Clinical and Experimental Pediatrics最新文献

{"title":"Cost-effectiveness of newborn screening for severe combined immunodeficiency: a systematic review.","authors":"Rezwanul Rana, Syed Afroz Keramat, Moin Ahmed","doi":"10.3345/cep.2025.00052","DOIUrl":"https://doi.org/10.3345/cep.2025.00052","url":null,"abstract":"<p><p>Severe combined immunodeficiency (SCID) is a rare genetic disorder that causes severe infections and death in early childhood. Newborn bloodspot screening (NBS) for SCID using the T-cell receptor excision circle assay can revolutionize the early detection and treatment of infants with SCID, leading to improved quality of life and life expectancy. This systematic review aimed to examine the cost-effectiveness of universal NBS for SCID. The MEDLINE, Embase, NHS Economic Evaluation Database, Health Technology Assessment, Scopus, and EconLit databases were searched for studies of the NBS for SCID published between January 2008 and March 2024. A standardized data extraction form was used to gather pertinent data such as characteristics, design, perspective, screening strategies and costs, health outcomes, incremental cost-effectiveness ratios, and sources of uncertainty. Eight studies met our inclusion criteria: six cost-utility analyses and two cost-effectiveness analyses. All studies were model-based economic evaluations. These studies indicated that universal NBS for SCID is highly likely to demonstrate health system and societal cost-effectiveness. The incremental cost-effectiveness ratio per quality-adjusted life-year gained ranged from $30,214-54,282 (USD 2022 value). Evidence suggests that early treatment of SCID is beneficial and that population-based NBS provides good value for the money. However, policymakers require better information about optimal treatment and treatment and screening costs to make informed decisions regarding competing healthcare priorities.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144031654","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":"Effect of vitamin C supplement in treatment of childhood pneumonia requiring hospitalization: A randomized controlled trial.","authors":"Chutima Phuaksaman, Katechan Jampachaisri, Klaita Srisingh","doi":"10.3345/cep.2024.01970","DOIUrl":"https://doi.org/10.3345/cep.2024.01970","url":null,"abstract":"<p><strong>Background: </strong>The role of vitamin C in children with community-acquired pneumonia (CAP) in children is controversial; moreover, a standard dose is lacking.</p><p><strong>Purpose: </strong>This study aimed to evaluate the ability of vitamin C to reduce symptom severity and length of hospital stay among children with CAP as well as determine its optimal dose.</p><p><strong>Methods: </strong>This randomized controlled clinical trial was conducted between July 2020 and October 2023. The participating patients were aged 6 months to 15 years, had CAP, and required hospitalization at Naresuan University Hospital. The patients were randomly assigned to placebo, low-dose vitamin C (15 mg/kg/dose every 6 h), and high-dose vitamin C (30 mg/kg/dose every 6 h) groups. Treatment was provided until discharge and doses maximized after 3 days. The patients' clinical symptoms and side effects were recorded every 12 h.</p><p><strong>Results: </strong>This study included 143 patients (median age, 3 years). The clinical severity score improved significantly in the low- and high-dose vitamin C versus placebo groups at 48-72 h. Vitamin C supplementation did not reduce the length of hospital stay in any group.</p><p><strong>Conclusion: </strong>Vitamin C supplementation did not reduce the length of hospital stay among patients with CAP who required hospitalization. However, it improved the mean clinical severity score, with the greatest reduction observed at 48 h post-treatment. A dose of 15 mg/kg was demonstrated effective with minimal side effects.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144056850","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":"Recent advances in understanding pathophysiology of non-nutritional stunting in very preterm infants.","authors":"Eduardo Cuestas, Alina Rizzotti","doi":"10.3345/cep.2024.01354","DOIUrl":"10.3345/cep.2024.01354","url":null,"abstract":"<p><p>Very preterm infants (VPIs) often experience extrauterine growth failure. Therefore, aggressive nutritional management of VPIs is recommended with the goal of achieving the postnatal growth of an equivalent fetus. However, VPIs frequently present postnatal length growth restriction at term-corrected age that remains lower than the standard weight and have greater fat mass and lower lean and bone mass than term-born infants. This condition differs from the classic pattern of infant undernutrition defined as a significantly lower weight for a given length. Moreover, it suggests that nonnutritional factors play a key role in length growth restriction. While weight faltering has been extensively studied, the significance of length growth failure in VPIs has only recently emerged. The nonnutritional factors underlying poor length growth in VPIs are currently not fully understood. In this review, we address recent advances in our understanding of the pathophysiology of length growth restriction, which has been identified as a major predictor of adverse neurodevelopmental and cognitive outcomes in VPIs. First, we review the shortand long-term consequences of poor length growth in VPIs; next, we highlight the effects of nonnutritional factors on postnatal length growth with focus on sustained neonatal inflammation; and finally, we discuss hypothesis and future lines of research attempting to understand the complex inflammatory-endocrine interactions and pathophysiological changes during early postnatal life, appropriately guide and apply clinical strategies aimed at optimizing length growth of VPIs, and identify evidence of the associations between sustained neonatal inflammation, stunting, and long-term health risks and the potential implications thereof.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"287-297"},"PeriodicalIF":3.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11969205/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142898956","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":"Global breastfeeding efforts: a long way to go.","authors":"Hye-Jung Shin","doi":"10.3345/cep.2024.01361","DOIUrl":"10.3345/cep.2024.01361","url":null,"abstract":"","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"300-302"},"PeriodicalIF":3.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11969202/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142629626","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":"Incidence of neural tube defects in tertiary care university hospital in Bangladesh.","authors":"Ismat Jahan, Md Arif Hossain, Shah Nizam Uddin, Sadeka Choudhuri Moni, Mohammad Kamrul Hassan, Sanjoy Kumer Dey, Mohammad Abdul Mannan, Mohammod Shahidullah","doi":"10.3345/cep.2024.01578","DOIUrl":"https://doi.org/10.3345/cep.2024.01578","url":null,"abstract":"<p><strong>Background: </strong>Although national population-based birth defect prevalence estimates are unavailable for Bangladesh specifically, data extrapolated from the March Dimes Global Birth Defects Report indicate a prevalence of neural tube defects (NTD) of 4.7 per 1000 live births.</p><p><strong>Purpose: </strong>This study aimed to determine the prevalence of NTD among infants born at a tertiary care multidisciplinary referral hospital in Bangladesh.</p><p><strong>Methods: </strong>Live born infants with NTD were prospectively enrolled in 2015-2021. Each enrolled NTD case was examined for type, location, and associated anomalies. The overall and annual prevalence rates were then calculated.</p><p><strong>Results: </strong>A total of 10,372 newborns were enrolled; of them, 68 had NTD (incidence, 6.4 [range, 4.59-11.2] per 1000 live births). The mean maternal age was 27.49 ± 4.72 years. Three-quarters of the NTD cases were detected at birth, and 94% of the mothers reported not taking periconceptional folic acid supplements. The meningomyelocele complex was the most frequent location. Two peaks in incidence were noted in 2017 and 2021 (10.28 and 11.2 per 1000 live births, respectively). The distribution of different NTD types included meningomyelocele at 53%, encephalocele at 26.6%, meningocele at 16%, and anencephaly at 4.4%. A male predominance was noted overall except for anencephaly. The most common location was the lumbosacrum (47%). The NTD was isolated in 20.59% (14/68) of cases and associated with other malformations in 80% (54/68) of cases.</p><p><strong>Conclusion: </strong>The incidence of NTD was 6.4 per 1000 live births at a leading tertiary care multidisciplinary referral center in Bangladesh. However, this figure might not reflect the incidence of NTD in the wider population.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144032490","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":"Impact of obesity on pulmonary function of preschool children: an impulse oscillometry study.","authors":"Anuvat Klubdaeng, Kanokporn Udomittipong, Apinya Palamit, Pawinee Charoensittisup, Khunphon Mahoran","doi":"10.3345/cep.2024.01053","DOIUrl":"10.3345/cep.2024.01053","url":null,"abstract":"<p><strong>Background: </strong>The increasing global prevalence of obesity poses significant public health problems, as obesity exerts adverse effects on many systems and lung function. However, research on the lung function of preschool children with obesity is limited and inconclusive. In addition, studies specific to obesity indices that influence lung function in young children with obesity are limited.</p><p><strong>Purpose: </strong>This study aimed to evaluate lung function of obese versus normal-weight preschool children using impulse oscillometry (IOS) and identify obesity indices predictive of altered lung function.</p><p><strong>Methods: </strong>We enrolled obese children aged 3-7 years as well as age- and sex-matched normal-weight controls. The participants underwent IOS assessments that measured the resistance at 5 Hz (R5) and 20 Hz (R20), the difference in resistance between these frequencies (R5-R20), reactance at 5 Hz (X5), resonance frequency, and reactance area (AX). We compared these parameters between groups and analyzed the correlations between obesity indices and IOS measures within the obese group using multiple linear regression.</p><p><strong>Results: </strong>The study included 68 participants (n=34 each group). In the obese group, significantly higher values were observed for R5 (adjusted for height, P=0.02; % predicted, P=0.01; z score, P<0.001), R5-R20 (absolute value, P=0.002; adjusted for height, P=0.001), and AX (z score, P=0.01). AX adjusted for height showed a greater trend (P=0.07). The waist-to-height ratio was the most robust independent predictor of total and peripheral airway resistance, with increases in R5 (b=1.65, P=0.02) and R5-R20 (b=1.39, P=0.03) and a near-significant correlation with AX (b=12.12, P=0.06).</p><p><strong>Conclusion: </strong>Preschool children with obesity exhibit impaired lung function, characterized by elevated total and peripheral airway resistance. Waist-to-height ratio was the strongest predictor of these changes.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"319-325"},"PeriodicalIF":3.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11969207/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142629710","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":"Occurrence of stroke in children and young adults in Indonesia: a multicenter private hospital study.","authors":"Jeanne Leman, Veli Sungono, Yosua Timotius Haryono, Muhammad Adam Mudzakir, Dewi Lestari Rahmawati, Callistus Bruce Henfry Sulay, Gilbert Sterling Octavius","doi":"10.3345/cep.2024.01214","DOIUrl":"10.3345/cep.2024.01214","url":null,"abstract":"<p><strong>Background: </strong>Most studies that estimate the occurrence of childhood stroke use heterogeneous methods and rely on International Classification of Diseases codes, a strategy that may be unreliable.</p><p><strong>Purpose: </strong>This study aimed to estimate the occurrence of childhood stroke in Indonesia using imaging and clinical data from a private hospital network.</p><p><strong>Methods: </strong>This cross-sectional study used consecutive retrospective multicenter data samples collected in 2019- 2023. The study cohort included children aged >28 days to young adults aged 24 years with confirmed ischemic or hemorrhagic stroke on computed tomography (CT) or magnetic resonance imaging (MRI). The 1-year occurrence was calculated, and the data analysis was performed using IBM SPSS Statistics ver. 26.0.</p><p><strong>Results: </strong>Over 5 years, the performance of 8,987 CT and 6,133 MRI scans resulted in the identification of 1,074 stroke cases. The average patient age was 14.8±7.0 years. Stroke occurrence was highest in 2021 (9.08%) and lowest in 2022 (5.91%). Male patients accounted for 67.9% of cases, with hemorrhagic strokes accounting for 83.4% of the total, primarily resulting from accidents (73.2%) and predominantly occurring in the frontal region (37.1%). A significant majority of cases (66.7%) were reported in Java. Males had a relative risk of 1.93 (95% confidence interval, 1.48-2.52; P<0.001) for hemorrhagic stroke versus females.</p><p><strong>Conclusion: </strong>The incidence of childhood stroke revealed critical epidemiological trends and disparities, emphasizing the need for targeted public health interventions and enhanced stroke prevention strategies in Indonesia.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"303-310"},"PeriodicalIF":3.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11969204/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142629756","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":"Cerebral organoid research for pediatric patients with neurological disorders.","authors":"Jin Eun, Jung Eun Lee, Seung Ho Yang","doi":"10.3345/cep.2024.01235","DOIUrl":"10.3345/cep.2024.01235","url":null,"abstract":"<p><p>Cerebral organoids derived from human induced pluripotent stem cells offer a groundbreaking foundation for the analysis of pediatric neurological diseases. Unlike organoids from other somatic systems, cerebral organoids present unique challenges, such as the high sensitivity of neuronal cells to environmental conditions and the complexity of replicating brain-specific architectures. Cerebral organoids replicate the human brain development and pathology, enabling research on conditions such as microcephaly, Rett syndrome, autism spectrum disorders, and brain tumors. This review explores the utility of cerebral organoids for modeling diseases and testing therapeutic interventions. Despite current limitations such as variability and lack of vascularization, recent technological advancements have improved the reliability and application of such interventions. Cerebral organoids provide valuable insight into the mechanisms underlying complex neural disorders and hold promise as novel treatment strategies for pediatric neurological diseases.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"269-277"},"PeriodicalIF":3.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11969208/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142751894","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":"Advancements and challenges in neonatal resuscitation: embracing laryngeal mask airways for improved outcomes.","authors":"Jang Hoon Lee","doi":"10.3345/cep.2024.01088","DOIUrl":"10.3345/cep.2024.01088","url":null,"abstract":"","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"298-299"},"PeriodicalIF":3.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11969200/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142751888","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":"Establishing an induced pluripotent stem cell bank using urine cells from pediatric patients with neurogenetic diseases.","authors":"Hien Bao Dieu Thai, WonWoo Jung, Sol Choi, Woo Joong Kim, JangSup Moon, ByungChan Lim","doi":"10.3345/cep.2024.01830","DOIUrl":"https://doi.org/10.3345/cep.2024.01830","url":null,"abstract":"<p><strong>Background: </strong>Inadequate knowledge of the fundamental mechanisms underlying pediatric neurological disorders impedes their effective treatment. Induced pluripotent stem cells (iPSCs) are essential for exploring the course of neurological diseases because they enable disease modeling at the cellular level.</p><p><strong>Purpose: </strong>This study aimed to generate an iPSC bank using urine cells (UCs) for clinical applications, particularly the study of pediatric neurogenetic diseases. Urine sample collections can benefit a large donor population because they use a non-invasive, painless, and simple technique that provides plentiful cells for iPSC generation.</p><p><strong>Methods: </strong>UCs were isolated from the urine of donors with specific diseases (n = 12; seven males, five females). The UCs were reprogrammed into iPSCs using episomal plasmid vectors and key transcription factors (OCT3/4, SOX2, KLF4, L-MYC, and LIN28). Quantitative polymerase chain reaction and immunocytochemical analyses confirmed the expression of pluripotent genes (OCT3/4, SOX2, NANOG, and LIN28) and proteins (OCT4, NANOG, SSEA-4, and TRA-1-60). Trilineage differentiation was investigated by immunostaining embryonic body-derived iPSCs for -tubulin III, smooth muscle actin, and alpha-fetoprotein. The genomic stability of the iPSCs was assessed using chromosomal microarray (CMA).</p><p><strong>Results: </strong>UCs were successfully isolated from patients with various early-onset neurogenetic diseases and reprogrammed into iPSCs. The iPSCs were confirmed as pluripotent and capable of trilineage differentiation as evidenced by the enhanced expression of relevant genes and proteins. The genomic profiles of the iPSCs were assessed using CMA, which revealed that four of the 12 lines exhibited pathogenic chromosomal deletions or duplications. Interestingly, repeated CMA tests using earlier-passage cells resulted in normal findings in one of the four iPSC lines. These findings highlight the need for genetic screening throughout the culture period.</p><p><strong>Conclusion: </strong>Here we used UCs to successfully develop an early-onset neurogenetic disease iPSC bank that offers an efficient protocol for expanding patient accessibility in pediatric neurogenetic research.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144042271","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}