Clinical and Experimental Pediatrics最新文献

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Protocolized sedation may reduce ventilation and sedation requirements in the pediatric intensive care unit: a systematic review and meta-analysis.
IF 3.2
Clinical and Experimental Pediatrics Pub Date : 2025-02-19 DOI: 10.3345/cep.2024.01711
Ambrus Szemere, Alíz Fazekas, Anna Réka Sebestyén, Rani Ezzeddine, Veronika Upor, Marie Anne Engh, Péter Hegyi, Zsolt Molnár, Klára Horváth
{"title":"Protocolized sedation may reduce ventilation and sedation requirements in the pediatric intensive care unit: a systematic review and meta-analysis.","authors":"Ambrus Szemere, Alíz Fazekas, Anna Réka Sebestyén, Rani Ezzeddine, Veronika Upor, Marie Anne Engh, Péter Hegyi, Zsolt Molnár, Klára Horváth","doi":"10.3345/cep.2024.01711","DOIUrl":"https://doi.org/10.3345/cep.2024.01711","url":null,"abstract":"<p><p>This study aimed to evaluate the effectiveness and safety of protocolized sedation in mechanically ventilated pediatric intensive care unit (PICU) patients. A comprehensive search was conducted in MEDLINE, CENTRAL, Embase, Web of Science, and Scopus from inception to October 18, 2023. Randomized controlled trials and observational studies that compared protocol-directed sedation management with conventional sedation regimens in pediatric patients who required invasive mechanical ventilation (IMV) for >24 h were included. Twenty-six studies (15,214 participants) were included. We found a statistically significant reduction in IMV duration (median difference [MD]=-13.88 h; 95% confidence interval [CI], -25.46 to -2.29; P=.022), PICU length of stay (MD=-0.64 days; 95% CI, -1.26 to -0.02; P=.045). We found significant reductions in the duration (MD= -1.28 days; 95% CI, -2.26 to -0.31; P=.016) and peak dose (MD=-0.05 mg/kg/h; 95% CI, -0.11 to 0.002; P=.044) of benzodiazepines. A significant increase was found in the odds of unplanned extubation (odds ratio=1.13; 95% CI, 1.02 to 1.26; P=.029). We found no significant results regarding the other outcomes. Our results suggest that protocolized sedation may reduce ventilation requirements and PICU length of stay; however, these findings were not confirmed by randomized controlled trials. Moreover, we observed a trend toward a reduction in sedative exposure and an increased odds of unplanned extubation.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143459933","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}
引用次数: 0
The Korea Infant Physical Growth Examination Survey (KIPGroS): a study protocol.
IF 3.2
Clinical and Experimental Pediatrics Pub Date : 2025-02-13 DOI: 10.3345/cep.2024.00297
Jong Woo Hahn, MinSoo Shin, Jin Gyu Lim, Yoon-Joo Kim, Ki Soo Kang, Narae Lee, Seong Hee Jeong, Mun Hui Jeong, Yeoun Joo Lee, Eui Kyung Choi, Jung Ok Shim, Jee Yoon Park, Chan-Wook Park, Joo Young Kim, Su Jin Jeong, Young Hwa Jung, Jaehyun Kim, Chang Won Choi, Ju Whi Kim, Seung Han Shin, Yun Jeong Lee, Young Ah Lee, Choong-Ho Shin, Seung-Sik Hwang, Young Eun Kim
{"title":"The Korea Infant Physical Growth Examination Survey (KIPGroS): a study protocol.","authors":"Jong Woo Hahn, MinSoo Shin, Jin Gyu Lim, Yoon-Joo Kim, Ki Soo Kang, Narae Lee, Seong Hee Jeong, Mun Hui Jeong, Yeoun Joo Lee, Eui Kyung Choi, Jung Ok Shim, Jee Yoon Park, Chan-Wook Park, Joo Young Kim, Su Jin Jeong, Young Hwa Jung, Jaehyun Kim, Chang Won Choi, Ju Whi Kim, Seung Han Shin, Yun Jeong Lee, Young Ah Lee, Choong-Ho Shin, Seung-Sik Hwang, Young Eun Kim","doi":"10.3345/cep.2024.00297","DOIUrl":"10.3345/cep.2024.00297","url":null,"abstract":"<p><p>Growth charts are important tools used to evaluate the growth status of children and estimate the nutritional and health status of the general population. In Korea, the national standardized growth charts were updated in 2017. However, the growth charts developed by the World Health Organization (WHO) are being adopted for children under 3 years of age despite a lack of evidence for their applicability to Koreans. Here we aimed to collect accurate physical measurement data, develop growth charts for infants and toddlers under 3 years of age based on breastfeeding, and evaluate the feasibility of adopting WHO growth standards as a reference in Korea. Beginning April 2020, six general and local hospitals were selected nationwide. Mothers and newborns without growth restriction-related factors were enrolled at birth, and they were followed up monthly until 12 months and every 3 months until 36 months. Data were collected on physical measurements; eating habits; childhood morbidity; perinatal factors; and socioeconomic, demographic, and environmental characteristics. All participating hospitals used the same standard equipment. The Korea Infant Physical Growth Examination Survey evaluated the suitability of the WHO growth charts and contributed to the next revision of the Korean growth charts. The results of this study provide a basis for developing health-related strategies for treating children in South Korea.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143442330","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}
引用次数: 0
Eosinophil-derived neurotoxin levels can predict allergic disease development and atopic march in children. 嗜酸性粒细胞衍生神经毒素水平可预测儿童过敏性疾病的发展和特应性进展。
IF 3.2
Clinical and Experimental Pediatrics Pub Date : 2025-02-03 DOI: 10.3345/cep.2024.01382
Zak Callaway, Chang-Keun Kim
{"title":"Eosinophil-derived neurotoxin levels can predict allergic disease development and atopic march in children.","authors":"Zak Callaway, Chang-Keun Kim","doi":"10.3345/cep.2024.01382","DOIUrl":"https://doi.org/10.3345/cep.2024.01382","url":null,"abstract":"<p><p>In some children, atopic manifestations begin with atopic dermatitis and progress to allergic asthma and allergic rhinitis; of them, a small subset experience food allergies as well. This progression shares genetic and environmental predisposing factors and immunological features, such as allergen-specific T helper type 2 responses, that manifest as specific immunoglobulin E production and eosinophil activation. Eosinophil-derived neurotoxin (EDN), which is released by eosinophils during this activation, shows promise as a reliable and accurate biomarker. EDN levels are elevated in a subset of patients with atopic march-associated conditions. Elevated EDN levels predict allergic disease development, demonstrating that EDN is a good biomarker for the prognosis, diagnosis, treatment, and monitoring of allergic diseases comprising atopic march. The early measurement of EDN would help identify those who are more likely to develop allergic diseases later in life. Thus, the early detection and treatment of elevated EDN could lead to better outcomes, including halting atopic march.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143123799","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}
引用次数: 0
Differential effects of dietary and physical activity interventions on adiposity of children with obesity.
IF 3.2
Clinical and Experimental Pediatrics Pub Date : 2025-02-03 DOI: 10.3345/cep.2024.01347
Anekchoke Tangtongsoong, Chonnikant Visuthranukul, Yuda Chongpison, Sirinuch Chomtho
{"title":"Differential effects of dietary and physical activity interventions on adiposity of children with obesity.","authors":"Anekchoke Tangtongsoong, Chonnikant Visuthranukul, Yuda Chongpison, Sirinuch Chomtho","doi":"10.3345/cep.2024.01347","DOIUrl":"https://doi.org/10.3345/cep.2024.01347","url":null,"abstract":"<p><strong>Background: </strong>Managing obesity in children remains challenging. In addition to body mass index (BMI), incorporating body composition into evaluations of post-obesity interventions would help assess changes in adiposity.</p><p><strong>Purpose: </strong>This study aimed to identify the relationship between dietary intake, physical activity, and changes in BMI z-scores and adiposity among children with obesity.</p><p><strong>Methods: </strong>Children aged 7-15 years with obesity received monthly dietary and physical activity instructions for 6 months. Three-day dietary records and physical activity questionnaires were collected at 0, 3, and 6 months. Body composition was measured using bioelectrical impedance analysis. These relationships were analyzed using uni- and multivariate linear regression analyses.</p><p><strong>Results: </strong>A total of 155 children with obesity completed the 6-month study. A higher total protein intake per ideal weight for height was significantly correlated with a decrease in BMI z-score and trunk fat mass index (trunk fat mass [FM] in kg/height in m2). Mean dietary fiber intake was negatively correlated with BMI z-score, FM, FM index (FMI, FM in kg/height in m2), and visceral fat area (VFA) at 6 months. Each 1 g/kg/day increase in protein intake and additional 1 g/day of dietary fiber intake led to a 0.191 kg/m2 (95% confidence interval [CI], -0.309 to -0.072) and 0.028 kg/m2 (-0.05 to -0.005) reduction in BMI z-score. Each 1 g/day increase in protein intake led to a reduction of 0.009 kg/m² in trunk FMI (-0.016 to -0.002) and 0.21 cm² in VFA (-0.418 to -0.002). The outcomes observed at 6 months were consistent with the overall 6-month findings, reinforcing the efficacy of the intervention.</p><p><strong>Conclusion: </strong>Our study showed beneficial effects of high dietary protein and fiber intakes on BMI z-scores and adiposity of children with obesity. Fine-tuning dietary interventions that emphasize appropriate protein and fiber intakes may be more practical for managing childhood obesity.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143123798","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}
引用次数: 0
COVID-19 vaccine hesitancy among parents of children with systemic lupus erythematosus.
IF 3.2
Clinical and Experimental Pediatrics Pub Date : 2025-02-03 DOI: 10.3345/cep.2024.01340
Karnchanit Sausukpaiboon, Nuanpan Penboon, Pornpimol Rianthavorn
{"title":"COVID-19 vaccine hesitancy among parents of children with systemic lupus erythematosus.","authors":"Karnchanit Sausukpaiboon, Nuanpan Penboon, Pornpimol Rianthavorn","doi":"10.3345/cep.2024.01340","DOIUrl":"https://doi.org/10.3345/cep.2024.01340","url":null,"abstract":"<p><strong>Background: </strong>Coronavirus disease 2019 (COVID-19) vaccination remains an essential strategy for reducing disease burden. Specific guidelines for vaccinating children with systemic lupus erythematosus (SLE) are currently unavailable, highlighting the gap in tailored recommendations for this population.</p><p><strong>Purpose: </strong>This study aimed to estimate parental intention to vaccinate children with SLE against COVID-19 and identify factors associated with this intention. It also explored parents' attitudes toward the vaccine.</p><p><strong>Methods: </strong>Seventy-four parents of patients aged 5-21 years who were diagnosed with SLE before 18 years of age were surveyed regarding their willingness to further vaccinate their children with SLE against COVID-19. The parents were categorized into vaccine acceptance (VA) and vaccine hesitancy (VH) groups and completed a validated six-item questionnaire designed to gauge their attitudes toward the vaccine. Vaccine Hesitancy Scale (VHS) scores were calculated with higher scores indicating increased VH. The adjusted odds ratios (aOR [95% confidence interval]) for VA-associated factors were determined using multivariate analysis.</p><p><strong>Results: </strong>Twenty-five parents (33.8%) were diagnosed with VH. Compared with the VH group, the VA group showed a higher frequency of previous COVID-19 vaccine uptake, completed immunization in children, and parental willingness to be vaccinated themselves. Children were older in the VA versus VH group. The mean total VHS score was significantly higher in the VH versus VA group. In a multivariate model of factors differing significantly between the VA and VH groups, parental willingness to vaccinate themselves (aOR, 5.0 [1.2-20.4]), patient age (aOR, 1.4 [1.1-1.9]), and VHS score on vaccine efficacy belief (aOR, 0.1 [0.0-0.5]) were significantly associated with VA.</p><p><strong>Conclusion: </strong>A significant proportion of parents were hesitant to vaccinate their children with SLE against COVID-19. These insights underscore the importance of developing targeted educational interventions to address specific parental concerns and improve vaccine uptake in children with SLE.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143123717","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}
引用次数: 0
Impact of Xmn1 polymorphism on hydroxyurea therapy in children with HbE-βnon-transfusion dependent thalassemia: a cohort study.
IF 3.2
Clinical and Experimental Pediatrics Pub Date : 2025-02-03 DOI: 10.3345/cep.2024.01284
Saheli Roy, Paramita Bhattacharya, Atanu Kumar Dutta, Mrinal Kanti Das
{"title":"Impact of Xmn1 polymorphism on hydroxyurea therapy in children with HbE-βnon-transfusion dependent thalassemia: a cohort study.","authors":"Saheli Roy, Paramita Bhattacharya, Atanu Kumar Dutta, Mrinal Kanti Das","doi":"10.3345/cep.2024.01284","DOIUrl":"10.3345/cep.2024.01284","url":null,"abstract":"<p><strong>Background: </strong>Fetal hemoglobin (HbF) inducers, among which hydroxyurea is the most extensively used, have shifted the paradigm toward the treatment of non-transfusion-dependent thalassemia (NTDT). Xmn1 polymorphism (rs7482144) is characterized by substitution (C>T) at -158 position of the γ-globin gene, which leads to CC, CT, or TT genotype. Recently, the role of the Xmn1 polymorphism as a modifier of hydroxyurea therapy has attracted immense research interest.</p><p><strong>Purpose: </strong>This study aimed to estimate the prevalence of the Xmn1 polymorphism and determine its impact on the efficacy of hydroxyurea therapy in children with NTDT in Eastern India.</p><p><strong>Methods: </strong>This observational ambispective cohort study involved the assessment of 50 patients with NTDT, of whom 28 qualified, who had been receiving hydroxyurea for less than a month. Relevant molecular analyses were performed, and data on the annual transfusion requirement (ATR), height, and HbF level before starting hydroxyurea treatment were derived from medical records. The same parameters were reassessed after six months of hydroxyurea therapy. Furthermore, patients were monitored for drug toxicity.</p><p><strong>Results: </strong>All patients included in this study exhibited HbE-β-thalassemia, thus implying it to be one of the commonest NTDT genotypes in Eastern India. The prevalence rates of CC and CT were 43% and 57%, respectively, and none of the patients harbored the TT genotype. Toxicity developed in 22% of patients; however, it was not significantly associated with the Xmn1 polymorphism. Significant decrease in ATR and increase in height were observed following hydroxyurea therapy in both groups. Nevertheless, the change was more marked in CT genotype (median ATR drop: 33%, increase in median height: 3.7%, pCT = 0.001) than in CC genotype (median ATR drop: 28%, increase in median height: 2.8%, pCC = 0.003).</p><p><strong>Conclusion: </strong>The T allele of the Xmn1 polymorphism had a favorable effect on the efficacy of hydroxyurea in patients with HbE-β-NTDT.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143123800","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}
引用次数: 0
Prevalence of childhood overweight and obesity in Malaysia: a systematic review and meta-analysis. 马来西亚儿童超重和肥胖症患病率:系统回顾和荟萃分析。
IF 3.2
Clinical and Experimental Pediatrics Pub Date : 2025-02-01 Epub Date: 2024-11-13 DOI: 10.3345/cep.2024.00899
Ker Yang Chua, Ker Yung Chua, Karuthan Chinna, Chooi Ling Lim, Maheeka Seneviwickrama
{"title":"Prevalence of childhood overweight and obesity in Malaysia: a systematic review and meta-analysis.","authors":"Ker Yang Chua, Ker Yung Chua, Karuthan Chinna, Chooi Ling Lim, Maheeka Seneviwickrama","doi":"10.3345/cep.2024.00899","DOIUrl":"10.3345/cep.2024.00899","url":null,"abstract":"<p><p>The incidence of childhood obesity is increasing worldwide. National surveys in Malaysia have shown similar trends. This review aimed to increase our understanding of the prevalence and associated factors of childhood overweight, obesity, and excess weight in Malaysia. A systematic review and meta-analysis were conducted of studies reporting the prevalence of overweight and obesity in Malaysian children aged <18 years. Systematic searches were conducted in October 2023 and repeated in March 2024 of the PubMed, Scopus, and Web of Science databases. The grey literature was also included. Study selection, data extraction, and quality assessments were conducted by paired independent researchers. A metaanalysis of prevalence and 95% confidence interval (CI) using a random-effects model and heterogeneity (I2) was calculated. Publication bias was investigated using Egger's test. This review included 33 studies (N=273,710) conducted between 1996 and 2022. Overall, the childhood overweight, obesity and excess weight prevalence was 13.9% (95% CI, 13.0%-14.8%), 11.8% (95% CI, 10.9%-12.8%), and 24.0% (95% CI, 22.3%-25.7%), respectively. Boys had a significantly higher proportion of obesity (12.5% [95% CI, 11.1%-14.1%] vs. 9.1% [95% CI, 8.1%-10.1%]) and excess weight (25.7% [95% CI, 23.5%-28.1%] vs. 20.7% [95% CI, 18.8%-22.8%]) than girls. Sarawak natives had a higher prevalence of obesity (17.7% [95% CI, 16.0%-19.5%]). Annually, the obesity prevalence increased by 0.3% (95% CI, 0.1%-0.6%; P=0.006) and excess weight prevalence increased by 0.6% (95% CI, 0.3%-1.0%; P=0.002). Heterogeneity was high (I2>90%), and publication bias was possible (P<0.001). Variability was not significantly affected by the critical appraisal score or sample size. In conclusion, the prevalence of childhood overweight, obesity, and excess weight in Malaysia doubled over 26 years. Future studies of the prevalence of childhood excess weight should follow a standardized reference for body mass index by age to ease interstudy comparisons.</p>","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"115-126"},"PeriodicalIF":3.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825116/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142629767","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}
引用次数: 0
Effect of metabolic syndrome on pulmonary dysfunction in children with asthma. 代谢综合征对哮喘儿童肺功能障碍的影响。
IF 3.2
Clinical and Experimental Pediatrics Pub Date : 2025-02-01 Epub Date: 2024-11-13 DOI: 10.3345/cep.2024.00360
Hyo-Bin Kim
{"title":"Effect of metabolic syndrome on pulmonary dysfunction in children with asthma.","authors":"Hyo-Bin Kim","doi":"10.3345/cep.2024.00360","DOIUrl":"10.3345/cep.2024.00360","url":null,"abstract":"","PeriodicalId":36018,"journal":{"name":"Clinical and Experimental Pediatrics","volume":" ","pages":"136-137"},"PeriodicalIF":3.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825108/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142629594","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}
引用次数: 0
Promising role of voxelotor in managing sickle cell disease in children: a narrative review. Voxelotor在治疗儿童镰状细胞病中的前景:综述。
IF 3.2
Clinical and Experimental Pediatrics Pub Date : 2025-02-01 Epub Date: 2024-11-13 DOI: 10.3345/cep.2024.00500
Amit Agrawal, Gaurav Jadon, Japna Singh, Dalwinder Janjua
{"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}
引用次数: 0
Clinical characteristics and associated factors of pediatric acute necrotizing encephalopathy: a retrospective study. 小儿急性坏死性脑病的临床特征和相关因素:一项回顾性研究。
IF 3.2
Clinical and Experimental Pediatrics Pub Date : 2025-02-01 Epub Date: 2024-11-11 DOI: 10.3345/cep.2024.00794
Huiling Zhang, Yilong Wang, Qianyun Ding, Xuekun Li, Sheng Ye
{"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}
引用次数: 0
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