Central venous catheters (CVCs) are essential in pediatric hematology-oncology, for the administration of chemotherapy and supportive therapy. Thrombocytopenia increases the risk of bleeding and current guidelines recommend prophylactic platelet transfusions below 40-50 × 109/L, though evidence is limited and transfusions entail risks and costs. Advances in procedural bundles and simulation-based training may enhance safety, enabling lower thresholds. This retrospective observational cohort study analyzed 274 pediatric patients undergoing CVC insertion (PICC, CICC, and FICC) at a tertiary center (2020-2023). Patients were stratified by platelet count using 50 × 109/L and 30 × 109/L thresholds. Postoperative complications, including bleeding and thrombosis, were compared between groups. All procedures employed pediatric-specific bundles, and operators received simulation-based ultrasound-guided PICC training to optimize safety. Complication rates did not differ significantly with platelets above or below 50 × 109/L (32.8% vs. 26.3%, p = 0.29) or 30 × 109/L (32.6% vs. 27.1%, p = 0.29). Findings indicate lowering the transfusion threshold to 30 × 109/L is safe. Bundles and simulation-based training likely contributed to low complication rates. A 30 × 109/L platelet transfusion threshold appears safe for pediatric CVC insertion. Emphasizing procedural bundles and simulation-based operator training may reduce transfusion needs, associated risks, and costs. Prospective multicenter studies are warranted to confirm safety and explore lower thresholds.

Carotid artery dissection is a rare but significant cause of pediatric stroke, often associated with trauma or underlying collagen vascular disorders. The management of these cases, both medical and surgical, is complex, particularly in bilateral dissections where collateral circulation may be insufficient. We describe a case of bilateral internal carotid artery occlusion that developed 10 days after an initial traumatic fall. The progression of ischemic stroke burden and fluctuating neurologic status, despite therapeutic anticoagulation and optimal medical management, led to the decision for endovascular intervention involving balloon angioplasty and stenting. Following the procedure, a rapid improvement in neurologic function was observed, with the patient exhibiting only mild deficits at discharge. This case highlights the complexities in managing pediatric carotid dissections, especially in unusual presentations. Our experience underscores the necessity for tailored treatment strategies, rigorous monitoring, and a multidisciplinary approach to optimize outcomes in pediatric stroke patients.

Introduction: Right ventricular outflow tract (RVOT) reconstruction in congenital heart diseases such as tetralogy of Fallot (TOF) often requires a transannular patch, which can lead to postoperative pulmonary regurgitation (PR). Persistent PR may result in significant complications, including right ventricular (RV) dysfunction, arrhythmias, and the need for reintervention. The use of autologous right atrial appendage (RAA) tissue to create a functional neovalve has emerged as a promising alternative. Methods: Twelve pediatric patients who underwent RVOT reconstruction with an RAA neovalve between July 2023 and March 2025 were retrospectively evaluated. Demographic, surgical, and echocardiographic data were analyzed to assess early clinical outcomes. Results: The median age at surgery was eight months (IQR: 6-11), with a median weight of 7.1 kg (range: 5.7-16). Primary diagnoses included TOF (n = 9), TOF with pulmonary atresia (n = 2), and absent pulmonary valve (n = 1). No in-hospital mortality occurred. Predischarge echocardiography showed no or trivial PR in eight patients and mild PR in four patients. At a mean follow-up of 12.8 months, all patients exhibited either no or mild PR, and RV function remained normal. No patient required reintervention. Conclusion: Neovalve reconstruction using autologous RAA tissue is a safe, feasible, and hemodynamically effective technique for RVOT repair in selected pediatric patients. Due to its autologous nature, growth potential, and favorable early outcomes, it offers a meaningful alternative to synthetic or allogenic materials. Further follow-up studies are necessary to evaluate its long-term durability.

Elamipretide (Forzinity™) is a mitochondrial cardiolipin binder being developed by Stealth BioTherapeutics for the treatment of a range of disorders featuring mitochondrial dysfunction. In September 2025, elamipretide was granted accelerated approval in the USA for use to improve muscle strength in adult and pediatric patients with Barth syndrome weighing ≥30kg. With this accelerated approval, elamipretide became the first disease-specific treatment approved for Barth syndrome, an ultra-rare X-linked recessive genetic disorder. Elamipretide is also under phaseIII clinical development for use in the treatment of dry age-related macular degeneration and mitochondrial myopathies. This article summarizes the milestones in the development of elamipretide leading to this first approval for Barth syndrome.

This study systematically evaluated the safety profile of tumor necrosis factor-alpha (TNF-α) inhibitors in pediatric patients using data from the U.S. Food and Drug Administration (FDA) Adverse Event Reporting System (FAERS) from Q1 2004 to Q3 2024.Through disproportionality analysis of adverse event (AE) reports for infliximab, etanercept, adalimumab, golimumab and certolizumab, we identified 852 significant safety signals spanning 27 system organ classes (SOCs). The most frequently reported SOCs included General Disorders and Administration Site Conditions (12,940 cases), Injury, Poisoning, and Procedural Complications (5,503 cases), and Gastrointestinal Disorders (4,346 cases).Key findings revealed that infection-related AEs and injection-site reactions were the predominant safety concerns. The median onset time of AEs was 235 days (IQR: 46–832 days), with 19.8% of cases occurring within the first month of treatment. Notably, 25.5% of reported AEs required hospitalization, while fatal and life-threatening outcomes accounted for 0.9% and 1.4% of cases, respectively. This comprehensive analysis confirms the multisystemic involvement and prolonged latency of TNF-α inhibitors-associated AEs in pediatric populations. While these agents remain vital for managing chronic inflammatory diseases, the findings advocate for enhanced clinical vigilance. We propose a tiered monitoring protocol prioritizing infection surveillance (e.g., serial inflammatory markers), systematic injection-site evaluations, and longitudinal organ function assessments, particularly during the initial treatment phase, to optimize therapeutic risk-benefit ratios.

The mean SUA level was significantly higher in the microalbuminuria group and the rate of microalbuminuria is higher in patients with T1DM when SUA was ≥ 3.98mg/dL. These findings indicate a potential link between elevated SUA levels and early renal involvement in pediatric T1DM, though the proposed cut-off should be interpreted with caution. • Serum uric acid levels have been associated with an increased risk of developing DKD and development of albuminuria has been linked to higher SUA levels in patients with diabetes. • The mean SUA level demonstrated a moderate discriminative ability for microalbuminuria, with an AUC of 0.66 (p = 0.008, 95% CI [0.56-0.76]), providing 63% sensitivity and 65% specificity at a cut-off value of 3.98mg/dL. • The rate of microalbuminuria is 3.06 times higher in pediatric patients with T1DM when SUA is ≥ 3.98mg/dL. • Considering the ongoing debate regarding the definition of elevated SUA levels even in healthy individuals, the proposed cut-off may serve as a preliminary reference for future studies exploring risk assessment and follow-up in this population.

Background: Pediatric patients with beta-thalassemia (BT) face unique immunologic challenges due to chronic transfusions and viral exposure. Hepatitis C virus (HCV), a common infection in polytransfused individuals, may influence immune polarization. However, the combined effect of chronic HCV and host immunogenetics on allergic sensitization remains incompletely understood. Objective: To assess total serum IgE levels and allergic manifestations in HCV-positive vs. HCV-negative BT patients, and explore associations with common polymorphisms in IL10, TLR7, IL4, and IFNG genes Methods: This cross-sectional observational study enrolled 46 BT patients (37 HCV-positive, 9 HCV-negative) and 50 healthy controls. Clinical allergy history, total IgE levels (ELISA), and skin prick tests (SPT) for aeroallergens were collected. Genotyping for IL10 −1082, TLR7 rs179008, IL4 −589, and IFNG +874 polymorphisms was performed. Associations between genotypes, HCV status, and IgE levels were analyzed descriptively due to small sample size Results: HCV-positive BT patients had lower mean IgE levels (18.73 ± 4.2 IU/mL) and fewer reported allergic symptoms (21.6%) compared to HCV-negative counterparts (118.76 ± 7.9 IU/mL; 55.5%). The IL10 −1082 AA and TLR7 rs179008 TT genotypes were more common in the HCV-positive group and were associated with lower IgE levels. No associations were noted for IL4 or IFNG variants. Splenectomy appeared to further modify IgE levels in HCV-negative patients. Due to limited power and absence of multivariate analysis, findings are exploratory. These preliminary observations may inform future studies of immune deviation in chronically infected pediatric cohorts. Conclusions: Chronic HCV infection may contribute to immune tolerance and reduced allergic expression in BT patients, potentially modulated by IL10 and TLR7 genotypes. Further studies with functional immune profiling and larger cohorts are required.

Bloodstream infections remain a major cause of morbidity and mortality worldwide, with the increasing threat of antimicrobial resistance (AMR) complicating treatment options. This study aimed to describe the frequency, distribution, and patterns of AMR among blood culture isolates over 5 years at the University Teaching Hospital of Kigali, in Rwanda. A retrospective cross-sectional surveillance analysis was performed on 1352 bacterial isolates from 8301 blood cultures conducted between January 1, 2020, and December 31, 2024. The distribution of pathogens, antimicrobial resistance profiles, and comparisons of resistance patterns between isolates from outpatient and hospitalized patients were analyzed using SPSS version 28. A p-value less than 0.05 was considered statistically significant. The most common isolates were Staphylococcus aureus (37.2%), Klebsiella pneumoniae (22.4%), Escherichia coli (13%), and Acinetobacter baumannii (11.6%). Nearly all isolates originated from inpatients (98.6%), with the pediatric unit accounting for 40.7%. Alarmingly high resistance rates were observed for ampicillin (94.2%), amoxicillin-clavulanic acid (92.5%), third-generation cephalosporins (79-86%), and ciprofloxacin (58.7%). Notably, vancomycin (1%) for Gram-positive bacteria, and polymyxin B (27.1%), imipenem (25.5%), and amikacin (15.6%) for Gram-negative bacteria generally exhibited lower resistance rates. Additionally, AMR was significantly more prevalent in isolates from hospitalized patients compared to ambulatory patients (p < 0.0001). This study reveals a substantial burden of AMR in blood culture isolates, particularly affecting hospitalized and pediatric patients. The high resistance rates to commonly used antibiotics highlight an urgent need for strengthened antimicrobial stewardship programs, improved infection prevention measures, and enhanced diagnostic laboratory capacity to guide therapy.

The application of personalized medication management for Voriconazole(VCZ), such as therapeutic drug monitoring (TDM), is the gold standard for attaining therapeutic goals in invasive fungal infections. Infectious disease (ID) pharmacists are the best for ensuring the recommendations are properly implemented. To assess the adherence of healthcare teams to VCZ dose individualization and the impact of the ID pharmacy team. This study included pediatric oncology patients who received VCZ in the inpatient department and had VCZ TDM level measurements for 3 months (from January to March 2019) and after implementing the (ID) pharmacy service during the same months in two different years (2020, 2021). This study included 989 patients who received VCZ; only 588/989 patients (59.5%) had VCZ TDM levels. Dose individualization was required for 62.6% (849/1357) levels, while 37.4% (508/1357) levels were in the therapeutic range. The overall compliance rate with the TDM recommendations was 92.9% (789/849). Additionally, there was a significant increase in VCZ dose individualization adherence after ID pharmacy implementation (88.6% (210/237) vs. 94.6% (579/612), p = 0.002). The rate of doctor acceptance of the ID pharmacist intervention was 94.7%. ID pharmacists' contributions are crucial and can increase clinicians' adherence to individualized dose recommendations.

To develop and validate a Pediatric Anatomo-Endocrine (PAE)-based nomogram for predicting severe postoperative endocrine dysfunction in children undergoing craniopharyngioma (CP) resection, integrating anatomical classification, surgical factors, and baseline endocrine status.This retrospective study included 370 pediatric CP patients (≤ 18 years) who underwent primary resection at a single center between 2017 and 2024. Patients were randomly divided into a primary (n = 300) and a validation (n = 70) cohort. Endocrine dysfunction was assessed based on the number and severity of hypothalamic-pituitary axis abnormalities. A novel three-tier PAE classification was developed from preoperative MRI. Logistic regression was used to identify predictors of severe dysfunction. A nomogram was constructed and validated via ROC, calibration, and decision curve analyses.In the primary cohort, 83.4% of patients had worsened endocrine dysfunction postoperatively, with severe dysfunction increasing from 20.3% to 79.3%. Independent predictors included PAE classification, baseline endocrine status, and pituitary stalk sacrifice. The nomogram demonstrated strong performance (AUC 0.84 in primary; 0.82 in validation). Among PAE subtypes, the Global subtype posed the highest risk, with an odds ratio of 16.30 versus the Sella-Infundibulum subtype.The PAE classification stratifies hypothalamic-pituitary involvement and serves as an independent predictor of endocrine outcomes. Although validated internally in a large single-center cohort, its long-term benefit remains to be confirmed. The proposed nomogram may provide a practical, non-invasive tool for individualized risk prediction and surgical planning, but requires external multicenter validation to establish generalizability.

Adults are not just big kids: adults have higher reoperation and complication rates following lumbar laminectomy for tethered cord release.

Idiopathic intracranial hypertension (IIH) in children is a rare but vision-threatening condition characterized by elevated intracranial pressure (ICP) without an identifiable cause. While medical therapy remains the first-line approach, a subset of pediatric patients remains refractory, necessitating surgical or endovascular interventions. Venous sinus stenting (VSS) has emerged as a promising, minimally invasive alternative to cerebrospinal fluid (CSF) shunting, but its safety and efficacy in pediatric populations are not well-defined. A systematic literature review was conducted following PRISMA guidelines, focusing on pediatric patients (< 18years) with medically refractory IIH treated with VSS. Databases searched included PubMed, ScienceDirect, Cochrane Library, and others, including studies published from January 1990 to September 2025. Data on patient selection, procedural outcomes, complications, and antiplatelet protocols were extracted and analyzed. Only studies rated ≥ 6 stars on the Newcastle-Ottawa Scale were included. Six primary studies were identified, reporting outcomes in 36 pediatric patients (mean age 13.32 ± 1.25years). VSS led to marked improvement in headache (87.5%), papilledema (89.5%), and tinnitus (100%), with reduced need for acetazolamide in 66.7%. Complications included one retroperitoneal hematoma, decreased flow in the vein of Labbe and minor hemorrhagic events related to antiplatelet therapy, including epistaxis, menorrhagia, and oral bleeding leading to early discontinuation of clopidogrel. Persistent or recurrent symptoms after stenting underscore the need for individualized, multimodal evaluation, as standardized selection criteria for pediatric IIH are still lacking. The estimated retreatment rate across studies was approximately 20%, with re-stenting or CSF shunting required in select cases. VSS appears to be a safe and effective treatment for selected pediatric IIH patients with venous sinus stenosis and demonstrable trans-stenotic gradients. However, its success relies heavily on appropriate patient selection and tailored antiplatelet management. Prospective pediatric studies and standardized guidelines are needed to clarify its optimal role in the therapeutic algorithm. Accordingly, our synthesis is intended to inform individualized decision-making in selected cases and does not constitute guideline-level recommendations.

Prevalence and epidemiology of enterovirus species in a pediatric oncology patient population.

Clinical features of cochlear implantation in Japan and factors affecting postoperative infection.

Combination of Perioperative Cilostazol and Aspirin in Pediatric Moyamoya: A Case Series.

Switching from oxcarbazepine to eslicarbazepine in pediatric patients with focal epilepsy is safe and well-tolerated.

Introduction: Peripheral Venous Catheters (PVCs) are routinely used in paediatric patients but are associated with catheterrelated infections. Adequate skin antisepsis prior to catheter insertion is critical in preventing these infections. Aim: To compare the efficacy of 2% chlorhexidine in alcohol (CHA) with 10% povidone-iodine in alcohol (PI) in reducing catheter-tip colonisation in paediatric patients. Materials and Methods: A double-blind randomised controlled trial was conducted at the Paediatric Ward of Acharya Vinoba Bhave Rural Hospital, Maharashtra, India, from May 2024 to December 2024. A total of 102 paediatric patients aged 1 month to 16 years, admitted to the paediatric ward with an indication for intravenous catheterisation, were randomly assigned to two groups: Group A received 2% CHA and Group B received 10% PI in alcohol before PVC insertion. Upon catheter removal, the distal tips were cultured for microbial colonisation. Parameters assessed included gender, age group, mean age, incidence of catheter-tip colonisation, and type of organism isolated. Statistical tests applied included Chi-square test, Independent t-test, and Fisher’s exact test. Results: Demographic data showed comparable gender distribution (M:F=31:20 in Group A, 30:21 in Group B) and mean age (6.6±3.05 years vs 7.14±3.42 years) between the two groups. Catheter-tip colonisation occurred in 1 (1.96%) patient in Group A and 6 (11.76%) in Group B (p-value=0.112). Staphylococcus was the predominant organism in both groups, while Klebsiella and Gram-positive Cocci (GPC) were additionally isolated in the PI group. Conclusion: Catheter-tip colonisation was higher in the PIalcohol group (6/51) compared with the CHA group (1/51); however, this difference did not reach statistical significance (p-value=0.112). A larger study is warranted to provide more definitive evidence.

Relapsed or refractory cases of pediatric acute myeloid leukemia (AML) have poor outcomes despite advancements in chemotherapy and hematopoietic stem cell transplantation (HSCT). While a second HSCT is often a salvage option, its outcomes vary widely, and prognostic factors remain unclear. This study aimed to evaluate outcomes and identify prognostic factors in pediatric patients with AML who underwent multiple HSCTs. We conducted a retrospective, multicenter study of 49 pediatric patients with AML who underwent two or more HSCTs at 18 Japan Association of Childhood Leukemia Study institutions during 2000-2019. Clinical data on patient demographics, disease status, transplant characteristics, and complications were collected. The primary endpoint was 5-year overall survival (OS) after the second HSCT. Kaplan-Meier and multivariate Cox regression analyses were performed. Of the 49 patients, three and 46 underwent three and two HSCTs, respectively. Among these 46 patients, 5-year OS after the second HSCT was 28.3%. Achieving hematological complete remission (CR) before the second HSCT was associated with significantly better outcomes (5-year OS: 45.0% vs. 0%, p < 0.01). Severe sinusoidal obstruction syndrome (SOS) after the second HSCT was a strong predictor of transplant-related mortality (5-year OS: 0% in severe SOS cases, p < 0.01). Patient age, donor type, conditioning regimen, and acute/chronic graft-versus-host disease were not significantly associated with survival in multivariate analysis. Achieving CR before the second HSCT is critical for long-term survival, while severe SOS significantly worsens prognosis. Optimized pre-transplant strategies to reduce SOS risk are essential to improve outcomes of pediatric patients with AML who undergo multiple HSCTs.

Goldenhar Syndrome (GS), also known as the Oculo-Auriculo-Vertebral Spectrum (OAVS), is a rare congenital disorder characterised by variable craniofacial and ocular anomalies due to aberrant development of the first and second branchial arches. This case series describes three paediatric patients who exhibited diverse phenotypic features of the syndrome, highlighting its clinical heterogeneity and the importance of individualised management. All three patients presented with epibulbar dermoids since birth, an ocular hallmark of GS. The first patient, a 12-year-old female, had a limbal dermoid, medial canthal swelling suggestive of nasolacrimal duct obstruction, a symblepharon, and microtia with preauricular tags. She was advised dacryocystorhinostomy and excision of the dermoid but was lost to follow-up. The second patient, a 15-year-old had a reddish limbal mass extending to the lateral canthus and preauricular tags, and a cosmetic symblepharon release was advised. The third patient, a 15-year-old had a non-progressive limbal dermoid with no significant adnexal involvement and was managed conservatively. None of the patients had associated spinal, cardiac, or neurological anomalies on systemic evaluation. This case series reinforces the phenotypic variability of GS and underscores the importance of early ophthalmic diagnosis, systemic screening, and a multidisciplinary approach to optimise functional and cosmetic outcomes in affected children.

Autoimmune manifestations in children with inborn errors of immunity in Morocco: A study from the national registry.