Pediatric neurology最新文献

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Early Response, Long-Term Seizure Outcome, and Very-Low-Dose Adrenocorticotrophic Hormone Therapy for Infantile Epileptic Spasms Syndrome With Down Syndrome 唐氏综合征 IESS 的早期反应、长期发作结果和极低剂量促肾上腺皮质激素疗法
IF 3.8 3区 医学
Pediatric neurology Pub Date : 2024-05-06 DOI: 10.1016/j.pediatrneurol.2024.04.025
Yuko Hirata MD, PhD , Shin-ichiro Hamano MD, PhD , Daishi Hirano MD, PhD , Ryuki Matsuura MD, PhD , Reiko Koichihara MD , Rikako Takeda MD , Hirokazu Takeuchi MD , Kenjiro Kikuchi MD, PhD
{"title":"Early Response, Long-Term Seizure Outcome, and Very-Low-Dose Adrenocorticotrophic Hormone Therapy for Infantile Epileptic Spasms Syndrome With Down Syndrome","authors":"Yuko Hirata MD, PhD ,&nbsp;Shin-ichiro Hamano MD, PhD ,&nbsp;Daishi Hirano MD, PhD ,&nbsp;Ryuki Matsuura MD, PhD ,&nbsp;Reiko Koichihara MD ,&nbsp;Rikako Takeda MD ,&nbsp;Hirokazu Takeuchi MD ,&nbsp;Kenjiro Kikuchi MD, PhD","doi":"10.1016/j.pediatrneurol.2024.04.025","DOIUrl":"10.1016/j.pediatrneurol.2024.04.025","url":null,"abstract":"<div><h3>Background</h3><p>Infantile epileptic spasms syndrome (IESS) with Down syndrome has good treatment response and good seizure outcomes with high-dose adrenocorticotrophic hormone (ACTH) therapy. We investigated the early treatment response of epileptic spasms (ES), long-term seizure outcome, and efficacy of very-low-dose ACTH therapy for IESS with Down syndrome.</p></div><div><h3>Methods</h3><p>We retrospectively investigated patients with Down syndrome and IESS between April 1983 and January 2023. We defined response to treatment as clinical remission and electrographic resolution of hypsarrhythmia after treatment for more than one month and early treatment as any treatment for ES within three months of initiation of treatment. Long-term seizure outcomes were determined by the presence of any type of seizure within one year of the last visit. We investigated the dosage and efficacy of very-low-dose ACTH therapy.</p></div><div><h3>Results</h3><p>Thirty patients were enrolled with a median follow-up period of 7.7 years (range: 1.3 to 19.1). The response and relapse rates in the early treatment were 83.3% and 16.0%, respectively. The seizure-free rate of long-term seizure outcomes was 80.0%. Long-term seizure outcomes correlated with early treatment response to ES. The response rate of very-low-dose ACTH therapy was 59.3%. The efficacy of ACTH therapy tended to be dose-dependent (<em>P</em> = 0.055).</p></div><div><h3>Conclusions</h3><p>Early treatment response to ES may be useful in predicting long-term seizure outcomes of IESS with Down syndrome. Very-low-dose ACTH therapy was the most effective treatment for ES and could exhibit dose-dependent efficacy. Depending on the IESS etiology, the ACTH dose could be reduced to minimize its side effects.</p></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141051245","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}
引用次数: 0
Long-Term Quality of Life After Pediatric Traumatic Brain Injury Treated in the Intensive Care Unit 在重症监护室治疗小儿脑外伤后的长期生活质量
IF 3.8 3区 医学
Pediatric neurology Pub Date : 2024-05-06 DOI: 10.1016/j.pediatrneurol.2024.04.030
Elina Kyösti MD , Era Mikkonen MD , Rahul Raj MD , Pasi Ohtonen MSci , Outi Peltoniemi MD , Markus B. Skrifvars MD , Tero Ala-Kokko MD
{"title":"Long-Term Quality of Life After Pediatric Traumatic Brain Injury Treated in the Intensive Care Unit","authors":"Elina Kyösti MD ,&nbsp;Era Mikkonen MD ,&nbsp;Rahul Raj MD ,&nbsp;Pasi Ohtonen MSci ,&nbsp;Outi Peltoniemi MD ,&nbsp;Markus B. Skrifvars MD ,&nbsp;Tero Ala-Kokko MD","doi":"10.1016/j.pediatrneurol.2024.04.030","DOIUrl":"10.1016/j.pediatrneurol.2024.04.030","url":null,"abstract":"<div><h3>Background</h3><p>To examine the long-term health-related quality of life (HRQL) after pediatric traumatic brain injury (TBI) treated in the intensive care unit (ICU).</p></div><div><h3>Methods</h3><p>This retrospective cohort study was conducted using data from four university hospital ICUs in Finland. Children aged &lt; 18 years with TBI treated in the ICU during 2003 to 2013 were included. Patients alive at the end of 2020 were sent two different HRQL questionnaires 15/16-dimensional (15D/16D) and RAND-36  and questions regarding chronic diseases, socioeconomical status, and the need for health care support. HRQL was defined as poor when the 15D/16D score total score was below the age- and sex-matched mean population score in Finland minus the minimal clinically important difference.</p></div><div><h3>Results</h3><p>A total of 150 of 337 (44%) patients responded (n = 144 15D/16D responses). Median follow-up time was 11 years. Seventy patients (49%) had a poor HRQL according to 15D/16D score. Patients with TBI had significantly poorer 15D scores in every dimension when compared with the matched population mean values. A higher Helsinki CT score, mechanical ventilation, and female sex were associated with poor long-term HRQL according to the 15D/16D. Patients with poor 15D/16D scores also needed significantly more health care services and medications and had more comorbidities than patients with normal scores. A poor 15D/16D score was associated with lower socioeconomic status.</p></div><div><h3>Conclusions</h3><p>Half of long-term pediatric ICU-treated TBI survivors had poor HRQL 11 years after injury. More severe head computed tomographic findings at admission and female sex associated with poor HRQL.</p></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0887899424001802/pdfft?md5=3426459f6eb339e900462f58fded00d1&pid=1-s2.0-S0887899424001802-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141056133","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}
引用次数: 0
Assessing the Role of Asthma on the Relationship between Neurodevelopmental Disabilities and Adverse Birth Outcomes 评估哮喘对神经发育障碍和不良出生结果之间关系的作用
IF 3.8 3区 医学
Pediatric neurology Pub Date : 2024-05-03 DOI: 10.1016/j.pediatrneurol.2024.04.023
Omobola Oluwafemi MPH , Sneha Manoharan BS , Luyu Xie PharmD, PhD , George Pro PhD , Rikinkumar S. Patel MD , George L. Delclos MD, PhD , Andrew Gelfand MD , Sarah E. Messiah PhD , David S. Lopez DrPH , Jenil Patel MBBS, MPH, PhD
{"title":"Assessing the Role of Asthma on the Relationship between Neurodevelopmental Disabilities and Adverse Birth Outcomes","authors":"Omobola Oluwafemi MPH ,&nbsp;Sneha Manoharan BS ,&nbsp;Luyu Xie PharmD, PhD ,&nbsp;George Pro PhD ,&nbsp;Rikinkumar S. Patel MD ,&nbsp;George L. Delclos MD, PhD ,&nbsp;Andrew Gelfand MD ,&nbsp;Sarah E. Messiah PhD ,&nbsp;David S. Lopez DrPH ,&nbsp;Jenil Patel MBBS, MPH, PhD","doi":"10.1016/j.pediatrneurol.2024.04.023","DOIUrl":"10.1016/j.pediatrneurol.2024.04.023","url":null,"abstract":"<div><h3>Background</h3><p>Investigating asthma as an effect modifier between adverse birth outcomes and neurodevelopmental disabilities (NDDs) across different races is crucial for tailored interventions and understanding variable susceptibility among diverse populations.</p></div><div><h3>Methods</h3><p>Data were collected through the National Survey of Children’s Health. This cross-sectional study included 131,774 children aged 0 to 17 years. Study exposures comprised adverse birth outcomes including preterm birth and low birth weight. Weighted prevalence estimates and odds ratios with 95% confidence intervals (CIs) among children with and without adverse birth outcomes were calculated for NDDs including attention-deficit/hyperactivity disorder, autism spectrum disorder, cerebral palsy, seizure, and several others including behavior problems. Adjusted odds ratios were stratified by asthma status and separate interactions were assessed for each outcome.</p></div><div><h3>Results</h3><p>Of 131,774 participants, 10,227 were born low birth weight (9.12%; 95% CI: 8.77% to 9.49%), 14,058 were born preterm (11.35%; 95% CI: 10.94% to 11.76%), and 16,166 participants had asthma (11.97%; 95% CI: 11.58% to 12.37%). There were 68,100 males (51.11%), 63,674 females (48.89%), 102,061 non-Hispanic Whites (NHW) (66.92%), 8,672 non-Hispanic Blacks (NHB) (13.97%), and 21,041 participants (19.11%) categorized as other. NHB children with adverse birth outcomes had higher prevalence of several NDDs compared to NHW children.</p></div><div><h3>Conclusions</h3><p>Asthma was not shown to be an effect modifier of the association between adverse birth outcomes and NDDs. Nevertheless, these results suggest that NDDs are more prevalent within US children with adverse birth outcomes, with higher rates among NHB compared to NHW children. These findings support screening for NDDs in pediatric health care settings among patients with adverse birth outcomes, particularly among those from ethnic minority backgrounds.</p></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-05-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141026774","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}
引用次数: 0
Pediatric Neuroglial Tumors: A Review of Ependymoma and Dysembryoplastic Neuroepithelial Tumor 小儿神经胶质瘤:上皮瘤和胚胎发育不全神经上皮瘤综述
IF 3.8 3区 医学
Pediatric neurology Pub Date : 2024-05-03 DOI: 10.1016/j.pediatrneurol.2024.04.022
Melissa Arfuso MD , Sandeepkumar Kuril MD , Harshal Shah , Derek Hanson MD
{"title":"Pediatric Neuroglial Tumors: A Review of Ependymoma and Dysembryoplastic Neuroepithelial Tumor","authors":"Melissa Arfuso MD ,&nbsp;Sandeepkumar Kuril MD ,&nbsp;Harshal Shah ,&nbsp;Derek Hanson MD","doi":"10.1016/j.pediatrneurol.2024.04.022","DOIUrl":"10.1016/j.pediatrneurol.2024.04.022","url":null,"abstract":"","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-05-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141055685","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}
引用次数: 0
Helicobacter pylori Infection in Children With Cerebral Palsy: A Cross-Sectional Study 脑瘫儿童的幽门螺杆菌感染:横断面研究
IF 3.8 3区 医学
Pediatric neurology Pub Date : 2024-05-03 DOI: 10.1016/j.pediatrneurol.2024.04.024
Sally M. Ismael MBBCH , Dina S. Atia MD , Salwa A. Atlam MD , Abeer A. Eltoukhy MD , Amira Hamed Darwish MD
{"title":"Helicobacter pylori Infection in Children With Cerebral Palsy: A Cross-Sectional Study","authors":"Sally M. Ismael MBBCH ,&nbsp;Dina S. Atia MD ,&nbsp;Salwa A. Atlam MD ,&nbsp;Abeer A. Eltoukhy MD ,&nbsp;Amira Hamed Darwish MD","doi":"10.1016/j.pediatrneurol.2024.04.024","DOIUrl":"10.1016/j.pediatrneurol.2024.04.024","url":null,"abstract":"<div><h3>Background</h3><p>Children with cerebral palsy (CP) frequently have associated disorders and complications, including gastrointestinal problems. <em>Helicobacter pylori</em> is a common infection worldwide, frequently associated with gastrointestinal manifestations.</p></div><div><h3>Methods</h3><p>To estimate the prevalence of <em>H</em>. <em>pylori</em> infection in children with CP<em>,</em> a cross-sectional study over an eight-month period was performed in the pediatric neurology outpatient clinic of Tanta University Hospital. The study included 100 patients with CP aged two to 17 years. All patients were tested for <em>H. pylori</em> antigen in stool by enzyme-linked immunosorbent assay.</p></div><div><h3>Results</h3><p>The mean age of studied children with CP was 7.03 ± 4.1 years; there were 57 males and 43 females. Spastic quadriplegic CP was the most common type of CP (34%). Forty-five children with CP were positive for <em>H. pylori</em> antigen in stool. Intellectual disability (ID), low sociodemographic scoring system, semisolid diet, and Eating and Drinking Ability Classification System (EDACS) levels 4 and 5 were significant predictors of <em>H. pylori</em> infection (odds ratio of 1.86, 2.63, 12, and 1.77, respectively, <em>P</em> &lt; 0.05). Vomiting, abdominal pain, and gastrointestinal tract bleeding were significantly more frequent in <em>H. pylori</em>-infected children with CP than noninfected children with CP (<em>P</em> value &lt; 0.05)</p></div><div><h3>Conclusion</h3><p><em>H. pylori</em> is a relatively common infection among children with CP. The main risk factors for <em>H. pylori</em> infection were low socioeconomic level, ID, semisolid diet, and EDACS levels 4 and 5.</p></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-05-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141035197","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}
引用次数: 0
Enterovirus-Related Parainfectious Acute Disseminated Encephalomyelitis Presenting With Dorsal Midbrain Syndrome: A Pediatric Case Report 肠道病毒相关副感染性急性播散性脑脊髓炎伴中脑背侧综合征:儿科病例报告。
IF 3.8 3区 医学
Pediatric neurology Pub Date : 2024-04-26 DOI: 10.1016/j.pediatrneurol.2024.04.017
H. Shafeeq Ahmed MBBS, Chinmayee J. Thrishulamurthy MS
{"title":"Enterovirus-Related Parainfectious Acute Disseminated Encephalomyelitis Presenting With Dorsal Midbrain Syndrome: A Pediatric Case Report","authors":"H. Shafeeq Ahmed MBBS,&nbsp;Chinmayee J. Thrishulamurthy MS","doi":"10.1016/j.pediatrneurol.2024.04.017","DOIUrl":"10.1016/j.pediatrneurol.2024.04.017","url":null,"abstract":"","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-04-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140958753","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}
引用次数: 0
Investigating Associations Between Nonadherence to Guideline-Recommended Treatment of Pediatric Seizures and Adverse Outcomes: A Canadian Feasibility Study 调查不遵守指南推荐的小儿癫痫发作治疗与不良后果之间的关系:加拿大可行性研究。
IF 3.8 3区 医学
Pediatric neurology Pub Date : 2024-04-26 DOI: 10.1016/j.pediatrneurol.2024.04.021
Meaghan Moreau MD , Helen Coo MSc , Niveditha Pattathil BHSc , Vinay Kukreti MD , Steven C. Brooks MD , Anupam Sehgal MD
{"title":"Investigating Associations Between Nonadherence to Guideline-Recommended Treatment of Pediatric Seizures and Adverse Outcomes: A Canadian Feasibility Study","authors":"Meaghan Moreau MD ,&nbsp;Helen Coo MSc ,&nbsp;Niveditha Pattathil BHSc ,&nbsp;Vinay Kukreti MD ,&nbsp;Steven C. Brooks MD ,&nbsp;Anupam Sehgal MD","doi":"10.1016/j.pediatrneurol.2024.04.021","DOIUrl":"10.1016/j.pediatrneurol.2024.04.021","url":null,"abstract":"<div><h3>Background</h3><p>Emerging evidence suggests that nonadherence to treatment guidelines for seizures may affect patient outcomes. We examined the feasibility of conducting a larger investigation to test this hypothesis in the pediatric population.</p></div><div><h3>Methods</h3><p>We retrospectively reviewed charts of patients aged ≤18 years who presented with seizure to the emergency departments of two Ontario hospitals in 2019 to 2021. Patients were grouped by seizure duration (&lt;5 minutes [n = 37], ≥5 minutes [n = 41]). We examined nonadherence to guideline-recommended treatment, adverse outcomes (hospitalization, length of stay, respiratory complications), and missing values for key variables.</p></div><div><h3>Results</h3><p>Of 78 patients, 34 (44%) did not receive guideline-recommended treatment. Nonadherence was similar in the two groups (&lt;5 minutes: 46%; ≥5 minutes: 41%). Common deviations included administering an antiseizure medication (ASM) for seizures of less than five minutes (46%), a delay (&gt;10 minutes) between the first and second ASM doses (50%), and use of a benzodiazepine for the third dose (45%). Hospitalizations were common in both seizure duration groups (∼90%), whereas respiratory complications were relatively rare. Time of seizure onset was missing in 51% of charts, and none contained the time of first contact with emergency services when patients were transported by ambulance.</p></div><div><h3>Conclusion</h3><p>We found evidence of substantial nonadherence to guideline-recommended treatment of pediatric seizures. Medical records do not contain sufficient information to comprehensively investigate this issue. A multicenter prospective study is the most feasible option to examine the association between nonadherence to guideline-recommended treatment and patient outcomes.</p></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-04-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140958755","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}
引用次数: 0
Sensory Symptoms Across the Lifespan in People With Cerebral Palsy 大脑性麻痹患者一生中的感觉症状。
IF 3.2 3区 医学
Pediatric neurology Pub Date : 2024-04-26 DOI: 10.1016/j.pediatrneurol.2024.04.019
Ariel M. Lyons-Warren MD, PhD , Danielle Guez-Barber MD, PhD , Sruthi P. Thomas MD, PhD , Evelyne K. Tantry , Aditya Mahat MBBS , Bhooma Aravamuthan MD, DPhil , Cerebral Palsy Research Network
{"title":"Sensory Symptoms Across the Lifespan in People With Cerebral Palsy","authors":"Ariel M. Lyons-Warren MD, PhD ,&nbsp;Danielle Guez-Barber MD, PhD ,&nbsp;Sruthi P. Thomas MD, PhD ,&nbsp;Evelyne K. Tantry ,&nbsp;Aditya Mahat MBBS ,&nbsp;Bhooma Aravamuthan MD, DPhil ,&nbsp;Cerebral Palsy Research Network","doi":"10.1016/j.pediatrneurol.2024.04.019","DOIUrl":"10.1016/j.pediatrneurol.2024.04.019","url":null,"abstract":"<div><h3>Background</h3><p>To estimate the prevalence of sensory symptoms in people with cerebral palsy (CP) across the lifespan.</p></div><div><h3>Methods</h3><p>In this cross-sectional study, the self-reported Sensory Processing Scale Inventory (SPS-I) was administered via Research Electronic Data Capture (REDCap) between February 1, 2022, and August 15, 2022, to people with CP or their caregivers enrolled in the online MyCP Community Registry. We determined the association between SPS-I scores and age (Pearson correlation) and functional status as assessed using five validated functional classification systems for CP (analysis of variance [ANOVA]). We hypothesized that sensory symptoms would differ between younger and older individuals with CP.</p></div><div><h3>Results</h3><p>Of 155 responses (28% response rate, age one to 76 years, 34% male), 97% reported at least one bothersome sensory symptom. Total sensory symptoms decreased with age (R<sup>2</sup> = 0.12, <em>P</em> &lt; 0.0001), driven by decreases in hyposensitivity symptoms (R<sup>2</sup> = 0.32, <em>P</em> &lt; 0.0001), primarily tactile hyposensitivity (R<sup>2</sup> = 0.29, <em>P</em> &lt; 0.0001). Sensory symptoms increased with greater functional impairment across all functional domains (ANOVA, <em>P</em> &lt; 0.0001). However, the age-specific decrease in hyposensitivities was most pronounced in people with the greatest gross motor functional impairment (R<sup>2</sup> = 0.70, <em>P</em> = 0.0004).</p></div><div><h3>Conclusion</h3><p>Our findings suggest that hyposensitivity, primarily tactile sensitivity, decreases with age in people with CP. Future work should assess whether decreased hyposensitivity contributes to other age-related changes in CP like increased pain.</p></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2024-04-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141458604","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}
引用次数: 0
Eculizumab in Adolescent Patients With Refractory Generalized Myasthenia Gravis: A Phase 3, Open-Label, Multicenter Study 易库珠单抗治疗难治性全身性肌无力青少年患者:一项开放标签、多中心 3 期研究
IF 3.8 3区 医学
Pediatric neurology Pub Date : 2024-04-26 DOI: 10.1016/j.pediatrneurol.2024.04.020
John F. Brandsema MD , Matthew Ginsberg MD , Hideki Hoshino MD , Masakazu Mimaki MD, PhD , Satoru Nagata MD , Vamshi K. Rao MD , Katherine Ruzhansky MD , Niraja Suresh MD , Emmanuelle Tiongson MD , Hideo Yamanouchi MD , Glen Frick MD, PhD , Eden Hicks PhD , Serena Liao PhD , James F. Howard Jr. MD , MG 303 Study Group
{"title":"Eculizumab in Adolescent Patients With Refractory Generalized Myasthenia Gravis: A Phase 3, Open-Label, Multicenter Study","authors":"John F. Brandsema MD ,&nbsp;Matthew Ginsberg MD ,&nbsp;Hideki Hoshino MD ,&nbsp;Masakazu Mimaki MD, PhD ,&nbsp;Satoru Nagata MD ,&nbsp;Vamshi K. Rao MD ,&nbsp;Katherine Ruzhansky MD ,&nbsp;Niraja Suresh MD ,&nbsp;Emmanuelle Tiongson MD ,&nbsp;Hideo Yamanouchi MD ,&nbsp;Glen Frick MD, PhD ,&nbsp;Eden Hicks PhD ,&nbsp;Serena Liao PhD ,&nbsp;James F. Howard Jr. MD ,&nbsp;MG 303 Study Group","doi":"10.1016/j.pediatrneurol.2024.04.020","DOIUrl":"https://doi.org/10.1016/j.pediatrneurol.2024.04.020","url":null,"abstract":"<div><h3>Background</h3><p>This study evaluated the efficacy and safety of eculizumab, a terminal complement C5 inhibitor, in juvenile generalized myasthenia gravis (gMG).</p></div><div><h3>Methods</h3><p>Adolescents aged 12 to 17 years with refractory anti-acetylcholine receptor (AChR) antibody-positive gMG received eculizumab (weekly induction [one to two doses of 600 mg or four doses of 900 mg] followed by maintenance doses [300 to 1200 mg] every two weeks for up to 26 weeks) in a phase 3, open-label multicenter study (NCT03759366). Change from baseline to week 26 in Quantitative Myasthenia Gravis (QMG) total score (primary end point) and secondary end points including Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score, Myasthenia Gravis Composite score, Myasthenia Gravis Foundation of America postintervention status, EuroQol 5-Dimensions (Youth) and Neurological Quality-of-Life Pediatric Fatigue questionnaire scores, as well as pharmacokinetics, pharmacodynamics, and safety, were recorded.</p></div><div><h3>Results</h3><p>Eleven adolescents (mean ± S.D. age 14.8 ± 1.8 years) were enrolled; 10 completed the primary evaluation period. Least-squares mean changes from baseline at week 26 were −5.8 (standard error [SE] 1.2; <em>P</em> = 0.0004) for QMG total score and −2.3 (SE 0.6; <em>P</em> = 0.0017) for MG-ADL total score. Overall, the primary and all secondary efficacy end point analyses met statistical significance from the first assessment and were sustained throughout. Complete terminal complement inhibition was sustained through 26 weeks in all patients. Treatment-emergent adverse events were all mild/moderate and predominantly unrelated to eculizumab.</p></div><div><h3>Conclusions</h3><p>Eculizumab was effective in reducing disease burden and was well tolerated in adolescents with refractory AChR antibody-positive gMG.</p></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-04-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0887899424001498/pdfft?md5=43c45e41d4d41443a7568c4e94f1bbc9&pid=1-s2.0-S0887899424001498-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141163797","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}
引用次数: 0
PRESenting a Challenge: Posterior Reversible Encephalopathy Syndrome in Pediatric Patients With Guillain-Barré Syndrome: A Case Series and Review of Literature 迎接挑战:吉兰-巴雷综合征(GBS)儿科患者的后可逆性脑病综合征(PRES)病例系列和文献综述
IF 3.8 3区 医学
Pediatric neurology Pub Date : 2024-04-25 DOI: 10.1016/j.pediatrneurol.2024.04.018
Rohini M. Surve MD, PDF , Kunal K. Sharma DM , Prachi Sharma DM , Roshan Nisal DM , Hima S. Pendharkar DM , Girish B. Kulkarni DM
{"title":"PRESenting a Challenge: Posterior Reversible Encephalopathy Syndrome in Pediatric Patients With Guillain-Barré Syndrome: A Case Series and Review of Literature","authors":"Rohini M. Surve MD, PDF ,&nbsp;Kunal K. Sharma DM ,&nbsp;Prachi Sharma DM ,&nbsp;Roshan Nisal DM ,&nbsp;Hima S. Pendharkar DM ,&nbsp;Girish B. Kulkarni DM","doi":"10.1016/j.pediatrneurol.2024.04.018","DOIUrl":"10.1016/j.pediatrneurol.2024.04.018","url":null,"abstract":"<div><h3>Background</h3><p>Guillain-Barré syndrome (GBS) is an autoimmune disorder characterized by demyelination of peripheral nerves. GBS-associated posterior reversible encephalopathy syndrome (PRES) is a rare and potentially life-threatening complication in the pediatric population. We aimed to report and analyze the clinical features, management, and outcomes of three cases of GBS-associated PRES in our setting in the light of the existing literature.</p></div><div><h3>Methods</h3><p>Medical records of 75 pediatric patients with GBS were reviewed for autonomic changes and GBS-associated PRES. Thirty-one developed dysautonomia while three were identified to have PRES. Clinical, radiological, laboratory, and treatment data were collected and analyzed.</p></div><div><h3>Results</h3><p>All three patients were male and presented with symptoms of acute flaccid paralysis and respiratory distress requiring mechanical ventilation. All three patients experienced various complications, including hypertension, seizures, and hyponatremia, and were subsequently diagnosed with PRES. Multimodal intensive care resulted in patient improvement and discharge in an ambulatory state after an average of 104 days of care.</p></div><div><h3>Conclusions</h3><p>GBS-associated PRES is a rare and potentially life-threatening complication that can occur in pediatric patients with GBS. Our findings suggest that early recognition, prompt intervention, and multimodal intensive care can improve patient outcomes. Further studies are needed to determine optimal treatment strategies for GBS-associated PRES.</p></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140783242","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}
引用次数: 0
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