Clinical and Experimental Pediatrics最新文献

Background: In recent years, minimally invasive methods have been increasingly utilized for surfactant administration in spontaneously breathing preterm infants with respiratory distress syndrome (RDS) managed with nasal continuous positive airway pressure owing to their feasibility and association with improved respiratory outcomes. However, data are limited from developing countries on the use and effectiveness of these techniques.

Purpose: The primary objective of this study was to evaluate the effect of less invasive surfactant administration (LISA) and intubation-surfactant-extubation (InSurE) techniques on the need for intubation and invasive mechanical ventilation (MV) within 72 hours of surfactant administration in preterm neonates with RDS. The secondary objectives were the effects of these methods on the need for a second surfactant dose, mortality rate, and other preterm morbidities.

Methods: This retrospective observational study was conducted in Southern India over 5 years. Clinical outcomes were analyzed in neonates with RDS at 24-34 weeks' gestation who received surfactants via the LISA or InSurE method.

Results: A total of 98 neonates were divided into the LISA group (n=54) and the InSurE group (n=44). The need for intubation and MV within 72 hours was significantly lower in the LISA versus InSurE group (18% vs. 64%, P=0.04; relative risk, 0.28; 95% confidence interval, 0.16-0.53). The duration of invasive ventilation was significantly shorter in the LISA group (P<0.001). We observed no significant intergroup differences in the need for a second surfactant dose (17% vs. 7%, P=0.14), bronchopulmonary dysplasia (3.7% vs. 8.8%, P=0.49), or mortality (14.5% vs. 13%, P=0.47).

Conclusion: LISA appears to be a less invasive and more effective alternative to InSurE, demonstrating the ability to reduce the need for intubation and invasive ventilation within the first 72 hours as well as the duration of invasive support in preterm infants with RDS.

Background: Third-generation cephalosporins remain the empirical mainstay for pediatric urinary tract infections (UTIs) in Korea, yet the resistance rate now approaches 30%, thereby threatening treatment effectiveness.

Purpose: To determine whether completing a cephalosporin regimen, despite in vitro resistance, increases early UTI recurrence rates.

Methods: We retrospectively reviewed the cases of children aged <24 months with their first Gram-negative UTI admitted in 2020-2024. Three exposure groups were defined: susceptible isolates treated with a third-generation cephalosporin; resistant isolates that received ≥5 days of antibiotics to which the isolated organism was susceptible (concordant); and resistant isolates that received <5 days of appropriate antibiotic therapy (discordant). The primary outcome was UTI recurrence within 2 months. Kaplan- Meier curves were generated, while multivariate Cox models adjusted for age, fever, acute cortical defects, and kidney anomalies were used to estimate hazard ratios (HRs).

Results: Among 989 children (mean age, 4.4 months), 424 (42.9%) had cefotaxime-resistant isolates; of them, 76 (17.9%) received concordant therapy and 348 (82.1%) received discordant therapy. The overall 2-month recurrence rate was 15.4% (95% confidence interval [CI], 13.0-17.7). Compared to the susceptible group, the concordant group did not show a significantly different relapse rate (adjusted HR [aHR], 1.09; 95% CI, 0.67-1.78), whereas the discordant group demonstrated an increased recurrence risk (aHR, 1.42; 95% CI, 1.08-1.86). An analysis of culture-confirmed recurrence yielded similar findings (discordant therapy aHR, 1.82; 95% CI, 1.29-2.56). No significant differences were observed when the analysis was restricted to febrile recurrence.

Conclusion: Completing a third-generation cephalosporin course when isolates are not susceptible to thirdgeneration cephalosporins can increase early UTI recurrence rates in Korean children. Reviewing susceptibility on day 5 and switching to an active oral agent may reduce recurrence and limit unnecessary broad-spectrum antibiotic exposure.

Background: Clinical, biochemical, histological, and immunological indicators are frequently used to diagnose autoimmune hepatitis (AIH), a chronic inflammatory liver disease affecting children. Wilson disease, which resembles AIH, is mainly evaluated using serum ceruloplasmin and copper levels. However, changes in these biomarkers have also been observed in AIH, raising the question of whether they could be useful for evaluating children with AIH.

Purpose: When selecting a treatment plan and estimating the long-term prognosis of patients with AIH, assessing the liver fibrosis stage is crucial. It is also crucial to identify noninvasive indicators of liver fibrosis, for which ceruloplasmin has been suggested as a biomarker in several liver diseases. Therefore, this study aimed to investigate the potential significance of serum ceruloplasmin and copper levels for identifying liver fibrosis in children with AIH.

Methods: One hundred children with AIH treated at Menoufia University's National Liver Institute Pediatric Hepatology, Gastroenterology, and Nutrition Department were enrolled. The duration of the study was 5 years (February 2020 to February 2025). The patients' histopathological, radiographic, laboratory, and clinical data were collected. We used the revised score to diagnose AIH. A Beckman Coulter AU480 chemistry analyzer was used to measure serum copper, while an enzyme-linked immunosorbent assay was used to measure serum ceruloplasmin.

Results: Serum ceruloplasmin levels were considerably lower in patients with advanced fibrosis (F3-4) than in those without advanced fibrosis (F0-2) (P<0.001). However, in patients with extensive fibrosis, the serum copper levels were considerably elevated (P<0.001). Compared to serum copper level, which had an area under a curve of 0.939 (95% confidence interval [CI], 0.887-0.991; P<0.001) and a cutoff of >24.7 mg/dL (90.8% sensitivity, 66.9% specificity), ceruloplasmin level had an area under a curve of 0.945 (95% CI, 0.889-1.00; P<0.001), suggesting that it could be a useful tool for the detection of advanced liver fibrosis in children.

Conclusion: To estimate the long-term prognosis of patients with AIH, it is crucial to assess liver fibrosis stage. It is crucial to identify noninvasive indicators of liver fibrosis, for which ceruloplasmin has been suggested as a biomarker. Therefore, serum copper and ceruloplasmin levels may provide important information for the identification of advanced liver fibrosis in children with AIH.

Background: The postacute sequelae of coronavirus disease 2019 (PASC), also known as pediatric long coronavirus disease (COVID), can significantly affect the quality of life (QoL) of affected children. Currently, there are no standardized screening tools to identify high-risk children. The Kinder Lebensqualität fragebogen (KINDL) is a psychometric method for measuring QoL in children.

Purpose: This study used the KINDL questionnaire and Children's Somatic Symptom Inventory-24 (CSSI-24) to evaluate symptom burden and establish a screening threshold for pediatric PASC.

Methods: We performed a cross-sectional study of children diagnosed with PASC defined as the presence of at least one persistent symptom lasting more than 4 weeks after a confirmed COVID-19 infection. Symptoms were assessed using a structured checklist developed by our team. The correlation between the KINDL and CSSI-24 scores was analyzed, and receiver operating characteristic (ROC) curve analysis was used to determine the optimal KINDL cutoff for identifying high-risk cases. QoL scores were also compared with those of historical cohorts to evaluate the impact of the PASC.

Results: We included 84 participants: 46 children (mean age, 8.74±1.77 years; 41.3% girls) and 38 adolescents (mean age, 14.50±1.56 years; 44.7% girls). KINDL and CSSI-24 scores showed a significant negative correlation (r=-0.44, P<0.001), suggesting that increased somatic symptoms were associated with a reduced QoL. The ROC analysis identified a KINDL cutoff score of 74.75 (area under the curve, 0.82) for detecting high-risk children. Compared to historical cohorts, children with PASC had QoL scores comparable to child-reported norms from 2010 but lower than parent-reported norms from the same year.

Conclusion: KINDL and CSSI-24 are practical tools for screening for pediatric PASC in outpatient settings. A KINDL cutoff of 74.75 may help clinicians identify children with PASC who require early intervention. Further studies in larger and more diverse populations are required to validate these findings.

Continuous glucose monitoring (CGM) has become a key component in the management of pediatric type 1 diabetes mellitus (T1DM) since it offers real-time glucose data that facilitate tighter glycemic control and reduce acute complications. Accumulating evidence and international guidelines highlight the clinical efficacy, safety, and feasibility of CGM use in children, particularly those with high adherence. Regular CGM use is associated with significant reductions in glycated hemoglobin, fewer hypo- and hyperglycemia episodes, and improved quality of life for both patients and their caregivers. Recent advances in CGM technology-including improved accuracy, extended sensor wear, factory calibration, and customizable alerts-have enhanced their usability in pediatric populations. In addition to established CGM metrics such as time in range, time below range, and glycemic variability, a novel parameter-time in tight range (also referred as time in normoglycemia), defined as the percentage of time with blood glucose readings within 70-140 mg/dL-has emerged as a potentially more sensitive marker of optimal glycemic control in children. This review provides a comprehensive overview of CGM technologies, including device types, performance metrics, and clinical evidence supporting their use for pediatric T1DM. It also examines recent advancements in Korea such as expanded insurance reimbursement and clinical integration. As CGM becomes more accessible and technologically advanced, it is expected to play an increasingly central role in optimizing long-term outcomes for children and adolescents with T1DM.

Neonatal ichthyosis-sclerosing cholangitis syndrome (NISCH) is an autosomal recessive disorder characterized by cholestasis, generalized ichthyosis, alopecia, and dental anomalies. As this is a rare syndrome, here we present a case caused by a novel mutation followed by a literature review of all published cases. This retrospective review includes all original articles on the clinical profiles of all 37 cases published through December 2024 using a PubMed search. The patient was a 2-month-old boy who presented with cholestasis, sparse hair, and generalized ichthyosis. Whole-exome sequencing revealed a novel pathogenic variation in exon 1 of the CLDN1 gene: (NM_021101.5) c.36dupT (p.Leu13SerfsTer56). The patient was symptomatically managed, and his condition improved. Among the 37 reported cases, the median age at diagnosis was 60 months (range, 1-636 months). Patients of Moroccan ethnicity were most commonly affected, and c.200_201delTT was the most common mutation. Among the clinical features, ichthyosis was universal (37 of 37 [100%]), followed by jaundice in 70.2% (26 of 37), pruritus in 38.2% (13 of 34), hepatomegaly in 43.3% (13 of 30), and splenomegaly in 23.8% (5 of 21) of patients. Portal hypertension (7 of 35 [20%]) and mental retardation (3 of 21 [14.2%]) were rare. The disease phenotype varied from no liver involvement or transient neonatal cholestasis to end-stage liver disease. Progressive liver disease was reported in 8 patients, five of whom underwent liver transplantation. NISCH is a rare syndrome with variable phenotypes ranging from no liver involvement and transient neonatal cholestasis to advanced liver disease requiring liver transplantation. Therefore, a multidisciplinary approach with close follow-up is required.

Background: Streptococcus pyogenes (group A Streptococcus [GAS]) is a common cause of bacterial pharyngitis and skin infections in children that can lead to severe and invasive GAS (iGAS) infections. The sudden acute respiratory syndrome coronavirus 2 pandemic coincided with an increase in iGAS cases, with emerging serotypes and risk factors like age, reduced postpandemic immunity, and viral coinfections. The treatment of iGAS with clindamycin and intravenous immunoglobulins (IVIG) is not well standardized, and pediatric data are limited. Linezolid is being explored as an alternative to clindamycin, although further research is required.

Purpose: This study aimed to evaluate the treatment of iGAS in hospitalized children with focus on the effectiveness of standard treatments and the role of alternative interventions in cases of treatment failure, including the use of linezolid or severe infections. Additionally, this study sought to identify the potential risk factors for pediatric intensive care unit (PICU) admission.

Methods: A retrospective observational study was conducted in children aged <18 years admitted to Meyer University Children's Hospital (September 2022 to September 2024) with confirmed or probable iGAS. Their anonymized general information, symptoms, laboratory test results, microbiological test results, coinfections, radiological examination results, antibiotic and nonantibiotic therapies, discharge information, and outcomes were collected.

Results: Forty-six children with confirmed/probable iGAS (median age, 53.7 months) were included. Of them, 34 (73.9%) had confirmed iGAS and 12 (26.1%) had probable iGAS. Sixteen children (34.8%) with iGAS were admitted to the PICU; of them, 2 died. All children received beta-lactam antibiotics; in 5 cases (10.9%), linezolid was administered after beta-lactam and clindamycin therapy failure. Thirty patients (65.2%) underwent surgery. Of the study population, 22% had preexisting conditions and 17% had viral respiratory coinfections. Elevated C-reactive protein and procalcitonin levels were independent risk factors for PICU admission. IVIG administered to 3 patients had varying outcomes.

Conclusion: Our findings highlight how treatment strategies and disease patterns have shifted in the postpandemic period. Pneumonia with a parapneumonic abscess or empyema has emerged as the most frequent clinical presentation, with nearly half of such cases requiring second-line linezolid therapy. Linezolid may be a valuable treatment alternative after beta-lactam and clindamycin failure. IVIG has been used in severe cases but often late, warranting further investigation into its optimal application.

Autism spectrum disorder (ASD) is a neurodevelopmental condition characterized by difficulty with communication and social interactions as well as restricted or repetitive behaviors. Over the last few decades, the prevalence of ASD has increased globally, with major differences in reporting, diagnosis, and interventions between developed and developing countries. The United States (U.S.) has seen a sharp rise in diagnosed ASD cases, with a current prevalence of approximately 1 in 31 children, due to improved awareness, early screening programs, and timely intervention. The U.S. healthcare system supports early intervention services through policies such as the Individuals with Disabilities Education Act and insurance mandates for ASD coverage. However, countries in Latin America, Africa, and Asia face challenges in ASD care, including limited access, stigma, underdiagnosis, and lack of resources. The World Health Organization Caregiver Skills Training, a global initiative, and the involvement of nongovernmental organizations are gradually bridging this gap. An interprofessional approach highlighting cross-cultural research, training providers, screening tools, referral options, policy implementation, and community-based care on a global scale will help reduce disparities in ASD care among countries. Making ASD care a global public health priority could help ensure developmental and mental healthcare equity. This study compares ASD care in the U.S. to that worldwide, highlighting the importance of global collaboration for early detection, service availability, and research.

Background: Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by a low platelet counts and an increased risk of bleeding. Moreover, the apoptotic mechanisms of platelets may influence their production and lifespan.

Purpose: To assess the involvement of apoptotic markers-specifically the B-cell lymphoma protein 2 family proteins Bak and Bcl-Xl in the pathogenesis of acute primary ITP in pediatric patients, and to evaluate the impact of intravenous immunoglobulin (IVIG) therapy on their expression.

Methods: This study included 24 children with acute primary ITP and 30 healthy controls. Patients were enrolled from the Hematology and Oncology Unit of Menoufia University Hospitals, Egypt. Two peripheral blood samples were obtained from each participant: one prior to receiving IVIG therapy and the other after treatment. Platelet-rich plasma was isolated, and Bak and Bcl-Xl gene expression levels were assessed using reverse transcription quantitative polymerase chain reaction.

Results: Before treatment, Bak gene expression and Bak/Bcl-Xl expression ratio were significantly higher in patients versus controls (P=0.001 and P<0.001, respectively), whereas Bcl-Xl gene expression was significantly lower (P=0.029). After treatment, Bak gene expression and the Bak/Bcl-Xl expression ratio decreased significantly (P<0.001 and P=0.001, respectively), whereas Bcl-Xl gene expression increased significantly (P<0.001).

Conclusion: Pediatric patients with acute primary ITP exhibited a heightened proapoptotic state, as indicated by an increased Bak expression and Bak/Bcl-Xl expression ratio, as well as a reduced Bcl-Xl expression. IVIG therapy appears to mitigate this pro-apoptotic effect, suggesting its ability to restore platelet homeostasis.