{"title":"Subacute-Aggressive-Onset Chronic Inflammatory Demyelinating Polyradiculoneuropathy as the Initial Presentation in a Pediatric Patient With Systemic Lupus Erythematosus: A 5-Year Follow-Up Case Report.","authors":"Zhiwei Yu, Yuan Xue, Hanyu Luo, Yuhang Li, Siqi Hong, Jiannan Ma, Li Jiang","doi":"10.1002/pdi3.70028","DOIUrl":"10.1002/pdi3.70028","url":null,"abstract":"","PeriodicalId":520221,"journal":{"name":"Pediatric discovery","volume":"3 3","pages":"e70028"},"PeriodicalIF":0.0,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12483291/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145208672","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}
Pediatric discoveryPub Date : 2025-09-14eCollection Date: 2025-12-01DOI: 10.1002/pdi3.70016
V A Raghu, Manju Vatsa, Neeraj Gupta, Hansraj, Ramesh Choudhary
{"title":"Development and Validation of a Nurse-Led Developmental Intervention Package to Improve the Neurodevelopmental Outcomes of Preterm Babies in Neonatal Intensive Care Unit.","authors":"V A Raghu, Manju Vatsa, Neeraj Gupta, Hansraj, Ramesh Choudhary","doi":"10.1002/pdi3.70016","DOIUrl":"10.1002/pdi3.70016","url":null,"abstract":"<p><p>Preterm birth is a major global health concern associated with high morbidity and mortality rates, along with long-term neurodevelopmental impairments. Developmental supportive care (DSC) plays a crucial role in optimizing neurodevelopmental outcomes in these vulnerable neonates. This study aimed to develop, validate, and assess the feasibility of a nurse-led developmental intervention package (NLDIP). A Medical Research Council (MRC) framework was used to develop and validate the NLDIP. The NLDIP was designed based on a comprehensive scoping review, expert consultations, and practical feasibility assessments in a neonatal intensive care unit (NICU). Validation was conducted through single-round Delphi technique with expert reviews from different fields. The content validity index value was 0.9 and a dry run was performed to assess compliance and feasibility. The NLDIP was systematically developed and validated, comprising three key components: standard DSC, age-appropriate multisensory stimulation (MSS) during hospitalization, and continued MSS at home. The findings demonstrate that the NLDIP is a valid, feasible, and well-structured intervention which can be effectively implemented by nurses in an integrated way to enhance the neurodevelopmental outcomes of preterm infants.</p>","PeriodicalId":520221,"journal":{"name":"Pediatric discovery","volume":"3 4","pages":"e70016"},"PeriodicalIF":0.0,"publicationDate":"2025-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12753019/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145879718","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}
Pediatric discoveryPub Date : 2025-08-24eCollection Date: 2026-03-01DOI: 10.1002/pdi3.70020
Yuehan Li, Yan Li, Jinying Xiang, Yinying Ren, Mi Zhou, Fang Deng, Zhou Fu, Fengxia Ding
{"title":"Glucocorticoid-Induced TNFR-Related Ligand as a Biomarker for Diagnosis and Severity of Asthma in Children.","authors":"Yuehan Li, Yan Li, Jinying Xiang, Yinying Ren, Mi Zhou, Fang Deng, Zhou Fu, Fengxia Ding","doi":"10.1002/pdi3.70020","DOIUrl":"10.1002/pdi3.70020","url":null,"abstract":"<p><p>Currently, there are no reliable pediatric biomarkers for assessing the severity of asthma. Therefore, the study aimed to assess the potential of glucocorticoid-induced TNFR-related ligand (GITRL) in assessing asthma severity. Bone marrow-derived dendritic cells (BMDCs) were isolated and treated with phosphate-buffered saline (PBS) or house dust mites (HDM). Flow cytometry, immunofluorescence, and RT-qPCR results revealed that GITRL expression was increased in HDM-treated BMDCs (<i>p</i> < 0.05). An asthma mouse model was established using HDM. Asthmatic mice exhibited significantly increased lung inflammation scores, serum IgE, total cell counts, and eosinophil counts in bronchoalveolar lavage fluid (BALF) (<i>p</i> < 0.05). Tissue immunofluorescence and immunohistochemistry indicated elevated GITRL expression levels (<i>p</i> = 0.03). Flow cytometry analysis of peripheral blood mononuclear cells (PBMCs) isolated from children showed that the proportion of GITRL + dendritic cells (DCs) and the mean fluorescence intensity (MFI) of GITRL were significantly increased in asthmatic children compared with healthy individuals (<i>p</i> < 0.01). RT-qPCR analysis showed that <i>GITRL</i> mRNA expression levels were significantly elevated in children with moderate-to-severe asthma compared to those with mild asthma (<i>p</i> = 0.006), and <i>GITRL</i> mRNA levels were higher in both groups of asthmatic children than in healthy controls (<i>p</i> < 0.05). Correlation analysis indicated that <i>GITRL</i> expression was negatively correlated with lung function and the Children's Asthma Control Test (C-ACT) score (<i>p</i> < 0.001). ROC analysis assessed its diagnostic value, with AUC values of 0.89 (asthma vs. healthy diagnosis) and 0.70 (distinguishing moderate-to-severe from mild asthma). These findings suggest that GITRL is an important biomarker for assessing asthma severity.</p>","PeriodicalId":520221,"journal":{"name":"Pediatric discovery","volume":"4 1","pages":"e70020"},"PeriodicalIF":0.0,"publicationDate":"2025-08-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13098139/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147794697","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}
Pediatric discoveryPub Date : 2025-07-24eCollection Date: 2025-09-01DOI: 10.1002/pdi3.70015
Ranganatha A Devaranavadagi, Gayatri Sasikumar, Pavitra Gangadharan Chandrasekaran, Netra S Kannur, H A Venkatesh, Suma Sriramanan, C N Radhakrishnan, Jayashree S Simha, Karthik Nagesh
{"title":"Early Forays Into Using Spinal Anesthesia for Inguinal Hernia Surgery in Preterm Infants: A Retrospective Analysis From a Single Center in Karnataka.","authors":"Ranganatha A Devaranavadagi, Gayatri Sasikumar, Pavitra Gangadharan Chandrasekaran, Netra S Kannur, H A Venkatesh, Suma Sriramanan, C N Radhakrishnan, Jayashree S Simha, Karthik Nagesh","doi":"10.1002/pdi3.70015","DOIUrl":"10.1002/pdi3.70015","url":null,"abstract":"<p><p>Neonates, particularly those born preterm, have a higher incidence of inguinal hernia and are at increased risk of cardiopulmonary complications associated with general anesthesia. To mitigate these risks, spinal anesthesia has been adopted in our institution for over two decades as the preferred technique for inguinal hernia repair in neonates. This retrospective study presents a 6-year experience with neonatal spinal anesthesia and includes both term and preterm infants undergoing inguinal surgery. We specifically analyzed cases in which spinal anesthesia was used in neonates with a postconceptional age below 50 weeks. Nineteen patients underwent inguinal surgery under spinal anesthesia in this timeframe. The gestational age at birth ranged from 27 to 38 weeks. The postmenstrual age at the time of surgery ranged from 35 to 46 weeks. The birth weight ranged from 740 to 3300 g, whereas the weight at surgery ranged from 1550 to 4900 g. A 26G hypodermic needle was used to give the spinal block, and 0.2 mL/kg of 0.5% heavy bupivacaine was injected. No cases required respiratory support and/or inotropic support during the procedure and postoperatively. None had apnea during/after surgery, including the four cases of bronchopulmonary dysplasia. None required general anesthesia. Spinal anesthesia for inguinal hernia repair is a safe and efficient method that obviates the necessity for NICU admission or any escalation in respiratory care, including in the cases of bronchopulmonary dysplasia.</p>","PeriodicalId":520221,"journal":{"name":"Pediatric discovery","volume":"3 3","pages":"e70015"},"PeriodicalIF":0.0,"publicationDate":"2025-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12483297/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145208843","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}
{"title":"Thiostrepton Suppresses the Progression of Rhabdomyosarcoma by Inhibiting the PI3K-AKT Signaling Pathway.","authors":"Yu Wang, Peng Hong, Zhiqiang Gao, Wei Ma, Zaihong Hu, Jie Lin, Kongkong Cui, Qinlin Shi, Xiao-Mao Tian, Guanghui Wei","doi":"10.1002/pdi3.70014","DOIUrl":"10.1002/pdi3.70014","url":null,"abstract":"<p><p>Rhabdomyosarcoma (RMS), the most common pediatric soft tissue sarcoma with 5-year survival below 30% in high-risk/metastatic cases, was investigated through integrated bioinformatics analysis (identifying 269 conserved differentially expressed genes in GEO datasets GSE28511/GSE141690) and experimentally validated thiostrepton (TST), a ribosomal-targeting antibiotic, as a potent therapeutic candidate via Connectivity Map analysis (<i>p</i> < 0.05, score ≈ -1). In-vitro studies demonstrated TST's dose-/time-dependent suppression of RMS proliferation (IC50 4.986-9.764 μmol/L), migration and invasion, G0/G1 cell cycle arrest, and apoptosis induction. In vivo, TST (3.4 mg/mL, 4 weeks) significantly inhibited tumor growth (<i>p</i> < 0.05 vs. phosphate buffered saline [PBS]) without organ toxicity. RNA sequencing identified the phosphatidylinositol 3-kinase/protein kinase B (PI3K-AKT) pathway as the primary suppressed pathway (False Discovery Rate [FDR] < 0.05), with concurrent downregulation of downstream regulators (<i>AKT, JAK, CDKs</i>). This was confirmed by PI3K activator 740 Y-P rescue experiments, which partially reversed the effects of TST (<i>p</i> < 0.05). These findings establish TST as a multi-mechanism PI3K-AKT inhibitor for refractory RMS while validating Connectivity Map (Cmap)-driven drug repurposing for pediatric oncology.</p>","PeriodicalId":520221,"journal":{"name":"Pediatric discovery","volume":"3 3","pages":"e70014"},"PeriodicalIF":0.0,"publicationDate":"2025-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12483292/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145208691","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}
Pediatric discoveryPub Date : 2025-06-23eCollection Date: 2025-06-01DOI: 10.1002/pdi3.70013
Wandi Wang, Lingman Wang, Chunxue Jiang, Shengxuan Zhang, Chen Tan, Liqiong Peng, Ran Ding, Bing Tian, Xiaojie Song, Li Jiang
{"title":"Protective Effects of the Ketogenic Diet on Cognitive Impairment Induced by Status Epilepticus in Rats: Modulation of Neuroinflammation Through the NF-κB Signaling Pathway.","authors":"Wandi Wang, Lingman Wang, Chunxue Jiang, Shengxuan Zhang, Chen Tan, Liqiong Peng, Ran Ding, Bing Tian, Xiaojie Song, Li Jiang","doi":"10.1002/pdi3.70013","DOIUrl":"10.1002/pdi3.70013","url":null,"abstract":"<p><p>Epilepsy is a chronic neurological disorder characterized by abnormal synchronized neuronal discharges, leading to cognitive dysfunction. The ketogenic diet (KD) has shown promise as an effective treatment for drug-resistant epilepsy (DRE), reducing seizures and improving cognitive and behavioral outcomes in patients. However, the precise neuroprotective mechanisms are not fully understood. This study aimed to investigate the effects of KD on cognitive impairment and hippocampal neurocircuit damage in rats with status epilepticus (SE), with a focus on a nuclear factor-kappa B (NF-κB) signaling. SE was induced using pilocarpine, and rats were assigned to KD and control groups. After 7 and 20 days of KD treatment, cognitive function was assessed using the elevated plus-maze, Morris water maze, novel object recognition, and Y-maze tests. Hippocampal tissue was analyzed for structural damage of neurocircuit. NF-κB pathway activation was evaluated by western blot and immunofluorescence. Results indicated that KD significantly improved cognitive performance and reduced hippocampal damage. Additionally, KD inhibited NF-κB pathway activation, evidenced by decreased levels of NF-κB, p-IκB, and proinflammatory cytokines. These findings suggest that KD may alleviate cognitive deficits and hippocampal damage by modulating the NF-κB signaling, providing insights into its neuroprotective mechanisms and potential as an alternative treatment for epilepsy.</p>","PeriodicalId":520221,"journal":{"name":"Pediatric discovery","volume":"3 2","pages":"e70013"},"PeriodicalIF":0.0,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12258113/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645136","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}
Pediatric discoveryPub Date : 2025-06-21eCollection Date: 2025-06-01DOI: 10.1002/pdi3.70012
Frankel Lin, Jasmine Huang, Sudipta Roy Chowdhury
{"title":"Acute Genitourinary Pain and Swelling in a Child-Look Out for the Rashes!","authors":"Frankel Lin, Jasmine Huang, Sudipta Roy Chowdhury","doi":"10.1002/pdi3.70012","DOIUrl":"10.1002/pdi3.70012","url":null,"abstract":"","PeriodicalId":520221,"journal":{"name":"Pediatric discovery","volume":"3 2","pages":"e70012"},"PeriodicalIF":0.0,"publicationDate":"2025-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12258110/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645129","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}
Pediatric discoveryPub Date : 2025-06-17eCollection Date: 2025-06-01DOI: 10.1002/pdi3.70011
Yassin El-Najjar, Mary-Joe Touma, Shuai Tan, Xiaoqin Zhou, Qi Liu
{"title":"A Review of the Current State of Off-Label Therapies for Pediatric Inflammatory Bowel Disease.","authors":"Yassin El-Najjar, Mary-Joe Touma, Shuai Tan, Xiaoqin Zhou, Qi Liu","doi":"10.1002/pdi3.70011","DOIUrl":"10.1002/pdi3.70011","url":null,"abstract":"<p><p>Off-label use of biologic therapies in patients with pediatric inflammatory bowel disease (IBD) has seen an increase in utilization. In this paper, we review the current state of off-label therapies in the pediatric IBD population. Real-world use of ustekinumab (UST), vedolizumab (VDZ), upadacitinib (UPA), tofacitinib, and ozanimod in the adult population could prove positive outcomes in the pediatric population. Established off-label therapies inch closer to comparable safety, efficacy, and outcomes in pediatric IBD use. Outcomes and use of newer biologic therapies in patients with pediatric IBD have improved with increased rates of steroid-free clinical remission (SFCR). Novel therapies, including Janus kinase (JAK) inhibitors and sphingosine-1-phosphate receptor (S1Pr) modulators, require further studies but could also prove effective.</p>","PeriodicalId":520221,"journal":{"name":"Pediatric discovery","volume":"3 2","pages":"e70011"},"PeriodicalIF":0.0,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12258086/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645128","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}
Pediatric discoveryPub Date : 2025-06-14eCollection Date: 2025-06-01DOI: 10.1002/pdi3.70010
Karen Spruyt
{"title":"SLEEP-WAKE: A Comprehensive Mnemonic for Systematic Screening of Sleep-Related Issues.","authors":"Karen Spruyt","doi":"10.1002/pdi3.70010","DOIUrl":"10.1002/pdi3.70010","url":null,"abstract":"","PeriodicalId":520221,"journal":{"name":"Pediatric discovery","volume":"3 2","pages":"e70010"},"PeriodicalIF":0.0,"publicationDate":"2025-06-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12258100/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645138","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}