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Question: Could hematopoietic stem cell transplantation (HSCT) be an alternative to enzyme replacement therapy (ERT) for type VI mucopolysaccharidosis (MPS VI)? Finding: HSCT is generally not offered due to reports of high toxicity and mortality. However, we detected fewer complications and graft-versus-host disease cases and no deaths with HSCT. Meaning: HSCT is both less expensive than ERT and permanent; thus, it should be considered an alternative treatment for MPS VI. |
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Functional gastrointestinal disorders (FGIDs) are associated with various somatic symptoms measured using a visual analogue scale and the Children’s Somatic Symptoms Inventory-24 questionnaire. Children with FGIDs exhibited more significant somatic symptoms than controls during acute illnesses. Gastrointestinal (GI) and non-GI manifestations are significantly more common in children with FGIDs. |
| Hematopoietic stem cell transplantation (HSCT) enhances the growth outcomes of pediatric patients with mucopolysaccharidosis, with early intervention leading to improved height, weight, and body mass index. However, achieving a standard adult height remains uncommon among these patients, even in cases of early HSCT. Growth hormone therapy provides short-term benefits but does not address long-term height deficits. Pubertal development is generally normal; however, precocious puberty and pubertal arrest may occur. |
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Question: Are serum amyloid A (SAA) and proadrenomedullin (proADM) levels early markers in critically ill children with sepsis? Finding: This prospective case-control study included 65 critically ill children with sepsis admitted to the pediatric intensive care unit and 31 controls. SAA and proADM levels were significantly higher in patients versus controls. Meaning: SAA and proADM are promising biomarkers for diagnosing and predicting outcomes in pediatric sepsis. |
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Question: Does the gut microbiota differ between very low birth weight (VLBW) infants with versus without bronchopulmonary dysplasia (BPD)? Finding: Common respiratory pathogens were notably elevated in the BPD group, whereas anaerobic and butyrate-producing taxa, key components of postbiotics, were dominant in the non-BPD group. Meaning: In gut-lung communication, the interplay between the intestinal and respiratory systems may implicate pro- and postbiotics in VLBW infants with BPD. |
| Protocolized sedation may reduce ventilation requirements, pediatric intensive care unit length of stay, and sedative exposure. However, it may increase the likelihood of unplanned extubation, highlighting the importance of incorporating preventive measures to mitigate this risk. |
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Question: Does the T allele of Xmn1 polymorphism favorably influence hydroxyurea efficacy in children of Eastern descent with fetal hemoglobin (HbE)-β nontransfusion dependent thalassemia (NTDT)? Finding: Decrease in transfusion requirement and increase in height following hydroxyurea therapy was noted in both groups, however, change in CT was more critical than that in CC genotype. Meaning: T allele of Xmn1 polymorphism favorably influences hydroxyurea efficacy in children with HbE-β NTDT. |
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Question: What are the most common enteric pathogens in acute diarrhea among children younger than 5 years of age, and which age group is most susceptible? Finding: Bacteria were the most common causative microorganisms of diarrhea, followed by viruses, parasites, and fungi. The 1–2-year age group was the most commonly affected. Meaning: There is a need to formulate preventive strategies targeting children exposed to enteric pathogens to limit diarrhea. |
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Question: What is the prevalence of an impaired trabecular bone score (TBS), a measure of bone microarchitecture, in adolescents with 21-hydroxylase deficiency (21OHD)? Do prednisolone and hydrocortisone affect TBS differently in this patient population? Finding: Impaired TBS was observed in 18% of participants. Prednisolone use negatively impacted TBS change. Meaning: Impaired TBS is prevalent among adolescents with 21OHD. Prednisolone impairs trabecular bone microarchitecture development. |
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Question: Do community members and health providers show different level of knowledge, attitude, and practice towards dengue vaccine? Finding: These 2 groups only differed in practice component, while the knowledge and attitude constituents were relatively low for both. Meaning: There is an urgent need to deliver educational interventions to raise awareness of community members and health providers regarding dengue vaccination. |
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Question: What are common tantrum behaviors in preschool children, and how frequently are problematic behaviors observed? Do problematic tantrums have a different emotional impact on caregivers compared to typical tantrums? Finding: Temper tantrums are common in preschool children, and verbal tantrums are the most common type. Meaning: Problematic tantrums, defined as tantrums exhibiting aggressive physical behavior, long duration (>15 minutes), or frequent occurrence (>3 days/wk), significantly affected caregivers’ emotions. |
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The incidence of childhood obesity is increasing worldwide. National surveys in Malaysia have shown similar trends. This review aimed to increase our understanding of the prevalence and associated factors of childhood overweight, obesity, and excess weight in Malaysia. A systematic review and meta-analysis were conducted of studies reporting the prevalence of overweight and obesity in Malaysian children aged <18 years.... |
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Voxelotor has promising ability to increase hemoglobin levels and reduce hemolysis markers in patients with sickle cell disease (SCD). Several preclinical and phase II/III trials have demonstrated its efficacy, dose-dependent responses, and tolerability in children. Ongoing trials are assessing its safety and effectiveness in various populations, including children younger than 12 years. These findings suggest its potential as a disease-modifying drug, warranting further exploration of its role in SCD management. |
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· The mortality rate of acute necrotizing encephalopathy was high. · Laboratory tests revealed that the fatal group had higher creatinine, lactate, activated partial thromboplastin time, thrombin time, interleukin (IL)-6, IL-10, creatine kinase, and D-dimer than survivors. · The fatal group displayed lower Glasgow Coma Scale scores and arterial pH. |
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· Milk and formula are common sources of microplastic in infants. · Water and air are the most common sources of microplastic pollution from infancy to adolescence. · Microplastic use by children of all ages can cause cell damage and affect their health. · Microplastics present in children can be quantified using a stereomicroscope and characterized using micro- Fourier transform infrared spectroscopy. |
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· Although there is broad consensus on many aspects regarding the symptoms, diagnosis, and treatment of cow's milk allergy, the impact of geographical, cultural, and socioeconomic factors remains unestablished. · Availability and cost of formula for the management of cow's milk allergy have a major impact on the therapeutic choice. · Region-specific guidelines for the treatment of cow's milk allergy are required. |
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This review aimed to determine whether pesticide exposure is associated with stunting in children. The 13 included studies agree that pesticide exposure is not correlated with stunting incidence regardless of substance type (organophosphate and pyrethroid). Heterogeneity appeared with age covariate as potential confounding. The evidence of this study is challeng-ing, as the adverse effects of pesticides grossly occurred. The protection of children is warranted for preventing future neurodevelopment issues. |
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Double-negative T (DNT) cells appear to be increased in several pediatric rheumatic diseases and this finding may be correlated with disease activity to some extent. However, due to significant heterogeneity in several methodological aspects, further investigations in rheumatic children are needed to assess the potential relevance of DNT cells as biomarkers and clarify their immunopathological role. |
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· This summary emphasizes the importance of the early diagnosis of Turner syndrome (TS) and presents a multidisciplinary approach to its prevention and management, high-lighting the need for customized care. · Advancements in immunogenetic research may improve our understanding of TS and improve its outcomes. · TS encompasses a wide array of medical challenges, including cardiovascular, endocrine, autoimmune, and mental health issues, as well as a heightened cancer risk. |
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· The safety and efficacy of the two-bag versus one-bag system for treating patients with diabetic ketoacidosis (DKA) < 21 years remains unestablished. · Our meta-analysis demonstrated similar safety outcomes but sooner DKA resolution and shorter mean response time for intravenous fluid changes for the two-bag system. · This preliminary evidence suggests that the two-bag system has some advantages in efficacy, but further studies are needed to evaluate their extent. |
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· A detailed history and comprehensive physical examination remain the cornerstones for establishing a diagnosis of global developmental delay/intellectual disability (GDD/ID). · Comprehensive surveillance and screening programs play a significant role in the early detection of GDD. · Whole-exome sequencing is highly recommended as first- or second-line testing for individuals with idiopathic GDD/ID. · Early intervention by a well-versed multidisciplinary team can significantly improve the outcomes and prognosis of GDD/ID. |
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Question: Does zinc supplementation along with probiotics affect disease severity or clinical outcomes of children with acute diarrhea? Findings: This study indicated that zinc supplementation and probiotics had no effect on clinical improvement or disease severity among pediatric patients with acute diarrhea. Meaning: Children who received probiotics plus zinc recovered faster than those who received probiotics only. |
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· We suggest offering long-term macrolides to children with noncystic fibrosis bronchiectasis with frequent exacerbations (conditional recommendation, moderate quality of evidence). · We do not recommend the routine use of mucolytic agents, inhaled corticosteroids, or nonsteroidal anti-inflammatory drugs to prevent exacerbation of bronchiectasis in children (inconclusive, very low quality of evidence). · We recommend the use of nebulized hypertonic saline to prevent exacerbations and improve the lung function of children with noncystic fibrosis bronchiectasis (weak recommendation, moderate quality of evidence). |
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Question: This systematic review attempts to discover the best magnetic resonance imaging (MRI) technique for myelin quantification in neonates by evaluating various MRI parameters and their reproducibility. Finding: Since the benefits of using synthetic MRI for quantifying myelin in neonates outweigh the very minor draw- backs, it is recommended. Meaning: The findings suggest the importance of identifying noninvasive MRI techniques available to assess myelin tissue in neonates, which aid in diagnosing neurodevelopmental disorders. |
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Question: What are the risk factors for prolonged diarrhea in children under 2 years old? Finding: History of antibiotic use, zinc deficiency, and elevated fecal alpha-1 antitrypsin levels were the main risk factors of prolonged diarrhea in children under 2 years old with acute diarrhea. Meaning: Rational antibiotic usage is necessary as well as thorough testing of serum zinc level and fecal alpha-1 antitrypsin levels. |
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· Orphan disease is a rare disease, primarily affecting newborn and children. Vast majority of orphan diseases has genetic background. · Orphan disease is individually rare. But as a whole, it is not rare, becoming a great socioeconomic burden. · The diagnosis of rare genetic disease has been problematic, but recent progress of genome analysis technologies makes it faster and more precise. · There are many unmet needs as to the curative treatment. However, the number of treatable rare diseases is growingly increasing owing to the development of biotechnology. · Most orphan drugs are extremely expensive because of numer ous hurdles during the process of drug development as well as small number of patients. |
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Several observational studies have shown that acute kidney injury affects up to 46% of children and adolescents who develop severe postinflammatory responses, such as multisystem inflammatory syndrome in childhood, due to coronavirus disease 2019 (COVID-19). Although causality has not been established, some cases of glomerulopathy or nephrotic syndrome occurring after COVID-19 infection or vaccination have been reported. Therefore, kidney complications associated with these conditions in children and adolescents warrant attention. |
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· Atopic dermatitis (AD) is characterized by a strong T helper (Th)2 response, although the extents of Th22, Th17/ interleukin (IL)-23, and Th1 responses vary among disease subtypes. · Children with moderate to severe AD may require early systemic therapy to reduce the systemic inflammation caused by increased Th2 cytokine levels. · Dupilumab, which blocks IL-4/IL-13 receptor, has equivalent efficacy for extrinsic and intrinsic AD and a favorable safety profile in infants and children aged 6 months and older. |
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· Primary pediatricians should play a key role in the diagnosis and treatment of attention-deficit/hyperactivity disorder (ADHD). · The Diagnostic and Statistical Manual of Mental Disorders, fifth edition, has lowered the diagnostic threshold for older teens and adults and a comorbid diagnosis with autism is now allowed. · The American Academy of Pediatrics had added recommendation-related comorbid conditions in its guideline and the Society of Developmental and Behavioral Pediatrics recently developed a complex ADHD guideline. · The European ADHD Guideline Group recently developed a guideline for managing ADHD during the coronavirus disease 2019 pandemic. |
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Question: This is the first meta-analysis to examine published evidence of the effectiveness of virtual reality at reducing pain during pediatric intravenous injections. Finding: Our results suggest that virtual reality effectively reduces pain associated with intravenous injections in pediatric patients. Meaning: These findings suggest the importance of virtual reality in decreasing the pain of intravenous injections among children. |
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