Min Kyung Shin, Yejun Son, Dong Keon Yon, Jinseok Lee
{"title":"Potential gut-brain axis-targeted therapies for autism spectrum disorder in children: opportunities and challenges.","authors":"Min Kyung Shin, Yejun Son, Dong Keon Yon, Jinseok Lee","doi":"10.1007/s12519-025-00924-4","DOIUrl":"10.1007/s12519-025-00924-4","url":null,"abstract":"<p><strong>Background: </strong>Autism spectrum disorder (ASD) lacks definitive treatment, but recent research has highlighted the potential of gut‒brain axis-targeted therapies for managing ASD symptoms in children. This review evaluated the effects of microbiota transplantation (MT), probiotics, dietary interventions, and nutritional supplements on ASD symptoms in children.</p><p><strong>Data sources: </strong>A systematic review was conducted via PubMed/MEDLINE, Scopus, and Web of Science to identify studies published up to June 2024. The inclusion criteria consisted of peer-reviewed articles encompassing both observational studies and interventional trials, and studies specifically targeted symptoms of ASD and included patients under the age of 18, with a minimum sample size of 20 participants.</p><p><strong>Results: </strong>Of the 3424 identified studies, 31 met the inclusion criteria. MT emerged as the most consistently effective intervention, showing improvements across multiple symptom domains, including behavior and social interaction, particularly for individuals with severe gastrointestinal (GI) issues. Probiotics have reported strain-specific efficacy, with some studies reporting behavioral improvements, but the results have been inconsistent. Dietary interventions, such as gluten-free casein-free and modified Atkins diets, have shown partial efficacy, particularly for individuals with cooccurring GI symptoms, with adherence challenges and variability in outcomes. Nutritional supplements yielded mixed outcomes, highlighting the need for personalized approaches. Despite promising findings, significant heterogeneity in study protocols and outcome measures underscores the need for standardized methodologies. Future research should prioritize standardization of these protocols. Long-term studies and longitudinal designs can help increase the reliability and practicality. Precision strategies based on individual microbiota compositions and genomics could optimize outcomes. Combined therapies should undergo rigorous evaluation. Reliable markers could improve cost-effectiveness by targeting therapies to responders. Broader research populations, economic evaluations, new technologies and interdisciplinary research will contribute to achieving a broader application and better outcomes.</p><p><strong>Conclusions: </strong>This review emphasizes the potential of gut‒brain axis-targeted therapies to improve the quality of life of children with ASD and their families. MT showed the most consistent improvements in managing pediatric ASD symptoms, with probiotics, dietary interventions, and nutritional supplements offering additional, albeit variable benefits. Efforts should be made to standardize the protocols, to conduct long-term studies, and to explore cost-effective solutions to ensure accessibility, particularly in resource-limited settings.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"447-467"},"PeriodicalIF":4.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144249906","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":"Advancing toward a curative frontier: an updated narrative review on stem-cell therapy in pediatric type 1 diabetes.","authors":"Samia Sulaiman, Abdallah Alaarag, Nadin Rayyan, Yousof Tuffaha, Nada Al-Awamleh, Khalid Adel Al Dojan","doi":"10.1007/s12519-025-00908-4","DOIUrl":"10.1007/s12519-025-00908-4","url":null,"abstract":"<p><strong>Background: </strong>Type 1 diabetes (T1D) is a chronic autoimmune disease primarily diagnosed in childhood, characterized by pancreatic β-cell destruction, severe insulin deficiency, and hyperglycemia. Current treatments, including insulin therapy and glucose-lowering medications, manage the condition but fall short of offering a cure. In this review we explore the potential of stem-cell therapy as a transformative and curative approach for T1D, focusing on its promise in regenerating β-cells and addressing challenges specific to the pediatric population.</p><p><strong>Data sources: </strong>A comprehensive review of the literature was conducted to evaluate stem-cell types: embryonic, perinatal, adult, induced pluripotent and cancer stem cells, and their role in T1D treatment. Particular emphasis was placed on methods for β-cell differentiation, advancements in autologous and allogeneic stem-cell transplantation and emerging strategies to overcome safety, efficacy, and economic barriers. Challenges such as immune rejection, tumorigenicity, and cost-effectiveness were analyzed, alongside novel solutions like immune-shielding and clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein-9 (Cas9) technology.</p><p><strong>Results: </strong>Stem-cell therapy presents a promising avenue for curing T1D, offering potential for β-cell regeneration and reduced dependence on exogenous insulin. However, challenges such as delayed β-cell functionality, immune responses, tumor risks, and high costs hinder widespread application.</p><p><strong>Conclusions: </strong>Advancements in personalized medicine, immune-shielding strategies, and cost reduction may pave the way for clinical success, especially in pediatric populations. Further research addressing these barriers is essential to establish stem-cell therapy as a viable and equitable treatment option.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"436-446"},"PeriodicalIF":4.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144026595","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":"Maternal inflammation during the second trimester of pregnancy with adverse birth outcomes: a prospective cohort study.","authors":"Wan-Qing Xiao, Si-Chi Zhu, Zhu-Gu Huang, Qi-Wen He, Zi-Yin Lyu, Xu-Tao Li, Qing-Qun Lyu, Ya-Shu Kuang, Xue-Ling Wei, Dong-Mei Wei, Jin-Hua Lu, Jian-Rong He, Xiu Qiu","doi":"10.1007/s12519-025-00909-3","DOIUrl":"10.1007/s12519-025-00909-3","url":null,"abstract":"<p><strong>Background: </strong>Distinct characteristics of maternal inflammation at midgestation related to fetal and neonatal health are not clear. This study aims to explore the associations between the maternal circulating inflammatory profile during the second trimester of pregnancy and a series of adverse birth outcomes.</p><p><strong>Methods: </strong>The present study was a prospective cohort study based on the Born in Guangzhou Cohort Study conducted in Guangzhou, China. Peripheral blood samples from women were obtained during the second trimester. Adverse newborn outcomes were collected through electronic medical records at birth and within the first week after birth. We used logistic, Poisson and generalized linear regressions to assess the impact of inflammatory indicators during pregnancy on different outcomes.</p><p><strong>Results: </strong>A total of 1567 singleton pregnant women were included. The proportions of preterm birth, small for gestational age (SGA), low birth weight (LBW), neonatal asphyxia and hyperbilirubinemia cases were 4.3%, 6.6%, 3.9%, 1.1% and 12.6%, respectively. The maternal circulating level of log2-transformed interleukin (IL)-7 was positively associated with preterm birth [adjusted odds ratio (OR) = 1.58, 95% confidence interval (CI) = 1.03-2.41]. A higher level of log2-transformed IL-2 was correlated with increased LBW risk (adjusted OR = 1.48, 95% CI = 1.09-2.02) and a decreased birth weight Z-score (adjusted β = - 0.06, 95% CI = - 0.11, - 0.01). IL-7 was associated with an increased risk of LBW (adjusted OR = 1.80, 95% CI = 1.14-2.86), whereas log2-transformed IL-15 was positively related to SGA (adjusted OR = 1.39, 95% CI = 1.02-1.91). There were no significant associations of maternal inflammatory indicators with neonatal asphyxia or hyperbilirubinemia.</p><p><strong>Conclusion: </strong>Maternal circulating IL-2, IL-7 and IL-15 levels during the second trimester of pregnancy were associated with preterm birth or birth weight.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"468-477"},"PeriodicalIF":4.5,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144226883","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":"Melatonin use in the pediatric population: an evolving global concern.","authors":"Judith Owens","doi":"10.1007/s12519-025-00896-5","DOIUrl":"https://doi.org/10.1007/s12519-025-00896-5","url":null,"abstract":"<p><strong>Background: </strong>The impact of melatonin on pediatric populations has not yet been widely researched. This narrative review is intended to summarize the current literature regarding the efficacy and safety of melatonin for children and potential pitfalls in its clinical use, in order to assist pediatric practitioners in making evidence-based recommendations that serve the best interests of their patients and families.</p><p><strong>Data sources: </strong>A literature review of melatonin in the pediatric population was conducted using PubMed search terms: melatonin, pediatrics, child/adolescent.</p><p><strong>Results: </strong>Melatonin use in the pediatric population world-wide has increased significantly over the past several decades. While a number of studies, largely in children with neurodevelopmental disorders with insomnia, have suggested that melatonin is generally safe and well-tolerated, a similar body of evidence is overall lacking for typically developing children and prospective studies regarding long-term adverse events are lacking. In addition, recent studies have raised concerns regarding the variable content of melatonin in over-the-counter products, as well as safety issues relating to accidental ingestions.</p><p><strong>Conclusions: </strong>Due to a number of concerns regarding inappropriate use, lack of efficacy and safety data across pediatric populations and variability in actual content, melatonin should be used with caution in children and only under medical supervision.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":""},"PeriodicalIF":6.1,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143985003","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}
Zhao-Yu Chen, Tian-Nan Zhang, Ji Li, Zhen-Jie Zhang, Hong-Mei Song
{"title":"Tofacitinib: a promising agent for the treatment of persistent rashes in juvenile dermatomyositis.","authors":"Zhao-Yu Chen, Tian-Nan Zhang, Ji Li, Zhen-Jie Zhang, Hong-Mei Song","doi":"10.1007/s12519-025-00901-x","DOIUrl":"10.1007/s12519-025-00901-x","url":null,"abstract":"","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"419-422"},"PeriodicalIF":6.1,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12103324/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143985017","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}
Zhi-Peng Shen, Zhong-Yuan Zhang, Nan Li, Liang Xu, Ye Chen
{"title":"Targeted therapy for pediatric glioma: RAF(t)ing in the molecular era.","authors":"Zhi-Peng Shen, Zhong-Yuan Zhang, Nan Li, Liang Xu, Ye Chen","doi":"10.1007/s12519-025-00889-4","DOIUrl":"10.1007/s12519-025-00889-4","url":null,"abstract":"<p><strong>Background: </strong>Pediatric gliomas are the most frequently occurring central nervous system tumors in children. While targeted therapies have been widely applied in the treatment of many adult cancers, their use in pediatric gliomas has lagged behind. However, recent advances in multiomics profiling of pediatric gliomas, coupled with the approval of inhibitors against Raf serine/threonine kinase (RAF), isocitrate dehydrogenase 1/2 (IDH1/2) and neurotrophic receptor tyrosine kinase (NTRK), have spurred significant progress in this field. In light of these developments, this review aims to provide a comprehensive overview of current advancements and the evolving landscape of targeted therapeutic strategies and approaches for pediatric gliomas.</p><p><strong>Data sources: </strong>Data analyzed in this study were obtained from the literature from PubMed, as well as other online databases and websites, including ClinicalTrials.gov and the Pediatric Neuro-Oncology Consortium.</p><p><strong>Results: </strong>Based on findings from multiomics profiling, significant insights have been gained into the genetic and molecular landscape of pediatric gliomas, enabling the identification of key mutations and potentially targetable lesions. These advancements provide rationales for the development of more precise treatment strategies and targeted therapies. Recent approvals of targeted therapies and ongoing clinical trials in pediatric gliomas are converging on the targeting of key signaling molecules and metabolic pathways.</p><p><strong>Conclusions: </strong>In the molecular era, targeted therapies offer new hope for more effective and personalized treatment options for pediatric glioma patients. By developing and tailoring treatments to target specific molecular and metabolic vulnerabilities, targeted therapies have the potential to improve the clinical management of pediatric gliomas, ultimately enhancing both the treatment experience and overall prognosis of these patients.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"338-351"},"PeriodicalIF":6.1,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144052600","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}
Yu-Meng Gao, Bo-Wen Xu, Ping Liu, Yu-Li Wang, Qing-You Zhang, Ying Liao, Jun-Bao Du, Hong-Fang Jin
{"title":"Nomogram and scoring models based on BMI z-score, serum chlorine and urine specific gravity levels for predicting oral rehydration solution efficacy in pediatric postural orthostatic tachycardia syndrome.","authors":"Yu-Meng Gao, Bo-Wen Xu, Ping Liu, Yu-Li Wang, Qing-You Zhang, Ying Liao, Jun-Bao Du, Hong-Fang Jin","doi":"10.1007/s12519-025-00898-3","DOIUrl":"10.1007/s12519-025-00898-3","url":null,"abstract":"<p><strong>Background: </strong>Oral rehydration solution (ORS) is predominantly utilized in the management of hypovolemic postural orthostatic tachycardia syndrome (POTS). This study aimed to identify effective indicators and develop models to assess the impact of ORS on pediatric patients diagnosed with POTS.</p><p><strong>Methods: </strong>We utilized a retrospective analysis of totally 158 pediatric patients with POTS receiving a 3-month course of ORS treatment. All patients were classified into training set (n = 98) and validation set (n = 60). Within the training set, univariate analysis and binary logistic regression were employed to select candidate predictors. To predict the efficacy of ORS in pediatric patients with POTS, a nomogram model and a scoring model were constructed and demonstrated. Additionally, the predictive ability and calibration performance were evaluated using receiver operating characteristic (ROC) curves, Hosmer-Lemeshow (H-L) goodness of fit test, and calibration plots. Decision curve analysis (DCA) was employed to assess the clinical applicability of the predictive models.</p><p><strong>Results: </strong>Body mass index (BMI) z-score, serum chlorine, and urine specific gravity (USG) before treatment were identified as significant and independent predictors of efficacy of ORS in pediatric patients with POTS. Consequently, these indicators were included in the predictive models. A nomogram model was constructed in the training set (AUC = 0.87, which yields a sensitivity of 84.5% and a specificity of 85.0%) and validated in the validation set (the sensitivity, specificity, and accuracy were 87.5%, 85%, and 86.7%, respectively). A scoring model was advanced in the training set (AUC = 0.88, which yields a sensitivity of 79.3% and a specificity of 82.5%) and validated in the validation set (the sensitivity, specificity, and accuracy were 77.5%, 80.0%, and 78.3%, respectively). The H-L test results indicated a good model fit. The calibration plots and DCA for both models exhibited excellent calibration and satisfactory net benefit.</p><p><strong>Conclusions: </strong>Based on pre-treatment BMI z-score, serum chlorine, and USG, a nomogram model and a scoring model were developed and validated. The models can effectively assess the efficacy of ORS in pediatric patients with POTS, offering an accurate and individualized therapeutic strategy.</p>","PeriodicalId":23883,"journal":{"name":"World Journal of Pediatrics","volume":" ","pages":"406-418"},"PeriodicalIF":6.1,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144039497","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}