Severe combined immunodeficiency (SCID) is a rare genetic disorder that causes severe infections and death in early childhood. Newborn bloodspot screening (NBS) for SCID using the T-cell receptor excision circle assay can revolutionize the early detection and treatment of infants with SCID, leading to improved quality of life and life expectancy. This systematic review aimed to examine the cost-effectiveness of... |
Background: Autoimmune hemolytic anemia (AIHA) is rare and characterized by hemolytic anemia with a positive direct antiglobulin test result after the exclusion of other causes. While adults often relapse within 1 year of first-line steroid therapy, children generally respond well. However, current treatment approaches lack substantial evidence and are primarily expert opinion–based.
Purpose: This study aimed to contribute our single-center... |
Background: Thiamin deficiency (TD) manifesting clinically as wet beriberi can significantly impair a patient's cardiac function. Children with heart disease who are receiving diuretic treatment may be at increased risk for severe clinical manifestations of TD.
Purpose: This study aimed to determine the prevalence of TD and evaluate the association between various factors with thiamin status in pediatric patients with... |
Background: In preterm newborns, neonatal respiratory distress syndrome (RDS) is among the main causes of respiratory failure and mortality. However, the effect of macrophage migration inhibitory factor (MIF) on neonatal developmental lung disease is not well documented in the literature. Moreover, little is known about the effects of growth differentiation factor-15 (GDF-15) on lung maturity in preterm infants.
Purpose: To evaluate... |
Background: Helicobacter pylori (H. pylori) infection is a common gastrointestinal pathogen associated with gastritis and peptic ulcers. The early detection of H. pylori–related gastritis is crucial to its effective management, especially in pediatric patients with dyspepsia.
Purpose: This study aimed to assess the expression of miRNA-146a and miRNA-125b as potential indicators of H. pylori–associated gastritis in children. Methods: This cross-sectional study... |
Background: Post-infectious bronchiolitis obliterans (PIBO) is a rare chronic obstructive pulmonary disease that occurs after a respiratory infection. Its diagnosis is generally based on clinical history, respiratory symptoms, and computed tomography (CT) findings.
Purpose: Here we evaluated the frequency of exacerbations, clinical progress, and inhaled corticosteroid (ICS) usage in children diagnosed with PIBO with or without comorbid bronchopulmonary dysplasia (BPD). Methods:... |
Myopia, among the most common vision disorders worldwide, is projected to affect approximately 50% of the world's population by 2050. Its prevalence is particularly high in East Asia, posing a considerable public health challenge. In particular, high myopia, defined as ≤6.0 diopters, significantly increases an individual's lifetime risk of vision-threatening complications. Moreover, recent studies revealed that non-ophthalmological factors such as... |
Background: The role of vitamin C in children with community-acquired pneumonia (CAP) in children is controversial; moreover, a standard dose is lacking.
Purpose: This study aimed to evaluate the ability of vitamin C to reduce symptom severity and length of hospital stay among children with CAP as well as determine its optimal dose. Methods: This randomized controlled clinical trial... |
Among neonates, hypoxic-ischemic encephalopathy is the most significant cause of mortality and hypoxia-ischemia is among the leading causes of brain damage. The microglia are primary mediators of neuroinflammation. NOD-like receptor family pyrin domain containing 3 (NLRP3) inflammasome activation is the first line of defense in the central nervous system. Numerous studies have shown that the NLRP3 inflammasome is activated and... |
Background: Inadequate knowledge of the fundamental mechanisms underlying pediatric neurological disorders impedes their effective treatment. Induced pluripotent stem cells (iPSCs) are essential for exploring the course of neurological diseases because they enable disease modeling at the cellular level.
Purpose: This study aimed to generate an iPSC bank using urine cells (UCs) for clinical applications, particularly the study of pediatric neurogenetic diseases.... |
Background: Although national population-based birth defect prevalence estimates are unavailable for Bangladesh specifically, data extrapolated from the March Dimes Global Birth Defects Report indicate a prevalence of neural tube defects (NTD) of 4.7 per 1000 live births.
Purpose: This study aimed to determine the prevalence of NTD among infants born at a tertiary care multidisciplinary referral hospital in Bangladesh. Methods: Live... |
Background: It is uncertain whether hematopoietic stem cell transplantation (HSCT), versus standard enzyme replacement therapy (ERT), is effective for type VI mucopolysaccharidosis (MPS VI).
Purpose: New related advances in HSCT prompted an examination of the transplant procedures performed in a recent cohort. Methods: This single-center retrospective study reviewed the medical records of 17 pediatric patients with MPS VI who underwent allogeneic... |
Background: Adequately powered studies in children are scarce and there are reports on the risk of carbon dioxide (CO2) retention after colonoscopy.
Purpose: This study investigated the efficacy and safety of CO2 insufflation in children undergoing colonoscopy. Methods: This prospective randomized clinical trial was conducted at a tertiary care hospital between March 2023 and July 2024. We recruited 200 consecutive children... |
Background: The neutrophil-to-lymphocyte ratio (NLR) is a marker of systemic inflammation associated with various diseases including respiratory conditions. However, the relationship between NLR and asthma in the pediatric population remains underexplored.
Purpose: This study aimed to explore the association between NLR and asthma in children and adolescents and assess its potential role as a predictive biomarker for pediatric asthma. Methods: We... |
Background: Functional gastrointestinal disorders (FGIDs) are associated with various gastrointestinal (GI) and non-GI symptoms, risk factors for which commonly include psychosocial and physical stresses.
Purpose: This study aimed to compare somatic symptom severity between children with FGIDs and healthy controls during acute illnesses. Methods: This was a prospective descriptive cross-sectional study whose inclusion criterion was age 4–18 years. Children were... |
Mucopolysaccharidosis (MPS) is a group of genetic disorders characterized by defective lysosomal enzyme activity that can result in growth abnormalities and other complications. Hematopoietic stem cell transplantation (HSCT), especially bone marrow transplantation (BMT), aims to restore enzyme function and improve growth parameters in patients with MPS. This systematic review evaluates the impact of HSCT on growth outcomes, including height, weight,... |
Background: Pro-adrenomedullin (pro-ADM), the most stable part of ADM, serves as an indirect marker of ADM levels. Serum amyloid A (SAA) is a protein produced primarily in the liver during acute inflammation.
Purpose: To assess the role of SAA and pro-ADM, individually and in combination, as diagnostic and prognostic markers in pediatric sepsis. Methods: This prospective case-control cohort study included... |
Background: Gut–lung crosstalk is a pathway involving interactions between the gastrointestinal, respiratory, and immune systems. The immune responses of the gut and lungs are intricately linked, and previous studies demonstrated that the gut microbiota can influence systemic immune responses in the respiratory system as well as bronchopulmonary dysplasia (BPD).
Purpose: To analyze the composition of the gut microbiota in very low... |
Background: Neonatal asphyxia is the primary cause of hypoxic-ischemic encephalopathy (HIE), a condition characterized by hypoxic and ischemic brain damage. A class of short noncoding RNAs known as microRNAs (miRNAs) have significant regulatory functions, can function as diagnostic and developmental indicators of diseases, and are involved in disease pathophysiology.
Purpose: To study the role of microRNA-410 and microRNA-498 in neonatal HIE... |
Background: To evaluate the outcomes of jaundiced neonates using two different total serum bilirubin (TSB) thresholds for discontinuing phototherapy.
Purpose: The study aims to evaluate the outcomes of jaundiced neonates by comparing two different total serum bilirubin (TSB) thresholds for discontinuing phototherapy. Methods: All consecutive jaundiced neonates in a tertiary care hospital with a gestational age of ≥35 weeks and ≥3... |
This study aimed to evaluate the effectiveness and safety of protocolized sedation in mechanically ventilated pediatric intensive care unit (PICU) patients. A comprehensive search was conducted in MEDLINE, CENTRAL, Embase, Web of Science, and Scopus from inception to October 18, 2023. Randomized controlled trials and observational studies that compared protocol-directed sedation management with conventional sedation regimens in pediatric patients who... |
In some children, atopic manifestations begin with atopic dermatitis and progress to allergic asthma and allergic rhinitis; of them, a small subset experience food allergies as well. This progression shares genetic and environmental predisposing factors and immunological features, such as allergen-specific T helper type 2 responses, that manifest as specific immunoglobulin E production and eosinophil activation. Eosinophil-derived neurotoxin (EDN), which... |
Background: Coronavirus disease 2019 (COVID-19) vaccination remains an essential strategy for reducing disease burden. Specific guidelines for vaccinating children with systemic lupus erythematosus (SLE) are currently unavailable, highlighting the gap in tailored recommendations for this population.
Purpose: This study aimed to estimate parental intention to vaccinate children with SLE against COVID-19 and identify factors associated with this intention. It also explored... |
Background: Fetal hemoglobin (HbF) inducers, among which hydroxyurea is the most extensively used, have shifted the paradigm toward the treatment of non-transfusion-dependent thalassemia (NTDT). Xmn1 polymorphism (rs7482144) is characterized by substitution (C>T) at -158 position of the γ-globin gene, which leads to CC, CT, or TT genotype. Recently, the role of the Xmn1 polymorphism as a modifier of hydroxyurea therapy... |
Background: Febrile neutropenia (FN) remains an important complication of cytotoxic chemotherapy for which an urgent and appropriate evaluation is imperative.
Purpose: To assess the diagnostic and prognostic roles of mid-regional pro-adrenomedullin (MR-ProADM) levels in predicting infection in patients with FN. Methods: This comparative cross-sectional study included 137 patients with chemotherapy-induced FN. Complete blood count, C-reactive protein (CRP), procalcitonin, and MR-ProADM were... |
Adenomatous Polyposis Coli (APC) is a tumor suppressor gene expressed throughout the body. APC mutations increase the risk of malignancy and are often characterized by syndromes that encompass a spectrum of neoplastic manifestations, such as familial adenomatous polyposis (FAP). We present a rare case of palatal peripheral nerve sheath tumor in the context of APC gene mutation. A 17-year-old male... |
Background: Diagnosing and predicting neonatal sepsis is challenging because of its nonspecific symptoms, lack of diagnostic criteria consensus, and absence of early, sensitive, and specific diagnostic laboratory tests.
Purpose: To evaluate the diagnostic and prognostic potential of adrenomedullin (ADM), interleukin-6 (IL-6), and C-reactive protein (CRP) in late-onset neonatal sepsis (LOS). Methods: We studied 53 neonates with culture-proven LOS by sampling... |
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