Translational pediatrics最新文献

Background: Resting energy expenditure (REE) refers to the energy consumption of the body in a resting state without skeletal muscle activity. This study aimed to examine the REE among children hospitalized with varying nutritional status.

Methods: This was a retrospective study. We enrolled 109 pediatric cases that underwent indirect calorimetry (IC) and divided into four groups: mild malnutrition group (15 cases), moderate malnutrition group (30 cases), severe malnutrition group (32 cases), and obesity group (32 cases). We compared and analyzed the measured REE (mREE) using IC with the predicted REE (pREE) using five energy equations. The paired t-test was used to compare the results of two samples. Pearson analysis was used to assess the correlation between two values. The agreement analysis was performed using the Bland-Altman method.

Results: There was no significant difference in mREE between the mild, moderate, and severe malnutrition groups, but each differed significantly from the obesity group. All populations exhibited significant correlation between the mREEs and all five energy equations, and the equation with the highest predictive accuracy was the Schofield equation, which achieved an accuracy of 47.7%. In subgroup analysis, there was no significant difference between mREE and pREE for each of the five equations in the mild, moderate malnutrition groups. Only the prediction result of the Liu equation was not significantly different from the mREE in the severe malnutrition group. The prediction accuracy of the Liu equation was relatively the highest (34.4%). However, in the obese group, there were significant differences in pREE and mREE between the Liu equation and Mifflin equation. Under different nutritional statuses, the results of the Bland-Altman analysis suggested that deviation values between REEs predicted by each equation and mREE were greater than ±10%.

Conclusions: There were differences in REE among children with different nutritional status. The results obtained from the five predictive energy equations deviated from the IC results. When REE cannot be measured by IC, it is essential to choose an appropriate predictive energy equation based on the nutritional status of the individual.

Background: Rebound hyperbilirubinemia (HBB) is still present in as high as 10% of newborn babies. However, the applicability of established prediction models for rebound HBB to Chinese newborns is unclear. This study aimed to establish a model to predict HBB rebound after phototherapy among Chinese neonates.

Methods: A retrospective cohort study was conducted on 1,035 HBB infants receiving phototherapy. Rebound HBB was defined as total serum bilirubin (TSB) returning to or above the American Academy of Pediatrics (AAP) phototherapy threshold within 72 hours after the end of phototherapy. The predictive effects of previously published two- and three-variable scores were verified. Neonates were randomly assigned in a 6:4 ratio to the training (n=621) group and the testing (n=414) group. All variables in the training set were used to select predictors by least absolute shrinkage and selection operator (LASSO) regression analysis. The internal validation of the prediction model was performed using the testing set. The model's predictive performance was evaluated by area under the curve (AUC), accuracy, sensitivity, and specificity, each with 95% confidence intervals (CIs). Receiver operating characteristic (ROC) and calibration curves were constructed to evaluate the discrimination ability and fitting effect of the prediction model, respectively.

Results: Rebound HBB was observed in 210 patients (20.3%). The AUC for the two- and three-variable scores were 0.498 (95% CI: 0.455-0.540) and 0.498 (95% CI: 0.457-0.540), respectively. Predictive factors for the risk of rebound HBB included formula feeding (>3 times/day), standard phototherapy irradiation time, TSB levels and age at termination of phototherapy, neonatal weight, and differences between TSB levels at the phototherapy termination and phototherapy threshold. The prediction model's AUC was 0.935 (95% CI: 0.911-0.958), the sensitivity was 0.880 (95% CI: 0.809-0.950), the specificity was 0.831 (95% CI: 0.790-0.871), and the accuracy was 0.841 (95% CI: 0.805-0.876).

Conclusions: The established model performed well in predicting rebound risk among Chinese infants with HBB, which may be beneficial in treating and managing HBB in infants.

Background: Diarrhea is the leading contributory factor of sickness and mortality among children under five and an economic burden for families. This study aimed to investigate the effects of mixed probiotics supplementation at different times (consecutive and alternate-hour) on intestinal microecology in Sprague-Dawley (SD) rats with acute diarrhea.

Methods: A total of 40 SD rats were randomly assigned to four groups, including the control group, model group, probiotic group A, and probiotic group B. An acute diarrhea model was induced by administration of 5% dextran sulfate sodium. Rats in probiotic group A and probiotic group B were fed with Clostridium butyricum (C. butyricum), Bifidobacterium infantis (B. infantis), and Saccharomyces boulardii (S. boulardii) for a total of 7 days. Probiotic group A was fed with all probiotics simultaneously. Probiotic group B was fed with C. butyricum and B. infantis simultaneously, and then after a 2-hour interval, with S. boulardii. Metagenomic next-generation sequencing was used to analyze the fecal samples from every rat. The metagenomic sequencing used in this experiment was used to evaluate the effect of probiotics on the composition as well as function of the gut microbiota in order to gain a deeper comprehension of probiotic-host interactions on health and disease.

Results: The structure of the gut microbiota in probiotic group A showed significant changes. Compared to the model group, the abundance of some beneficial bacteria had increased, including Actinobacteria (P=0.048), Lactobacillus (P=0.050), and Lactobacillus johnsonii (P=0.042), and many opportunistic pathogenic bacteria has decreased, such as Ruminococcus (P=0.001). Compared to the control group, the abundance of some beneficial bacteria had increased, including Fusobacteria (P=0.02) and Phascolarium (P=0.002), and there was a reduction in the abundance of many opportunistic pathogenic bacteria such as Roseburia (P=0.03), Lachnoclosterium (P=0.009), and Oscillibacter_sp_1-3 (P=0.002). In addition, metagenomic analysis showed that as well as an up-regulation of glycoside hydrolase expression, amino acid and inorganic ion transport, and metabolism-related pathways, there was a down-regulation of cell motility.

Conclusions: Simultaneous administration of probiotics may have more positive implications in improving the gut microbiota of acute diarrhea rats.

Background: Previously, we developed the Guidelines for the Management of Pediatric Off-Label Use of Drugs in China in addressing the need for standardization of pediatric off-label drug use. As the implementation of recommendations in Guidelines among healthcare professionals is weak, it is important to identify barriers to guideline implementation for developing appropriate strategies for specific settings and target groups. This study aimed to assess the difficulty and urgency in implementing the recommendations in the Guideline, identifying the factors affecting the implementation of these recommendations to realize the clinical translation of the Guideline.

Methods: A cross-sectional study was conducted from March 1 to June 17, 2022. Pediatricians, pharmacists, and health managers from all 31 mainland Chinese provinces were involved. The electronic questionnaires were distributed nationwide by The Clinical Pharmacology Group of the Pediatric Society of the Chinese Medical Association and the National Clinical Research Center for Child Health. Data analysis, including frequency, percentages, averages, and standard deviations was performed using Microsoft Excel 16.54. Chi-squared tests, multi-factor logistic regression, and linear regression were analyzed in SPSS 23.0. A Sankey diagram was constructed using R software.

Results: A total of 869 valid questionnaires were collected from 491 participating organizations. More than half of the recommendations were implemented, and 12 recommendations were implemented more in tertiary hospitals than in secondary hospitals. The mean urgency scores of all 21 recommendations were over 5. The mean difficulty scores of all 21 recommendations were over 4. The percentage of the most urgent was 44.33%, and the least urgent was 1.45%. The most difficult portion was 12.03%, and the least difficult was 5.74%. Factors impacting the urgency and difficulty of guideline implementation were different, with common influences including the position, education level of clinicians and hospital level.

Conclusions: The recommendations in the Guideline for the Management of Pediatric Off-Label Use of Drugs are considered highly urgent for implementation in China. Nevertheless, the study revealed challenges in applying all 21 recommendations within clinical practice. The key factors affecting implementation include the position, education, experience, and hospital level of healthcare professionals. It is recommended to facilitate implementing the recommendations by sharing experience across various hospital levels, starting from high-level hospitals and extending to primary healthcare settings. Moreover, adjustments to the professional structure within hospitals are needed to enhance the management of off-label drug use in pediatric patients.

Background: Adenoid hypertrophy is a prevalent cause of upper airway obstruction in children, potentially leading to various otolaryngological complications and even systemic sequelae. The lateral nasopharyngeal radiograph is routinely employed for the diagnosis of adenoid hypertrophy. This study aimed to evaluate the accuracy and reliability of deep learning, using lateral nasopharyngeal radiographs, for the diagnosis of adenoid hypertrophy in pediatric patients.

Methods: In the retrospective study, the lateral nasopharyngeal X-ray images were collected from children receiving therapy in the Children's Hospital of Soochow University, the 983th Hospital of Joint Logistics Support Forces of Chinese PLA and the Suzhou Wujiang District Children's Hospital from January 2023 to November 2023. Five deep learning models, i.e., AlexNet, VGG16, Inception v3, ResNet50 and DenseNet121, were used for model training and validation. Receiver operating characteristic (ROC) curve analyses were used to evaluate the performance of each model. The best algorithm was compared with interpretations from three radiologists on 208 images in the internal validation group.

Results: The lateral nasopharyngeal X-ray images were collected from 1,188 children, including 705 males (59.3%) and 483 females (40.7%), aged 8 months to 13 years, with a mean age of 5.57±2.66 years. Among the five deep learning models, DenseNet-121 performed the best, with area under the curve (AUC) values of 0.892 and 0.872, with accuracy of 0.895 and 0.878, sensitivity of 0.870 and 0.838, and specificity of 0.913 and 0.906 in the internal and external validation groups, respectively. The diagnostic performance of DenseNet-121 was higher than that of the junior and mid-level radiologists (0.892 vs. 0.836, 0.892 vs. 0.869), close to the senior radiologist (0.892 vs. 0.901). However, Delong's test revealed no significant difference between DenseNet121 and each radiologist in the validation group (P=0.24, P=0.52, P=0.79).

Conclusions: All the five deep learning models in the study showed good performance for the diagnosis of adenoid hypertrophy, with DenseNet121 being the best, which was clinically relevant for the automatic identification of adenoid hypertrophy.

Background: Microvascular free-tissue transfer is a widely used technique for surgical site reconstruction following head and neck mass resection. While it is commonly used in adults, the rarity of head and neck cancers in children makes free flap reconstruction relatively rare in this population. Free flap reconstruction allows for coverage of large defects which may result from wide resections performed to avoid exposing pediatric patients to primary radiotherapy.

Case description: We present two pediatric oncologic cases using free flap reconstruction. The first, a 4-year-old male, presented with a rapidly enlarging tongue lesion that was diagnosed as a biphasic synovial sarcoma. The mass was resected, and a radial forearm free flap was placed. The second, a 9-year-old male, presented with jaw pain and a mandibular mass. Biopsy confirmed a diagnosis of Ewing sarcoma. The patient was treated initially with chemotherapy, followed by surgical resection and placement of an osteocutaneous fibular free flap. Both patients are disease-free 5 years post-operation.

Conclusions: In each patient, complete resection of the primary tumor was performed eliminating the need for radiotherapy with utilization of free flap reconstruction. As advances in medicine enable better evaluation of clear margins resulting in larger resection defects, free tissue transfer provides a useful reconstructive option in the pediatric population.

Background: Primary mediastinal choriocarcinoma (PCC) is a rare, highly vascular invasive, and prognostically unfavorable malignant tumor. When occurring outside the gonads, primary choriocarcinoma is commonly found in midline locations such as the mediastinum or retroperitoneum. Currently, there is no standardized treatment strategy for PCC. In the case reported herein, we employed tislelizumab and chemotherapy in the treatment of a patient with PCC, and as in March 2024, the patient remained survive.

Case description: A 15-year-old boy who presented with symptoms of fever and cough for a year. Chest computed tomography (CT) scan showed a relatively large soft tissue shadow in the right upper anterior mediastinum, measuring approximately 5.4 cm × 3.8 cm. The patient's soft tissue exhibited unclear demarcation from surrounding mediastinal structures and was accompanied by lung metastasis. The patient underwent a fine needle aspiration biopsy for a mediastinal mass, and the pathology results indicated a germ cell tumor with solid malignant components in the mediastinum, along with pulmonary metastasis of the solid malignancy. The patient's serum levels of beta-human chorionic gonadotropin (β-HCG) were elevated at 125,554 mIU/mL (normal range: <5 mIU/mL), and alpha-fetoprotein (AFP) was 75.8 ng/mL (normal range, 0.605-7 ng/mL). The patient's cranial magnetic resonance imaging (MRI) plain scan indicated multiple scattered abnormal signals in both cerebral hemispheres. Subsequently, the patient was transferred to Children's Hospital of Nanjing Medical University for his further treatment. During the treatment period, we employed various therapeutic approaches, including chemotherapy, radiotherapy and tislelizumab therapy. After five cycles of tislelizumab treatment, the patient's symptoms of cerebral edema significantly improved, β-HCG levels decreased. Brain MRI of the patient revealed multiple abnormal signals within the skull, with some lesions showing reduction in size and significant improvement in the surrounding edema zones. The clinical symptoms of the patient improved and he achieved partial remission (PR). At the moment, the patient is living with the disease.

Conclusions: The effectiveness of chemotherapy for PCC is limited. Tislelizumab may potentially serve as salvage treatment options for PCC.