World Journal of Pediatrics最新文献

{"title":"Management of pulmonary thromboembolism in children: an evidence-based expert consensus.","authors":"Li-Nan Zeng, Ying-Xue Zou, Hai-Lin Zhang, Li-Na Chen, De-Hui Chen, Xin-Xin Chen, Xing Chen, Zhi-Min Chen, Xiao-Yan Dong, Liang Huang, Yi Ji, Yong-Mei Jiang, Zhi-Ping Li, En-Mei Liu, Shu-Hua Luo, Xiao-Feng Ni, Guang-Min Nong, Yun Peng, Su-Yun Qian, Tian-You Wang, Xin-Yu Yuan, Hao Zhang, Hong Zhang, Xiao-Bo Zhang, De-Yu Zhao, Shun-Ying Zhao, Xiu-Fang Zhao, Kai-Yu Zhou, Quan Lu, Ling-Li Zhang, Han-Min Liu","doi":"10.1007/s12519-025-00987-3","DOIUrl":"10.1007/s12519-025-00987-3","url":null,"abstract":"<p><strong>Background: </strong>Pulmonary thromboembolism is rare in children, but can be life-threatening. Timely diagnosis and treatment of pulmonary thromboembolism are crucial for reducing mortality associated with pulmonary thromboembolism in children. While guidelines for pulmonary thromboembolism in adults are available, guidelines for standardized diagnosis and management of pulmonary thromboembolism in children are not. This expert consensus aims to provide recommendations for the management of pulmonary thromboembolism in children based on the current best available evidence.</p><p><strong>Data sources: </strong>Following the World Health Organization Handbook for Guideline Development, the expert panel consisted of 30 members from different clinical areas. The panel identified clinical questions through systematic reviews and expert discussions, systematically reviewed evidence on pulmonary thromboembolism in children, and evaluated the quality of the evidence using the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach. Using the GRADE Evidence to Decision Framework, the panel made recommendations, considering the effects of interventions, resource use, values and preferences, equity, acceptability, and feasibility.</p><p><strong>Results: </strong>The epidemiology, classification, and pathophysiology characteristics are summarized. The expert panel developed 33 recommendations addressing 20 questions related to diagnosis steps, treatment approaches such as anticoagulant therapy, thrombolysis therapy, catheter-based interventional therapy, surgical embolectomy, multidisciplinary team, and treatment of patients with comorbidities, prognosis, education, as well as follow-up. Among these, 18 are weak recommendations based on very low quality evidence, and 15 are good practice statements.</p><p><strong>Conclusions: </strong>The expert panel provided recommendations for pulmonary thromboembolism in children based on available evidence, which was generally low in quality and volume. The panel urges further research on early identification and diagnosis strategies, preventive and therapeutic regimens, and long-term management for pulmonary thromboembolism.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"330-348"},"PeriodicalIF":4.5,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13076377/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146258899","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Modeling Williams syndrome from a neurodevelopmental perspective: recent advances, model-based translational insights and future directions.","authors":"Ya-Yue Chen, Wei-Jun Chen, Rui Zhang, Chai Ji, Yu-Han Zhang, Da-Qing Ma, Qiao-Juan Shi, Yi-Cheng Xie","doi":"10.1007/s12519-026-01020-x","DOIUrl":"10.1007/s12519-026-01020-x","url":null,"abstract":"<p><strong>Background: </strong>Williams syndrome (WS; OMIM #194,050) is a multisystem pediatric genetic disorder caused by a heterozygous microdeletion of a 1.5-1.8 Mb region at chromosome 7q11.23, encompassing 26 to 28 genes. Clinical hallmarks include cardiovascular anomalies, distinctive craniofacial morphology and neurodevelopmental deficits characterized by hypersociability, cognitive impairment and anxiety. Although causative therapies for WS still remain elusive, advances in gene editing and forebrain organoids have already greatly furthered our understanding of the underlying mechanisms.</p><p><strong>Data sources: </strong>This narrative review was conducted by searching for papers using PubMed/MEDLINE. Relevant publications were identified using single and/or combined keywords including: Williams syndrome, 7q11.23, microdeletion, microduplication, atypical deletion, neurodevelopment, neuroanatomy, neuroimaging. cognitive impairment, mouse models, GTF2I, GTF2IRD1, CLIP2, LIMK1, NCF1, EIF4H, STX1A/B, FZD9, HIP1, CLDN3, FKBP6, organoid, induced pluripotent stem cell (iPSC) and forebrain organoids.</p><p><strong>Results: </strong>Mouse models including multigene deletion strains recapitulating the WS critical region and single-gene knockout strains targeting Gtf2i, Gtf2ird1, Clip2 and Limk1 replicate key WS neurodevelopmental phenotypes, substantially contributing to mechanistic studies and therapeutic screening. In addition, forebrain organoids derived from patients or generated by gene editing have provided human-specific insights into progenitor dynamics, synaptic function, and ribosome biogenesis.</p><p><strong>Conclusions: </strong>This review synthesizes recent progress in WS modeling in the context of neurodevelopmental impairments. While animal models and forebrain organoids have substantially accelerated both mechanistic understanding and translational research in WS, effective diagnostic and therapeutic approaches are still unavailable. Integration of animal models and forebrain organoids, together with the advanced technologies, will be essential for biomarker discovery and development of mechanism-based therapeutic approaches.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"284-302"},"PeriodicalIF":4.5,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13076566/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147475824","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Integrative metabolomics and machine learning identify biomarkers of adolescent social anxiety disorder.","authors":"Jun-Yu Lai, Bei-Bei Yang, Pei-Jun Ju, Ying Sun, Xiu-Jia Sun, Wen-Hong Cheng, Jing-Hong Chen","doi":"10.1007/s12519-025-00984-6","DOIUrl":"10.1007/s12519-025-00984-6","url":null,"abstract":"<p><strong>Background: </strong>Social anxiety disorder (SAD) is one of the most prevalent anxiety disorders in adolescents but remains underdiagnosed due to the lack of objective diagnostic tools. This study aimed to identify serum metabolomic alterations in adolescent SAD patients and to develop an interpretable diagnostic model.</p><p><strong>Methods: </strong>In this cross-sectional study, serum samples were collected from 78 adolescents, including 42 drug-naive, first-episode SAD patients and 36 matched healthy controls. Untargeted metabolomic profiling was performed, and feature selection was conducted via least absolute shrinkage and selection operator regression, followed by logistic regression for model construction.</p><p><strong>Results: </strong>Among the 661 detected metabolites, 46 differed significantly between groups, mainly within amino acid and energy metabolism pathways. Five key metabolites, 2-hydroxybutanoic acid, L-alanine, L-asparagine, glutamine and beta-tocopherol, were selected for model construction. The diagnostic model achieved an area under the curve of 0.934 in the training set, but external validation is still lacking, and the findings should be interpreted as hypothesis-generating.</p><p><strong>Conclusions: </strong>Adolescents with SAD exhibit distinct metabolic profiles, and a preliminary diagnostic model was developed. Exploratory microbiota-related observations suggested potential links between gut microbial activity, host metabolism, and anxiety phenotypes, but these findings remain preliminary and outside the scope of the present study. Overall, these findings provide hypothesis-generating support for further investigation of gut-metabolism-brain interactions and highlight the need for larger, externally validated studies to advance biomarker development.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"349-361"},"PeriodicalIF":4.5,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145347817","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"From treatment to health: reforming the child health care system in China in an era of low fertility.","authors":"Jian Wang, Dan Shan, Guo-Dong Ding, Yong-Jun Zhang, Kun Sun","doi":"10.1007/s12519-026-01029-2","DOIUrl":"10.1007/s12519-026-01029-2","url":null,"abstract":"","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"281-283"},"PeriodicalIF":4.5,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147475802","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prevalence of pediatric cannabidiol prescribing in the United States.","authors":"Yifan Li, Chloe Lessard, Fábio A Nascimento, Jacob T Borodovsky, Dawn Gano, Robert Trestman, Anita Kablinger, Pallawi Jyotsana, Jacob Steinle, Ruth Ling, Lisa Gong, Kevin Y Xu, Binx Yezhe Lin","doi":"10.1007/s12519-026-01028-3","DOIUrl":"10.1007/s12519-026-01028-3","url":null,"abstract":"","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"372-376"},"PeriodicalIF":4.5,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13035319/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147522301","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Use of transcranial Doppler to determine brain death or death by neurologic criteria in children: a narrative review.","authors":"Na Tan, Jie Wu, Su-Yun Qian","doi":"10.1007/s12519-025-01015-0","DOIUrl":"10.1007/s12519-025-01015-0","url":null,"abstract":"<p><strong>Background: </strong>Transcranial Doppler (TCD) detects characteristic waveforms indicating cerebral circulatory arrest and has been widely applied as an ancillary test for adult brain death or death by neurologic criteria. However, its application in children remains controversial due to anatomical differences and limited evidence. This review outlines the current role of transcranial Doppler in confirming pediatric brain death or death by neurologic criteria, offering practical insights and directions for future research.</p><p><strong>Methods: </strong>A literature review was conducted on the use of transcranial Doppler to confirm pediatric brain death or death by neurologic criteria. This included original studies, meta-analyses, reviews, clinical guidelines, consensus statements, position papers, and legislation. Databases searched included PubMed, Embase, Cochrane Library, Web of Science, Google Scholar, China National Knowledge Infrastructure and Wanfang, covering records from inception to July 5, 2025. Search terms included \"transcranial Doppler\", \"TCD\", \"cerebral circulatory arrest\", \"death by neurologic criteria\", and \"brain death\".</p><p><strong>Results: </strong>The use of transcranial Doppler varies across countries for confirming pediatric brain death or death by neurologic criteria. Some recommend transcranial Doppler, while others do not. Diagnostic criteria, including vessel selection, interpretation of absent blood flow signals and the number of tests, also vary. Pediatric studies support the clinical value of transcranial Doppler in the determination of brain death or death by neurologic criteria, but small sample sizes, methodological inconsistencies, and false results due to hemodynamic instability, open fontanelles or skull defects limit firm conclusions. Transcranial color-coded Doppler may improve diagnostic accuracy in certain cases.</p><p><strong>Conclusions: </strong>Transcranial Doppler demonstrates considerable potential in confirming pediatric brain death or death by neurologic criteria, particularly in resource-limited or bedside settings. Given the current variability in clinical practice and the limitations of existing evidence, further large-scale, prospective studies are warranted to validate its role in this indication.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"303-314"},"PeriodicalIF":4.5,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146087429","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Artificial intelligence in diagnosis of pediatric neurodevelopmental disorders: a scoping review.","authors":"María Alejandra Nieto Ramírez, Mateo Mariño Rodríguez, María José Castro Salas, Erwin Hernando Hernández Rincón","doi":"10.1007/s12519-025-00999-z","DOIUrl":"10.1007/s12519-025-00999-z","url":null,"abstract":"<p><strong>Background: </strong>Neurodevelopmental disorders are a group of conditions that affect key areas of development and may significantly impact a child's quality of life. This underscores the importance of accurate diagnostic tools to improve outcomes. Artificial intelligence (AI) has shown measurable effectiveness for enhancing the diagnosis and monitoring of neurodevelopmental disorders. This scoping review aims to summarize the current evidence on the use of AI technologies, including deep learning, supervised machine learning, decision support systems, and biosignal analysis, in improving diagnostic accuracy for pediatric neurodevelopmental disorders.</p><p><strong>Data sources: </strong>A systematic search was conducted across PubMed, LILACS, MEDLINE, Google Scholar, and psychology-indexed journals, covering publications from 2000 to January 2025. Keywords and Medical Subject Headings terms were used to search for and select studies, applying specific inclusion and exclusion criteria. Selection followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews guidelines and included clinical studies, reviews, and validation research. The data were extracted and synthesized descriptively.</p><p><strong>Results: </strong>Twenty-two studies were included. Deep learning models achieved diagnostic accuracies exceeding 85% in most studies in neuroimaging interpretation, whereas supervised machine learning improved the subtype classification of autism spectrum disorder and attention deficit hyperactivity disorder. Decision support systems have increased diagnostic efficiency, and biosignal-based AI has shown potential in identifying physiological markers related to neurodevelopmental disorders.</p><p><strong>Conclusions: </strong>AI technologies may significantly contribute to improving early diagnosis and clinical decision-making in pediatric neurodevelopment. However, variability in study design, population, and algorithm standardization remains a challenge. AI technologies are also facing ethical concerns such as data privacy and security, interpretability, equity and access, and algorithmic bias. Further multicenter validation and regulatory frameworks are essential for clinical translation.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"315-329"},"PeriodicalIF":4.5,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13076480/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146067595","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Gene-based burden testing implicates four novel susceptibility genes associated with isolated short stature in pediatric patients.","authors":"Fei Xiao, Ming-Yue Cai, Bing-Yu Yang, Wu-Di Gu, Li-Li Wang, Hai-Ying Wu, Rong-Rong Xie, Feng-Yun Wang, Xiu-Li Chen, Lin-Qi Chen, Dan-Dan Zhang, Qing Wang, Hong-Ying Wang, Yu Jin, Xue-Qian Wang, Ting Chen","doi":"10.1007/s12519-026-01021-w","DOIUrl":"10.1007/s12519-026-01021-w","url":null,"abstract":"<p><strong>Background: </strong>Idiopathic short stature (ISS), a common cause of unexplained growth failure in children, remains poorly characterized at the genetic level. This study aimed to investigate the contribution of rare variant burdens in growth-related genes and pathways to the etiology of ISS using next-generation sequencing and gene-based burden testing, thereby identifying novel genetic contributors to the polygenic landscape of ISS.</p><p><strong>Methods: </strong>We analyzed 212 pediatric patients with short stature who remained undiagnosed following trio-based whole-exome sequencing. The comparison cohort included 352 healthy adults with normal stature and 4327 internal samples from the Exome Aggregation Consortium database. Gene-based burden testing was performed using an optimized TRAPD (testing rare variants using public data) framework. Functional enrichment analyses, including Kyoto Encyclopedia of Genes and Genomes and Gene Ontology pathway analyses, were conducted to delineate the biological processes associated with the identified candidate genes.</p><p><strong>Results: </strong>Under a dominant inheritance model, 3907 genes were significantly enriched in rare variants (P < 0.05), whereas 85 genes were significantly enriched under a recessive model (P < 0.05). The top 10 most significantly associated genes identified through primary modeling included FCGBP, FRAS1, MPDZ, and OBSCN, among which highly significant signals were identified (P < 1 × 10^-⁹). Pathway analyses revealed enrichment in steroid hormone biosynthesis, ascorbate and aldarate metabolism, pentose and glucuronate interconversions, and porphyrin metabolism. Key genes such as OBSCN, FRAS1, and MPDZ were involved in multiple enriched pathways.</p><p><strong>Conclusions: </strong>This study implicates rare variant burdens in growth-related genes as contributors to ISS pathogenesis, highlighting key genes (OBSCN, FCGBP, FRAS1, and MPDZ) and pathways involved. These findings suggest that the dysregulation of hormonal signaling, the extracellular matrix, and muscle-skeletal interactions impairs linear growth, suggesting potential diagnostic and therapeutic targets for ISS.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"362-371"},"PeriodicalIF":4.5,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147345260","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Integrated cognitive-motor exercise for core symptoms and executive functions in children with attention deficit hyperactivity disorder: a randomized clinical trial.","authors":"Fei-Long Zhu, Zheng-Hao Dong, Hao-Yuan Lu, Dong-Qing Kuang, Bao-Hua Xu, Li Yang, Yu-Feng Wang, Ming Zhang, Yuan-Chun Ren","doi":"10.1007/s12519-026-01019-4","DOIUrl":"https://doi.org/10.1007/s12519-026-01019-4","url":null,"abstract":"<p><strong>Background: </strong>Attention deficit hyperactivity disorder (ADHD) is a prevalent neurodevelopmental disorder. While physical exercise is a promising non-pharmacological intervention, the efficacy of integrating cognitive demands with physical activity remains underexplored. This trial compared the effects of integrated cognitive-motor exercise, aerobic exercise, and a minimal intervention on core symptoms and executive functions (EFs) in children with ADHD.</p><p><strong>Methods: </strong>In this randomized, controlled, multicenter trial, 107 children with ADHD (aged 6-10 years) were allocated to one of three groups for 12 weeks: (1) integrated cognitive-motor exercise (EG1, n = 36); (2) aerobic exercise (EG2, n = 35); or (3) wait-list control (CG, n = 36). Both EG1 and EG2 performed their respective interventions three times per week in 45-minute sessions. The primary outcomes were inattention and hyperactivity-impulsivity symptoms and EFs (inhibitory control, working memory, and cognitive flexibility). The analysis followed the intention-to-treat principle with linear mixed models.</p><p><strong>Results: </strong>Compared with the CG, both exercise groups presented significant, comparable reductions in inattention and hyperactivity-impulsivity symptoms (all P ≤ 0.01). However, EG1 demonstrated superior improvements in specific EFs. For inhibitory control (Stroop color-word interference), EG1 resulted in a significantly greater reduction in color-word interference time than both EG2 [β = - 6.24, 95% confidence interval (CI) = - 12.28 to - 0.20, P = 0.045] and CG (β = - 13.97, 95% CI = - 19.97 to - 7.97, P < 0.001). For immediate working memory, the improvement in EG1 was greater than that in both EG2 (β = 2.09, 95% CI = 0.33-3.85, P = 0.032) and CG (β = 3.57, 95% CI = 1.83-5.31, P < 0.001). Both exercise groups improved similarly in cognitive flexibility compared with the CG. Parental satisfaction was significantly greater in EG1 than in EG2 (P < 0.001). No adverse events were reported.</p><p><strong>Conclusions: </strong>A structured integrated cognitive-motor exercise intervention is an effective and safe non-pharmacological treatment for children with ADHD. Compared with aerobic exercise alone, it not only alleviates core symptoms but also yields superior benefits for key EFs, specifically inhibitory control and immediate working memory.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2026-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146167031","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Small-molecule therapies for pediatric inflammatory bowel disease: toward precision medicine.","authors":"Ying Chen, Yang Wang, Jing Guo, Ling-Fen Xu, Xu Teng","doi":"10.1007/s12519-025-01001-6","DOIUrl":"10.1007/s12519-025-01001-6","url":null,"abstract":"<p><strong>Background: </strong>Pediatric inflammatory bowel disease (pIBD) often begins early in life, progresses rapidly, and is associated with impaired growth and delayed development. These challenges demand treatment strategies that address both intestinal inflammation and the broader developmental needs of children.</p><p><strong>Data sources: </strong>This review summarizes current advances in small-molecule therapies for pIBD based on published clinical trials, real-world studies, and mechanistic investigations retrieved from PubMed and clinical trial registries. Special emphasis is placed on Janus kinase (JAK) inhibitors and sphingosine-1-phosphate (S1P) modulators, which represent the main translational research focus in pediatric IBD.</p><p><strong>Results: </strong>JAK inhibitors such as tofacitinib and upadacitinib have demonstrated promising efficacy in pediatric patients with refractory disease, although their use remains off-label worldwide. Long-term safety concerns persist, including infection risk, developmental effects, and potential risks of malignancy or major adverse cardiovascular events. S1P modulators such as ozanimod are under clinical evaluation in children, but robust long-term data are still lacking. Emerging technologies such as single-cell and spatial profiling have begun to reveal age-dependent remodeling of gut immune architecture, emphasizing the importance of developmentally informed therapeutic approaches.</p><p><strong>Conclusions: </strong>Small-molecule therapies offer a promising and mechanistically precise direction for the management of pIBD. Future progress will depend on age-specific clinical trials, physiologically based pharmacokinetic modeling, and biomarker discovery through integrated multiomics. Collaborative multicenter research is essential to optimize the safety and efficacy of these agents in children.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"212-224"},"PeriodicalIF":4.5,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12923488/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145688273","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}