Clinical and Experimental Pediatrics最新文献

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.

Background: Cholestasis is characterized by disrupted bile flow and can lead to severe liver disease in newborns, of which biliary atresia (BA) is a common cause. The gut microbiome plays a crucial role in aggravating liver injury in BA and non-BA cholestasis. However, information is lacking regarding the differences in gut microbiome composition between patients with BA and non-BA cholestasis.

Purpose: This study aimed to assess the gut microbiome profile of infants with BA versus those with non-BA cholestasis and healthy controls in an Indonesian population.

Methods: We investigated the changes in the microbial composition of fecal samples from 12 infants with BA and 8 with non-BA cholestasis and compared them with those of 8 age-matched healthy controls (HCs). Fecal DNA from all the participants was subjected to 16S rRNA amplicon sequencing.

Results: The fecal microbiome at the phylum level differed between the BA and non-BA cholestasis groups with increased Proteobacteria and decreased Firmicutes. At the genus level, the BA group was enriched with Bacteroides, unclassified Enterobacteriaceae, and Dialister (P<0.05), whereas the non-BA group was enriched with Klebsiella, Chryseobacterium, Acinetobacter, and Pseudomonas (P<0.05). Parabacteroides, unclassified Lachnospiraceae, Actinomyces, Anaerococcus, Clostridium innocuum group, Collinsella, Gemella, and Peptostreptococcaceae (P<0.05) were more enriched in the HC than in the other 2 groups. Detected cytomegalovirus in fecal samples was associated with significant microbial shifts, including increased Lactobacillus, decreased Escherichia-Shigella, and altered Faith's phylogenetic diversity, highlighting its potential role in gut microbiome modulation. Microbial alterations in patients with BA versus non-BA cholestasis were significantly correlated with liver function indicators.

Conclusion: The BA and non-BA groups showed specific genus enrichment, highlighting the urgent need to identify potential treatments to inhibit the progression of liver injury in infants with cholestasis.

Background: Pediatric chylothorax poses management challenges, with conservative treatment as the first-line approach and surgery reserved for persistent cases. However, data remain limited on factors related to conservative treatment success and optimal surgical timing.

Purpose: This study aimed to evaluate the success rate of conservative treatment, identify the associated factors, and determine the optimal timing for surgical intervention.

Methods: We retrospectively reviewed pediatric chylothorax cases at Siriraj Hospital treated between January 2012 and December 2022. Clinical and laboratory parameters, treatment modalities, and outcomes were analyzed.

Results: Thirty pediatric patients were included (median age, 1.7 months); 67% were male. Conservative treatment had an overall success rate of 83.3% (25 of 30) and a 95% (19 of 20) success rate among patients with chylothorax after congenital heart disease surgery. Among the factors associated with successful conservative treatment, chylothorax caused by surgery was associated with a significantly higher success rate than nonsurgical causes (91.7% vs. 50%, respectively; P=0.04). The successful group exhibited a significantly lower peak pleural fluid flow rate than the unsuccessful group (26.8 mL/kg/day vs. 91 mL/kg/day, P=0.002). A time-to-event analysis showed that the success rate of conservative treatment for postsurgical chylothorax was 78% at 14 days, suggesting that 2 weeks may be the optimal timing for surgical intervention.

Conclusion: Conservative treatment is an effective first-line treatment for pediatric chylothorax. The etiology and peak flow rate of pleural fluid drainage are associated with its success. Optimized surgical intervention timing is crucial for improving outcomes.

Background: Rickets is a growth disorder that imposes a global health burden and causes disability in affected children. However, issues related to the clinical epidemiology and mortality risk of nutritional versus hereditary rickets have not been fully investigated in large population studies, particularly in Asia.

Purpose: This study aimed to investigate the nationwide incidence, demographic characteristics, and mortality-related risk factors of pediatric rickets stratified by nutritional and hereditary subtypes.

Methods: This study utilized data of subjects aged 0-18 years taken from Taiwan's National Health Insurance Research Database. The database includes records of 31,488,321 individuals from January 1, 2008, to December 31, 2018. We analyzed all cases and conducted subgroup analyses of nutritional and hereditary rickets to examine how different etiologies affect the risk of mortality (ROM).

Results: Among the 1,551 patients with rickets, nutritional rickets accounted for twice as many cases as hereditary rickets. Nutritional rickets primarily affects preschoolers without sex-based differences, whereas hereditary rickets is often diagnosed later with a male predominance. ROM in rickets is associated with a low household income, anemia, chronic kidney disease (CKD), hyperparathyroidism secondary to renal tubulopathy, and a prolonged length of hospital stay (LOS). Between 2012 and 2018, the overall incidence of rickets increased and the mortality rates decreased.

Conclusion: Increasing incidence and decreasing mortality rates of rickets were noted, suggesting improvements in clinical awareness and disease management. influencing ROM, such as family income, anemia, CKD, hyperparathyroidism secondary to renal tubulopathy, and LOS are important considerations in the clinical care of rickets.

Background: Positional plagiocephaly (PP) is a common cranial asymmetry of infancy. Its treatment options include conservative management and helmet therapy. However, the efficacy of each, particularly at achieving a normal cranial shape, remains uncertain.

Purpose: This study aimed to compare the efficacy of conservative management and helmet therapy for PP.

Methods: We retrospectively analyzed 199 infants with PP treated in 2015-2024. A total of 72 patients with a minimum treatment duration of 90 days and minimum plagiocephaly severity level of 2 (Children's Healthcare Atlanta Plagiocephaly Severity Scale) were included. Of them, 36 received conservative management and 36 received helmet therapy. Each infant underwent three-dimensional surface scanning of the cranium (StarScanner).

Results: The mean±standard deviation age at treatment initiation was 31.9±6.6 weeks in the helmet group versus 21.0±5.7 weeks in the conservative management group (P<0.001). The average treatment duration was 21.9 (interquartile range [IQR], 15.3-31.4) weeks vs. 20.6 (IQR, 14.1-26.6) weeks (P=0.171), respectively. The monthly correction speed of the cranial vault asymmetry index (CVAI) was comparable between groups (0.66±2.09 vs. 0.64±0.55, P=0.964). Plagiocephaly degree was reduced to level 1 in 9 of 36 patients (25%) who received helmet therapy versus 4 of 36 patients (11%) in the conservative management group (P=0.220), whereas a reduction in severity level was observed in 24 of 36 (67%) versus 15 of 36 (42%), respectively (P=0.058). In the helmet group, an earlier treatment initiation was significantly associated with a greater severity level reduction (r=-0.480, P=0.003). A longer treatment duration showed a trend toward a greater reduction in CVAI (r=0.331, P=0.052). In the conservative management group, both earlier treatment initiation (r=-0.537, P<0.001) and longer treatment duration (r=0.381, P=0.022) correlated significantly with improved outcomes.

Conclusion: Conservative management and helmet therapy reduced cranial asymmetry with no significant difference in correction speed. An early treatment initiation was the strongest predictor of improvement, while a longer treatment duration was associated with better outcomes. A trend toward a greater reduction in severity level was observed with helmet therapy, suggesting its potential benefits in more severe cases.

Respiratory syncytial virus (RSV) is a leading cause of acute lower respiratory tract infections (LRTIs) in infants and young children. In April 2024, the Korea Ministry of Food and Drug Safety approved nirsevimab (Beyfortus), a long-acting monoclonal antibody, as a passive immunization to prevent RSV-associated LRTI among infants and young children. Nirsevimab was launched in Korea in February 2025. This report summarizes the recommendations of the Committee on Infectious Diseases of the Korean Pediatric Society regarding the use of nirsevimab. We recommend a single dose of nirsevimab for all neonates born during the RSV season (October to March), as well as all infants younger than 6 months at the start of the RSV season (i.e., those born between April and September of that year). Nirsevimab should be administered shortly after birth (within the first week of life) to neonates born during the RSV season and just before or early in the season (late September to October) to infants entering their first RSV season. Nirsevimab may also be considered for children younger than 2 years of age who are at increased risk of severe RSV disease and entering their second RSV season.

Background: Critically ill children are at risk of postoperative malnutrition. Thus, optimal nutritional therapy is essential for preventing morbidity development and reducing mortality rates among this population. An adequate protein intake increases anabolism. However, data on the effect of enteral protein supplementation on nitrogen balance (NB) and intestinal fatty acid-binding protein (I-FABP) levels in postoperative critically ill children remain limited.

Purpose: This study aimed to analyze whether an increased protein intake via enteral nutrition improves NB and reduces serum I-FABP levels among postoperative critically ill children.

Methods: This double-blind randomized controlled trial examined critically ill children aged 1-5 years who received early postoperative enteral nutrition. A total of 76 subjects were randomized into a standard-protein group (3.0 g/100 mL) or a high-protein group (4.35 g/100 mL). NB was assessed on days 1 and 3, while I-FABP levels were measured before and after 72 h of enteral feeding.

Results: The high-protein group showed a significantly greater increase in average NB (283.4 [standard deviation, 82.5] mg/kg/day) compared to the standard-protein group (114.7 [standard deviation, 53] mg/kg/day) (P<0.0001). However, no significant decrease in I-FABP levels was noted in either group despite the above- and below-average NB improvements.

Conclusion: High-protein enteral supplementation improves NB in postoperative critically ill children without causing adverse side effects.

Background: Homocysteine metabolism is crucial to maintaining vascular and metabolic homeostasis, yet its dysregulation in pediatric β-thalassemia major (β-TM) remains poorly understood.

Purpose: This study investigated the prevalence and determinants of hyperhomocysteinemia in pediatric β-TM with a focus on vitamin B9 (folate), B12, and B6 deficiencies, oxidative stress marker levels, and the impact of splenectomy.

Methods: A cross-sectional study was conducted of 92 pediatric β-TM patients. Levels of plasma homocysteine, vitamins B9, B12, and B6, and oxidative stress marker (protein carbonyls, thiols, nitrotyrosine, and nitric oxide metabolites) levels were measured. The patients were grouped based on their splenectomy status. The MTHFR C677T polymorphism was genotyped in a subset of patients (n=39). The statistical analyses included t tests, analysis of variance, Pearson's correlation, and multivariate regression.

Results: Overall, 93% of patients had hyperhomocysteinemia (≥15 μM), with the values of 50% exceeding 30 μM. Homocysteine levels were negatively correlated with folate levels (r=-0.22, P=0.03) and weakly correlated with B12 levels (r=-0.18, P=0.08). Vitamin B6 levels were not significantly associated with homocysteine levels. Post-splenectomy, patients had significantly higher homocysteine levels (43.3 μM vs. 32.3 μM, P=0.002) but lower nitrotyrosine levels (P=0.035), suggesting reduced nitrative stress. The MTHFR C677T genotype did not significantly influence homocysteine levels in our cohort.

Conclusion: Hyperhomocysteinemia is prevalent in pediatric β-TM, driven primarily by severe folate and B12 deficiencies. Splenectomy exacerbates hyperhomocysteinemia but reduces nitrative stress, indicating complex metabolic shifts postsplenectomy. These findings highlight the need for routine homocysteine monitoring and targeted vitamin supplementation to mitigate the potential vascular risks of pediatric thalassemia.