Aims and Scope Translational Pediatrics (Transl Pediatr, tp.amegroups.com; tp.amegroups.org, Print ISSN 2224-4336, Online ISSN 2224-4344) is an open access, peer-reviewed, monthly journal (quarterly published from Jul. 2012 to Oct. 2019, bimonthly published from Dec. 2019 to Dec. 2020). TP publishes articles in all aspects of health care in newborns, children and adolescents. Contributions pertinent to pediatrics are also included from related fields such as nutrition, surgery, respiration, dentistry, urology, public health, child health services, human genetics, basic sciences, psychology, psychiatry, education, sociology, and nursing. Currently, TP has been indexed by PubMed/PubMed Central, Scopus and Web of Science [Science Citation Index Expanded (SCIE)]. The SCIE impact factor 2025 for TP is 2. TP is the official journal of the Society of Translational Medicine (STM). 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Example of statements in published articles: https://actr.amegroups.org/article/view/8875; https://med.amegroups.org/article/view/8358/html. Contact with Editorial Office Mira Wu, Corresponding Editor, Translational Pediatrics Email: editor@thetp.org Publisher Information TP is published by AME Publishing Company. Addresses: Hong Kong branch office: Flat/RM C 16F, Kings Wing Plaza 1, NO. 3 on Kwan Street, Shatin, NT, Hong Kong, China. Updated on June 17, 2026
Need for increasing uptake of influenza vaccination and investing in novel vaccines for paediatric influenza prevention
Do financial incentives improve children’s long-term adherence to medication in asthma?
Extended-phase anticoagulation for pediatric venous thromboembolism after EINSTEIN Jr
The utility of sugammadex in infants: a shifting clinical paradigm
New study does not support use of dexmedetomidine-remifentanil to change neurodevelopmental outcomes of sevoflurane
Expanding preventive options for pediatric migraine: the use of anti-CGRP monoclonal antibodies for children and adolescents with migraine
Pediatric sedation is not adult sedation in small: lessons from a PK/PD analysis of remimazolam
Irinotecan plus temozolomide for pediatric relapsed or refractory solid tumors: a retrospective cohort study
Background: Relapsed or refractory pediatric solid tumors carry a poor prognosis, and effective salvage therapies are limited. The combination of irinotecan and temozolomide (IT) has shown promise in early studies, but real‑world data in Chinese pediatric populations are lacking. This study aims to evaluate the efficacy and safety of the IT regimen in children with recurrent or refractory solid tumors. Methods: This single‑center retrospective cohort study included 86 pediatric patients with relapsed or refractory malignant solid tumors treated at Beijing Jingdu Children’s Hospital (March 2021–March 2025). Inclusion criteria were pathologically confirmed recurrence/progression, adequate organ function, and no prior IT therapy. Patients received irinotecan (50 mg/m2/day, intravenous, days 1–5) and temozolomide (100–150 mg/m2/day, oral, days 1–5) in 21‑day cycles. Tumor response was assessed every two cycles using RECIST 1.1. Primary endpoints were objective response rate (ORR) and progression‑free survival (PFS); secondary endpoints were disease control rate (DCR), overall survival (OS), and safety. Survival was estimated by Kaplan‑Meier with time zero set to IT initiation. Results: Of 86 patients (median age 7 years, range 1–18 years; 52 neuroblastoma), the median number of prior chemotherapy lines was 3 (range 1–7). ORR was 23.3% [95% confidence interval (CI): 15.1–33.6%] and DCR 52.4% (95% CI: 41.3–63.3%). For neuroblastoma (n=52), ORR was 31.0% (95% CI: 19.5–44.5%) and DCR 67.3% (95% CI: 53.7–78.8%). Median PFS was 5.5 months (95% CI: 3.8–8.1) and median OS 9.8 months (95% CI: 6.5–14.2). The 1‑year OS rate was 36.8% (95% CI: 27.5–49.4%) and 1‑year PFS rate 22.4% (95% CI: 14.7–34.4%). Grade III–IV adverse events included neutropenia (25.2% of cycles), thrombocytopenia (26.0%), diarrhea (3.6%), and liver dysfunction (4.0%). No treatment‑related deaths occurred. Conclusions: The IT regimen demonstrates clinical activity and was found to be tolerable in this cohort. These findings are hypothesis‑generating and warrant confirmation in prospective controlled studies.
Early physical growth and neurodevelopment in full-term small for gestational age infants: reference points from propensity score matching and restricted cubic splines
Background: Full-term small for gestational age (SGA) infants are at risk for impaired neurodevelopment, but quantitative descriptions of growth-related developmental patterns and heterogeneity within this population remain limited. This study aimed to examine the associations of 1-year neurodevelopment with physical growth and birth-related factors in full-term SGA and appropriate for gestational age (AGA) infants, and to explore heterogeneity within the SGA group using age- and sex-adjusted relative length at 1 year. We also sought to assess whether SGA status remained associated with developmental quotient (DQ) scores after accounting for measured covariates and estimate model-derived weight-ratio reference points corresponding to the average DQ levels of AGA infants. Methods: In this retrospective cross-sectional study, 448 SGA and 2,376 AGA children were included. Birth data, anthropometric measurements (weight, length, and head circumference) at 1 year, and Gesell Developmental Scale (GDS) scores were collected. Generalized linear models (GLMs) were used to examine factors associated with DQ in the SGA and AGA groups separately. Heterogeneity within the SGA group was further explored by stratifying infants according to age- and sex-adjusted relative length at 1 year. Two-step propensity score matching (PSM) with weighted GLMs was used to compare DQ scores between SGA and AGA infants after matching the measured covariates. Restricted cubic spline (RCS) models were applied to assess nonlinear associations between the 1-year weight ratio (SGA/AGA) and DQ. Results: In the SGA group, 1-year weight was positively associated with DQ across multiple domains, including fine motor, adaptive behavior, language, and personal-social behavior (all P<0.01). Across age- and sex-adjusted relative-length strata, lower relative length was accompanied by lower weight, shorter length, smaller head circumference, and lower DQ scores across all five GDS domains (all P≤0.002). After matching the measured covariates, the SGA and AGA groups showed no statistically significant differences in DQ scores across domains (all P>0.05). Weight ratio showed nonlinear associations with DQ in all five domains (all P<0.001). The model-derived reference points at which the predicted mean DQ was closest to the average AGA DQ for the gross motor, fine motor, adaptive behavior, and language domains were 0.901, 0.909, 0.864, and 0.890, respectively. The personal-social domain showed a U-shaped pattern, with the lower crossing point at 0.741. Conclusions: In this clinic-based retrospective cross-sectional sample, neurodevelopmental scores at 1 year in full-term SGA infants were more closely associated with postnatal growth status than with SGA status after matching. Relative length at 1 year also revealed substantial heterogeneity within the SGA group. The weight-ratio values identified by spline models may serve as statistical reference points for developmental comparison; however, they should not be interpreted as causal or universal clinical targets. Because development was assessed only at 1 year, these findings should be interpreted cautiously.
Novel inflammatory biomarkers and disease severity in hospitalized children with Mycoplasma pneumoniae pneumonia: a retrospective study
Background: A resurgence of Mycoplasma pneumoniae (M. pneumoniae) infections has been observed worldwide, including large outbreaks in China during 2023–2024, with a higher incidence of severe and complicated pneumonia cases. Identifying clinical and laboratory indicators associated with disease severity may assist clinicians in early risk stratification. This study aimed to explore the association between novel inflammatory biomarkers and disease severity in hospitalized children with M. pneumoniae pneumonia (MPP). Methods: We conducted a retrospective analysis of 866 hospitalized children with MPP. For each case, we calculated the neutrophil-to-lymphocyte ratio (NLR), monocyte-to-lymphocyte ratio (MLR), platelet-to-lymphocyte ratio (PLR), systemic immune-inflammation index (SII), and systemic inflammation response index (SIRI). Multivariable logistic regression analysis was performed to identify factors associated with severe MPP. Receiver operating characteristic (ROC) curves were used to evaluate the discriminatory performance of these biomarkers for distinguishing severe cases from non-severe MPP. Results: Compared with the non-severe group, children with severe MPP showed significantly higher levels of NLR, PLR, and SII (all P<0.001), whereas SIRI levels were lower (P=0.01). Patients with severe MPP were also older and had longer fever duration (P<0.001). In ROC analysis, SII demonstrated limited discriminatory ability for identifying severe MPP. The combination of SII and fever duration showed the highest area under the curve (AUC) among the evaluated indices. Conclusions: Elevated SII and prolonged fever duration were associated with increased disease severity in children with MPP. Although the discriminatory performance of SII alone was limited, inflammatory indices may provide additional information for clinical risk stratification when interpreted alongside clinical features. Further prospective studies are needed to validate these findings.
Comparative effectiveness of structured clinician-guided and self-guided parent training for preschool attention-deficit/hyperactivity disorder (ADHD) risk: a randomized controlled trial
Background: Attention-deficit/hyperactivity disorder (ADHD) in preschool-aged children presents a significant developmental and public health challenge globally. In Thailand, early identification and intervention are constrained by diagnostic uncertainty, limited standardized care, and cultural misalignment with Western-based treatment models. Although parent management training (PMT) is the recommended first-line non-pharmacological intervention for early-onset ADHD, its effectiveness and adaptability in collectivist, resource-limited contexts remain underexplored. This study aimed to evaluate the efficacy of a culturally adapted, group-based PMT program for Thai caregivers of preschoolers at risk for ADHD, with the goal of reducing symptoms and enhancing positive parenting compared to standard educational materials. Methods: This single-blind randomized controlled trial (RCT) enrolled caregivers of preschool children aged 3–5 years identified as being at risk for ADHD using the Thai ADHD Screening Scales (THASS). Participants were randomly assigned to either a culturally adapted group-based PMT intervention or an educational materials control condition. Outcomes were assessed at baseline, 2 weeks, and 6 weeks using standardized caregiver-reported measures. Primary efficacy analyses were conducted using generalized estimating equations (GEE) to account for correlations between repeated outcome measurements over time. Results: The intervention group demonstrated significant reductions in ADHD symptoms (Cohen’s d>1.2; P<0.001), with sustained improvements across hyperactivity-impulsivity and inattention domains through 6 weeks. Positive parenting scores improved at 2 weeks (d=0.74), though gains were partially reduced at 6 weeks. No adverse events occurred, and retention was 100%, indicating strong acceptability of the intervention model. Conclusions: This study provides the first controlled evidence for a culturally adapted, group-based PMT intervention in Southeast Asia. Its feasibility, brevity, and alignment with cultural values support its potential as a scalable early intervention model in low-resource, collectivist settings. Trial Registration: Thai Clinical Trials Registry (TCTR20260202021).
Associations of picky eating levels with dietary diversity, nutrient intake, and eating behaviors in preschool children with autism spectrum disorder: a cross-sectional study
Background: Picky eating is common among children with autism spectrum disorder (ASD). However, the specific effects of different levels of picky eating on nutritional status, dietary diversity, and eating behaviors in preschool-aged children with ASD remain unclear. This study aimed to compare these aspects between children with low versus high levels of picky eating. Methods: A cross-sectional study design was employed, enrolling 130 children with ASD aged 3 to 6 years who met the DSM-5 diagnostic criteria [Diagnostic and Statistical Manual of Mental Disorders Fourth Edition (DSM-V)]. Trained personnel measured each child's height and weight using standardized instruments. Validated dietary diversity scores and preschool eating behavior scales were used to assess dietary diversity and nutrient intake and eating behaviors. energy and macronutrient intake were quantified through three non-consecutive 24-hour dietary recalls. Children were divided into a low picky eating group and a high picky eating group based on the degree of picky eating for comparative analysis. Results: There were no statistically significant differences in height and weight indicators between the two groups, with most children falling within the normal range of growth and development. However, distinct differences were observed in nutrient intake and eating behaviors: children in the low picky eating group showed significantly higher total dietary diversity scores (P<0.05). Regarding nutrient intake, children in the low picky eating group exhibited higher intake levels of total energy, polyunsaturated fatty acids, and dietary fiber compared to those in the high picky eating group; in specific food categories, intake of starchy tubers, fruits, nuts, and legumes was also significantly higher. Regarding eating behaviors, children in the low picky eating group showed lower scores for picky eating and problematic eating behaviors, whereas exhibited heightened satiety responsiveness, with all differences reaching statistical significance (P<0.05). Conclusions: The degree of picky eating is a key factor influencing dietary quality and eating behaviors in preschool-age children with ASD. Low levels of picky eating are associated with more comprehensive nutrient intake, richer dietary diversity, and more favorable eating behavior patterns. Early identification and intervention targeting picky eating behavior are of significant importance for improving the nutritional status of children with ASD.
Analysis of differences in response to long-acting growth hormone therapy and influencing factors between patients with idiopathic short stature (ISS) and growth hormone deficiency (GHD)
Background: Long-acting growth hormone [pegylated recombinant human growth hormone (PEG-rhGH)] is increasingly used for pediatric short stature, yet its differential effects between growth hormone deficiency (GHD) and idiopathic short stature (ISS) remain unclear. This study aims to compare the 52-week efficacy and safety of PEG-rhGH in prepubertal children with GHD versus ISS within a Chinese clinical cohort. Methods: A single-center retrospective cohort study enrolled prepubertal children (3–10 years for girls, 3–11 years for boys) with GHD/ISS (June 2023–June 2024). All received once-weekly PEG-rhGH [starting dose: 0.2 mg/kg/week, adjusted by insulin-like growth factor 1 standard deviation score (IGF-1 SDS)/height velocity (HV)] and completed 12-month follow-up. Primary endpoint: 12-month Δheight standard deviation score (ΔHt-SDS); secondary endpoints: HV, ΔIGF-1 SDS, target height achievement rate. Safety was assessed via adverse events (AEs) and metabolic/hepatorenal parameters. Regression analyses identified influencing factors. Results: There were 132 children (68 GHD, 64 ISS) included, with balanced baselines. At 12 months: (I) efficacy: GHD had higher ΔHt-SDS (0.92±0.31 vs. 0.65±0.27, P<0.001), HV (9.8±1.6 vs. 8.2±1.4 cm/year, P<0.001), target height achievement rate (64.7% vs. 43.8%, P=0.009), and ΔIGF-1 SDS (1.8±0.7 vs. 1.1±0.6, P<0.001); (II) safety: AE incidence was similar (GHD: 32.4%, ISS:35.9%, P=0.63), with no serious AEs. ISS had slightly higher fasting insulin (15.8±4.2 vs. 12.3±3.5 µU/mL, P<0.001) and low-density lipoprotein cholesterol (LDL-C) (2.8±0.5 vs. 2.5±0.4 mmol/L, P=0.002), all within normal ranges; (III) influencing factors: diagnosis type (β=0.32), baseline IGF-1 (β=0.21), and PEG-rhGH dose (β=0.18) independently affected ΔHt-SDS (all P<0.01). Conclusions: GHD children respond better to PEG-rhGH than ISS children; both have good safety. Diagnosis type is the key efficacy factor, supporting subtype-specific PEG-rhGH strategies.
Family quality of life among parents of children diagnosed with atopic dermatitis: a cross-sectional study at a tertiary hospital in Jeddah
Background: Atopic dermatitis (AD) is an immune-mediated inflammatory process characterized by pruritus and skin dryness. It is considered one of the most common chronic conditions in pediatrics, affecting an estimated 20% of children worldwide. While previous studies showed AD has a clear and significant impact on caregivers of children with AD, our geographical area still needs similar evidence, given the scarcity of studies in this field. As far as our knowledge goes, this is the first study of its kind in Saudi Arabia. This study aimed to assess family quality of life among parents and legal guardians of children with AD attending a university hospital in Saudi Arabia, in order to support a more comprehensive family-centered approach to disease management. Methods: Our study is a single-center, cross-sectional, caregiver survey-based study measuring the family quality of life of caregivers of AD patients using the Family Dermatology Life Quality Index (FDLQI). We hypothesized that caregivers in our population would have impaired quality of life, consistent with global data. We recruited caregivers of children (aged 0–14 years) diagnosed with AD at the pediatric allergy clinic of King Abdulaziz University Hospital, Jeddah, between July 2023 and August 2024 via telephone contact. Family quality of life was measured using the validated FDLQI, scored 0–30, with higher scores indicating greater impairment. Results: Our results indicate that FDLQI scores, reflecting impaired family quality of life, were higher among mothers (mean 11.08±6.2) compared to fathers (mean 8.16±3.8), but the overall difference between caregivers was not statistically significant. The overall mean FDLQI score among all participants was 10.22±5.84. While no significant differences were identified by caregiver role, child age, disease duration or comorbidities, the overall FDLQI score (10.22±5.84) indicates moderate family quality of life impairment. These findings provide the first local evidence from Saudi Arabia; null results should be interpreted with caution given the limited sample size and the lack of data on disease severity. Conclusions: Family quality of life is impaired among caregivers of children with AD in Jeddah, regardless of child age, disease duration, or parental role, underscoring the need for universal psychosocial support in pediatric dermatology practice. Our findings provide foundational local evidence and highlight the need for larger longitudinal studies in the region.
Transcriptomic remodeling of bone marrow mesenchymal stromal cells in pediatric B-cell acute lymphoblastic leukemia: a four-gene signature
Background: Pediatric B-cell acute lymphoblastic leukemia (B-ALL) is molecularly heterogeneous and influenced by the bone marrow microenvironment. Mesenchymal stromal cells (MSCs) provide critical niche signals, yet their transcriptomic remodeling in B-ALL is poorly defined. Methods: We analyzed GSE101425 (GPL570) MSC microarrays (rather than leukemic blasts) from pediatric B-ALL at diagnosis (day 0, n=35), remission (n=29), relapse (n=6), and healthy donors (n=16). Differential expression (day 0 vs. healthy), Random Forest feature ranking, consensus clustering, and Gene Ontology (GO), Kyoto Encyclopedia of Genes and Genomes (KEGG), and Gene Set Enrichment Analysis (GSEA) analyses were performed. Within-cohort discrimination used 5-fold cross-validated logistic regression with bootstrapped area under the receiver operating characteristic curve (AUC) confidence intervals (CIs) (CCN4 mapped to WISP1 on GPL570). Public single-cell RNA sequencing (scRNA-seq) data provided cellular context. Peripheral blood real-time quantitative PCR (RT-qPCR) served as an exploratory assessment of systemic expression, not MSC validation. Results: We identified 78 differentially expressed genes (DEGs) in B-ALL-associated MSCs. A four-gene MSC signature [Dickkopf WNT Signaling Pathway Inhibitor 1 (DKK1), Regulator of G Protein Signaling 2 (RGS2), Cellular Communication Network Factor 4/WNT1-Inducible Signaling Pathway Protein 1 (CCN4/WISP1), Lysozyme (LYZ)] distinguished active B-ALL MSCs from healthy MSCs with cross-validated AUC 0.883 (95% CI: 0.783–0.966) and separated samples into two transcriptional states with distinct pathway enrichment, including extracellular matrix and immune-related programs. Single-cell data localized RGS2 and LYZ to immune compartments, whereas DKK1 and CCN4/WISP1 were low, supporting stromal specificity. Peripheral blood RT-qPCR showed decreased RGS2 and LYZ, whereas CCN4 showed a nonsignificant upward trend (P=0.053), underscoring compartment-dependent patterns. Conclusions: These data delineate transcriptional remodeling of bone marrow MSCs in pediatric B-ALL and nominate a four-gene MSC-associated signature. By centering on stromal MSC transcriptomes rather than leukemic blasts, we target a complementary microenvironmental layer of B-ALL biology that is underrepresented in blast-centric transcriptomics. The findings are hypothesis-generating and require independent MSC cohorts and functional validation.
Efficacy and safety of inhaled ambroxol hydrochloride solution in Chinese pediatric patients with acute lower respiratory tract infections: a real-world, multicenter, open-label, single-arm study
Background: Although randomized controlled trials (RCTs) have confirmed the mucolytic efficacy of ambroxol hydrochloride solution for inhalation (AHSI) in selected cohorts, their stringent exclusion criteria often omit children with comorbidities and complex presentations encountered in routine practice. Consequently, real-world evidence is needed to evaluate the effectiveness and safety of AHSI in a broader, clinically representative pediatric population with acute lower respiratory tract infections (ALRTIs). XXXXXXXXXXXX. Methods: This real-world, multicenter, open-label, single-arm study enrolled hospitalized patients aged ≥6 months with ALRTI (acute bronchitis, bronchiolitis, or pneumonia) and symptom duration <7 days across 62 centers in China (April 2021–April 2022). Key inclusion criteria included a cough score ≥2 (0–4 scale), tenacious sputum, and difficulty expectorating. Major exclusions comprised severe pneumonia, bronchial asthma, interstitial lung disease, significant hepatic or renal dysfunction [alanine aminotransferase (ALT) >1.5× upper limit of normal (ULN), total bilirubin (TBil) or serum creatinine (Scr) > ULN], other severe comorbidities, known hypersensitivity to ambroxol, or recent trial participation. Participants received weight-based doses of nebulized AHSI twice daily for 7 days as add-on to standard care. Follow-up visits occurred at day 4 and day 7 (end of treatment). Primary endpoints were the cough improvement rate (defined by a reduction in cough score) and the overall clinical response rate (investigator-assessed improvement). Secondary endpoints included changes from baseline in cough, throat rales, and pulmonary auscultation scores. Safety assessments comprised monitoring of adverse events (AEs) (coded with MedDRA), vital signs, and laboratory tests (hematology, biochemistry, urinalysis) at baseline and day 7. Results: A total of 2,599 children were enrolled [full analysis set (FAS)]. At baseline, mean age was 3.60±2.50 years, 57.6% were male, and symptom scores were: cough 2.10±0.30, throat sputum 1.65±0.59, lung auscultation 1.44±0.70. In the FAS, the cough improvement rate was 96.73% [95% confidence interval (CI): 96.05–97.41] and the clinical response rate was 94.73% (95% CI: 93.87–95.59). All symptom scores decreased significantly from baseline to day 7 (P<0.001). Drug-related AEs (DRAEs) occurred in 0.39% of patients, predominantly mild-to-moderate rash, transient liver enzyme elevations, and gastrointestinal events; no serious DRAEs were reported. Outcomes were consistent across pneumonia and bronchitis subgroups and across various nebulizer types. Conclusions: This large real-world study demonstrated that a 7-day course of AHSI, added to standard care, was associated with clinically meaningful improvements in respiratory symptoms and a favorable safety profile in children with ALRTI. The consistent effects across disease subtypes and nebulizer devices underscore the practical utility of AHSI in diverse pediatric settings. While the single-arm design limits causal inference, these findings provide robust real-world evidence supporting AHSI as an effective expectorant option. Prospective confirmation through RCTs will further define its role in first-line therapy.
Adrenal AKR1C3-mediated androgen activation contributes to hyperandrogenism in prepubertal ovariectomized rats
Background: Hyperandrogenism during childhood is a key feature of several pediatric endocrine disorders, including premature adrenarche and polycystic ovary syndrome (PCOS). The adrenal gland contributes significantly to circulating androgens, yet the regulation of adrenal steroidogenic enzymes during the prepubertal period remains poorly understood. This study aimed to investigate adrenal androgen synthesis in a prepubertal rat model of ovarian removal and identify key enzymes responsible for hyperandrogenism. Methods: Female Sprague-Dawley rats underwent either ovariectomy or sham surgery on postnatal day (PND) 15. Serum steroid hormone levels were measured by enzyme-linked immunosorbent assay (ELISA). Adrenal tissue was analyzed by RNA sequencing to screen for differentially expressed genes (DEGs) involved in steroidogenesis; findings were subsequently validated by quantitative real-time polymerase chain reaction (qPCR), western blot, and immunohistochemistry (IHC). The functional role of a candidate gene in steroid synthesis was assessed through in vitro overexpression and in vivo pharmacological inhibition. Results: Serum testosterone levels increased on PND 25 and 30 after ovariectomy, whereas dehydroepiandrosterone (DHEA) concentrations decreased. Transcriptomic analysis revealed that adrenal AKR1C3 was the most significantly DEG between OVX and sham-operated controls. Its expression was elevated in OVX rats from PND 25 to PND 30, as confirmed by qPCR, western blot, and IHC. In vitro, AKR1C3 overexpression converted exogenous androstenedione to testosterone. Conversely, in vivo administration of the AKR1C3 inhibitor ASP9521 (2 mg/kg.day) to OVX rats reduced serum testosterone levels. Additionally, adrenal expression of CYP17A1, another key enzyme in androgen biosynthesis, was also increased in OVX rats from PND 25 to 30. Conclusions: This study demonstrates that prepubertal ovarian removal upregulates adrenal AKR1C3 and CYP17A1, leading to enhanced adrenal androgen synthesis and systemic hyperandrogenism. These findings provide mechanistic insights into adrenal-derived hyperandrogenism with potential clinical implications for pediatric endocrine disorders such as premature adrenarche and PCOS.
Procalcitonin in predicting acute kidney damage in children with initial febrile urinary tract infection: a retrospective study
Background: Urinary tract infection (UTI) is the most common bacterial infection in children, with 10–15% progressing to irreversible renal scarring. This study evaluated the predictive value of inflammatory markers for acute renal damage in children with first febrile UTI. Methods: This study conducted a retrospective analysis of 143 children with initial febrile UTI who underwent technetium-99m labelled dimercaptosuccinic acid (99mTc-DMSA) scintigraphy for kidney damage assessment and had serum C-reactive protein (CRP), white blood cell (WBC) count, absolute neutrophil count, neutrophil percentage (NEUT%), and procalcitonin (PCT) measured. The efficiency of above inflammatory markers in predicting acute kidney damage was calculated and compared. Results: Univariate regression analysis revealed significant associations between acute kidney damage and CRP, absolute neutrophil count, and PCT. Multivariate regression identified both CRP [odds ratio (OR) 1.013, 95% confidence interval (CI): 1.003–1.024, P=0.02] and PCT (OR 1.671, 95% CI: 1.066–2.621, P=0.03) as independent predictors, with area under the curve (AUC) values of 0.718 and 0.815, respectively. Notably, a PCT cutoff >0.26 ng/mL demonstrated superior predictive value (sensitivity 87.1%, specificity 69.0%) compared to other markers. Conclusions: Early measurement of inflammatory markers, particularly PCT, have significant predictive value for acute kidney damage in pediatric febrile UTI. While both PCT and CRP serve as reliable predictors, PCT demonstrates superior diagnostic accuracy, providing a new method to identify early kidney damage in children presenting with first febrile UTI for primary hospitals without single photon emission computer scanner or gamma camera.
The early predictive value of Cys-C, lactic acid, and urinary microglobulins in acute kidney injury following neonatal asphyxia
Background: Neonatal asphyxia is a critical perinatal complication closely associated with acute kidney injury (AKI), which seriously affects the prognosis of neonates. Early and accurate biomarkers are urgently needed for timely identification and risk prediction of AKI in asphyxiated newborns. This study aimed to explore the predictive value of cord blood and urinary renal injury markers for neonatal AKI after asphyxia. Methods: A total of 85 neonates with asphyxia admitted to Jinjiang City Hospital (Fujian Branch of Shanghai Sixth People’s Hospital) from January 2023 to March 2025 were analyzed. All patients underwent renal injury marker testing, including cord blood cystatin C (Cys-C) and lactic acid, urinary β2-microglobulin (β2-MG), transferrin (TRF), and α1-microglobulin (α1-MG). Neonates were classified into an AKI group and a non-AKI group based on AKI occurrence. Levels of serum Cys-C, lactic acid, and renal injury marker were compared between the two groups. The predictive value of serum Cys-C, lactic acid, urinary β2-MG, TRF and α1-MG levels, and their individual or combined effects on neonatal AKI was evaluated. Results: The incidence of AKI in asphyxiated neonates was 42.35% (36/85). The AKI group had a significantly higher proportion of severe asphyxia compared to the non-AKI group (P<0.05). Blood Cys-C, lactic acid, urinary β2-MG, TRF and α1-MG levels were significantly elevated in the AKI group (P<0.05). The combination of blood Cys-C, lactic acid levels with urinary β2-MG, TRF, α1-MG yielded a sensitivity of 75%, a specificity of 98.0% and an area under the curve (AUC) of 0.909 for predicting AKI in asphyxia newborns, both significantly higher than individual indicators (P<0.05), The specificity was-consistent to that of single indicators. Conclusions: The study shows that Cys-C, lactic acid, urinary β2-MG, TRF, and α1-MG are effective early indicators for monitoring renal injury after neonatal asphyxia resuscitation. Each of these indicators has predictive value for AKI in asphyxia newborns, with the combined five indicators demonstrating even higher predictive value, providing a more accurate assessment of early renal function status in such infants.
Clinical phenotype, gonadal development, and comorbidity spectrum in 43 children with triple X syndrome: a single-center retrospective descriptive case series with cytogenetic refinement in patients with and without X-monosomy-containing cell lines
Background: Triple X syndrome (TXS) is a common yet under-diagnosed sex chromosome aneuploidy with significant phenotypic heterogeneity. Previous studies have typically described TXS as a homogeneous entity, lacking karyotype-stratified analysis. This study aimed to compare clinical phenotypes, gonadal development, and comorbidity profiles across karyotype subgroups in pediatric TXS patients. Methods: In this single-center retrospective chart review with descriptive subgroup characterization (no formal between-group hypothesis tests performed given the small subgroup sizes), we analyzed clinical data from 43 TXS patients (aged 3 days to 16 years) diagnosed at Jiangxi Provincial Children’s Hospital between January 2019 and November 2024. Patients were stratified into three subgroups: non-mosaic group, X-monosomy mosaic group (containing 45,X cell lines), and non-X-monosomy mosaic group (47,XXX/46,XX without 45,X cell lines). Karyotyping was performed by G-banding analysis of peripheral blood lymphocytes (≥20 metaphases routinely; ≥30 if mosaicism was suspected); fluorescence in situ hybridization (FISH) counting ≥200 interphase nuclei was used in 5 selected cases (where mosaicism was suspected, the phenotype was atypical, or a critical clinical decision required cytogenetic refinement) to confirm or refine mosaicism, and a 5% threshold was applied for retaining low-frequency cell lines. Data included karyotype, anthropometric measurements [including height standard deviation score (HtSDS) and body mass index (BMI) standard deviation score (BMISDS)], sex hormones [follicle-stimulating hormone (FSH), luteinizing hormone (LH), estradiol (E2), anti-Müllerian hormone (AMH)], uterine/ovarian ultrasound, and bone age. Results: Of 43 patients, 31 (72.1%) were non-mosaic, 9 (20.9%) were X-monosomy mosaic, and 3 (7.0%) were non-X-monosomy mosaic. The median age at diagnosis was 7.58 years and median height was 117.6 cm. Fifteen patients (34.9%) experienced spontaneous menarche at a mean age of 11.13±1.28 years, which appeared earlier than the commonly cited general population average (~12.0 years), although a formal statistical comparison was not performed. For descriptive purposes, mean bone age difference (chronological age − bone age) was +1.36 years in the X-monosomy mosaic subgroup (n=7 with bone age data) and −0.10 years in the non-mosaic subgroup (n=20 with bone age data); these subgroup-level descriptions are presented in parallel without formal between-group testing. Comorbidity profiles by subgroup are reported descriptively: across the cohort, neuropsychiatric disorders were the most prevalent comorbidity (11/43, 25.6%); within the X-monosomy mosaic subgroup, skeletal anomalies (3/9, 33.3%) and renal anomalies (2/9, 22.2%) were observed, a pattern consistent with features known in Turner syndrome. One patient was diagnosed with premature ovarian insufficiency (POI) (peak FSH 57.16 U/L, AMH 0.12 ng/mL). Conclusions: In this descriptive single-center case series, patients with X-monosomy-containing cell lines were observed to have phenotypic features overlapping both Turner syndrome (disproportionate bone age delay, renal/skeletal anomalies) and typical 47,XXX (neuropsychiatric findings). Given the small subgroup sizes and the single-center design, these subgroup-level descriptions are presented to inform future hypothesis-driven studies rather than to support formal between-group conclusions. We outline a tentative, practice-informed framework intended to support—rather than replace—individualized multidisciplinary follow-up; prospective multi-center validation is required before adoption as a clinical management algorithm.
Routine admission biomarkers for identifying co-infection and stratifying severity in pediatric macrolide-resistant Mycoplasma pneumoniae pneumonia: a retrospective cohort study
Background: Early identification of co-infection and timely risk stratification remain difficult in children with pneumonia caused by macrolide-resistant Mycoplasma pneumoniae (MRMP). This study aimed to investigate whether routinely available admission parameters were associated with bacterial and/or viral co-infection and severe disease in children with MRMP pneumonia. Methods: This single-center retrospective cohort study included children with MRMP pneumonia admitted to The Sixth Affiliated Hospital of Harbin Medical University from August 1 to December 31, 2023. MRMP was confirmed by targeted next-generation sequencing (t-NGS) detection of M. pneumoniae with macrolide resistance-associated mutations. Patients were first classified as having MRMP mono-infection or MRMP co-infection with bacteria and/or viruses based on clinical, laboratory, imaging, and t-NGS findings. The co-infection group was then further divided into mild and severe subgroups according to disease severity. Demographic characteristics and admission laboratory parameters were compared between groups. Logistic regression and receiver operating characteristic (ROC) analyses were performed. Results: A total of 278 children with MRMP pneumonia were enrolled, including 78 with mono-infection and 200 with co-infection. In the first-stage analysis, platelet (PLT) levels on admission were higher in the co-infection group than in the mono-infection group [290.50 (243.00, 362.00) vs. 265.00 (220.50, 326.25)×109/L, P=0.03]. However, no independent factor associated with co-infection was identified by multivariable analysis, and the discriminative performance of PLT was limited [area under the curve (AUC) =0.584]. Among the 200 co-infected patients, 159 were mild and 41 were severe. Compared with the mild group, the severe group had higher neutrophil percentage (NEUT%), D-dimer, lactate dehydrogenase (LDH), and procalcitonin (PCT) levels, but lower lymphocyte count (LYM) and albumin (ALB) levels on admission. Multivariable logistic regression showed that ALB was independently associated with lower odds of severe disease [odds ratio (OR) =0.800, 95% confidence interval (CI): 0.710–0.900, P<0.001], whereas PCT was independently associated with a higher odds (OR =9.440, 95% CI: 1.110–80.340, P=0.04). The combination of ALB and PCT yielded an AUC of 0.720 for identifying severe co-infection, compared with 0.704 for ALB alone and 0.704 for PCT alone. Conclusions: In children with MRMP pneumonia, PLT showed limited discriminatory value for identifying bacterial and/or viral co-infection, whereas lower ALB and higher PCT on admission were associated with severe disease among co-infected children.
Characteristics and influencing factors of bronchoalveolar lavage fluid cellular patterns in children with Mycoplasma pneumoniae pneumonia: a cross-sectional study
Background: Mycoplasma pneumoniae pneumonia (MPP) is a common cause of community-acquired pneumonia in children. Clinical assessment currently relies mainly on blood inflammatory markers. Bronchoalveolar lavage fluid (BALF) cellular analysis provides an important window to directly assess local pulmonary inflammation. Small studies have demonstrated BALF neutrophil predominance in pediatric MPP. However, large-scale studies on the characteristics and influencing factors of BALF cellular patterns are lacking. In particular, it remains unclear whether local MP-DNA load independently associates with neutrophil predominance or whether common blood inflammatory markers correlate with BALF cellular patterns. This large cross-sectional study aimed to characterize BALF cellular patterns and investigate influencing factors. Methods: This single-center cross-sectional study consecutively enrolled children with confirmed MPP (positive BALF MP-DNA) hospitalized at Tianjin Children’s Hospital from November 2023 to April 2024. Diagnosis followed the Chinese Guidelines for the Diagnosis and Treatment of Mycoplasma pneumoniae Pneumonia in Children (2023 Edition). BALF cytological examination were performed in accordance with the Consensus of Chinese Experts on Morphological Examination of Bronchoalveolar Lavage Fluid Cells [2023]. Clinical and laboratory covariates were extracted from medical records. Spearman’s rank correlation, Kruskal-Wallis H test, Mann-Whitney U test and multivariable linear regression were used for statistical analysis. Results: (I) Among 238 children (mean age 7.79±2.76 years; 49.2% male), median BALF neutrophil proportion was 80.0% [interquartile range (IQR), 71.8%, 84.0%], lymphocyte proportion 3.0% (IQR, 2.0%, 5.0%), and macrophage proportion 13.5% (IQR, 11.0%, 19.0%). (II) Univariable analysis showed a positive correlation between BALF MP-DNA load and neutrophil proportion (rs =0.268, P<0.001). Multivariable linear regression confirmed that MP-DNA load remained independently associated with BALF neutrophil proportion [B=5.435; 95% confidence interval (CI): 3.073–7.796; P<0.001]. The high MP-DNA load group (≥107 copies/mL) had a significantly higher neutrophil proportion than the medium‑load group (Z=−3.924, P<0.001). (III) BALF neutrophil proportion was not significantly correlated with common blood inflammatory markers [leukocyte, neutrophil, lymphocyte counts, procalcitonin (PCT), lactate dehydrogenase (LDH), and ferritin] in univariable analysis (all P>0.05). C-reactive protein (CRP) showed a weak univariable correlation (rs =0.144, P=0.03), but it was not independently associated in multivariable analysis (P=0.17). (IV) Exploratory analysis of pleural effusion indicated its presence was associated with a lower BALF lymphocyte proportion in univariable analysis (Z=−2.093, P=0.04); multivariable regression was not feasible due to limited cases (n=38). Conclusions: In children with MPP, BALF cellular patterns are characterized by neutrophil predominance, which independently associated with local MP-DNA load, but not with most common blood inflammatory markers. The findings indicate BALF cellular patterns provide complementary information for assessing pulmonary immune status and inflammatory phenotypes in pediatric MPP, while not establishing causality or therapeutic benefit.
Clinical characteristics and therapeutic experiences of magnetic foreign body ingestion in children: a single-center retrospective study
Background: XXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXXX. The study aims to summarize the clinical features and treatment experiences associated with the ingestion of multiple magnetic foreign bodies (MMFB) in children. Methods: A retrospective study was performed on 160 pediatric patients diagnosed with MMFB ingestion at our hospital between June 2016 and August 2025. Results: Among the 38.8% (62/160) of patients who received conservative treatment, foreign bodies passed through the anus in 50 cases, were removed by gastroscope in 8 cases, and were extracted by colonoscopy in 1 case. In three patients, the foreign bodies were partially removed by gastroscopy, with the remainder passing spontaneously. Surgical treatment was required in 61.2% (98/160) of patients. In the surgical group, the incidence of gastrointestinal perforation was 92.9% (91/98), and 65.3% (64/98) were symptomatic. Of the 52.0% (51/98) of patients who underwent laparoscopic surgery, 56.8% (29/51) required conversion to laparotomy. A total of 48.0% (47/98) of patients underwent direct laparotomy. The surgical group had a median of seven beads [interquartile range (IQR): 4–12], which was significantly higher than that in the conservative group, which had a median of three beads (IQR: 2–6) (P<0.001). A marked difference in hospital stay length was observed between the surgical and conservative groups [11.5 days (IQR: 10–13 days) vs. 2 days (IQR: 1–3 days); P<0.001]. Furthermore, on multivariable analysis, vomiting [odds ratio (OR) =8.79, P=0.01] and abdominal pain (OR =5.10, P=0.02) were independent predictors of surgical intervention; the number of magnetic beads also independently predicted surgery, with each additional bead increasing the odds by 6% (OR =1.06 per bead, P=0.04). For gastrointestinal perforation, vomiting emerged as the strongest predictor (OR =13.67, P=0.002), followed by abdominal pain (OR =3.88, P=0.04) and number of beads (OR =1.06 per bead, P=0.02). A threshold of four or more magnets conferred substantially increased risk for both outcomes. Conclusions: Surgical intervention is required in more than 60% of children with MMFB ingestion, especially when vomiting is present or the magnet count is four or more. Conservative management may be considered in asymptomatic children with fewer magnets, however, timely surgery is critical once symptoms develop.
CaMKIIα inhibition facilitates functional recovery after preterm hypoxic-ischemic brain injury by disrupting microglial GPNMB signaling
Background: Preterm brain injury involves white matter damage and aberrant synaptic development, yet the mechanisms linking these pathologies remain unclear. Our previous work demonstrated that hypoxia-ischemia (HI) upregulates microglial glycoprotein non-metastatic melanoma protein B (GPNMB), which is released and correlates with abnormal synaptogenesis and neurobehavioral deficits. However, the downstream signaling pathways by which GPNMB impairs synaptic function are unknown. Given the central role of hippocampal Ca2+/calmodulin-dependent protein kinase II (CaMKII) and its substrate, the glutamate receptor A1 (GluA1) subunit of the α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) receptor, in synaptic signaling and spine development, we investigated whether they mediate GPNMB-induced impairments in immature brain post-HI. Methods: Preterm HI brain injury was induced in P3 C57BL/6 mice using a modified Rice-Vannucci model. Microglia-specific GPNMB overexpression mice were achieved via crosses between Cx3cr1-CreERT2 and R26-CAG-LSL-GPNMB mice. The α-isoform of calmodulin-dependent protein kinase II (CaMKIIα) inhibitor KN93 (10 mg/kg, i.p.) was administered. Synaptic markers [synaptophysin (SYN), postsynaptic density protein 95 (PSD95), GluA1], signaling molecules [p-CaMKIIα, cAMP response element-binding protein (CREB), brain-derived neurotrophic factor (BDNF)], and ultrastructure were assessed by western blot, immunofluorescence, and electron microscopy. Protein interaction was predicted by AlphaFold 3. Long-term neurobehavioral function was evaluated using open field, rotarod, and Morris water maze tests. Results: HI increased the expression levels of synaptic proteins (SYN, PSD95), damaged the synaptic ultrastructure, and induced neurobehavioral deficits. All these deficits were exacerbated by microglial GPNMB overexpression, which was accompanied by increased expression levels of p-CaMKIIα, CaMKIIα and GluA1. Immunofluorescence analysis revealed that GPNMB overexpression significantly enhanced the co-localization and fluorescence intensity of GluA1 and PSD95 in the hippocampal CA1, CA3 and DG subregions, which was reversed by KN93 administration. KN93 reversed GPNMB-induced upregulation of p-CaMKIIα, CaMKIIα and GluA1, and enhanced CREB1 and BDNF expression. It also restored synaptic structure and ameliorated behavioral deficits. In the HI injury model, KN93 administration significantly reduced HI-induced upregulation of p-CaMKIIα, t-CaMKIIα, PSD95, and SYN, and improved behavioral outcomes in both wild-type and GPNMB-overexpressing HI mice, with a more pronounced effect in the latter group. Conclusions: Microglial GPNMB aggravates post-HI synaptic and behavioral deficits by promoting CaMKIIα phosphorylation and GluA1 dysregulation. Inhibition of CaMKIIα using KN93 reinstates synaptic integrity and neurobehavioral performance, demonstrating that the GPNMB-CaMKIIα-GluA1 axis is a pathogenic pathway and a potential target for intervention in preterm brain injury.
Quantitative assessment of multidimensional health disparities and their determinants among vulnerable children
Background: Despite global efforts to advance health equity and social development, significant gaps remain in safeguarding children’s health. This study quantitatively assessed multidimensional health disparities between vulnerable children and non-vulnerable children. The aim was to provide empirical evidence to inform the development of strategies for reducing health disparities, promoting health equity, and improving welfare outcomes for this population. Methods: A cross-sectional survey design was employed to assess general health outcomes and to investigate the potential impact of familial structural factors among vulnerable children. Data were collected from students in 14 primary schools located in Shanxi Province, China. Of the 1,560 questionnaires distributed, 1,516 were returned, and 1,464 were valid, yielding a response validity rate of 96.6%. Among the respondents, 283 met the criteria for classification as vulnerable children. Results: Significant differences in physical and psychological health were observed between vulnerable children residing in urban and rural settings. Functional capacities presented a tendency to decline with increasing grade level. Learning ability and self-concept demonstrated a downward trend as grade level increased. Psychological concerns were prevalent and varied by sex. Families of vulnerable children faced challenges related to economic status, cultural capital, and family structure, with family composition demonstrating a potential impact on health outcomes. Greater parental level of involvement was associated with improved quality of life (QoL), while children with psychological needs were at a potential elevated risk for adverse outcomes. Conclusions: Children identified as vulnerable demonstrated pronounced disadvantages in psychosocial well-being, physical health, and living conditions. Key factors associated with general health status included household economic conditions, parental marital and educational status, and the degree of parental attention to the psychological needs of children. Enhancing the general health and QoL for vulnerable children necessitates targeted policy interventions, enhanced family-based support, and expanded access to community-based health and social services.
Identification of mechanical ventilation-related risk factors for retinopathy of prematurity in preterm infants
Background: Retinopathy of prematurity (ROP) remains a leading cause of preventable childhood blindness. While gestational age (GA) and oxygen therapy are established risk factors, prior research has largely treated oxygen exposure as an aggregate variable, without disaggregating the impact of specific respiratory support modalities. This study investigated whether the mode and duration of respiratory support independently influence ROP risk. Methods: We conducted a single-center retrospective cohort study of 83 preterm infants (GA <28 weeks) admitted to the neonatal intensive care unit (NICU) at Jeonbuk National University Hospital (2014–2024). Modality-specific durations [conventional ventilation, high-frequency oscillatory ventilation (HFOV), non-invasive neurally adjusted ventilatory assist (NIV-NAVA), nasal continuous positive airway pressure (NCPAP), and high-flow nasal cannula (HFNC)] and time-weighted average (TWA) ventilator settings [fraction of inspired oxygen (FiO2), mean airway pressure (MAP), positive end-expiratory pressure (PEEP), and peak inspiratory pressure (PIP)] at postnatal days 1, 3, 7, and 10 were extracted from continuous electronic medical records (EMRs). Associations with ROP were evaluated using Firth’s penalized logistic regression, with and without GA adjustment. Results: ROP was diagnosed in 52 infants (62.7%). In unadjusted analyses, GA [odds ratio (OR) =0.54; 95% confidence interval (CI): 0.35–0.80; P<0.001] and NCPAP duration (OR =1.04; 95% CI: 1.01–1.09; P=0.01) were significantly associated with ROP, alongside TWA PEEP on days 7 and 10. After GA adjustment, NCPAP duration remained the sole independently associated variable (OR =1.04; 95% CI: 1.00–1.08; P=0.04). No other modality, TWA parameter (including FiO2), or respiratory severity index showed an independent association after adjustment for GA. Conclusions: NCPAP duration is independently associated with ROP in infants born before 28 weeks, with each additional day associated with a 4% increase in risk after adjustment for GA. This association was independent of mean FiO2, suggesting that oxygenation variability during spontaneous breathing may be a more proximal pathophysiological driver; however, as oxygen saturation (SpO2) variability was not directly measured, this interpretation should be regarded as hypothesis-generating. NCPAP duration may serve as a practical EMR-derived metric for ROP risk stratification in the NICU.
Thyroid disease burden among individuals aged 0–19 years in low-SDI countries, 1990–2035: a systematic analysis of the Global Burden of Disease Study 2023
Background: Thyroid diseases can adversely affect growth, neurodevelopment, and long-term health during the first two decades of life. Because low Socio-demographic Index (SDI) countries may experience substantial disability despite comparable prevalence, clarifying this burden is essential for equitable pediatric endocrine service planning. This study aims to evaluate the prevalence and disability-adjusted life years (DALYs) burden of non-neoplastic thyroid diseases among individuals aged 0–19 years in 50 low-SDI countries from 1990 to 2023 and project trends to 2035. Methods: We used Global Burden of Disease (GBD) 2023 estimates derived from vital registration systems, household surveys, disease registries, hospital records, and published literature. Prevalence counts, prevalence rates, DALY counts, DALY rates, and age-sex-country-year-specific population denominators were extracted for individuals aged 0–19 years. Temporal trends were assessed using joinpoint regression and average annual percent change (AAPC). Age- and sex-specific patterns, Spearman correlations with SDI, slope index of inequality, concentration index, frontier efficiency gaps, and Bayesian age-period-cohort (BAPC) projections were analysed. All rates are reported per 100,000 population with 95% uncertainty intervals (UI). Results: The total population aged 0–19 years in the 50 low-SDI countries increased from approximately 440.5 million in 1990 to 847.7 million in 2023. Over the same period, prevalent cases increased from 6.22 million (95% UI: 4.38–8.80) to 11.39 million (95% UI: 7.87–16.58), while prevalence rates remained stable (AAPC =−0.06). Total DALYs increased from 0.19 million (95% UI: 0.13–0.29) to 0.32 million (95% UI: 0.23–0.49), whereas DALY rates decreased significantly (AAPC =−0.46). Females bore higher burden across all ages, peaking at 15–19 years, and neonates had persistently high DALY rates (130.2 per 100,000). Country-level heterogeneity was substantial; Haiti had the highest prevalence rate (2,752.0 per 100,000) and DALY rate (85.6 per 100,000), whereas Mali and Nepal showed the smallest efficiency gaps for prevalence and DALY rate, respectively. DALY rates negatively correlated with SDI (R =−0.36, P=0.01). By 2035, prevalent cases are projected to reach 18.37 million (95% UI: 0.18–36.56) and DALYs to reach 0.37 million (95% UI: 0.002–0.75). Conclusions: In low-SDI countries, stable prevalence rates mask a rapidly expanding absolute pediatric thyroid disease burden driven by population growth. Reducing avoidable disability requires integrating maternal iodine nutrition, dried blood spot newborn screening for congenital hypothyroidism, school-based detection for adolescents, and affordable levothyroxine and other essential thyroid medicines into universal health coverage benefit packages.
Gamma-aminobutyric acid supplementation combined with gonadotropin-releasing hormone analog therapy versus gonadotropin-releasing hormone analog therapy alone for linear growth in children with growth retardation: a retrospective cohort study
Background: Growth retardation is a significant clinical challenge in children with central precocious puberty (CPP) or early and fast puberty (EFP) in children receiving gonadotropin-releasing hormone agonist (GnRHa) therapy and represents an important unmet need in pediatric endocrine practice, particularly where access to recombinant human growth hormone is limited. Methods: This single-center retrospective cohort study included 150 pediatric patients with CPP or EFP (mean age 9.0 years, 83.3% female) who showed growth retardation [growth velocity (GV) <5 cm/year] despite adequate GnRHa suppression of hormone levels. The intervention group (n=99) received 100 mg of gamma-aminobutyric acid (GABA) orally before bedtime in addition to standard GnRHa therapy, while the control group (n=51) received GnRHa alone. Baseline covariates, including age, height standard deviation score (HtSDS), bone age/chronological age (BA/CA) ratio, sex, target height, growth velocity (GV), insulin-like growth factor-1 (IGF-1) levels, and duration of prior GnRHa treatment, were collected from medical records at enrollment using standardized chart review protocols. Follow-up assessments were conducted at 3, 6, and 12 months. The primary outcome was the change in height standard deviation score (ΔHtSDS) after 12 months. Secondary outcomes included GV, predicted adult height (PAH), and adverse events (AE). Differences between groups were analyzed using Welch’s t-test. A robustness test used analysis of covariance (ANCOVA) after adjusting for baseline age, HtSDS, BA/CA, sex, target height, GV, IGF-1, and months of GnRHa treatment. Results: A total of 150 children were enrolled. Baseline characteristics were well balanced between groups (mean age: 9.0 vs. 9.0 years; mean HtSDS: 0.13 vs. 0.13; BA/CA ratio: 1.05 vs. 1.05; GnRHa duration: 18.5 vs. 17.8 months; all P>0.05). At 12 months, ΔHtSDS in the GnRHa + GABA group was significantly higher than that in the control group (0.53±0.22 vs. 0.05±0.12; difference 0.48, 95% confidence interval (CI): 0.43–0.53, P<0.001). The GABA group also showed a significantly higher ΔPAH value (+2.75 cm; 95% CI: 2.55–2.95, P<0.001). GV was similar during months 0–3 (4.15±0.48 vs. 4.16±0.48 cm/year; P=0.90) but was higher with GABA during months 3–6 (4.79±0.53 vs. 4.18±0.48; P<0.001) and months 6–12 (5.98±0.64 vs. 4.19±0.49; P<0.001). No clinically meaningful heterogeneity of treatment effect was observed across prespecified subgroups. AE rates were similar (22.2% vs. 17.6%, P=0.67). Conclusions: GABA supplementation was associated with safe and significant improvements in linear growth in children with growth retardation during GnRHa therapy, providing a potentially practical adjunctive treatment for this clinical challenge.
Development and external validation of machine learning models to predict insulin resistance among iron-deficient children and adolescents
Background: Insulin resistance (IR) represents a critical metabolic complication in iron-deficient children, yet existing predictive models target general or overweight pediatric populations and do not account for iron-deficiency as a distinct risk modifier. This study aimed to develop and externally validate machine learning (ML) models using routinely available clinical parameters to address this diagnostic gap. Methods: We utilized data from 222 iron-deficient children and adolescents aged 6 to 17 years from the China Health and Nutrition Survey (CHNS) for model training, and 125 cases from two hospitals for external validation. Iron-deficiency was defined using age- and sex-specific soluble transferrin receptor (sTfR) thresholds, with exclusion of elevated high-sensitivity C-reactive protein (hs-CRP) (≥5 mg/L) or missing metabolic variables. IR was defined as Homeostatic Model Assessment for Insulin Resistance (HOMA IR) exceeding 3.0. Least Absolute Shrinkage and Selection Operator (LASSO) regression selected nine predictors from 27 candidate variables (demographics, anthropometrics, blood pressure, hematology, glucose metabolism, lipids, hepatic and renal function). Four ML algorithms [logistic regression (LR), random forest (RF), k-nearest neighbor (KNN), and extreme gradient boosting (XGBoost)] were developed and evaluated by area under the curve, sensitivity, specificity, and calibration, with five-fold repeated cross-validation for internal validation. SHapley Additive exPlanations (SHAP) analysis quantified predictor contributions. Results: XGBoost achieved optimal discriminative performance with an external validation area under the receiver operating characteristic curve (AUC) of 0.940 [95% confidence interval (CI): 0.889–0.991], outperforming other algorithms. RF demonstrated the highest training AUC (0.993, 95% CI: 0.987–1.000) with near-perfect sensitivity (0.985, 95% CI: 0.920–1.000) but showed limited generalization capacity given minimal training-validation divergence. LR and KNN achieved lower validation AUC values of 0.832 (95% CI: 0.743–0.922) and 0.823 (95% CI: 0.740–0.905), respectively. XGBoost was selected as the final model based on superior specificity (0.967, 95% CI: 0.906–0.993) and tighter CIs, indicating more stable performance estimation. Fasting glucose (mean |SHAP| =0.707) and triglycerides (0.383) emerged as dominant predictors, while albumin demonstrated a protective association [odds ratio (OR) 0.86, 95% CI: 0.78–0.95]. Conclusions: This study establishes an externally validated, interpretable ML framework for predicting IR among iron-deficient youth using routine clinical data. While the XGBoost model demonstrates promising discriminative performance and geographic generalizability, the modest sample size and single-province validation limit immediate deployment readiness. Prospective multi-site validation is required before any consideration of clinical implementation as a developmental screening framework.
Comparative effectiveness of low-dose versus high-dose vitamin D on bone metabolic markers in preterm infants: a retrospective cohort study
Background: Recommendations for vitamin D (VD) supplementation in preterm infants remain inconsistent, and the dose range used in clinical practice is broad. Evidence comparing commonly used regimens in infants born at less than 32 weeks’ gestation remains limited, particularly for early biochemical outcomes during the first postnatal month. Methods: This single-center retrospective cohort study included preterm infants with a gestational age of less than 32 weeks who were discharged from the Neonatal Intensive Care Unit of the Women’s Hospital, Zhejiang University School of Medicine between April 1, 2024 and May 31, 2025. Infants received either 500 IU/day (n=124) or 900 IU/day (n=115) of VD starting during postnatal days 5 to 8. Eligible infants had no prior VD supplementation and available 4-week outcome data; infants with major congenital anomalies, severe hepatic or renal dysfunction, major metabolic disorders, or protocol non-adherence were excluded. Serum 25-hydroxyvitamin D [25(OH)D] was measured by liquid chromatography-tandem mass spectrometry. Calcium, phosphorus, and alkaline phosphatase (ALP) were assessed within the first week after birth and again at 4 weeks of postnatal age. Major neonatal complications were also recorded. Results: Baseline characteristics were similar between groups, including gestational age, birth weight, parenteral nutrition duration, and first-week calcium, phosphorus, and ALP concentrations (all P>0.05). At 4 weeks of postnatal age, serum 25(OH)D concentrations were similar between the 500 IU/day and 900 IU/day groups (median (interquartile range), 31.46 (26.18–39.52) ng/mL versus 32.52 (24.94–38.57) ng/mL; P=0.771), and VD status distribution did not differ significantly. In gestational-age-stratified analysis, ALP was higher in the 900 IU/day group among infants born at 28+0 to 31+6 weeks’ gestation, whereas no clear between-group difference was observed in infants born at less than 28 weeks’ gestation. Calcium and phosphorus concentrations, as well as the incidences of major neonatal complications, did not differ significantly between groups. Conclusions: In preterm infants born at less than 32 weeks’ gestation, 500 IU/day and 900 IU/day VD supplementation were associated with similar serum 25(OH)D concentrations and broadly comparable calcium-phosphorus biochemical findings by 4 weeks of postnatal age. The 900 IU/day regimen was associated with higher ALP in some analyses, particularly in infants born at 28+0 to 31+6 weeks’ gestation. These findings are limited to the first month after birth.
Efficacy and safety of oral propranolol combined with intralesional injection of lauromacrogol for infantile hemangioma: a systematic review and meta-analysis
Background: XXXXXXXXXXXXXX. This study aimed to identify the efficacy and safety of oral propranolol (PRO) combined with intralesional injection of lauromacrogol (LAU) for infantile hemangioma (IH) by comparison with PRO or LAU monotherapy. Methods: PubMed, Web of Science, Chinese National Knowledge Infrastructure and VIP databases were searched up to April 23, 2026. Endpoints of efficacy included the cure rate (reduction in size >75%), significant rate (reduction in size >50%), effective rate (reduction in size >25%) and treatment time. The incidence of adverse reactions was investigated to evaluate the safety. Results: Eight available studies were included, with four randomized controlled trials (RCTS) and four retrospective studies. A total of 668 patients were enrolled, among whom 318 patients were in the PRO + LAU group. By comparison with patients in the PRO group, patients in the PRO + LAU group revealed higher cure rate [odds ratio (OR) =3.22, 95% confidence interval (CI): 1.40–7.41, P=0.006], significant effective rate (OR =3.61, 95% CI: 1.94–6.74, P<0.001), shorter treatment time [mean difference (MD) =−72.81 days, 95% CI: −106.55 to −39.08, P<0.001] and lower incidence of adverse reaction (OR =0.55, 95% CI: 0.33–0.94, P=0.03). Furthermore, the cure rate of patients in the PRO + LAU group was significantly higher than that of patients in the LAU group (OR =3.69, 95% CI: 1.05–12.93, P=0.04) and the incidence of adverse reaction did not differ significantly between the two groups (P=0.40). Conclusions: Oral PRO combined with intralesional injection of LAU appears to improve certain efficacy outcomes and may shorten treatment duration compared with monotherapy, with no clear increase in adverse reactions. However, the overall certainty of evidence is limited.
FOS regulation of T-cell activation and the mechanism of inflammatory injury of coronary endothelium in Kawasaki disease
Background: Kawasaki disease (KD) is a pediatric systemic vasculitis often causing coronary lesions driven by aberrant T-cell activation. While FOS modulates T cells, its specific function in KD remains undefined. This study aims to investigate the role of FOS in T-cell activation and coronary endothelial inflammation in KD. Methods: The study integrated transcriptomic profiling of T cells from patients with KD and a murine model of Candida albicans cell wall extract (CAWS)-induced vasculitis to characterize FOS expression and vasculitis. Mechanistically, we employed lentiviral modulation of FOS in activated JURKAT cells co-cultured with human coronary artery endothelial cells (HCAECs) to delineate the impact of FOS on T-cell activation and endothelial inflammation. Results: Compared to controls, FOS expression was significantly upregulated in peripheral blood T cells of acute KD patients (P<0.001). FOS levels were also elevated in peripheral blood T cells and cardiac inflammatory regions of the KD model mice, and inhibition of FOS expression attenuated vasculitis. CD3/28 magnetic bead stimulation increased FOS expression in JURKAT cells, along with elevated levels of inflammatory cytokines interleukin-6 and tumor necrosis factor. Co-culture of activated JURKAT cells with HCAECs resulted in marked endothelial inflammation. Conversely, knocking down FOS in JURKAT cells prior to activation and co-culture mitigated endothelial inflammation. Conclusions: FOS contributes to the development and progression of coronary endothelial inflammation in KD by modulating T-cell activation. Targeting FOS may represent a potential therapeutic strategy for mitigating KD-associated coronary artery injury.
Prevalence of respiratory complications in long-term survivors of childhood and adolescent cancer: a systematic review and meta-analysis
Background: Improved survival after childhood and adolescent cancer has led to a growing population of long-term survivors, yet the burden of late respiratory complications remains poorly quantified. In this study, the prevalence of respiratory complications in long-term survivors was quantified, and associated risk factors were identified. Methods: A systematic review and meta-analysis were performed. PubMed, Embase, Web of Science, Cochrane Central Register of Controlled Trials (CENTRAL), China National Knowledge Infrastructure (CNKI), Wanfang, and VIP Chinese Science and Technology Journal Database were searched from inception to December 30, 2025, for studies reporting respiratory outcomes in 5-year survivors diagnosed with malignancy before age 21 years. The primary outcome was prevalence, defined as the proportion of survivors affected at the time of assessment. Two reviewers independently screened studies, extracted data, and assessed risk of bias using the Newcastle-Ottawa Scale (NOS). Pooled prevalence was estimated using random-effects models. Results: A total of 11 studies involving 128,509 survivors were included. Study quality was generally high (NOS scores 7–9). The pooled prevalence of any respiratory complication was 35% [95% confidence interval (CI): 30–40%], though limited by extreme heterogeneity (I2=99.0%). Prevalence estimates for specific complications were: reduced forced vital capacity (FVC), 19% (95% CI: 7–32%); reduced forced expiratory volume in one second (FEV1), 19% (95% CI: 2–35%); impaired diffusion capacity, 39% (95% CI: 9–70%); restrictive ventilatory dysfunction, 14% (95% CI: 6–22%); and symptomatic respiratory disease, 14% (95% CI: 10–19%). Higher prevalence was observed in survivors receiving high-risk chemotherapy, those diagnosed before 2000, and those with longer follow-up. Conclusions: Respiratory complications affect approximately one-third of long-term survivors. Higher prevalence was associated with greater treatment intensity and longer follow-up. Lower prevalence among more recently diagnosed survivors suggests evolving treatment strategies may be reducing long-term pulmonary toxicity.
Preliminary experience with oral thrombopoietin-receptor agonists, primarily hetrombopag, in pediatric immune thrombocytopenia: a pilot single-center retrospective report
Background: Although thrombopoietin receptor agonists (TPO-RAs) are effective second-line treatments for pediatric immune thrombocytopenia (ITP), real-world data on newer agents like hetrombopag remain scarce. This study aimed to describe a single-center retrospective experience with oral TPO-RAs, predominantly hetrombopag, in children with ITP who were refractory to or relapsed after first-line therapy. Methods: We conducted a single-center retrospective study of 18 children with ITP treated with oral TPO-RAs (hetrombopag, eltrombopag, or avatrombopag) at Qingdao Women and Children’s Hospital from January 2023 to June 2024. Platelet counts, bleeding grades, use of rescue therapy, and adverse events (AEs) were extracted from medical records and summarized descriptively; statistical analyses were performed. Results: Platelet counts increased after treatment initiation, with statistically significant differences from baseline at prespecified time points (week 1, month 1, month 3, and month 6; all Holm-adjusted P≤0.005). Bleeding grades (Buchanan & Adix overall grade 0–4) did not show a statistically significant improvement at the group level (P=0.10) or individual level (P=0.056). No statistically significant associations were detected between response and selected baseline factors (age, sex, disease duration, infection status, bleeding at baseline, or antibody status), although the study was underpowered for these analyses. Most recorded AEs were mild to moderate. Conclusions: In this preliminary, pilot-scale hetrombopag-predominant cohort, oral TPO-RA use was associated with increased platelet counts; however, the limited sample size and lack of statistically significant improvement in bleeding scores preclude definitive conclusions regarding the overall clinical utility of these agents beyond their ability to increase platelet counts. Given the retrospective design, heterogeneity of exposure, and limited sample size, the findings are hypothesis-generating and should be confirmed in prospective studies.
Salidroside alleviates TNF-α-induced endothelial inflammatory injury by modulating NF-κB/NLRP3 inflammasome-related signaling: an integrated network pharmacology and experimental study
Background: Kawasaki disease (KD) is an acute febrile vasculitis in children, and vascular endothelial injury is a central event in the development of coronary artery lesions. Salidroside (SAL), a natural active compound extracted from Rhodiola rosea, has anti-inflammatory, anti-apoptotic, and immunomodulatory properties. However, its mechanism in KD-related vascular inflammatory injury remains unclear. This study aimed to investigate the protective effects and potential mechanisms of SAL in endothelial injury relevant to KD. Methods: Potential targets of SAL were predicted using reverse virtual screening and SwissTargetPrediction. KD-related targets were collected from the GeneCards and DisGeNET databases. Overlapping targets were subjected to Gene Ontology (GO), Kyoto Encyclopedia of Genes and Genomes (KEGG), and protein-protein interaction analyses. Key targets were further evaluated by molecular docking. Human coronary artery endothelial cells (HCAECs) were stimulated with tumor necrosis factor-α (TNF-α) to establish an endothelial inflammatory injury model. RNA-sequencing (RNA-seq) was performed to identify differentially expressed genes after SAL treatment. The protective effects of SAL were then assessed by Cell Counting Kit-8 assay, wound-healing assay, Annexin V-fluorescein isothiocyanate (FITC)/propidium iodide (PI) flow cytometry, reverse transcription quantitative polymerase chain reaction, and western blotting. Results: Network pharmacology identified 226 potential SAL targets and 2,686 KD-related targets, with 56 overlapping targets. KEGG enrichment and RNA-seq indicated that SAL may regulate a broader inflammatory network, particularly interleukin (IL)-17 and TNF signaling pathways. Molecular docking showed stable binding of SAL to matrix metalloproteinase-9 (MMP-9). In TNF-α-stimulated HCAECs, SAL significantly improved cell viability, inhibited excessive migration, and reduced inflammatory cell death. SAL also decreased the messenger RNA (mRNA) expression of IL-1β, IL-6, MMP-9, interleukin-17 receptor A (IL-17RA), intercellular adhesion molecule-1 (ICAM-1), and NOD-like receptor family pyrin domain containing 3 (NLRP3), and reduced the protein levels of IL-6, phosphorylated nuclear factor kappa B (NF-κB) p65, NLRP3, gasdermin D (GSDMD), and Caspase-1 (P<0.05). Conclusions: SAL alleviates TNF-α-induced endothelial inflammatory injury in HCAECs. Its protective effects are associated with suppression of inflammatory signaling, inhibition of abnormal migration and cell death, and attenuation of NF-κB/NLRP3 inflammasome-related molecules. These findings provide preliminary experimental evidence for further investigation of SAL in KD-related vascular inflammation.
Precision myeloablation with TDM-guided busulfan in pediatric primary immunodeficiencies: a real-world study of 28 patients
Background: Optimizing busulfan (Bu) exposure is critical for balancing engraftment and toxicity in pediatric patients with primary immunodeficiencies (PID) undergoing hematopoietic stem cell transplantation (HSCT). This study evaluates the outcomes of a therapeutic drug monitoring (TDM)-guided, personalized Bu/fludarabine (Flu) regimen. Methods: We retrospectively analyzed 28 pediatric PID patients [11 severe combined immunodeficiency (SCID) and 17 with non-SCID] who underwent first allogeneic HSCT (allo-HSCT) between March 2022 and September 2025. All patients received Flu (150–180 mg/m2) and TDM-guided Bu with target cumulative area under the curve (AUC) of 60–70 mg·h/L for SCID and 85–95 mg·h/L for non-SCID. Primary endpoints were overall survival (OS) and event-free survival (EFS). Secondary endpoints included engraftment, regimen-related toxicity (RRT), graft-versus-host disease (GVHD), and immune reconstitution. Results: Only 11/28 patients (39.3%) achieved target AUC after the first Bu dose, confirming the necessity of TDM. With a median follow-up of 829.5 days [interquartile range (IQR), 376–1,046 days], 2-year OS and EFS were 96.42%. Sustained engraftment with complete or high-level donor chimerism was achieved in 96.42% of patients. Severe RRT was minimal: grade 3–4 mucositis (n=1, 3.5%) and definite veno-occlusive disease (VOD) (n=1). No grade 3–4 acute GVHD occurred. Chronic GVHD (cGVHD) occurred in 7/27 patients (25.9%), predominantly pulmonary-limited. Cytomegalovirus (CMV) reactivation occurred in 11 patients (39.2%) without CMV disease. All surviving patients discontinued immunoglobulin replacement within one year. Conclusions: A risk-adapted, TDM-guided Bu/Flu conditioning regimen achieves an optimal balance between efficacy and toxicity in pediatric PID HSCT. With a 2-year OS of 96.4%, minimal severe RRT, and universal intravenous immunoglobulin (IVIG) independence by 12 months, this precision myeloablative approach should be considered the preferred standard for this vulnerable population.
Efficacy and influencing factors of recombinant human growth hormone therapy in children with Turner syndrome: a single-center retrospective cohort study
Background: Real-world evidence regarding the optimal initiation timing and safety of recombinant human growth hormone (rhGH) in children with Turner syndrome (TS) remains limited. This study aimed to evaluate the efficacy of rhGH therapy in children with TS and to identify clinically relevant factors influencing treatment outcomes, thereby providing evidence to inform individualized therapeutic strategies. Methods: This retrospective cohort study included 50 pediatric patients with TS (3–14 years) who received rhGH therapy for ≥6 months. Eligibility criteria included a confirmed TS karyotype and height <−2 standard deviations (SDs). Baseline characteristics, laboratory parameters, and karyotypes were recorded. Patients were followed up every 6 months for up to 36 months, with height-related indicators [height standard deviation score (HtSDS), change in HtSDS (ΔHtSDS), growth velocity (GV)] and safety parameters [glycated hemoglobin (HbA1c), homeostatic model assessment for insulin resistance (HOMA-IR)] monitored. We used a generalized estimating equation (GEE) model to identify factors associated with rhGH treatment efficacy and then examined the interaction between follow-up duration and age at treatment initiation. Results: Baseline characteristics showed a median age of 7 [5–11] years and a mean HtSDS of −3.18±0.93. After 36 months of treatment, the mean height increased from 114.50±14.90 cm at baseline to 132.82±11.77 cm, whereas HtSDS improved to −1.80±0.99 (mean ΔHtSDS 1.20±0.67). Safety evaluation in a stable 12-month cohort (n=33) showed stable HbA1c levels (P=0.87) and a physiological increase in HOMA-IR (P=0.049) within the normal clinical range. GEE analysis revealed that treatment duration was positively correlated with HtSDS and ΔHtSDS, but negatively correlated with GV (all P<0.05). Baseline HtSDS was positively associated with follow-up HtSDS (β=0.797, P<0.001). Interaction analysis indicated a significant interaction between initiation age and follow-up duration: patients who started therapy earlier exhibited more rapid increases in HtSDS and ΔHtSDS during the early phase (6–12 months), whereas those with later initiation showed a greater decline in GV at 36 months. Conclusions: rhGH therapy appears to be effective and safe for enhancing linear growth in children with TS. The therapeutic response is time-dependent and appears to be optimized by earlier initiation and higher baseline height.
Clinical features and initial treatment outcomes of pediatric B-cell acute lymphoblastic leukemia with P2RY8::CRLF2 fusion positivity: a case series
Background: P2RY8::CRLF2 fusion is a recurrent CRLF2 (cytokine receptor-like factor 2) rearrangement in pediatric B-cell acute lymphoblastic leukemia (B-ALL) and is frequently associated with Philadelphia chromosome-like features and kinase pathway activation. However, clinical data on Chinese children with this lesion remain limited, and its early response under contemporary risk-adapted chemotherapy is not fully defined. We report a single-center case series to describe the clinical, molecular, and early treatment characteristics of this rare subgroup. Case Description: Seven consecutive children with newly diagnosed P2RY8::CRLF2 fusion-positive B-ALL treated between September 2017 and July 2024 were retrospectively reviewed. The median age at diagnosis was 3 years and 11 months, and the median initial white blood cell (WBC) count was 25.65×109/L. All patients had precursor B-cell immunophenotype. Concurrent genetic abnormalities included RAS pathway mutations in three patients, PAX5 fusions in two, and a JAK mutation in one. Two patients were treated according to the CCCG-ALL-2015 protocol and five according to the CCCG-ALL-2020 protocol. One patient received ruxolitinib during induction, and another patient with poor early response received ruxolitinib plus blinatumomab. Five patients achieved minimal residual disease (MRD) negativity on day 19, and six achieved MRD <0.01% with disappearance of the fusion transcript by day 46; the remaining patient became MRD-negative after salvage immunotherapy. After a median follow-up of 32 months, all patients remained in continuous complete remission (CR), although three were still receiving therapy. Conclusions: Pediatric P2RY8::CRLF2 fusion-positive B-ALL may show molecular heterogeneity, including recurrent RAS pathway lesions. Favorable early responses were observed under contemporary intensified chemotherapy with individualized targeted or immunotherapeutic interventions. Because of the small sample size, single-center design, and limited follow-up, these findings should be interpreted cautiously and require validation in multicenter cohorts.
Direct internal jugular vein incision for salvage of ‘stuck’ port catheters in pediatric leukemia: a single-center experience
Background: Totally implantable venous access ports (TIVAPs) are essential for long-term venous access in pediatric acute lymphoblastic leukemia (ALL). However, catheter-related calcified fibroblastic sheaths often make port removal difficult, and conventional traction may fail. Evidence regarding safe salvage surgery for calcified sheath-related stuck ports in children remains limited. This study aimed to investigate the etiology of difficult port removal and to evaluate the safety and feasibility of direct internal jugular vein (IJV) incision for fibroblastic sheath dissection and port extraction in pediatric patients with ALL. Case Description: This is a retrospective analysis of five pediatric ALL patients with difficult port removals due to calcified fibroblastic sheaths between January 2022 and December 2024. All patients had indwelling 6 Fr TIVAPs for 25–44 months and failed conservative traction attempts after 3–4 weeks of therapeutic low-molecular-weight heparin (LMWH). All underwent direct internal jugular vein (IJV) incision for fibroblastic sheath dissection and port extraction. All procedures were completed with complete removal of the sheath and catheter, and no perioperative complications occurred. Operative time was 35–60 minutes (mean 45.5±8.7 minutes), blood loss was 3–10 mL (mean 6.2±2.1 mL), and hospital stay was 2–3 days (mean 2.3±0.5 days). At 1–3 months of follow-up, all patients had patent IJV flow without sheath recurrence, full neck range of motion, no pain or neurologic deficits, and excellent cosmetic outcomes. Conclusions: Calcified adherent fibroblastic sheaths are the primary cause of difficult port removal in pediatric ALL. Direct IJV incision for fibroblastic sheath dissection appears to be a feasible and effective last-resort salvage procedure, offering a definitive solution for such complex cases.
Treatment strategies and prognostic outcomes in craniosynostosis with concurrent hydrocephalus: a case series
Background: Craniosynostosis complicated with hydrocephalus is a rare pediatric condition with complex and debated treatment protocols. The optimal sequence and combination of surgical interventions remain controversial, particularly in cases where both conditions coexist. Understanding effective management strategies is crucial to improving outcomes in these patients. Case Description: We analyzed nine pediatric patients treated at our institution for combined craniosynostosis and hydrocephalus. The cohort included seven males and two females, aged 8 to 45 months. Seven patients had syndromic craniosynostosis, while two had non-syndromic forms. All were admitted due to abnormal facial features. At admission, six had normal head circumference, two had reduced, and one had increased head circumference. Elevated intracranial pressure was observed in eight patients. All underwent cranioplasty, with cerebrospinal fluid leakage occurring in two cases. Five patients required ventriculoperitoneal (VP) shunt placement due to persistent hydrocephalus after surgery. Follow-up, averaging 53.6 months, showed improved cranial shape and symptom relief in eight patients, and resolution or significant mitigation of hydrocephalus in all cases. Conclusions: Craniosynostosis frequently coexists with hydrocephalus, particularly in syndromic cases with normal head circumference. Based on our institutional experience, initial cranioplasty was commonly performed to address cranial deformities and was associated with improvement in some patients. However, persistent hydrocephalus in certain cases required additional intervention, such as VP shunting.
Gut and respiratory microbiomes in asthma and allergic diseases: a narrative review of mechanistic insights, gut-lung axis interactions and therapeutic opportunities
Background and Objective: Asthma and allergic diseases are increasingly prevalent chronic inflammatory disorders characterized by immune dysregulation, epithelial barrier impairment, and marked clinical heterogeneity. Increasing evidence suggests that both the gut microbiome and the respiratory microbiome are associated with disease initiation, phenotype expression, and exacerbation risk. This narrative review aims to synthesize current evidence on microbiome alterations associated with asthma and allergic diseases, with particular emphasis on mechanistic pathways, bidirectional gut-lung axis interactions, and microbiome-targeted therapeutic opportunities. Methods: We conducted a narrative review of recent English-language literature on the gut microbiome, respiratory microbiome, asthma, allergic diseases, microbial metabolites, and microbiome-based interventions. Relevant studies and reviews were identified through literature screening and were selected for their relevance to early-life microbial colonization, disease-associated dysbiosis, immune regulation, gut-lung axis biology, and translational strategies. Key Content and Findings: Current evidence indicates that early-life gut microbial colonization, airway microbial dysbiosis, and altered metabolite production are associated with allergic susceptibility, inflammatory phenotype, exacerbation risk, and disease progression. The strength of evidence differs across domains: human cohort and clinical studies most strongly support associations between early-life microbial patterns, airway dysbiosis, and disease phenotypes, whereas many mechanistic pathways remain supported primarily by preclinical or experimental data. Key mechanisms include mucosal microbiome-immune crosstalk, local airway epithelial-microbial interactions, short-chain fatty acid-mediated immune regulation, tryptophan and bile acid signaling, epithelial barrier dysfunction, viral-microbiome interactions, and epigenetic modulation. The gut-lung axis provides a bidirectional framework linking intestinal and airway microbial ecosystems through immune, metabolic, inflammatory, infectious, and treatment-related pathways. Emerging interventions show different levels of evidence and should not be interpreted as equally mature therapeutic strategies. Conclusions: The gut and respiratory microbiomes are important components of the pathogenic network underlying asthma and allergic diseases and may represent future targets for prevention and therapy. However, many reported microbial signatures remain associative, and stronger standardization, longitudinal validation, functional studies, and evidence-stratified clinical trials are needed before microbiome-informed precision medicine can be broadly implemented in routine care.
Artificial intelligence in congenital heart surgery: a scoping review and primer for surgeons
Background: Congenital heart surgery (CHS) encompasses a wide spectrum of complex cardiac defects, many of which demand specialised perioperative management and tailored surgical planning. Artificial intelligence (AI), including machine learning (ML), is gaining prominence as a tool to optimise clinical decision-making and achieve better outcomes. This scoping review aims to map and summarise the existing applications of AI modalities in CHS. Methods: A comprehensive search of MEDLINE, Embase, and Web of Science was performed, combining terms for AI with terms for congenital heart disease and surgery. Results: A total of 2,871 articles were retrieved from the search, of which 93 studies were included. The majority of studies focused on outcome prediction and imaging-based applications. Smaller proportions addressed decision-making and data augmentation, omics integration, benchmarking and quality improvement. The majority of studies examined heterogeneous congenital heart disease (CHD) populations, with tetralogy of Fallot (TOF) and single ventricle physiology most frequently represented. Conclusions: AI applications in CHS are rapidly expanding across diverse domains, with early studies showing encouraging potential to support diagnostics, guide surgical decision-making, and improve perioperative outcomes. However, most models remain in the preliminary stage with limited external validation. Emerging advances in AI may further accelerate progress, but careful evaluation and integration are essential to translate this promise into tangible clinical benefits.
Regenerative medicine approaches for children with cerebral palsy: a systematic review of clinical safety and effectiveness
Background: Cerebral palsy (CP) is a leading cause of childhood disability, and current interventions limited to symptomatic management. Regenerative medicine has been proposed as a promising to enhance neuroplasticity and improve outcomes. This systematic review aimed to synthesize clinical evidence regarding the effectiveness and safety of regenerative medicine interventions in children with CP. Methods: A systematic search of PubMed, Web of Science, Cochrane Library, and ScienceDirect [2010–2025] identified studies evaluating regenerative therapies in pediatric CP. Eligible studies included randomized controlled trials (RCTs), interventional studies, cohort studies, and case series ≥3 participants. Risk of bias was assessed using RoB-2 and ROBINS-I. Due to heterogeneity, findings were synthesized narratively. Results: Eighteen studies including 1,087 children were analyzed. Most interventions involved umbilical cord (UC)-derived products (73%), followed by bone marrow-derived cellular therapies and granulocyte colony-stimulating factor-based protocols. Cord-derived therapies demonstrated moderate improvements in motor outcomes in selected trials, combination therapies (cord blood with erythropoietin) demonstrated the greatest motor and cognitive gains, while bone marrow approaches showed more variable effects. Adverse events were primarily mild and transient; however, long-term safety data were limited. Conclusions: Regenerative medicine represents a promising adjunctive strategy for treating CP in children, particularly with regard to products derived from UC blood. However, the variability in study design, small sample sizes, and short follow-up periods limit the possibility of drawing definitive conclusions.
Peri-ictal magnetic resonance imaging findings in pediatric seizures: a scoping review on pearls and pitfalls from a heterogeneous population
Background: Peri-ictal magnetic resonance imaging (MRI) brain abnormalities are increasingly recognized, yet their temporal evolution and imaging features in children remain incompletely characterized. This scoping review aims to map and synthesize two decades of evidence on MRI-based seizure-related changes in pediatric patients, providing an overview of the existing literature, reported imaging findings, diagnostic challenges, and knowledge gaps. Methods: PubMed and MEDLINE databases were searched (January 2000 to September 2025) for studies evaluating MRI changes in focal seizures (FS), prolonged febrile seizures (PFS), or status epilepticus (SE) in children aged 0-18 years. Studies reporting diffusion weighted images/apparent diffusion coefficient maps (DWI/ADC), T2/fluid attenuated inversion recovery (FLAIR), arterial spin labelling (ASL), functional MRI (fMRI), or diffusion tensor imaging (DTI) changes were considered. Results: Twenty-seven studies were included, most focusing on children with PFS and SE, whereas focal seizures were underreported, particularly extratemporal ones. Acute MRI findings (<1-week) most commonly showed hippocampal or limbic swelling with T2/FLAIR hyperintensity and diffusion restriction, consistent with edema, particularly following prolonged seizures. ASL reported cases of early focal hyperperfusion concordant with the seizure focus, often persisting longer than diffusion abnormalities. Subacute changes (days 4–10) included evolving edema and post-ictal hypoperfusion, while chronic findings included hippocampal atrophy, mesial temporal sclerosis (MTS), and persistent white matter abnormalities on DTI. Thalamic, basal ganglia, and subcortical white matter involvement have been hypothesized to reflect excitotoxic or inflammatory propagation beyond the seizure onset zone. Early DWI/ADC changes correlated with seizure duration, and when involving the hippocampus predicted MTS and long-term cognitive sequelae in some studies. Conclusions: Peri-ictal MRI abnormalities in children are most frequently described in PFS and SE, where recurring patterns of acute edema and later structural changes have been reported. However, existing evidence is heterogeneous and weighted toward early-life and acute symptomatic populations, limiting generalizability. Associations between early imaging changes and longer-term outcomes remain inconsistent. Advanced imaging techniques, largely underreported, may provide additional insights into seizure-related network alterations, although their prognostic relevance remains uncertain. Overall, larger, seizure-type specific longitudinal studies are needed before definitive conclusions can be made.
Current applications and future perspectives of family-integrated care in the pediatric intensive care unit: a narrative review from concept to practice
Background and Objective: Family-integrated care (FIC), grounded in the principles of family-centered care (FCC), has become an important approach for supporting children and families during pediatric intensive care. Although post-intensive care syndrome in pediatrics (PICS-p) provides one important rationale for family engagement, the scope of FIC extends beyond PICS-p to include bedside participation, communication, shared decision-making, rehabilitation, psychosocial support, and transition planning. This narrative review aims to synthesize current applications, reported benefits, implementation barriers, and future priorities for FIC in the pediatric intensive care unit (PICU). Methods: We conducted a narrative review of literature on FIC/FCC in pediatric critical care. Searches were performed in PubMed/MEDLINE, CINAHL, Embase, Web of Science, and the Cochrane Library, with emphasis on contemporary literature from 2015 to April 2026 and inclusion of selected foundational papers when conceptually necessary. Eligible evidence included observational, qualitative, mixed-methods, quality-improvement, pre-post, interventional, guideline, and review literature relevant to family participation in PICU care. Key Content and Findings: The review identifies several recurring themes: the transition from restricted visitation to partnership-based care; practical family participation in routine care, communication, shared decision-making, rehabilitation, and end-of-life contexts; patient- and family-centered outcomes; structural, cultural, linguistic, and resource-related barriers; and the role of interdisciplinary teams, institutional policy, staff education, and digital tools in supporting implementation. Conclusions: Current evidence suggests that FIC may improve family experience, communication, parental confidence, and selected psychosocial outcomes, while its effects on long-term child outcomes and PICS-p require further pediatric-specific evaluation. Future work should prioritize clearer definitions, standardized implementation models, inclusive study designs, and pragmatic outcome measures that reflect both child and family priorities.
Entertainment interventions for pediatric fracture patients during cast room procedures: a scoping review
Background: Children undergoing cast room procedures are usually awake and often experience anxiety, fear, and behavioral resistance, which may hinder the smooth completion of the procedure. Entertainment interventions, as distraction strategies, have been increasingly applied in clinical practice. However, the types of interventions, implementation methods, outcome indicators, assessment tools, and intervention effects remain unclear, limiting their clinical application. Therefore, this study aimed to conduct a scoping review of entertainment interventions used during cast room procedures for pediatric fracture patients to inform clinical practice and future research. Methods: PubMed, Web of Science, Embase, and Cochrane Library databases were searched from inception to October 1, 2025. Studies involving entertainment interventions for children (aged ≤18 years) with fractures during cast room procedures were screened according to predefined inclusion and exclusion criteria. The included literature was systematically organized and narratively analyzed. Results: A total of 16 studies were included, covering 8 types of entertainment interventions [music, iPad-game, iPad-video, instructional video, virtual reality (VR)-based distraction intervention, VR-game, VR-video, and therapeutic play]. Anxiety and pain were the most commonly reported outcome indicators, followed by fear and behavioral responses, while heart rate was the most frequently used physiological indicator. Standard assessment tools included: heart rate response and the Visual Analogue Scale (VAS) for anxiety; the Numerical Rating Scale (NRS) for pain; the Child Fear Scale (CFS) for fear; and self-created behavior scales for behavioral changes. The results indicated that music, VR-game, iPad-video, VR-video, VR-based distraction intervention and therapeutic play demonstrated positive effects in relieving anxiety; VR-game and instructional video may reduce pain; music and iPad-video showed positive effects in reducing heart rate; VR-game and VR-video reduced fear; and therapeutic play enhanced behavioral performance. Conclusions: Entertainment interventions vary in implementation approaches and effectiveness. Except for iPad-game interventions, other interventions generally demonstrated positive effects on children’s procedural experience in the cast room. Clinical staff may select the most appropriate intervention based on department resources and children’s specific responses, such as anxiety, pain, and behavioral resistance. Future studies should develop or optimize new entertainment strategies, expand the range of interventions, and explore individualized strategies for children of different ages, genders, and fracture types to further improve treatment experiences.
Maternal-fetal transmission of Zika virus and long-term neurodevelopmental outcomes in children: a narrative review
Background and Objective: The Zika virus (ZIKV), a flavivirus transmitted through mosquito bites, presents significant global health challenges due to its potential to cause severe congenital malformations, particularly neurological disorders. It is imperative to comprehend the transmission pathways and the long- term impact of ZIKV on children’s neurodevelopment. This narrative review aims to summarize current evidence on maternal-fetal transmission of ZIKV and its neurodevelopmental consequences in offspring. Methods: A narrative review with systematic elements was conducted using PubMed, Scopus, and Web of Science [2015–2024]. Relevant studies on ZIKV vertical transmission and neurodevelopmental outcomes were screened, with independent data extraction and quality assessment performed by multiple reviewers. Key Content and Findings: Twenty-one studies with diverse designs met the inclusion criteria. The research suggests that human decidual cells facilitate the transmission of ZIKV, a virus specific to the fetus, primarily infecting the fetus during the first trimester via the placenta. Congenital Zika syndrome (CZS) was prevalent, characterized by ocular and joint abnormalities, cerebral atrophy, and microcephaly. Children born without microcephaly also exhibited impaired cognitive, motor, and linguistic development later in life. Other neurological manifestations included Guillain-Barré syndrome, encephalitis, and seizures. Conclusions: Maternal-fetal transmission of ZIKV, particularly during the early stages of pregnancy, results in various long-term neurodevelopmental abnormalities in children. Enhancing outcomes necessitates multimodal therapies, early detection, and routine neurodevelopmental follow-up. Further research is essential to elucidate the underlying mechanisms and improve prevention strategies.
Current situation, challenges and future trends of pediatric palliative care from a multi-dimensional perspective: a narrative review
Background and Objective: Pediatric palliative care (PPC) provides holistic support for children and adolescents with life-threatening or life-limiting conditions and their families. This narrative review summarizes PPC concepts, current service models, key implementation challenges, evidence in major practice areas, and future directions, with attention to global differences and the Chinese context. Methods: A narrative review was conducted using a targeted PubMed search and supplementary checking of key consensus documents, guidelines, reviews, and organizational reports published from 2010 to 2025. Findings were synthesized thematically rather than quantitatively. Key Content and Findings: The review is organized across four domains: foundational concepts and target populations; global service models and regional differences; major challenges involving culture, access, workforce, policy, financing, and ethics; and key practice areas including symptom management, psychosocial-spiritual support, family-centered decision-making, early integration, digital health, and capacity building. PPC has developed rapidly in high-income settings, but access remains uneven, and low-resource regions face persistent barriers related to workforce, essential medicines, reimbursement, and cultural acceptance. Conclusions: PPC should be integrated early into serious pediatric illness care while remaining adaptable to local health-system capacity, culture, and family needs. Future progress requires clearer definitions, stronger methods, tiered service models, professional training, equitable access to essential medicines, context-sensitive terminology, and implementation research in underrepresented regions.
Best evidence summary for the prevention of intraventricular hemorrhage in preterm infants: a systematic review
Background and Objective: With continuous advances in neonatal intensive care, the survival of preterm infants has improved substantially. However, intraventricular hemorrhage (IVH) remains one of the most serious complications of prematurity and is associated with death and long-term neurodevelopmental impairment. A growing body of evidence suggests that timely preventive interventions can reduce the incidence and severity of IVH. This study aimed to systematically retrieve, appraise, and synthesize the best available evidence on IVH prevention in preterm infants, so as to provide practical references for perinatal and neonatal intensive care. Methods: Based on the “6S” evidence pyramid model, we searched multiple databases and resources. Evidence was searched in clinical decision support resources, guideline repositories, evidence-based databases, and major English and Chinese literature databases, including British Medical Journal (BMJ), UpToDate, National Institute for Health and Care Excellence (NICE), Guidelines International Network (GIN), Medlive, American Heart Association (AHA), American Academy of Pediatrics (AAP), Joanna Briggs Institute databases (JBI), Cochrane Library, Cumulative Index to Nursing and Allied Health Literature (CINAHL), PubMed, Web of Science, SinoMed, Wanfang Data, and China National Knowledge Infrastructure (CNKI). The search timeframe was from January 2015 to December 2025. Eligible publications included clinical decisions, guidelines, systematic reviews, evidence summaries, expert consensus documents, best practice implementation projects, and randomized controlled trials (RCTs). We extracted evidence from the included literature, summarized and graded it into evidence summaries. Key Content and Findings: A total of 20 publications were included, comprising 2 clinical decision resources, 4 guidelines, 6 systematic reviews, 2 evidence summaries, 2 expert consensus documents, 1 best practice implementation report, and 3 RCTs. A total of 46 pieces of best evidence were synthesized and categorized into ten domains: prevention principles, risk factor identification, antenatal preparation, immediate intrapartum and postnatal management, thermoregulation, oxygen therapy support, blood pressure monitoring and assessment, glycemic management, care bundles, and training and education. Conclusions: This study synthesizes 46 pieces of best evidence for preventing IVH in preterm infants, providing a robust foundation for clinical practice. However, clinical translation may encounter barriers related to local healthcare policies, equipment constraints, and human resources. When implementing these recommendations, practitioners must carefully evaluate the source context, evidence grading, and institutional adaptability to ensure effective evidence-based practice.
Risk factors and predictive models for perioperative acute kidney injury in children: a narrative review
Background and Objective: Perioperative acute kidney injury (AKI) is a serious complication in children, with an incidence of 5–30% and up to 40% in neonates after cardiac surgery. It increases mortality and the risk of chronic kidney disease. This narrative review synthesizes current evidence on risk factors and predictive models for perioperative AKI in children, aiming to inform early risk stratification and preventive care. Methods: A literature search was conducted up to March 2024 using PubMed/MEDLINE, Embase, Web of Science, and Cochrane Library. The search combined terms related to AKI, pediatrics, the perioperative period, risk factors, and prediction models. Studies focusing on pediatric patients (≤18 years) were included. Key Content and Findings: Key risk factors include young age, congenital heart disease, exposure to nephrotoxic medications, and major surgeries like those using cardiopulmonary bypass. The review evaluates predictive models, from traditional statistical methods to machine learning models that incorporate novel biomarkers such as neutrophil gelatinase-associated lipocalin and kidney injury molecule-1 for earlier detection. Promising biomarkers like urinary L-FABP and TIMP-2×IGFBP7 are also highlighted. Integrating these tools into clinical workflows can guide proactive management. Conclusions: Early identification of high-risk children is crucial. While predictive modeling is advancing, a gap remains in models specifically validated for pediatric populations. Future research should focus on multicenter studies to refine pediatric-specific models, validate novel biomarkers, and develop personalized approaches. Implementing evidence-based, risk-stratified care has the potential to significantly improve outcomes and long-term renal health for children undergoing surgery.
Advances in behavioral vision training for the treatment of childhood amblyopia: a narrative review
Background and Objective: Childhood amblyopia is a common neurodevelopmental disorder that impairs visual function and binocular integration. Traditional treatments, such as occlusion and pharmacological penalization, are often limited by poor adherence, incomplete restoration of binocular function, and variable response across patients. Behavioral vision training, grounded in principles of neural plasticity, has emerged as a promising alternative. This narrative review synthesizes the literature on behavioral vision training for pediatric amblyopia, focusing on its theoretical basis, main technical modalities, effects on multidimensional visual outcomes, comparison with traditional therapy, combination strategies, adherence, and influencing factors. Methods: The PubMed and Web of Science databases were searched for literature published from January 1, 2015 to December 31, 2025. Search terms included combinations related to children, amblyopia, behavioral vision training, perceptual learning, dichoptic training, digital therapeutics, and virtual reality (VR). Studies were selected based on predefined inclusion and exclusion criteria, with data extraction and quality assessment performed independently by two reviewers. The heterogeneity of intervention protocols and outcome measures precluded quantitative synthesis, making a narrative review the appropriate approach. Key Content and Findings: A total of 100 original studies were included, comprising randomized controlled trials (RCTs), prospective and retrospective cohort studies, and single-arm interventional trials. The evidence base remains moderate in quality, with substantial heterogeneity in training protocols, outcome measures, and follow-up durations. Behavioral vision training encompasses monocular perceptual learning, dichoptic training, gamified digital therapeutics, and VR-based interventions. These approaches have demonstrated varying degrees of efficacy in improving best-corrected visual acuity (BCVA), stereopsis, contrast sensitivity (CS), and other visual functions. Direct comparisons with traditional occlusion therapy yield mixed results: some well-designed noninferiority trials show comparable or superior outcomes under specific conditions, while others report no advantage. Combination strategies integrating behavioral training with occlusion may provide synergistic benefits, particularly for children with suboptimal response to monotherapy. Adherence rates vary considerably (range 56–98%) and are influenced by training design, supervision, and delivery platform. Age, amblyopia subtype, and training dose appear to be important modifiers of treatment response. Key challenges include the lack of standardized protocols, insufficient long-term follow-up data, heterogeneity in study designs, and emerging safety considerations regarding ocular biometric changes. Conclusions: Behavioral vision training represents a promising and evolving therapeutic approach for childhood amblyopia, offering the potential to address the limitations of traditional occlusion therapy through active, engaging, and mechanism-targeted interventions. The evidence supports its effectiveness in improving both monocular and binocular outcomes, but the field requires high-quality, long-term RCTs to establish standardized protocols, optimize patient selection, and clarify long-term safety profiles.
Perioperative management of emergency non-cardiac surgery in a pediatric patient with Eisenmenger syndrome: a case report
Background: Eisenmenger syndrome (ES) is a late-stage complication of untreated congenital heart disease. It is characterized by irreversible pulmonary arterial hypertension (PAH), right-to-left shunting, and cyanosis. Pediatrics with ES who undergo emergency non-cardiac surgery are at an extremely high perioperative risk. Potential complications include pulmonary hypertension crisis (PHC), right ventricular failure, and sudden cardiac death. There is limited evidence guiding perioperative management in this population. Case Description: An 11-year-old girl required emergency surgery under general anesthesia for right ovarian torsion. She had a history of congenital heart disease, which was complicated by severe pulmonary hypertension (pulmonary artery pressure: 105/49 mmHg) and ES. Anesthesia was inducted with etomidate, midazolam, vecuronium, and sufentanil, and maintained with propofol, remifentanil and sevoflurane. Intraoperatively, hemodynamic parameters remained stable. However, the patient developed a postoperative PHC, which was characterized by ventricular tachycardia, hypotension, and hypoxemia. Prompt treatment with amiodarone, treprostinil, sodium bicarbonate, and supplemental oxygen successfully restored sinus rhythm and hemodynamic stability. The patient was discharged on postoperative day 11. Conclusions: Emergency non-cardiac surgery poses an extremely high anesthetic risk for pediatrics with ES. It is critical to avoid factors that elevate pulmonary vascular resistance, ensure meticulous monitoring, and recognize and treat PHC early. This case underscores the importance of personalized anesthetic planning and multidisciplinary perioperative management for this vulnerable population.
Anesthetic management for double lung transplantation in an 8-year-old male with extreme malnutrition (BMI 11.4 kg/m2) following hematopoietic stem cell transplantation: a case report
Background: Severe malnutrition is a prevalent comorbidity in pediatric lung transplantation (LTx) and is historically considered as a relative contraindication due to high perioperative mortality. While current guidelines emphasize the hazards of a low body mass index (BMI), literature detailing specific anesthetic strategies for severely malnourished patients remains limited. This report delineates an anesthetic protocol designed to safely navigate a candidate with an extremely low BMI through surgical stress, demonstrating the clinical value of precision management over rigid anthropometric cutoffs. Case Description: An 8-year-old male with end-stage bronchiolitis obliterans syndrome (BOS) following hematopoietic stem cell transplantation (HSCT) presented for double LTx. The chief complaint was progressive chest tightness and dyspnea for 11 months, 3 years post HSCT. He exhibited profound somatic depletion with an extremely low BMI of 11.4 kg/m2. The anesthetic strategy aimed to mitigate the dual risks of altered pharmacokinetics and limited myocardial reserve. Bispectral index (BIS) monitoring was utilized for the precise anesthetic depth titration, preventing the relative overdose and drug accumulation common in sarcopenic patients. Intraoperatively, profound hypotension and bradycardia occurred during pulmonary artery (PA) and left atrial clamping. This critical hemodynamic intolerance was successfully managed via transesophageal echocardiography (TEE)-guided optimization and vasopressor support. The patient was extubated on postoperative day 2 and discharged from the intensive care unit (ICU) on day 6, demonstrating favorable long-term pulmonary function and catch-up growth. Conclusions: Successfully navigating pediatric LTx in cases of extreme malnutrition (BMI <16 kg/m2) does not imply that such profound frailty is universally reversible. Rather, it underscores the feasibility of a highly specialized, multidisciplinary management protocol. The integration of BIS-guided titration and TEE-directed hemodynamic support is essential to bridge physiologically vulnerable candidates to postoperative rehabilitation.
A female manifesting carrier of DMD with exon 45 deletion: a case report
Background: Duchenne muscular dystrophy (DMD) is a rare X-linked recessive hereditary disease that overwhelmingly affects males, while symptomatic female carriers are rarely encountered in clinical practice. Persistent elevation of liver enzymes is often misattributed to primary liver injury in young children, which may delay the recognition of underlying myogenic disorders like DMD. Case Description: We herein report the case of a 3-year-old female manifesting carrier of DMD. The patient initially presented with continuously raised liver transaminases and drastically elevated serum creatine kinase (CK). Further physical examination found delayed motor developmental milestones. Genetic testing confirmed an unreported novel deletion variant in exon 45 of the DMD gene. After all hepatic pathogenic factors were ruled out, the child was finally diagnosed with symptomatic DMD carrier status. Conclusions: For pediatric cases featuring unexplained hypertransaminasemia combined with high CK values, clinicians should stay highly alert to the possibility of DMD. Dynamic serial CK monitoring and full-scale genetic analysis should be arranged without delay to achieve early diagnosis and timely intervention, which can effectively optimize long-term prognosis for affected children.
Bilateral active chorioretinal lesions and fundus hemorrhage in a 23-month-old male with CYBB mutation: a case report
Background: Chronic granulomatous disease (CGD) is a rare primary immunodeficiency disease caused by genetic mutations. Clinical manifestations of CGD include recurrent infections, granuloma formation, and noninfectious inflammatory responses in multiple organs, while ocular involvement is uncommon. Chorioretinal lesions in cases of CGD are mostly inactive and tend not to affect vision, while active chorioretinal lesions and fundus hemorrhage are rare. Case Description: A 23-month-old male child with CYBB mutation was admitted to hospital due to fever and decreased visual acuity. He had poor eye contact with others and needed help walking. Physical examination indicated reduced pursuit to light in the left eye and no pursuit to light in the right eye. Syphilis, tuberculosis, cytomegalovirus, herpes simplex virus and mass lesions were excluded. Dilated ophthalmoscopy revealed bilateral chorioretinal lesions and extensive fundus hemorrhage in the right eye. Ultimately, the patient lacked light perception in the right eye and had poor light perception in the left eye. Conclusions: Chorioretinal lesions in CGD are mostly inactive. Although rare, active lesions with fundus hemorrhage can cause visual loss. Recurrent infections may be a trigger for chorioretinal lesions. When patients with CGD have decreased visual acuity, timely fundoscopic evaluation by an ophthalmologist is necessary.
Alström syndrome in a Chinese girl: a case report and literature review
Background: Alström syndrome (ALMS) is a rare autosomal recessive genetic disorder caused by homozygous or compound heterozygous mutations in the ALMS1 gene. The encoded ALMS1 protein is widely distributed, leading to the complex clinical manifestations of this disease. Currently, there are no effective curative treatments-disease-modifying or curative therapies. Methods: Here, we report a case of ALMS in a Chinese female patient featuring nystagmus, visual impairment, diabetes mellitus, hepatosplenomegaly, mixed hearing loss, ovarian pedicle torsion, and acanthosis nigricans. Comprehensive clinical data—including anthropometric, laboratory, and imaging findings—were collected. Whole-exome sequencing identified biallelic pathogenic variants in the ALMS1 gene, confirming the diagnosis. For glycemic control, the patient was managed with a combination of insulin aspart, insulin detemir, and metformin. Results: Whole-exome sequencing identified compound heterozygous mutations in the ALMS1 gene, leading to erroneous ALMS1 protein coding and confirming ALMS. The variant ALMS1 c.4839dupA was identified as a novel likely pathogenic mutation. Currently, there is no definitive treatment for this disease, and management primarily focuses on symptomatic support. Conclusions: We identified a novel likely pathogenic variant in the ALMS1 gene as the underlying cause of ALMS in this Chinese patient. The clinical presentation highlights the remarkable phenotypic variability of the disorder, a key factor contributing to frequent diagnostic delays or errors. Consequently, early genetic confirmation is imperative to guide appropriate and timely interventions that can significantly improve patient care and quality of life.
Reply to: A mono-organ POLG1-related mitochondrial DNA depletion syndrome must be confirmed by quantitative real-time polymerase chain reaction (PCR) or droplet digital PCR