review; 90% of these were conducted in patients from Asian hospitals. Most of the studies (18/21; 85.7%) were retrospective, and two-thirds included fewer than 1000 patients. Significant heterogeneity in study design and parameter selection was observed across the studies. Resistance to intravenous immunoglobulin emerged as a key factor in AI-based models for predicting CALs. Only five models demonstrated a sensitivity > 80%, and four studies provided access to the underlying algorithms and datasets. Challenges such as small sample sizes, class imbalance, and the need for multicenter validation currently limit the clinical applicability of machine-learning-based predictive models. The effectiveness of AI models is heavily influenced by the quantity and quality of data, labeling accuracy, and the completeness of the training datasets. Additionally, issues such as noise and missing data can negatively affect model performance and generalizability. These limitations highlight the need for rigorous validation and open access to model code to ensure transparency and reproducibility. Collaboration and data sharing will be essential for refining AI algorithms, improving patient stratification, and optimizing treatment strategies.

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引用次数: 0
Tapinarof Cream 1%: Pediatric First Approval.
IF 3.4 3区 医学 Q1 PEDIATRICS Pub Date : 2025-03-29 DOI: 10.1007/s40272-025-00689-3
Sheridan M Hoy

The aryl hydrocarbon receptor (AhR) has an integral role in maintaining skin homeostasis. Tapinarof cream 1% (VTAMA®) is an AhR agonist developed by Dermavant Sciences, an Organon Company, as a once-daily topical treatment for plaque psoriasis and atopic dermatitis (AD). It was first approved in May 2022 in the USA for the topical treatment of plaque psoriasis in adults. It was then approved in June 2024 in Japan for the topical treatment of plaque psoriasis in adults and AD in adults and pediatric patients 12 years of age and older. In December 2024, it was approved in the USA for the topical treatment of AD in adults and pediatric patients 2 years of age and older. This article summarizes the milestones in the development of tapinarof cream 1% leading to this first pediatric approval.

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引用次数: 0
Unveiling the Complexities of Pediatric Asthma Treatment: Evidence, Controversies, and Emerging Approaches.
IF 3.4 3区 医学 Q1 PEDIATRICS Pub Date : 2025-03-22 DOI: 10.1007/s40272-025-00694-6
Maria Elisa Di Cicco, Diego Peroni, Gian Luigi Marseglia, Amelia Licari

Pediatric asthma remains a prevalent and challenging chronic condition globally, affecting quality of life and imposing significant burdens on families and healthcare systems. Despite advancements in understanding asthma pathophysiology and treatment, key controversies persist in optimizing management strategies. Inhaled corticosteroids (ICS) are the cornerstone of treatment, reducing inflammation and preventing exacerbations. While concerns about growth suppression exist, evidence suggests that this effect is primarily associated with high doses and prolonged use, rather than standard maintenance therapy. Nonetheless, adherence to ICS remains suboptimal, necessitating strategies to ensure effective and sustained treatment. The introduction of maintenance and reliever therapy (MART) with ICS-formoterol has offered improved outcomes by simplifying regimens and reducing reliance on short-acting beta-agonists (SABA). However, evidence supporting MART and ICS-SABA regimens in younger children is limited, highlighting gaps in pediatric-focused research. Biologics targeting inflammatory pathways, such as omalizumab, mepolizumab, and dupilumab, represent a personalized approach for severe asthma but face challenges including high costs, limited long-term safety data, and uncertainty regarding their ability to modify disease progression. In addition, the complexity of treatment decisions is compounded by insufficient biomarkers and age-specific evidence to guide therapy. Addressing these gaps requires robust clinical studies and improved adherence strategies tailored to pediatric populations. This review critically examines current pharmacological strategies, unresolved issues, and evolving approaches in asthma management, emphasizing the need for personalized and evidence-based care. Enhancing treatment outcomes for pediatric asthma necessitates balancing therapeutic benefits with minimal adverse effects and leveraging ongoing research to inform future practice.

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引用次数: 0
Emerging Treatments in Neonatal Fungal Infections: Progress and Prospects.
IF 3.4 3区 医学 Q1 PEDIATRICS Pub Date : 2025-03-21 DOI: 10.1007/s40272-025-00688-4
Lucy Eletel, Talia Thomas, Emily A Berry, Gregory L Kearns

Fungal infections in neonates are potentially life threatening. The differential diagnosis for neonatal rashes is extensive, with common culprits including both bacteria and fungi. Candida albicans is the predominant fungal pathogen, causing infections that range from superficial disease to severe systemic conditions, including sepsis and meningitis. Neonates, especially those who are preterm, are particularly susceptible because of developmentally immature immune systems and the use of invasive procedures and devices in neonatal intensive care units. Congenital cutaneous candidiasis, acquired in utero or during delivery, can lead to disseminated infection with high mortality rates. Early diagnosis and prompt antifungal treatment are crucial but challenging because of subtle clinical presentations, making accurate identification of the offending organism essential for selecting the appropriate treatment. Candida species account for the majority of neonatal fungal infections, with different species necessitating distinct treatments because of varying susceptibility profiles. Aspergillus, another significant pathogen, poses high mortality risks and can present either cutaneously or systemically. Malassezia, though less common, primarily affects preterm infants with catheter-related fungemia. Other fungal species, including Zygomycetes, Trichosporon, and Cryptococcus, rarely produce neonatal infections but are noteworthy for consideration. Treatment of fungal infection is critical despite the relative paucity of information regarding the clinical pharmacology of many antifungal drugs in neonates. We review the major antifungal agents (e.g., amphotericin B, the echinocandins, the azoles) and provide pharmacologic and dosing information. Finally, preventive strategies, including the use of stringent aseptic techniques and careful clinical monitoring, are essential to mitigate both the incidence and severity of these infections in neonates and infants in the first months of life.

新生儿真菌感染可能危及生命。新生儿皮疹的鉴别诊断范围很广,常见的罪魁祸首包括细菌和真菌。白色念珠菌是最主要的真菌病原体,引起的感染范围从浅表疾病到严重的全身性疾病,包括败血症和脑膜炎。新生儿,尤其是早产儿,由于免疫系统发育不成熟,加上新生儿重症监护室使用侵入性程序和设备,特别容易受到感染。先天性皮肤念珠菌病在子宫内或分娩时感染,可导致播散性感染,死亡率很高。早期诊断和及时抗真菌治疗至关重要,但由于临床表现不明显,准确识别病原菌对选择适当的治疗方法至关重要。在新生儿真菌感染中,白色念珠菌占大多数,由于对不同菌种的敏感性不同,因此需要采取不同的治疗方法。曲霉菌是另一种重要的病原体,具有很高的致死风险,可通过皮肤或全身感染。马拉色菌(Malassezia)虽然不常见,但主要影响导管相关真菌血症的早产儿。其他真菌种类,包括接合菌、三孢子菌和隐球菌,很少造成新生儿感染,但也值得考虑。尽管许多抗真菌药物在新生儿中的临床药理信息相对匮乏,但真菌感染的治疗至关重要。我们回顾了主要的抗真菌药物(如两性霉素 B、棘白菌素类、唑类),并提供了药理和剂量信息。最后,预防策略(包括使用严格的无菌技术和仔细的临床监测)对于降低新生儿和婴儿出生后最初几个月内此类感染的发生率和严重程度至关重要。
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引用次数: 0
Macitentan: Pediatric First Approval.
IF 3.4 3区 医学 Q1 PEDIATRICS Pub Date : 2025-03-15 DOI: 10.1007/s40272-025-00692-8
Susan J Keam

Macitentan (Opsumit®), an endothelin receptor antagonist (ERA) developed by Johnson & Johnson, is well established worldwide as monotherapy or combination therapy for the long-term treatment of pulmonary arterial hypertension (PAH). In September 2024, based on phase 3 clinical data in patients aged < 18 years, macitentan received its first pediatric approval in the EU as monotherapy or in combination for the long-term treatment of PAH in pediatric patients aged 2 years to < 18 years with WHO functional class (FC) II to III. Macitentan has also been approved in the UK for this indication. This article summarizes the milestones in the development of macitentan leading to the first pediatric approval for the long-term treatment of PAH.

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引用次数: 0
Diagnosis and Oral Sirolimus Treatment of Fibro-Adipose Vascular Anomaly in Pediatric Patients: A Case Series and Comprehensive Review. 小儿纤维脂肪血管异常的诊断和口服西罗莫司治疗:病例系列和全面回顾。
IF 3.4 3区 医学 Q1 PEDIATRICS Pub Date : 2025-03-08 DOI: 10.1007/s40272-025-00686-6
Rui He, Jie Yin, Nan Zhang, Yanni Wang, Yun Peng, Bin Zhang

Background: Fibro-adipose vascular anomaly (FAVA) is a rare and complex vascular malformation that, to date, has hardly been studied, especially in children. The diagnosis and management of FAVA is complicated, and no treatment guidelines have yet been published.

Objectives: This study aimed to analyze the clinical manifestations and diagnostic and genetic evidence of FAVA and to explore safe and effective treatment with sirolimus in pediatric patients.

Methods: We retrospectively analyzed the clinical manifestations and examination data of 18 pediatric patients with FAVA who presented at the Vascular Anomaly Center from September 2019 to February 2023 and summarized the basis on which a diagnosis of FAVA was made. A genetic examination was completed in five cases. A total of 12 cases were treated with oral sirolimus. We analyzed changes in skin lesions before and after treatment and recorded the occurrence of adverse reactions.

Results: Of the 18 patients, 15 were girls and 3 were boys. Most lesions (15 cases) were in the lower extremities, accompanied by varying degrees of chronic pain, functional impairment, contractures, and other functional disorders. Imaging findings can be divided into three categories: focal, focal infiltrative, and diffuse. Histopathological manifestations were malformed vascular fibro-adipose tissue. A genetic examination of five cases identified a PIK3CA somatic mutation. After oral sirolimus treatment, pain and dysfunction associated with the lesions were significantly improved, the lesion volume dramatically diminished, and no obvious adverse reactions occurred.

Conclusions: With the help of imaging, and histopathological and somatic genetic examinations, FAVA can be promptly diagnosed and treated to avoid serious dysfunction. The efficacy and safety of oral sirolimus in the treatment of FAVA deserves further study.

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引用次数: 0
Cardioprotective Effect of Nigella sativa in Pediatric Patients with Type 1 Diabetes Mellitus: A Randomized Controlled Study.
IF 3.4 3区 医学 Q1 PEDIATRICS Pub Date : 2025-03-06 DOI: 10.1007/s40272-025-00687-5
Dalia El-Afify, Doaa El Amrousy

Background and objective: Nigella sativa is a widely used medicinal plant with several potential therapeutic uses. This study aimed to investigate the possible beneficial cardioprotective effect of Nigella sativa in pediatric patients with type 1 diabetes mellitus.

Methods: Sixty children and adolescents with type 1 diabetes were randomized into two groups: group I (n = 30) who received Nigella sativa seed oil 450 mg twice daily after meals for 3 months in addition to insulin, and group II (n = 30) who received insulin alone. Echocardiographic examinations were performed before and after the treatment. The lipid profile, malondialdehyde, nitric oxide, tumor necrosis factor-α, transforming growth factor-β, and troponin I were also measured before and after Nigella sativa treatment.

Results: After 3 months of Nigella sativa administration, group I had significantly lower cholesterol and low-density lipoprotein-cholesterol, malondialdehyde, nitric oxide, tumor necrosis factor-α, transforming growth factor-β, and troponin I levels compared with their pretreatment levels and compared with group II. In addition, group I had a significantly higher left ventricular E'/A' ratio and two-dimensional left ventricular global longitudinal strain (2D-LV GLS) compared with baseline values and compared with group II after treatment.

Conclusions: Nigella sativa can improve subclinical left ventricular dysfunction in pediatric patients with type 1 diabetes mellitus.

Clinical trial registration: this clinical trial was registered at www.pactr.org with ID: PACTR202302478939306.

{"title":"Cardioprotective Effect of Nigella sativa in Pediatric Patients with Type 1 Diabetes Mellitus: A Randomized Controlled Study.","authors":"Dalia El-Afify, Doaa El Amrousy","doi":"10.1007/s40272-025-00687-5","DOIUrl":"https://doi.org/10.1007/s40272-025-00687-5","url":null,"abstract":"<p><strong>Background and objective: </strong>Nigella sativa is a widely used medicinal plant with several potential therapeutic uses. This study aimed to investigate the possible beneficial cardioprotective effect of Nigella sativa in pediatric patients with type 1 diabetes mellitus.</p><p><strong>Methods: </strong>Sixty children and adolescents with type 1 diabetes were randomized into two groups: group I (n = 30) who received Nigella sativa seed oil 450 mg twice daily after meals for 3 months in addition to insulin, and group II (n = 30) who received insulin alone. Echocardiographic examinations were performed before and after the treatment. The lipid profile, malondialdehyde, nitric oxide, tumor necrosis factor-α, transforming growth factor-β, and troponin I were also measured before and after Nigella sativa treatment.</p><p><strong>Results: </strong>After 3 months of Nigella sativa administration, group I had significantly lower cholesterol and low-density lipoprotein-cholesterol, malondialdehyde, nitric oxide, tumor necrosis factor-α, transforming growth factor-β, and troponin I levels compared with their pretreatment levels and compared with group II. In addition, group I had a significantly higher left ventricular E'/A' ratio and two-dimensional left ventricular global longitudinal strain (2D-LV GLS) compared with baseline values and compared with group II after treatment.</p><p><strong>Conclusions: </strong>Nigella sativa can improve subclinical left ventricular dysfunction in pediatric patients with type 1 diabetes mellitus.</p><p><strong>Clinical trial registration: </strong>this clinical trial was registered at www.pactr.org with ID: PACTR202302478939306.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143567885","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Prevalence and Temporal Trends of Attention Deficit Hyperactivity Disorder Medication Fills During Pregnancy and Breastfeeding in Denmark. 丹麦孕期和哺乳期注意缺陷多动障碍药物填充的患病率和时间趋势。
IF 3.4 3区 医学 Q1 PEDIATRICS Pub Date : 2025-03-01 Epub Date: 2025-01-13 DOI: 10.1007/s40272-024-00671-5
Malene Galle Madsen, Jin Liang Zhu, Trine Munk-Olsen, Theresa Wimberley, Henrik Larsson, Anna-Sophie Rommel, Xiaoqin Liu, Mette-Marie Zacher Kjeldsen, Sarah Kittel-Schneider, Veerle Bergink, Kathrine Bang Madsen

Background and objectives: Females of reproductive age are increasingly using attention deficit hyperactivity disorder (ADHD) medication, but its use during pregnancy and breastfeeding is largely unknown. The aim of this study is to examine the prevalence of ADHD medication fills during pregnancy and breastfeeding, including characteristics of these females and cohort differences over time.

Methods: We conducted a descriptive study using Danish nationwide registers. Within cohorts of pregnant and breastfeeding females, we calculated the prevalence of ADHD medication (methylphenidate, amphetamine, dexamfetamine, lisdexamfetamine, modafinil, atomoxetine, clonidine and guanfacine) fills and described sociodemographic and clinical characteristics across groups with fills, no fills and previous fills. Cohort differences in ADHD medication fills during pregnancy for 2005-2010, 2011-2016 and 2017-2022 were examined.

Results: In this cohort of 1,077,279 pregnancies, ADHD medication fills increased from 0.08 to 7.71 per 1000 individuals between 2005 and 2022. Among 446,485 breastfeeding females, fills increased from 0.55 to 3.67 per 1000 individuals from 2012 to 2022. Compared with the group with no fills, females filling ADHD medication during pregnancy and breastfeeding were younger, had lower levels of education, were more often smoking during pregnancy, utilised more psychiatric healthcare and had concurrent fills of other psychotropic medication. Cohort differences over time revealed that females filling ADHD medication during pregnancy in 2017-2022 were older, had higher levels of education, smoked less during pregnancy, had fewer psychiatric contacts and were less likely to fill other psychotropic medications compared with females in the earlier cohorts.

Conclusions: Results showed an increasing prevalence of ADHD medication fills during pregnancy and breastfeeding in Denmark over time, surpassing the increase observed generally in females of reproductive age filling ADHD medication. Results revealed a difference in characteristics of females filling ADHD medication during pregnancy over time, suggesting a shift in pregnancy treatment patterns.

背景和目的:育龄女性越来越多地使用注意缺陷多动障碍(ADHD)药物,但其在怀孕和哺乳期间的使用情况在很大程度上是未知的。本研究的目的是检查怀孕和哺乳期间ADHD药物填充的患病率,包括这些女性的特征和随时间的队列差异。方法:我们使用丹麦全国登记册进行了一项描述性研究。在怀孕和哺乳期女性队列中,我们计算了ADHD药物(哌甲酯、安非他明、地塞米安、利地塞米安、莫达非尼、托莫西汀、克拉定和胍法辛)填充的患病率,并描述了填充组、未填充组和以前填充组的社会人口学和临床特征。研究了2005-2010年、2011-2016年和2017-2022年妊娠期间ADHD药物填充的队列差异。结果:在该队列的1,077,279例妊娠中,ADHD药物填充率在2005年至2022年间从0.08 / 1000增加到7.71 / 1000。在446485名母乳喂养的女性中,从2012年到2022年,每1000人的填充物从0.55增加到3.67。与没有服用ADHD药物的女性相比,在怀孕和哺乳期间服用ADHD药物的女性更年轻,受教育程度更低,在怀孕期间更经常吸烟,使用更多的精神保健,同时服用其他精神药物。随着时间的推移,队列差异显示,与早期队列中的女性相比,在2017-2022年怀孕期间服用ADHD药物的女性年龄更大,受教育程度更高,怀孕期间吸烟更少,精神病接触更少,服用其他精神药物的可能性更小。结论:结果显示,随着时间的推移,丹麦怀孕和哺乳期间ADHD药物填充的患病率越来越高,超过了育龄女性填充ADHD药物的普遍增长。结果显示,女性在怀孕期间服用ADHD药物的特征随着时间的推移而有所不同,这表明怀孕治疗模式发生了转变。
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引用次数: 0
Dexmedetomidine for Less Invasive Surfactant Administration: Another Promising Medicine in an Ever-Growing Toolbox? 右美托咪定用于微创表面活性剂给药:在不断增长的工具箱中另一个有前途的药物?
IF 3.4 3区 医学 Q1 PEDIATRICS Pub Date : 2025-03-01 Epub Date: 2025-01-08 DOI: 10.1007/s40272-024-00678-y
Christian A Maiwald, Karel Allegaert
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引用次数: 0
Visceral Pain in Preterm Infants with Necrotizing Enterocolitis: Underlying Mechanisms and Implications for Treatment. 坏死性小肠结肠炎早产儿的内脏疼痛:潜在的机制和治疗意义。
IF 3.4 3区 医学 Q1 PEDIATRICS Pub Date : 2025-03-01 Epub Date: 2025-01-03 DOI: 10.1007/s40272-024-00676-0
Judith A Ten Barge, Gerbrich E van den Bosch, Rebeccah Slater, Nynke J van den Hoogen, Irwin K M Reiss, Sinno H P Simons

Necrotizing enterocolitis (NEC) is a relatively rare but very severe gastrointestinal disease primarily affecting very preterm infants. NEC is characterized by excessive inflammation and ischemia in the intestines, and is associated with prolonged, severe visceral pain. Despite its recognition as a highly painful disease, current pain management for NEC is often inadequate, and research on optimal analgesic therapy for these patients is lacking. Insight into the mechanisms underlying intestinal pain in infants with NEC-visceral pain-could help identify the most effective analgesics for these vulnerable patients. Therefore, this comprehensive review aims to provide an overview of visceral nociception, including transduction, transmission, modulation, and experience, and discuss the implications for analgesic therapy in preterm infants with NEC. The transmission of visceral pain differs from that of somatic pain, contributing to the diffuse nature of visceral pain. Studies evaluating the effectiveness of analgesics for treating visceral pain in infants are scarce. However, research in visceral pain models highlights agents that may be particularly effective for treating visceral pain based on their mechanisms of action. Further research is necessary to determine whether agents that have shown promise for treating visceral pain in preclinical studies and adults are effective in infants with NEC as well.

坏死性小肠结肠炎(NEC)是一种相对罕见但非常严重的胃肠道疾病,主要影响极早产儿。NEC的特点是肠道过度炎症和缺血,并伴有长期严重的内脏疼痛。尽管NEC被认为是一种非常痛苦的疾病,但目前对NEC的疼痛管理往往不足,并且缺乏对这些患者的最佳镇痛治疗的研究。深入了解nec婴儿肠道疼痛(内脏疼痛)的潜在机制,可以帮助确定对这些易感患者最有效的镇痛药。因此,本综述旨在提供内脏痛觉的概述,包括传导、传递、调节和经验,并讨论对NEC早产儿镇痛治疗的影响。内脏疼痛的传递不同于躯体疼痛,这有助于内脏疼痛的弥漫性。评估镇痛药治疗婴儿内脏疼痛的有效性的研究很少。然而,内脏疼痛模型的研究强调了基于其作用机制的药物可能对治疗内脏疼痛特别有效。需要进一步的研究来确定在临床前研究和成人中显示出治疗内脏疼痛的药物是否对NEC婴儿有效。
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引用次数: 0
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Pediatric Drugs
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