Search Results (132)

Pediatric asthma is a multifactorial and heterogeneous disease determined by the dynamic interplay of genetic susceptibility, environmental exposures, and immune dysregulation. Recent advances have highlighted the pivotal role of epigenetic mechanisms, in particular, DNA methylation, histone modifications, and non-coding RNAs, in the regulation of inflammatory pathways contributing to asthma phenotypes and endotypes. This review examines the role of respiratory viruses such as respiratory syncytial virus (RSV), rhinovirus (RV), and other bacterial and fungal infections that are mediators of infection-induced epithelial inflammation that drive epithelial homeostatic imbalance and induce persistent epigenetic alterations. These alterations lead to immune dysregulation, remodeling of the airways, and resistance to corticosteroids. A focused analysis of T2-high and T2-low asthma endotypes highlights unique epigenetic landscapes directing cytokines and cellular recruitment and thereby supports phenotype-specific aspects of disease pathogenesis. Additionally, this review also considers the role of miRNAs in the control of post-transcriptional networks that are pivotal in asthma exacerbation and the severity of the disease. We discuss novel and emerging epigenetic therapies, such as DNA methyltransferase inhibitors, histone deacetylase inhibitors, miRNA-based treatments, and immunomodulatory probiotics, that are in preclinical or early clinical development and may support precision medicine in asthma. Collectively, the current findings highlight the translational relevance of including pathogen-related biomarkers and epigenomic data for stratifying pediatric asthma patients and for the personalization of therapeutic regimens. Epigenetic dysregulation has emerged as a novel and potentially transformative approach for mitigating chronic inflammation and long-term morbidity in children with asthma. Full article

Background/Objectives: Medulloblastoma is the most common malignant brain tumor in children and comprises four molecular subtypes—WNT, SHH, Group 3, and Group 4—each with distinct genetic, epigenetic, and metabolic features. Increasing evidence highlights the critical role of metabolic reprogramming and epigenetic alterations in driving tumor progression, therapy resistance, and clinical outcomes. This review aims to explore the interplay between metabolic and epigenetic mechanisms in medulloblastoma, with a focus on their functional roles and therapeutic implications. Methods: A comprehensive literature review was conducted using PubMed and relevant databases, focusing on recent studies examining metabolic pathways and epigenetic regulation in medulloblastoma subtypes. Particular attention was given to experimental findings from in vitro and in vivo models, as well as emerging preclinical therapeutic strategies targeting these pathways. Results: Medulloblastoma exhibits metabolic adaptations such as increased glycolysis, lipid biosynthesis, and altered amino acid metabolism. These changes support rapid cell proliferation and interact with the tumor microenvironment. Concurrently, epigenetic mechanisms—including DNA methylation, histone modification, chromatin remodeling, and non-coding RNA regulation—contribute to tumor aggressiveness and treatment resistance. Notably, metabolic intermediates often serve as cofactors for epigenetic enzymes, creating feedback loops that reinforce oncogenic states. Preclinical studies suggest that targeting metabolic vulnerabilities or epigenetic regulators—and particularly their combination—can suppress tumor growth and overcome resistance mechanisms. Conclusions: The metabolic–epigenetic crosstalk in medulloblastoma represents a promising area for therapeutic innovation. Understanding subtype-specific dependencies and integrating biomarkers for patient stratification could facilitate the development of precision medicine approaches that improve outcomes and reduce long-term treatment-related toxicity in pediatric patients. Full article

This narrative review explores the integration of theranostics and artificial intelligence (AI) in neuro-oncology, addressing the urgent need for improved diagnostic and treatment strategies for brain tumors, including gliomas, meningiomas, and pediatric central nervous system neoplasms. A comprehensive literature search was conducted through PubMed, Scopus, and Embase for articles published between January 2020 and May 2025, focusing on recent clinical and preclinical advancements in personalized neuro-oncology. The review synthesizes evidence on novel theranostic agents—such as Lu-177-based radiopharmaceuticals, CXCR4-targeted PET tracers, and multifunctional nanoparticles—and highlights the role of AI in enhancing tumor detection, segmentation, and treatment planning through advanced imaging analysis, radiogenomics, and predictive modeling. Key findings include the emergence of nanotheranostics for targeted drug delivery and real-time monitoring, the application of AI-driven algorithms for improved image interpretation and therapy guidance, and the identification of current limitations such as data standardization, regulatory challenges, and limited multicenter validation. The review concludes that the convergence of AI and theranostic technologies holds significant promise for advancing precision medicine in neuro-oncology, but emphasizes the need for collaborative, multidisciplinary research to overcome existing barriers and enable widespread clinical adoption. Full article

Advances in molecular diagnostics have enabled precision medicine approaches in pediatric neuro-oncology, with small-molecule drugs emerging as promising therapeutic candidates targeting specific genetic and epigenetic alterations in central nervous system (CNS) tumors. This review provides a focused overview of several small-molecule agents under investigation or in early clinical use, including ONC201, tazemetostat, vorasidenib, CDK inhibitors, selinexor, and aurora kinase A inhibitors, among others. Highlighted are their mechanisms of action, pharmacokinetic properties, early efficacy data, and tolerability in pediatric populations. Despite encouraging preclinical and early-phase results, most agents face limitations due to study heterogeneity, lack of large-scale pediatric randomized trials, and challenges in drug delivery to the CNS. The review underscores the critical need for robust prospective clinical trials for the integration of these therapies into pediatric neuro-oncology care. Full article

Climate change and air pollution are reshaping viral circulation patterns and increasing host vulnerability, amplifying the burden of respiratory illness in early childhood. This narrative review synthesizes current evidence on how environmental exposures, particularly to nitrogen dioxide, ozone, and fine particulate matter, contribute to the incidence and severity of bronchiolitis, with a focus on biological mechanisms, epidemiological trends, and public health implications. Bronchiolitis remains one of the leading causes of hospitalization in infancy, with Respiratory Syncytial Virus (RSV) being responsible for the majority of severe cases. Airborne pollutants penetrate deep into the airways, triggering inflammation, compromising mucosal defenses, and impairing immune function, especially in infants with pre-existing vulnerabilities. These interactions can intensify the clinical course of viral infections and contribute to more severe disease presentations. Children in urban areas exposed to high levels of traffic-related emissions are disproportionately affected, underscoring the need for integrated public health interventions. These include stricter emission controls, urban design strategies to reduce exposure, and real-time health alerts during pollution peaks. Prevention strategies should also address indoor air quality and promote risk awareness among families and caregivers. Further research is needed to standardize exposure assessments, clarify dose–response relationships, and deepen our understanding of how pollution interacts with viral immunity. Bronchiolitis emerges as a sentinel condition at the crossroads of climate, environment, and pediatric health, highlighting the urgent need for collaboration across clinical medicine, epidemiology, and environmental science. Full article

Background: Residency training is a formative and rigorous experience, with burnout rates reported at 76%. Formal peer support groups have shown improvement in burnout among healthcare workers with anxiety and depression. Objective: Implement a peer support program for emergency medicine (EM) residents and characterize utilization of metrics by demographics, burnout rates of participants, and overall session impact. Methods: An IRB-approved, longitudinal, prospective cohort study of 73 EM and EM/Pediatrics residents post-graduate year (PGY) 1–5 from July 2021–June 2022 was performed. Resident peer leaders were trained using a novel curriculum to lead peer support groups. Residents were invited to participate in biweekly sessions, with optional pre- and post-session surveys measuring demographics, burnout, themes discussed, and how they felt after sessions (Patients’ Global Impression of Change scale). Results: There were 134 attendances over 20 sessions, averaging 6 residents per session. Of 73 total residents, 37 (50%) participated at least once. All levels of training were represented, with half being female, 20% underrepresented in medicine, and 14% LGBTQ+. Overall burnout rates were unchanged for first-time attendances (49%, n = 18) vs. recurrent (50%, n = 11). Females had higher burnout at both baseline (60%, n = 15) and recurrent sessions (69%, n = 13). Following sessions, 94% of participants reported feeling immediately better and 100% of leaders felt prepared leading peer support sessions. Conclusions: This study demonstrates that residents utilize peer support, with many returning more than once. Despite stable burnout rates, 94% of participants felt immediately better after the session, suggesting that peer support is a valuable resource for residents actively experiencing burnout. Full article

Background/Objectives: A 2-year-old male presented to the emergency department (ED) with vomiting and abdominal discomfort following ingestion of multiple magnets from a sibling’s bracelet. This case highlights the risks associated with magnet ingestion and the need for coordinated multidisciplinary care and public health intervention. Methods: Radiographs revealed magnets in the oropharynx, stomach, and small bowel. Emergency physicians coordinated care with otolaryngology, gastroenterology, and general surgery. Results: Laryngoscopy successfully removed two magnets from the uvula, and endoscopy retrieved 30 magnets from the stomach. General surgery performed a diagnostic laparoscopy, identifying residual magnets in the colon. Gastroenterology attempted a colonoscopy but was unable to retrieve magnets due to formed stool, leading to bowel preparation and serial imaging. The patient eventually passed 12 magnets per rectum without surgical intervention. Conclusions: This case emphasizes the importance of multidisciplinary collaboration in managing magnet ingestion, a preventable cause of serious gastrointestinal injury. Recent studies highlight the increasing incidence and severity of such cases due to accessibility and inadequate regulation. These findings underscore the need for public awareness and adherence to management protocols to mitigate morbidity and mortality in pediatric patients. Full article

Fetal surgery has emerged as a viable option for the management of selected congenital anomalies that result in severe or lethal outcomes if left untreated until birth. Conditions such as spina bifida, urinary tract obstruction, congenital cystic adenomatoid malformation, diaphragmatic hernia, sacrococcygeal teratoma, and twin–twin transfusion syndrome have shown improved prognosis after in utero intervention, open, or fetoscopically. Despite significant advances in surgical methods and anesthesia, preterm labor remains a primary concern. Stem cell transplantation and in utero gene therapy are developing, and they have the potential to expand the treatment window, as they minimize maternal complications. Hematopoietic stem cell transplantation, which is based on the immaturity of the fetal immune system, is a promising treatment for inherited disorders. Although many procedures of fetal interventions are now established, their safety and efficacy must be ensured and this requires optimal patient selection and choice of appropriate timing for intervention, adherence to ethical principles, and continuous research. Therefore, a multidisciplinary team, including specialists in maternal–fetal medicine, pediatric surgery, anesthesiology, neonatology, psychosocial support, and bioethics, is essential to guide comprehensive, patient-centered care. Fetal surgery is an evolving field that offers hope for conditions previously considered untreatable before birth. Full article

Genetic dystonias are a heterogeneous group of movement disorders characterized by involuntary, sustained muscle contractions that cause repetitive movements and abnormal postures. Often beginning in childhood, they can significantly affect quality of life. Although individually rare, genetic causes are collectively relevant in pediatric dystonias, with over 250 associated genes. Among these, TOR1A, SGCE, and KMT2B are the most frequently reported in pediatric forms. Diagnosis is challenging due to the wide clinical and genetic variability. Recent advances in genetic testing, including whole-exome and whole-genome sequencing, have improved the early identification of causative variants. Functional data on selected mutations are helping to refine genotype–phenotype correlations. Management typically requires a multidisciplinary approach. Symptomatic treatments include anticholinergics, benzodiazepines, and botulinum toxin, while deep brain stimulation can be effective in refractory cases, especially in patients with TOR1A variants. Disease-modifying therapies are also emerging, such as gene therapy for AADC deficiency, highlighting the potential of precision medicine. This review provides an updated overview of pediatric genetic dystonias, with a focus on differential diagnosis and treatment strategies. Early and accurate diagnosis, together with personalized care, is key to improving outcomes in affected children. Full article

Sugammadex represents a significant advancement in neuromuscular blockade management, enabling rapid, predictable, and highly effective reversal of steroidal neuromuscular blockers such as rocuronium and vecuronium. This review critically examines recent advances in sugammadex research, particularly over the last decade, detailing its pharmacological profile, clinical efficacy, and safety compared to traditional reversal agents, like neostigmine. Its expanding clinical applications across operating rooms, critical care units, and emergency medicine are discussed, emphasizing dosing recommendations and clinical utility in special patient populations, including individuals with renal impairment, pediatric, obstetric, and obese patients. Economic considerations are explored, highlighting sugammadex’s cost-effectiveness through reduced postoperative complications and enhanced operational efficiency, despite higher initial costs. Finally, the review outlines ongoing research directions, including emerging reversal agents, advanced neuromuscular monitoring technologies, and potential future clinical applications, underscoring sugammadex’s evolving role in improving patient safety and anesthetic practice. Full article

Background/Objectives: Accurate prehospital assessment of total body surface area burned (TBSA-B) is crucial for pediatric burn management, guiding resuscitation, fluid therapy, and transfer decisions. This study evaluates the accuracy of prehospital TBSA-B estimations compared to in-hospital expert assessment and examines their impact on prehospital management. Methods: This retrospective study analyzed 104 pediatric burn cases (median 17 months; 5 days–14 years) from 2017 to 2021. The primary endpoint was the difference between prehospital TBSA-B estimation and clinical measurement, with a clinically significant discrepancy defined as >5%. Secondary endpoints included the relationship between TBSA-B estimation and fluid therapy, analgesia, and hospital stay duration. Results: Prehospital TBSA-B estimations ranged from 2% to 40% (mean: 13.9%, SD = 4.4%) with scalds being the most common burn type (90.4%). Bland–Altman analysis showed a mean TBSA-B overestimation (bias) of 6.35%, with limits of agreement ranging from −6.97% (CI: −9.42 to −4.51) to 19.67% (CI: 17.21 to 22.12). No significant patterns in overestimation were associated with age, gender, or burn location. Fluid therapy volumes were independent of prehospital TBSA-B estimates, and analgesic administration varied by gender, with girls receiving less analgesia than boys, but showed no association with burn extent or severity. Hospital stay duration correlated proportionally with in-hospital assessed TBSA-B. Conclusions: Prehospital TBSA-B estimation was systematically overestimated, yet it did not influence fluid therapy decisions. Gender differences were observed in analgesic administration, while hospital stay duration was directly related to burn extent. These findings highlight the need for improved training and standardized tools to enhance prehospital burn assessment in pediatric patients. Full article

Brain organoids are self-organized, three-dimensional (3D) aggregates derived from human embryonic stem cells, induced pluripotent stem cells, or primary organs with cell types and cellular architectures resembling those of the developing human brain. Recent studies have shown the use of region-specific brain organoids for modeling various diseases ranging from neurodevelopmental and neurodegenerative diseases to different brain cancers, which have numerous applications in fundamental research and the development of new drugs, personalized treatment, and regenerative medicine. Consequently, the use of brain organoids in drug discovery is complex and challenging and still an emerging area in this field. This review article summarizes the primary stem cells used in brain organoid generation, region-specific brain organoids, and the functional assays used in their characterization. In addition, we discuss the use of brain organoids in modeling neurodevelopmental and neurodegenerative diseases and pediatric brain cancers, as well as the application of organoids, assembloids, and tumoroids in cancer neuroscience. We further explore the recent advances in using brain organoids in high-throughput screening to improve their use for drug discovery. Full article

Artificial intelligence (AI) is rapidly transforming pediatric oncology by creating new means to improve the accuracy and efficacy of cancer diagnosis and treatment in children. This review critically examines current applications of AI technologies like machine learning (ML) and deep learning (DL) to the main types of pediatric cancers. However, the application of AI to pediatric oncology is prone to certain challenges, including the heterogeneity and rarity of pediatric cancer data, rapid technological development in imaging, and ethical concerns pertaining to data privacy and algorithmic transparency. Collaborative efforts and data-sharing schemes are important to surpass these challenges and facilitate effective training of AI models. This review also points to emerging trends, including AI-based radiomics and proteomics applications, and provides future directions to realize the full potential of AI in pediatric oncology. Finally, AI is a promising paradigm shift toward precision medicine in childhood cancer treatment, which can enhance the survival rates and quality of life for pediatric patients. Full article

Background and Objectives: Artificial intelligence (AI) has seen rapid integration into various areas of medicine, particularly with the advancement of machine learning (ML) and deep learning (DL) techniques. In pediatric orthopedics, the adoption of AI technologies is emerging but still not comprehensively reviewed. The purpose of this study is to review the latest evidence on the applications of artificial intelligence in the field of pediatric orthopedics. Materials and Methods: A literature search was conducted using PubMed and Web of Science databases to identify peer-reviewed studies published up to March 2024. Studies involving AI applications in pediatric orthopedic conditions—including spinal deformities, hip disorders, trauma, bone age assessment, and limb discrepancies—were selected. Eligible articles were screened and categorized based on application domains, AI models used, datasets, and reported outcomes. Results: AI has been successfully applied across several pediatric orthopedic subspecialties. In spinal deformities, models such as support vector machines and convolutional neural networks achieved over 90% accuracy in classification and curve prediction. For developmental dysplasia of the hip, deep learning algorithms demonstrated high diagnostic performance in radiographic interpretation. In trauma care, object detection models like YOLO and ResNet-based classifiers showed excellent sensitivity and specificity in pediatric fracture detection. Bone age estimation using DL models often matched or outperformed traditional methods. However, most studies lacked external validation, and many relied on small or single-institution datasets. Concerns were also raised about image quality, data heterogeneity, and clinical integration. Conclusions: AI holds significant potential to enhance diagnostic accuracy and decision making in pediatric orthopedics. Nevertheless, current research is limited by methodological inconsistencies and a lack of standardized validation protocols. Future efforts should focus on multicenter data collection, prospective validation, and interdisciplinary collaboration to ensure safe and effective clinical integration. Full article

Background: Considerable mortality and morbidity rates linked to AGE are well documented in the literature. Many inflammatory markers have been studied in the context of research on AGE as tools to predict the clinical course of the disease and determine the need for the use of antimicrobials. This study focuses on CRP, PCT, and WBC counts as inflammatory markers of AGE. Methods: A retrospective chart review study was conducted at King Abdullah Specialized Children’s Hospital, Jeddah. Using a non-probability consecutive sampling technique, all patients under the age of 14 diagnosed with gastroenteritis over four years (2020–2024) were included. Results: The sample population consisted of 84 individuals. Pathogen prevalence was identified in only 15%. Salmonella was the most frequently identified bacterial pathogen. While the WBC count and ESR were reassuring in most cases, the CRP and PCT measurements were almost always elevated. Compared to the stronger association observed with WBC counts, the correlation between PCT levels and ED visits were less significant. Higher CRP levels were associated with an increased use of antibiotics. Conclusion: The results of this study highlight that CRP is useful in identifying patients who are likely to have bacterial AGE and require antibiotics. Moreover, the WBC count is a helpful tool in predicting those likely to present to the ED again. Full article