Clinical and Experimental Pediatrics最新文献

{"title":"Promising role of voxelotor in managing sickle cell disease in children: a narrative review.","authors":"Amit Agrawal, Gaurav Jadon, Japna Singh, Dalwinder Janjua","doi":"10.3345/cep.2024.00500","DOIUrl":"10.3345/cep.2024.00500","url":null,"abstract":"<p><p>Sickle cell disease (SCD) is characterized by chronic hemolytic anemia and intermittent vasoocclusive crises. To date, 4 disease-modifying drugs have been approved for the treatment of SCD: hydroxyurea (an S-phase inhibitor), L-glutamine (an amino acid), crizanlizumab (a P-selectin inhibitor), and voxelotor (a hemoglobin S polymerization inhibitor). Preclinical studies suggested that voxelotor effectively treats SCD and sickle cell anemia (SCA). In a phase III trial, voxelotor-treated patients showed significantly elevated hemoglobin levels (>1 g/dL from baseline) compared to placebo-treated patients. The group that received voxelotor also showed a greater decrease in hemolytic markers but a comparable incidence of side effects. Six ongoing clinical trials also sought to ascertain the effectiveness and safety of high-dose voxelotor when administered to children younger than 12 years. Studies assessing their long-term efficacy and safety are needed to fully understand the role of voxelotor in treating SCD/SCA. In this review, we discuss the mechanisms, trials to date, and future treatment directions of voxelotor.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"106-114"},"PeriodicalIF":3.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825115/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142629768","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":"Clinical characteristics and associated factors of pediatric acute necrotizing encephalopathy: a retrospective study.","authors":"Huiling Zhang, Yilong Wang, Qianyun Ding, Xuekun Li, Sheng Ye","doi":"10.3345/cep.2024.00794","DOIUrl":"10.3345/cep.2024.00794","url":null,"abstract":"<p><strong>Background: </strong>In the clinic, pediatric acute necrotizing encephalopathy (ANE) primarily affects children under 5 years of age and is characterized by severe brain damage and high mortality. However, some challenges remain regarding the diagnosis and treatment of ANE. In the present study, we analyzed the clinical characteristics and related factors of ANE with the aim of providing improved diagnostic and treatment strategies.</p><p><strong>Purpose: </strong>The main purpose of this study is to gain a deeper understanding of the clinical characteristics and pathophysiology of ANE through a retrospective study, and to provide clinical practitioners with more effective. diagnostic and treatment strategies to improve patient survival rates and quality of life.</p><p><strong>Methods: </strong>Thirty-four pediatric ANE patients admitted to Zhejiang University School of Medicine Hospital between February 2019 and December 2023 were included in this study. To identify the factors associated with mortality, clinical, laboratory and imaging data were analyzed with independent-sample t tests, Mann-Whitney U tests, Fisher exact probability tests and receiver operating characteristic curve analyses.</p><p><strong>Results: </strong>In this cohort of 34 patients, the most common symptoms were fever, seizures, altered consciousness, vomiting, diarrhea and shock. The mortality rate was 55.9%. Laboratory tests revealed that patients who died had higher creatinine, lactate, activated partial thromboplastin time, thrombin time, interleukin (IL)-6, IL-10, creatine kinase, and D-dimer than survivors. Imaging examinations predominantly revealed symmetrical lesions in the thalamus. The fatal group displayed lower Glasgow Coma Scale (GCS) scores and severe complications. Other factors related to mortality included the arterial pH, GCS score and hospitalization duration.</p><p><strong>Conclusion: </strong>The most common symptoms of ANE are fever, seizures, altered consciousness, vomiting, diarrhea and shock, and ANE has a high mortality rate. The GCS score and arterial pH are critical biomarkers for assessing the severity of ANE.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"153-162"},"PeriodicalIF":3.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825111/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142629586","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":"Differences in immune cells and gene expression in human milk by parity on integrated scRNA sequencing.","authors":"Dae Yong Yi, Hong-Jai Park, Min Sun Shin, Hyoungsu Kim, Sang Jin Lee, Insoo Kang","doi":"10.3345/cep.2024.01585","DOIUrl":"10.3345/cep.2024.01585","url":null,"abstract":"<p><strong>Background: </strong>Human breast milk (HBM) is an important source of tolerogenic immune mediators that influence the infant immune system. HBM-derived immune components are affected by various factors; however, few studies have examined the relationship between parity and immune cell profiles of HBM.</p><p><strong>Purpose: </strong>This study aimed to clarify the effects of parity on HBM immune cell heterogeneity and gene expression by integrating and analyzing publicly available single-cell RNAsequencing (scRNA-seq) datasets.</p><p><strong>Methods: </strong>We clarified the effects of parity on HBM immune cell heterogeneity and gene expression by integrating and analyzing publicly available scRNA-seq datasets.</p><p><strong>Results: </strong>The proportion of innate immune cells was significantly higher in the primiparous versus multiparous group, whereas the proportion of adaptive immune cells was significantly higher in the multiparous group (P=0.021). The 2 immune clusters were reannotated and classified into monocyte, T/B cell, and CD45¯ groups. The proportions of monocytes and T/B cells were higher in the primiparous and multiparous groups, respectively. In a gene set enrichment analysis of monocytes, genes with a direct role in the infant immune system and immune response-related genes were more highly expressed in the primiparous group.</p><p><strong>Conclusion: </strong>Our results support the parity-dependent differences in gene expression between innate and adaptive immune cells.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"141-152"},"PeriodicalIF":3.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825117/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142984930","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":"Evaluation of pediatric migraine triggers: a single-center study.","authors":"Hey-Joon Son, Joo-Ok Jin, Kon-Hee Lee","doi":"10.3345/cep.2024.00738","DOIUrl":"10.3345/cep.2024.00738","url":null,"abstract":"<p><strong>Background: </strong>Pediatric migraines are primarily treated with medications. However, recognizing the triggers related to patient behaviors and lifestyle is also important.</p><p><strong>Purpose: </strong>This study aimed to evaluate the factors that trigger pediatric migraines.</p><p><strong>Methods: </strong>This study included 102 pediatric patients with migraine diagnosed using the third edition of the International Classification of Headache Disorders who visited our pediatric headache clinic between November 2021 and October 2022. We reviewed the patients' clinical features, imaging studies, screening tests for behaviors and emotions, and questionnaires regarding triggers. Statistical analyses were performed using the independent sample Student t test and linear-by-linear association test.</p><p><strong>Results: </strong>The 102 patients (44 males, 58 females; mean age, 12.0±2.9 years) were classified into migraine without aura (n=58) and migraine with aura (n=44) groups. Sleep disturbances were the most frequent trigger (76.5%). Other triggers included academic stress (66.7%), motion sickness (62.7%), and fatigue (52.9%). Academic stress was the most significant trigger for pain severity in 44.1% of patients. Abnormal behavioral or psychiatric assessment results were associated with a higher number of triggers.</p><p><strong>Conclusion: </strong>This study identified sleep disturbance and academic stress as common triggers of pediatric migraine, with academic stress being the most intense. Clinicians should understand these triggers and advise patients to avoid them by changing their lifestyles, if possible.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"163-169"},"PeriodicalIF":3.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825112/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142629611","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":"Effect of high-frequency oscillatory ventilation with intermittent sigh breaths on carbon dioxide levels in neonates.","authors":"Kulthida Baingam, Anucha Thatrimontrichai, Manapat Praditaukrit, Gunlawadee Maneenil, Supaporn Dissaneevate","doi":"10.3345/cep.2024.01011","DOIUrl":"10.3345/cep.2024.01011","url":null,"abstract":"<p><strong>Background: </strong>High-frequency oscillatory ventilation (HFOV) minimizes ventilator-induced lung injuries. Spontaneous sigh breathing may augment the functional residual capacity, increase lung compliance, and recruit atelectatic alveoli.</p><p><strong>Purpose: </strong>To evaluate the difference in the partial pressure of carbon dioxide (PaCO2) in neonates receiving invasive HFOV as the primary mode of respiratory support before versus after sigh breaths (Sighs).</p><p><strong>Methods: </strong>This prospective study was conducted between January and December 2023. Intubated preterm and term neonates who underwent HFOV with an available arterial line were enrolled in this study after informed parental consent was obtained. Sighs were set at a frequency of 3 breaths/min and pressure of 5 cmH2O above the mean airway pressure for 2 hours. Arterial blood gas was collected before and after Sighs and analyzed using 2 dependent tests.</p><p><strong>Results: </strong>Thirty neonates with a mean gestational age of 33.6±4.1 weeks and median date of intervention of 1.88 days (interquartile range, 0.87-3.79 days) were enrolled. The mean PaCO2 level was significantly lower in the HFOV with Sighs group (45.2±6.6 mmHg) versus the HFOV alone group (48.8±3.1 mmHg) with a mean difference (MD) of -3.6 mmHg (95% confidence interval [CI], -6.3 to -0.9; P=0.01). Subgroup analyses indicated the ability of Sighs to reduce the PaCO2 level in neonates with respiratory distress syndrome (n=15; MD [95% CI]=-4.2 [-8.2 to -0.2] mmHg; P= 0.04).</p><p><strong>Conclusion: </strong>Sighing can reduce PaCO2 levels in neonates ventilated with HFOV, particularly those with respiratory distress syndrome.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"178-184"},"PeriodicalIF":3.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825110/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142629590","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":"Characteristics of temper tantrums in 1-6-year-old children and impact on caregivers.","authors":"Warangkana Prutipaisan, Issarapa Chunsuwan, Tippawan Hansakunachai, Paskorn Sritipsukho","doi":"10.3345/cep.2024.00766","DOIUrl":"10.3345/cep.2024.00766","url":null,"abstract":"<p><strong>Background: </strong>Temper tantrums are common behavioral difficulties in children. Although they are generally considered a normal part of development, certain characteristics- such as aggression, prolonged duration, and frequent occurrences-have been linked to psychological issues and can negatively impact both the child and their caregivers.</p><p><strong>Purpose: </strong>To study the prevalence and characteristics of temper tantrums in children aged 1-6 years at daycare and in kindergarten in Thailand as well as the impact of problematic and nonproblematic tantrums on their caregivers' emotional well-being.</p><p><strong>Methods: </strong>This cross-sectional descriptive study was conducted in 2021. The main caregivers of the participants completed self-reported questionnaires that collected their demographic information and the temper tantrum characteristics and impacts on the caregivers' emotions.</p><p><strong>Results: </strong>Data from 211 children were included in this study. The mean child age was 4.4±1.2 years. Two hundred one parents (95.3%) reported that their children had at least one tantrum behavior, of which verbal were the most common (94.5%). One hundred and 11 children (55.2%) had tantrums defined as problematic: exhibiting aggressive physical behavior, duration >15 minutes, frequency >3 days/ wk. The mean emotional burden scores of the children's problematic and nonproblematic temper tantrums on their parents were 23.3±8.4 and 17.7±8.3 (maximum, 55; P=0.001), respectively, showing a statistically significant difference.</p><p><strong>Conclusion: </strong>Tantrums are common in children aged 1-6 years, but their expression varies. Problematic tantrums were reported for approximately half of the children and significantly impacted their caregivers' emotions. Therefore, children with problematic tantrums and their families should receive assistance.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"170-177"},"PeriodicalIF":3.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825109/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142629582","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":"Value of transabdominal ultrasonography for diagnosing functional constipation in children: a systematic review and meta-analysis.","authors":"Duc Long Tran, Phu Nguyen Trong Tran, Paweena Susantitaphong, Phichayut Phinyo, Palittiya Sintusek","doi":"10.3345/cep.2024.00927","DOIUrl":"10.3345/cep.2024.00927","url":null,"abstract":"<p><p>Transabdominal ultrasonography is increasingly used as a novel modality for detecting pediatric functional constipation (FC). This systematic review and metaanalysis aimed to assess the diagnostic parameters of FC including rectal diameter (RD) and anterior rectal wall thickness. A systematic search was conducted of the Ovid MEDLINE, Embase, Scopus, and PubMed databases through September 29, 2023, to identify studies comparing RD and anterior wall thickness using transabdominal ultrasonography in children with versus without FC. Metaanalyses were performed using random-effects models to calculate the weighted mean differences (MDs) in RD and anterior wall thickness. Comprehensive Meta-Analysis ver. 3, R, and Review Manager ver. 5.4.1 software were used to assess the optimal cutoff, sensitivity, specificity, and area under the curve (AUC). Fourteen studies involving 1,255 children (mean age, 6.21±2.3 years) were included. The mean RD was significantly larger in constipated children versus controls (MD, 10.35 mm; 95% confidence interval [CI], 6.97-13.74; P<0.001; I2=94%). A meta-regression showed no significant effects of age, weight, or height on RD. An optimal RD cutoff point of 31 mm was suggested by a pooled analysis with an AUC of 0.86 (95% CI, 0.8-0.91; P<0.001), sensitivity of 0.75 (95% CI, 0.59-0.86), and specificity of 0.84 (95% CI, 0.68-0.93). The mean anterior rectal wall thickness was greater among constipated children than among controls (MD, 0.44; 95% CI, -0.26 to 1.13; P=0.22), but this difference was not statistically significant. RD measured using transabdominal ultrasonography with a cutoff point of 31 mm exhibited good diagnostic accuracy for diagnosing FC in children.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"127-135"},"PeriodicalIF":3.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825114/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142629773","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":"Absolute versus functional iron deficiency.","authors":"Hye Lim Jung","doi":"10.3345/cep.2023.01732","DOIUrl":"10.3345/cep.2023.01732","url":null,"abstract":"","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"138-140"},"PeriodicalIF":3.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825113/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142629573","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":"Screen time and neurodevelopment in preschoolers: addressing a growing concern in pediatric practice.","authors":"Soongang Park, Hyewon Woo","doi":"10.3345/cep.2024.01536","DOIUrl":"https://doi.org/10.3345/cep.2024.01536","url":null,"abstract":"","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-01-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142984949","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":"Peripheral nerve sheath tumors in the head and neck in patients with APC gene deletion mutations: a case report and scoping review of the literature.","authors":"Koral Blunt, Monirah Albathi, Miriam Conces, Tendy Chiang","doi":"10.3345/cep.2024.01375","DOIUrl":"10.3345/cep.2024.01375","url":null,"abstract":"<p><p>Adenomatous Polyposis Coli (APC) is a tumor suppressor gene expressed throughout the body. APC mutations increase the risk of malignancy and are often characterized by syndromes that encompass a spectrum of neoplastic manifestations, such as familial adenomatous polyposis (FAP). We present a rare case of palatal peripheral nerve sheath tumor in the context of APC gene mutation. A 17-year-old male with a significant history of FAP presented to our clinic for with globus sensation for 5 months with increasing discomfort. Flexible nasolaryngoscopy revealed a pedunculated lesion attached to the posterior surface of the soft palate. Imaging was obtained and confirmed a soft tissue homogenous mass contiguous with the soft palate. Endoscopic-assisted transoral resection was performed and pathologic features were consistent with schwannoma. We also discuss the spectrum of benign neoplastic lesions. Current literature fails to describe pharyngeal masses in the setting of APC gene mutations. The purpose of this case report is to describe a patient presentation of a symptomatic pharyngeal tumor with a known APC gene mutation and explore the differential diagnoses that must be considered.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-01-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142984931","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}