Pub Date : 2024-12-14DOI: 10.1007/s00431-024-05905-9
Giovanni Taccetti, Vito Terlizzi, Silvia Campana, Daniela Dolce, Novella Ravenni, Cristina Fevola, Michela Francalanci, Valeria Galici, Anna Silvia Neri
Bacterial infections of the lower airways are the main cause of mortality and morbidity in cystic fibrosis. The most frequently isolated pathogens are S. aureus and P. aeruginosa; bacterial co-infections are frequently observed. The aim of this review is to provide, in the current context, the indications regarding the best antibiotic strategy to adopt in subjects affected by CF infected with the most common pathogens. We selected relevant publications (guidelines, systematic reviews and clinical studies published so far on these topics) and we analysed the sampling methods used and antibiotic strategies adopted. Oropharyngeal sampling methods are considered less sensitive for pathogen detection than sputum. In non-expectorating people, induced sputum is considered equivalent to two-lobe bronchoalveolar lavage, which is considered invasive. Antibiotic treatment against the main pathogens can consist in eradication treatment in the early stages of infection, chronic suppressive therapy and treatment of the pulmonary exacerbations. This scheme is valid for P. aeruginosa but remains to be demonstrated for the other pathogens. For S. aureus, no evidence-based therapeutic strategies on how to treat the different stages of bacterial infection have been established with certainty. With regard to the treatment of the other classic pathogens (B. cepacia complex, A. xylosoxidans and S. maltophilia), no evidence-based indications exist and decision is left to the clinician. The recent introduction of highly effective modulators on the CFTR protein, in addition to the favourable effects described in regulatory trials, has led to a reduction in bacterial isolations; the real effect of which in clinical practice has still to be assessed on the basis of scientific data. CONCLUSIONS: The reliability of culture examination depends on sampling methods, and expectorated sputum continues to be the best method as it is simple and non-invasive. P. aeruginosa is the pathogen for which antibiotic strategies for the various stages of infection appear best established, and the efficacy of early eradication treatment and chronic suppressive therapy have been underlined in clinical trials and systematic reviews. The recent introduction of modulators into clinical practice, despite their widely described efficacy, has not yet led to suggestions for changes in antibiotic strategies against the pathogens most frequently isolated.
{"title":"Antibiotic treatment of bacterial lung infections in cystic fibrosis.","authors":"Giovanni Taccetti, Vito Terlizzi, Silvia Campana, Daniela Dolce, Novella Ravenni, Cristina Fevola, Michela Francalanci, Valeria Galici, Anna Silvia Neri","doi":"10.1007/s00431-024-05905-9","DOIUrl":"10.1007/s00431-024-05905-9","url":null,"abstract":"<p><p>Bacterial infections of the lower airways are the main cause of mortality and morbidity in cystic fibrosis. The most frequently isolated pathogens are S. aureus and P. aeruginosa; bacterial co-infections are frequently observed. The aim of this review is to provide, in the current context, the indications regarding the best antibiotic strategy to adopt in subjects affected by CF infected with the most common pathogens. We selected relevant publications (guidelines, systematic reviews and clinical studies published so far on these topics) and we analysed the sampling methods used and antibiotic strategies adopted. Oropharyngeal sampling methods are considered less sensitive for pathogen detection than sputum. In non-expectorating people, induced sputum is considered equivalent to two-lobe bronchoalveolar lavage, which is considered invasive. Antibiotic treatment against the main pathogens can consist in eradication treatment in the early stages of infection, chronic suppressive therapy and treatment of the pulmonary exacerbations. This scheme is valid for P. aeruginosa but remains to be demonstrated for the other pathogens. For S. aureus, no evidence-based therapeutic strategies on how to treat the different stages of bacterial infection have been established with certainty. With regard to the treatment of the other classic pathogens (B. cepacia complex, A. xylosoxidans and S. maltophilia), no evidence-based indications exist and decision is left to the clinician. The recent introduction of highly effective modulators on the CFTR protein, in addition to the favourable effects described in regulatory trials, has led to a reduction in bacterial isolations; the real effect of which in clinical practice has still to be assessed on the basis of scientific data. CONCLUSIONS: The reliability of culture examination depends on sampling methods, and expectorated sputum continues to be the best method as it is simple and non-invasive. P. aeruginosa is the pathogen for which antibiotic strategies for the various stages of infection appear best established, and the efficacy of early eradication treatment and chronic suppressive therapy have been underlined in clinical trials and systematic reviews. The recent introduction of modulators into clinical practice, despite their widely described efficacy, has not yet led to suggestions for changes in antibiotic strategies against the pathogens most frequently isolated.</p>","PeriodicalId":11997,"journal":{"name":"European Journal of Pediatrics","volume":"184 1","pages":"82"},"PeriodicalIF":3.0,"publicationDate":"2024-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11645307/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142822013","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-12-14DOI: 10.1007/s00431-024-05846-3
Janna-Lina Kerth, Maurus Hagemeister, Anne C Bischops, Lisa Reinhart, Juergen Dukart, Bert Heinrichs, Simon B Eickhoff, Thomas Meissner
The integration of artificial intelligence (AI) and machine learning (ML) has shown potential for various applications in the medical field, particularly for diagnosing and managing chronic diseases among children and adolescents. This systematic review aims to comprehensively analyze and synthesize research on the use of AI for monitoring, guiding, and assisting pediatric patients with chronic diseases. Five major electronic databases were searched (Medline, Scopus, PsycINFO, ACM, Web of Science), along with manual searches of gray literature, personal archives, and reference lists of relevant papers. All original studies as well as conference abstracts and proceedings, focusing on AI applications for pediatric chronic disease care were included. Thirty-one studies met the inclusion criteria. We extracted AI method used, study design, population, intervention, and main results. Two researchers independently extracted data and resolved discrepancies through discussion. AI applications are diverse, encompassing, e.g., disease classification, outcome prediction, or decision support. AI generally performed well, though most models were tested on retrospective data. AI-based tools have shown promise in mental health analysis, e.g., by using speech sampling or social media data to predict therapy outcomes for various chronic conditions.
Conclusions: While AI holds potential in pediatric chronic disease care, most reviewed studies are small-scale research projects. Prospective clinical implementations are needed to validate its effectiveness in real-world scenarios. Ethical considerations, cultural influences, and stakeholder attitudes should be integrated into future research.
What is known: • Artificial Intelligence (AI) will play a more dominant role in medicine and healthcare in the future and many applications are already being developed.
What is new: • Our review provides an overview on how AI-driven systems might be able to support children and adolescents with chronic illnesses. • While many applications are being researched, few have been tested on real-world, prospective, clinical data.
人工智能(AI)与机器学习(ML)的结合已显示出在医疗领域的各种应用潜力,尤其是在儿童和青少年慢性疾病的诊断和管理方面。本系统综述旨在全面分析和综合有关使用人工智能监测、指导和辅助儿科慢性病患者的研究。研究人员检索了五大电子数据库(Medline、Scopus、PsycINFO、ACM、Web of Science),并人工检索了灰色文献、个人档案和相关论文的参考文献列表。所有以人工智能在儿科慢性病护理中的应用为主题的原创研究、会议摘要和论文集均被纳入其中。有 31 项研究符合纳入标准。我们提取了所使用的人工智能方法、研究设计、人群、干预措施和主要结果。两名研究人员独立提取数据,并通过讨论解决差异。人工智能的应用多种多样,包括疾病分类、结果预测或决策支持等。虽然大多数模型都是在回顾性数据上进行测试,但人工智能的表现普遍良好。基于人工智能的工具在心理健康分析方面大有可为,例如利用语音采样或社交媒体数据预测各种慢性疾病的治疗结果:结论:虽然人工智能在儿科慢性病护理方面具有潜力,但大多数综述研究都是小规模的研究项目。结论:虽然人工智能在儿科慢性病护理中具有潜力,但大多数综述研究都是小规模的研究项目,需要进行前瞻性的临床实施,以验证其在现实世界中的有效性。在未来的研究中应考虑伦理因素、文化影响和利益相关者的态度:- 人工智能(AI)未来将在医学和医疗保健领域发挥更重要的作用,许多应用已在开发之中:- 我们的综述概述了人工智能驱动的系统如何为患有慢性疾病的儿童和青少年提供支持。- 虽然许多应用正在研究中,但很少有应用在真实世界、前瞻性临床数据上进行过测试。
{"title":"Artificial intelligence in the care of children and adolescents with chronic diseases: a systematic review.","authors":"Janna-Lina Kerth, Maurus Hagemeister, Anne C Bischops, Lisa Reinhart, Juergen Dukart, Bert Heinrichs, Simon B Eickhoff, Thomas Meissner","doi":"10.1007/s00431-024-05846-3","DOIUrl":"10.1007/s00431-024-05846-3","url":null,"abstract":"<p><p>The integration of artificial intelligence (AI) and machine learning (ML) has shown potential for various applications in the medical field, particularly for diagnosing and managing chronic diseases among children and adolescents. This systematic review aims to comprehensively analyze and synthesize research on the use of AI for monitoring, guiding, and assisting pediatric patients with chronic diseases. Five major electronic databases were searched (Medline, Scopus, PsycINFO, ACM, Web of Science), along with manual searches of gray literature, personal archives, and reference lists of relevant papers. All original studies as well as conference abstracts and proceedings, focusing on AI applications for pediatric chronic disease care were included. Thirty-one studies met the inclusion criteria. We extracted AI method used, study design, population, intervention, and main results. Two researchers independently extracted data and resolved discrepancies through discussion. AI applications are diverse, encompassing, e.g., disease classification, outcome prediction, or decision support. AI generally performed well, though most models were tested on retrospective data. AI-based tools have shown promise in mental health analysis, e.g., by using speech sampling or social media data to predict therapy outcomes for various chronic conditions.</p><p><strong>Conclusions: </strong>While AI holds potential in pediatric chronic disease care, most reviewed studies are small-scale research projects. Prospective clinical implementations are needed to validate its effectiveness in real-world scenarios. Ethical considerations, cultural influences, and stakeholder attitudes should be integrated into future research.</p><p><strong>What is known: </strong>• Artificial Intelligence (AI) will play a more dominant role in medicine and healthcare in the future and many applications are already being developed.</p><p><strong>What is new: </strong>• Our review provides an overview on how AI-driven systems might be able to support children and adolescents with chronic illnesses. • While many applications are being researched, few have been tested on real-world, prospective, clinical data.</p>","PeriodicalId":11997,"journal":{"name":"European Journal of Pediatrics","volume":"184 1","pages":"83"},"PeriodicalIF":3.0,"publicationDate":"2024-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11645428/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142822014","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-12-13DOI: 10.1007/s00431-024-05917-5
Helen Michaela de Oliveira, Mariano Gallo Ruelas, Lucas Mendes Barbosa, Camilo André Viana Diaz, Gustavo Roberto Minetto Wegner, Bruno Francisco Minetto Wegner, André Vieira da Cruz
Neonatal hyperbilirubinemia is a prevalent condition, with a risk of serious complications. Phototherapy is the standard treatment for significant cases, but its limitations highlight the need for additional options. Zinc sulfate has emerged as a potential adjunctive treatment. Our objective is to evaluate the efficacy of zinc sulfate as an adjunct to phototherapy in neonates with hyperbilirubinemia. PubMed, Embase, and CENTRAL were searched for studies published up to September 2024. Eligible studies were randomized clinical trials (RCTs) enrolling neonates with hyperbilirubinemia that evaluated the combined use of phototherapy and zinc sulfate. This study followed PRISMA guidelines, with independent extraction of data by two reviewers. Risk of bias was assessed using the RoB2 tool, and the quality of evidence was evaluated using the GRADE approach. Eleven RCTs comprising 1,349 neonates were included. A total of 690 (51.1%) neonates received zinc sulfate. Zinc sulfate significantly reduced bilirubin levels at 24 h (MD = -0.76 mg/dL; 95% CI = -1.30 to -0.22; P < .01; I2 = 82%), 48 h (MD = -0.88 mg/dL; 95% CI = -1.60 to -0.17; P = 0.02; I2 = 76%) and at 72 h (MD = -1.19 mg/dL; 95% CI = -2.29 to -0.09; P = .003; I2 = 94%). Subgroup analysis indicated that term neonates with normal birth weight benefited most from the intervention, while preterm and low-birth-weight infants showed no significant difference.Conclusion: Zinc sulfate effectively reduces serum bilirubin levels in neonates with hyperbilirubinemia when used alongside phototherapy, especially in term neonates.Trial registration number and date of registration: PROSPERO, CRD42024586259, 09/13/2024. The Impact of Zinc Sulfate on the Treatment of Neonatal Hyperbilirubinemia: An Updated Systematic Review and Meta-Analysis"; CRD42024586259; Link: https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=586259.
{"title":"Zinc sulfate on neonatal hyperbilirubinemia: an updated systematic review and meta-analysis.","authors":"Helen Michaela de Oliveira, Mariano Gallo Ruelas, Lucas Mendes Barbosa, Camilo André Viana Diaz, Gustavo Roberto Minetto Wegner, Bruno Francisco Minetto Wegner, André Vieira da Cruz","doi":"10.1007/s00431-024-05917-5","DOIUrl":"10.1007/s00431-024-05917-5","url":null,"abstract":"<p><p>Neonatal hyperbilirubinemia is a prevalent condition, with a risk of serious complications. Phototherapy is the standard treatment for significant cases, but its limitations highlight the need for additional options. Zinc sulfate has emerged as a potential adjunctive treatment. Our objective is to evaluate the efficacy of zinc sulfate as an adjunct to phototherapy in neonates with hyperbilirubinemia. PubMed, Embase, and CENTRAL were searched for studies published up to September 2024. Eligible studies were randomized clinical trials (RCTs) enrolling neonates with hyperbilirubinemia that evaluated the combined use of phototherapy and zinc sulfate. This study followed PRISMA guidelines, with independent extraction of data by two reviewers. Risk of bias was assessed using the RoB2 tool, and the quality of evidence was evaluated using the GRADE approach. Eleven RCTs comprising 1,349 neonates were included. A total of 690 (51.1%) neonates received zinc sulfate. Zinc sulfate significantly reduced bilirubin levels at 24 h (MD = -0.76 mg/dL; 95% CI = -1.30 to -0.22; P < .01; I<sup>2</sup> = 82%), 48 h (MD = -0.88 mg/dL; 95% CI = -1.60 to -0.17; P = 0.02; I<sup>2</sup> = 76%) and at 72 h (MD = -1.19 mg/dL; 95% CI = -2.29 to -0.09; P = .003; I<sup>2</sup> = 94%). Subgroup analysis indicated that term neonates with normal birth weight benefited most from the intervention, while preterm and low-birth-weight infants showed no significant difference.Conclusion: Zinc sulfate effectively reduces serum bilirubin levels in neonates with hyperbilirubinemia when used alongside phototherapy, especially in term neonates.Trial registration number and date of registration: PROSPERO, CRD42024586259, 09/13/2024. The Impact of Zinc Sulfate on the Treatment of Neonatal Hyperbilirubinemia: An Updated Systematic Review and Meta-Analysis\"; CRD42024586259; Link: https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=586259.</p>","PeriodicalId":11997,"journal":{"name":"European Journal of Pediatrics","volume":"184 1","pages":"79"},"PeriodicalIF":3.0,"publicationDate":"2024-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142817476","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-12-13DOI: 10.1007/s00431-024-05913-9
Winnie Wan Yee Tso, Yuliang Wang, Daniel Yee Tak Fong, Mike Yat Wah Kwan, Patrick Ip, Jasper Fuk Woo Chan, Lok Kan Leung, Jason Ying Kuen Chan, Sabrina Siu Ling Tsao, Christy Shuk Kuen Chau, Ka Man Yip, Ka Yi Hui, Jaime Sou Rosa Duque, Yu Lung Lau, Tatia Mei Chun Lee
This study aims to develop and validate the Post-COVID Symptom Scale for Children/Youth (PCSS-C/Y), which is a comprehensive tool for measuring the symptom burden of post-COVID-19 conditions-persistent symptoms after SARS-CoV-2 infection, commonly known as Long COVID-and its impact on health-related quality of life among children and adolescents. Parents of children and adolescents, adolescents, and young adults with and without a history of COVID-19 were invited to fill in a questionnaire from October 2022 to June 2023. There were 386 valid parent proxy-reported responses, 433 valid adolescent self-reported responses, and 324 valid young adult self-reported responses included in the final analysis. The PCSS-C/Y demonstrated stable factor structure and good internal consistency in different sampling groups. The scale score was negatively associated with Paediatric Quality of Life Inventory (PedsQL) scores (young adult self-report, adjusted R2 = 0.394; adolescent self-report, adjusted R2 = 0.219; parent-report, adjusted R2 = 0.292), while it was positively associated with Strengths and Difficulties Questionnaire (SDQ) scores (young adult self-report, adjusted R2 = 0.195; adolescent self-report, adjusted R2 = 0.154; parent-report, adjusted R2 = 0.239). The scale can also discriminate the post-infected cases and control cases, Cohen's d = 0.41, 0.50, and 0.38 for adult self-report, adolescent self-report, and parent-report, respectively. Conclusions: The PCSS-C/Y is a valid and reliable tool for quantifying the diverse symptomatology of post-COVID-19 conditions in children and adolescents. It provides quantifiable measurements that enable clinicians to monitor post-COVID-19 symptoms in children and young people and facilitates the development of interventions for post-COVID-19 conditions.
{"title":"Development and validation of the Post-COVID Symptom Scale for Children/Youth (PCSS-C/Y).","authors":"Winnie Wan Yee Tso, Yuliang Wang, Daniel Yee Tak Fong, Mike Yat Wah Kwan, Patrick Ip, Jasper Fuk Woo Chan, Lok Kan Leung, Jason Ying Kuen Chan, Sabrina Siu Ling Tsao, Christy Shuk Kuen Chau, Ka Man Yip, Ka Yi Hui, Jaime Sou Rosa Duque, Yu Lung Lau, Tatia Mei Chun Lee","doi":"10.1007/s00431-024-05913-9","DOIUrl":"10.1007/s00431-024-05913-9","url":null,"abstract":"<p><p>This study aims to develop and validate the Post-COVID Symptom Scale for Children/Youth (PCSS-C/Y), which is a comprehensive tool for measuring the symptom burden of post-COVID-19 conditions-persistent symptoms after SARS-CoV-2 infection, commonly known as Long COVID-and its impact on health-related quality of life among children and adolescents. Parents of children and adolescents, adolescents, and young adults with and without a history of COVID-19 were invited to fill in a questionnaire from October 2022 to June 2023. There were 386 valid parent proxy-reported responses, 433 valid adolescent self-reported responses, and 324 valid young adult self-reported responses included in the final analysis. The PCSS-C/Y demonstrated stable factor structure and good internal consistency in different sampling groups. The scale score was negatively associated with Paediatric Quality of Life Inventory (PedsQL) scores (young adult self-report, adjusted R<sup>2</sup> = 0.394; adolescent self-report, adjusted R<sup>2</sup> = 0.219; parent-report, adjusted R<sup>2</sup> = 0.292), while it was positively associated with Strengths and Difficulties Questionnaire (SDQ) scores (young adult self-report, adjusted R<sup>2</sup> = 0.195; adolescent self-report, adjusted R<sup>2</sup> = 0.154; parent-report, adjusted R<sup>2</sup> = 0.239). The scale can also discriminate the post-infected cases and control cases, Cohen's d = 0.41, 0.50, and 0.38 for adult self-report, adolescent self-report, and parent-report, respectively. Conclusions: The PCSS-C/Y is a valid and reliable tool for quantifying the diverse symptomatology of post-COVID-19 conditions in children and adolescents. It provides quantifiable measurements that enable clinicians to monitor post-COVID-19 symptoms in children and young people and facilitates the development of interventions for post-COVID-19 conditions.</p>","PeriodicalId":11997,"journal":{"name":"European Journal of Pediatrics","volume":"184 1","pages":"81"},"PeriodicalIF":3.0,"publicationDate":"2024-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11645425/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142822015","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-12-13DOI: 10.1007/s00431-024-05916-6
Carlos Delgado-Miguel, Ada García Morán, Lara Fuentes Gómez, Mercedes Díaz, Miriam Miguel-Ferrero, Juan Carlos López-Gutiérrez
Early debridement of partial-thickness burns and coverage with skin substitutes is currently the standard of care in children, although there is currently no "gold standard" skin substitute. Our aim is to compare the effectiveness of three different skin substitutes, analyzing the medium- and long-term outcomes.
Methods: A retrospective study was conducted on burn patients under 18 years admitted to our Burn Unit between 2015 and 2021, who were divided into 3 groups according to the type of skin substitute used (EZ-derm®, Biobrane®, and Suprathel®). Demographic and clinical data and short- and long-term outcomes were analyzed. Effectiveness was analyzed by escharectomy and grafting rate during acute management and long-term follow-up reintervention rate. A total of 378 patients were included (179 EZ-derm® group, 107 Biobrane® group, and 92 Suprathel® group). No differences in demographics or burn characteristics were observed between the groups. Patients treated with Suprathel® had a significantly shorter hospital stay (median 4 days (IQR 2-9)), a lower rate of escharectomy and grafting during acute management (21.1%), and a lower long-term follow-up reintervention rate (18.5%) when compared to the EZ-derm® group (median stay 9 days (IQR 6-13); escharectomy and graft 24.6% and reintervention 26.8%) and to the Biobrane® group (median stay 9 days (IQR 7-14); escharectomy and graft 32.1% and reintervention 26.2%).
Conclusion: Treatment of partial-thickness burns with Suprathel® is associated with a shorter hospital stay, lower need for escharectomy and grafting, and lower need for long-term reintervention. Therefore, it should be considered the treatment of choice for pediatric partial-thickness burns.
What is known: • Different types of skin substitutes are available for the treatment of skin burns in paediatric patients.
What is new: • Suprathel® is linked to a reduction in hospital stays, a lower need for escharectomy and grafting, and a lower likelihood of requiring long-term re-interventions.
{"title":"Comparison of the effectiveness of three different skin substitutes for the treatment of pediatric burns.","authors":"Carlos Delgado-Miguel, Ada García Morán, Lara Fuentes Gómez, Mercedes Díaz, Miriam Miguel-Ferrero, Juan Carlos López-Gutiérrez","doi":"10.1007/s00431-024-05916-6","DOIUrl":"https://doi.org/10.1007/s00431-024-05916-6","url":null,"abstract":"<p><p>Early debridement of partial-thickness burns and coverage with skin substitutes is currently the standard of care in children, although there is currently no \"gold standard\" skin substitute. Our aim is to compare the effectiveness of three different skin substitutes, analyzing the medium- and long-term outcomes.</p><p><strong>Methods: </strong>A retrospective study was conducted on burn patients under 18 years admitted to our Burn Unit between 2015 and 2021, who were divided into 3 groups according to the type of skin substitute used (EZ-derm®, Biobrane®, and Suprathel®). Demographic and clinical data and short- and long-term outcomes were analyzed. Effectiveness was analyzed by escharectomy and grafting rate during acute management and long-term follow-up reintervention rate. A total of 378 patients were included (179 EZ-derm® group, 107 Biobrane® group, and 92 Suprathel® group). No differences in demographics or burn characteristics were observed between the groups. Patients treated with Suprathel® had a significantly shorter hospital stay (median 4 days (IQR 2-9)), a lower rate of escharectomy and grafting during acute management (21.1%), and a lower long-term follow-up reintervention rate (18.5%) when compared to the EZ-derm® group (median stay 9 days (IQR 6-13); escharectomy and graft 24.6% and reintervention 26.8%) and to the Biobrane® group (median stay 9 days (IQR 7-14); escharectomy and graft 32.1% and reintervention 26.2%).</p><p><strong>Conclusion: </strong>Treatment of partial-thickness burns with Suprathel® is associated with a shorter hospital stay, lower need for escharectomy and grafting, and lower need for long-term reintervention. Therefore, it should be considered the treatment of choice for pediatric partial-thickness burns.</p><p><strong>What is known: </strong>• Different types of skin substitutes are available for the treatment of skin burns in paediatric patients.</p><p><strong>What is new: </strong>• Suprathel® is linked to a reduction in hospital stays, a lower need for escharectomy and grafting, and a lower likelihood of requiring long-term re-interventions.</p>","PeriodicalId":11997,"journal":{"name":"European Journal of Pediatrics","volume":"184 1","pages":"80"},"PeriodicalIF":3.0,"publicationDate":"2024-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142817474","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-12-12DOI: 10.1007/s00431-024-05862-3
Enrico Pistritto, Federica M F Schera, Emilia Vassilopoulou, Antonio Corsello, Ilaria Alberti, Sebastiano A G Lava, Céline Betti, Mario G Bianchetti, Carlo Agostoni, Pietro Camozzi, Gregorio P Milani
Adolescent binge drinking is increasingly common. This study investigates the anomalies in glucose, sodium, calcium, potassium, and acid-base homeostasis induced by binge drinking in adolescents. The records of teenagers who sought medical attention for binge drinking (ethanol level ≥ 0.80 g/L) at the Pediatric Emergency Department, Ca' Granda Ospedale Maggiore Policlinico, Milan (Italy), spanning the years 2013 to 2023 were retrospectively analyzed. For this analysis, cases were selected if documented blood chemistry encompassed sodium, potassium, total calcium, glucose, acid-base balance, and lactic acid (only for those with metabolic acidosis). Included were 173 adolescents (female-to-male ratio 0.94), 13.2 to 18.4, median 16.4 years of age. Hypoglycemia (≤ 3.3 mmol/L; N = 1, 0.6%), hyponatremia (≤ 134 mmol/L; N = 7, 4.0%), hypernatremia (≥ 146 mmol/L; N = 3, 1.7%), hypocalcemia (≤ 2.19 mmol/L; N = 0) hypercalcemia (≥ 2.61 mmol/L; N = 0), and hyperkalemia (≥ 5.1 mmol/L; N = 0) were infrequent. Acute respiratory acidosis (pCO2 ≥ 46 mm Hg; pH < 7.40; N = 101, 58%) was the most common acid-base imbalance, followed by respiratory alkalosis (pCO2 ≤ 34 mm Hg; pH > 7.40; N = 10, 5.6%), and metabolic acidosis (HCO3- ≤ 19 mmol/L, pH < 7.40; N = 9, 5.2%). The lactic acid level was increased (≥ 2.1 mmol/L) in all cases with metabolic acidosis. Metabolic alkalosis (HCO3- ≥ 28 mmol/L, pH > 7.40) never occurred. Hypokalemia (≤ 3.4 mmol/L; N = 56, 32%) was prevalent, particularly in adolescents with normal acid-base equilibrium or metabolic acidosis, rather than respiratory acidosis or alkalosis.Conclusion: Adolescents who engage in binge drinking often experience a disrupted acid-base balance and hypokalemia, while glucose, sodium and calcium levels are rarely affected. What is known? • Binge drinking is becoming increasingly common among adolescents. • Conflicting data regarding the type and prevalence of biochemical disorders induced by binge drinking are available in this age group. What is new? • Acute respiratory acidosis is prevalent in adolescents with binge drinking, whereas respiratory alkalosis, metabolic acidosis, and hypoglycemia are uncommon. • Hypokalemia develops frequently.
{"title":"Impact of adolescents' binge drinking on blood chemistry.","authors":"Enrico Pistritto, Federica M F Schera, Emilia Vassilopoulou, Antonio Corsello, Ilaria Alberti, Sebastiano A G Lava, Céline Betti, Mario G Bianchetti, Carlo Agostoni, Pietro Camozzi, Gregorio P Milani","doi":"10.1007/s00431-024-05862-3","DOIUrl":"10.1007/s00431-024-05862-3","url":null,"abstract":"<p><p>Adolescent binge drinking is increasingly common. This study investigates the anomalies in glucose, sodium, calcium, potassium, and acid-base homeostasis induced by binge drinking in adolescents. The records of teenagers who sought medical attention for binge drinking (ethanol level ≥ 0.80 g/L) at the Pediatric Emergency Department, Ca' Granda Ospedale Maggiore Policlinico, Milan (Italy), spanning the years 2013 to 2023 were retrospectively analyzed. For this analysis, cases were selected if documented blood chemistry encompassed sodium, potassium, total calcium, glucose, acid-base balance, and lactic acid (only for those with metabolic acidosis). Included were 173 adolescents (female-to-male ratio 0.94), 13.2 to 18.4, median 16.4 years of age. Hypoglycemia (≤ 3.3 mmol/L; N = 1, 0.6%), hyponatremia (≤ 134 mmol/L; N = 7, 4.0%), hypernatremia (≥ 146 mmol/L; N = 3, 1.7%), hypocalcemia (≤ 2.19 mmol/L; N = 0) hypercalcemia (≥ 2.61 mmol/L; N = 0), and hyperkalemia (≥ 5.1 mmol/L; N = 0) were infrequent. Acute respiratory acidosis (pCO<sub>2</sub> ≥ 46 mm Hg; pH < 7.40; N = 101, 58%) was the most common acid-base imbalance, followed by respiratory alkalosis (pCO<sub>2</sub> ≤ 34 mm Hg; pH > 7.40; N = 10, 5.6%), and metabolic acidosis (HCO<sub>3</sub><sup>-</sup> ≤ 19 mmol/L, pH < 7.40; N = 9, 5.2%). The lactic acid level was increased (≥ 2.1 mmol/L) in all cases with metabolic acidosis. Metabolic alkalosis (HCO<sub>3</sub><sup>-</sup> ≥ 28 mmol/L, pH > 7.40) never occurred. Hypokalemia (≤ 3.4 mmol/L; N = 56, 32%) was prevalent, particularly in adolescents with normal acid-base equilibrium or metabolic acidosis, rather than respiratory acidosis or alkalosis.Conclusion: Adolescents who engage in binge drinking often experience a disrupted acid-base balance and hypokalemia, while glucose, sodium and calcium levels are rarely affected. What is known? • Binge drinking is becoming increasingly common among adolescents. • Conflicting data regarding the type and prevalence of biochemical disorders induced by binge drinking are available in this age group. What is new? • Acute respiratory acidosis is prevalent in adolescents with binge drinking, whereas respiratory alkalosis, metabolic acidosis, and hypoglycemia are uncommon. • Hypokalemia develops frequently.</p>","PeriodicalId":11997,"journal":{"name":"European Journal of Pediatrics","volume":"184 1","pages":"77"},"PeriodicalIF":3.0,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11638386/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142812450","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-12-12DOI: 10.1007/s00431-024-05920-w
Andrea de Oliveira Campos Amaral, Armanda de Oliveira Pache de Faria, Fabiana Rabe Carvalho, Luis Antonio Bataglin Dalcastel, Simone Saraiva de Abreu Almeida, Alexandre Ribeiro Fernandes, Luis Guillermo Coca Velarde, Solange Artimos de Oliveira, Claudete Aparecida Araújo Cardoso, Maria Elisa Vieira da Cunha Ramos Miterhof, Renata Artimos de Oliveira Vianna
This study aimed to evaluate the association between microcephaly and hearing disorders in children with exposed or suspected exposure to Zika virus (ZIKV) during the intrauterine period. In this cross-sectional study, we enrolled children exposed or suspected of being exposed to ZIKV during intrauterine period, admitted to the hospital between April 2016 and July 2018, and followed up until September 2021. All children underwent at least one automated auditory brainstem response (AABR) test. For analysis, the patients were divided into four groups: those with microcephaly, without microcephaly, suspected ZIKV infection, and controls. Other causes of microcephaly were excluded. Hearing impairment was assessed using the AABR to determine associations with microcephaly or central nervous system (CNS) abnormalities. Of the 134 children included, 34 (25.4%) were diagnosed with congenital Zika syndrome (CZS), of whom 28 (82.4%) had microcephaly, and the remaining six (17.6%) without microcephaly. Among the 28 children with microcephaly, 3 (10.7%) had abnormal AABR. Among CZS children without microcephaly (n = 6), 1 (16.7%) had abnormal AABR (Fisher's exact test p = 0.56).Conclusion: In our study population, that hearing impairment assessed using the AABR test was not associated with microcephaly or severe CNS alterations.
{"title":"Association between microcephaly and hearing disorders in children exposed or suspected of exposure to the Zika virus during the intrauterine period.","authors":"Andrea de Oliveira Campos Amaral, Armanda de Oliveira Pache de Faria, Fabiana Rabe Carvalho, Luis Antonio Bataglin Dalcastel, Simone Saraiva de Abreu Almeida, Alexandre Ribeiro Fernandes, Luis Guillermo Coca Velarde, Solange Artimos de Oliveira, Claudete Aparecida Araújo Cardoso, Maria Elisa Vieira da Cunha Ramos Miterhof, Renata Artimos de Oliveira Vianna","doi":"10.1007/s00431-024-05920-w","DOIUrl":"10.1007/s00431-024-05920-w","url":null,"abstract":"<p><p>This study aimed to evaluate the association between microcephaly and hearing disorders in children with exposed or suspected exposure to Zika virus (ZIKV) during the intrauterine period. In this cross-sectional study, we enrolled children exposed or suspected of being exposed to ZIKV during intrauterine period, admitted to the hospital between April 2016 and July 2018, and followed up until September 2021. All children underwent at least one automated auditory brainstem response (AABR) test. For analysis, the patients were divided into four groups: those with microcephaly, without microcephaly, suspected ZIKV infection, and controls. Other causes of microcephaly were excluded. Hearing impairment was assessed using the AABR to determine associations with microcephaly or central nervous system (CNS) abnormalities. Of the 134 children included, 34 (25.4%) were diagnosed with congenital Zika syndrome (CZS), of whom 28 (82.4%) had microcephaly, and the remaining six (17.6%) without microcephaly. Among the 28 children with microcephaly, 3 (10.7%) had abnormal AABR. Among CZS children without microcephaly (n = 6), 1 (16.7%) had abnormal AABR (Fisher's exact test p = 0.56).Conclusion: In our study population, that hearing impairment assessed using the AABR test was not associated with microcephaly or severe CNS alterations.</p>","PeriodicalId":11997,"journal":{"name":"European Journal of Pediatrics","volume":"184 1","pages":"78"},"PeriodicalIF":3.0,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142812444","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-12-12DOI: 10.1007/s00431-024-05888-7
Karin Blomberg, Małgorzata Farnik, Mats Eriksson
The aim of this study was to translate, adapt, and psychometrically evaluate the Quality of Life in a Child's Chronic Disease Questionnaire (QLCCDQ) for the Swedish context. The QLCCDQ was translated into Swedish and adapted to the Swedish context. Data for psychometric testing were obtained through a survey of parents of children and adolescents (n = 627) with chronic diseases: asthma and type 1 diabetes mellitus, with a total of 173 responses (27.6%). Face and content validity of the instrument was assessed, and floor and ceiling effects were measured. Internal consistency was measured with Cronbach's alpha and an exploratory factor analysis (EFA) was conducted. The EFA gave a two-factor solution with an eigenvalue > 1 explaining 73.9% of total variance for the Swedish version. The new subscales are family life and activities (eight questions) and emotions and symptoms (four questions). Three questions concerning anxiety, worry, and guilt loaded < 0.6 and were excluded.
Conclusion: The study concludes that the Swedish version of the QLCCDQ is a reliable and valid questionnaire. The instrument may be useful for clinical screening of families who have the greatest need for supportive interventions. However, this should be further evaluated.
What is known: • A child's chronic disease influences quality of life of its family members. • Few instruments are designed to measure the impact on families.
What is new: • The Swedish version of the Quality of Life in a Child's Chronic Disease Questionnaire has two subscales compared to the original's five. • The instrument may potentially be useful for clinical screening of families who have the greatest need for supportive interventions.
{"title":"Translation, adaptation, and psychometric evaluation of the Quality of Life in a Child's Chronic Disease Questionnaire for the Swedish context.","authors":"Karin Blomberg, Małgorzata Farnik, Mats Eriksson","doi":"10.1007/s00431-024-05888-7","DOIUrl":"10.1007/s00431-024-05888-7","url":null,"abstract":"<p><p>The aim of this study was to translate, adapt, and psychometrically evaluate the Quality of Life in a Child's Chronic Disease Questionnaire (QLCCDQ) for the Swedish context. The QLCCDQ was translated into Swedish and adapted to the Swedish context. Data for psychometric testing were obtained through a survey of parents of children and adolescents (n = 627) with chronic diseases: asthma and type 1 diabetes mellitus, with a total of 173 responses (27.6%). Face and content validity of the instrument was assessed, and floor and ceiling effects were measured. Internal consistency was measured with Cronbach's alpha and an exploratory factor analysis (EFA) was conducted. The EFA gave a two-factor solution with an eigenvalue > 1 explaining 73.9% of total variance for the Swedish version. The new subscales are family life and activities (eight questions) and emotions and symptoms (four questions). Three questions concerning anxiety, worry, and guilt loaded < 0.6 and were excluded.</p><p><strong>Conclusion: </strong>The study concludes that the Swedish version of the QLCCDQ is a reliable and valid questionnaire. The instrument may be useful for clinical screening of families who have the greatest need for supportive interventions. However, this should be further evaluated.</p><p><strong>What is known: </strong>• A child's chronic disease influences quality of life of its family members. • Few instruments are designed to measure the impact on families.</p><p><strong>What is new: </strong>• The Swedish version of the Quality of Life in a Child's Chronic Disease Questionnaire has two subscales compared to the original's five. • The instrument may potentially be useful for clinical screening of families who have the greatest need for supportive interventions.</p>","PeriodicalId":11997,"journal":{"name":"European Journal of Pediatrics","volume":"184 1","pages":"76"},"PeriodicalIF":3.0,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11634934/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142812511","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-12-12DOI: 10.1007/s00431-024-05911-x
Jakob Thestrup, Jette Led Sørensen, Barbara Hoff Esbjørn, Jane Hybschmann, Thomas Leth Frandsen, Patricia DeCosta, Line Klingen Gjærde
Communication skills are a vital but often neglected part of paediatric training. To make communication training more responsive to patient needs, children and adolescents should be involved in developing the communication curriculum for healthcare professionals, though this is rarely the case. The present study explored children and adolescents' perceptions of healthcare professionals to identify recommendations for healthcare professionals to contribute to formulating goals, learning objectives, and competencies for an interprofessional paediatric communication curriculum. We used narrative and play-based interviews to include the perceptions of preschool children aged 3-6 years (n = 8) and an online questionnaire to explore those of schoolchildren and adolescents aged 5-18 years (n = 54). We did a thematic analysis of the qualitative interview data and open-ended questionnaire responses, which showed that preschool children found familiar approaches, physical contact, and their parents comforting and that healthcare professionals should use playful methods, child-friendly words, and tangible rewards. Schoolchildren and adolescents preferred healthcare professionals who were friendly, patient, attentive, communicated clearly, and engaged them in conversation. They did not like when healthcare professionals appeared stressed, did not keep their promises, or forced them to do something.
Conclusions: We condensed perceptions of children and adolescents into tips and statements to be used in further development of a communication curriculum for paediatric healthcare professionals. Our findings emphasize that paediatric communication training should focus on healthcare professionals' attitude and appearance, strategies for building trust and engaging patients in treatment and care, the use of age-appropriate communication, and understanding the cognitive development of children and adolescents.
What is known: • Communication is a core competence that all paediatric healthcare professionals must practice and maintain. • Children and adolescents can contribute to curriculum development, but only few studies have done so.
What is new: • The perspectives of children and adolescents indicate that education programmes on paediatric communication should focus on the attitude and appearance of healthcare professionals, strategies to build trust and engage patients, age-appropriate communication, and understanding the cognitive development of children and adolescents. • Children and adolescents aged 3-18 years can contribute to the development of goals, learning objectives, and competencies for paediatric communication training, which may help promote more patient-centred approaches in practice.
{"title":"Paediatric patient perceptions of healthcare professionals: contributions to a communication curriculum.","authors":"Jakob Thestrup, Jette Led Sørensen, Barbara Hoff Esbjørn, Jane Hybschmann, Thomas Leth Frandsen, Patricia DeCosta, Line Klingen Gjærde","doi":"10.1007/s00431-024-05911-x","DOIUrl":"10.1007/s00431-024-05911-x","url":null,"abstract":"<p><p>Communication skills are a vital but often neglected part of paediatric training. To make communication training more responsive to patient needs, children and adolescents should be involved in developing the communication curriculum for healthcare professionals, though this is rarely the case. The present study explored children and adolescents' perceptions of healthcare professionals to identify recommendations for healthcare professionals to contribute to formulating goals, learning objectives, and competencies for an interprofessional paediatric communication curriculum. We used narrative and play-based interviews to include the perceptions of preschool children aged 3-6 years (n = 8) and an online questionnaire to explore those of schoolchildren and adolescents aged 5-18 years (n = 54). We did a thematic analysis of the qualitative interview data and open-ended questionnaire responses, which showed that preschool children found familiar approaches, physical contact, and their parents comforting and that healthcare professionals should use playful methods, child-friendly words, and tangible rewards. Schoolchildren and adolescents preferred healthcare professionals who were friendly, patient, attentive, communicated clearly, and engaged them in conversation. They did not like when healthcare professionals appeared stressed, did not keep their promises, or forced them to do something.</p><p><strong>Conclusions: </strong>We condensed perceptions of children and adolescents into tips and statements to be used in further development of a communication curriculum for paediatric healthcare professionals. Our findings emphasize that paediatric communication training should focus on healthcare professionals' attitude and appearance, strategies for building trust and engaging patients in treatment and care, the use of age-appropriate communication, and understanding the cognitive development of children and adolescents.</p><p><strong>What is known: </strong>• Communication is a core competence that all paediatric healthcare professionals must practice and maintain. • Children and adolescents can contribute to curriculum development, but only few studies have done so.</p><p><strong>What is new: </strong>• The perspectives of children and adolescents indicate that education programmes on paediatric communication should focus on the attitude and appearance of healthcare professionals, strategies to build trust and engage patients, age-appropriate communication, and understanding the cognitive development of children and adolescents. • Children and adolescents aged 3-18 years can contribute to the development of goals, learning objectives, and competencies for paediatric communication training, which may help promote more patient-centred approaches in practice.</p>","PeriodicalId":11997,"journal":{"name":"European Journal of Pediatrics","volume":"184 1","pages":"75"},"PeriodicalIF":3.0,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11634935/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142812466","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-12-10DOI: 10.1007/s00431-024-05897-6
Ting Zhao, Hong-Jian Li, Hui-Lan Zhang, Jing Yu, Jie Feng, Long Cui, Ke-Fang Sun, Yan Sun, Lu-Hai Yu
To evaluate the effects of cytochrome P450 family 2 subfamily C member 9 (CYP2C9) and cytochrome P450 family 2 subfamily C member 19 (CYP2C19) polymorphisms on the plasma concentrations, efficacy, and safety of lacosamide (LCM) among pediatric patients with epilepsy. This prospective study was conducted at two institutions. It included 215 pediatric patients with epilepsy who were under LCM. LCM plasma concentrations were quantified using validated ultra-performance liquid chromatography. CYP2C9 and CYP2C19 polymorphisms were analyzed in all pediatric patients in our hospital's Institute of Clinical Pharmacy research laboratory through polymerase chain reaction, agarose gel electrophoresis detection, gel recovery, and other steps. Seizure frequencies were recorded 3, 6, and 12 months after initiating LCM therapy and compared with the baseline monthly frequency. Clinical information, including efficacy, toxicity, and concomitant drugs, was collected. A total of 158 pediatric patients (73.5%) responded to LCM therapy. Of them, 77 patients reported adverse events while under LCM. The LCM plasma concentration was linearly correlated with its daily dose (r = 0.26, p < 0.001). Patients with adverse events reported higher LCM plasma concentrations (7.9 ± 4.0 µg/mL) than patients without adverse events (6.8 ± 3.0 µg/mL; p < 0.05). The poor metabolizer (PM) group demonstrated the highest concentration-to-dose ratio (1.7 ± 0.7 μg·mL-1·kg·mg-1) than the extensive metabolizer, intermediate metabolizer, and ultra-rapid metabolizer groups (0.8 ± 0.4, 1.0 ± 0.5, and 0.8 ± 0.4 μg·mL-1·kg·mg-1, respectively). The PM group comprised the highest proportion of patients with effective LCM (9/11, 81.8%) and adverse events (7/11, 63.6%).
Conclusion: LCM plasma concentrations were strongly associated with its clinical efficacy and toxicity. CYP2C19 polymorphisms affect the plasma concentration and treatment efficacy in pediatric patients with epilepsy. CYP2C19 PMs with two no-function alleles are likely to have higher LCM plasma concentrations.
What is known: • LCM is metabolized by CYP2C19, CYP2C9, and CYP3A4 into pharmacologically inactive O-desmethyl-lacosamide; it primarily undergoes renal elimination. • Plasma LCM concentrations in patients treated with the recommended dose vary widely between and within individuals variability.
What is new: • CYP2C19 polymorphisms affect the plasma concentration and treatment efficacy in Chinese pediatric patients with epilepsy. • CYP2C19 PMs with two no-function alleles are likely to have higher plasma LCM concentrations.
{"title":"Effects of CYP2C19 and CYP2C9 polymorphisms on the efficacy and plasma concentration of lacosamide in pediatric patients with epilepsy in China.","authors":"Ting Zhao, Hong-Jian Li, Hui-Lan Zhang, Jing Yu, Jie Feng, Long Cui, Ke-Fang Sun, Yan Sun, Lu-Hai Yu","doi":"10.1007/s00431-024-05897-6","DOIUrl":"10.1007/s00431-024-05897-6","url":null,"abstract":"<p><p>To evaluate the effects of cytochrome P450 family 2 subfamily C member 9 (CYP2C9) and cytochrome P450 family 2 subfamily C member 19 (CYP2C19) polymorphisms on the plasma concentrations, efficacy, and safety of lacosamide (LCM) among pediatric patients with epilepsy. This prospective study was conducted at two institutions. It included 215 pediatric patients with epilepsy who were under LCM. LCM plasma concentrations were quantified using validated ultra-performance liquid chromatography. CYP2C9 and CYP2C19 polymorphisms were analyzed in all pediatric patients in our hospital's Institute of Clinical Pharmacy research laboratory through polymerase chain reaction, agarose gel electrophoresis detection, gel recovery, and other steps. Seizure frequencies were recorded 3, 6, and 12 months after initiating LCM therapy and compared with the baseline monthly frequency. Clinical information, including efficacy, toxicity, and concomitant drugs, was collected. A total of 158 pediatric patients (73.5%) responded to LCM therapy. Of them, 77 patients reported adverse events while under LCM. The LCM plasma concentration was linearly correlated with its daily dose (r = 0.26, p < 0.001). Patients with adverse events reported higher LCM plasma concentrations (7.9 ± 4.0 µg/mL) than patients without adverse events (6.8 ± 3.0 µg/mL; p < 0.05). The poor metabolizer (PM) group demonstrated the highest concentration-to-dose ratio (1.7 ± 0.7 μg·mL<sup>-1</sup>·kg·mg<sup>-1</sup>) than the extensive metabolizer, intermediate metabolizer, and ultra-rapid metabolizer groups (0.8 ± 0.4, 1.0 ± 0.5, and 0.8 ± 0.4 μg·mL<sup>-1</sup>·kg·mg<sup>-1</sup>, respectively). The PM group comprised the highest proportion of patients with effective LCM (9/11, 81.8%) and adverse events (7/11, 63.6%).</p><p><strong>Conclusion: </strong>LCM plasma concentrations were strongly associated with its clinical efficacy and toxicity. CYP2C19 polymorphisms affect the plasma concentration and treatment efficacy in pediatric patients with epilepsy. CYP2C19 PMs with two no-function alleles are likely to have higher LCM plasma concentrations.</p><p><strong>What is known: </strong>• LCM is metabolized by CYP2C19, CYP2C9, and CYP3A4 into pharmacologically inactive O-desmethyl-lacosamide; it primarily undergoes renal elimination. • Plasma LCM concentrations in patients treated with the recommended dose vary widely between and within individuals variability.</p><p><strong>What is new: </strong>• CYP2C19 polymorphisms affect the plasma concentration and treatment efficacy in Chinese pediatric patients with epilepsy. • CYP2C19 PMs with two no-function alleles are likely to have higher plasma LCM concentrations.</p>","PeriodicalId":11997,"journal":{"name":"European Journal of Pediatrics","volume":"184 1","pages":"73"},"PeriodicalIF":3.0,"publicationDate":"2024-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142806406","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}