Archives De Pediatrie最新文献

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Kidney outcomes in Shiga toxin-associated hemolytic uremic syndrome in childhood: A retrospective single-center study from 1999 to 2017 儿童志贺毒素相关溶血性尿毒症综合征的肾脏结局:1999年至2017年的回顾性单中心研究:儿童典型溶血性尿毒症综合征的肾脏结局。
IF 1.8 4区 医学
Archives De Pediatrie Pub Date : 2024-01-01 DOI: 10.1016/j.arcped.2023.09.016
Astrid Viennet , Jean-Baptiste Pretalli , Rachel Vieux , François Nobili
{"title":"Kidney outcomes in Shiga toxin-associated hemolytic uremic syndrome in childhood: A retrospective single-center study from 1999 to 2017","authors":"Astrid Viennet ,&nbsp;Jean-Baptiste Pretalli ,&nbsp;Rachel Vieux ,&nbsp;François Nobili","doi":"10.1016/j.arcped.2023.09.016","DOIUrl":"10.1016/j.arcped.2023.09.016","url":null,"abstract":"<div><h3>Background</h3><p><span>Shiga toxin-associated hemolytic uremic syndrome (STEC</span><img><span><span>HUS) is the main cause of acute kidney injury in children and may be responsible for </span>adverse outcomes despite an apparent quiescent period.</span></p></div><div><h3>Objective</h3><p><span>To describe the medium- and long-term kidney outcomes of pediatric STEC</span><img>HUS in a French region.</p></div><div><h3>Methods</h3><p>A single-center, descriptive, retrospective study of STEC<img><span>HUS cases that occurred at Besançon University Hospital between 1999 and 2017 in children up to 17 years of age was conducted. The primary study endpoint was the proportion of chronic kidney disease (CKD) cases at 5 years of follow-up.</span></p></div><div><h3>Results</h3><p><span><span>We included 98 consecutive patients. Among the 71 patients at the 5-year follow-up, we found 24 (34 %) patients with no adverse kidney outcome, 18 (25 %) with moderate adverse kidney outcome, and one (1.4 %) with severe adverse kidney outcome. Among the 96 patients at 1 year from the diagnosis, these figures were, respectively, 25 (26 %), 51 (53 %), and two (2 %); and among the 38 patients at 10 years, they were, respectively, nine (24 %), 12 (32 %), and one (3 %). The </span>glomerular filtration rate level and oliguria–anuria beyond 8 days at baseline were significantly associated with more severe kidney outcomes at 10 years (</span><em>p</em> = 0.03 and 0.005, respectively). Two patients died during the acute phase. Overall, 33 patients (34 %) were lost to follow-up.</p></div><div><h3>Conclusion</h3><p>Adverse kidney outcomes may appear many years after an episode of STEC<img><span>HUS despite an apparent quiescent period. Regular long-term monitoring is required. The challenge is to reduce the proportion of patients lost to follow-up with potentially severe adverse kidney outcomes and no evaluation or treatment.</span></p></div>","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138292471","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cow's milk-based infant formula supplements in breastfed infants and primary prevention of cow's milk allergy: A commentary of the Committee on Nutrition of the French Society of Pediatrics 母乳喂养婴儿的牛奶配方补充剂和牛奶过敏的初级预防:法国儿科学会营养委员会的评论。
IF 1.8 4区 医学
Archives De Pediatrie Pub Date : 2023-11-01 DOI: 10.1016/j.arcped.2023.07.005
Christophe Dupont , Alain Bocquet , Sandra Brancato , Martin Chalumeau , Dominique Darmaun , Arnaud de Luca , François Feillet , Marie-Laure Frelut , Dominique Guimber , Alexandre Lapillonne , Agnès Linglart , Noel Peretti , Jean-Christophe Roze , Umberto Siméoni , Dominique Turck , Jean-Pierre Chouraqui , Committee on Nutrition of the French Society of Pediatrics
{"title":"Cow's milk-based infant formula supplements in breastfed infants and primary prevention of cow's milk allergy: A commentary of the Committee on Nutrition of the French Society of Pediatrics","authors":"Christophe Dupont ,&nbsp;Alain Bocquet ,&nbsp;Sandra Brancato ,&nbsp;Martin Chalumeau ,&nbsp;Dominique Darmaun ,&nbsp;Arnaud de Luca ,&nbsp;François Feillet ,&nbsp;Marie-Laure Frelut ,&nbsp;Dominique Guimber ,&nbsp;Alexandre Lapillonne ,&nbsp;Agnès Linglart ,&nbsp;Noel Peretti ,&nbsp;Jean-Christophe Roze ,&nbsp;Umberto Siméoni ,&nbsp;Dominique Turck ,&nbsp;Jean-Pierre Chouraqui ,&nbsp;Committee on Nutrition of the French Society of Pediatrics","doi":"10.1016/j.arcped.2023.07.005","DOIUrl":"10.1016/j.arcped.2023.07.005","url":null,"abstract":"<div><p><span>The role of nutritional interventions for the primary prevention of cow's milk allergy (CMA) remains debated as well as the role of early introduction of allergenic foods, which is largely encouraged from the beginning of complementary feeding. Considering the introduction of cow's milk protein (CMP), current recommendations suggest avoidance of any cow's milk formula (CMF) supplements in breastfed infants in the </span>maternity<span> ward. By contrast, based on poor evidence, some authors support systematic supplements of CMP in breastfed children at risk of allergy from the first week of life. The Committee on Nutrition of the French Society of Pediatrics considers that such a proposal requires more clinical studies and mainly randomized and placebo-controlled clinical trials before becoming a recommendation.</span></p></div>","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10244584","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Outcomes after aortic coarctation repair in neonates weighing less than 2000 g 体重小于2000 g新生儿主动脉缩窄修复后的结果。
IF 1.8 4区 医学
Archives De Pediatrie Pub Date : 2023-11-01 DOI: 10.1016/j.arcped.2023.08.002
Victoria Butler , Zahra Belhadjer , Régis Gaudin , Olivier Raisky , Lucile Houyel , Damien Bonnet
{"title":"Outcomes after aortic coarctation repair in neonates weighing less than 2000 g","authors":"Victoria Butler ,&nbsp;Zahra Belhadjer ,&nbsp;Régis Gaudin ,&nbsp;Olivier Raisky ,&nbsp;Lucile Houyel ,&nbsp;Damien Bonnet","doi":"10.1016/j.arcped.2023.08.002","DOIUrl":"10.1016/j.arcped.2023.08.002","url":null,"abstract":"<div><h3>Background</h3><p><span>Preterm birth is common in children with </span>congenital heart disease<span>. However, data on how to manage low-birth-weight infants with aortic coarctation are scarce and outcomes are poorly reported. Surgery is often delayed in these infants because gaining weight is supposed to improve mortality and to reduce the risk for recoarctation.</span></p></div><div><h3>Methods</h3><p>All infants weighing less than 2000 g who underwent repair for aortic coarctation at our institution between January 2017 and December 2020 were included in a retrospective study. Baseline characteristics, medical and surgical management, and outcomes, including recoarctation, death, and complications of preterm birth, were analyzed.</p></div><div><h3>Results</h3><p><span>A total of 15 patients had coarctation repair at a median age of 15 days and at a median weight of 1585 g. Infants with a birth weight &lt;1200 g were operated on later and did not have higher recoarctation rates compared to those with a birth weight &gt;1200 g. The recoarctation rate was 26.6% and one infant died of an extracardiac cause. Concerning prematurity-related complications, we observed 40% of bronchopulmonary dysplasia<span>, 40% of intraventricular hemorrhage, and 27% of </span></span>retinopathy of prematurity. These complications were more prevalent in children with a birth weight of &lt;1200 g.</p></div><div><h3>Conclusion</h3><p>Delaying surgery beyond 15 days to gain weight does not appear to decrease the risk of recoarctation and may be deleterious in low-birth-weight infants who are exposed for a longer period to risk factors of prematurity-related complications.</p></div>","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10297153","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Perceptions of pediatric residents and pediatricians about ethical dilemmas: The case of Turkey 儿科住院医师和儿科医生对伦理困境的看法:以土耳其为例。
IF 1.8 4区 医学
Archives De Pediatrie Pub Date : 2023-11-01 DOI: 10.1016/j.arcped.2023.06.012
Nihal Durmaz , Betül Ulukol , Serap Şahinoğlu
{"title":"Perceptions of pediatric residents and pediatricians about ethical dilemmas: The case of Turkey","authors":"Nihal Durmaz ,&nbsp;Betül Ulukol ,&nbsp;Serap Şahinoğlu","doi":"10.1016/j.arcped.2023.06.012","DOIUrl":"10.1016/j.arcped.2023.06.012","url":null,"abstract":"<div><h3>Background</h3><p>This study determined the knowledge, attitudes, and behaviors of pediatricians and pediatric residents regarding issues of ethics, professional education, clinical ethical principles, and consent they encounter in health service delivery.</p></div><div><h3>Methods</h3><p>Participants in the study were 134 pediatricians and pediatric residents from three hospitals in Ankara, Turkey. Participants were asked questions regarding their sociodemographic characteristics, their knowledge and views of ethics and ethical education, whether they had ever encountered an ethical problem, their beliefs about obtaining consent from pediatric patients and their families, and case-based questions. All data were collected and evaluated.</p></div><div><h3>Results</h3><p>Of the participants in the study, 82 (61.2%) were residents, 41 (30.6%) were pediatricians, and 11 (8.2%) were faculty lecturers. The data revealed that 10% of the pediatricians and pediatric residents received ethical education, apart from medical ethics/deontology education at medical school, and 90.3% required further education on children and ethical problems. It was determined that 89% of residents and 78% of pediatricians needed help with the ethical problems they encountered during the diagnostic and treatment processes. Overall, 65.7% of the participants stated that the ethical problems they encountered affected the efficiency of health service delivery. It was observed that residents were more affected by ethical issues than pediatricians were. The present study revealed that pediatric residents and pediatricians need ethics education and there is a need to establish ethics counseling centers in hospitals. There is also a need for further studies in pediatrics and ethics.</p></div>","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10264108","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Evolution of screen use among youth between 2012 and 2020 in Switzerland 2012年至2020年瑞士青少年屏幕使用情况的演变。
IF 1.8 4区 医学
Archives De Pediatrie Pub Date : 2023-11-01 DOI: 10.1016/j.arcped.2023.09.001
Maëlle Mora-Monteros , Joan-Carles Suris , Lorraine Chok , Agnieszka Siwiak , Sophie Stadelmann , Yara Barrense-Dias
{"title":"Evolution of screen use among youth between 2012 and 2020 in Switzerland","authors":"Maëlle Mora-Monteros ,&nbsp;Joan-Carles Suris ,&nbsp;Lorraine Chok ,&nbsp;Agnieszka Siwiak ,&nbsp;Sophie Stadelmann ,&nbsp;Yara Barrense-Dias","doi":"10.1016/j.arcped.2023.09.001","DOIUrl":"10.1016/j.arcped.2023.09.001","url":null,"abstract":"<div><h3>Aim</h3><p>To compare the evolution of screen and Internet use by youths between 2012 and 2020 in Switzerland.</p></div><div><h3>Methods</h3><p>Cross-sectional studies of 10th-graders (age 13–14) were performed in Switzerland in 2012 and 2020, and compared in bivariate and multivariate analyses on sociodemographic, schooling, physical activity, emotional well-being, and screen use variables.</p></div><div><h3>Results</h3><p>We found that screen use had shifted to smartphones with 71.7 % of youths primarily using this device in 2020 compared to 23.2 % in 2012. In association with this change, young people's screen time had increased dramatically with an odds ratio (OR) of 11.90 for adolescents spending more than 4 h in front of screens in 2020 compared to 2012. No changes were found in the score on the Internet Addiction Test (IAT) to detect problematic screen use and for adolescents’ emotional well-being. Furthermore, youths in 2020 engaged in less physical activity lasting 60 min daily, but the frequency of their extracurricular sport participation remained unchanged.</p></div><div><h3>Conclusions</h3><p>Young people spend more time on screens, especially because of an increase in smartphone use in 2020. However, youths do not seem to show more problematic behaviors regarding screen use, nor has this development affected their emotional well-being. The daily and continuous use of new devices is now an integral part of young people's lives. This process seems to be part of the growth of the digital world. However, Internet and screen addiction scales should be adapted to ensure that adolescents in need of help and counseling are identified.</p></div>","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0929693X23001471/pdfft?md5=034cd478f79892bf20780d29e3cfe3c7&pid=1-s2.0-S0929693X23001471-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41121992","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Evaluation of the growth response of children with growth hormone deficiency according to the peak growth hormone levels in provocation tests 根据激发试验中生长激素峰值水平评估生长激素缺乏儿童的生长反应。
IF 1.8 4区 医学
Archives De Pediatrie Pub Date : 2023-11-01 DOI: 10.1016/j.arcped.2023.08.005
Zeynep Donbaloğlu , Berna Singin , Sezer Acar , Aynur Bedel , Ebru Barsal Çetiner , Bilge Aydın Behram , Mesut Parlak , Hale Tuhan
{"title":"Evaluation of the growth response of children with growth hormone deficiency according to the peak growth hormone levels in provocation tests","authors":"Zeynep Donbaloğlu ,&nbsp;Berna Singin ,&nbsp;Sezer Acar ,&nbsp;Aynur Bedel ,&nbsp;Ebru Barsal Çetiner ,&nbsp;Bilge Aydın Behram ,&nbsp;Mesut Parlak ,&nbsp;Hale Tuhan","doi":"10.1016/j.arcped.2023.08.005","DOIUrl":"10.1016/j.arcped.2023.08.005","url":null,"abstract":"<div><h3>Background</h3><p><span>We aimed to evaluate the relation between the peak growth hormone (GH) levels in provocation tests and response to recombinant human GH (rhGH) therapy </span>in patients<span> with GH deficiency (GHD).</span></p></div><div><h3>Methods</h3><p>This was a cross-sectional, single-center, and retrospective study. A total of 135 patients under the age of 16 years who were diagnosed with GHD through insulin tolerance tests<span> and L-DOPA stimulation tests and who received rhGH therapy for at least 2 years in the Pediatric Endocrinology Clinic of Akdeniz University Hospital between 1997 and 2021 were included in the study.</span></p></div><div><h3>Results</h3><p>The patients were divided into two groups: idiopathic GHD (group I, <em>n</em><span> = 119) and multiple pituitary hormone deficiencies or organic pathology on magnetic resonance imaging (group II, </span><em>n</em> = 16). The patients in group I were classified into three subgroups according to the peak GH values in the provocation tests (group Ia: peak GH &lt;3 μg/L, <em>n</em> = 34; group Ib: peak GH between 3 and 7 μg/L, <em>n</em> = 71; group Ic: peak GH between 7 and 10 μg/L, <em>n</em><span> = 34). The median age was 11.5 years in group I (8.8 in group Ia, 12.1 in group Ib, 12.3 in group Ib) and 8.8 years in group II. The height standard deviation score (SDS) was −2.93 in group I (−2.85 in group Ia, −2.99 in group Ib, −2.94 in group Ic) and −3.79 in group II. The median Δheight SDS was 0.61 in group I and 1.05 in group II at the end of the first year of treatment and 0.31 in group I and 0.45 in group II at the end of the second year (</span><em>p</em> = 0.005 and <em>p</em> = 0.074, respectively). When the subgroups of group I were compared, height SDS, Δheight SDS, and height velocity (HV) SDS were all higher in group Ia at the end of the first year of rhGH therapy (<em>p</em> = 0.040, <em>p</em> = 0.029, and <em>p</em> = 0.005, respectively). The height SDS was still significantly higher in group Ia (<em>p</em> = 0.033) while the HV SDS and Δheight SDS were similar between the groups at the end of the second year of therapy (<em>p</em> = 0.164 and <em>p</em> = 0.522, respectively). There was a statistically significant association between the first-year HV SDS and the peak GH value in provocation tests in multiple regression analyses (<em>p</em>&lt;0.001). In addition, the final model revealed that height SDS and weight SDS at the start of the treatment and the first-year HV SDS are the factors with a statistically significant effect on the second-year HV SDS (<em>p</em> = 0.022, <em>p</em> = 0.001, and <em>p</em>&lt;0.001, respectively).</p></div><div><h3>Conclusion</h3><p>Our findings show that the lower the GH peak in provocation tests, the better the response to treatment. The best HV was observed in the first year of rhGH therapy, and the diagnosis should be checked in those patients who had a low first-year HV and did not have a severely low GH pe","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41169158","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Current limitations of gene therapy for rare pediatric diseases: Lessons learned from clinical experience with AAV vectors 目前罕见儿科疾病基因治疗的局限性:从AAV载体临床经验中吸取的教训
IF 1.8 4区 医学
Archives De Pediatrie Pub Date : 2023-11-01 DOI: 10.1016/S0929-693X(23)00227-0
Antoine Gardin , Giuseppe Ronzitti
{"title":"Current limitations of gene therapy for rare pediatric diseases: Lessons learned from clinical experience with AAV vectors","authors":"Antoine Gardin ,&nbsp;Giuseppe Ronzitti","doi":"10.1016/S0929-693X(23)00227-0","DOIUrl":"https://doi.org/10.1016/S0929-693X(23)00227-0","url":null,"abstract":"<div><p><span>Gene therapy using adeno-associated viral (AAV) vectors is a promising therapeutic strategy for multiple inherited diseases. Following intravenous injection, AAV vectors carrying a copy of the missing gene or the genome-editing machinery reach their target cells and deliver the genetic material. Several </span>clinical trials<span><span> are currently ongoing and significant success has already been achieved with at least six AAV gene therapy products with market approval in Europe and the United States. Nonetheless, clinical trials and preclinical studies have uncovered several limitations of </span>AAV gene transfer<span>, which need to be addressed in order to improve the safety and enable the treatment<span> of the largest patient population. Limitations include the occurrence of immune-mediated toxicities, the potential loss of correction in the long run, and the development of neutralizing antibodies against AAV vectors preventing re-administration. In this review, we summarize these limitations and discuss the potential technological developments to overcome them.</span></span></span></p><p>© 2023 Published by Elsevier Masson SAS on behalf of French Society of Pediatrics.</p></div>","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138472495","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Gene therapy for neurodegenerative disorders in children: dreams and realities 儿童神经退行性疾病的基因治疗:梦想与现实
IF 1.8 4区 医学
Archives De Pediatrie Pub Date : 2023-11-01 DOI: 10.1016/S0929-693X(23)00225-7
Odile Boespflug-Tanguy , Caroline Sevin , Francoise Piguet
{"title":"Gene therapy for neurodegenerative disorders in children: dreams and realities","authors":"Odile Boespflug-Tanguy ,&nbsp;Caroline Sevin ,&nbsp;Francoise Piguet","doi":"10.1016/S0929-693X(23)00225-7","DOIUrl":"https://doi.org/10.1016/S0929-693X(23)00225-7","url":null,"abstract":"<div><p><span><span>Gene therapy encompasses the administration of biological medicinal products containing recombinant nucleic acids<span><span><span>, mainly DNA, with the aim of treating or curing diseases. This represents a unique therapeutic strategy to reach the brain, in order to prevent or halt a neurodegenerative process. During the past decade, active multidisciplinary research has started to solve many issues for gene therapy in neurodegenerative disorders in terms of vectors, modes of administration, and expression of the therapeutic DNA. The engineering of </span>hematopoietic stem cells (HSC) with </span>lentivirus vectors<span> for ex vivo gene therapy has demonstrated efficiency in reaching the brain through their transformation into microglial/macrophages cells with a long-term gene expression of the therapeutic vector as an alternative to autologous HSC transplants. Two </span></span></span>drugs based on this strategy have been approved to date. The first is for </span>metachromatic leukodystrophy<span><span><span><span><span> (MLD), a severe lysosomal storage disease, and provides high levels of the deficient enzyme; the second one is for cerebral forms of X-linked </span>adrenoleukodystrophy<span> (X-ALD), and works by halting the neuroinflammation process. However, due to the long-lasting effect of the procedure, the therapy is applicable only to pre- or pauci/oligo-symptomatic patients. </span></span>In vivo gene therapy<span> via direct injection into the brain or the cerebrospinal fluid, but also by </span></span>intravenous injection<span>, represents a more efficient approach; however, many challenges remain to be solved despite the approval of two drugs: one for the early infantile form of spinal muscular atrophy (SMA), in which the gene product injected intravenously is able to prevent spinal </span></span>motoneuron<span><span> neurodegeneration. The second one, for aromatic L-amino acid decarboxylase (AADC) deficiency, provides the defective enzyme to the </span>basal ganglia via intraparenchymal injection. The production of vectors able to reach the brain target cells with a sufficiently high expression remains a major bottleneck. In parallel, efforts must continue in order to better define (i) the natural history and clinical outcomes of many neurodegenerative disorders with childhood onset, and (ii) the mechanisms involved in the neurodegenerative process.</span></span></p><p>© 2023 Published by Elsevier Masson SAS on behalf of French Society of Pediatrics.</p></div>","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138472499","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Title Page 标题页
IF 1.8 4区 医学
Archives De Pediatrie Pub Date : 2023-11-01 DOI: 10.1016/S0929-693X(23)00217-8
{"title":"Title Page","authors":"","doi":"10.1016/S0929-693X(23)00217-8","DOIUrl":"https://doi.org/10.1016/S0929-693X(23)00217-8","url":null,"abstract":"","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138472570","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The prevalence of vitamin D deficiency and the factors affecting vitamin D levels in children admitted to the outpatient clinic of pediatric endocrinology in Bolu Province 勃鲁省儿科内分泌门诊儿童维生素D缺乏的患病率及影响维生素D水平的因素。
IF 1.8 4区 医学
Archives De Pediatrie Pub Date : 2023-11-01 DOI: 10.1016/j.arcped.2023.08.010
Yasemin Baranoglu Kilinc , Semih Bolu
{"title":"The prevalence of vitamin D deficiency and the factors affecting vitamin D levels in children admitted to the outpatient clinic of pediatric endocrinology in Bolu Province","authors":"Yasemin Baranoglu Kilinc ,&nbsp;Semih Bolu","doi":"10.1016/j.arcped.2023.08.010","DOIUrl":"10.1016/j.arcped.2023.08.010","url":null,"abstract":"<div><h3>Background</h3><p>The present study aimed to determine the rate of vitamin D deficiency<span><span> in children who presented to the pediatric endocrinology outpatient clinic in Bolu and to investigate the factors affecting </span>vitamin D levels.</span></p></div><div><h3>Methods</h3><p>Vitamin D levels of 1008 children and adolescents were retrospectively analyzed according to age group (0–1, 1–10, and 10–18 years), gender, season, month, obesity and other diseases, and deficiency category. Moreover, calcium, phosphorus, alkaline phosphatase<span>, and parathyroid hormone levels were evaluated. Comparisons and correlation analyses between related groups were performed.</span></p></div><div><h3>Results</h3><p>The mean vitamin D level of the patients was 16.35±9.56 ng/mL and was lower in girls (14.90±9.56 ng/mL) than in boys (18.68±9.63 ng/mL, <em>p</em>&lt;0.001). Overall, 18.3% of the children and adolescents had vitamin D insufficiency, 52.3% had vitamin D deficiency, and 3.5% had severe vitamin D deficiency. Vitamin D levels were lower in 10–18-year age group than in the other age groups (<em>p</em>&lt;0.001), and levels were higher in summer and autumn than in winter and spring (<em>p</em><span>&lt;0.001). Vitamin D levels of participants with obesity (14.3 ± 8.3 ng/mL) were significantly lower than normal-weight participants with no health problems (15.9 ± 8.3 ng/mL, </span><em>p</em> = 0.004). There was a negative correlation between vitamin D, alkaline phosphatase, and parathyroid hormone levels in the 1–10-year age group, but a positive correlation between vitamin D, alkaline phosphatase, and calcium levels in the 10–18-year age group.</p></div><div><h3>Conclusion</h3><p>The rate of vitamin D deficiency is high among children and adolescents who presented to the endocrine outpatient clinic in Bolu. The season appears to be an important factor affecting vitamin D levels as well as the relationship between vitamin D and parathyroid hormone. Obese children and adolescents living in this region may be advised to take vitamin D supplements in winter and spring.</p></div>","PeriodicalId":55477,"journal":{"name":"Archives De Pediatrie","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41142675","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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