Pediatric neurology最新文献

{"title":"Clinical Characteristics and Prognostic Factors of Anti-GM1 Antibody-Positive Guillain-Barré Syndrome Spectrum Disorders in Children","authors":"Jiaqi Yan BS ,&nbsp;Lamei Chen MS ,&nbsp;Peijiao Liu BS ,&nbsp;Hailun Peng MS ,&nbsp;Li Jiang MD ,&nbsp;Yue Hu MD","doi":"10.1016/j.pediatrneurol.2025.03.007","DOIUrl":"10.1016/j.pediatrneurol.2025.03.007","url":null,"abstract":"<div><h3>Background</h3><div>The study aimed to analyze the clinical features and risk factors for poor prognosis of Guillain-Barré syndrome (GBS) spectrum disorders in children positive for anti-tetrahexose monosialoganglioside (GM1) antibody.</div></div><div><h3>Methods</h3><div>We collected data for children with anti-GM1 antibody-positive GBS spectrum disorders in Affiliated Children's Hospital of Chongqing Medical University between July 2018 and March 2024; 1:1 matching was performed for combined anti-ganglioside or anti-sulfatide antibody. The patients underwent comparative clinical characterization to determine the antibody phenotype-clinical phenotype and to analyze the possible risk factors for the poor prognosis of the disorders.</div></div><div><h3>Results</h3><div>Thirty-seven pediatric patients were recruited. Anti-GM1 antibody-positive GBS spectrum disorders were preceded by a prodromal event (25 of 37, 67.6%). The first symptom was mainly limb weakness (20 of 37, 54.1%), which could be predominately accompanied by autonomic nerve involvement (21 of 37, 56.8%). Seven features showed statistically significant differences (<em>P</em> &lt; 0.05) between the positive group and the negative one, including cranial nerve involvement, bulbar palsy, low lower limb muscle strength at discharge, axonal type of electrophysiological typing, and clinical typing of acute motor axonal neuropathy. The GBS disability scores at discharge and at one month after discharge were higher than those in the control group. The shorter time to peak (&lt;7.5 days) was identified as an independent risk factor for poor short-term prognosis of the disorders.</div></div><div><h3>Conclusions</h3><div>Anti-GM1 antibody-positive GBS spectrum disorders have a relatively specific antibody phenotype-clinical phenotype. The shorter time to peak (&lt;7.5 days) is an independent risk factor for poor short-term prognosis of the disorders in children.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"167 ","pages":"Pages 42-51"},"PeriodicalIF":3.2,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143800145","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}

{"title":"Dimethyl Fumarate for Pediatric-Onset Multiple Sclerosis: A Systematic Review","authors":"Masoud Etemadifar MD ,&nbsp;Hasan Kaveyee MD ,&nbsp;Yasin Ebne-Ali-Heydari MD ,&nbsp;Parto Zohrabi MD ,&nbsp;Pantea Miralaei MD ,&nbsp;Nahad Sedaghat MD ,&nbsp;Amir Mohammad Jozaie MD ,&nbsp;Mehri Salari MD ,&nbsp;Aryana Ramezani MD","doi":"10.1016/j.pediatrneurol.2025.03.008","DOIUrl":"10.1016/j.pediatrneurol.2025.03.008","url":null,"abstract":"<div><h3>Background</h3><div>Dimethyl fumarate (DMF) is an oral disease-modifying therapy (DMT) being used for adult patients with multiple sclerosis (MS) with acceptable safety and efficacy profile. The use of DMF for pediatric-onset multiple sclerosis (POMS) is being considered especially for patients who prefer a self-administered oral DMT. To systematically review the experience with DMF in POMS regarding the safety and efficacy profile.</div></div><div><h3>Methods</h3><div>This systematic review was conducted based on the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. Three different databases were searched (PubMed, Scopus, and Web of Science) to find relevant articles until the end of May 15, 2024. Data were screened and extracted by two independent authors.</div></div><div><h3>Results</h3><div>From 344 studies, six studies with 316 patients were included in the systematic review. DMF had an acceptable effect on reducing relapses and magnetic resonance imaging activity. Gastrointestinal discomfort and facial flushing were the most reported adverse events related to the use of DMF in patients with POMS.</div></div><div><h3>Conclusions</h3><div>DMF could be considered for patients with POMS both as first-line and switching therapy. Despite a recent tendency toward high-efficacy DMTs for POMS, DMF remains a good option for certain cases.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"167 ","pages":"Pages 52-59"},"PeriodicalIF":3.2,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143814971","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}

{"title":"Brain Folate Depletion as a Cause of Metabolic Stroke in Dihydropteridine Reductase Deficiency: A Suggestive Case Report","authors":"Giacomina Ricciardi MD ,&nbsp;Mario Mastrangelo MD, PhD ,&nbsp;Claudia Carducci MD ,&nbsp;Antonio Gambardella MD ,&nbsp;Francesco Pisani MD ,&nbsp;Vincenzo Leuzzi MD","doi":"10.1016/j.pediatrneurol.2025.03.006","DOIUrl":"10.1016/j.pediatrneurol.2025.03.006","url":null,"abstract":"","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"167 ","pages":"Pages 6-8"},"PeriodicalIF":3.2,"publicationDate":"2025-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143746767","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}

{"title":"Postconcussion Clinical Insomnia is Associated With Heightened Symptom Severity and Delayed Recovery in Children and Adolescents","authors":"Maree Cassimatis BAppSc (ExPhys) ,&nbsp;Rhonda Orr PhD ,&nbsp;Andrew Fyffe BExPhys ,&nbsp;Gary Browne MD","doi":"10.1016/j.pediatrneurol.2025.03.004","DOIUrl":"10.1016/j.pediatrneurol.2025.03.004","url":null,"abstract":"<div><h3>Background</h3><div>There is limited evidence on the prevalence and implications of specific types of sleep disturbance in pediatric concussion. This study aimed to (1) identify the prevalence of postconcussion clinical insomnia (PCCI) in children, (2) determine the impact of PCCI on concussion recovery outcomes, and (3) ascertain clinical characteristics associated with PCCI.</div></div><div><h3>Methods</h3><div>A cross-sectional study was conducted in patients (n = 164, aged 9-17 years) presenting to a pediatric tertiary referral concussion clinic from January 2021 to December 2022. PCCI was identified using the Insomnia Severity Index. Characteristics, including symptom severity, cognitive function, sleep hygiene behavior, and exercise tolerance, were compared between patients presenting with and without insomnia postinjury. A subgroup analysis of recovered patients was undertaken to determine the impact of PCCI on concussion recovery duration.</div></div><div><h3>Results</h3><div>Over one third of patients (n = 59, 36%) presented with PCCI. Symptom severity was three times greater in patients with PCCI compared with patients without insomnia postconcussion (<em>P</em> &lt; 0.001). Patients with PCCI had inferior cognitive function in verbal memory (<em>P</em> = 0.01) and visual memory (<em>P</em> = 0.02) cognitive test domains and poorer sleep hygiene behavior (<em>P</em> &lt; 0.001). The subgroup analysis of recovered patients (n = 113) revealed that patients with PCCI had a significantly prolonged recovery duration, taking over one month longer to recover compared with patients without insomnia postinjury (mean recovery duration: 89 vs 57 days, <em>P</em> = 0.004).</div></div><div><h3>Conclusions</h3><div>PCCI is an important contributing factor of symptom burden and severity in pediatric concussion.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"167 ","pages":"Pages 17-23"},"PeriodicalIF":3.2,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143768030","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}

{"title":"Developmental, Endocrine, and Ophthalmologic Outcomes in Children Prenatally Diagnosed With Midline Brain Malformations","authors":"McKenna L. Coletti MD ,&nbsp;Jennifer C. Keene MD, MBA, MS ,&nbsp;Allison R. Smego MD ,&nbsp;Marielle P. Young MD ,&nbsp;Betsy Ostrander MD","doi":"10.1016/j.pediatrneurol.2025.03.005","DOIUrl":"10.1016/j.pediatrneurol.2025.03.005","url":null,"abstract":"<div><h3>Background</h3><div>Midline brain malformations (MBMs) are commonly prenatally diagnosed and associated with endocrinologic, ophthalmic, and adverse developmental outcomes.</div></div><div><h3>Methods</h3><div>A retrospective review was conducted of all neonates identified prenatally with suspected MBMs between 2018 and 2022 at the only multidisciplinary referral center in a multistate region. Abnormalities were categorized as isolated versus complicated absent cavum septum pellucidum (ASP) (N = 11 vs N = 13) or corpus callosum abnormalities (AgCC) (N = 11 vs N = 43) or holoprosencephaly spectrum (N = 12). We assessed subsequent diagnoses and outcomes using a standardized assessment pathway.</div></div><div><h3>Results</h3><div>Infants with holoprosencephaly were significantly more likely to die than patients with isolated ASP or AgCC (<em>P</em> = 0.02). Surviving infants with holoprosencephaly had universal developmental delay, significantly more than the 10% seen in isolated ASP or AgCC (<em>P</em> = 0.007), and infants with isolated MBMs were significantly more likely to be alive and without endocrine, ophthalmologic, developmental, or epileptic diagnoses at last follow-up than other groups (isolated ASP = 67%, AgCC 82%). The median time to diagnosis of optic nerve hypoplasia was 3 days and initial identification of endocrine concerns was 7 days. There were no significant differences between rates of diagnosis for endocrine, ophthalmologic, or epileptic complications between groups, with all MBMs demonstrating a risk for complications.</div></div><div><h3>Conclusions</h3><div>Our study shows the importance of multidisciplinary screening in all infants with midline brain defects. Most infants with isolated ASP or AgCC did not have MBM-associated diagnoses at last follow-up, but all groups had comorbidities and would benefit from multispecialty postnatal monitoring.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"167 ","pages":"Pages 82-88"},"PeriodicalIF":3.2,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143824008","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}

{"title":"Pediatric Immune-Mediated Necrotizing Myopathy: A Single-Center Retrospective Cohort Study","authors":"Yikang Wang PhD ,&nbsp;Mengting Yang MD ,&nbsp;Yawen Zhao MD ,&nbsp;Yiming Zheng MD ,&nbsp;Hongjun Hao PhD ,&nbsp;Feng Gao MD ,&nbsp;Hui Xiong MD ,&nbsp;Wei Zhang MD ,&nbsp;Zhaoxia Wang MD ,&nbsp;Yun Yuan MD","doi":"10.1016/j.pediatrneurol.2025.03.002","DOIUrl":"10.1016/j.pediatrneurol.2025.03.002","url":null,"abstract":"<div><h3>Background</h3><div>Immune-mediated necrotizing myopathy (IMNM) is a type of idiopathic inflammatory myopathies (IIMs), and the data concerning the phenotypes of pediatric IMNM are very limited. The present study aimed to elucidate the characteristics of pediatric IMNM.</div></div><div><h3>Methods</h3><div>We examined 116 pediatric patients with IIMs through a muscle biopsy-oriented registration study. Anti-signal recognition particle (anti-SRP) and anti-3-hydroxy-3-methylglutaryl-CoA reductase (anti-HMGCR) antibodies were detected via an immunoblot assay. A retrospective clinical, imaging, and myopathological analysis of 55 pediatric patients with IMNM was conducted.</div></div><div><h3>Results</h3><div>The cohort included 38 girls and 17 boys with a median age of 7 years. Acute/subacute onset occurred in 40.0% and chronic onset in 60.0% of the patients. Proximal and neck weakness were common symptoms. The frequencies of anti-HMGCR, anti-SRP, and seronegative myopathies were 61.8%, 20.0%, and 18.2%, respectively. Chronic onset was more common in anti-HMGCR myopathy than in anti-SRP myopathy (<em>P</em> = 0.003). Thigh magnetic resonance imaging revealed generalized muscle edema, and the severity of fatty infiltration correlated with disease duration. Necrotizing myopathy was most common among patients with seronegative IMNM, followed by patients with anti-HMGCR and anti-SRP myopathies. Dystrophic pathology was most common among patients with anti-SRP myopathy, followed by patients with anti-HMGCR myopathy and seronegative IMNM. After steroids combined with multiple immunosuppressant therapies, 18 of 39 (46.2%) patients achieved complete or partial remission. The percent of complete remission was significantly lower in patients with anti-HMGCR myopathy compared with those with seronegative or anti-HMGCR myopathies (<em>P</em> = 0.030).</div></div><div><h3>Conclusions</h3><div>IMNM is common in Chinese pediatric patients with IIMs. Most patients have anti-HMGCR antibodies, are more commonly female, have chronic onset and proximal weakness, lack other organ manifestations, have disease course-related muscle fatty infiltration, and have a poor response to immunosuppression.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"167 ","pages":"Pages 33-41"},"PeriodicalIF":3.2,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143792745","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}

{"title":"Renewing the Commitment to Diversity in Neurology: Resisting the Backlash","authors":"Joshua A. Budhu ,&nbsp;Diana M. Cejas","doi":"10.1016/j.pediatrneurol.2025.02.006","DOIUrl":"10.1016/j.pediatrneurol.2025.02.006","url":null,"abstract":"","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"165 ","pages":"Pages A6-A7"},"PeriodicalIF":3.2,"publicationDate":"2025-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143619439","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}

{"title":"IMPAIRED CEREBRAL AUTOREGULATION IN CHILDREN","authors":"Carlos Castillo-Pinto MD ,&nbsp;Priscilla Yu MD ,&nbsp;Mark S. Wainwright MD, PhD ,&nbsp;Matthew P. Kirschen MD, PhD","doi":"10.1016/j.pediatrneurol.2025.03.003","DOIUrl":"10.1016/j.pediatrneurol.2025.03.003","url":null,"abstract":"<div><div>Managing acute brain injury involves protecting the brain from secondary injury by addressing the mismatch between metabolic demand and cerebral perfusion. Observational studies have associated impaired cerebral autoregulation, a physiological process governing the regulation of cerebral blood flow, with unfavorable neurological outcomes in both pediatric and adult populations. We review the pathophysiology of cerebral autoregulation and discuss methods for assessing and monitoring it in children after acquired brain injury. We also examine the current research investigating the relationship between impaired cerebral autoregulation and outcomes following traumatic brain injury, cardiac arrest, cardiopulmonary bypass, and extracorporeal membrane oxygenation. Furthermore, we outline potential areas for future research in cerebral autoregulation and its clinical implications for pediatric patients with brain injuries.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"167 ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2025-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143768032","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}

{"title":"Editorial Board and Masthead","authors":"","doi":"10.1016/S0887-8994(25)00055-4","DOIUrl":"10.1016/S0887-8994(25)00055-4","url":null,"abstract":"","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"165 ","pages":"Pages A1-A2"},"PeriodicalIF":3.2,"publicationDate":"2025-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143619436","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}

{"title":"A Postmarketing Pharmacovigilance Study of Fenfluramine: Adverse Event Data Mining and Analysis Based on the US Food and Drug Administration Public Data Open Project (openFDA)","authors":"Tianyu Chen MNS ,&nbsp;Qiying Chen BM ,&nbsp;Yuezhen Zhang BM ,&nbsp;Ting Liu BM","doi":"10.1016/j.pediatrneurol.2025.03.001","DOIUrl":"10.1016/j.pediatrneurol.2025.03.001","url":null,"abstract":"<div><h3>Background</h3><div>A postmarketing analysis of the adverse events (AEs) associated with fenfluramine (FFA) was conducted using the US Food and Drug Administration's Open Public Data Program (openFDA).</div></div><div><h3>Methods</h3><div>The openFDA database was queried to retrieve FFA AE reports. Two algorithms, namely, the reporting odds ratio (ROR) and proportional reporting ratio, were employed for the purpose of detecting potential safety signals.</div></div><div><h3>Results</h3><div>From the openFDA data platform, a total of 6,269,521 AE reports were collected during the study period; the number of AE reports with FFA as the primary suspect was 2386. Of these, 1526 (63.96%) were reported by consumers or non–health professionals, 2009 (84.20%) were reported by the United States, 1053 (44.13%) were unknown indications, and serious AEs were reported in 1315 cases (55.11%). A total of 62 signals were generated. The top 10 signals included atonic seizures (ROR of 918.52, 95% confidence interval [CI]: 670.65-1257.99), seizure clusters (ROR of 787.02, 95% CI: 595.26-1040.56), mitral valve thickening (ROR of 773.94, 95% CI: 463.47-1292.38), pulmonary valve incompetence (ROR of 600.71, 95% CI: 432.09-835.13), echocardiogram abnormal (ROR of 417.13, 95% CI: 307.87-565.16), change in seizure presentation (ROR of 287.55, 95% CI: 214.81-384.91), tricuspid valve incompetence (ROR of 221.42, 95% CI: 179.68-272.84), aortic valve incompetence (ROR of 176.59, 95% CI: 131.89-236.45), tonic convulsion (ROR of 173.68, 95% CI: 110.28-273.54), and myoclonic epilepsy (ROR of 158.05, 95% CI: 102.60-243.46).</div></div><div><h3>Conclusions</h3><div>This study employed the openFDA database to identify safety signals associated with FFA, thereby offering significant insights for clinical monitoring and risk identification in patients undergoing FFA therapy.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"166 ","pages":"Pages 96-102"},"PeriodicalIF":3.2,"publicationDate":"2025-03-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143705752","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}