Pediatric Investigation最新文献

Importance: The safety and efficacy of the bath-plug technique for the closure of cerebrospinal fluid (CSF) leaks in children remain unknown.

Objective: We undertook this study to ascertain whether the bath-plug technique was safe and effective for the repair of CSF leaks.

Methods: We retrospectively reviewed patients who underwent endoscopic repair of CSF leaks with the fat graft as a plug-in at Beijing Children's Hospital from March 2016 to May 2020. Demographic data, medical history, defect sites and sizes, interventions, and clinical outcomes were analyzed. One representative clinical case was additionally selected to highlight the procedure and the healing process.

Results: A total of 18 pediatric patients were included in this study. The group was composed of 11 boys and seven girls, aged from 5 to 123 months. The etiologies included congenital CSF leaks (n = 9) and head trauma (n = 9). Among all patients, 12 fistulas (66.7%) were located at the cribriform plate area, two (11.1%) at the roof of the ethmoid sinuses, two (11.1%) in the sphenoid sinus, and two (11.1%) at the frontal sinus. The maximum diameters of fistulas ranged from 5 to 20 mm, with a median value of 8 mm. Encephaloceles were identified in 14 (77.8%) patients. No hydrocephalus was recognized. All CSF leaks were successfully repaired with a bath-plug technique. Follow-up ranged from 50 to 70 months. No surgical complications were encountered in any patient.

Interpretation: Bath-plug technique is safe and reliable for the endoscopic management of CSF leaks in children. Meticulous peri-operative preparations are important for pediatric patients.

Importance: Patients with Kawasaki disease (KD) experience various extracardiovascular injury complications, which may affect their outcomes.

Objective: To investigate the incidence and clinical characteristics of extracardiovascular complications in children with KD.

Methods: The clinical data of patients diagnosed with KD in the First Affiliated Hospital of Guangxi Medical University from January 2003 to January 2021 were reviewed. The clinical characteristics and extracardiovascular complications were compared among patients stratified by age, intravenous immunoglobulin (IVIG) therapy responsiveness, and coronary status.

Results: A total of 511 patients with KD were included, 357 (69.9%) were aged 1-5 years. Children aged <1 year (21.5%) and boys (70.8%) were more likely to have coronary artery lesions (CALs). The incidence of incomplete KD was lowest in 1-5-year-old patients (19.6%). Involvement of the hematological system gradually decreased with age (<1 year, 51.8%; 1-5 years, 36.7%; >5 years, 29.5%), whereas the involvement of the joints gradually increased with age (<1 year, 2.7%; 1-5 years, 6.2%; >5 years, 20.5%). Nervous system involvement was more common in IVIG non-responders (15.7% [13/83] vs. 5.4% [23/428], P = 0.001). However, there were no significant differences in extracardiovascular injury complications between patients with or without CALs.

Interpretation: KD can involve multiple organ injuries as well as cardiovascular complications, and nervous systerm involvement may be more common in patients unresponsive to IVIG.

Importance: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is considered the only effective treatment for chronic active Epstein-Barr virus infection (CAEBV). The clinical efficacy and safety of allo-HSCT with different conditioning regimens in children with CAEBV remain unclear.

Objective: To evaluate the effectiveness and safety of allo-HSCT with the modified myeloablative conditioning (MAC) regimen for children with CAEBV and also the factors affecting the outcomes.

Methods: We retrospectively analyzed children with CAEBV who underwent allo-HSCT with the modified MAC regimen at Beijing Children's Hospital, Capital Medical University from October 2016 to June 2021. Data related to the clinical manifestations, engraftment, and outcome were extracted from the medical records.

Results: The cohort comprised 41 patients (24 males, 17 females) with a median transplantation age of 92.6 (60.4, 120.7) months and a median follow-up time of 28.2 (15.3, 40.2) months. Four patients (9.8%) died, among which three died from primary disease relapse, and one died from grade IV acute graft-versus-host diseases (aGVHD) after stopping treatment. The 3-year overall survival (OS) and 3-year event-free survival (EFS) rates were 88.8% ± 5.4% and 85.0% ± 5.7%, respectively. The 3-year OS and EFS did not significantly differ between the patients with hemophagocytic lymphohistiocytosis (HLH) and the patient without HLH (87.7% ± 6.8% vs. 91.7% ± 8.0%, P = 0.790; 85.0% ± 6.9% vs. 84.6% ± 10.0%, P = 0.921), or among the patients with complete remission, partial remission, and activity disease before HSCT (all P > 0.05). Multivariate analysis showed that grade III-IV aGVHD was a risk factor for mortality (Hazards ratio: 11.65, 95% confidence interval: 1.00, 136.06; P = 0.050).

Interpretation: Allo-HSCT with the modified MAC regimen is safe and effective for pediatric CAEBV. This treatment benefits patients with HLH or active disease. Patients with Grade III-IV aGVHD may be associated with worse outcomes.

Newborn screening (NBS) is a public health service aimed at identifying infants with severe genetic disorders, thus providing effective treatment early enough to prevent or ameliorate the onset of symptoms. Current NBS uses biochemical analysis of dried blood spots, predominately with time-resolved fluorescence immunoassay and tandem mass spectrometry, which produces some false positives and false negatives. The application of enzymatic activity-based testing technology provides a reliable screening method for some disorders. Genetic testing is now commonly used for secondary or confirmatory testing after a positive result in some NBS programs. Recently, next-generation sequencing (NGS) has emerged as a robust tool that enables large panels of genes to be scanned together rapidly. Rapid advances in NGS emphasize the potential for genomic sequencing to improve NBS programs. However, some challenges still remain and require solution before this is applied for population screening.

Using the US National Inpatient Sample dataset (2010 to 2018), we compared outcomes of neonates with Tetralogy of Fallot who had early primary surgical repair (1726 neonate) and those who had staged palliative intervention with transcatheter (1702 neonate) or surgical palliative shunt (2661 neonate).

Importance: Effective screening strategies for early-onset neonatal sepsis (EONS) have the potential to reduce high volume parenteral antibiotics (PAb) usage in neonates.

Objective: To compare management decisions for EONS, between CG149 National Institute for Health and Care Excellence (NICE) guidelines and those projected through the virtual application of the Kaiser Permanente sepsis risk calculator (SRC) in a level 2 neonatal unit at a district general hospital (DGH).

Methods: Hospital records were reviewed for maternal and neonatal risk factors for EONS, neonatal clinical examination findings, and microbial culture results for all neonates born at ≥34 weeks' gestation between February and July 2019, who were (1) managed according to CG149-NICE guidelines or (2) received PAb within 72 h following birth at a DGH in Winchester, UK. SRC projections were obtained using its virtual risk estimator.

Results: Sixty infants received PAb within the first 72 h of birth during the study period. Of these, 19 (31.7%) met SRC criteria for antibiotics; 20 (33.3%) met the criteria for enhanced observations and none had culture-proven sepsis. Based on SRC projections, neonates with '≥1 NICE clinical indicator and ≥1 risk factor' were most likely to have a sepsis risk score (SRS) >3. Birth below 37 weeks' gestation (risk ratio [RR] = 2.31, 95% confidence interval [CI]: 1.02-5.22) and prolonged rupture of membranes (RR = 3.14, 95% CI: 1.16-8.48) increased the risk of an SRS >3.

Interpretation: Screening for EONS on the SRC could potentially reduce PAb usage by 68% in term and near-term neonates in level 2 neonatal units.