中国当代儿科杂志最新文献

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[Relationship between common myositis-specific antibodies and clinical features in children with juvenile dermatomyositis]. 【小儿皮肌炎常见肌炎特异性抗体与临床特征的关系】。
中国当代儿科杂志 Pub Date : 2025-07-15 DOI: 10.7499/j.issn.1008-8830.2502077
Su-Yun Cheng, Jia-Min Lu, Feng Li
{"title":"[Relationship between common myositis-specific antibodies and clinical features in children with juvenile dermatomyositis].","authors":"Su-Yun Cheng, Jia-Min Lu, Feng Li","doi":"10.7499/j.issn.1008-8830.2502077","DOIUrl":"10.7499/j.issn.1008-8830.2502077","url":null,"abstract":"<p><strong>Objectives: </strong>To investigate the distribution of myositis-specific antibodies (MSA) in juvenile dermatomyositis (JDM) and the relationship between MSA and clinical features of JDM.</p><p><strong>Methods: </strong>Clinical data of 72 children with JDM hospitalized from January 2020 to April 2025 were reviewed retrospectively, all of whom had been tested for MSA. The relationship between common MSA subtypes and clinical features was analyzed.</p><p><strong>Results: </strong>Among the 72 children, 45 (62%) were positive for MSA, including 27 anti-NXP2-positive cases (38%), 10 anti-MDA5-positive cases (14%), and 3 anti-cN1A-positive cases (4%). Compared with the MSA-negative group, the anti-MDA5-positive patients showed significantly higher incidence rates of fever, arthritis, and interstitial lung disease (<i>P</i><0.05). The anti-NXP2-positive patients exhibited significantly higher incidence rates of calcinosis, fever, soft tissue edema, and interstitial lung disease than the MSA-negative patients (<i>P</i><0.05). Compared with the anti-MDA5-positive group and MSA-negative group, the anti-NXP2-positive group had significantly higher levels of creatine kinase and creatine kinase isoenzyme (<i>P</i><0.017) and a significantly lower score of the Childhood Myositis Assessment Scale (<i>P</i><0.017).</p><p><strong>Conclusions: </strong>The positive rate of MSA is high in children with JDM, with different subtypes correlating with specific clinical manifestations and organ involvement. Detection of MSA is crucial for diagnosis and clinical management of JDM.</p>","PeriodicalId":39792,"journal":{"name":"中国当代儿科杂志","volume":"27 9","pages":"1076-1081"},"PeriodicalIF":0.0,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12447943/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145082026","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
[Genetic profiling and intervention strategies for phenylketonuria in Gansu, China: an analysis of 1 159 cases]. [中国甘肃1 159例苯丙酮尿的基因分析及干预策略]。
中国当代儿科杂志 Pub Date : 2025-07-15 DOI: 10.7499/j.issn.1008-8830.2502040
Chuan Zhang, Pei Zhang, Bing-Bo Zhou, Xing Wang, Lei Zheng, Xiu-Jing Li, Jin-Xian Guo, Pi-Liang Chen, Ling Hui, Zhen-Qiang DA, You-Sheng Yan
{"title":"[Genetic profiling and intervention strategies for phenylketonuria in Gansu, China: an analysis of 1 159 cases].","authors":"Chuan Zhang, Pei Zhang, Bing-Bo Zhou, Xing Wang, Lei Zheng, Xiu-Jing Li, Jin-Xian Guo, Pi-Liang Chen, Ling Hui, Zhen-Qiang DA, You-Sheng Yan","doi":"10.7499/j.issn.1008-8830.2502040","DOIUrl":"10.7499/j.issn.1008-8830.2502040","url":null,"abstract":"<p><strong>Objectives: </strong>To investigate the molecular epidemiology of children with phenylketonuria (PKU) in Gansu, China, providing foundational data for intervention strategies.</p><p><strong>Methods: </strong>A retrospective analysis was conducted on 1 159 PKU families who attended Gansu Provincial Maternity and Child Care Hospital from January 2012 to December 2024. Sanger sequencing, multiplex ligation-dependent probe amplification, whole exome sequencing, and deep intronic variant analysis were used to analyze the <i>PAH</i> gene.</p><p><strong>Results: </strong>For the 1 159 children with PKU, 2 295 variants were identified in 2 318 alleles, resulting in a detection rate of 99.01%. The detection rates were 100% (914/914) in 457 classic PKU families, 99.45% (907/912) in 456 mild PKU families, and 96.34% (474/492) in 246 mild hyperphenylalaninemia families. The 2 295 variants detected comprised 208 distinct mutation types, among which c.728G>A (14.95%, 343/2 295) had the highest frequency, followed by c.611A>G (4.88%, 112/2 295) and c.721C>T (4.79%, 110/2 295). The cumulative frequency of the top 23 hotspot variants reached 70.28% (1 613/2 295), and most variant alleles were detected in exon 7 (29.19%, 670/2 295).</p><p><strong>Conclusions: </strong>Deep intronic variant analysis of the <i>PAH</i> gene can improve the genetic diagnostic rate of PKU. The development of targeted detection kits for <i>PAH</i> hotspot variants may enable precision screening programs and enhance preventive strategies for PKU.</p>","PeriodicalId":39792,"journal":{"name":"中国当代儿科杂志","volume":"27 7","pages":"808-814"},"PeriodicalIF":0.0,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12291574/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144691837","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
[Research progress on the cGAS-STING signaling pathway in immune-mediated inflammatory diseases in children]. [cGAS-STING信号通路在儿童免疫介导炎性疾病中的研究进展]。
中国当代儿科杂志 Pub Date : 2025-07-15 DOI: 10.7499/j.issn.1008-8830.2412098
Xin-Yue Wei, Xiao-Juan Gong, Hong Ji
{"title":"[Research progress on the cGAS-STING signaling pathway in immune-mediated inflammatory diseases in children].","authors":"Xin-Yue Wei, Xiao-Juan Gong, Hong Ji","doi":"10.7499/j.issn.1008-8830.2412098","DOIUrl":"10.7499/j.issn.1008-8830.2412098","url":null,"abstract":"<p><p>The cyclic GMP-AMP synthase (cGAS)-stimulator of interferon gene (STING) signaling pathway is a crucial component of the immune system. It detects abnormal cytosolic double-stranded DNA and promotes the expression of type I interferons and other inflammatory factors, thereby protecting the body from pathogenic infections. In children, an immature immune system or genetic mutations can lead to immune dysregulation, increasing the risk of autoimmune diseases (AID) and autoinflammatory diseases. Recent studies have shown that aberrant activation of the cGAS-STING signaling pathway is associated with the development of AID and autoinflammatory diseases in children. This review summarizes the research progress on the cGAS-STING signaling pathway in childhood AID and autoinflammatory diseases, aiming to provide new directions for clinical diagnosis and treatment.</p>","PeriodicalId":39792,"journal":{"name":"中国当代儿科杂志","volume":"27 7","pages":"881-887"},"PeriodicalIF":0.0,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12291564/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144691843","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
[Febrile infection-related epilepsy syndrome caused by hemophagocytic lymphohistiocytosis: a case report]. 噬血细胞性淋巴组织细胞增多症致发热性感染相关性癫痫综合征1例。
中国当代儿科杂志 Pub Date : 2025-07-15 DOI: 10.7499/j.issn.1008-8830.2503079
Xiao-Lu Deng, Li-Fen Yang, Xia Wang, Hui Zhang, Jian He, Jing Peng
{"title":"[Febrile infection-related epilepsy syndrome caused by hemophagocytic lymphohistiocytosis: a case report].","authors":"Xiao-Lu Deng, Li-Fen Yang, Xia Wang, Hui Zhang, Jian He, Jing Peng","doi":"10.7499/j.issn.1008-8830.2503079","DOIUrl":"10.7499/j.issn.1008-8830.2503079","url":null,"abstract":"<p><p>The patient was a girl, aged 10 years, who was admitted due to fever for 5 days and pancytopenia in peripheral blood for 2 days. Bone marrow examination showed the presence of phagocytic activity, and peripheral blood tests showed pancytopenia, an increase in ferritin, a reduction in fibrinogen, increases in triglyceride and sCD25, and a reduction in natural killer cell activity, which led to the diagnosis of hemophagocytic lymphohistiocytosis (HLH). On the day of admission, the child developed convulsions and rapidly progressed to refractory status epilepticus, which was consistent with the manifestations of febrile infection-related epilepsy syndrome. HLH was controlled after active immunotherapy, with the sequela of refractory epilepsy, and her cognitive function was essentially within normal limits. This article reports the condition of febrile infection-related epilepsy syndrome caused by HLH for the first time in China, in order to improve the awareness of this disease among clinicians.</p>","PeriodicalId":39792,"journal":{"name":"中国当代儿科杂志","volume":"27 7","pages":"864-869"},"PeriodicalIF":0.0,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12291570/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144691836","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
[Dynamic changes in serum microRNA-15b and vascular endothelial growth factor in preterm infants with bronchopulmonary dysplasia and their value in assessing neurodevelopment]. [支气管肺发育不良早产儿血清microRNA-15b和血管内皮生长因子的动态变化及其在神经发育评估中的价值]。
中国当代儿科杂志 Pub Date : 2025-07-15 DOI: 10.7499/j.issn.1008-8830.2412039
Qian Chen, Pei-Pei Zhang, Qing-Hua Lu, Zhen-Xia Wan, Lei Huang
{"title":"[Dynamic changes in serum microRNA-15b and vascular endothelial growth factor in preterm infants with bronchopulmonary dysplasia and their value in assessing neurodevelopment].","authors":"Qian Chen, Pei-Pei Zhang, Qing-Hua Lu, Zhen-Xia Wan, Lei Huang","doi":"10.7499/j.issn.1008-8830.2412039","DOIUrl":"10.7499/j.issn.1008-8830.2412039","url":null,"abstract":"<p><strong>Objectives: </strong>To investigate the dynamic changes in serum microRNA-15b (miR-15b) and vascular endothelial growth factor (VEGF) in preterm infants with mild or moderate-to-severe bronchopulmonary dysplasia (BPD), as well as their value in assessing short-term neurodevelopment.</p><p><strong>Methods: </strong>A retrospective analysis was conducted on the medical data of 156 preterm infants with BPD who were admitted to the neonatal intensive care unit from January 2020 to February 2023. According to the severity of BPD, they were divided into a mild group (<i>n</i>=88) and a moderate-to-severe group (<i>n</i>=68). Serum levels of miR-15b and VEGF were measured on postnatal days 1, 7, 14, and 28. Repeated measures analysis of variance was used to assess the dynamic changes in serum levels of miR-15b and VEGF. The mediating effect of VEGF between miR-15b and short-term neurological development was tested and analyzed using the stepwise regression method and the Bootstrap method. Logistic regression analysis was used to identify factors influencing adverse neurodevelopmental outcomes.</p><p><strong>Results: </strong>In the mild group, there was a significant reduction in the serum level of miR-15b and a significant increase in VEGF over time (<i>P</i><0.05), while in the moderate-to-severe group, there was a significant increase in miR-15b and a significant reduction in VEGF over time (<i>P</i><0.05). Serum miR-15b and VEGF levels were important factors influencing neurodevelopmental outcomes, showing independent correlations (<i>P</i><0.001). The mediating effect analysis indicated that miR-15b indirectly affected short-term neurodevelopment by inhibiting VEGF expression [indirect effect: -0.705 (95%<i>CI</i>: -1.178 to -0.372)], with the indirect effect accounting for 54.36% of the total effect.</p><p><strong>Conclusions: </strong>There are different changing trends in serum levels of miR-15b and VEGF in preterm infants with mild and moderate-to-severe BPD. miR-15b primarily influences neurodevelopment through VEGF.</p>","PeriodicalId":39792,"journal":{"name":"中国当代儿科杂志","volume":"27 9","pages":"1062-1070"},"PeriodicalIF":0.0,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12447931/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145081862","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
[Clinical and genetic features of 5 neonates with centronuclear myopathy caused by MTM1 gene variation]. [5例新生儿MTM1基因变异致核中心性肌病的临床及遗传特点]。
中国当代儿科杂志 Pub Date : 2025-07-15 DOI: 10.7499/j.issn.1008-8830.2501068
Tian Xie, Jia-Jing Ge, Zi-Ming Zhang, Ding-Wen Wu, Yan-Ping Xu, Li-Ping Shi, Xiao-Lu Ma, Zheng Chen
{"title":"[Clinical and genetic features of 5 neonates with centronuclear myopathy caused by <i>MTM1</i> gene variation].","authors":"Tian Xie, Jia-Jing Ge, Zi-Ming Zhang, Ding-Wen Wu, Yan-Ping Xu, Li-Ping Shi, Xiao-Lu Ma, Zheng Chen","doi":"10.7499/j.issn.1008-8830.2501068","DOIUrl":"10.7499/j.issn.1008-8830.2501068","url":null,"abstract":"<p><strong>Objectives: </strong>To study clinical manifestations and gene mutation features of neonates with centronuclear myopathy.</p><p><strong>Methods: </strong>A retrospective analysis was conducted on the medical data of 5 neonates with centronuclear myopathy diagnosed in the Neonatal Intensive Care Unit of Children's Hospital, Zhejiang University School of Medicine from January 2020 to August 2024. The data included gender, gestational age, birth weight, Apgar score, clinical manifestations, creatine kinase level, electromyography, genetic testing results and the outcomes of the infants.</p><p><strong>Results: </strong>All 5 male neonates had a history of postpartum asphyxia and resuscitation. They all presented with hypotonia, myasthenia, and respiratory failure; two neonates also had swallowing dysfunction. Of the five neonates, three had normal creatine kinase levels, while two had slightly elevated levels. Electromyography was performed for three neonates, among whom two had myogenic damage. <i>MTM1</i> gene mutations were identified by genetic testing in all five neonates, including two nonsense mutations and three missense mutations, among which one variant had not been previously reported. Four mutations were inherited from the mother, and the other one was a <i>de novo</i> mutation. The five neonates showed no clinical improvement following treatment, failed weaning from mechanical ventilation, and ultimately died after withdrawal of life-sustaining therapy.</p><p><strong>Conclusions: </strong>Centronuclear myopathy caused by <i>MTM1</i> gene mutation often has a severe phenotype and a poor prognosis, and it should be considered for neonates with hypotonia and myasthenia after birth. Genetic testing should be performed as soon as possible.</p>","PeriodicalId":39792,"journal":{"name":"中国当代儿科杂志","volume":"27 9","pages":"1071-1075"},"PeriodicalIF":0.0,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12447940/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145081934","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
[Non-Down-syndrome-related acute megakaryoblastic leukemia in children: a clinical analysis of 17 cases]. 儿童非唐氏综合征相关性急性巨核母细胞白血病17例临床分析
中国当代儿科杂志 Pub Date : 2025-07-15 DOI: 10.7499/j.issn.1008-8830.2502082
Ding-Ding Cui, Ye-Qing Tao, Xiao-Pei Jia, An-Na Lian, Qiu-Xia Fan, Dao Wang, Xue-Ju Xu, Guang-Yao Sheng, Chun-Mei Wang
{"title":"[Non-Down-syndrome-related acute megakaryoblastic leukemia in children: a clinical analysis of 17 cases].","authors":"Ding-Ding Cui, Ye-Qing Tao, Xiao-Pei Jia, An-Na Lian, Qiu-Xia Fan, Dao Wang, Xue-Ju Xu, Guang-Yao Sheng, Chun-Mei Wang","doi":"10.7499/j.issn.1008-8830.2502082","DOIUrl":"10.7499/j.issn.1008-8830.2502082","url":null,"abstract":"<p><strong>Objectives: </strong>To investigate the clinical features and prognosis of children with non-Down-syndrome-related acute megakaryoblastic leukemia (non-DS-AMKL).</p><p><strong>Methods: </strong>A retrospective analysis was conducted on the medical data of 17 children with non-DS-AMKL who were admitted to Children's Hospital of The First Affiliated Hospital of Zhengzhou University from January 2013 to December 2023, and their clinical features, treatment, and prognosis were summarized.</p><p><strong>Results: </strong>Among the 17 children with non-DS-AMKL, there were 8 boys and 9 girls. Fourteen patients had an onset age of less than 36 months, with a median age of 21 months (range:13-145 months). Immunophenotyping results showed that 16 children were positive for CD61 and 13 were positive for CD41. The karyotype analysis was performed on 16 children, with normal karyotype in 6 children and abnormal karyotype in 9 children, among whom 5 had complex karyotype and 1 had no mitotic figure. Detected fusion genes included <i>EVI1</i>, <i>NUP98-KDM5A</i>, <i>KDM5A-MIS18BP1</i>, <i>C22orf34-BRD1</i>, <i>WT1</i>, and <i>MLL-AF9</i>. Genetic alterations included <i>TET2</i>, <i>D7S486</i> deletion (suggesting 7q-), <i>CSF1R</i> deletion, and <i>PIM1</i>. All 17 children received chemotherapy, among whom 16 (94%) achieved complete remission after one course of induction therapy, and 1 child underwent hematopoietic stem cell transplantation (HSCT) and remained alive and disease-free. Of all children, 7 experienced recurrence, among whom 1 child received HSCT and died of graft-versus-host disease. At the last follow-up, six patients remained alive and disease-free.</p><p><strong>Conclusions: </strong>Non-DS-AMKL primarily occurs in children between 1 and 3 years of age. The patients with this disorder have a high incidence rate of chromosomal abnormalities, with complex karyotypes in most patients. Some patients harbor fusion genes or gene mutations. Although the initial remission rate is high, the long-term survival rate remains low.</p>","PeriodicalId":39792,"journal":{"name":"中国当代儿科杂志","volume":"27 9","pages":"1113-1118"},"PeriodicalIF":0.0,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12447936/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145081996","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
[Guideline for the diagnosis and treatment of common neonatal diseases in primary healthcare institutions: neonatal transport (2025)]. [初级卫生保健机构常见新生儿疾病诊断和治疗指南:新生儿转运(2025年)]。
中国当代儿科杂志 Pub Date : 2025-07-15 DOI: 10.7499/j.issn.1008-8830.2412152
{"title":"[Guideline for the diagnosis and treatment of common neonatal diseases in primary healthcare institutions: neonatal transport (2025)].","authors":"","doi":"10.7499/j.issn.1008-8830.2412152","DOIUrl":"10.7499/j.issn.1008-8830.2412152","url":null,"abstract":"<p><p>Neonatal transport is a crucial aspect of clinical work in neonatology, aimed at timely and safely transferring high-risk neonates from birth facilities or primary healthcare institutions to neonatal centers equipped for critical care. This ensures timely diagnosis and treatment, thereby reducing mortality and complications and improving outcomes. Currently, there is significant regional variation in neonatal transport practices across China. In response, the Subspecialty Group of Neonatology of Society of Pediatrics of Chinese Medical Association and the Editorial Board of <i>Chinese Journal of Contemporary Pediatrics</i> have jointly developed the \"Guideline for the diagnosis and treatment of common neonatal diseases in primary healthcare institutions: neonatal transport (2025)\". This guideline addresses 10 clinical issues related to neonatal transport and formulates 18 recommendations based on the best available evidence and expert consensus. It aims to provide a systematic approach to neonatal transport in primary care settings, tailored to the national context of China, offering guidance and decision-making support for primary healthcare providers.</p>","PeriodicalId":39792,"journal":{"name":"中国当代儿科杂志","volume":"27 7","pages":"759-769"},"PeriodicalIF":0.0,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12291573/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144691838","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
[Application of umbilical cord mesenchymal stem cells in the treatment of severe immune-mediated thrombocytopenia after allogeneic hematopoietic stem cell transplantation in children]. 【脐带间充质干细胞在治疗儿童异基因造血干细胞移植后严重免疫介导性血小板减少症中的应用】。
中国当代儿科杂志 Pub Date : 2025-07-15 DOI: 10.7499/j.issn.1008-8830.2503173
Bo Zhang, Zuo Luan, Xiang-Feng Tang, Nan-Hai Wu
{"title":"[Application of umbilical cord mesenchymal stem cells in the treatment of severe immune-mediated thrombocytopenia after allogeneic hematopoietic stem cell transplantation in children].","authors":"Bo Zhang, Zuo Luan, Xiang-Feng Tang, Nan-Hai Wu","doi":"10.7499/j.issn.1008-8830.2503173","DOIUrl":"10.7499/j.issn.1008-8830.2503173","url":null,"abstract":"<p><p>This report describes two cases of severe immune-mediated thrombocytopenia after allogeneic hematopoietic stem cell transplantation (HSCT) who were treated with umbilical cord mesenchymal stem cells (UC-MSCs). Case 1 was a child with severe aplastic anemia who underwent haploidentical bone marrow and peripheral blood HSCT, with a chimerism rate of 99.8% on day +25 and severe immune-mediated thrombocytopenia on day +60. After intravenous immunoglobulin (IVIG) pulse therapy, platelet count increased temporarily but then decreased, while cyclosporine, methylprednisolone, and rituximab had a poor therapeutic effect. Case 2 was a child with Gaucher's disease who underwent unrelated umbilical cord blood HSCT, with a chimerism rate of 96.35% on day +41 and severe immune-mediated thrombocytopenia on day +153. After three sessions of IVIG pulse therapy, the platelet count increased initially but subsequently decreased. Therapies with dexamethasone, prednisone, cyclosporine, and recombinant human thrombopoietin also yielded a poor response. Both children received three sessions of UC-MSCs infusion, and platelet counts increased and were subsequently maintained within the normal range. Case 1 has been followed up for 10 years and remains in disease-free survival. UC-MSCs infusion may be effective for severe immune-mediated thrombocytopenia that is unresponsive to first- and second-line therapies after HSCT and could potentially improve the quality of life and disease-free survival rate.</p>","PeriodicalId":39792,"journal":{"name":"中国当代儿科杂志","volume":"27 9","pages":"1128-1133"},"PeriodicalIF":0.0,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12447928/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145081790","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
[Granulomatous primary central nervous system vasculitis in a child]. [儿童原发性肉芽肿性中枢神经系统血管炎]。
中国当代儿科杂志 Pub Date : 2025-07-15 DOI: 10.7499/j.issn.1008-8830.2503053
Ya-Nan Zhang, Chang-Hong Ding, Shu-Hong Ren, Wei-Hua Zhang, Fang Liu, Nan Zhang, Yu-Juan Zhang
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