Hepatolenticular degeneration, also known as Wilson's disease, is a type of autosomal recessive genetic disorder of copper metabolism. The causative gene, ATP7B, is located on the long arm of chromosome 13 and encodes a P-type ATPase that is involved in copper transport. Pathogenic mutations in the ATP7B gene sequence lead to the diminished or lost function of the ATP7B protein, resulting in pathological copper deposition in organs such as the liver, brain, kidneys, and cornea. Currently, the treatment of Wilson's disease primarily involves oral medications to promote copper excretion or reduce copper absorption so as to alleviate the state of illness. However, pharmacological treatment has objective limitations, including the need for lifelong therapy and varying degrees of adverse drug reactions in some patients. Gene therapy can fully correct the genetic defect, restore ATP7B protein function, achieve a curative effect, and improve the patient's quality of life.

Objective:To compare the clinical effects of plasma exchange（PE）combined with continuous veno-venous hemodiafiltration（CVVHDF）at different time points in children with Kawasaki disease shock syndrome（KDSS）.Methods:Thirty-five children with KDSS admitted to the intensive care unit of Hunan Children's Hospital from January 2018 to December 2022 were enrolled.According to whether PE combined with CVVHDF was performed within 24 hours after reaching the blood purification criteria for KDSS，the patients were divided into the early treatment group(8 cases) and the control group (27 cases).The clinical and laboratory indicators and prognosis were compared between the two groups.Results:There were no statistically significant differences in age，gender，mean arterial pressure，and pediatric critical illness score between the two groups before treatment（ P>0.05）.In the early treatment group,the vasoactive inotropic score (VIS) gradually decreased,and was significantly lower than that in the control group after 24 hours of treatment.The duration of vasopressor use，pediatric logistic organ dysfunction score（PELOD），inflammatory markers，total hospitalization time，and PICU stay were all lower in the early treatment group than in the control group（ P<0.05）.The incidence of coronary artery involvement within 6 months post-discharge was lower in the early treatment group than in the control group（ P<0.05）.Among the 12 children who underwent PE combined with CVVHDF，four cases were in the late treatment group.The duration of CVVHDF，PICU stay，and PELOD scores were lower in the early treatment group than in the late treatment group（ P<0.05）.Additionally，the concentrations of interleukin-6，tumor necrosis factor-α，heparin-binding protein，and N-terminal pro-brain natriuretic peptide before PE were lower in the early treatment group than in the late treatment group（ P<0.05）. Conclusion:Early PE combined with CVVHDF treatment for KDSS patients can reduce inflammatory response，shorten the course of the disease，and reduce the duration of vasopressor use.However，most patients' conditions can be controlled with active conventional treatment.

Objective:To compare the clinical effects of plasma exchange（PE）combined with continuous veno-venous hemodiafiltration（CVVHDF）at different time points in children with Kawasaki disease shock syndrome（KDSS）.Methods:Thirty-five children with KDSS admitted to the intensive care unit of Hunan Children's Hospital from January 2018 to December 2022 were enrolled.According to whether PE combined with CVVHDF was performed within 24 hours after reaching the blood purification criteria for KDSS，the patients were divided into the early treatment group(8 cases) and the control group (27 cases).The clinical and laboratory indicators and prognosis were compared between the two groups.Results:There were no statistically significant differences in age，gender，mean arterial pressure，and pediatric critical illness score between the two groups before treatment（ P>0.05）.In the early treatment group,the vasoactive inotropic score (VIS) gradually decreased,and was significantly lower than that in the control group after 24 hours of treatment.The duration of vasopressor use，pediatric logistic organ dysfunction score（PELOD），inflammatory markers，total hospitalization time，and PICU stay were all lower in the early treatment group than in the control group（ P<0.05）.The incidence of coronary artery involvement within 6 months post-discharge was lower in the early treatment group than in the control group（ P<0.05）.Among the 12 children who underwent PE combined with CVVHDF，four cases were in the late treatment group.The duration of CVVHDF，PICU stay，and PELOD scores were lower in the early treatment group than in the late treatment group（ P<0.05）.Additionally，the concentrations of interleukin-6，tumor necrosis factor-α，heparin-binding protein，and N-terminal pro-brain natriuretic peptide before PE were lower in the early treatment group than in the late treatment group（ P<0.05）. Conclusion:Early PE combined with CVVHDF treatment for KDSS patients can reduce inflammatory response，shorten the course of the disease，and reduce the duration of vasopressor use.However，most patients' conditions can be controlled with active conventional treatment.

Hepatolenticular degeneration, also known as Wilson's disease, is a type of autosomal recessive genetic disorder of copper metabolism. The causative gene, ATP7B, is located on the long arm of chromosome 13 and encodes a P-type ATPase that is involved in copper transport. Pathogenic mutations in the ATP7B gene sequence lead to the diminished or lost function of the ATP7B protein, resulting in pathological copper deposition in organs such as the liver, brain, kidneys, and cornea. Currently, the treatment of Wilson's disease primarily involves oral medications to promote copper excretion or reduce copper absorption so as to alleviate the state of illness. However, pharmacological treatment has objective limitations, including the need for lifelong therapy and varying degrees of adverse drug reactions in some patients. Gene therapy can fully correct the genetic defect, restore ATP7B protein function, achieve a curative effect, and improve the patient's quality of life.

Objective:To explore the effect of case study combined with scenario simulation in teaching clinical internship of radiation oncology.Methods:This study selected 144 undergraduate students majoring in clinical medicine who interned at the Department of Radiotherapy at Sun Yat-sen University Cancer Center from November 2022 to May 2023 as the research subjects. They were randomly divided into a control group and an observation group. The control group received traditional teaching, while the observation group received case study combined with scenario simulation teaching, with 72 students in each group. The two groups of trainees were evaluated for theoretical and skill assessment scores, core competencies, and teaching satisfaction. The t-test and chi-square test were performed using SPSS 22.0. Results:The theoretical and skill scores of the trainees in the observation group [(88.27±3.40) and (92.69±3.65)] were higher than those in the control group [(83.01±10.38) and (87.20±3.74)], and the differences were statistically significant. Compared with the control group, the observation group showed higher core competency scores in eight dimensions, including learning initiative and enthusiasm, knowledge comprehension ability, critical thinking ability, clinical specialty practice ability, analytical and problem-solving ability, doctor-patient communication, professional ethics, and teamwork ability, and the differences were statistically significant ( P<0.05). The satisfaction scores of the observation group trainees of teaching plan, teaching content, teaching methods, teaching results, and teaching ability were higher than those of the control group, and the differences were statistically significant ( P<0.05). Conclusions:The application of case study combined with scenario simulation teaching clinical internship of radiation oncology can help improve the theoretical and skill scores of medical students, as well as their core competencies and teaching satisfaction. This is a teaching method worthy of application and promotion.

Objective:To construct a risk prediction model for the inter-hospital transfer of critically ill children using machine learning methods, identify key medical features affecting transfer outcomes, and improve the success rate of transfers.Methods:A prospective study was conducted on critically ill children admitted to the pediatric transfer center of Hunan Children's Hospital from January 2020 to January 2021. Medical data on critical care features and relevant data from the Pediatric Risk of Mortality (PRISMⅢ) scoring system were collected and processed. Three machine learning models, including logistic regression, decision tree, and Relief algorithm, were used to construct the risk prediction model. A back propagation neural network was employed to build a referral outcome prediction model to verify and analyze the selected medical features from the risk prediction model, exploring the key medical features influencing inter-hospital transfer risk.Results:Among the 549 transferred children included in the study, 222 were neonates (40.44%) and 327 were non-neonates (59.56%). There were 50 children in-hospital deaths, resulting in a mortality rate of 9.11%. After processing 151 critical care medical feature data points, each model selected the top 15 important features influencing transfer outcomes, with a total of 34 selected features. The decision tree model had an overlap of 72.7% with PRISMⅢ indicators, higher than logistic regression (36.4%) and Relief algorithm (27.3%). The training prediction accuracy of the decision tree model was 0.94, higher than the accuracy of 0.90 when including all features, indicating its clinical utility. Among the top 15 important features selected by the decision tree model, the impact on transfer outcomes was ranked as follows based on quantitative feature violin plots: base excess, total bilirubin, ionized calcium, total time, arterial oxygen pressure, blood parameters (including white blood cells, platelets, prothrombin time/activated partial thromboplastin time), carbon dioxide pressure, blood glucose, systolic blood pressure, heart rate, organ failure, lactate, capillary refill time, temperature, and cyanosis. Eight of these important features overlapped with PRISMⅢ indicators, including systolic blood pressure, heart rate, temperature, pupillary reflex, consciousness, acidosis, arterial oxygen pressure, carbon dioxide pressure, blood parameters, and blood glucose. The decision tree was used to select the top 15 medical features with high impact on the neonatal and non-neonatal datasets, respectively. A total of 19 features were selected, among which there were 8 differences and 11 overlap terms between the important features of the neonatal and non-neonatal.Conclusions:Machine learning models could serve as reliable tools for predicting the risk of inter-hospital transfer of critically ill children. The decision tree model exhibits superior performance and helps identify key medical features affecting inter-hospital transfer risk, thereby improving the success rate of inter-hospital transfers for critically ill children.

Objective:To investigate the value of the ratio of heparin binding protein (HBP) to albumin (ALB) in diagnosis and predict the severity of Kawasaki disease shock syndrome (KDSS).Methods:This study was a retrospective study. Pediatric patients with Kawasaki disease (KD) admitted to the Children's Intensive Care Unit and department of Pediatric Emergency Center of Hunan Children's Hospital from January 2019 to May 2022 were enrolled. The HBP/ALB ratio was calculated according to HBP and serum ALB. The children were divided into three groups (low, medium, and high ratio groups) according to the median and upper and lower quartiles of the HBP/ALB ratio. The differences of each index among the three groups were compared. The receiver operating characteristic curves were drawn to evaluate the clinical value of the HBP/ALB ratio in diagnosis of KDSS and the severity of the disease.Results:A total of 111 cases were included in this study, including 28 cases in the low ratio group, 56 cases in the medium ratio group, and 27 cases in the high ratio group. There were 24 cases with coronary artery damage, 87 cases without coronary artery damage, 27 cases with abnormal ECG findings, and 17 children with KDSS (including 5 cases in the medium ratio group, and 12 cases in the high ratio group). The incidence of KDSS, coronary involvement, and abnormal electrocardiogram proportions in the high ratio group were significantly higher than those in the other two groups. Compared with low and medium ratio groups, the levels of cardiac troponin I, N-terminal pro-brain natriuretic peptide, lactate, stroke output variation, trends in thoracic fluid content, white blood cell count, C-reactive protein, procalcitonin, and D-dimer levels were higher in the high ratio group, while ALB and blood sodium levels were lower in the high ratio group (all P<0.05). There was no significant difference in above indicators between the low and medium ratio groups (all P>0.05). The HBP/ALB ratio had a higher area under the curve, sensitivity, and specificity (0.942, 0.882, and 0.883, respectively) in predicting KDSS compared to HBP alone (0.776, 0.842, and 0.670, respectively). Conclusion:The HBP/ALB ratio could reflect the severity of children with KD and has certain clinical value for prognostic evaluation.

Objective:To explore the effect of case study combined with scenario simulation in teaching clinical internship of radiation oncology.Methods:This study selected 144 undergraduate students majoring in clinical medicine who interned at the Department of Radiotherapy at Sun Yat-sen University Cancer Center from November 2022 to May 2023 as the research subjects. They were randomly divided into a control group and an observation group. The control group received traditional teaching, while the observation group received case study combined with scenario simulation teaching, with 72 students in each group. The two groups of trainees were evaluated for theoretical and skill assessment scores, core competencies, and teaching satisfaction. The t-test and chi-square test were performed using SPSS 22.0. Results:The theoretical and skill scores of the trainees in the observation group [(88.27±3.40) and (92.69±3.65)] were higher than those in the control group [(83.01±10.38) and (87.20±3.74)], and the differences were statistically significant. Compared with the control group, the observation group showed higher core competency scores in eight dimensions, including learning initiative and enthusiasm, knowledge comprehension ability, critical thinking ability, clinical specialty practice ability, analytical and problem-solving ability, doctor-patient communication, professional ethics, and teamwork ability, and the differences were statistically significant ( P<0.05). The satisfaction scores of the observation group trainees of teaching plan, teaching content, teaching methods, teaching results, and teaching ability were higher than those of the control group, and the differences were statistically significant ( P<0.05). Conclusions:The application of case study combined with scenario simulation teaching clinical internship of radiation oncology can help improve the theoretical and skill scores of medical students, as well as their core competencies and teaching satisfaction. This is a teaching method worthy of application and promotion.

Objective:To explore the clinical value of serum insulin combined with cardiac-related markers in evaluating the severity of sepsis associated encephalopathy (SAE).Methods:The clinical data of 130 children with sepsis who admitted to the Pediatric Intensive Care Unit of Hunan Children's Hospital from January 2018 to December 2021 were analyzed retrospectively, and the differences of serum insulin and cardiac-related markers in children with sepsis and SAE were compared.Results:The levels of serum insulin, creatine kinase isoenzyme, hypersensitive troponin T, and N-terminal cerebral urine peptide in the SAE group were significantly higher than those in the non-SAE group ( P<0.05), but there was no significant difference in heart rate and lactic acid ( P>0.05). The levels of serum insulin, creatine kinase isoenzyme, hypersensitive troponin T, N-terminal cerebral urine peptide and lactic acid in the death group were significantly higher than those in the survival group ( P<0.05), while the heart rate was not significantly different ( P>0.05). The area under ROC curve of serum insulin, creatine kinase isoenzyme, hypersensitive troponin T, and N-terminal cerebral urine peptide in predicting SAE were 0.841, 0.599, 0.700, and 0.667, respectively; in terms of judging the prognosis of sepsis, the area under ROC curve were 0.647, 0.669, 0.645, and 0.683, respectively; and in terms of judging the prognosis of children with SAE, the areas under the ROC curve were 0.509, 0.682, 0.666 and 0.555, respectively. Binary logistic regression equation was established with serum insulin, creatine kinase isoenzyme, hypersensitive troponin T, and N-terminal cerebral urine peptide: Y=8.153×NT-proBNP+1.704×CTnT-hs+27.121×insulin+0.946×CK-MB+1.573. The area under the ROC curve of the new variable Y in predicting sepsis SAE, evaluating the prognosis of sepsis, and predicting the prognosis of children with sepsis and SAE was 0.890, 0.756, and 0.729, respectively. Conclusions:Serum insulin, creatine kinase isoenzyme, hypersensitive troponin T, and N-terminal cerebral urine peptide can be used alone to determine the severity of sepsis and sepsis in children with SAE. The combined value of the four indicators is obviously better than that of the single indicator. The combined application of the four indicators may better evaluate the severity of sepsis and SAE.

Objective To investigate the efficacy and application of bronchoalveolar lavage in chil-dren with severe pneumonia undergoing mechanical ventilation. Methods Using a prospective randomized controlled clinical study, 202 children with severe pneumonia received mechanical ventilation in Hunan Children′s Hospital from January 2016 to January 2018 were selected as the subjects. According to the digital method,all cases were divided into treatment group (101 cases) and control group (101 cases) randomly. The patients in the control group were given conventional treatment ( anti-infection and symptomatic thera-py) . The treatment group was treated with bronchoalveolar lavage on the basis of conventional treatment. The basic situation,the respiratory function before and after the treatment,the inflammation index,the curative effect and the prognosis of two groups were analyzed. Results There were no significant differences between the two groups in gender,age,course pre-admission,pediatric critical illness score,respiratory function and in-flammation index ( P>0. 05 ) . The respiratory function indexes of the treatment group were obviously im-proved 2 hours after the treatment and the PaO2 ,PaO2/FiO2 and SaO2 were significantly higher than those of the control group[PaO2:(82. 4 ± 6. 4) mmHg(1 mmHg=0. 133 kPa) vs. (74. 0 ± 5. 5) mmHg, PaO2/FiO2:(360. 2 ± 21. 3) mmHg vs. (332. 6 ± 23. 5) mmHg,SaO2:(94. 9 ± 8. 2)％ vs. (88. 6 ± 10. 3)％], while the PaCO2 were significantly lower than the control group [ ( 37. 3 ± 10. 3 ) mmHg vs. ( 45. 8 ± 5. 5 ) mmHg],and the differences were statistically significant (P<0. 05). Five days after treatment,the WBC, PCT and CRP of treatment group were significantly lower than those in the control group[WBC:(8. 5 ± 2. 4) × 109/L vs. (11. 7 ± 3. 5) × 109/L,PCT:(1. 2 ± 0. 7) μg/L vs. (2. 3 ± 0. 9) μg/L,CRP:(9. 1 ± 3. 2) mg/L vs. (16. 5 ± 4. 7) mg/L,P<0. 05,respectively]. The total effective rate in the treatment group was significantly higher than that in the control group[93. 1％(94/101)vs. 81. 2％(82/101)]. Mechanical venti-lation duration and PICU stay in treatment group were significantly shorter than those in the control group [(148. 5 ±30. 6)h vs. (159. 6 ±47. 3)h,(220. 8 ±49. 7)h vs. (330. 7 ±94. 6)h]. The positive rate of patho-genic bacteria was significantly higher than that in the control group [79. 2％(80/101)vs. 62. 4％(63/101), P<0. 05],but there was no significant difference in the 28 days mortality of the two groups[5. 0％(5/101) vs. 5. 9％(6/101),P>0. 05]. Conclusion The bronchoalveolar lavage can improve the respiratory func-tion,reduce the inflammatory reaction,shorten mechanical ventilation duration and PICU stay in children with severe pneumonia undergoing mechanical ventilation obviously. It is worth popularizing in the PICU because of the improvement of curative effect in these children.