Background：Cardiovascular complications have become the leading cause of death in children with Duchenne muscular dystrophy(DMD). Electrocardiography(ECG) is emerging as an early sensitive indicator for monitoring myocardial involvement in this population.
Objective：To analyze ECG characteristics in children with DMD.
Design：Retrospective cohort study.
Methods：The characteristics of 486 ECG in 304 children with genetically diagnosed DMD were retrospectively analyzed, and compared with 112 ECG indices of 112 normal children in each age group.
Main outcome measures：The incidence and type of ECG abnormalities in children with DMD.
Results：The overall incidence of abnormal ECG in children with DMD was 38.7% (188/486). ECG abnormalities mainly include sinus tachycardia, left ventricular hyper voltage/left ventricular hypertrophy, right ventricular hypertrophy, double ventricular hypertrophy, STT changes, T wave changes, pathological Q waves,incomplete right bundle branch block(ICRBBB), complete right bundle branch block(CRBBB), ventricular block, atrial premature beats/ventricular premature beats and short PR syndrome.No long QT syndrome and other types of conduction block were identified. The incidence of abnormal ECG in children with DMD regardless of LVEF increased significantly with age (P<0.05), and children with DMD had significantly faster heart rate, significantly shortened PR interval, and significantly increased amplitude of RV1 and RV5, compared to agematched healthy children (P<0.05).
Conclusions：ECG abnormalities are common manifestations in children with DMD, and their incidence gradually increases with age, and increased heart rate, shortened PR interval, and increased RV1 amplitude are early characteristic changes in ECG abnormalities in children with DMD.
Background：Retinopathy of prematurity (ROP) can cause irreversible visual impairment in severe cases, and there is a lack of appropriate risk prediction model for identifying severe ROP specifically tailored to the Chinese preterm population.
Objective：To construct and validate an early risk prediction model for severe ROP in premature infants with gestational age ≤ 34 weeks based on the Chinese neonatal population.
Design：Retrospective cohort study.
Methods：From January 2020 to March 2022, premature infants admitted to the Neonatal Intensive Care Unit (NICU) with a gestational age of ≤ 34 weeks and who underwent a complete eye examination were included. The cohort was divided into training and validation sets using the June 2021 admission time as the cutoff point. Clinical information was collected at the time of the first postnatal eye examination, and regular screenings and followups were conducted according to the ROP screening guidelines. Severe ROP is defined as having stage 3 or higher lesions, positive for plus disease, or requiring laser and cryotherapy treatment. All other cases are categorized as mild ROP. In the training set, clinical variables with collinearity were excluded based on the Pearson correlation coefficient and the final predictive factors were determined through Lasso regression and logistic regression analysis, then the prediction model was constructed and presented as a nomogram. In the validation set, the area under the curve (AUC) of receiver operating characteristic (ROC) was used to evaluate the discrimination and determine the optimal cutoff value of the prediction model, in which the sensitivity and specificity of the model were calculated. Based on the total population, clinical efficacy of the model was finally assessed by decision curve analysis (DCA).
Main outcome measures：The predictive performance of the model for identifying premature infants who will progress to severe ROP.
Results：There were 1,380 cases in the training set (76 cases with severe ROP) and 592 cases in the validation set (36 cases with severe ROP). The differences in the distribution of baseline information between these two groups were not statistically significant. The nomogram prediction model established by including five predictors of gestational age, birth weight, prenatal corticosteroids, invasive mechanical ventilation, and lateonset sepsis had an AUC of 0.87 (95% CI: 0.83 to 0.90) and 0.88 (95% CI: 0.82 to 0.94) in the training and validation sets, respectively. When the predictive threshold was set at 0.04, the model had a sensitivity of 0.833 (95% CI: 0.7120.955) and a specificity of 0.786 (95% CI: 0.7520.820) in the validation set. The clinical decision curve demonstrates that the model has good clinical utility and provides higher net benefits when the threshold probability ranges from 5% to 40%.
Conclusions：The severe ROP prediction model established has a certain reference value for evaluating the risk of severe ROP in neonates with gestational age ≤ 34 weeks.
Background：The disturbance coefficient(DC) is the main technical parameter of noninvasive brain edema dynamic monitoring instruments, and there is no normal reference value for children and adolescents at home abroad.
Objective：To establish the reference intervals of DC for children and adolescents aged 116 years.
Design：Multicenter prospective crosssectional survey.
Methods：From March 2020 to March 2023, children and adolescents aged 116 years without craniocerebral injury were recruited from 6 hospitals 2 kindergarten and primary schools in Chongqing, Sichuan and Shenzhen. The noninvasive brain edema dynamic monitor was used for continuous monitoring for 15 minutes, and the monitoring average was taken as the final DC value of the subjects. Generalized Additive Models for Location, Scale, and Shape (GAMLSS) were used to draw P1, P5, P10, P25, P50, P75, P90, P95, P99 percentile values and percentile curves for disturbance coefficients of different ages. Independent sample ttest was used to analyze the difference of DC between boys and girls, and analysis of variance was used to calculate the DC mean value in different age groups and the difference of DC in different age groups. The changes of DC with age, weight and head circumference were explored by drawing scatter plots and Loess local weighted nonparametric regression curves.
Main outcome measures：Disturbance coefficient.
Results：A total of 1 247 children and adolescents with no cranial injuries were included in the study, including 787 males (63.1%) and 460 females. The observed fitted percentile curves of disturbance coefficients for children and adolescents of different ages showed that the level of disturbance coefficients gradually increased with age, and the growth of disturbance coefficient curves slowed down and eventually stopped after 5 years of age. In the overall population, there was no significant difference in DC levels between males and females (P>0.05), and there was no significant difference in DC among children and adolescents of different ages and genders. Correlation analysis showed that when age＜5 years old, body weight<18.0 kg and head circumference<51.0 cm, DC was significantly positively correlated with age, body weight and head circumference (r=0.663, 0.454 and 0.474, respectively). On the contrary, when age≥5 years old, body weight≥18.0 kg and head circumference≥51.0 cm, DC did not change significantly and gradually stabilized at 107.
Conclusions：The study constructed the percentile curve of DC in children and adolescents aged 1 to 16 years old. When the age was ＜5 years old, the DC increased with age, and when the age was ≥5 years old, the DC gradually stabilized at 107.
Background：Antibiotics are a frequently used drug category in NICU. The Antibacterial Drug Management Project (ASP) can promote the rationalization of the use of antibiotics in NICU.
Objective：To explore the effectiveness of implementing ASP to reduce the use of antibiotics in newborns with pneumonia.
Design：Quality improvement research.
Methods：This study focuses on infants with neonatal pneumonia and divides them into a control group and an intervention group before and after the implementation of ASP starting from January 1, 2019. The control group was treated with antibiotics selected by the attending physician based on empirical antimicrobial spectrum, and special pathogens or drugs were used after consultation with infectious disease physicians. The intervention group implemented NICU's characteristic ASP through the MDTASP team, which includes neonatologists, clinical pharmacists, infectious disease physicians, microbiology laboratory physicians, and radiologists. The MDTASP team conducted regular rounds, medication evaluations, and guidance on antibiotic use. When the sample size of the intervention group and the control group meet a 1∶1 ratio, inclusion will be terminated.
Main outcome measures：Proportion of combined use of antibiotics in newborns with pneumonia.
Results：From January 1, 2018 to June 30, 2018, 229 cases were retrospectively included in the control group, and from January 1, 2019 to May 30, 2019, 229 cases were prospectively included in the intervention group. There was no statistically significant difference between the two groups in terms of general information of the child and basic information of the mother. The proportion of combined use of antibiotics in the control group and intervention group was 24.5% (56/229) and 7.0% (16/229), respectively（χ2=26.367, P＜0.001). Compared with the intervention group, the days of antimicroial therapy was shortened from 12 (360) days to 8 (035) days (P＜0.001), and the length of hospital stay was shortened from 10 (733) days to 9 (546) days (P=0.005). There was no statistically significant difference in the proportion of necrotizing enterocolitis and death between the two groups after the use of antibacterial drugs.
Conclusions：ASP can reduce the use of antimicrobial agents so as to decrease unnecessary antimicrobial exposure in neonatal department.
Background：Preschoolers are experiencing a key period of shaping healthy dietary behaviors, for which daily monitoring and timely intervention is urgently required. However, there is a lack of efficient tools for quantifying this level in China so far.
Objective：To introduce a scale questionnaire named Nutrition Quotient for Preschoolers from the South Korea (NQ-P), and evaluate its reliability and validity.
Design：Crosssectional study.
MethodsThe Chinese version of NQ-P was obtained after forward and backward translation, research group review and modification. Guardians of preschoolers in two kindergartens were crosssectionally investigated and the data was collected for reliability and validity analysis.
Main outcome measures;NQ-P(Chinese version) scores.
Results:The Chinese version of NQ-P used the same scoring and weighting method as the original version, which included 14 items and 3 dimensions (balance, moderation, environment). A total of 440 guardians of preschoolers aged 36.5 years completed the study (97.3%). Among 440, 51.8% were boys, and among those who filled the questionnaires, 317(72.0%) were mothers, 115(26.1%) were fathers, and 282 of them were primary meal planners. Confirmatory factor analysis showed CFI were 0.768 for NQ-P total score, 0.9050.909 for 3 dimensions, RMSEA were 0.063 for NQ-P total score and 0.0310.043 for 3 dimensions. Cronbach's α were 0.7060.774 for NQ-P scores. Retest Kappa were 0.790.98 for 14 items.
Conclusions:The Chinese version of NQ-P has acceptable validity and reliability, and can be used to initially evaluate the dietary quality of preschoolers by investigating their guardians.
Background：Primary testicular lymphoblastic lymphoma(LBL) in children is rare and mostly secondary, and the previous treatment regimens were mainly chemotherapy combined with local radiotherapy of testis. Modern treatment regimens use high dose methotrexate (HDMTX) and other systematic chemotherapy without testicular radiotherapy.
Objective：To summarize the clinical features of pediatric lymphoblastic lymphoma (LBL) with testicular involvement and to investigate the clinical efficacy and prognosis of ALLlike regimen containing HDMTX.
Design：Case series report.
Methods：The clinical data were collected from 17 pediatric LBL patients with neoplastic lesions and testicular involvement who were treated from January 2009 to April 2017, and chemotherapy was performed with NHL2009LBL regimen (from the modified BFM90 protocol) in lymphoma treatment regimen at Beijing Children's Hospital (BCH) Affiliated to Capital Medical University. The clinical characteristics and the curative effect were analyzed.
Main outcome measures：5year overall survival rate(OS) and eventfree survival rate (EFS).
Results：Of 277 children with LBL in the same period, 17 were associated with testicular involvement(6.1%), including 5 cases of TLBL and 12 cases of BLBL, with a median age of 6.2(2.613.7) years old. The clinical stages were stage Ⅲ (4 cases) and stage Ⅳ (13 cases). There were 5 cases with mediastinal mass, 3 cases with central nervous system involvement, 12 cases with bone marrow involvement (8 cases of which had reached the leukemia stage), 11 cases of inguinal, pelvic, sciatic/pubic area invasion and 7 cases of giant tumor. Primary testicular lymphoma was found in 1 case and secondary in 16 cases. Bilateral testicular invasion occurred in 6 cases and unilateral testicular invasion in 11 cases. After induction remission treatment, the testicular tumor was evaluated to disappear. The median followup time was 103(3129) months. Among the 17 children, 6 cases relapsed (only 1 case of testicular relapsed who had complete remission after treatment according to the standard protocol and longterm diseasefree survival). Two cases died of recurrence. The 5year OS was (87.5±8.3)%, and the 5year EFS was (68.8±11.6)%. Compared with the overall survival rate of LBL during the same period, the difference was not statistically significant.
Conclusions：The clinical symptoms of testicular involvement in pediatric LBL are hidden. Routine Bultrasound examination before chemotherapy can improve the diagnosis rate. The risk of testicular involvement is increased in patients with groin area, pelvis and pelvic invasion. Continued complete remission can be achieved with regular chemotherapy without testicular radiotherapy, and testicular function can be preserved.
Background Nusinersen has been officially used for the treatment of 5q spinal muscular atrophy (SMA) in China recently. By now there are few domestic case reports on the treatment of SMA with nusinersen.
Objective To investigate the efficacy, safety and levels of neurofilament light chain protein (NFL) in cerebrospinal fluid with treatment of nusinersen in children with 5q spinal muscular atrophy.
Design Case series report.
Methods Patients with 5q SMA who were initially treated with nusinersen as monotherapy for not less than 14 months in the Department of Neurology, Children's Hospital of Shandong University from January 2020 to June 30, 2023 were enrolled. Multiple ligation dependent probe amplification (MLPA) or fluorescence quantitative PCR (qPCR) methods were used to detect exon 7 of SMN1 gene. Clinical classification is based on the age of symptom onset and the maximum motor function achieved. Cerebrospinal fluid samples for the detection of neurofilament light chain protein (NFL) level were collected by routine lumbar punctures or ultrasound-guided lumbar punctures, and nusinersen was administrated via intrathecal injection. The Children′s Hospital of Philadelphia infant test of neuromuscular disorders (CHOP INTEND), the Revised Upper Limb Module Test (RULM), the Hammersmith functional motor scale expanded (HFMSE), and the 6-minute walk test (6MWT) were performed by a professionally trained and qualified rehabilitation physician before and after treatment. General information such as gender, date of birth, date of symptom onset, and adverse events during treatment were collected.
Main outcome measures The scores of motor function scale after 14 months treatment with nusinersen.
Results In total, 68 SMA pediatric patients with treatment duration not less than 14 months were included in this study, including 32 girls (47.0%). There were 9 cases of type I (13.2%), 40 cases of typeⅡ (58.8%), and 19 cases of type Ⅲ (27.9%). The median age of disease onset is 10 (7,13.8) months, and the median age of diagnosis is 17.5 (12,37) months. Homozygous deletion of exon 7 in SMN1 gene occurred in 64 cases (94.1%), and intragenic mutation of SMN1 gene occurred in 4 cases. The improvement of motor function in 60 children (88.2%) had clinical significance, including 7 cases of type I, 37 cases of typeⅡ, and 16 cases of type Ⅲ. In total, 90.0% (18/20) cases showed a score improvement by ≥4 points on the CHOP INTEND scale, while 97.8% (44/45) cases showed a score improvement by ≥3 points on the HFMSE scale. In total, 92.3% (12/13) cases showed a distance extension by ≥30 meters on the 6MWT; 66.7% (12/18) cases showed a score improvement by ≥2 points on the RULM scale. Two cases achieved new motor function milestones after treatment. The NFL level in the cerebrospinal fluid of 15 children with SMA typeⅡ declined to 69.2 (40.5, 89.3) pg·mL-1 after the loading dose therapy (184th day after first drug administration) comparing with 176.6 (104.5,199.6) pg·mL-1 before treatment, the difference is statistically significant. Adverse events during treatment included fever in 3 cases with 1 case of type Ⅲ, 2 cases of type Ⅱ, all of which were relieved spontaneously; respiratory tract infection in 2 cases with 1 case of type I and 1 case of type Ⅱ, which were relieved after treatment; constipation, myalgia, vertigo and cough occurred in 1 type Ⅱ case each, all of which were relieved spontaneously and 1 case of typeⅡ with abnormal liver function, which normalized after 7 days treatment. No serious adverse event occurred.
Conclusions Nusinersen can improve the motor function of children with 5q SMA after 14 months of treatment with good safety. The NFL level in cerebrospinal fluid of children with SMA typeⅡ can be significantly decreased after the nusinersen loading dose therapy.
Background Over the past two decades, implementing perioperative feeding protocols in infants with critical congenital heart disease (CCHD) has a certain effect on fostering their physical development and enhancing their clinical outcomes. Nevertheless, the existence of diverse feeding protocols and their appropriate application to different infant populations and feeding stages remains a pending issue.
Objective To evaluate the effect of standardized perioperative feeding protocols on the physical development and clinical outcomes of critically ill infants with CCHD.
Design Systematic review and meta-analysis.
Methods Systematic retrieval was conducted across foreign language databases, including PubMed, Embase, The Cochrane Library, and Web of Science, as well as Chinese databases, such as SinoMed, China National Knowledge Infrastructure, and Wanfang Database. CCHD infants adhering to a standardized feeding protocol during the perioperative period were included. The outcomes evaluated included the incidence of necrotizing enterocolitis (NEC), physical development indicators, hospital length of stay (LOS), ICU LOS and mechanical ventilation duration. The study type consisted of randomized controlled trial (RCT), non-randomized trial, cohort study, case series report, and systematic review/meta-analysis. The literature was searched from the inception of the database up to December 11, 2022. Three individuals conducted literature screening and data extraction based on the predefined criteria. The risk of bias assessment for non-randomized studies and randomized clinical trials was performed using ROBINS-I and ROB 2, respectively. Meta-analysis was carried out using RevMan 5.4 and R version 4.2.2.
Main outcome measures The incidence of NEC.
Results A total of 11 studies conducted in the US from 2000 to 2020 were included. One article employed a non-randomized control trial, 9 used case before-after study design, and 1 used self-before-after study design. The standardized feeding protocol was implemented in 3 studies during the pre-operation period, 4 during the post-operation period, and 5 during the perioperative period. The gestational age of participants ranged from 35 to 41 weeks. Among the included articles, 8 reported the incidence of NEC as the primary outcome. Separate meta-analyses were conducted by different stages of intervention implementation. In a meta-analysis of two articles that implemented standardized protocols during the peri-operation period, the result revealed that there was no statistically significant difference in the incidence of NEC between the two groups［odds ratio (OR)=0.57 (95% CI: 0.19-1.76)］. The results of the other 2 meta-analyses were as follows: one for 3 articles implementing intervention during the pre-operation period showed no statistically significant difference［OR=0.95 (95% CI: 0.48-1.88)］ , and the other for 3 articles during the post-operation also showed no statistically significant difference［OR=0.69 (95% CI: 0.35-1.34)］. Among the 4 articles that reported the weight-for-age z-score at discharge, a meta-analysis of 3 of them found no statistically significant difference between the two groups, WMD=-0.10 (95% CI: -0.37- 0.17). Ten articles reported LOS. A meta-analysis of LOS for intervention during the pre-operation period revealed that LOS was significantly shorter in the study group, WMD=-6.83 (95% CI: -12.13--1.53). However, other meta-analyses of ICU LOS (2 articles) and mechanical ventilation duration (3 articles) all revealed no statistically significant differences between the two groups. The critical appraisal of the included articles indicated that 4 of them were at moderate risk of bias, 6 were at high risk, and 1 was at critical risk.
Conclusions A standardized perioperative feeding protocol implemented in surgical infants with CCHD can not reduce the incidence of NEC, improve the weight-for-age z-score at discharge, and cut down the LOS, ICU LOS, and mechanical ventilation duration.
Background Sitting height/leg length ratio (SH/LL) and Sitting height/height ratio (SH/H) are the important indicators of body proportion assessment. Their scientific assessment will supply valuable information in clinical practices, but the detailed data on their growth reference standards have not been reported.
Objective To construct the SH/LL and SH/H growth charts for Chinese children and adolescents from birth to 18 years.
Design A cross-sectional survey.
Methods Stature and sitting height of 92 494 healthy urban children aged 0-18 years were measured in two national surveys in 2005 in China, which named The National Survey on the Physical Growth and Development of Children in the Nine Cities of China and The Chinese National Survey on Student's Constitution and Health. Leg length was calculated by height minus sitting height and the SH/LL ratio and SH/H ratio were calculated. Growth reference standards of SH/LL and SH/H were constructed using the LMS method.
Main outcome measures Reference values of SH/LL and SH/H.
Results The Z-scores growth charts of SH/LL and SH/H (-3SD, -2SD, -1SD, Median, +1SD, +2SD, +3SD) were constructed. The median of SH/LL declined from birth (2.00) to 13 years in boys (111) and to 11 years in girls (1.13), then increased slightly to the age of 18 (1.16 in boys and 1.18 in girls). A similar growth pattern was found in the SH/H, that is, the median of SH/H was 0.67 at birth and declined to the lowest point (0.53) with age, then increased slightly to 0.54 at 18 years old.
Conclusions The growth charts of SH/LL and SH/H are used in assessing body proportions for Chinese children and adolescent individuals, and they could be used in conjunction with the growth charts of height, weight for Chinese children aged 0-18 years.
Background Prenatal screening for congenital heart disease (CHD) can not only reduce the number of critical birth defects with poor prognosis, but also facilitate the early evaluation, tracking and management of CHD fetuses.
Objective To investigate the diagnostic accuracy of fetal CHD by ultrasound screening in the first and second trimester.
Design Diagnostic test accuracy.
Methods This research covers the pregnant women and their fetuses who registered, received examinations and gave birth in Jiangsu Kunshan Maternity and Child Care Centre between Aug.2018 and Dec. 2021. The fetal or neonatal cardiac ultrasound was used as the gold standard for diagnosis, and the standard to be further tested was the positive ultrasound result in the first and second trimesters.
Main outcome measures Diagnostic accuracy parameters.
Results During the study period, a total of 18 326 pregnant women underwent birth examination in our hospital, among which 3 534 (19.3%) had incomplete delivery data, and 14 960 fetal data were included in the analysis. Among 882 fetuses positive for ultrasound screening in the first and second trimester, 92 were lost to followup (10.2%), and 790 fetuses underwent ultrasound, cardiac ultrasound, or chromosome examination with induced labor and abortion in 182 cases. Of the 608 live births, 103 were diagnosed with CHD by fetal echocardiography and 4 were diagnosed with CHD by postnatal echocardiography.Based on 14 868 cases of fetal CHD screening, 171 cases were true positive, 48 cases were false negative, 14 030 cases were true negative, and 619 cases were false positive. The sensitivity was 78.1%(95%CI:71.9%83.3%), and the specificity was 95.8%(95%CI:95.4%96.1%). The false negative rate was 21.9%(95%CI: 16.7%28.1%).There were 14 078 negative fetuses in the first and second trimester of pregnancy with no lost followup cases, and 505 fetuses in the first and second trimester of pregnancy with positive ultrasound screening but normal results by cardiac ultrasound or chromosome examination. Screened by dual index method after birth, 14 335 negative fetuses and 244 positive fetuses were found, and 52 CHD cases were confirmed by neonatal cardiac ultrasound.In 219 cases of confirmed CHD, (1) CHD featured by atrial septal defect (secondary perforation) and congenital bilobal aortic valve malformation could not be found by fetal ultrasound and needed to be diagnosed by neonatal ultrasound; (2) Among 64 cases of induced labor, the 100% induced labor rate was found in CHD of complex type, right ventricular double outlet, ventricular septal defect (coarctation of aorta), atrial septal dilatation tumor, aortic valve stenosis, aortic arch rupture, pulmonary artery suspension, absence of inferior vena cava, ventricular septal defect (pulmonary stenosis), endocardial fibroplasia, right heart dysplasia, single ventricle, severe tricuspid valve stenosis, right pulmonary artery abnormality originating from the ascending aorta, and 80% induced labor rate was found in CHD of tetralogy of Fallot (8/10), complete atrioventricular septal defect (5/6), complete transposition of great arteries (4/5);(3)There were 9 cases of CHD with chromosomal abnormalities with 2 cases of ventricular septal defect (2.4%), 1 case of complex CHD (5%), 1 case of tetralogy of Fallot (10%), 1 case of right heart dysplasia (50%), and 1 case of single ventricle (100%).
Conclusions After CHD screening in the first and second trimester, even fetuses with negative screening should be screened with two indicators after birth. Fetuses with positive screening should be closely examined with fetal cardiac ultrasound so as to fully realize the integrated prenatal and postpartum management mode of CHD.
