European Journal of Pediatrics最新文献

Carbapenem-resistant multidrug-resistant (MDR) and pandrug-resistant (PDR) Gram-negative infections represent a critical therapeutic challenge in pediatric intensive care units (PICUs), where effective treatment options are extremely limited. This study aimed to describe the real-world clinical outcomes and safety of ceftazidime-avibactam (CZA) in critically ill children with MDR/PDR infections. We conducted a retrospective observational study of pediatric patients aged 1 month to 18 years who received CZA for microbiologically confirmed MDR or PDR Gram-negative infections in a tertiary PICU between February 2021 and January 2025. Twenty-one critically ill children (median age 55 months, IQR 11-126) were included; two-thirds had underlying chronic conditions and 23.8% were immunosuppressed. Klebsiella pneumoniae was the predominant pathogen (85.7%). Microbiological clearance in follow-up cultures was observed in 85.7% of patients with available microbiological data, and clinical improvement was noted in most patients in this critically ill population. Infection-related mortality was 19% and was primarily associated with host-related factors, including younger age and immunosuppression. Resistance emergence during therapy was observed in one patient. Such resistance development during CZA treatment has been reported rarely in the literature and may involve mechanisms such as mutations in carbapenemase enzymes or alterations in bacterial permeability. In our study, resistance was detected during ongoing therapy in a single patient with Klebsiella pneumoniae, highlighting the importance of close microbiological monitoring in critically ill patients receiving prolonged treatment.

Conclusion:  CZA was used as a salvage treatment in critically ill children with MDR and PDR Gram-negative infections, with favorable clinical and microbiological outcomes observed. These real-world data support its cautious incorporation into pediatric antimicrobial stewardship strategies in PICU settings.

What is known: • Ceftazidime-avibactam is a promising agent against carbapenem-resistant Enterobacterales in adults. • Pediatric data remain limited, especially in critically ill children with MDR or PDR infections.

What is new: • Ceftazidime-avibactam was used as salvage therapy for multidrug- and pandrug-resistant Gram-negative infections in critically ill children. • Clinical and microbiological response was achieved in a high-risk PICU population despite prolonged hospitalization and extensive prior antibiotic exposure.

Purpose: Haematuria and proteinuria may point to kidney diseases, but may also be found incidentally. Among schoolchildren, many studies suggest their prevalence to around 1%. In neonates, the frequency and persistence of haematuria and/or proteinuria in the general population have so far not been investigated systematically.

Methods: In the course of the prospective KUNO-Kids Health Study, urine samples were collected and analysed by dipstick in asymptomatic neonates on days three to five after birth. Those with positive findings underwent a maximum of two follow-ups until 16 weeks of age and factors associated with haematuria and/or proteinuria were explored.

Results: Of 509 participants with a urine sample available, 27% (n = 139) exhibited positive results. Of these, 58% (n = 81) had isolated haematuria, 21% (n = 29) had isolated proteinuria, and 21% (n = 29) had both. Of all children with positive urine tests, 76% (n = 105) underwent a first follow-up (mean 7 weeks later), and only in 1.9% (n = 2) was a positive result found. In the second follow-up (mean 2 weeks later), no positive results were detected anymore. Positive urine test results were more common in females and after vaginal delivery (p < 0.001 and p = 0.037, respectively).

Conclusion: The present study identified haematuria and/or proteinuria in a significant proportion of healthy newborns shortly after birth, but results returned to normal within weeks in all participants available for follow- up. Thus, isolated findings of haematuria and/or proteinuria in newborns should be interpreted with caution.

What is known: • Previous studies have reported the prevalence and persistence of haematuria and/or proteinuria in school- age children, but data on haematuria and/or proteinuria in neonates are scarce.

What is new: • Positive urinary test results for haematuria and/or proteinuria are common after birth and were found in 27%. • Positive urinary test results returned to normal within 16 weeks in all children tested.

This study aims to investigate the utilization of online support communities by caregivers of children with Tic disorders (TD) and explore its relationship with children's quality of life, anxiety levels, and caregivers' quality of life. In this cross-sectional study, 330 TD children and their caregivers from the TD online support communities were included. They filled out general information questionnaires, self-assessment scales for quality of life and anxiety, tic severity scales, and answered several open-ended questions. Open-ended questions were used to explore caregivers' experiences with online support communities through qualitative analysis. Statistical analyses included univariate analysis, multifactorial regression, and Spearman correlation to examine the relationship between participation time and quality of life, anxiety. In qualitative analysis, three themes document the experience of using TD online support community. In the correlation analysis, there is a significant positive correlation between participation time and caregiver quality of life. Similarly, the longer the participation time, the higher the quality of life and lower the anxiety level of TD children. We also found that frequent access to online support communities can reduce anxiety and depression among caregivers. Higher per capita monthly household income and the education level of caregivers can improve the quality of life of TD children and caregivers, reducing anxiety and depression.

Conclusion:  Online support communities play a significant role in improving the quality of life of TD children and their caregivers, reducing anxiety, and providing valuable emotional and informational support.

What is known: • Online support communities often provide members with information support, emotional support, online support, and respect support. • TD online support community is an online community dedicated to providing information exchange and resource sharing for TD patients and their caregivers, playing a crucial role in improving patients' lives.

What is new: • Quantitatively demonstrate that long-term participation in TD online support communities is associated with improved quality of life for children with TD and their caregivers, as well as reduced anxiety levels in children with tic disorders.

Chemotherapy-related toxicity has been associated with body composition, yet there is no consensus on which CT-derived muscle parameter or handgrip strength (HGS) best predicts toxicity in pediatric cancer patients. This study evaluated whether CT-derived body composition parameters and nutritional status independently predict early chemotherapy-related infections and hematologic toxicity in children and adolescents with cancer. In this prospective observational cohort, patients aged 7-18 years undergoing chemotherapy with available CT/PET-CT scans were included. Anthropometric data (weight, height, arm circumference, triceps skinfold thickness) and HGS were collected. At the third lumbar vertebra (L3), skeletal muscle mass (SMM), skeletal muscle index (SMI), total adipose tissue, and muscle radiodensity were measured; psoas area was assessed at L4. Associations were analyzed using χ2 tests and logistic regression adjusted for sex, age, tumor stage, and type. Forty-eight patients were included (mean age 12.6 ± 3.3 years; 64.6% male); 64.6% had hematologic malignancies and 53.6% had advanced disease. Most were classified as having normal nutritional status (50-64.6%). Hematologic (100%) and gastrointestinal (85.4%) toxicities were most frequent. Low SMM and SMI were correlated with a higher risk of infection (OR 4.20; 95% CI 1.23-14.27 and OR 3.92; 95% CI 1.17-13.20, respectively) and fever (OR 4.05; 95% CI 1.21-13.54 and OR 5.82; 95% CI 1.67-20.25, respectively). After adjustment, only low SMI remained independently associated with fever (OR 4.74; 95% CI 1.09-20.61; p = 0.038).

Conclusions: Low muscle mass was associated with early infection and fever-related toxicity, suggesting a role of muscle quantity in immune function during pediatric cancer treatment.

What is known: • Body composition in children and adolescents changes during chemotherapy is associated with treatment-related toxicity. • Muscle status is crucial for chemotherapy tolerance and treatment response.

What is new: • Low skeletal muscle mass was associated with a higher risk of toxicities, including fever. • No association was found between anthropometric parameters, HGS and toxicities, indicating that further studies are needed.

To describe and assess diaphragmatic function by ultrasound during the weaning and post-extubation phases in preterm newborns. This cross-sectional study included intubated preterm newborns eligible for extubation. Inspiratory diaphragmatic thickness (IDT), expiratory diaphragmatic thickness (EDT), diaphragmatic thickening fraction (DTF), and diaphragmatic excursion (DE) were evaluated at three time points: T1-during invasive mechanical ventilation (IMV); T2-during the spontaneous breathing trial (SBT); and T3-during nasal intermittent positive pressure ventilation (NIPPV). Friedman test and Wilcoxon signed-rank post hoc test were used for comparisons (p < 0.05). Fifty preterm newborns were evaluated. Forty-five (mean gestational age 30.9 ± 2.2 weeks; mean weight 1377.5 ± 432 g) received IMV for 4 ± 3 days and were successfully extubated (extubation failure rate 10%). Due to the small number of failures (n = 5), only descriptive analyses were performed. In successfully extubated newborns, IDT and DE increased at T2 and T3 compared to T1 (p < 0.05), with IDT highest at T2. No significant changes were observed in EDT or DTF, which remained consistently elevated.Conclusion: In preterm newborns successfully extubated, diaphragmatic thickness and excursion increased during the SBT and remained elevated after extubation. These findings may support the use of diaphragmatic ultrasound to guide extubation readiness and monitor post-extubation adaptation in preterm infants.

Pediatric advanced chronic kidney disease (CKD) profoundly affects not only the patient but the entire family system. This study explores how affected families-including patients, siblings, and primary caregivers-perceive and experience advance CKD as a chronic, life-limiting illness with life-prolonging but non-curative treatment options. A qualitative study was conducted involving seven families recruited from three pediatric nephrology centers. Semi-structured interviews were conducted with patients with CKD ≥ 4, at least one sibling and one parent per family. Both, patients and siblings, were aged ≥ 14 years. Data were analyzed using qualitative content analysis. Five major themes were identified: families' understanding of CKD as a chronic life-limiting disease, coping with CKD and its uncertain course, the perspectives of healthy siblings, family dynamics, and communication. Even in stable medical conditions like a functioning kidney transplant, participants acknowledged CKD as a lifelong condition without definitive cure options. Siblings frequently adapted flexibly to family needs, occasionally adapting their role to fill structural gaps. Family members involved in caregiving-whether parents or siblings-frequently expressed feeling insufficiently informed. In contrast, patients and siblings not engaged in caregiving generally felt well informed.

Conclusion:  By capturing family-wide perspectives, this study contributes new evidence on intra-family dynamics and supports the need for family-centered care that integrates structured communication, sibling involvement in clinical practice, and screening for family-level risk factors.

What is known: • Pediatric advanced CKD impacts family functioning. • Siblings are at risk for psychological distress but are often overlooked in care.

What is new: • Additional stressors beyond CKD can increase caregiving burden and compromise family coping. • Information needs are tied to the level of responsibility and involvement in disease management. • Siblings often adopt flexible roles to stabilize the family system.

This study aimed to compare key pathological parameters of the liver and cyst wall between prenatally and postnatally diagnosed choledochal cyst children and analyze the characteristics and clinical implications of pathological changes in these groups. Between June 2023 and December 2023, 85 patients were reviewed: group 1, prenatally diagnosed group (n = 44); group 2, postnatally diagnosed group (n = 41). Patient demographics, liver pathology parameters, and cyst wall pathology parameters were evaluated and compared. The overall rate of advanced fibrosis was 16.5%. The fibrosis rate was comparable between group 1 and group 2, but bile plug formation was more severe in group 1. The degree of cyst wall fibrosis in the middle segment was higher in group 1. The distribution pattern of smooth muscle fibers was predominantly scattered in both groups. Group 1 showed a more severe absence of smooth muscle fibers in both the proximal and middle segments. Cyst diameter and length were positively correlated with the stage of liver fibrosis. The degree of cyst wall fibrosis was negatively correlated with smooth muscle bundle thickness. Two patients (2.4%) exhibited both intestinal metaplasia and pyloric gland metaplasia.

Conclusion: Children diagnosed prenatally demonstrated more severe hepatic fibrosis, bile plug formation, cyst wall fibrosis, and smooth muscle deficiency. Early diagnosis and surgery are recommended to avoid postoperative complications.

What is known: • Choledochal cyst is the most common congenital malformation in pediatric hepatobiliary disorders, with half of all cases diagnosed prenatally. • Prenatally diagnosed patients exhibit distinct pathological characteristics, but relative studies are very rare.

What is new: • Prenatally diagnosed cases showed a higher incidence of hepatic fibrosis, bile plug formation, cyst wall fibrosis, and smooth muscle deficiency. • Two patients exhibited both intestinal metaplasia and pyloric gland metaplasia.

The purpose of this study is to evaluate if non-invasive high-frequency oscillation ventilation (NHFOV) is non-inferior to nasal continuous positive airway pressure (CPAP) as primary non-invasive respiratory support (NRS) in preterm neonates of ≥ 30 weeks' gestation with respiratory distress syndrome (RDS). In this open-label randomized controlled trial (RCT) with a non-inferiority design conducted in a lower middle-income country, 142 preterm neonates were randomized to receive NHFOV (n = 71) or CPAP (n = 71) at equivalent pressures after lung recruitment with nasal mask (NM) interface. A non-inferiority margin of 20% was pre-specified and a two-sided 90% confidence interval (CI) standardly used in non-inferiority trials was chosen. For the outcome treatment failure (requirement of an alternate NRS as rescue), the event rate was 4.2% in both groups, risk difference (RD) with 90% CI being 0.00 (- 0.06 to + 0.06), and that for IMV requirement was - 0.01 (- 0.04 to 0.01). Since upper limits of CIs for both primary outcomes were well below the non-inferiority margin, NHFOV was proven to be non-inferior compared to CPAP. Duration of primary NRS was significantly lesser (median difference (MD) (95% CI), 7 h lesser (- 14 to 0); p = 0.03) and ventilator-free days for primary NRS were significantly higher (MD (95% CI), 0.30 days (0.00 to 0.60); p = 0.02) in the NHFOV group.

Conclusion:  In preterm neonates of ≥ 30 weeks' gestation with RDS, NHFOV delivered through NM at equivalent pressures is non-inferior to CPAP when used as primary NRS. The finding of shorter NRS duration with NHFOV needs to be proven in adequately powered trials. Use of NM interface with equivalent pressures addresses the lacunae in the current literature on NHFOV and provides a rigorous comparison between the two NRS modes.

Trial registration: www.ctri.nic.in , id CTRI/2024/10/074939, registered on 8 October 2024.

What is known: • CPAP as initial respiratory support for preterm neonates with RDS is the standard of care in LMICs. • NHFOV is more efficacious than CPAP when used as a post-extubation respiratory support modality, evidence for the same being uncertain when used as primary support.

What is new: • NHFOV is non-inferior to CPAP as primary support in preterm neonates ≥ 30 weeks with RDS for the outcomes of treatment failure and IMV requirement with equivalent pressures after lung recruitment and nasal mask interface. • A superiority design RCT comparing these two non-invasive respiratory support modalities in this subgroup of preterm neonates may not be feasible. The low baseline event rate of IMV necessitates an impractically large sample size to achieve adequate power.

Although lung ultrasound (LUS) is considered non-invasive in nature, handling and probe pressure may provoke pain and transient physiological changes in preterm infants. The aim of this study was to determine whether oral sucrose combined with a pacifier reduces procedural pain and stress in preterm infants undergoing bedside LUS. A two-arm randomized controlled trial was conducted between November 2020 and January 2023. Preterm infants undergoing LUS were randomized to receive either oral sucrose-plus-pacifier or pacifier-alone, administered 2 min before the scan. Pain was evaluated using the premature infant pain profile (PIPP) at four time points: baseline (30 s before intervention), 30 s into scanning, midpoint of the scan, and 30 s post-procedure. Physiological parameters (heart rate, oxygen saturation, and apneic episodes) and duration of the procedure were recorded. Group differences in PIPP scores were analyzed using repeated-measures ANOVA. Out of 60 infants enrolled, 30 infants were randomized in each group with data available on 29 infants in the sucrose-plus-pacifier group and 27 in the pacifier-alone group. Baseline demographics and clinical characteristics were comparable between groups. No statistically significant differences were observed in PIPP scores and physiological parameters at any time point between groups. Repeated-measures ANOVA showed a significant change in PIPP scores across procedural phases (p < 0.0001), with no differences between groups (p = 0.16) or group-by-phase interaction, indicating similar PIPP score trajectories in both groups (p = 0.21). Conclusion: Among preterm infants, sucrose did not add benefit to pacifier-alone for reducing procedural pain and stress during lung ultrasound.Trial registration: NCT05717088. What is Known • Lung ultrasound (LUS) is a bedside, radiation-free imaging modality increasingly used in preterm infants, but probe pressure and handling may still provoke mild procedural discomfort and physiological changes. • Oral sucrose is an established non-pharmacologic analgesic for painful neonatal procedures; however, its benefit during minimally invasive procedures such as LUS remains uncertain. What is New • In this randomized controlled trial, oral sucrose did not provide additional analgesic benefit beyond pacifier use alone during bedside LUS in preterm infants. • These findings suggest that non-nutritive sucking alone may be sufficient for comfort during LUS, supporting its classification as a minimally distressing neonatal bedside procedure.