Diagnosing and treating rare diseases in children is a major challenge for pediatricians globally. There is a lack of adequate knowledge of these conditions and diagnostic testing is not easily accessible, which frequently results in delays in care. The knowledge, experiences and challenges faced by pediatricians in Tanzania are not known. This study used a nationwide cross-sectional online survey to describe the knowledge of pediatricians in Tanzania on rare diseases, their experiences, and the challenges they face in treating these children. The survey tool was shared on the Pediatric Association of Tanzania WhatsApp group where most pediatricians are registered. 168 pediatricians completed the survey, giving a response rate of 52%. All of them had encountered a child with a presumed rare disease in their career, with 60% having seen one in the 6 months preceding the survey. The commonest presumed rare condition encountered was genetic/metabolic, and the most common difficulty (97%) encountered was lack of access to diagnostic testing. A third of respondents reported that rare diseases were taught in university and 60% felt unprepared to look after these children. Three quarter of respondents could not access to experts to advise them on management. Presumed rare diseases are commonly encountered by pediatricians in Tanzania, and there are challenges in diagnostic testing, gaps in training, lack of confidence in providing care and inability to access experts on rare disease management. To improve care of children with rare diseases, diagnostic testing should be made available, accessible and affordable. A review of medical training curricula should be done to incorporate rare disease education and skill development. Platforms and pathways to connect pediatricians with regional and global experts should be put in place to provide timely and appropriate care to children with rare diseases.
Invasive aspergillosis (IA) is a major cause of morbidity and mortality in immunocompromised children. Pediatric data are limited, and treatment guidelines often rely on adult studies. This study aimed to assess clinical characteristics and treatment outcomes of pediatric IA. We conducted a retrospective cohort study of pediatric patients (0-18 years) diagnosed with IA at a tertiary center in Türkiye between 2010 and 2022. Data on demographics, underlying conditions, Aspergillus species, antifungal susceptibility, treatment regimens, and outcomes were analyzed. Survival analysis was performed using Kaplan-Meier curves. Fifty-five children met criteria for proven (69%) or probable (31%) IA after excluding 22 possible cases. Median age was 8.6 years; mortality did not differ significantly by age, sex, or underlying condition. Hematologic malignancies were the most common comorbidity (35%). Aspergillus fumigatus was the predominant species (57%), and no antifungal resistance was detected. Classical risk factors; central venous catheter use (53%), neutropenia (51%), and recent chemotherapy (35%) were frequent but showed no association with mortality. Lung involvement was the most common presentation (71%), followed by sinus (31%) and central nervous system involvement (13%). Thirty-day mortality was 11%, 12-week mortality was 20% (11/55), and overall mortality reached 29%. Pediatric IA remains associated with substantial morbidity and mortality, although outcomes in this cohort were more favorable than those reported in many prior pediatric series. No single clinical, microbiological, or radiological factor reliably predicted mortality, underscoring the complexity of risk stratification. Early diagnosis and timely antifungal therapy remain essential, and multicenter prospective studies are needed to optimize treatment approaches.
Health informatics and artificial intelligence (AI) technologies are increasingly influencing pediatric health care delivery across diverse health system contexts. These technologies offer opportunities to improve diagnostic accuracy, personalized treatment approaches, and access to care globally. This viewpoint examines how health and public health informatics frameworks, when integrated with AI technologies, may help address persistent challenges in global pediatric care delivery. This paper is a viewpoint informed by selected published studies and international digital health guidance rather than a systematic review. Evidence from clinical implementations suggests that AI applications embedded in standardized electronic health records can facilitate improved pediatric diagnostic processes. For instance, machine learning-based algorithms to diagnose serious bacterial infections among febrile infants have shown high diagnostic accuracy and reduced unnecessary invasive procedures in certain clinical contexts. Case studies from the Pediatric Emergency Care Applied Research Network decision rules, neonatal intensive care units, and autism screening programs reflect diverse applications of AI-enabled clinical decision support across pediatric settings. However, there are concerns regarding implementation due to limitations in interoperability of health information systems, gaps in data standardization, inadequate digital infrastructure in resource-limited settings, and issues related to algorithmic bias and equitable access. We argue that strategic development of interoperable health information systems, standardized data governance frameworks, and equitable digital infrastructure is essential to responsibly realize the potential of AI-enhanced pediatric care at scale.
Socioeconomic disadvantage is associated with increased disease severity and worse clinical outcomes among children with acute appendicitis. We sought to evaluate the association between neighborhood-level child opportunity and complicated appendicitis (CA) among a retrospective pediatric cohort. We hypothesized that lower neighborhood-level opportunity, measured by the Child Opportunity Index (COI), is associated with higher incidence of CA and hospital length of stay (LOS). We performed a retrospective review of children (<18 y) with appendicitis in the North Carolina Discharge Database, from 2019 to 2021. Patients were categorized as having simple appendicitis or CA, based on the presence of perforation, gangrene, or abscess. Multivariate regression analysis was used to identify independent predictors of CA and LOS. A total of 876 children with acute appendicitis and available zip code data were identified. Most children had public (n = 463, 52.9%) health insurance coverage. Compared to children with simple appendicitis, those with CA were significantly younger (9.9 ± 4.4 versus 11.9 ± 3.8 y; P < 0.0001), and with a higher proportion from very low (15.3% versus 12.2%) and low (23.1% versus 15.9%) COI neighborhoods. Hospital LOS was significantly longer for children with CA (3.7 ± 5.2 versus 1.6 ± 2.1 d; P < 0.001). On multivariate regression analysis, COI was not independently associated with odds of CA (adjusted odds ratio: 1.003; 95% confidence interval: 1.00-1.01) or hospital LOS (adjusted relative risk: 0.001; 95% confidence interval: -0.001 to 0.003). In a statewide cohort of pediatric patients, COI was not independently associated with CA. Our findings suggest that high insurance coverage improves access to acute pediatric surgical care and may mitigate disparities linked to child neighborhood level opportunity.
The establishment of a Center of Excellence in robotic pediatric urology is a deliberate, phased evolution-from individual pioneering procedures to an institution capable of achieving sustained long-term outcomes at par with established standards, training surgeons globally, and driving innovation. This article chronicles a single-surgeon, two-decade journey at the University of Chicago Medicine Comer Children's Hospital across five phases: foundation and initial implementation with critical adult-pediatric partnership; program expansion, care standardization, and infant application; maturation into a Center of Clinical Excellence with durable outcomes; emergence as a training and regional hub through a mini-fellowship, proctorship program, live surgical demonstrations, and multi-institutional collaboration with the Pediatric Urology Robotic Surgery (PURS) group; and a future vision centered on global health equity, telesurgery, and artificial intelligence. This narrative serves as a framework for programs on a similar trajectory.
This Viewpoint discusses the importance of evidence-based dietary guidelines for the health of children and adolescents, summarizes the strengths of the 2025-2030 Dietary Guidelines for Americans as well as concerns and challenges, and suggests government policy actions needed from the perspective of pediatric health care professionals and researchers.
The optimal dosing strategy for valganciclovir (VGCV) prophylaxis against cytomegalovirus (CMV) infection in pediatric patients after kidney transplantation (KT) remains uncertain because of the narrow therapeutic window between antiviral efficacy and safety. This study included pediatric patients who received VGCV prophylaxis after KT at a single center between December 2023 and December 2024. VGCV was administered once daily for 200 days using a reduced-dose regimen. Approximately 50% of the U.S. Food and Drug Administration-recommended dose was used for high-risk patients defined as donor-positive and recipient-negative, and 33% for intermediate-risk patients defined as recipient-positive. The incidence of CMV infection within 200 days after KT and VGCV-related adverse events (AEs) were evaluated. Nine patients with a median age of 8 years were included, with a median observational period of 431 days after KT. CMV infection within 200 days occurred in four patients, all of whom were high-risk patients. No CMV disease occurred during the observational period. Neutropenia occurred in six patients, including five high-risk patients and one intermediate-risk patient. Three high-risk patients developed febrile neutropenia. VGCV dose reduction and/or discontinuation due to hematologic AEs was required in four patients. The reduced-dose VGCV prophylaxis regimen was associated with a high incidence of CMV infection and AEs in high-risk patients, indicating a suboptimal balance between efficacy and safety. In intermediate-risk patients, the regimen appeared effective and tolerable. Further optimization of prophylactic strategies is required, particularly for high-risk pediatric recipients.
The 2026 Conference on Retroviruses and Opportunistic Infections provided data on several investigational antiretroviral compounds with the potential for twice-yearly dosing. A complete yearly regimen seems likely to be available in the coming years. Data on doravirine/islatravir showed that this regimen is an effective 2-drug regimen in those initiating antiretroviral therapy (ART) and in those switching from a suppressive regimen. Several abstracts on ART resistance were presented, focusing on the emergence of resistance to integrase strand transfer inhibitors and to lenacapavir. Recent data highlighted new strategies for the empiric treatment of severe pneumonia in infants with HIV, the potential for long-term ART-free remission using broadly neutralizing antibodies and very early initiation of ART, the evaluation of simplified and long-acting ART regimens, and optimizing transition services for adolescents with perinatally acquired HIV to adult care.
Evidence on the association between social media use and vaccine hesitancy has been inconsistent, and research on the mechanisms underlying this relationship remains limited. This study aimed to examine whether confidence, complacency, and convenience (3Cs) mediate the association between social media use and influenza vaccine hesitancy. An online questionnaire survey was conducted among 464 pediatric healthcare workers in a tertiary hospital in December 2024. The questionnaire assessed social media use (including trust and exposure), vaccine hesitancy, and the 3Cs. Reliability and validity were examined. Structural equation modeling (SEM) was used to estimate the direct and indirect effects of social media use on vaccine hesitancy. The model included social media trust, social media exposure, the 3Cs, and vaccine hesitancy as key variables. Convenience sampling was used. A total of 439 valid questionnaires were included in the analysis. The measures of vaccine hesitancy, social media use, and 3Cs demonstrated good reliability and validity. The Cronbach's α coefficients were 0.797, 0.928, and 0.859, respectively, and the corresponding Kaiser-Meyer-Olkin (KMO) values were 0.908, 0.882, and 0.91. Confidence significantly mediated the association between social media use and vaccine hesitancy, accounting for 34.9% of the total effect of social media trust on vaccine hesitancy (p = .001) and 30.7% of the total effect of social media exposure on vaccine hesitancy (p = .001). Greater trust in social media and greater exposure to vaccine-related information may be associated with lower influenza vaccine hesitancy among pediatric healthcare workers, primarily through increased vaccine confidence. Governments and health institutions should make better use of social media to disseminate evidence-based vaccine information through official channels and curb the spread of misinformation.
Mechanical ventilation of children sets the highest demands on the performance of anesthesia workstations. Different ventilator technologies are available, each representing unique pneumatic characteristics. We hypothesized that precision of delivered ventilation parameters depends on the ventilator type. Six modern anesthesia workstations (GE HealthCare Carestation 650; Mindray Bio-Medical Electronics Co. Ltd. Wato EX-65 Pro and A9; Dräger Medical Atlan A350 and Perseus A500; Getinge Flow-c) were tested in physical models simulating the respiratory systems of a preterm baby, a neonate, an infant and a toddler. Volume-controlled and pressure-controlled ventilation were investigated with and without airway leakage. Delivered tidal volume, peak inspiratory pressure, positive end-expiratory pressure and inspiratory to expiratory ratio were compared to the set values. Data are presented descriptively, a difference ≥ 10 % between delivered and the set values was considered clinically relevant. Deviations of the delivered volume from the set tidal volume during volume-controlled ventilation ranged between -19.5 ± 0.6 % and +35.1 ± 3.0 % in the preterm baby model, but decreased with increasing tidal volume. Positive end-expiratory pressure and peak inspiratory pressure during pressure-controlled ventilation did not differ relevantly from the set values in any device or lung model. Expiratory ratios varied widely in most devices leading to relevant differences in tidal volume during pressure-controlled ventilation. With leakage, tidal volume was on average 7 % lower during volume-controlled ventilation but 2 % lower during pressure-controlled ventilation. Precision of ventilation with modern anesthesia workstations varies depending on the ventilator type, ventilation mode and the set parameters. Pressure-controlled ventilation is advantageous in the six workstations tested considering the good agreement between the applied and the set parameters and leakage compensation. Our results imply precision superiority of pressure-controlled ventilation over volume-controlled ventilation in preterm babies and neonates, independent from the ventilator technology in use.
Ventriculo-pyeloureteral (VPU) shunting is a rare salvage procedure for hydrocephalus when traditional ventriculoperitoneal (VP) and ventriculoatrial (VA) shunts fail. We report the successful use of a self-expanding, nitinol-based ureteral stent (Allium Medical Solutions, Israel) to facilitate VPU shunting in a toddler with complex multicystic hydrocephalus. A premature infant born with duodenal atresia developed severe post-hemorrhagic hydrocephalus resistant to multiple VP and VA shunt revisions due to extensive peritoneal adhesions and recurrent atrial thrombosis. A VPU shunt was attempted as a last resort but was complicated by urinoma formation and obstruction due to the small caliber of the toddler's ureter relative to the catheter. To salvage the procedure, a large-caliber Allium ureteral stent was deployed to passively dilate the ureter, creating a protected channel for the shunt catheter. At 7 months follow-up, the patient demonstrated stable neurological function, effective CSF drainage, and no vesicoureteral reflux. This case highlights that VPU shunting combined with an Allium stent is a viable last-resort option, preventing ureteral obstruction by the shunt catheter.
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Central Nervous System (CNS) tumors are the leading solid malignancies in children, 50% of CNS tumors in children are glial tumors, with two-thirds of these gliomas being categorized as low-grade gliomas (LGG). We aimed to review the clinical profile, management, and outcomes of pediatric LGG cases. A retrospective cohort study was conducted at Aga Khan University Hospital between 2013 and 2025 including patients aged ≤ 18 years diagnosed with LGG. A total of 191 patients were identified, with a slight preponderance of male patients (51.3%). Median age was 10 years (IQR 6-14) with the larger age group being 10-18 years (50.3%). Median duration of presenting symptoms was 4 months (IQR 1-18). Headaches (n = 112, 58.6%) and vomiting (n = 93, 48.7%) were the primary presenting complaints. Tumors were predominantly supratentorial (n = 115, 60.2%). The most common histopathology was Pilocytic Astrocytoma (n = 119). Upfront surgery was done in 163 (85.3%) patients; Gross Total Resection (GTR) was achieved in 85 patients (52.1%). Twenty-six (13.7%) patients received Chemotherapy while 9 (4.8%) had radiation therapy. Disease didn't progress in 131 (68.6%) patients while progression and relapse occurred in 36 (18.8%) and 7 (3.7%) patients, respectively. Seventeen (8.9%) patients did not have any follow-op scans. The Overall Survival (OS) was 95.3%, with only 9 deaths recorded. Biopsy was deferred to 28 patients (14.7%), a group primarily comprising Subependymal Giant Cell Astrocytomas (SEGA) (n = 17, 60.7%), Tectal Plate Glioma (n = 3, 10.7%), and Optic Path Glioma (n = 5, 17.8%). Kaplan-Meier analysis estimated a median EFS of 84.2 months (95% CI: 70.9-97.5 months). However, this estimate should be interpreted cautiously given the relatively short median follow-up and substantial right-censoring. Despite heterogeneity in histopathology and location, outcomes were comparable to those reported in the literature, underscoring the effectiveness of current practices while highlighting the need for improved follow-up and comprehensive outcome reporting in resource-limited settings.
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Despite evidence that enhanced recovery protocols (ERPs) improve outcomes in adults undergoing surgery, adoption for pediatric populations has lagged. To assess the implementation and clinical effectiveness of a consensus-based ERP for pediatric patients undergoing elective gastrointestinal (GI) surgery. A prospective type 2 hybrid implementation-effectiveness, stepped-wedge, cluster-randomized by entry date into implementation phase, trial of pediatrics patients, 10 to 18 years of age, undergoing elective GI surgery at 18 US sites from September 2019 to June 2024. Sites were randomized into 3 groups, each spending at least 9 months in a control phase, with usual care, followed by an implementation phase at 6-month intervals that included a 21-element ERP supported by a structured Implementation Toolkit, based on 5 Active Implementation Frameworks (5AIFs), and a sustainment phase (12-24 months). Implementation was facilitated by a 1-year, group-based Learning Collaborative curriculum, a repository of tools, ERP adherence feedback, and implementation report cards. Site-level scores were created based on 5AIFs domains. ERP adherence was assessed by ERP elements delivered at patient and site level. The primary effectiveness outcome, postoperative length of stay (LOS), and secondary effectiveness outcomes (including opioid use, time to regular diet, complications, readmission, and patient-reported health-related quality of life [HRQOL]) were evaluated across study phases (baseline, implementation, and sustainability). Correlations between site-level implementation scores and fidelity were estimated. Of the 597 enrolled pediatric patients (median [IQR] age, 15 [13-17] years; 274 [45.9%] female; 323 [54.1%] male), 433 (72.5%) had inflammatory bowel disease. No significant differences were found by study phase in LOS or secondary outcomes, except shorter time to regular diet and decreased opioid use during hospitalization. Patients who received at least 13 ERP elements had shorter median LOS (-1.14 days [95% CI -2.01 to -0.27]) and fewer complications (adjusted odds ratio, 0.48 [95% CI, 0.28-0.82]). Patient-level adherence increased by study phase (number of ERPs: 11 [10-13], 14 [12-15], and 14 [13-15], [P < .001]). ERP integration into order sets and site culture were moderately correlated with fidelity. This stepped-wedge cluster-randomized trial found that despite multifaceted implementation strategies, a pediatric GI surgery ERP did not significantly reduce LOS. However, when accounting for implementation fidelity at the patient level, it resulted in significantly lower LOS and complications. ClinicalTrials.gov Identifier: NCT04060303.
The ambulatory arterial stiffness index (AASI) has emerged as an ambulatory blood pressure monitoring (ABPM) measure of stiffness and is supposedly useful in younger subjects. The objective of our study was to evaluate the relationships between the AASI and indices of arterial stiffness in a pediatric population at risk of hypertension. Cross-sectional study of children/adolescents (8-18 years) whose pulse wave velocity (PWV: carotid-to-femoral cf-PWV and heart-finger hf-PWV), augmentation index (AIx; normalized at 75 bpm: AIx75), systemic arterial stiffness (aortic pulse pressure/stroke volume, measured via pulse contour analysis), and ABPM were measured. At-risk populations were potential vascular remodeling (preterm birth, n = 44 and chronic kidney diseases, n = 7) and potential hyperkinetic causes (congenital central hypoventilation syndrome, n = 14 and psychostimulant treatment, n = 10). The mean age of the 75 participants was 12.3 ± 2.5 years (34 girls), and their mean AASI was 0.33 ± 0.17. AASI did not correlate with cf-PWV, hf-PWV, AIx, or systemic arterial stiffness. In contrast, the AASI correlated with both systolic and diastolic BP dipping at night (R =  - 0.23; p = 0.048 and R =  - 0.33; p = 0.004, respectively). Systemic arterial stiffness correlated with hf-PWV and AIx75 (R = 0.35; p = 0.004 and R =  - 0.34; p = 0.013, respectively). Based on ABPM, 15/75 (20%) participants had hypertension, and they had higher cf-PWV than participants without hypertension (5.64 ± 0.70 vs 4.92 ± 0.78 m/s, p = 0.002) and not different AASI values (0.34 ± 0.14 vs 0.32 ± 0.18, p = 0.756). AASI is not a measure of arterial stiffness in children at risk of secondary hypertension. • The ambulatory arterial stiffness index (AASI) has emerged as an ambulatory blood pressure monitoring measure of stiffness and is supposedly useful in younger subjects. Few adult studies validated the concept of AASI as a marker of arterial stiffness, and recent studies suggested the need for AASI measurement in pediatric subjects. • Our cross-sectional study, which used different methods for the assessment of vascular remodeling, shows that the AASI is not a reliable marker of arterial stiffness in children at risk of secondary hypertension.
Traumatic spinal cord injury (TSCI) without concurrent vertebral fracture is a phenomenon often described in pediatrics. There is limited evidence regarding factors that predispose adults to this rarer injury. To determine different factors that increase the odds of TSCI without vertebral fracture. Retrospective case-control study. Participating sites of the Spinal Cord Injury Model Systems (SCIMS) database. Patients aged 18 or older presenting at a registered SCIMS center following TSCI between 2006 and 2021. None. Logistic regression identified variables associated with TSCI without vertebral fracture (OR > 1). This study included 2,524 patients, of whom 501 did not suffer a vertebral fracture. Patients with incomplete SCI at cervical levels and those of more advanced age were less likely to have a concurrent vertebral fracture. Penetrating injury (OR = 4.13 [95% CI: 2.72, 6.28], P < 0.0001) and medical/surgical complications (OR = 50.30 [95% CI: 27.80, 95.90], P < 0.0001) dramatically increased the odds of suffering SCI without vertebral fracture compared to non-penetrating injuries. Overall results were similar when only blunt injuries were analyzed, except that complete injuries at T1-S5 (OR = 0.41 [95% CI: 0.17, 0.96], P = 0.044) were associated with vertebral fracture. Adults may experience biomechanical stresses similar to those proposed in pediatric populations. The findings of this study can be used to screen patients with suspected spinal cord injury without obvious signs of spinal trauma.