Pediatrics
● American Academy of Pediatrics (AAP)
Preprints posted in the last 90 days, ranked by how well they match Pediatrics's content profile, based on 10 papers previously published here. The average preprint has a 0.01% match score for this journal, so anything above that is already an above-average fit.
Hurst, J. H.; Zhao, C.; Raynor, E. M.; Lee, J.; Gitomer, S. A.; Woods, C. W.; Kelly, M. S.; Smith, M. J.; Goldstein, B. A.
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Background and ObjectivesRecurrent acute otitis media (rAOM; defined as [≥]3 AOM episodes in 6 months or [≥]4 episodes in 12 months) affects 10-15% of children in the United States and is a leading cause of healthcare utilization and antibiotic prescriptions. Prospective identification of children at risk of rAOM could help target interventions and identify new risk factors to guide preventive approaches. We therefore sought to develop predictive models to identify children at risk of rAOM using electronic health records (EHR) data. MethodsWe extracted retrospective EHR data for children who were born at Duke University Health System (DUHS) hospitals between January 1, 2014, and June 30, 2022, and who had at least one AOM episode during the study period. We used LASSO to build predictive models for development of rAOM at each episode and identified factors associated with rAOM. ResultsWe identified 6,566 children who met the study criteria, including 1,634 (24.8%) who met criteria for rAOM. A model using only data available at the first AOM episode had an area under the curve (AUC) of 0.75 (0.73, 0.77) and an Area Under the Precision Recall Curve (AUPRC) of 0.41 (95% CI 0.37, 0.46), indicating moderate discriminative ability. At the time of the first AOM episode, features associated with subsequent rAOM development included age, number of prior antibiotic prescriptions, and diagnosis of gastroesophageal reflux disease (GERD). Further, children who developed rAOM were more likely to experience treatment failure than children who did not meet rAOM criteria across all episodes. ConclusionsOur findings indicate that clinical exposures and patient characteristics documented in the EHR distinguish children who are at risk of developing rAOM. Such models could be deployed within EHR systems to identify children who would benefit from early evaluation by an otolaryngologist and audiologist.
Xia, N.; Henningfeld, J.; Steuart, R.
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BackgroundChildren with tracheostomies experience frequent and recurrent acute respiratory infections (ARIs). While cultured respiratory pathogens can inform ARI diagnosis, it is unknown if their presence in the airway affects future ARI risk. ObjectiveTo identify predictors of frequent (3+) ARIs within 36 months of tracheostomy. MethodsWe conducted a single-center, retrospective cohort study of children with tracheostomies placed between 2010-2016. Medical records were reviewed for each encounter in which a respiratory culture was obtained over the 3 years post-tracheostomy. ARIs were defined using encounter ICD-9/10 codes. Logistic and Poisson regression were used to model the association between clinical and microbiologic predictor variables with having frequent (3+) ARIs and the total number of ARIs per child. Mediation analysis using stepwise regression models further evaluated the role of P. aeruginosa. ResultsAmong 436 children, 631 diagnosed ARIs occurred within 36 months of tracheostomy; 20.2% of children had 3+ ARIs. Pseudomonas aeruginosa was isolated in 25% of children and was more common among those with 3+ ARIs compared with 0-2 ARIs (56.8% vs 20.7%, p<0.001). Those with early P. aeruginosa isolation were more likely to have 3+ ARIs (aOR 3.38, 95% CI 1.97-5.81), and this relationship persisted when analyzing ARIs and P. aeruginosa counts. Identification of P. aeruginosa partially mediated the relationship of ventilator dependence with ARI frequency. ConclusionIsolation of P. aeruginosa, particularly early and repeated isolation, is associated with frequent ARIs in the 3 years after tracheostomy and is an important partial mediator. Findings may inform risk stratification and targeted treatment strategies.
BAFUNYEMBAKA, G.; Nacher, M.; Maniassom, C.; Houdouin, V.; Nathan, N.; Elenga, N.
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BackgroundAsthma is a frequent comorbidity in children with sickle cell disease and has been associated with an increased risk of acute complications, particularly vaso-occlusive crises and acute chest syndrome. However, determinants of clinical severity among children with sickle cell disease and confirmed asthma remain poorly characterized, especially in tropical settings. This study aimed to identify factors associated with clinical severity in this population. MethodsWe conducted an observational study among children with sickle cell disease followed in French Guiana. The analysis was restricted to children with confirmed asthma. Clinical severity was defined as the occurrence of at least two hospitalizations during the 12 months preceding evaluation for vaso-occlusive crises and/or acute chest syndrome. Factors associated with severity were assessed using univariate and multivariate logistic regression analyses. ResultsA total of 138 children with sickle cell disease and confirmed asthma were included, of whom 49 (35.5%) presented a severe clinical form. In multivariate analysis, no variable was independently associated with clinical severity. However, a trend toward an increased risk of severe disease was observed among children living in rural areas (adjusted OR = 1.94; 95% CI: 0.77-4.86), while a trend toward a protective effect was observed for Strongyloides stercoralis infection (adjusted OR = 0.18; 95% CI: 0.02-1.51). Allergic sensitization, although frequent (64.5%), was not associated with clinical severity after adjustment (adjusted OR = 0.66; 95% CI: 0.31-1.44). ConclusionAmong children with sickle cell disease and confirmed asthma, more than one third experience severe clinical disease. Severity does not appear to be driven by allergy but may be influenced by environmental and contextual factors specific to tropical settings. These findings support a stratified approach to sickle cell-associated asthma to identify high-risk children and prevent avoidable acute complications.
Villatoro, C.; Yonts, A. B.; Barter, T.; Mohandas, S.; Malone, L. A.
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BackgroundPediatric long COVID is associated with substantial symptom burden, yet evidence-based pharmacologic treatments remain limited. Low-dose naltrexone (LDN) has been proposed as a potential symptomatic therapy, but data in pediatric populations is lacking. MethodsWe conducted a retrospective analysis of pediatric and young adult patients ([≤]25 years) with a clinical diagnosis of long COVID who were prescribed LDN between July 2020 and July 2025 at three multidisciplinary pediatric long COVID programs in the United States. Deidentified clinical data were extracted from medical records. Outcomes included symptom prevalence, dosing practices, treatment continuation or discontinuation, adverse effects, and available patient-reported quality-of-life measures (PedsQL and PROMIS(R)). FindingsThe study included 62 patients (mean age, 15.6 years [range, 8-23]; 53.2% male and 46.8% female). Fatigue was nearly universal (98.4%), followed by headaches (87.1%), brain fog (74.2%), dizziness/lightheadedness (67.7%), anxiety (66.1%), and post-exertional malaise (56.5%). LDN-treated patients demonstrated a higher prevalence of neurocognitive and autonomic symptoms, compared to general clinic cohorts. Most patients (71.0%) reported no adverse effects; the most common were vivid dreams (9.7%) and insomnia (9.7%). At follow-up, 66.1% of patients remained on LDN. Medication discontinuation was attributed to perceived lack of benefit (43.8%) or side effects (25.0%). Baseline quality-of-life measures at initiation showed marked impairment: PedsQL Physical Health (M=38.0, SD=20.9) and Multidimensional Fatigue (M=35.7, SD=15.8) scores were low. PROMIS scores indicated reduced physical functioning (M=36.8, SD=8.7) and cognitive functioning (M=40.8, SD=7.6), with elevated fatigue (M=68.0, SD=10.4) and pain interference (M=58.6, SD=8.2) relative to population norms. The study was not designed to assess efficacy. InterpretationLDN was primarily prescribed to patients with prominent fatigue, neurocognitive symptoms, and autonomic dysfunction, and was generally well tolerated. These findings provide descriptive evidence of real-world prescribing practices and support the need for clinical trials to systematically evaluate LDNs efficacy in pediatric long COVID.
Weill, O.; Lucas, N.; Bailey, B.; Marquis, C.; Gravel, J.
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Objectives: Acute gastroenteritis is a leading cause of pediatric emergency department (ED) visits. While ondansetron reduces vomiting, intravenous rehydration, and hospital admissions, its efficacy when initiated at triage remains unclear. We aimed to evaluate whether triage nurse-initiated administration of ondansetron in children with suspected gastroenteritis reduces the proportion of patients requiring observation following initial physician assessment. Methods: We conducted a randomized, double-blind, placebo-controlled trial in a tertiary pediatric ED in Canada. Children aged 6 months to 17 years presenting with morae than 3 episodes of vomiting in the preceding 24 hours (including 1 within 2 hours of arrival), were eligible. At triage, we randomized participants to receive liquid ondansetron or a color- and taste-matched placebo. The primary outcome was the proportion of patients requiring observation after the first physician evaluation. Secondary outcomes included post-intervention vomiting, ED length of stay, patient comfort, and 48-hour return visits. The trial was registered at ClinicalTrials.gov (NCT03052361). Results: Recruitment was stopped prematurely due to the COVID-19 pandemic. Ninety-one participants were randomized to ondansetron (n= 44) or placebo (n= 47). Overall, 40 patients (45%) were discharged immediately after the initial physician assessment, with no difference between the ondansetron and placebo groups (44% vs. 45%; absolute difference -1%, 95% CI: -20% to 19%). No significant differences were observed in all secondary outcomes. Conclusion: In this trial, triage nurse-initiated ondansetron administration did not reduce the need for ED observation in children with presumed gastroenteritis. While being underpowered, this study could inform researchers planning larger clinical trials.
Aravamuthan, B. R.; Bailes, A. F.; Baird, M.; Bjornson, K.; Bowen, I.; Bowman, A.; Boyer, E.; Gelineau-Morel, R.; Glader, L.; Gross, P.; Hall, S.; Hurvitz, E.; Kruer, M. C.; Larrew, T.; Marupudi, N.; McPhee, P.; Nichols, S.; Noritz, G.; Oleszek, J.; Ramsey, J.; Raskin, J.; Riordan, H.; Rocque, B.; Shah, M.; Shore, B.; Shrader, M. W.; Spence, D.; Stevenson, C.; Thomas, S. P.; Trost, J.; Wisniewski, S.
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Objective Cerebral palsy (CP) affects approximately 1 million Americans and 18 million individuals worldwide, yet contemporary US epidemiologic data remains limited. We aimed to use Cerebral Palsy Research Network (CPRN) clinical registry to describe demographics and clinical characteristics of individuals with CP across the US and determine associations with gross motor function and genetic etiology. Methods Registry subjects were included if they had clinician-confirmed CP and prospectively entered data for Gross Motor Function Classification System (GMFCS) Level, gestational age, genetic etiology, CP distribution, and tone/movement types. Logistic regression was used to determine which of these variables plus race, sex, ethnicity, and age were associated with GMFCS level and genetic etiology. Results A total of 9,756 children and adults with CP from 22 CPRN sites met inclusion criteria. Participants were predominantly White (73.0%), male (57.3%), non-Hispanic (87.8%), and younger than 18 years (73.7%). Most were classified as GMFCS levels I-III (55.6%), born preterm (52.8%), had spasticity (83.8%), and had quadriplegia (41.9%); 12.2% were identified as having a genetic etiology. Tone/movement types, CP distribution, and gestational age were significantly associated with both GMFCS level and genetic etiology (p<0.001). Compared to White individuals, Black individuals were more likely to have greater gross motor impairment (p<0.001). Conclusion In this large US cohort, clinical and demographic factors, including race, were associated with gross motor function and genetic etiology in CP. These findings highlight persistent disparities and demonstrate the value of a national clinical registry for informing prognostication, quality improvement efforts, and targeted genetic testing strategies.
Chen, W.; Ballarin, S.; Fioletova, M.; Bhosale, C. R.; Matthews, T.; Potter, A. K.; Forbes, J.; Blavo, C.
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Objective To evaluate physician knowledge, attitudes, and practices regarding viral exanthem diagnosis and mandatory reporting requirements among practicing physicians in major metropolitan regions of Florida. Study Design An IRB-exempt cross-sectional survey was distributed via REDCap to licensed physicians and residents in family medicine, internal medicine, pediatrics, and infectious disease across Florida. The 19-question survey assessed demographic characteristics, knowledge of viral exanthem diagnosis (measles, rubella, roseola), reporting requirements, physician attitudes, and clinical practices. Knowledge scores were compared by specialty using ANOVA with Tukey post-hoc analysis. Multivariate analysis and linear regression assessed associations between physician confidence and knowledge scores. Results A total of 162 physicians responded, with 146 complete responses included in analysis. Participants included pediatrics (n=74), family medicine (n=48), and internal medicine (n=24). The overall mean knowledge score was 78.5% (SD 20.5). Pediatricians demonstrated the highest scores (82.7%) compared with internal medicine (76.4%) and family medicine (73.3%), with pediatricians scoring significantly higher than family physicians (p=0.04). Differences in vignette-based diagnostic knowledge and mandatory reporting knowledge were not statistically significant across specialties. Roseola was the most commonly diagnosed viral exanthem (66%), followed by measles (30%) and rubella (17%). Most physicians (91.4%) expressed interest in additional training. Conclusions Although overall physician knowledge of viral exanthem diagnosis and reporting was high, clinically meaningful gaps remain, particularly in differentiating similar rash presentations. Pediatricians demonstrated higher knowledge scores than family physicians. Enhanced physician education may improve diagnostic accuracy and public health reporting as vaccination rates decline and outbreaks of vaccine-preventable viral exanthems increase.
Temple, A.-M.; Golden, D.; Temple, J.; D'Adamo, C.
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BackgroundPediatric eczema is a highly prevalent condition that often causes substantial suffering among affected children and their families. Numerous modifiable lifestyle and environmental risk factors for the condition have been identified, although these risk factors and related interventions have generally been studied in isolation. The goal of this study was to evaluate the effects of an integrative program for parents of children with eczema that simultaneously addressed multiple lifestyle and environmental risk factors. MethodsChildren with eczema diagnosis who began the online eczema program and provided outcomes data from May 2024 to May 2025 were eligible. The primary outcome was the Patient-Oriented Scoring Atopic Dermatitis (PO-SCORAD), a validated measure of eczema symptoms and burden. Outcomes were assessed at baseline and at one month, two months, and six months after beginning the program. Changes in mean PO-SCORAD scores from baseline throughout the duration of the study were assessed with analysis of variance (ANOVA). Multivariate linear regression modeling of PO-SCORAD scores using population-averaged generalized estimating equations (GEE) were also constructed accounting for baseline PO-SCORAD scores and adjusting for age, sex, presence of any allergy, use of topical corticosteroids, and use of antihistamines. Results197 participants were included in the study. The mean baseline PO-SCORAD score was 51.4, which is considered severe eczema. PO-SCORAD scores improved over the course of the study (p<0.0001) and there were statistically significant and clinically meaningful improvements noted after one month (11.3 points, 22.0% improvement), two months (17.8 points, 34.6% improvement), and six months (27.2 points, 52.9% improvement) in the program (p<0.0001). After accounting for baseline PO-SCORAD scores and covariates in regression modeling, there was a 22.5-point (p<0.0001) improvement in PO-SCORAD scores from baseline to final assessment. There was a 31.4-point decrease in PO-SCORAD scores from baseline to final assessment (p<0.0001, 47.2% improvement) among the subgroup of participants with severe eczema symptoms at baseline. ConclusionsAn online program focusing on modifiable lifestyle and environmental modifications was associated with clinically meaningful symptom improvements among children with eczema. Symptoms improved relatively quickly and the greatest improvements were noted among children with severe symptoms at baseline.
Shireman, J.; Mukherjee, N.; Brackman, K.; Kurtz, N.; Patniak, A.; McCarthy, L.; Gonugunta, N.; Ammanuel, S.; Dey, M.
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Objectives: Academic medical institutions are the gatekeepers of the physician workforce and shape the future of medicine by regulating medical school admissions as well as residency training. Although broadly the field of medicine is seeing more representation from traditionally underrepresented groups, the critical decision-making platform of academic medicine continues to be uncharacteristically homogeneous, represented mainly by white males. This is even more pronounced in surgical subspecialties, such as academic neurosurgery. This study aims to quantify this phenomenon, uncover its driving factors, and define opportunities for improvement. Methods: Using a mixed research methodology, academic neurosurgical faculty in the U.S were identified, and their demographic data was collected. An internet search using Google Scholar and Scopus was conducted to determine scholarly activity using number of publications and h-index. Results: We found a significant increase in female faculty in academic neurosurgery within the last decade. Comparing the faculty rank amongst male and female faculty, we found that the majority of female faculty are at the assistant professor level (n=36/79; 45.6%) while male faculty are more at the full professor rank (n=265/582; 45.5%). A similar trend was seen for under-represented minority neurosurgery faculty. Strong scholarly activity corelated with a departmental chair position for male faculty, however, this trend was not true for female faculty. There was a significant difference in the number of publications and h-index in female vs male faculty, but only when including male faculty outliers at the full professor level. Conclusion: Slowly but steadily, academic neurosurgery is making progress towards a more diverse and representative workforce in the U.S that better reflects the patient population. Facilitating timely progression of females and URM neurosurgeons into senior professorship and academic leadership roles will further advance this essential progress.
Santorelli, G.; Cheung, R. W.; Bhopal, S.; Wright, J.
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Objective To examine ethnic differences in the incidence and age-related trajectories of childhood health conditions from birth to adolescence within a UK birth cohort. Design Longitudinal population-based birth cohort with linkage to primary care electronic health records. Setting Born in Bradford (BiB), a multi-ethnic birth cohort in Bradford, UK. Participants 13,282 children (36% White British, 44% Pakistani British, 20% other ethnicity) born 2007 to 2011 with linked primary care records and over 1 year follow-up. Main outcome measures Incident diagnoses of atopic conditions (asthma, eczema, allergic rhinoconjunctivitis), overweight/obesity, common mental health disorders (anxiety, depression), and neurodevelopmental disorders (including ADHD and autism). Incidence rates, Kaplan-Meier cumulative incidence, and Cox regression hazards ratios (HRs) were estimated. Results Atopic conditions emerged early (median onset 5 to 6 years) and were more common among Pakistani British children, with higher hazards of eczema (HR 2.29, 95% CI 2.01 to 2.61), allergic rhinoconjunctivitis (HR 2.27, 2.00 to 2.58), and asthma (HR 1.35, 1.22 to 1.50). Overweight/ obesity developed later (median 9 to 10 years) and were also more frequent in Pakistani British children (HR 1.25, 1.16 to 1.35). In contrast, common mental health disorders emerged predominantly in early adolescence (median around 13 years), and both mental health and neurodevelopmental diagnoses were more frequently recorded among White British children; Pakistani British children had lower hazards of neurodevelopmental diagnoses (HR 0.28, 0.23 to 0.35) and mental health disorders (HR 0.53, 0.41 to 0.70). Conclusions Ethnic differences in childhood health are condition-specific and vary by age of onset, emerging at distinct stages. These findings inform the timing of prevention, service planning, and research into underlying mechanism.
Dolin, P.; Keogh, K. A.; Rowell, J.; Edmonds, C.; Kielar, D.; Meyers, J.; Esterberg, E.; Nham, T.; Chen, S. Y.
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Purpose: We evaluated healthcare resource utilization (HCRU) and costs in patients with eosinophilic granulomatosis with polyangiitis (EGPA). Methods: Patients with newly diagnosed EGPA (2017--2021), [≥]12 months' pre-diagnosis health plan enrollment, and [≥]1 inpatient or [≥]2 outpatient claims with an EGPA diagnosis were included. Follow-up was from EGPA diagnosis until disenrollment or database end. HCRU and health insurer payment costs during follow-up were compared with those for matched cohorts of general insured patients without EGPA (comparison A) and without EGPA but with severe uncontrolled asthma (SUA; comparison B). Results: In comparison A, all-cause HCRU was higher in the EGPA cohort (n = 213) versus matched patients (n = 779) for all clinical encounters/pharmacy claim types; annualized, mean total all-cause costs were 16-fold higher ($117,563/patient) versus matched patients ($7,520/patient). In comparison B, all-cause HCRU was higher for the EGPA cohort (n = 182) versus the matched SUA cohort (n = 640) for all clinical encounters/pharmacy claim types, with 5-fold higher mean total all-cause costs ($118,127/patient vs $22,286/patient). In both EGPA cohorts, HCRU and associated costs increased between the baseline and follow-up periods. Conclusions: These findings highlight the need for more effective treatments to reduce the clinical and economic burden of EGPA.
Lee, D.; Lee, C.; Oh, S. S.; Lee, K.; Hyun, C. S.; Shin, J. I.; An, S.; Ioannidis, J.
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BackgroundParticipating in research during medical school is supported by institutional programs and may influence subsequent professional development. ObjectiveWe aimed to describe the current status and heterogeneity of scholarly research programs for medical students in the United States, including expectations, support, and key structural features. MethodsWe conducted a cross-sectional web audit of official webpages for all accredited US MD- and DO-granting medical schools (search performed September 2024 to January 2025). Extracted variables included participation requirements, mentorship, timing and duration (overall and dedicated research time), expected scholarly outputs, funding sources, stipend information, and stated program goals. We compared Carnegie tier R1 (Very high research activity) versus other institutions, QS Top-50 versus other institutions, and MD versus DO schools using {chi}2/Fisher exact tests for 2x2 tables and exact trend or Freeman-Halton tests for multicategory variables. ResultsPrograms were identified for all 202 institutions. Funding was explicitly mentioned by 61.9% (125/202) of programs, 27.0% (51/189) were compulsory, 98.9% (188/190) reported faculty mentorship, and 91.0% (171/188) were exclusive for medical students. Program duration, dedicated time, expected outcomes, stipend reporting, funding sources, and stated goals varied widely. Carnegie R1 institutions had longer duration (P=.002) and tended to report external funding more often than other institutions (25/104, 24.0% vs 9/98, 9.2%; OR 3.13, 95% CI 1.38-7.10; P=.008). QS Top-50 institutions were more likely to require compulsory participation than other institutions (11/19, 57.9% vs 40/170, 23.5%; OR 4.47, 95% CI 1.68-11.87; P=.003). No significant differences were observed between MD and DO programs across most measured characteristics. ConclusionsScholarly research programs for medical students are ubiquitous across US medical schools but heterogeneous in structure, expectations, and support. Research-intensive and top-ranked institutions may have more external funding and sometimes may put together longer and compulsory programs Further evaluation of student experiences and outcomes is warranted.
Gao, S.; Gao, J.; Miles, K.; Madan, J. C.; Pasternack, M.; Wald, E. R.; Gunther, S. H.; Frankovich, J.
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Background Group A streptococcus (GAS) infections have been associated with neuropsychiatric disorders in epidemiologic studies and animal models, but data in US health care populations are limited. GAS is also associated with autoimmune sequelae, including acute rheumatic fever (ARF)/Sydenham chorea (SC), poststreptococcal reactive arthritis (PSRA), poststreptococcal glomerulonephritis (PSGN), and guttate psoriasis (GP). Epstein-Barr virus (EBV) has been linked to systemic lupus erythematosus (SLE) and multiple sclerosis (MS) and the complexity of these associations parallels that of GAS-associated conditions, providing a useful comparison. Objectives 1) Assess the association between a positive GAS test and incident neuropsychiatric diagnoses within 1 year in a large US health care database. 2) Assess the validity of the same database in detecting well-established disease associations while avoiding false associations. Design, Setting, Participants Retrospective cohort study using TriNetX data from US health care organizations. Patients with positive or negative tests were propensity score-matched (GAS cohort n=178,301; EBV cohort n=64,854). Patients with documented neuropsychiatric diagnoses prior to testing were excluded. To approximate a primary care population, inclusion required at least one well-visit. Exposures Positive vs negative GAS test; positive vs negative EBV test (separate cohorts). Main Outcomes and Validations Main outcome: incident neuropsychiatric diagnoses within 1 year of GAS testing. Positive control outcomes: ARF/SC, PSRA, PSGN, and GP (for GAS cohort); SLE and MS (for EBV cohort). Negative control outcomes: conditions without known association with GAS. Results After matching, a positive GAS test was associated with attention-deficit/hyperactivity disorder (ADHD) (RR: 1.09; 95% CI: 1.03-1.15). Among established poststreptococcal conditions, only GP was associated with prior GAS (RR: 1.75; 95% CI: 1.06-2.89). Case counts were insufficient to evaluate ARF/SC, PSRA, and PSGN. Negative control outcomes showed no association. In the EBV cohort, no association was observed with SLE, and MS showed a decreased risk. Conclusions and Relevance A positive GAS test was associated with ADHD but not with other neuropsychiatric disorders. The database detected poststreptococcal GP but did not identify most established postinfectious autoimmune associations, likely reflecting rarity, heterogeneity, and diagnostic complexity. These findings begin to describe the range of real-world health care databases to evaluate postinfectious neuropsychiatric risk.
Dias, J. M. L.; More, R. P.; Butler, D.; Aldus, C.; Brown, J.; French, C. E.; Dolling, H.; Raymond, L.; Rowitch, D. H.; Aiken, C. E.
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ImportanceWhole genome sequencing (WGS) is increasingly used to diagnose severely ill children, yet the long-term impact of a genetic diagnosis on healthcare utilization and resource allocation remains poorly understood. ObjectiveTo determine the influence of a genetic diagnosis via WGS on long-term healthcare utilization metrics in severely ill children. DesignA retrospective cohort study using data from the Next Generation Children study (2016-2020) with record linkage and analysis of primary care records conducted between 2022 and 2024. SettingA multicenter study involving primary care and hospital records linked via the UK National Health Research Institute (NIHR) Rare Disease Bioresource, Cambridge, UK. ParticipantsA referred sample of 270 severely ill children who underwent WGS. Exposure(s)Receipt of a genetic diagnosis (87/270; 32%) compared to those who remained undiagnosed (183/270; 68%) following WGS. Main Outcome(s) and Measure(s)Comparison of 36 healthcare utilization parameters, including hospitalizations, primary care prescriptions, and diagnostic tests. ResultsAmong the 270 children analyzed, those receiving a genetic diagnosis (n=87) exhibited significantly higher overall healthcare utilization compared to undiagnosed peers (n=183). This included increased hospital admissions and outpatient visits, particularly for neurodevelopmental and seizure-related conditions. Diagnosed children received a higher volume of neurological, gastrointestinal, and nutritional prescriptions. The most pronounced differences in utilization were observed in children initially diagnosed in neonatal (NICU) or pediatric (PICU) intensive care settings. While genetic diagnosis was not associated with reduced healthcare costs during the study period, it was linked to more targeted, condition-specific medical care. Conclusions and RelevanceWGS diagnosis facilitates the integration of specialist care and the alignment of healthcare resources with the specific needs of children with complex disorders. These findings suggest that while costs may not decrease immediately, a diagnosis enables more precise and targeted clinical management. Key PointsO_ST_ABSQuestionC_ST_ABSDoes a genetic diagnosis through whole genome sequencing influence long-term healthcare utilization in severely ill children? FindingsIn this cohort study of 270 children, those who received a genetic diagnosis demonstrated significantly greater overall healthcare utilization, including more hospitalizations and targeted prescriptions, compared with undiagnosed children. MeaningA genetic diagnosis facilitates the integration of specialized, condition-specific care, helping to align healthcare resources with the individual needs of children with complex disorders.
Bieling, F.; Kirchgatter, A. M.; Bauer, A.; Weiss, C.; Mueller, H.; Matzel, K.; Rowald, A.; Besendoerfer, M.; Diez, S. M.
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Objectives. To compare the efficacy and safety of invasive sacral neuromodulation (SNM) and noninvasive enteral neuromodulation (ENM) in children with refractory gastrointestinal motility disorders (GMD). Materials and Methods. This prospective interventional trial enrolled pediatric patients with GMD between 2019 and 2024 at a single tertiary referral center. Children with inflammatory bowel disease or mechanical causes of GMD were excluded. Participants received either SNM via an implanted device or ENM via surface electrodes. Stimulation was delivered at 14 Hz, 210 s pulse width, with individualized intensity (median 1.0 mA for SNM; 6.0 mA for ENM). Primary outcomes were abdominal pain, fecal incontinence, defecation frequency, and stool consistency. Treatment success was defined as clinically significant improvement in at least two of these four domains. Quality of life was assessed at baseline and 12 weeks. Safety outcomes were monitored over a 12-month follow-up. Results. Of 70 eligible patients, 48 completed the study (18 SNM; 30 ENM). Diagnoses included Hirschsprung disease, functional constipation, and congenital neuronal malformations. Severe comorbidities were more frequent in the SNM group (45%) than the ENM group (3%; P = .0018). Treatment success was observed in 80% of ENM and 83% of SNM patients (P = 1.00). No significant differences were found between groups for individual outcomes. No major complications occurred. Minor adverse events were comparable (ENM 27% vs SNM 17%; P = .50). Conclusions. Both SNM and ENM are effective and safe options for treating pediatric GMD and may be considered within a multimodal therapeutic approach.
yin, h.; He, S.; Wu, Z.; Tan, W.; Du, F.; Yang, C.; Yu, H.
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Methods: Using Global Burden of Disease (GBD) data, we analyzed prevalence, incidence, mortality, and disability-adjusted life years (DALYs) rates across global and 21 GBD regions from 1990-2023. Joinpoint regression identified temporal trends, age-period-cohort models analyzed effect contributions, Das Gupta decomposition quantified demographic and epidemiological impacts, inequality indices assessed health equity, and Bayesian models projected 2024-2038 trends. Results: In 2023, the global number of children and adolescents with asthma reached 131 million, with an age-standardized prevalence rate (ASPR) of 1,789.9 per 100,000. From 1990 to 2023, the global ASPR and age-standardized incidence rate (ASIR) of asthma in children and adolescents showed an upward trend, while the age-standardized mortality rate (ASMR) and age-standardized disability-adjusted life years (DALYs) rate (ASDR) exhibited a downward trend. Among the 0-14 age group, the disease burden was greater in males than in females, whereas in the 15-19 age group, males had a lower disease burden than females. Projections indicate that over the next 15 years, the overall disease burden will continue to decline; however, female mortality rates and DALYs rates are projected to show an upward trend. Conclusions: The increasing prevalence and incidence rates, coupled with declining mortality and DALYs rates of asthma among children and adolescents globally, underscore the necessity for targeted public health interventions. These findings provide crucial insights for early diagnosis, treatment optimization, and global health policy formulation.
Vollmuth, Y.; Soric, B.; Beer, J.; Behrends, U.; Paolini, M.; Blaschek, A.; Meyer-Buehn, M.; Klein, C.; Huebner, J.; Dobler, G.; Schober, T.
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BackgroundThe epidemiology of suspected pediatric meningoencephalitis has shifted in the era of conjugate vaccines and multiplex PCR diagnostics, with viral pathogens now predominating over bacterial causes. Updated epidemiologic data are essential to adapt diagnostic and therapeutic algorithms to current clinical practice. MethodsThis retrospective single-center study included children and adolescents <18 years who underwent lumbar puncture with cerebrospinal fluid multiplex PCR for suspected central nervous system infection at a tertiary-care pediatric hospital in Germany between 2016 and 2024. Clinical, laboratory, and outcome data were extracted from electronic medical records. Cerebrospinal fluid was analyzed using the BioFire(R) FilmArray(R) Meningitis/Encephalitis Panel. Statistical analyses included descriptive statistics, nonparametric group comparisons, receiver operating characteristic analyses. ResultsAmong 1,198 included children, definite bacterial meningitis was diagnosed in 13 (1.1%), definite viral meningitis in 80 (6.7%), aseptic meningitis of unknown etiology in 131 (11.0%), confirmed/probable encephalitis in 53 (4.4%), and possible encephalitis in 34 (2.8%). Bacterial meningitis accounted for 5.8% of all meningitis cases. A causative pathogen was identified in all bacterial meningitis cases, most commonly Streptococcus pneumoniae (n = 7). Enterovirus (n = 52) and parechovirus (n = 9) predominated in viral meningitis, whereas an infectious etiology was identified in only 13 of 53 confirmed/probable encephalitis cases. The Bacterial Meningitis Score showed a sensitivity of 80.0% and a specificity of 57.6%. The recently published UK-ChiMES-pre- and post-lumbar puncture scores demonstrated sensitivities of 84.6% and 76.9% and specificities of 86.3% and 92.7%, respectively. DiscussionBacterial meningitis was rare in this contemporary cohort, while viral and etiologically unresolved infections predominated despite routine multiplex PCR diagnostics. Clinical prediction scores supported risk stratification, with the UK-ChiMES-pre-lumbar puncture score showing the most favorable balance between sensitivity and specificity and potential to guide diagnostic decisions and antiinfective therapy.
Bianchina, N.; Fischer, C.; Rai, K.; Clawson, J.; McBeth, L.; Gottenborg, E.; Keniston, A.; Burden, M.
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BackgroundHigh workload among healthcare workers has increasingly been correlated with poor patient outcomes, inefficient operational and financial outcomes, and burnout. Despite growing literature exploring causes of attending physician workload, there is limited understanding of trainee-specific measures. ObjectiveWe aimed to characterize elements contributing to trainee workload and perceived challenges and satisfiers to the trainee workday as a foundation for better understanding and measuring trainee work experience. MethodsInternal Medicine and Medicine-Pediatrics residents at an academic medical center were invited to participate in focus groups discussing contributors to inpatient workload and work experience between March and April 2024. A qualitative content analysis identified key metrics of trainee workload and work experience, which were then consolidated into overarching domains. A structured, multi-round rating process ranked the perceived relevance of each metric. ResultsTwenty residents participated across six focus groups. Analysis of focus groups yielded 297 workload metrics across 28 unique domains. Seventeen domains had metrics identified as highly relevant (median 6-7; IQR < 1) including autonomy, communication, disruptions, task switching, documentation, emotional burden, patient factors, professional fulfillment, rounding, teaming, and work-life balance. ConclusionsResident physicians highlighted complex interactions between clinical factors, work design, and psychosocial dynamics that contribute to their sense of workload. This creates opportunities to develop unique measures of workload to understand the trainee experience better. Further studies are needed to capture the generalizability of these findings and the relationship between these workload domains and patient, organizational, and trainee outcomes with the aim of implementing evidence-based work design.
Luff, A.; Shields, M.; Hirschtick, J.; Ingle, M.; Crosh, C.; Marsh, M.; Modave, F.; Fitzpatrick, V.
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BackgroundWell-child visits (WCVs) are essential for preventive care, yet missed appointments often lead to delayed interventions. We developed and validated models to predict next-visit nonattendance using routine electronic health record data. MethodsUsing data from two Chicago-area pediatric practices, Practice A (1,215 patients; 3,654 visits) and Practice B (1,271 patients; 3,044 visits), we compared regularized logistic regression, random forest, and XGBoost models. Predictors included visit context, prior utilization, and patient characteristics. Models were trained on Practice A and validated on Practice B. ResultsMissed-next-visit rates were 16.2%(A) and 20.7%(B). In external validation, performance was similar across models (AUC 0.66-0.68). At the threshold maximizing F1 score, recall ranged from 0.54-0.71. The LASSO logistic regression model identified six key predictors: timepoint, visit delay, prior no-shows, schedule lead time, new patient status, and immunization refusal. SHAP values confirmed these process measures as among the most influential features across all models. ConclusionPredicting WCV nonattendance is feasible using routine data. A simple logistic regression model performs comparably to complex algorithms, offering a practical pathway for clinical integration. By identifying at-risk families during a current appointment, this may enable clinicians to provide proactive support to support preventive care before a lapse occurs. ImpactO_LIMissed well-child visits are common, leading to an increasing number of preventable acute care visits, delayed recognition of developmental delays, and missed opportunities to initiate early intervention C_LIO_LIA multimodal approach is needed to support well-child visit attendance C_LIO_LIMachine learning is an emerging tool to predict well-child visit no show rates with implications for future interventions to support families at risk for missing well-child visits and promote positive health outcomes C_LI
Narayanan, N.; Murrill, M. T.; Burrough, W.; Mochizuki, T.; Panina, C.; Tamerat, M.; Fink, J.; Wu, I. L.; Salcedo, K.; Katrak, S. S.; Mayo, T.; Chitnis, A.; Hsieh, C.; Noor, Z.; Lewis, G.; Jaganath, D.
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ObjectiveTo evaluate whether new tuberculosis (TB) tools in the electronic health record (EHR) can support latent TB infection (LTBI) screening, testing and treatment among children and adolescents in a primary care setting. Study DesignThis retrospective cohort study included children and adolescents between the ages 1-25 years who had a well-child or well-adolescent visit at a Federally Qualified Health Center in Oakland, California, from December 2021 to December 2022. Four new EHR tools were introduced for the completion and documentation of TB risk factor screening, testing and treatment. Data were extracted from the EHR to identify gaps in these steps, and logistic regression was used to examine factors associated with completion of TB infection screening and testing. Acceptability was evaluated using provider satisfaction surveys before and after the implementation of TB EHR tools. ResultsOf 5,879 individuals (median age of 9 years at first visit, interquartile range (IQR) 4-13 years), 94% completed TB risk factor screening. Among those with a new risk factor, 59% had a TB infection test ordered and 96% completed testing. Ten participants (3%) tested positive, all initiated LTBI treatment, and most (n=7, 70%) completed treatment. Overall, 5,162 (88%) individuals completed their LTBI care cascade. Younger children ages 1-4 years were more likely to be screened for TB risk factors, but were less likely to be tested. Provider satisfaction increased from 40% to 71% for risk factor screening, and 36% to 77% for test ordering. ConclusionEHR tools supported completion of the pediatric LTBI care cascade, while also increasing provider satisfaction. EHR-based solutions show promise as part of multi-component strategies to address gaps in LTBI care for children and adolescents.