中华儿科杂志最新文献

{"title":"[Clinical evaluation of instantly generated inhaled nitric oxide in the treatment of neonatal pulmonary arterial hypertension].","authors":"C Y Ma, J Du, L Chen, X Y Xiang, M Y Hei, Z X Yang","doi":"10.3760/cma.j.cn112140-20250710-00596","DOIUrl":"https://doi.org/10.3760/cma.j.cn112140-20250710-00596","url":null,"abstract":"<p><p><b>Objective:</b> To explore the efficacy and safety of instantly generated inhaled nitric oxide (iNO) for treating neonatal pulmonary arterial hypertension (PAH) complicated with severe hypoxic respiratory failure. <b>Methods:</b> This single-center, single-arm, prospective study included 32 neonates with PAH complicated with hypoxic respiratory failure who were hospitalized in the neonatal intensive care unit (NICU) of Beijing Children's Hospital Affiliated to Capital Medical University from March 2023 to March 2025 and received immediate iNO generation therapy. The demographic data, maternal pregnancy, mechanical ventilation parameters, arterial blood gas indexes, other hospitalization data and safety indexes of iNO treatment were collected. The time point for starting iNO treatment was set as 0 h, and the observation time points were 1, 6, 12, 24, 48 h after treatment and when iNO treatment was stopped. The positive reaction of iNO treatment was defined as the decrease of oxygenation index (OI)>10% or the increase of arterial partial pressure of oxygen (PaO<sub>2</sub>)>10% after treatment. The OI, mechanical ventilation parameters, arterial blood gas index changes and treatment positive reaction ratio were analyzed to evaluate the effectiveness of iNO treatment, and the nitrogen dioxide concentration, methemoglobin (MetHb) concentration and other indicators were analyzed to evaluate the safety of iNO treatment. Paired <i>t</i> test or Wilcoxon signed rank sum test was used to compare the observation indexes at different treatment times. Friedman test was used to compare the concentration of nitrogen dioxide and MetHb at multiple treatment times. Receiver operating characteristic (ROC) curve was used to analyze the best cut-off value of OI related indexes to distinguish the treatment outcome of iNO. <b>Results:</b> Among 32 neonates, 18 (56%) were males and 14 (44%) were females, the gestational age was 38 (35, 39) weeks, the birth weight was 3.1 (2.3, 3.4) kg, and the age of enrollment was 3 (2, 8) days. The OI and the mean airway pressure at 48 h after treatment were both lower than those at 0 h ((10.4±2.0 <i>vs.</i> 22.6±2.5, 13.0 (12.0, 14.0) <i>vs</i>. 14.0 (13.0, 16.0) cmH<sub>2</sub>O, 1 cmH<sub>2</sub>O=0.098 kPa, both <i>P</i><0.05). The fraction of inspired oxygen at 24 and 48 h after treatment were both lower than those at 0 h (both <i>P</i><0.05). The PaO2 at 6, 12, 24 and 48 h after treatment were all higher than those at 0 h (all <i>P</i><0.05). The proportion of positive reactions to iNO treatment was 20 neonates (63%), 22 neonates (69%), 23 neonates (72%), 23 neonates (72%) and 26 neonates (8%) at 1, 6, 12, 24, 48 h after treatment, respectively. No occurrence of methemoglobinemia, excessive nitrogen dioxide concentration, or device related adverse events were observed. Out of 32 neonates, a total of 24 neonates (75%) were cured or improved and discharged according to medical advice, while 8 neonates (25%) died in the ho","PeriodicalId":60813,"journal":{"name":"中华儿科杂志","volume":"63 11","pages":"1234-1239"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145294426","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Interpretation of the guidelines for the diagnosis and treatment of children with hemophilia (2025)].","authors":"M Zhou, X J Li, R H Wu","doi":"10.3760/cma.j.cn112140-20250721-00664","DOIUrl":"https://doi.org/10.3760/cma.j.cn112140-20250721-00664","url":null,"abstract":"","PeriodicalId":60813,"journal":{"name":"中华儿科杂志","volume":"63 11","pages":"1189-1193"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145294538","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Early differentiation of Kawasaki disease shock syndrome and septic shock in children].","authors":"H Y Ge, S Liu, J Chen, W P Gao, S Y Huang, F Li, F Lyu, D Qu","doi":"10.3760/cma.j.cn112140-20250615-00514","DOIUrl":"https://doi.org/10.3760/cma.j.cn112140-20250615-00514","url":null,"abstract":"<p><p><b>Objective:</b> To explore the differences in early clinical features between Kawasaki disease shock syndrome (KDSS) and septic shock (SS). <b>Methods:</b> A retrospective case-control study was conducted. Clinical data was collected from 64 children who were diagnosed with KDSS or SS and admitted to the Department of Critical Care Medicine of Capital Center for Children's Health, Capital Medical University from January 2018 to February 2025. Mann-Whitney <i>U</i> test, <i>χ</i>² test, or Fisher's exact test were used to compare the differences in clinical features, treatment, and outcomes between children with KDSS and SS. Lasso regression was applied to screen predictive variables, and multivariable logistic regression analysis was performed to identify factors associated with KDSS. Receiver operating characteristic (ROC) curve was used to evaluate the predictive value of parameters for KDSS. <b>Results:</b> Among the 64 children (30 males and 34 females), the age w 3.6 (1.2, 6.5) years old. There were 51 cases in the SS group and 13 cases in the KDSS group. Compared to children with SS, children with KDSS had a longer pre-shock fever duration, lower lactate levels and serum albumin levels, and higher soluble interleukin-2 receptor (sIL-2R) levels (all <i>P</i><0.05). Additionally, they exhibited a higher incidence of coronary involvement, pericardial effusion, and ascites, a higher utilization rate of intravenous immunoglobulin, and a lower utilization rate of invasive mechanical ventilation (all <i>P</i><0.05). There was no significant difference in in-hospital mortality between KDSS and SS (<i>P</i>=0.574). Multivariate logistic regression analysis identified pre-shock fever duration and sIL-2R as independent factors associated with KDSS (<i>OR</i>=1.52 and 1.54 (per 1 000 U increase), 95%<i>CI</i> 1.12-2.05 and 1.06-2.24, respectively; botb <i>P</i><0.05). ROC curve analysis showed that the areas under the curve for pre-shock fever duration and sIL-2R in identifying KDSS were 0.83 (95%<i>CI</i> 0.73-0.94, <i>P</i>=0.001) and 0.70 (95%<i>CI</i> 0.53-0.87, <i>P</i>=0.042), respectively. The optimal cutoff values were 3.5 d and 3.8×10⁶ U/L, with sensitivities of 0.91 and 0.82, and specificities of 0.71 and 0.62, respectively. <b>Conclusions:</b> Children with KDSS have higher incidences of coronary involvement, pericardial effusion, and ascites compared to those with SS. Pre-shock fever duration and sIL-2R may serve as potential early indicators for distinguishing KDSS from SS.</p>","PeriodicalId":60813,"journal":{"name":"中华儿科杂志","volume":"63 11","pages":"1229-1233"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145294566","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Clinical characteristics analysis of Chikungunya fever in children].","authors":"J Bai, X G Ye, J X Chen, W H Zhou, S T Gong, W Y Luo, H Y Liu, M H Li, Y Q Zhang, T Z Lu, Y Y Zheng, Z G Liu","doi":"10.3760/cma.j.cn112140-20250814-00756","DOIUrl":"https://doi.org/10.3760/cma.j.cn112140-20250814-00756","url":null,"abstract":"<p><p><b>Objective:</b> To investigate the clinical characteristics of children with Chikungunya fever. <b>Methods:</b> This retrospective cohort study analyzed clinical data of 91 children with Chikungunya fever at the Department of Pediatrics, Foshan llbnen and Children Hospital between July 2025 and August 2025. The patients were divided into four groups based on onset-age: 0-<1 year, 1-<3 years, 3-<6 years, and 6-14 years. One-way ANOVA and chi-square tests were used to compare the clinical features of children with Chikungunya fever at different ages. <b>Results:</b> Among the 91 children with chikungunya fever, 55 were male and 36 were female, with an onset age of 6 (2, 11) years, age groups comprised 0-<1 year (10 cases), 1-<3 years (13 cases), 3-<6 years (17 cases) and 6-14 years (51 cases). Fever occurred in 87 cases (96%), with 50 cases (57%) had high fever. Skin rash was observed in 89 cases (98%), and 60 cases (67%) had a generalized rash. Joint pain was reported in 57 cases (63%), among which 35 cases (61%) had pain in two or more locations, with the knee involved in 21 cases (37%), the ankle in 15 cases (26%), and the wrist in 6 cases (11%).The knee was the most commonly affected joint 21 cases (37%), followed by the ankle 15 cases (26%) and wrist 6 cases (10%). Joint ultrasound was performed in 31 cases (34%), all showed joint effusion, including 8 cases (26%) without complaints of joint pain. The incidence of high fever was significantly lower in the 3-<6 years and 6-14 years groups compared to the 0-<1 year group (both <i>P</i><0.05). The 6-14 years group also had a lower incidence of high fever than the 1-<3 years group (<i>P</i><0.05). The 1-<3 years group had longer duration of fever than the 3-<6 years and 6-14 years groups (both <i>P</i><0.05). The incidence of joint pain was higher in the 3-<6 years and 6-14 years groups compared to the 1-<3 years group (both <i>P</i><0.05), and higher in the 6-14 years group than in the 3-<6 years group (<i>P</i>=0.007). Among all 91 children, 22 cases (24%) had abnormal liver function, 49 cases (54%) showed elevated lactate dehydrogenase (LDH), and 2 cases (2%) had elevated creatine kinase. The proportions of elevated aspartate aminotransferase (AST) and LDH were higher in the 0-<1 year and 1-<3 years groups compared to the 3-<6 years and 6-14 years groups (all <i>P</i><0.05). <b>Conclusions:</b> The clinical characteristics of children with Chikungunya fever vary among children of different ages. Children in the 0-<3 years are more prone to high fever with longer duration and generalized maculopapular rash, while the children in the 6-14 years have have a higher proportion of joint pain, and joint ultrasound revealed effusion in all examined children. AST and LDH levels are elevated in the 0-<3 years groups.</p>","PeriodicalId":60813,"journal":{"name":"中华儿科杂志","volume":"63 11","pages":"1224-1228"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145294429","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Recent advances in novel small molecule drugs for pediatric inflammatory bowel disease].","authors":"J Y He, S N Shu","doi":"10.3760/cma.j.issn112140-20250305-00176","DOIUrl":"https://doi.org/10.3760/cma.j.issn112140-20250305-00176","url":null,"abstract":"","PeriodicalId":60813,"journal":{"name":"中华儿科杂志","volume":"63 11","pages":"1266-1270"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145294534","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Long-term efficacy observation of nicotinamide in the treatment of early-onset progressive encephalopathy with brain edema and (or) leukoencephalopathy-2 caused by NAXD gene variation].","authors":"C L Xu, F Fang, J Zhou, H Wang, W H Zhang, S Gong, H F Jiang, Z M Liu, J W Li","doi":"10.3760/cma.j.cn112140-20250327-00260","DOIUrl":"https://doi.org/10.3760/cma.j.cn112140-20250327-00260","url":null,"abstract":"<p><p><b>Objective:</b> To summarize the long-term efficacy of nicotinamide in treating pediatric early-onset progressive encephalopathy with brain edema and (or) leukoencephalopathy-2 (PEBEL2) caused by NAXD gene variation<b>.</b> <b>Methods:</b> This was a case report conducted from February 2019 to January 2025. The long-term efficacy of nicotinamide was observed by following up a child with PEBEL2 who received the treatment in the Department of Neurology, Beijing Children's Hospital Affiliated to Capital Medical University. The clinical data included changes in skin lesions, neurological symptoms. The modified Rankin scale (mRS) was used to evaluate the recovery of neurological function. <b>Results:</b> A boy was diagnosed with PEBEL2 caused by NAXD gene variation via genetic testing at Beijing Children's Hospital Affiliated to Capital Medical University in February 2019, when he was 4 years and 6 months of age. Immediately after diagnosis, nicotinamide treatment was initiated at an initial dose of 100 mg/d, which was increased by 100 mg per week and gradually increased to 500 mg/d; meanwhile, other therapeutic drugs were gradually discontinued. After 1 year and 8 months of treatment, the child's skin lesions had completely resolved; at the 2-year follow-up, dystonia in both upper limbs and swallowing dysfunction was alleviated significantly; by 2.5-year follow-up, his cognitive function also showed improvement. When the child was treated with 500 mg/d for 3 years, a rash appeared around the mouth. After the dose was reduced to 250 mg/d, the rash resolved, and the dose of 250 mg/d was maintained until the last follow-up. At the last follow-up in January 2025, the child was 10 years and 5 months of age. His mRS score decreased from 5 (before treatment) to 4. During the 6-year of continuous nicotinamide treatment, the child's condition remained stable without progression. Drug-related skin rashes occurred, but no severe drug-related adverse reactions were observed. <b>Conclusions:</b> PEBEL2 is a treatable mitochondrial disease. Nicotinamide treatment can effectively improve skin lesions and neurological symptoms in PEBEL2 patients, and the long-term administration demonstrates a favorable safety profile.</p>","PeriodicalId":60813,"journal":{"name":"中华儿科杂志","volume":"63 11","pages":"1246-1249"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145294555","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Guidelines for the diagnosis and treatment of children with hemophilia (2025)].","authors":"","doi":"10.3760/cma.j.cn112140-20250619-00534","DOIUrl":"https://doi.org/10.3760/cma.j.cn112140-20250619-00534","url":null,"abstract":"","PeriodicalId":60813,"journal":{"name":"中华儿科杂志","volume":"63 11","pages":"1170-1181"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145294541","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[The efficacy of blinatumomab in the treatment of pediatric B-cell acute lymphoblastic leukemia: a multicenter study].","authors":"W L Yan, J Lu, H Wang, L H Yu, H D Feng, B Li, W W Jia, J Wang, W T Hu, X Tang, J Fan, Y J Guan, X L Li, Y L You, Y M Tang, X J Xu","doi":"10.3760/cma.j.cn112140-20250330-00267","DOIUrl":"https://doi.org/10.3760/cma.j.cn112140-20250330-00267","url":null,"abstract":"&lt;p&gt;&lt;p&gt;&lt;b&gt;Objective:&lt;/b&gt; To investigate the efficacy and toxicity of blinatumomab in the first-line and second-line treatment of pediatric B-cell acute lymphoblastic leukemia (B-ALL). &lt;b&gt;Methods:&lt;/b&gt; A multi-center retrospective cohort study was conducted to analyze clinical data from 323 pediatric B-ALL patients treated with blinatumomab across 14 hospitals in China from May 2021 to July 2023. Patients were divided into four groups based on the treatment phase and disease status when blinatumomab used: relapsed/refractory group, post-consolidation minimal residual disease (MRD)-positive group, early MRD-positive group, and MRD-negative group. Blinatumomab for the relapsed/refractory group was considered as second-line treatment, while the other three groups as first-line treatment. The remission rate, the MRD negativity rate after treatment, the survival rates and the incidence of severe adverse events were compared across these groups. Patients who received blinatumomab for more than 7 days were included in the efficacy analysis. Survival analysis was performed using the Kaplan-Meier method, and Log-Rank test was used to compare the survival rates among groups. &lt;b&gt;Results:&lt;/b&gt; Among the 323 patients, 191 (59.1%) were male, with the age of 6.2 years. There were 117 patients in the relapsed/refractory group, 62 cases in the post-consolidation MRD-positive group group, 43 cases in the early MRD-positive group, and 101 cases in the MRD negative group. In the relapsed/refractory group, the complete remission rate and MRD negativity rate after one course of blinatumomab were 71.4% (35/49) and 81.5% (75/92) for the 49 children without complete remission and the 92 children with flow cytometry-positive MRD, respectively. In the post-consolidation MRD-positive group, the MRD negativity rates after one course of blinatumomab were 100.0% (27/27), 12/16, and 9/19 for patients with MRD positivity detected by flow cytometry, polymerase chain reaction, and next-generation sequencing, respectively. In the early MRD-positive group, the MRD negativity rates were 96.7% (29/30) and 9/9 for flow cytometry and next-generation sequencing, respectively. The 2-year overall survival rate and event-free survival rate for the 319 children evaluable for efficacy were (90.6±1.7)% and (87.6±1.9)%, respectively, with the relapsed/refractory group showing significantly lower overall survival rates and event-free survival rate compared to the other groups(&lt;i&gt;χ&lt;/i&gt;&lt;sup&gt;2&lt;/sup&gt;=21.40, 26.21,both &lt;i&gt;P&lt;/i&gt;&lt;0.001). Grade 3 or higher adverse events occurred in 128 cases (39.6%), with hematological toxicity observed in 101 cases, while cytokine release syndrome (CRS), infection, and neurotoxicity occurred in 11, 26 and 8 cases, respectively. In addition, there were statistically significant differences in the grade 3 or higher CRS among the four groups (&lt;i&gt;χ&lt;/i&gt;&lt;sup&gt;2&lt;/sup&gt;=8.03, &lt;i&gt;P&lt;/i&gt;&lt;0.05). &lt;b&gt;Conclusion:&lt;/b&gt; Blinatumomab can clear MRD more effectively and achieve superior surviva","PeriodicalId":60813,"journal":{"name":"中华儿科杂志","volume":"63 11","pages":"1194-1200"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145294649","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Research progress of chimeric antigen receptor T-cell therapy in the treatment of neuroblastoma].","authors":"G T He, Q Wei, B H Wu, X Li, S Yang","doi":"10.3760/cma.j.cn112140-20250507-00389","DOIUrl":"https://doi.org/10.3760/cma.j.cn112140-20250507-00389","url":null,"abstract":"","PeriodicalId":60813,"journal":{"name":"中华儿科杂志","volume":"63 11","pages":"1258-1261"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145294556","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"[Expert consensus on the management of obesity in infants and young children​​ (2025)].","authors":"","doi":"10.3760/cma.j.cn112140-20250606-00482","DOIUrl":"https://doi.org/10.3760/cma.j.cn112140-20250606-00482","url":null,"abstract":"","PeriodicalId":60813,"journal":{"name":"中华儿科杂志","volume":"63 11","pages":"1182-1188"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145294580","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}