To describe site-level variation in the performance and outcomes of tracheal intubation (TI) in pediatric emergency departments (PEDs) using the National Emergency Airway Registry for Pediatric Emergency Medicine (NEAR4PEM). A multicenter observational study of TI was performed between April 2018 and September 2023. TI data were obtained from the 16 PEDs contributing to the NEAR4PEM registry. Standard NEAR4PEM operational definitions were used. Site-level variation in TI performance and outcomes were reported using summary statistics and interquartile range (IQR), including first-attempt success (FAS) and adverse airway outcomes (AAOs). AAOs were defined as adverse tracheal intubation-associated events or oxygen desaturation (SpO2 <80%). A total of 1,729 TIs were performed. Median annual site visits were 52,500 (IQR: 39,750, 64,750). Median site admission rate was 14% (IQR: 11.7, 17). The proportion of infants was median 28.5% (IQR: 23, 32) and patients with a difficult airway feature was median 25% (IQR: 14, 37). A trainee performed a median 75% (IQR 71, 93) of first attempts. There was substantial variation among sites in TI practice (median, IQR): use of apneic oxygenation 38% (26, 53), use of a video laryngoscope 87% (67, 93), preinduction vagolytic use in infants 33% (5, 46), and TIs without paralysis 13% (10, 23). FAS per site was a median of 72% (IQR: 68, 76). The proportion of TIs with an AAO by site was median 24% (IQR: 16, 30). Considerable variation exists in performance and outcomes of TI across the PEDs participating in NEAR4PEM. These findings will be used to inform future research and quality improvement efforts.

Whether interventions to improve hydroxyurea adherence in youth with sickle cell disease (SCD) also improve health-related quality of life (HRQoL) has not been determined. We prospectively examined changes in generic and disease-specific HRQoL over a 12-month period in youth who participated in "Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment (HABIT)" randomized controlled multi-site efficacy trial. The HABIT intervention was led by community health workers and augmented by tailored text message reminders. Improvements in generic and disease-specific HRQoL were secondary HABIT outcomes. Intervention efficacy and sustainability were measured as changes in HRQoL from Months 0 to 9 and from Months 9 to 12 of the trial, respectively. Data were analyzed for within-group and between-group changes. Fifty youth, 24 assigned to intervention and 26 to the control group, mean age of 13.3±1.9years, participated in the trial. There were no differences between groups at study entry. At Month 0, total generic and disease-specific HRQoL scores were 71.2±15.6 and 62.7±19.0, respectively. At 9months, compared to controls, the intervention group significantly improved generic HRQoL total score (p = 0.04) and two subscales (emotional, p = 0.03, and social, p = 0.01), and one disease-specific HRQoL subscale (treatment, p = 0.006). HRQoL improvements were not sustained at 12months. Findings of this study contribute to the evolving understanding of the impact of interventions to improve hydroxyurea adherence on HRQoL in youth with SCD. Further research directed to enhancing intervention sustainability is needed to maintain hydroxyurea adherence and HRQoL improvements to reduce health disparities for youth with SCD.

To identify a concise set of screening questions that effectively predict severe obstructive sleep apnea (OSA). Secondary analysis of prospectively collected data from two large pediatric clinical trials. Children ages 5 to 9 from the Pediatric Adenotonsillectomy Trial for Snoring (June 2016 to January 2021) and the Childhood Adenotonsillectomy Trial (October 2007 to June 2012). Screening questions and tonsil size were analyzed against polysomnography (PSG) markers, including the apnea-hypopnea index (AHI), obstructive AHI (OAHI), and respiratory disturbance index (RDI). Positive predictive values (PPVs) for severe OSA and negative predictive values (NPVs) for lack of severe OSA were calculated for various question combinations. The top combinations with a PPV of 100% and the highest NPV were identified. The top four three-question combinations, consistently among the highest NPV sets across all outcomes, achieved NPVs of 95.97% to 96.64% and PPVs of 100%. These combinations assessed the following key factors: breath-holding spells, choking/gasping, mouth breathing, aggressive/hyperactive behavior, discipline problems, and growth delays. In univariate analysis, larger tonsils were more common in children with moderate or severe OSA, but tonsil size was not an independent predictor of severe disease in multivariable models. This study demonstrates that SDB can be effectively predicted or ruled out using just three key screening questions. Incorporating these into preoperative evaluations, alongside history, physical examination, and tonsil size, may improve efficiency in clinical practice, guide PSG referral, and inform perioperative decisions such as the need for inpatient admission and the optimal surgical setting.

Ganciclovir remains the primary therapeutic for cytomegalovirus (CMV) infections in early infancy, but its pharmacokinetics and dosing in very preterm infants with end-organ CMV disease have not been fully evaluated. Premature infants with confirmed CMV infection and receiving ganciclovir as standard of care were enrolled into a pharmacokinetic sampling study. All were <32 weeks gestational age and >500 grams at enrollment. Plasma for ganciclovir quantitation was collected at steady-state at 0, 1, and between 2-3, 5-7, and 10-12 hours post-dose. Specimens were shipped, analyzed, and pharmacokinetic parameters calculated in real-time. Noncompartmental and modeling approaches were used for analysis. Eighteen infants were enrolled; mean gestational age at delivery was 26.7 weeks, and mean age and weight at enrollment were 42 days and 1519.0 grams, respectively. Seventeen completed pharmacokinetic assessments. Geometric mean dose and resulting 12 hour area-under-the-curve (AUC12) were 5.19 mg/kg and 52.7 mgxh/L, respectively. A total of 85 ganciclovir concentration-time data points were available for modeling. A one-compartment power covariate model with weight and serum creatinine on clearance and weight on distribution volume was used. Noncompartmental and modeled pharmacokinetic parameters were similar. These are the first intravenous ganciclovir population pharmacokinetic data with covariate assessments in premature infants being treated for CMV disease. Results suggest an intravenous dose of 5 mg/kg/dose every 12 hours may be an appropriate starting regimen for treatment of premature infants with congenital CMV. Additional data are needed in this and other populations to better define optimal ganciclovir exposure targets.

ObjectiveAutophagy is a fundamental biological pathway with vital roles in intracellular homeostasis. During autophagy, defective cargoes including mitochondria are targeted to lysosomes for clearance and recycling. Recessive truncating variants in the autophagy gene EPG5 have been associated with Vici syndrome, a severe early‐onset neurodevelopmental disorder with extensive multisystem involvement. Here, we aimed to delineate the extended, age‐dependent EPG5‐related disease spectrum.MethodsWe investigated clinical, radiological, and molecular features from the largest cohort of EPG5‐related patients identified to date, complemented by experimental investigation of cellular and animal models of EPG5 defects.ResultsThrough worldwide collaboration, we identified 211 patients, 97 of them previously unpublished, with recessive EPG5 variants. The phenotypic spectrum ranged from antenatally lethal presentations to milder isolated neurodevelopmental disorders. A novel Epg5 knock‐in mouse model of a recurrent EPG5 missense variant featured motor impairments and defective autophagy in brain areas particularly relevant for the neurological disorders in milder presentations. Novel age‐dependent neurodegenerative manifestations in our cohort included adolescent‐onset parkinsonism and dystonia with cognitive decline, and myoclonus. Radiological features suggested an emerging continuum with brain iron accumulation disorders. Patient fibroblasts showed defects in PINK1‐Parkin‐dependent mitophagic clearance and α‐synuclein overexpression, indicating a cellular basis for the observed neurodegenerative phenotypes. In Caenorhabditis elegans, EPG5 knockdown caused motor impairments, defective mitophagic clearance, and changes in mitochondrial respiration comparable to observations in C. elegans knockdown of parkinsonism‐related genes.InterpretationOur findings illustrate a lifetime neurological disease continuum associated with pathogenic EPG5 variants, linking neurodevelopmental and neurodegenerative disorders through the common denominator of defective autophagy. ANN NEUROL 2025;98:932–950

The growing interest in complementary and alternative medicine (CAM) has sparked discussions about its role in modern pediatric healthcare. While some parents may turn to herbal supplements, essential oils, and other remedies for symptom management, questions remain regarding their safety, efficacy, and regulation for pediatric use. Pediatricians should understand the benefits and limitations of CAM to effectively inform families. Recent studies suggest that certain CAM therapies may offer relief for pediatric conditions like anxiety and digestive disorders. However, a lack of research and standardized regulation raises concerns about their clinical value, appropriate dosing, and misinformation. While parental interest has led to integration of CAM into some mainstream settings, further studies are needed to establish better evidence-based guidelines for use in pediatric patients. CAM continues to garner attention as families seek holistic alternatives to traditional healthcare. While CAM therapies may offer benefit when used alongside conventional medicine, they do not rival first-line treatments for pediatric disorders. Providers must be wary of unverified claims and risks. Incorporating evidence-based research and regulatory oversight can help maximize the well tolerated and effective use of CAM.

The mandated accessibility of the electronic health record (EHR), including direct patient access through a portal, has created a unique and important opportunity for clinicians to examine their communication. This development underscores the importance of understanding how language - both written and verbal - may inadvertently reinforce biases, perpetuate existing disparities, and potentially distance patients from care. Indeed, the language clinicians use - whether written or verbal - is not merely descriptive; it is a powerful intervention that can either build trust or perpetuate harm. While often unintentional, the misuse of language in a clinical context is a significant and modifiable driver of health disparities. We will examine the mechanisms by which biased words erode patient care and then propose concrete strategies to foster communication that are precise, respectful, and actively promotes health equity. The connection between language in a clinical context and bias, whether spoken or written, is increasingly acknowledged in medical education and research literature. This evolving field is especially pertinent to pediatric care, where communication plays a critical role in health outcomes. This review defines stigmatizing language and explores how linguistic patterns can influence the physician-patient relationship. We delve into the ways the use of stigmatizing language reinforces broader societal frameworks, shapes clinicians' attitudes, and healthcare disparities. We illustrate how shifting towards person-centered language can transform linguistic patterns. As our digital age continues to evolve, with increasing reliance on social media and artificial intelligence, we urge physicians to model language that fosters inclusivity by offering strategies, at both the individual and system levels, to integrate into their healthcare communication.