Shanghai Children's Medical Research Articles

The efficacy of muscle function training combined with occlusal inducer in the treatment of malocclusion in children with OSA

Objective:This study aims to investigate the clinical effectiveness of muscle function training combined with occlusal inducers in the treatment for children's malocclusion after obstructive sleep apnea（OSA） surgery. Methods:A total of 40 pediatric patients who underwent surgery for OSA at Shanghai Children's Medical Center, affiliated with Shanghai Jiao Tong University School of Medicine, from January 2020 to December 2021 were involved in this study. They were divided into a treatment group（n=20） and a control group（n=20）. The treatment group received muscle function training combined with occlusal inducers, while the control group received muscle function training alone. Cephalometric measurements of hard tissues were compared between the two groups before and 12 months after surgery. Additionally, the OSA-18 questionnaire, which includes 18 items to assess the life quality of children with OSA, was filled out before surgery, 6 months after surgery, and 12 months after surgery by these patients. Results:①The scores of sleep disorders, physical symptoms, emotional status, daytime sleepiness and energy status and the degree of influence on guardians in the two groups were significantly improved at 12 months after operation（P<0.05）. The scores of sleep disorders, physical symptoms, emotional status and the degree of influence on guardians in the treatment group were better than those in the control group（P<0.05）. ②Cephalometric data at 12 months after operation showed that the upper and lower alveolar seat angle（ANB）, Overbite, upper and lower central incisor angle（U1-L1） and Overjet in the treatment group were lower than those in the control group at 12 months after operation, and the difference was statistically significant（P<0.05）. Conclusion:Children with OSA can improve the dentition irregularity by muscle function training combined with occlusal inducer after operation, and the effect is better than that of muscle function training alone.

Efficacy of peritoneal drainage in very-low-birth-weight neonates with Bell's stage II necrotizing enterocolitis: A single-center retrospective study.

Currently, pediatric surgeons are challenged by a lack of consensus on the optimal management strategy (conservative or surgical) for children with Bell's stage II necrotizing enterocolitis (NEC). To evaluate the clinical efficacy of peritoneal drainage in very-low-birth-weight (VLBW) neonates with modified Bell's stage II NEC. This was a retrospective analysis of 102 NEC (modified Bell's stage II) neonates born with VLBW who were treated at the Fujian Children's Hospital (Fujian Branch of Shanghai Children's Medical Center) between January 2017 and January 2020; these included 24 cases in the peritoneal drainage group, 36 cases in the exploratory laparotomy group, and 42 cases in the conservative treatment group. The general characteristics were comparable in the three groups (P > 0.05). Compared with conservative treatment, peritoneal drainage was associated with significantly shorter fasting time, abdominal distension relief time, fecal occult blood (OB) negative conversion time, and reduced hospital length of stay (HLOS) (P < 0.05 for all). Despite some advantages of peritoneal drainage over conservative treatment in terms of cure, conversion to laparotomy, intestinal perforation, intestinal stenosis, and abdominal abscess rates, the differences were not statistically significant (P > 0.05). Compared to exploratory laparotomy, the fecal OB negative conversion time was significantly shorter in the peritoneal drainage group (P < 0.05); similarly, the exploratory laparotomy group showed longer fasting time, abdominal distension relief time, HLOS, and higher complication rate compared to peritoneal drainage group, but the between-group differences were not statistically significant (P > 0.05). Peritoneal drainage, an easy-to-operate procedure, can improve the clinical symptoms of VLBW neonates with Bell's stage II NEC and help reduce the HLOS.

Experience of Pediatric Nurses in Parent-Child Isolation Units of COVID-19 Designated Hospitals: A Qualitative Study.

The omicron pandemic in Shanghai has created unprecedented challenges for pediatric medical institutions, and the work of pediatric nurses has changed rapidly due to the introduction of parent-child treatment. This study aimed to explore the experiences of pediatric nurses in the parent-child isolation unit of COVID-19-designated hospitals and provide a basis for developing feasible interventions as the next step. Using phenomenological research methods, 12 nurses working in the parent-child isolation unit of Shanghai Children's Medical Center affiliated with Shanghai Jiao Tong University School of Medicine from April 1, 2022, to June 15, 2022, were selected by purposive sampling. Semi-structured interviews and data analysis were conducted using Colaizzi's 7-step analysis. Data analysis revealed three major themes and 11 subthemes. The main themes were "risk factors", "protection factors", and "resilience strategies". The sub-themes were "challenge of caring for both adults and children simultaneously", "lack of adult expertise leads to inadequate coping skills", "change in the care population increased communication difficulties", "physical and psychological distress", "social support", "social recognition", "awareness of responsibilities and roles", "team coming together", "hunger for knowledge", "health promotion", and "psychological adjustment strategies". Hospital managers should optimize hospital management strategies, pay attention to multi-departmental and multidisciplinary team cooperation, reduce the burden on pediatric nurses, improve the work and rest environment, mobilize the hospital support system, and maintain nurse's physical and mental health, establish a warm parent-child isolation unit encourages nurses to listen to the patients' voices and adopt diversified communication methods, and strengthen the publicity of the nursing profession, improve social support and recognition, and enhance the sense of self-worth and mission.

Safety and efficacy of transcatheter closure of Fontan fenestration with atrial septal occluder

Objective: To assess the safety and efficacy of transcatheter fenestration closure following Fontan procedure with an atrial septal occluder. Methods: This is a retrospective study. The study sample consists of all consecutive patients who underwent closure of a fenestrated Fontan baffle at Shanghai Children's Medical Center Affiliated to Shanghai Jiaotong University School of Medicine between June 2002 and December 2019. The indications of Fontan fenestration closure included that normal ventricular function, targeted drugs for pulmonary hypertension and positive inotropic drugs were not required prior the procedure; and the Fontan circuit pressure was less than 16 mmHg (1 mmHg=0.133 kPa) and no more than a 2 mmHg increase during test occlusion of the fenestration. Electrocardiogram and echocardiography were reviewed at 24 hours, 1, 3, 6 months and annually thereafter post procedure. Follow-up information including clinical events and complications related to Fontan procedure was recorded. Results: A total of 11 patients, including 6 males and 5 females, aged (8.9±3.7) years old were included. The types of Fontan were extracardiac conduits (7 cases) and intra-atrial duct (4 cases). The interval between percutaneous fenestration closure and the Fontan procedure was (5.1±2.9) years. One patient reported recurrent headache after Fontan procedure. Successful fenestration occlusion with atrial septal occluder was achieved in all patients. Compared with prior closure, Fontan circuit pressure ((12.72±1.90) mmHg vs. (12.36±1.63) mmHg, P<0.05), and aortic oxygen saturation ((95.11±3.11)% vs. (86.35±7.26)%, P<0.01) were increased. There were no procedural complications. At a median follow-up of (3.8±1.2) years, there was no residual leak and evidence of stenosis within the Fontan circuit in all patient. No complication was observed during follow-up. One patient with preoperative headache did not have recurrent headache after closure. Conclusions: If the Fontan pressure is acceptable by test occlusion during the catheterization procedure, Fontan fenestration could be occluded with the atrial septum defect device. It is a safe and effective procedure, and could be used for occlusion of Fontan fenestration with varying sizes and morphologies.

Therapeutic efficacy of hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome in 60 children

Objective: To evaluate the therapeutic efficacy of hematopoietic stem cell transplantation (HSCT) for Wiskott-Aldrich syndrome (WAS), and to analyze the factors related to the outcomes. Methods: The clinical data of 60 children with WAS received HSCT in Shanghai Children's Medical Center from January 2006 to December 2020 were retrospectively analyzed. All cases were treated with a myeloablative conditioning regimen with busulfan and cyclophosphamide, and a graft-versus-host disease (GVHD) prevention regimen based on cyclosporine and methotrexate. Implantation, GVHD, transplant-related complications, immune reconstitution and survival rate were observed. Survival analysis was performed by Kaplan-Meier method, and Log-Rank method was used for univariate comparison. Results: The 60 male patients had main clinical features as infection and bleeding. The age at diagnosis was 0.4 (0.3, 0.8) years, and the age at transplantation was 1.1 (0.6, 2.1) years. There were 20 cases of human leukocyte antigen matched transplantation and 40 mismatched transplantation; 35 patients received peripheral blood HSCT, and 25 cord blood HSCT. All cases were fully implanted. The incidence of acute GVHD (aGVHD) was 48% (29/60) and only 2 (7%) developed aGVHD of grade Ⅲ; the incidence of chronic GVHD (cGVHD) was 23% (13/56), and all cases were limited. The incidence of CMV and EBV infection was 35% (21/60) and 33% (20/60) respectively; and 7 patients developed CMV retinitis. The incidence of sinus obstruction syndrome was 8% (5/60), of whom 2 patients died. There were 7 cases (12%) of autoimmune hemocytopenia after transplantation. Natural killer cells were the earliest to recover after transplantation, and B cells and CD4+T cells returned to normal at about 180 days post HSCT. The 5-year overall survival rate (OS) of this group was 93% (95%CI 86%-99%), and the event free survial rate (EFS) was 87% (95%CI 78%-95%). EFS of non-CMV reactivation group is higher than that of CMV reactivation group (95% (37/39) vs.71% (15/21), χ2=5.22, P=0.022). Conclusions: The therapeutic efficacy of HSCT for WAS is satisfying, and the early application of HSCT in typical cases can achieve better outcome. CMV infection is the main factor affecting disease-free survival rate, which can be improved by strengthening the management of complications.

Clinical characteristics of anomalous aortic origin of a coronary artery in children

Objective: To analyze the clinical characteristics, diagnosis and treatment of anomalous aortic origin of a coronary artery (AAOCA) in children. Methods: There were 17 children diagnosed with AAOCA from January 2013 to January 2022 in Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine.Their clinical manifestations, laboratory and imaging data, treatment and prognosis were retrospectively analyzed. Results: These 17 children included 14 males and 3 females, with the age of (8.7±3.5) years. There were 4 anomalous left coronary artery (ALCA) and 13 anomalous right coronary artery (ARCA). Seven children presented with chest pain or chest pain after exercise, three patients presented with cardiac syncope, one complained chest tightness and weakness, and the other six patients had no specific symptoms. Cardiac syncope and chest tightness occurred in patients with ALCA. Fourteen children had the dangerous anatomical basis of myocardial ischemia caused by coronary artery compression or stenosis on imaging. Seven children had coronary artery repair, of whom two were ALCA and five were ARCA. One patient had received heart transplantation because of heart failure. The incidence of adverse cardiovascular events and poor prognosis in ALCA group was higher than that in ARCA group (4/4 vs. 0/13, P<0.05). They were followed up in the outpatient department regularly for 6 (6, 12) months; except for the one who lost visit, the rest of the patients had a good prognosis. Conclusions: Cardiogenic syncope or cardiac insufficiency usually occurs in ALCA, and adverse cardiovascular events and poor prognosis are more common in ALCA than in ARCA. Early surgical treatment should be considered for children with ALCA and ARCA accompanied by myocardial ischemia.

Genomic Analysis Revealed the International and Domestic Transmission of Carbapenem-Resistant Klebsiella pneumoniae in Chinese Pediatric Patients.

Carbapenem-resistant Klebsiella pneumoniae (CRKP) poses a severe threat to public health worldwide. Based on the genomic analysis of 198 CRKP isolates collected at Shanghai Children's Medical Center over the last 8 years (2013 to 2021), we reported the clinical risk, genetic diversity, and prevalence of antimicrobial resistance (AMR) of CRKP in pediatric patients at the genomic level. We found that the blaNDM genes were the predominant carbapenemase genes, followed by blaKPC-2 and blaIMP. All of the carbapenemases were disseminated mainly by four main types of plasmids, among which one plasmid was associated with a higher risk of bloodstream infections. Notably, we tracked disease outbreaks caused by recent introductions of ST14 CRKP from southeast Asia or western countries, and we reported frequent, repetitive introductions of ST11 from other domestic hospitals that were associated interhospital movement of the patients. The cocirculation of K. pneumoniae and AMR plasmids in hospitals highlights the importance of genome sequencing for monitoring and controlling CRKP infections. IMPORTANCE Carbapenem-resistant Klebsiella pneumoniae (CRKP) infection in pediatric patients differs from that in adults patients in terms of both genetic and phenotypic features, which remain to be elucidated. We present a summary of prevalent CRKP isolates from Chinese pediatric patients over 8 years, demonstrating the prevalence and clinical importance of New Delhi metallo-β-lactamase genes in pediatric patients, mainly describing the genomic features of two predominant CRKP clones (ST11 and ST14) in Chinese children, and identifying four carbapenemase-encoding plasmids that contribute to the transmission of most carbapenemase genes in hospitals. Overall, our research provides valuable information about the international and domestic transmission of CRKP isolates that are prevalent in Chinese children and shows the urgent need for genome sequencing-based surveillance systems for monitoring the transmission of CRKP.

Absence of terminal deoxynucleotidyl transferase expression in T-ALL/LBL accumulates chromosomal abnormalities to induce drug resistance.

T-acute lymphoblastic leukemia/lymphoma (T-ALL/LBL) is a malignant neoplasm of immature lymphoblasts. Terminal deoxynucleotidyl transferase (TDT) is a template-independent DNA polymerase that plays an essential role in generating diversity for immunoglobulin genes. T-ALL/LBL patients with TDT- have a worse prognosis. However, how TDT- promotes the disease progression of T-ALL/LBL remains unknown. Here we analyzed the prognosis of T-ALL/LBL patients in Shanghai Children's Medical Center (SCMC) and confirmed that TDT- patients had a higher rate of recurrence and remission failure and worse outcomes. Cellular experiments demonstrated that TDT was involved in DNA damage repair. TDT knockout delayed DNA repair, arrested the cell cycle and decreased apoptosis to induce the accumulation of chromosomal abnormalities and tolerance to abnormal karyotypes. Our study demonstrated that the poor outcomes in TDT- T-ALL/LBL might be due to the drug resistance (VP16 and MTX) induced by chromosomal abnormalities. Our findings revealed novel functions and mechanisms of TDT in T-ALL/LBL and supported that hematopoietic stem cell transplantation (HSCT) might be a better choice for these patients.

Carbapenem-resistant Pseudomonas aeruginosa infections in critically ill children: Prevalence, risk factors, and impact on outcome in a large tertiary pediatric hospital of China.

Carbapenem-resistant Pseudomonas aeruginosa (CRPA) is a major cause of healthcare-associated infections worldwide, but comprehensive study of clinical characteristics for CRPA infections among critically ill children remains limited in China. The objective of this study was to determine the epidemiology, risk factors, and clinical outcomes of CRPA infections among critically ill pediatric patients in a large tertiary pediatric hospital in China. A retrospective case-control study of patients with P. aeruginosa infections was conducted in the three intensive care units (ICUs) of Shanghai Children's Medical Center from January 2016 to December 2021. All patients with CRPA infection in the ICUs were enrolled as case patients. Patients with carbapenem-susceptible P. aeruginosa (CSPA) infection were randomly selected as control patients in a ratio of 1:1. Clinical characteristics of those inpatients were reviewed through the hospital information system. Univariate and multivariate analyses were performed to evaluate risk factors associated with the development of CRPA infections and mortality of P. aeruginosa infections. A total of 528 cases of P. aeruginosa infection in the ICUs were enrolled in the 6-year study. The prevalence of CRPA and MDRPA (multidrug-resistance P. aeruginosa) was 18.4 and 25.6%, respectively. Significant risk factors related to CRPA infection were the length of hospitalization >28 days (OR = 3.241, 95% CI 1.622-6.473, p = 0.001), receiving invasive operations (OR = 2.393, 95% CI 1.196-4.788, p = 0.014) and a blood transfusion (OR = 7.003, 95% CI 2.416-20.297, p < 0.001) within 30 days before infection. Conversely, birth weight ≥2,500 g (OR = 0.278, 95% CI 0.122-0.635, p = 0.001) and breast nursing (OR = 0.362, 95% CI 0.168-0.777, p = 0.009) were significant protective factors against CRPA infections. The in-hospital mortality rate was 14.2%, and no difference in mortality was observed between patients with CRPA and CSPA infections. Platelet < 100 × 109/L (OR = 5.729, 95% CI 1.048-31.308, p = 0.044) and serum urea <3.2 mmol/L (OR = 5.173, 95% CI 1.215-22.023, p = 0.026) were independent predictors for the mortality due to P. aeruginosa infection. Our findings provide insights into CRPA infections among critically ill children in China. They provide guidance in identifying patients that may be at high risk for a resistant infection and emphasize the importance of antimicrobial stewardship and infection control in hospitals.

Molecular genetic characteristics of a family which coinheritance of rare-88 C>G (HBB:c.-138 C>G) β-thalassemia mutation with α-thalassemia and review of the literature

The molecular genetic characteristics of a family with rare -88 C>G (HBB: c.-138 C>G) β-thalassemia gene mutation were studied using cohort study. The cohort study was conducted from June to August 2022 by Prenatal Diagnosis Center of Sanya Women and Children's Hospital Managed by Shanghai Children's Medical Center. The phenotype and genotype were analyzed by hematological cytoanalyzer, automatic electrophoretic analysis system, and next-generation sequencing (NGS). And then, Sanger sequencing was used to verify the rare gene results. The results showed that the proband, her father, her uncle and her younger male cousin had discrete microcytosis (MCV 70.1 fl, 71.9 fl, 73.1 fl and 76.6 fl, respectively) and hypochromia (MCH 21.5 pg,22.0 pg,22.6 pg and 23.5 pg, respectively), elevated hemoglobin A2 level (5.3%, 5.4%, 5.4% and 5.5%, respectively), slightly elevated or normal fetal hemoglobin (Hb F), but no anemia. The proband was identified to have co-inherited ɑ-thalassemia (Hb Westmead gene heterozygous mutation, ɑwsɑ/ɑɑ) and β-thalassemia with a rare -88 C>G (HBB: c.-138 C>G) heterozygous mutation (β-88 C>G/βN). Her mother had the same α-thalassemia as the proband. Her father, her uncle and her younger male cousin had the same rare -88 C>G heterozygous mutations as the proband. While her grandmother and younger brother were not carrier of thalassemia. In conclusion, 4 cases of rare -88 C>G(HBB:c.-138 C>G) heterozygous mutation had been detected in a Chinese family. Carriers of this beta-thalassemia are clinically asymptomatic. This study enriches the knowledge of the thalassemia mutation spectrum in Chinese people and provides valuable information for genetic counseling, prenatal diagnosis, and prevention of thalassemia, providing a scientific basis for improving the quality of birth population and preventing birth defects.

Epidemiology and clinical characteristics of pediatric sepsis in PICUs of China: A national cross-sectional study.

Epidemiology and clinical characteristics of pediatric sepsis in PICUs of China: A national cross-sectional study.

A cross-sectional study evaluating health-related quality of life of Chinese pediatric patients with hematological malignancies using EQ-5D-Y.

The study aimed to assess health-related quality of life (HRQoL) and to estimate the health utility of pediatric patients with hematological malignancies (HMs) in China. A cross-sectional study recruited a series of pediatric inpatients diagnosed with HM from November 2018 to May 2019 in the Shanghai Children's Medical Center. Subjects were interviewed to collect sociodemographic information about themselves and their guardians. The EQ-5D-Y was completed by each patient to rate their own HRQoL, which later derived the health utility. The health status was also assessed by clinicians following the Eastern Cooperative Oncology Group (ECOG) system. Upon the descriptive analysis and univariate analysis, multivariate generalized linear models were built to explore the associations of risk factors with HRQoL measures of utility, Visual Analog Scale (VAS) score, and the five EQ-5D-Y domains. The 96 subjects had a mean age of 10.5 years and included 62 (64.4%) boys. There were 46 (47.9%) and 25 (26.0%) children diagnosed with acute lymphoblastic leukemia and non-Hodgkin's lymphoma, respectively. The means (SD) of utility and EQ-VAS scores were 0.88 (0.10) and 85.8 (15.1), respectively. Twenty-six (27.1%) patients were graded poor health by the ECOG standard (score 2/3). Both univariate and multivariate analyses found strong correlations between ECOG and HRQoL. After adjusting for covariates, poor ECOG score was significantly associated with an impaired utility and VAS of -0.103 and -8.65, respectively. With regard to individual HRQoL domains, worse ECOG was more likely to report health problems with an increased risk of 2.94 to 12.50; residence, income, guardians' education, and disease duration were also found to be significantly related to either the utility or certain health domains. The HRQoL of Chinese pediatric patients with HM is considered relatively poor and of great concern to healthcare. With the strong correlations between EQ-5D-Y-related HRQoL measures and the traditional clinical index ECOG, the EQ-5D-Y is able to provide valuable evidence for clinical decision-making at the individual level. At the same time, its health utility can inform resource allocation at a macro level.

Prognosis of bronchial asthma in children with different pulmonary function phenotypes: A real-world retrospective observational study.

To follow up on the changes in pulmonary function phenotypes in children with asthma in the first year after diagnosis, and explore the risk factors of poor control in children with good treatment compliance. Children who were diagnosed with asthma in the Respiratory Department of Shanghai Children's Medical Center from January 1, 2019 to December 31, 2020 and were re-examined every 3 months after diagnosis for 1 year were continuously included, regardless of gender. We collected the clinical data, analyzed clinical characteristics of the different pulmonary function phenotypes at baseline and explored risk factors of poor asthma control after 1 year of standardized treatment. A total of 142 children with asthma were included in this study, including 54 (38.0%) with normal pulmonary function phenotype (NPF), 75 (52.8%) with ventilation dysfunction phenotype (VD), and 13 (9.2%) with small airway dysfunction phenotype (SAD) in the baseline. Among them, there were statistically significant differences in all spirometry parameters, age, and course of disease before diagnosis (P < 0.05), and a negative correlation between age (r 2 = -0.33, P < 0.001), course of disease before diagnosis (r 2 = -0.23, P = 0.006) and FEV1/FVC. After 1-year follow-up, large airway function parameters and small airway function parameters were increased, fractional exhaled nitric oxide (FeNO) was decreased, the proportion of NPF was increased, the proportion of VD was decreased (P < 0.05), while there was no significant difference in the proportion of SAD. After 1 year of standardized treatment, 21 patients (14.8%) still had partly controlled or uncontrolled asthma. Our results showed that the more asthma attacks occurred within 1 year (OR = 6.249, 95% CI, 1.711-22.818, P = 0.006), the more times SAD presented at baseline and Assessment 1-4 (OR = 3.092, 95% CI, 1.222-7.825, P = 0.017), the higher the possibility of incomplete control of asthma. About 15% of the children with good treatment compliance were still not completely controlled after 1 year of treatment, which is closely associated with persistent small airway dysfunction.

Health utility of children with acute lymphoblastic leukemia in China.

Acute lymphoblastic leukemia is the most common cancer in children. As the 5-year survival rate has been improved to over 80%, more emphasis is now placed on reducing therapy toxicities and enhancing health-related quality of life (HRQoL) of patients during treatment. Our objective was to measure health utility of pediatric acute lymphoblastic leukemia (pALL) patients in China, examine utility weights of different treatment phases and influencing factors of health utility, as well as identify which aspects of HRQoL were most impaired. A cross-sectional study was conducted in Shanghai Children's Medical Center (SCMC) Affiliated to Shanghai Jiao Tong University School of Medicine in China from April to November 2021. Primary caregivers of 247 patients completed the assessment by CHU9D-CHN and health utility scores were computed for all the patients and stratified by treatment phases. Various multivariable models were constructed and the best was chosen to identify independent factors associated with utility scores. Factors affecting the most impaired dimensions were also examined. The overall mean (SD) health utility score was 0.79 (±0.17) and significantly increased from induction (0.73 [±0.19], P < 0.001) to consolidation (0.74 [±0.18]), and to maintenance (0.82 [±0.16]). After adjusting for potentially influencing factors, utility scores in induction (Beta = -0.086, P = 0.005) and consolidation (Beta = -0.074, P = 0.043) were constantly lower than those during maintenance. In item-level analysis, lower age and induction phase were found to be significantly associated with high severity reported on the "school work/homework" dimension. Additionally, only the induction phase (vs. maintenance, OR = 2.24, P = 0.016) was independently associated with the high severity level reported on the "able to join in activities" dimension. This is the first study that measured health utility of children with pALL in China. Mean health utility scores increased from induction to maintenance. These provided important utility estimates that help inform future health economic models. The phrasing of "School work/homework" in CHU9D-CHN could be further improved. More efforts are needed to design and implement specific interventions targeting at the dimension "able to join in activities" for enhancing HRQoL of children with pALL in China.

Clinical and laboratory features of childhood-onset primary Sjögren's syndrome: A retrospective study from China.

The initial presentations of childhood-onset primary Sjögren's syndrome (C-pSS) vary, making diagnosis challenging. We aimed to improve the diagnosis and evaluation of C-pSS by summarizing its clinical and laboratory features. A total of 49 patients with C-pSS between July 2015 and August 2022 in the Department of Rheumatology and Immunology of Shanghai Children's Medical Centre were enrolled in this study. Their clinical manifestations and laboratory examinations of these patients were compared based on the presence or absence of thrombocytopenia and parotitis and whether the immunological markers, including anti-nuclear antibodies (ANA), rheumatoid factor (RF), anti-Ro52/SSA antibodies (anti-SSA/Ro52), anti-Ro60/SSA antibodies (anti-SSA/Ro60), and anti-Ro/SSB antibodies (anti-SSB), were positive. The mean age at C-pSS diagnosis was 10.34 ± 3.45years, and the ratio of boys to girls was 1:6. In the thrombocytopenia group, parotitis (P = 0.044), organ involvement except for hematology (P = 0.002), positive anti-SSB (P = 0.004), and positive RF (P = 0.001) were less frequently observed. Complement C4 (P = 0.038) and white blood cells (P = 0.002) levels decreased and increased significantly, respectively. Anti-SSB (P = 0.010) and RF (P = 0.004) positivity were independent potential protective factors against thrombocytopenia in patients with C-pSS. In the parotitis group, higher ANA titers (P = 0.027), higher focus scores on labial gland biopsy (P = 0.024), and positive RF (P = 0.001), anti-SSA/Ro60 (P = 0.003), and anti-SSB (P = 0.001) were observed more frequently. Furthermore, positive anti-SSB (P = 0.012) and positive RF (P = 0.028) were independent risk factors for parotitis in patients with C-pSS. The hemoglobin level was significantly lower in patients with positive anti-SSA/Ro52 and positive anti-SSA/Ro60 results (P = 0.022 and P = 0.029, respectively), while immunoglobulin G level was significantly higher in patients in the same group (P = 0.048 and P = 0.007, respectively). Positive anti-SSB and positive RF values may be independent potential protective factors of thrombocytopenia in patients with C-pSS. In contrast, positive anti-SSB and positive RF were independent risk factors of parotitis in patients with C-pSS. More studies are needed to reveal the diagnostic role and pathogenic mechanism of immunological markers in C-pSS.

Anatomical attention-based prediction of postoperative pulmonary venous obstruction via CTA images.

Total anomalous pulmonary venous connection (TAPVC) is a rare congenital heart disease, with which some patients suffer from postoperative pulmonary venous obstruction (PPVO), requiring particular follow-up strategies and treatments. PPVO prediction has important clinical significance, while building a PPVO prediction model is challenging due to limited data and class imbalance distribution. Inspired by the anatomical evidence of PPVO, which is related to the structure of the left atrium (LA) and pulmonary vein (PV), we design an effective multi-task network for PPVO classification. The proposed method incorporates clinical priors and merits of the segmentation-based network into the classification task. The features learned from segmenting LA and PV are concatenated into the PPVO classification branch to constrain the learning of discriminative features. Anatomical-guided attention is applied in the aggregation of these features to restrict them focusing on TAPVC-related regions. To deal with the imbalance classification problem of PPVO, a novel classification loss derived by masked class activation map (MCAM) is designed to improve the classification performance. Computed tomography angiography (CTA) images of 146 patients diagnosed with supracardiac TAPVC in Shanghai Children's Medical Center and Guangdong Provincial People's Hospital were enrolled in this work. The comprehensive experiments demonstrate the effectiveness and generalization of our proposed method. The automatic PPVO prediction model shows the potential application in helping clinicians develop follow-up strategies, thereby improving the survival rate of TAPVC patients.

Effectiveness of Paxlovid in the treatment of the SARS-CoV-2 Omicron variant infection in children with hematologic malignancies: a retrospective cohort study.

Patients with hematologic malignancies (HMs) may be immunocompromised after receiving anti-tumor therapy. Those who also have the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) virus infection face many challenges, including a lack of effective antiviral drugs. This study aimed to investigate the clinical features of the SARS-CoV-2 Omicron variant infection in children with HMs, and the effectiveness of Paxlovid. A retrospective, non-randomized study was conducted on pediatric patients with HMs infected with the SARS-CoV-2 Omicron variant who had been admitted to the Shanghai Children's Medical Center, Shanghai, China from December 1, 2022 to March 1, 2023. The Paxlovid-treated group (Group P) comprised 21 patients, and the non-Paxlovid-treated group (Group N) comprised 21 patients. The patients' demographic data, clinical features, and therapeutic outcomes were collected. Statistical tests were used to evaluate the effectiveness of the treatment and related factors. The clinical course of the SARS-CoV-2 Omicron variant infection for most of the children with HMs was non-severe (97.6%), and only one child progressed to severe disease (2.4%). The most common symptoms were fever (66.7%) and cough (52.4%). Compared with the children in Group N, those in Group P had worse clinical characteristics, including those who previously underwent hematopoietic stem cell transplantation (HSCT) or chimeric antigen receptor T (CAR-T) cell treatment (71.4% vs. 28.6%, P=0.005), and those in the myelosuppressive phase (57.1% vs. 4.8%, P<0.001). Most of the children in Group P were treated with more than two types of antibiotics (76.2% vs. 42.9%, P=0.02). The patients treated with Paxlovid within 5 days of diagnosis had a median viral clearance time of 5 days [interquartile range (IQR), 4-8 days], which was significantly shorter than that of the patients who were not treated with Paxlovid (P=0.03). There were no significant differences in the clinical outcomes between the two groups after the propensity score matching (PSM) analyses. Eight patients (19%) had repeat-positive (re-positive) test results. No factor was found to be statistically significant in predicting re-positive test results based on the binary logistic regression analysis. Administering Paxlovid within 5 days of the diagnosis of the SARS-CoV-2 Omicron variant infection in children may effectively shorten the clearance time of the virus, but there is still the possibility the patients may have re-positive test results.

Infection with the SARS-CoV-2 Omicron variant in children with congenital heart disease: A case series study during Shanghai epidemic.

ObjectiveTo analyze the clinical characteristics and prognostic factors of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) Omicron variant infections in children with congenital heart disease (CHD).MethodsA retrospective analysis was performed on SARS-CoV-2 Omicron-infected children with CHD who were admitted to Shanghai Children's Medical Center from April 1, 2022 to May 31, 2022. The clinical, laboratory and imaging data, and the nucleic acid conversion time of the children in this group were collected and analyzed.ResultsThirteen patients were included in this study and had an average age of 1.1 (0.16–14) years. Among the patients, 3 patients were preoperatively treated, and 10 were postoperatively treated. According to the severity of the disease, 1 patient was diagnosed with the moderate type, and the remaining 12 patients were diagnosed with the mild type. The clinical symptoms were mostly associated with upper respiratory tract infections, including 13 with fever (100%), 8 with cough (61.8%), 5 with sputum production (38.5%), 1 of shortness of breath (7.7%), etc. All patients were successfully discharged from the hospital, with 16.4 ± 2.9 days needed to obtain cycle threshold (CT) values ≥35 in nucleic acid testing and 17.5 ± 3.6 days of hospitalization.ConclusionsFor vulnerable patients such as children with CHD, SARS-CoV-2 Omicron variant infections mostly present with mild upper respiratory tract symptoms with negative or mildly changed chest imaging. Through appropriate treatment of the underlying disease in the quarantine ward, patients might obtain good outcomes, even after long periods of hospitalization.

Analysis of 42 cases of childhood superior vena cava syndrome associated with mediastinal malignancy

Objective: To summarize the clinical features, management and outcome of superior vena cava syndrome (SVCS) associated with mediastinal malignancy in children. Methods: Clinical data of 42 children of SVSC associated with mediastinal malignancy in Shanghai Children's Medical Center from January 2015 to December 2021 were collected and analyzed retrospectively. The clinical manifestations, pathological diagnosis, disease diagnosis process, and prognosis were summarized. Results: Among 42 children of SVCS associated with mediastinal malignancy, there were 31 males and 11 females. The age at diagnosis was 8.5 (1.9, 14.9) years. Cough and wheezing (33 cases, 79%), orthopnea (19 cases, 45%) and facial edema (18 cases, 43%) occurred most commonly. T-cell lymphoblastic lymphoma (T-LBL) was the most frequent pathological diagnosis (25 cases, 60%), followed by T-cell acute lymphoblastic leukemia (T-ALL) (7 cases, 17%), anaplastic large cell lymphoma (4 cases, 10%) and diffuse large B-cell lymphoma (2 cases, 5%), peripheral T-lymphoma, Hodgkin lymphoma, Ewing's sarcoma and germ cell tumor (1 case each). Pathological diagnosis was confirmed by bone marrow aspiration or thoracentesis in 14 cases, peripheral lymph node biopsy in 6 cases, and mediastinal biopsy in 22 cases. Twenty-seven cases (64%) had local anesthesia. Respiratory complications due to mediastinal mass developed in 3 of 15 cases who received general anesthesia. Of the 42 cases, 27 cases had sustained remission, 1 case survived with second-line therapy after recurrence, and 14 cases died (2 cases died of perioperative complications and 12 cases died of recurrence or progression of primary disease). The follow-up time was 36.7 (1.2, 76.1) months for 27 cases in continuous complete remission. The 3-year overall survival (OS) and events free survival (EFS) rates of 42 children were 59% (95%CI 44%-79%) and 58% (95%CI 44%-77%) respectively. Conclusions: SVCS associated with mediastinal malignancy in children is a life-threatening tumor emergency with high mortality. The most common primary disease is T-LBL. The most common clinical symptoms and signs are cough, wheezing, orthopnea and facial edema. Clinical management should be based on the premise of stable critical condition and confirm the pathological diagnosis through minimal invasive operation.

Association between body mass index at diagnosis and outcomes in Chinese children with newly diagnosed acute lymphoblastic leukemia.

Studies of the association between body mass index (BMI) at diagnosis and treatment outcome in children with acute lymphoblastic leukemia (ALL) have yielded inconsistent results. Hence, we conducted a retrospective study in a large cohort of Chinese children with ALL treated with contemporary protocols. A total of 1437 children (62.1% male; median age at diagnosis 5.7 years, range: 2.3-16.3 years) were enrolled in two consecutive clinical trials at the Shanghai Children's Medical Center. The rates of overall survival, event-free survival, relapse, treatment-related mortality, and adverse events were compared among patients who were underweight (BMI < 5th percentile), at a healthy weight (5th to 85th percentile), overweight (>85th to <95th percentile), and obese (≥95th percentile). At diagnosis, 91 (6.3%) patients were underweight, 1070 (74.5%) were at a healthy weight, 91 (6.3%) were overweight, and 185 (12.9%) were obese. No significant association was found between weight status and 5-year overall survival, event-free survival, or relapse in the overall cohort. When analyzed as a continuous variable, a higher BMI Z-score was associated with treatment-related mortality (hazard ratio 1.33 (95% confidence interval [CI], 1.05-1.68%), p= 0.02). The treatment-related mortality rate was higher in the overweight (5.5%, 95% CI 0.8-10.2%) and obese (3.2%, 95% CI 0.6-5.8%) groups compared with the underweight (0.0%) and healthy-weight groups (1.9%, 95% CI 1.1-2.7%; p=0.04). Multivariable analysis showed that children who were overweight had a higher risk of treatment-related mortality (hazard ratio 3.8, 95% CI 1.3-11.4). While body weight status was not associated with event-free survival or overall survival, overweight patients were at higher risk of treatment-related mortality.