Frontiers in Pediatrics最新文献

Background: The prognosis for pediatric patients with relapsed or refractory sarcoma remains poor, and standard salvage therapies are lacking. This study evaluated the efficacy and toxicity of the gemcitabine and docetaxel (GEMDOX) combination in this patient population.

Methods: We retrospectively analyzed 36 pediatric patients treated with GEMDOX at our institution between 2015 and 2025. Patients received gemcitabine (1,000 mg/m² on days 1 and 8) and docetaxel (100 mg/m² on day 8) in 21-day cycles.

Results: The median age was 13.5 years, with osteosarcoma being the most common diagnosis (58.3%). GEMDOX was administered predominantly as a third-line regimen (58.3%). The objective response rate (ORR) at the final assessment was 5.8%, and the disease control rate (DCR) was 14.6%. The median progression-free survival (PFS) and overall survival (OS) were 5.72 months (95% CI, 3.95-7.48) and 12.33 months (95% CI, 8.97-15.66), respectively. The most common Grade 3-4 toxicities were neutropenia (22.2%) and febrile neutropenia (19.4%), both of which were manageable with G-CSF support. No treatment-related mortality occurred.

Conclusions: Although the objective response rate was modest in this heavily pretreated cohort, GEMDOX demonstrated a manageable safety profile. It represents a viable palliative option with a manageable toxicity profile for pediatric patients with relapsed/refractory sarcoma when curative options are limited. However, given its intensive nature, optimal efficacy may be better achieved when utilized earlier in the relapse setting rather than as a late-line rescue therapy.

Invasive aspergillosis is a severe opportunistic infection in immunocompromised children, particularly those receiving chemotherapy for hematologic malignancies. We report the case of a 2-year-old girl with acute lymphoblastic leukemia who developed massive hemoptysis during consolidation chemotherapy. Thoracic computed tomography revealed a saccular pseudoaneurysm of the proximal left subclavian artery. Surgical resection and autologous vein graft replacement were performed, and Aspergillus flavus DNA was detected in the resected tissue using Aspergillus-specific polymerase chain reaction. The patient received dual antifungal therapy with liposomal amphotericin B and voriconazole, followed by long-term voriconazole prophylaxis. She made a full recovery. This case highlights the importance of considering angioinvasive aspergillosis in immunocompromised children presenting with hemoptysis and lung lesions. Early recognition and multidisciplinary management are critical to preventing fatal vascular complications.

Introduction: Some individuals with spinal muscular atrophy (SMA) transition from nusinersen to risdiplam during disease-modifying therapy (DMT) due to factors such as treatment convenience, economic considerations, and adverse events (AEs). This study evaluates the safety and effectiveness of switching DMTs by analyzing real-world clinical data from multiple centers in China.

Methods: Patients with 5q-SMA who switched from nusinersen to risdiplam were enrolled from four medical institutions in Jiangsu Province. The reasons for switch, as well as any adverse events experienced, were documented. Assessments of motor function were conducted prior to treatment, following the switch, and at four-month intervals subsequently.

Results: A total of eleven patients were included in this retrospective analysis. RULM scores showed maintains improvement following the switch compared to baseline measurements prior to treatment initiation. No significant adverse events were reported after the switch.

Conclusion: Despite the small sample size and lack of a control group, these findings suggest that switching from nusinersen to risdiplam in real-world clinical settings is safe and allows for continued improvement of motor function in SMA patients.

Introduction: Juvenile idiopathic arthritis (JIA) is the commonest rheumatologic disease in children and frequently affects the knee joint. Synovial inflammation and tenosynovitis are key pathological features, and ultrasound plays an increasingly important role in their assessment. Superb Microvascular Imaging (SMI) is a novel Doppler technique with enhanced sensitivity to low-velocity microvascular flow, but evidence on its repeatability in JIA remains limited. This study aimed to evaluate intra- and inter-observer repeatability of knee SMI in children with JIA.

Methods: In this prospective multicenter study (June 2023-October 2024), 76 children with JIA were examined (Hannover Medical School and St. Josef-Stift Sendenhorst). Each underwent three standardized SMI scans: two by the same and one by a different examiner. Synovial vascularity was graded using the Pediatric OMERACT scoring system. Intra- and inter-observer reliability measures were calculated using intra-class correlation coefficients (ICC). Agreement between longitudinal and transverse suprapatellar planes was assessed using weighted kappa statistics, and correlations with clinical disease activity were analyzed via logistic regression.

Results: Intra-observer reliability was excellent (ICC = 0.972, 95% CI: 0.956-0.982). Inter-observer reliability was strong (ICC = 0.828-0.928), regardless of examiner experience. Agreement between imaging planes was substantial (κ = 0.72, p = 0.32). Synovial vascularity scores correlated significantly with clinical measures of active arthritis (OR = 1.182, p = 0.0004), particularly with swelling (OR = 1.249, p < 0.0001).

Discussion: SMI demonstrates excellent repeatability for assessing synovial vascularity in JIA. Its reliability, examiner independence, and non-invasive nature support its use for routine monitoring and longitudinal disease evaluation in pediatric rheumatology.

Background: Intraventricular Hemorrhage (IVH) is one of the common and serious complications of Very Low Birth Weight Infant (VLBW) that may lead to long-term neurodevelopmental deficits. Although several studies have been conducted to explore its risk factors, the results have been inconsistent. The aim of this study was to identify the major risk factors for intraventricular Hemorrhage in VLBW by systematic evaluation and Meta-analysis of the available evidence.

Methods: PubMed, Web of Science, Embase, Cochrane Library were systematically searched, and observational studies (case-control and cohort studies) were included from the time of library construction to 20 January 2025, and the literature that met the criteria were screened and relevant data were extracted. Meta-analysis was performed using Stata 15.0 software to assess the combined odds ratio (OR) and 95% confidence interval (CI) for each risk factor.

Results: A total of 21 studies included 6 case-control studies, 15 cohort studies, involving a total of 13,800 patients, The results of the meta-analysis showed that hypotension [OR = 3.64, 95%CI (1.87, 7.08)], patent ductus arteriosus (PDA) [OR = 1.86, 95%CI (1.46, 2.36)], vaginal delivery [OR = .10, 95%CI (1.61, 2.72)], neonatal thrombocytopenia[OR = 2.43, 95%CI (1.79, 3.30)], pulmonary hemorrhage [OR = 2.45, 95%CI (1.43, 4.20)], mechanical [OR = 2.09, 95%CI (1.40, 3.10)], sepsis[OR = 2.28, 95%CI (1.77, 2.95)] were a risk factor for the development of IVH in VLBW. While antenatal corticosteroids [OR = 0.68, 95%CI (0.55, 0.84)] was a protective factor for the development of IVH in VLBW.

Conclusion: This study indicates that hypotension, patent ductus arteriosus (PDA), antenatal corticosteroid use, vaginal delivery, neonatal thrombocytopenia, pulmonary hemorrhage, mechanical ventilation, and sepsis constitute the primary risk factors for IVH in VLBW infants. Although these factors exhibit strong clinical associations, current understanding of IVH pathogenesis remains largely dependent on preclinical studies. Integrating clinical and preclinical evidence facilitates a more comprehensive understanding of IVH etiology and informs early intervention strategies.

Systematic review registration: identifier CRD42025633474.

Background: This multicenter study investigated the association between birthweight discordance (BWD) and extrauterine growth restriction (EUGR) in preterm twins, and evaluated the modifying role of small for gestational age (SGA).

Methods: Data from 2,496 infants (1,248 twin pairs) admitted to 22 Chinese NICUs (2018-2020) were analyzed. BWD was calculated as the percentage difference in birthweight between larger and smaller twins, categorized into four groups (≤15%, 15%-20%, 20%-25%, >25%). EUGR was defined as discharge weight below the 10th percentile for corrected gestational age and sex (Fenton's chart). A generalized linear mixed model was employed to analyze the association between BWD and EUGR. Modification analysis was performed to assess the effect of SGA on this association.

Results: BWD of >25% was associated with a significantly increased risk of EUGR compared to BWD ≤15% (adjusted OR = 1.59, 95% CI: 1.05-2.41). Stratified analysis revealed a consistent association in SGA infants (OR = 1.38, 95% CI: 1.30-1.47).

Conclusion: Findings highlight BWD as a critical risk factor for EUGR, particularly in SGA twins. This association suggests that future research should investigate whether tailored monitoring and nutritional interventions in NICUs could help mitigate these growth disparities.

Objective: The current diagnostic and prognostic assessment of congenital diaphragmatic hernia (CDH) in neonates remains challenging. This study aimed to evaluate the utility of neonatal ultrasonography in the diagnosis and prognostic prediction of CDH in infants.

Materials and methods: A retrospective analysis was conducted on clinical data from 152 infants diagnosed with CDH and admitted to the Department of Neonatal Surgery at Children's Hospital between 2017 and 2023. The cohort included 86 (56.6%) males and 66 (43.4%) females. Multivariate logistic regression was employed to identify factors associated with CDH prognosis. Receiver operating characteristic (ROC) curve analysis was performed to assess the predictive value of significant ultrasonographic indicators.

Results: Multivariate logistic regression identified four factors as significant predictors of mortality: diaphragmatic defect length >4 cm [odds ratio [OR] = 2.41, 95% confidence interval [CI]: 1.08-10.58], the presence of hepatic herniation (OR = 2.61, 95% CI: 1.16-5.87), absence of a hernial sac (OR = 4.86, 95% CI: 2.00-11.76), and concomitant lung ultrasound abnormalities (OR = 10.86, 95% CI: 1.28-21.85). The combination of these four parameters demonstrated strong predictive performance for mortality, with an area under the ROC curve of 0.860 (95% CI: 0.786-0.935).

Conclusion: Diaphragmatic defect length, hepatic herniation, hernial sac status, and lung ultrasound findings serve as valuable prognostic indicators in infants with CDH. Integrating these four parameters enhances prognostic accuracy and may support clinical decision-making.

Background: The incidence of obstructive sleep apnea (OSA) in children has increased in recent years. Many primary hospitals have not polysomnography which can diagnose OSA. To investigate the lowest blood oxygen saturation at night (LSaO₂) combine with obstructive sleep apnea 18 items survey (OSA-18) scale to initially screen the sleep status of children with OSA.

Methods: A retrospective study of 189 children aged 4-12 years with sleep-disordered breathing was conducted. All children were monitored using polysomnography and divided into the simple snoring and OSA groups (mild, moderate, and severe). Their parents completed the OSA-18 scale. Correlation among three indices [LSaO2, OSA-18 scale, and obstructive apnea hypoventilation index (OAHI)] was assessed. Subsequently, series and parallel tests were used to understand the sensitivity and specificity of diagnosis.

Results: There was no statistical differences in sex (P = 0.909) and age (P = 0.894), and a significant difference in OSA-18 scores between the simple snoring and OSA groups (P = 0.014) but not in the LSaO2 (P = 0.409). OSA-18 and LSaO2 scores of the mild, moderate, and severe groups were significantly different (P < 0.05), and LSaO2 was correlated with the OAHI. Use of the OSA-18 scale combined with LSaO2 to assess the boundary value of mild and moderate-to-severe OSA was better than that of each index independently. The sensitivity and specificity the parallel test were 85.7% and 62.7%, respectively, can better predict OSA than series test.

Conclusion: The OSA-18 scale combined with LSaO2 has diagnostic value for the diagnosis of OSA, and can be used as a prediction tool for OSA.

Background: Breastfeeding promotion is a cornerstone of neonatal nursing, as it plays a pivotal role in safeguarding the health and fostering the development of newborns. This study aimed to assess neonatal nurses' Knowledge, Attitudes, and Practices (KAP) regarding breastfeeding for hospitalized neonates and identify factors influencing these domains.

Methods: A cross-sectional survey was conducted among neonatal nurses between July 1 and August 30, 2025. Eligible nurses were recruited via a rigorous screening process, and data were collected using a validated KAP scale. Benjamini-Hochberg false discovery rate (FDR) adjustment addressed multiple comparison biases, and comprehensive regression assumption checks (multicollinearity, residual normality, homoscedasticity) were performed to ensure result validity.

Results: A total of 122 neonatal nurses were included. Based on predefined scoring benchmarks (Low: ≤90; Moderate: 91-140; High: ≥141), participants demonstrated a moderate level of overall breastfeeding-related KAP (mean ± SD: 134.28 ± 14.02). Hierarchical regression analysis revealed: (1) Knowledge was significantly predicted by age (β = 0.304, p = 0.017), years of clinical experience (β = 0.433, p = 0.040), educational level (β = 0.385, p = 0.015), and specialized breastfeeding training (β = 0.402, p = 0.007); (2) Attitudes were significantly influenced by number of children (β = 0.224, p = 0.018), professional title (β = 0.196, p = 0.002), and specialized training (β = 0.264, p = 0.001); (3) Practices were significantly associated with years of clinical experience (β = 0.380, p = 0.028), professional title (β = 0.504, p = 0.011), educational level (β = 0.436, p = 0.020), and specialized training (β = 0.329, p = 0.001). Specialized breastfeeding training emerged as a consistent positive predictor across all KAP dimensions (medium effect sizes), explaining 56.0%-63.6% of the variance in the models (adjusted R ²).

Conclusion: Neonatal nurses exhibited positive attitudes but moderate knowledge and practice levels regarding breastfeeding, with targeted gaps in evidence-based care and parental education. These findings highlight the need for tailored training programs-prioritizing younger nurses, those with less experience, lower educational/professional titles, and untrained individuals-to enhance breastfeeding-related competence.