Glycogen storage disease type Ⅱ (GSDⅡ) is a rare autosomal recessive disorder.Infant onset of GSDⅡ usually accompanies progressive cardiac hypertrophy and muscle weakness, and eventually dies of cardiopulmonary failure.GSDⅡ is mainly screened and diagnosed by enzymatic and genetic tests.Enzyme replacement therapy (ERT) is the only currently approved treatment of GSDⅡ, which can effectively improve the function of the affected organs and the survival.Gene therapy and substrate reduction therapy for GSDⅡ are also undergoing basic or clinical research.This review summarizes the current research status of the diagnosis and treatment of GSDⅡ at home and abroad, focusing on the influencing factors for the efficacy of specific treatment (especially ERT), dosing regimen, and ways to improve the efficacy.

Objective:To investigate the dynamic trend of platelet（PLT）count and mean platelet volume（MPV）in children with severe community-acquired pneumonia（SCAP）in PICU and their correlation with prognosis.Methods:A retrospective study was conducted in 215 SCAP children who were admitted to the PICU of Beijing Children's Hospital Affiliated to Capital Medical University from January 2016 to December 2019.According to the disease outcome，the patients were divided into improvement group ( n=184) and unrecovered group ( n=31).The changes of PLT count and MPV at admission，on the 2nd，3rd，and 7th days of hospitalization and before discharge were observed，and the relationship between changes in PLT parameters and poor prognosis was analyzed. Meanwhile，the correlation between thrombocytopenia on admission and on the 7th day of hospitalization and prognosis was further explored. Results:The PLT count of improvement group at admission，on the 2nd，3rd，and 7th days of hospitalization and at discharge[(328±159, 329±137, 362±159, 439±168, 510±171)×10 9/L] were significantly higher than those of unrecovered group [(210±142, 207±152, 267±143, 260±162, 343±159)×10 9/L]（ P＜0.05）.Although the MPV of improvement group [(10.9±1.9)fL] on admission was significantly lower than that of the unrecovered group[(12.7±2.5) fL]（ P＜0.05），there was no significant difference in MPV between two groups on the 2nd，3rd，7th days of hospitalization and discharge（ P＞0.05）.In addition，compared with the admission，children in improvement group had significantly higher PLT count on the 7th day of hospitalization and before discharge（ P＜0.05），but there was no significant change in unrecovered group（ P＞0.05）.Compared with SCAP patients with thrombocytopenia at admission (PLT＜100×10 9/L)( n=22),those with thrombocytopenia on 7th day of hospitalization had a significant higher rate of non recovery( P＜0.05). Conclusion:The occurrence of thrombocytopenia on admission and after 7 days of hospitalization in children with SCAP is associated with poor prognosis.No significant increase or decrease in PLT count after 7 days of hospitalization is often indicative of poor prognosis.Dynamic monitoring of PLT parameter changes may help to better judge the prognosis of severe pneumonia.

Objective:To summarize and analyze the clinical characteristics, treatment and prognosis of glycogen storage disease type Ⅱ(GSD Ⅱ) patients admitted to pediatric intensive care units(PICU), and to improve the pediatricians' understanding of children with severe GSD Ⅱ.Methods:Children with GSD Ⅱ admitted to PICU at Beijing Children's Hospital of Capital Medical University between January 2010 and December 2021 were included. Patient's data were collected through the electronic medical record system.After the patient was discharged,telephone follow-ups were conducted regularly for over a year.Results:A total of eight patients with a median age of 30.5 months were included. There were four patients with infantile GSD Ⅱ, whose median age of onset was 5.5 months. There were four patients with late-onset GSD Ⅱ, whose median age of onset was 36.0 months. Eight patients required continuous noninvasive/invasive respiratory support. Three patients with infantile GSD Ⅱ required respiratory support within one month of onset, and three patients with late onset GSD Ⅱ required respiratory support within one year of onset. A total of six patients had cardiac arrest during the course of the disease. One patient was regularly treated with enzyme replacement therapy during hospitalization but his condition did not improve significantly. Three patients were discharged following medical advice,including one patient continuing noninvasive respiratory support after discharge, and two patients requiring onging invasive respiratory support.A total of four children died,including one being an in-hospital death,and three occuring within one year after hospital discharge. A total of 14 genotypes were detected in eight patients, of which three were newly discovered gene mutations.Conclusion:The children with GSD Ⅱ admitted to PICU have severe respiratory dysfunction and need continuous respiratory support during the early stage of the disease. The incidence of cardiopulmonary arrest caused by infection and respiratory muscle weakness is high. It is recommended to closely monitor the lung function and cardiac function of such children, and actively give the prevention and treatment of infectious diseases. Whether enzyme replacement therapy can benefit patients with severe GSD Ⅱ and whether the newly identified mutations correlate with disease severity needs to be further evaluated.

Objective:To summarize the clinical characteristics of suicidal behavior in children and provide a reference for further developing strategies for preventing childhood suicide.Methods:The medical records of children hospitalized in the pediatric intensive care unit（PICU）at Beijing Children's Hospital for suicidal behavior in the electronic medical record system from January 1,2018,to December 31,2022 were retrospectively selected.Clinical data of children with suicidal behavior were collected.Results:A total of 3 249 patients were admitted to the PICU,including 62 suicide patients.There were 20(32.3%) males,and 42(67.7%) females,with a male-to-female ratio of 1:2.1.The average age was (13.1 ± 1.5) years old,with a minimum age of 7.9 years and a maximum age of 15.7 years.There were 17 (27.4%) children with previously diagnosed mental illnesses.Notably 58.1% children had triggering factors for suicidal behavior.Analysis of suicide methods,53(85.5%) cases were intentional drug ingestion,six(9.7%) cases were jumping from height,and three(4.8%) cases were hanging.Additionally 26.4% cases of poisoning were male,while 66.7% of cases of jumping from height and hanging were male.There was a statistical difference in suicide methods between genders (χ 2=5.704, P=0.025).The poisoning ingestions were classified as 20(37.7%) cases of toxins; 14(26.4%) cases of antidepressants; 10(18.9%) cases of over-the-counter drugs; five(9.4%) cases of other prescription drugs; three(5.7%) cases of sedative drugs; one(1.9%) case of daily chemical.Sources of ingestions: 18(34.0%) cases were purchased personally (pharmacies or online shopping); 16(30.2%) cases were storing medicines at home; 13(24.5%) cases were daily taking antidepressants.Compared with non-poisoning patients,poisoning patients had a significantly shorter PICU duration (Z=-2.884, P=0.004).The total mortality rate of children admitted to PICU due to suicide was 16.1%(10/62).There was a statistical difference in the mortality rate among different suicide methods (χ 2=7.883, P=0.019). Conclusion:School aged children and boys are more likely to choose impulsive and more harmful suicide behaviors such as jumping from heights and hanging,and adolescent girls are more likely to choose intentional poisoning as their suicide method.Attention should be paid to the suicide risk of different age and gender groups.Intentional drug ingestion is the main method of suicide in children.Herbicide poisoning and antidepressant drug poisoning are the leading causes of suicide death.The death risk of suicidal behavior in children may be reduced by controlling the way of getting pesticides (especially herbicides) and obtaining maximum doses of antidepressants.

Objective:To investigate respiratory virus infection in children with septic shock in pediatric care units (PICU) in China and its influence on clinical outcomes.Methods:The clinical data of children with septic shock in children′s PICU from January 2018 to December 2019 in 10 Chinese hospitals were retrospectively collected. They were divided into the pre-COVID-19 and post-COVID-19 groups according to the onset of disease, and the characteristics and composition of respiratory virus in the 2 groups were compared. Matching age, malignant underlying diseases, bacteria, fungi and other viruses, a new database was generated using 1∶1 propensity score matching method. The children were divided into the respiratory virus group and non-respiratory virus group according to the presence or absence of respiratory virus infection; their clinical characteristics, diagnosis, and treatment were compared by t-test, rank sum test and Chi-square test. The correlation between respiratory virus infection and the clinical outcomes was analyzed by logistic regression. Results:A total of 1 247 children with septic shock were included in the study, of them 748 were male; the age was 37 (11, 105) months. In the pre-and post-COVID-19 groups, there were 530 and 717 cases of septic shock, respectively; the positive rate of respiratory virus was 14.9% (79 cases) and 9.8% (70 cases); the seasonal distribution of septic shock was 28.9% (153/530) and 25.9% (185/717) in autumn, and 30.3% (161/530) and 28.3% (203/717) in winter, respectively, and the corresponding positive rates of respiratory viruses were 19.6% (30/153) and 15.7% (29/185) in autumn, and 21.1% (34/161) and 15.3% (31/203) in winter, respectively. The positive rates of influenza virus and adenovirus in the post-COVID-19 group were lower than those in the pre-COVID-19 group (2.1% (15/717) vs. 7.5% (40/530), and 0.7% (5/717) vs. 3.2% (17/530), χ2=21.51 and 11.08, respectively; all P<0.05). Rhinovirus virus were higher than those in the pre-Covid-19 group (1.7% (12/717) vs. 0.2% (1/530), χ2=6.51, P=0.011). After propensity score matching, there were 147 cases in both the respiratory virus group and the non-respiratory virus group. Rate of respiratory failure, acute respiratory distress, rate of disseminated coagulation dysfunction, and immunoglobulin usage of the respiratory virus group were higher than those of non-respiratory virus group (77.6% (114/147) vs. 59.2% (87/147), 17.7% (26/147) vs. 4.1% (6/147), 15.6% (25/147) vs. 4.1% (7/147), and 35.4% (52/147) vs. 21.4% (32/147); χ2=11.07, 14.02, 11.06 and 6.67, all P<0.05); and PICU hospitalization of the former was longer than that of the later (7 (3, 16) vs. 3 (1, 7)d, Z=5.01, P<0.001). Univariate logistic regression analysis showed that the presence of respiratory viral infection was associated with respiratory failure, disseminated coagulation dysfunction, the use of mechanical ventilation, and the use of immunoglobulin and anti-respiratory viral drugs ( OR=2.42, 0.22, 0.25, 0.56 and 1.12, all P<0.05). Conclusions:The composition of respiratory virus infection in children with septic shock is different between pre and post-COVID-19. Respiratory viral infection is associated with organ dysfunction in children with septic shock. Decreasing respiratory viral infection through respiratory protection may improve the clinical outcome of these children.

Objective:To summarize the clinical characteristics and prognosis of severe infant botulism and evaluate the therapeutic effect of botulinum antitoxin in the pediatric intensive care unit (PICU).Methods:The clinical data of 8 cases diagnosed with infantile botulism were retrospectively analyzed in the PICU of Beijing Children′s Hospital from October 2019 to August 2023. Data of basic demographic information, clinical manifestations, laboratory tests, treatment and prognosis of each child were collected and analyzed using descriptive statistical methods.Results:Eight laboratory-confirmed cases of infant botulism were included in this study, all of which were male infants with an age of 6.0 (3.3,6.8) months. Three of the children were from Inner Mongolia Autonomous Region, 2 of them were from Hebei, and the other 3 were from Beijing, Shandong and Xinjiang Uyghur Autonomous Region, respectively. All the patients were previously healthy. In 4 of these cases, the possible cause was the ingestion of either honey and its products or sealed pickled food by the mother or child before the onset of the disease. The first symptom was poor milk intake (4 cases), followed by shallow shortness of breath (7 cases), limb weakness (7 cases) and so on. The typical signs were bilateral dilated pupils (8 cases) and decreased limb muscle strength (8 cases). The main subtype was type B (7 cases), and only 1 case was classified as type A. Six of the children were treated with antitoxin therapy for a duration of 24 (19, 49) d. Seven of them had invasive mechanical ventilation. All the patients survived upon discharge with a follow-up period of 29 d to 3 years and 8 months. Six patients had fully recovered, and 2 recently discharged patients were gradually recovering.Conclusions:For infants with suspected contact or ingestion of botulinum and presented with bilateral pupillary paralysis, muscle weakness and clear consciousness, the stool should be collected for diagnostic testing using a mouse bioassay as soon as possible. Type B was the most common type. The antitoxin treatment was effectiveness and the prognosis was well.

Objective:To analyze the diagnosis and treatment of children with rare diseases in the pediatric intensive care unit (PICU), the distribution of disease types and populations, clinical characteristics, and the use of orphan drugs.Methods:A retrospective case summary was conducted. Data were collected from 105 children aged 29 days to <18 years with a confirmed diagnosis of rare diseases according to the "First Batch of Rare Disease Catalogue in China" who were admitted to the PICU of Beijing Children′s Hospital, Capital Medical University from January 2020 to December 2022. Data including general information, auxiliary examinations, and treatment details for each patient were collected from the hospital′s electronic medical record system. Patients were divided into age groups: infancy (29 days to<1 year), early childhood (1 to <3 years), preschool age (3 to<7 years), school age (7 to<13 years), and adolescence (13 to<18 years) . The chi-square test was used to compare gender distribution differences among various rare diseases. Results:A total of 105 patients with 130 cases meeting the diagnostic criteria were included, accounting for 4.7% (130/2 754) of the total admissions to the PICU. The age at PICU admission was 5.3 (0.8, 9.5) years and there were 81 cases in male. The 3 most common types of diseases were endocrine, nutritional, and metabolic diseases (37 cases); followed by neurological disorders(32 cases); and congenital malformations, deformities, and chromosomal abnormalities(17 cases). The 5 most common rare diseases were methylmalonic acidemia (14 cases), mitochondrial encephalomyopathy (14 cases), atypical hemolytic uremic syndrome (12 cases), autoimmune encephalitis (12 cases), and idiopathic cardiomyopathy (9 cases). The distributions of common rare diseases varied among different age groups. In infants, atypical hemolytic uremic syndrome was most common (6 children). There was no statistically significant difference regarding gender among children with mitochondrial encephalomyopathy (13.6% (11/81) vs. 6.1% (3/49), χ2=1.77, P=0.184). Respiratory failure (36 cases) was the primary reason for rare diseases children to be admitted to the PICU. A total of 95 cases underwent mechanical ventilation, 39 cases received multidisciplinary collaborative diagnosis and treatment, and only 6 children received orphan drug therapy during their stay in the PICU. Conclusions:Rare diseases are not uncommon in PICU. Endocrine, nutritional and metabolic disorders, neurological disorders, congenital malformations, deformities, and chromosomal abnormalities were common. Methylmalonic acidemia, mitochondrial encephalomyopathy, atypical hemolytic uremic syndrome and autoimmune encephalitis have higher cases. Many children with rare diseases in the PICU have complex conditions those are challenging to treat, requiring multidisciplinary collaboration. The utilization rate of orphan drugs among children with rare diseases in PICU needs to be improved.

Objective:To investigate the clinical features, pathogen composition, and prognosis of septic shock in pediatric intensive care units (PICU) in China.Methods:A multicenter retrospective cohort study. A retrospective analysis was conducted on the clinical data of children with septic shock from 10 hospitals in China between January 2018 and December 2021. The clinical features, pathogen composition, and outcomes were collected. Patients were categorized into malignant tumor and non-malignant tumor groups, as well as survival and mortality groups. T test, Mann Whitney U test or Chi square test were used respectively for comparing clinical characteristics and prognosis between 2 groups. Multiple Logistic regression was used to identify risk factors for mortality. Results:A total of 1 247 children with septic shock were included, with 748 males (59.9%) and the age of 3.1 (0.9, 8.8) years. The in-patient mortality rate was 23.2% (289 cases). The overall pathogen positive rate was 68.2% (851 cases), with 1 229 pathogens identified. Bacterial accounted for 61.4% (754 strains) and virus for 24.8% (305 strains). Among all bacterium, Gram negative bacteria constituted 64.2% (484 strains), with Pseudomonas aeruginosa and Enterobacter being the most common; Gram positive bacteria comprised 35.8% (270 strains), primarily Streptococcus and Staphylococcus species. Influenza virus (86 strains (28.2%)), Epstein-Barr virus (53 strains (17.4%)), and respiratory syncytial virus (46 strains (17.1%)) were the top three viruses. Children with malignant tumors were older and had higher pediatric risk of mortality (PRISM) Ⅲ score, paediatric sequential organ failure assessment (pSOFA) score (7.9 (4.3, 11.8) vs. 2.3 (0.8, 7.5) years old, 22 (16, 26) vs. 16 (10, 24) points, 10 (5, 14) vs. 8 (4, 12) points, Z=11.32, 0.87, 4.00, all P<0.05), and higher pathogen positive rate, and in-hospital mortality (77.7% (240/309) vs. 65.1% (611/938), 29.7% (92/309) vs. 21.0% (197/938), χ2=16.84, 10.04, both P<0.05) compared to the non-tumor group. In the death group, the score of PRISM Ⅲ, pSOFA (16 (22, 29) vs. 14 (10, 20) points, 8 (12, 15) vs. 6 (3, 9) points, Z=4.92, 11.88, both P<0.05) were all higher, and presence of neoplastic disease, positive rate of pathogen and proportion of invasive mechanical ventilation in death group were also all higher than those in survival group (29.7% (87/289) vs. 23.2% (222/958), 77.8% (225/289) vs. 65.4% (626/958), 73.7% (213/289) vs. 50.6% (485/958), χ2=5.72, 16.03, 49.98, all P<0.05). Multiple Logistic regression showed that PRISM Ⅲ, pSOFA, and malignant tumor were the independent risk factors for mortality ( OR=1.04, 1.09, 0.67, 95% CI 1.01-1.05, 1.04-1.12, 0.47-0.94, all P<0.05). Conclusions:Bacterial infection are predominant in pediatric septic shock, but viral infection are also significant. Children with malignancies are more severe and resource consumptive. The overall mortality rate for pediatric septic shock remains high, and mortality are associated with malignant tumor, PRISM Ⅲ and pSOFA scores.

Objective:To understand the work experience and service quality of "Internet plus Nursing Service" led by tertiary hospitals to lay the foundation for building an "Internet plus Nursing Service" quality control index system suitable for China's national conditions.Methods:Using field research method, combining purposive sampling with open sampling, from September 2021 to February 2022, the 11 "Internet plus Nursing Service" visiting nurses from 10 residential areas in Shibei District, Shinan District, Laoshan District, Chengyang District and Jimo District of Qingdao were observed for 5 months.Results:The work experience of "Internet plus Nursing Service" visiting nurses: meeting the rigid needs of society and patients, and nurses′ professional value and satisfaction were multiplied; there were still shortcomings in the regulatory mechanism, and information technology construction needed to be continuously strengthened; there were still hidden dangers in patient safety and nurse professional safety issues. Six key factors influencing the quality of Internet plus nursing service were analyzed: non-standard preparation of articles before visiting, non-standard dress for door-to-door service, inadequate preparation for pre operation evaluation, lack of empathy during operation, weak awareness of infection prevention outside the hospital, and failure to carry out health education based on patients′ conditions after operation.Conclusions:The Internet plus nursing service model is dominated by the tertiary hospitals in Qingdao, which has realized two-way referral between the community and the superior hospitals, and has effectively improved the quality of extended care. The acceptance and satisfaction of patients and their families are high, and nurses′work enthusiasm and professional value have increased significantly. It is still necessary to establish and improve theInternet plus nursing service work standards to achieve the homogenization and diversification of nursing services, promote the comprehensive quality of community medical staff.

Objective:To analyze the clinical characteristics of children aged two years old and above with respiratory syncytial virus (RSV) infection in pediatric intensive care unit (PICU).Methods:Children who had RSV infection admitted to PICU at Children′s Hospital of Zhengzhou University from March 2019 to December 2021 were divided into older age group(≥two years old) and younger age group(0.05); In younger age group, there were 132(64.8%) cases of fever and 125(64.8%) cases of wheezing; In older age group, there were 31(93.9%) cases of fever and 13(39.4%) cases of wheezing; the incidence of fever was higher in older age group than that in younger age group( χ2=9.147, P<0.05), and the incidence of wheezing was lower than that in younger age group( χ2=7.631, P<0.05), and the differences were statistically significant.(2) There were 15 (45.5%) cases of simple infection and 18 (54.5%) cases of mixed infection in older age group, and the P/F was significantly different between children with simple infection and those with mixed infection ( P<0.05); there were 14 (42.4%) cases of combined underlying diseases and 19 (57.6%) cases without underlying diseases, and the length of stay in PICU was different between children with combined underlying diseases and those without underlying diseases ( P<0.05), and there was no significant difference in mechanical ventilation or total length of stay ( P>0.05). Conclusion:RSV infection in children aged two years old and above in PICU occurres mostly between two and six years of age, with a relatively low incidence of wheezing and common fever; RSV infection alone may have a greater impact on oxygenation, and combined underlying diseases can affect the length of stay in PICU.