Translational pediatrics最新文献

Background: Despite improving outcomes in pediatric acute lymphoblastic leukemia (ALL), pegaspargase (PEG-ASP) carries a risk of cerebral venous sinus thrombosis (CVST), for which optimal anticoagulation management in children remains undefined. This retrospective case series aimed to analyze the incidence, associated factors, and management outcomes of PEG-ASP-associated CVST in pediatric ALL patients. Among 741 ALL patients treated at a single center over a 3-year period, the incidence of PEG-ASP-associated CVST was 1.4% (11 cases).

Case description: Eleven pediatric ALL patients who developed PEG-ASP-associated CVST were stratified by disease severity [intensive care unit (ICU) admission vs. non-ICU] and anticoagulation duration (>3 vs. ≤3 months). ICU-admitted patients received significantly higher cumulative PEG-ASP doses (4,000 IU/m²) compared to ward-managed patients (2,250 IU/m²), and a strong positive correlation (r=0.75) was observed between cumulative PEG-ASP dose and disease severity. Extended anticoagulation (>3 months) was associated with improved laboratory parameters, including notably lower triglyceride and D-dimer levels at 8 weeks, along with increased antithrombin (AT)-III levels. All patients were managed conservatively with low-molecular-weight heparin without thrombectomy or discontinuation of PEG-ASP. Outcomes were excellent, with 90.9% survival, complete neurological recovery (modified Rankin Scale grade 0), and no cases of thrombosis recurrence or leukemia relapse.

Conclusions: The study highlights a significant association between cumulative PEG-ASP dose and CVST severity in this pediatric ALL cohort. The findings support the feasibility and safety of extended anticoagulation therapy alongside continued chemotherapy, without interrupting PEG-ASP treatment, leading to favorable clinical and neurological outcomes.

Background: Early postnatal hypothermia (EPH) remains a significant clinical concern in critically ill transported neonates. A previous regional study reported an alarming EPH incidence rate of 82.1%, with wide variation across delivery facilities, underscoring the urgent need for quality improvement (QI) in early body temperature management. This study aimed to reduce the percentage of EPH in critically ill transported newborns by 20% to 25% within 1 year.

Methods: A standardized, evidence-based thermal care bundle and comprehensive staff training were implemented using multiple Plan-Do-Study-Act (PDSA) cycles starting May 1, 2023. Neonates transported from six referral hospitals in the pre-QI phase (January 1, 2022-April 30, 2023) were compared with those in the intervention period (May 1-December 31, 2024). Clinical characteristics, EPH incidence, and early outcomes within 7 days were analyzed.

Results: A total of 1,247 neonates transferred from six referral hospitals were included, with 457 newborns in the pre-QI group and 790 in the post-QI group. Baseline characteristics were similar except for higher maternal intrapartum fever in the post-QI group (1.0% vs. 3.1%, P=0.02). Following implementation of the thermal management protocol, the incidence of EPH significantly decreased from 82.1% to 59.0% (adjusted P<0.001). A marked reduction in severe intraventricular hemorrhage (IVH) (grade ≥3) was observed, from 2.6% to 0.2% (adjusted P=0.002). No significant difference was observed in mortality, pulmonary hemorrhage, shock, hypoglycemia, disseminated intravascular coagulation (DIC), nor invasive mechanical ventilation use.

Conclusions: Implementing a standardized thermal management protocol substantially reduces EPH incidence in critically ill transported neonates. Avoiding EPH is beneficial for improving short-term outcomes.

Background: Pediatric cardiac magnetic resonance imaging (MRI) examinations often present significant challenges. artificial intelligence-assisted compressed sensing (ACS) technique is widely used to reduce scan time and improve image quality. Our purpose was to explore the value of the ACS technique for detecting coronary artery aneurysms (CAA) in children with Kawasaki disease (KD), as compared to the united compressed sensing (uCS) technique.

Methods: Sixty patients with KD complicated with suspected coronary artery disease (CAD) who underwent 3T cardiovascular magnetic resonance (CMR) were recruited. Contrast-enhanced magnetic resonance angiography (MRA) images were obtained by both ACS and uCS techniques. The two sequences' scan time, subjective and objective image quality, and diagnostic performance for detecting CAA were measured and compared.

Results: Sixty participants (39 males and 21 females; mean age ± standard deviation, 7±3 years) completed two sequences. The ACS technique exhibited a significantly shorter scan time compared to the uCS technique group (230.6±35.7 vs. 335.3±70.8 seconds, P<0.001). The subjective image quality scores, signal-to-noise ratios (SNRs), and contrast-to-noise ratios (CNRs) of the ACS technique were significantly higher than those of the uCS technique (all P<0.05). The sensitivity of the ACS technique coronary MRA was found to be 96% (53/55), the specificity was 80% (4/5), the positive predictive value (PPV) was 98% (53/54), the negative predictive value (NPV) was 67% (4/6) and the accuracy was 95% (57/60) on the basis of each person. The area under the receiver operating characteristic curve (AUC-ROC) of the ACS technique MRA images was larger than that of the uCS technique group on a per-patient basis (0.824 vs. 0.796, P<0.001), a per-vessel basis (0.894 vs. 0.823, P<0.001) and a per-segment basis (0.897 vs. 0.836, P<0.001).

Conclusions: The ACS technique shows excellent diagnostic performance at 3T imaging with shorter scan time and better image quality compared with the uCS technique.

Background: Human bocavirus 1 (HBoV1) is a common pediatric respiratory virus, yet, its potential to cause severe systemic illness as a sole pathogen and its specific effects on the urinary system are not fully recognized. This report describes the first case of pediatric sepsis, meeting the 2024 Phoenix sepsis criteria, caused by high-load HBoV1 viremia, which was characterized by a novel triphasic urinary course.

Case description: A previously healthy 4-year-old girl presented with fever and cough and developed asymptomatic sterile pyuria on illness day 4. On day 7, her condition deteriorated to sepsis (Phoenix Sepsis Score of 2), with acute respiratory distress and a depressed level of consciousness. Blood metagenomic next-generation sequencing (mNGS) identified high-load HBoV1 viremia (7,513 reads) as the sole pathogen, with negative blood and urine cultures. During the septic peak, urinary tract ultrasonography was normal; however, follow-up imaging on day 13 revealed delayed-onset, non-obstructive pyelectasis and increased post-void residual (PVR) volume. These functional abnormalities were resolved completely within 2.5 months.

Conclusions: HBoV1 can act as a sole pathogen to cause pediatric sepsis. The observed triphasic urinary course-early sterile pyuria, imaging quiescence at the sepsis peak, and delayed functional impairment-supports an inflammation-mediated pathogenesis rather than direct viral invasion. This case highlights the critical role of advanced molecular diagnostics in identifying viral etiologies in culture-negative sepsis and underscores the necessity of longitudinal functional surveillance, as clinically significant organ dysfunction may manifest after the acute inflammatory phase has resolved.

Background: Cupriavidus gilardii is a multidrug-resistant (MDR) pathogen found in soil and water. Human infection is extremely rare, with only two pediatric cases reported to date, and its clinical features and the antimicrobial strategies remain unclear. The pathogen's MDR nature often poses a therapeutic challenge. This case provides valuable clinical evidence regarding the management of C. gilardii infection in immunocompromised pediatric patients.

Case description: A 4-year-old boy with acute lymphoblastic leukemia developed severe immunosuppression after reinduction cyclophosphamide, cytarabine (Ara-C), mercaptopurine (CAM) chemotherapy according to the SCCCG-ALL-2023 protocol. The patient initially presented with recurrent fever, septic shock, and progressive respiratory distress. Despite an aggressive empirical antibiotic regimen including meropenem, imipenem, piperacillin-tazobactam, linezolid, and levofloxacin, his condition continued to worsen. Blood cultures and metagenomic next-generation sequencing (mNGS) subsequently confirmed C. gilardii infection. Based on susceptibility testing, therapy was adjusted to a combination of ceftazidime-avibactam (CAZ-AVI) and tigecycline. After this adjustment, the patient showed marked clinical improvement, with decreased inflammatory indicators and nearly completed clearance of the pathogen. Unfortunately, on hospital day 26, he developed sudden massive hemoptysis due to Aspergillus pulmonary artery invasion and died despite emergency treatment.

Conclusions: This case demonstrates that CAZ-AVI may offer effective antimicrobial control for C. gilardii infection in immunocompromised pediatric patients. Although the patient succumbed to secondary fungal complications, successful microbiological control demonstrates that CAZ-AVI may serve as a potential salvage therapy for rare MDR Gram-negative bacteria and provides clinical insight into the management of uncommon pediatric infections.

Background: Early identification of individuals at high risk for autism spectrum disorder (ASD) is crucial for optimizing intervention strategies and improving outcomes. This study aims to develop a risk prediction model integrating biopsychosocial factors through a systematic review with multicenter validation.

Methods: A comprehensive search was conducted across PubMed, Cochrane Library, and Embase for articles on biopsychosocial ASD risk factors during 2010-2023. Two reviewers independently extracted data. Meta-regression analysis of 37 systematic reviews/meta-analyses identified 18 potential risk factors by Stata 16.0. Four core variables were included in the prediction model, while 14 were excluded due to low-quality evidence or insufficient data after screening. Multivariate logistic regression with least absolute shrinkage and selection operator (LASSO) variable selection derived model weights. External validation was performed in a Chinese cohort (n=1,175) from two tertiary hospitals. Model discrimination was assessed via receiver operating characteristic (ROC) curves and clinical utility by decision curve analysis (DCA).

Results: Analysis of 37 systematic reviews identified four independent predictors of ASD risk: adverse childhood experiences (ACEs) [odds ratio (OR) =2.11; 95% confidence interval (CI): 1.61-2.77], preterm birth (OR =3.3; 95% CI: 1.24-7.60), antidepressant exposure during pregnancy (OR =1.17; 95% CI: 1.08-1.21), and perinatal antibiotic exposure (OR =1.52; 95% CI: 1.09-2.12). The risk model formula was: 0.82 × (ACEs) + 1.19 × (preterm birth) + 0.42 × (antidepressant exposure) + 0.21 × (perinatal antibiotic exposure). External validation showed excellent discrimination [area under the curve (AUC) =0.78; 95% CI: 0.75-0.81]. DCA confirmed significantly higher net clinical benefit compared to universal intervention strategies.

Conclusions: This study developed a risk prediction model integrating biopsychosocial factors, providing an evidence-based tool for early identification of individuals at high risk for ASD.

Background: Mycoplasma pneumoniae (MP), characterized by periodic outbreaks, causes community-acquired pneumonia in Chinese children. Non-pharmaceutical interventions (NPIs) during the coronavirus disease 2019 (COVID-19) pandemic significantly altered MP transmission. Following the relaxation of NPIs, MP infections rebounded globally, with trends toward younger individuals and increased severity. In this study, we aimed to identify new changes in the epidemiological characteristics, clinical phenotypes, and treatment strategies of MP infection in the post-pandemic era in China.

Methods: In this retrospective study, we analyzed the clinical data of 541 pediatric patients with MP nucleic acid samples who were treated at a general hospital in Chongqing, China between January 2018 and December 2024. Participants were divided into an observation group [2023-2024] and a control group [2018-2022] based on their hospitalization dates. Differences in the group characteristics were compared.

Results: After the relaxation of NPIs, the 2023 hospitalization rate was 5.1 times higher than that before the pandemic (11.16% vs. 2.29%). The peak age of onset shifted from 5-15 to 3-7 years. The observation group had a significantly higher proportion of severe cases (74.34% vs. 40.00%), more intense fever and cough symptoms, multilobar involvement (66.89%), and a higher post-discharge residual cough rate (64.69% vs. 20.00%). In the observation group, tetracycline usage increased (57.38% in children <8 years), glucocorticoid use increased (63.82% vs. 23.53%), and treatment duration was extended.

Conclusions: The post-pandemic era witnessed a surge in MP infections in younger patients, with more severe cases requiring adjusted treatment strategies. This study provides critical evidence for the clinical management and public health decision-making for MP pneumonia.

Background: Angle Class II Division 1 malocclusion in adolescents can impair oral health and facial aesthetics, yet evidence on clear aligner therapy is limited. This study aimed to evaluate the clinical efficacy of clear aligner therapy combined with Class II intermaxillary traction for treating Angle Class II Division 1 malocclusion in adolescents during the early permanent dentition stage.

Methods: A total of 30 adolescents (14 males and 16 females; age range, 12-15 years; mean age: 12.6 years) diagnosed with Angle Class II Division 1 malocclusion in early permanent dentition were enrolled. All patients received non-extraction treatment using clear aligners combined with Class II intermaxillary traction. Treatment outcomes were assessed by comparing pre- and post-treatment cephalometric measurements using statistical analysis.

Results: The mean treatment duration was 28.6 months. All patients achieved satisfactory occlusal outcomes, including improved overjet, overbite, and Class I molar relationship, along with notable enhancement in facial profile aesthetics. Post-treatment cephalometric analysis revealed statistically significant improvements (P<0.05) in most parameters, including sella-nasion-point B angle (SNB), point A-nasion-point B angle (ANB), gonion-pogonion distance (Go-Pog) distance, U1-SN angle, U1-NA angle, U1-L1 angle, U1-NA distance, L1-MP angle, L1-NB angle, L1-NB distance, overjet, overbite, U1-PP distance, L1-MP distance, PTM-U6 distance, upper lip to E-plane (UL-EP), lower lip to E-plane (LL-EP), nasolabial angle (NLA), and facial convexity angle (FCA). No significant changes were observed in sella-nasion-point A angle (SNA), MP-SN, or MP-FH angles (P>0.05).

Conclusions: Clear aligner therapy, when combined with Class II intermaxillary traction, is an effective approach for correcting Angle Class II Division 1 Malocclusion in adolescents. It not only improves occlusal relationships but also enhances facial soft tissue profile.

Background: Acute kidney injury (AKI) is a serious postoperative complication in hospitalized neonates. We aimed to develop and evaluate a machine learning (ML) model for predicting the risk of postoperative AKI in neonates.

Methods: The clinical records of 2,025 neonates were collected, and the patients were randomly divided into training and test sets. The outcome variable was the occurrence of postoperative AKI, and the models incorporated 25 predictive variables, including demographics, intraoperative infusions, and postoperative indicators. ML models were developed using six different algorithms on the training set, and their performance was assessed on the test set using the area under the curve (AUC) of the receiver operating characteristic (ROC) curve. The model with the best AUC was selected for validation in the test set. The association between the risk factors and postoperative AKI was interpreted using the SHapley Additive exPlanations (SHAP) method.

Results: A total of 110 neonatal patients (5.43%) developed AKI following surgery. Patient age, operation duration, and urine output were the three most important predictors of AKI. Among the tested models, the logistic regression (LR) algorithm was the best predictor of postoperative AKI, achieving the highest AUC [median, 0.807; 95% confidence interval (CI): 0.701-0.897] and the highest sensitivity (median, 0.733; 95% CI: 0.5-0.938). The SHAP method was used to illustrate the prediction process of the LR model for neonatal postoperative AKI at the level of individual patients.

Conclusions: The ML model that uses the LR algorithm with eight commonly measured variables could serve as a tool to predict postoperative AKI in neonates.