Pediatric neurologyPub Date : 2025-01-10DOI: 10.1016/j.pediatrneurol.2025.01.004
Lisa Pabst MD , Melissa Chung MD , Thomas Murray DO , Melissa Hutchinson MD , Melica Nikahd MS , Guy Brock PhD , Alicia Zha MD , Warren Lo MD
{"title":"Is There a SPOT for Remote Pediatric Stroke Evaluation in the World of Telestroke Networks? A Pilot Feasibility Study of Performing the Pediatric National Institutes of Health Stroke Scale Over Televideo","authors":"Lisa Pabst MD , Melissa Chung MD , Thomas Murray DO , Melissa Hutchinson MD , Melica Nikahd MS , Guy Brock PhD , Alicia Zha MD , Warren Lo MD","doi":"10.1016/j.pediatrneurol.2025.01.004","DOIUrl":"10.1016/j.pediatrneurol.2025.01.004","url":null,"abstract":"<div><h3>Background</h3><div>Telestroke assessments are widely used to remotely assess adults with suspected stroke, although they have not been studied in children. SPOT, the Study of Performing the PedNIHSS Over Televideo, tested the feasibility of assessing the Pediatric National Institutes of Health Stroke Scale (PedNIHSS) by televideo in children.</div></div><div><h3>Methods</h3><div>Children aged 2 to 17 years with and without strokes were recruited and examined in the outpatient neurology clinic. The PedNIHSS was assessed by separate neurologists via televideo and at the bedside. Intraclass correlation coefficients (ICCs) were used to analyze inter-rater reliability.</div></div><div><h3>Results</h3><div>Twenty children were recruited; mean age was 9.2 years (range, 2 to 17 years). Six had chronic stroke. By bedside examination, the total PedNIHSS score ranged from 0 to 8, with a mean of 1.65. By televideo, the PedNIHSS was identical to the bedside examination in 12 of 20 (60%) of the children and identical or within 1 point in 19 of 20 (95%) with excellent overall inter-rater reliability (ICC = 0.92; 95% confidence interval: 0.81, 0.97). The mean time to complete bedside and remote examinations was 5 and 7 minutes, respectively.</div></div><div><h3>Conclusions</h3><div>Performing the PedNIHSS over televideo is feasible, accurate, and requires similar time as a bedside evaluation. Limitations of this study include a small sample size and the overall low burden of neurological deficits. Future studies assessing the reliability of performing the PedNIHSS over televideo should include children with acute neurological deficits in the acute care setting across multiple sites.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"164 ","pages":"Pages 72-77"},"PeriodicalIF":3.2,"publicationDate":"2025-01-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143059663","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":"Early Corticosteroid Use and Short-Term Outcomes in Pediatric Bacterial Meningitis: A Nationwide Study in Japan, 2014 to 2022","authors":"Satoko Kameda MD , Hayato Yamana MD, MPH, PhD , Yusuke Sasabuchi MD, MPH, PhD , Nobuaki Michihata MD, MPH, PhD , Shotaro Aso MD, MPH, PhD , Hiroki Matsui MPH, PhD , Kiyohide Fushimi MD, PhD , Hideo Yasunaga MD, PhD , Takahide Kohro MD, PhD","doi":"10.1016/j.pediatrneurol.2025.01.002","DOIUrl":"10.1016/j.pediatrneurol.2025.01.002","url":null,"abstract":"<div><h3>Background</h3><div>The benefit of early corticosteroid use in pediatric bacterial meningitis is uncertain, except for its effect on hearing loss caused by <em>Haemophilus influenzae</em> type b (Hib) meningitis. We aimed to evaluate the association between early corticosteroid use and the short-term outcomes amid a background of decreased Hib infection incidence.</div></div><div><h3>Methods</h3><div>We conducted a retrospective cohort study using data from a nationwide inpatient database in Japan. We identified children younger than 15 years with bacterial meningitis who were discharged between April 2014 and March 2022. The primary outcome was a composite of in-hospital death and neurological sequelae, defined as tracheostomy during hospitalization or disturbed consciousness, mechanical ventilation, tube feeding, or antiepileptic drugs at discharge. The secondary outcome was a recorded diagnosis of hearing loss. Stabilized inverse probability of treatment weighting using propensity scores was performed to compare the outcomes between patients with and without intravenous corticosteroid use on the day of admission.</div></div><div><h3>Results</h3><div>Of the 1310 eligible patients, 454 (35%) received early corticosteroids. Overall, 170 patients (13%) had composite outcomes, including seven deaths (0.5%). Hearing loss was observed in seven patients. After propensity score weighting, no significant difference was observed between patients with and without early corticosteroid use in the composite outcome (14.2% vs 13.5%, respectively; risk difference: 0.7%; 95% confidence interval: −3.3% to 4.6%) or hearing loss (0.7% vs 0.5%, respectively; risk difference: 0.2%; 95% confidence interval: −0.7% to 1.2%).</div></div><div><h3>Conclusions</h3><div>Early corticosteroid use in children with bacterial meningitis was not associated with reduced deaths or neurological sequelae.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"164 ","pages":"Pages 97-104"},"PeriodicalIF":3.2,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143075294","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}
Pediatric neurologyPub Date : 2025-01-09DOI: 10.1016/j.pediatrneurol.2024.12.012
Zhirong Wei MM , Tinghong Liu MM , Suhui Kuang MD , Jiaqi Wang MM , Shuli Liang PhD
{"title":"Effectiveness of Vagus Nerve Stimulation in Patients With Dravet Syndrome: A Case Series and Meta-Analysis","authors":"Zhirong Wei MM , Tinghong Liu MM , Suhui Kuang MD , Jiaqi Wang MM , Shuli Liang PhD","doi":"10.1016/j.pediatrneurol.2024.12.012","DOIUrl":"10.1016/j.pediatrneurol.2024.12.012","url":null,"abstract":"<div><h3>Objective</h3><div>This case series and meta-analysis aimed to determine effectiveness of vagus nerve stimulation (VNS) in patients with Dravet syndrome (DS).</div></div><div><h3>Methods</h3><div>All patients with DS and VNS were included in this case series. For meta-analysis, a literature search of electronic databases PubMed, Cochrane, and Embase was performed. Critical Appraisal Tools were used to evaluate study quality. Responder (>50% seizure reduction from baseline) rate was the main outcome, which was quantitatively synthesized at specific follow-up points (at least six months). Subgroup analyses were conducted based on duration of epilepsy, proportion of males, and duration of follow-up. Additionally, cognitive and behavior changes were analyzed as secondary outcomes in case series only.</div></div><div><h3>Results</h3><div>Of the case series, two of four patients responded. In the meta-analysis, of 1183 studies retrieved in the initial search, nine comprising 92 patients were included. The overall responder rate reached 0.55 (95% confidence interval [CI] 0.45 to 0.64). Subgroup analyses were performed. The responder rates in groups with duration of epilepsy groups greater than or equal to six years and less than six years were 0.38 (95% CI 0.14 to 0.61) and 0.65 (95% CI 0.47 to 0.82) (<em>P</em> = 0.07). No heterogeneity was found in responder rate in groups with different proportion of males (>50%, ≤50%) or two groups with different duration of follow-up (greater than or equal to three years, less than three years) (<em>P</em> > 0.1).</div></div><div><h3>Conclusions</h3><div>VNS was an effective option in patients with DS who failed to respond to medications. Patients with short duration of epilepsy (less than six years) were prone to respond to VNS.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"164 ","pages":"Pages 105-114"},"PeriodicalIF":3.2,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143075296","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}
Pediatric neurologyPub Date : 2025-01-09DOI: 10.1016/j.pediatrneurol.2024.12.017
Liping Yu MM , Yanlin Li MM , Hanxue Yang PhD , Yonghua Cui PhD , Ying Li PhD
{"title":"The Premonitory Urge to Tic in Children and Adolescents: Measuring, Describing, and Correlating","authors":"Liping Yu MM , Yanlin Li MM , Hanxue Yang PhD , Yonghua Cui PhD , Ying Li PhD","doi":"10.1016/j.pediatrneurol.2024.12.017","DOIUrl":"10.1016/j.pediatrneurol.2024.12.017","url":null,"abstract":"<div><h3>Background</h3><div>This study aimed to explore the premonitory urges (PUs) experienced by children with tic disorders (TDs), with the aim of describing and correlating these urges with various factors.</div></div><div><h3>Methods</h3><div>First-episode and drug-naive patients with TDs were recruited. We conducted a comprehensive study utilizing the Premonitory Urge for Tics Scale to measure the severity of PUs. Regression analysis was performed to explore the relationships between PUs and other relevant factors, such as demographic characteristics, tic severity, and comorbidities.</div></div><div><h3>Results</h3><div>The linear regression model revealed that age (<em>β</em> = 0.090, <em>P</em> = 0.004), the severity of motor tics (<em>β</em> = 0.112, <em>P</em> < 0.001), the severity of vocal tics (<em>β</em> = 0.074, <em>P</em> = 0.020), and the severity of tic-related impairments (<em>β</em> = 0.112, <em>P</em> = 0.001) were significant predictors of PUs.</div></div><div><h3>Conclusions</h3><div>This study provides insights into the nature of PUs in children with TD. Future research should focus on PUs across different age groups and develop and evaluate targeted treatments that aim to reduce the severity of tics.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"164 ","pages":"Pages 66-71"},"PeriodicalIF":3.2,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143047366","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}
Pediatric neurologyPub Date : 2025-01-07DOI: 10.1016/j.pediatrneurol.2025.01.005
Roger Esmel-Vilomara MD , Eulàlia Turón-Viñas MD, PhD , Marta Pujol-Sanjuan MD , Simona Giorgi MD, PhD , María Pérez-Fernández RN , Alejandra Pérez-Restrepo RN, PhD , José Ángel Aibar , Susana Boronat MD, PhD
{"title":"Cardiac Implications in Dravet Syndrome: Can Electrocardiogram and Echocardiography Detect Hidden Risks?","authors":"Roger Esmel-Vilomara MD , Eulàlia Turón-Viñas MD, PhD , Marta Pujol-Sanjuan MD , Simona Giorgi MD, PhD , María Pérez-Fernández RN , Alejandra Pérez-Restrepo RN, PhD , José Ángel Aibar , Susana Boronat MD, PhD","doi":"10.1016/j.pediatrneurol.2025.01.005","DOIUrl":"10.1016/j.pediatrneurol.2025.01.005","url":null,"abstract":"<div><h3>Background</h3><div>Dravet syndrome (DS) is a severe developmental and epileptic encephalopathy associated with loss-of-function variants in the <em>SCN1A</em> gene. Although predominantly expressed in the central nervous system, <em>SCN1A</em> is also expressed in the heart, suggesting a potential link between neuronal and cardiac channelopathies. Additionally, DS carries a high risk of sudden unexpected death in epilepsy (SUDEP). This study investigates electrocardiographic (EKG) and echocardiographic findings in patients with DS to assess potential cardiac risks.</div></div><div><h3>Methods</h3><div>This prospective study recruited 34 patients with DS with confirmed <em>SCN1A</em> pathogenic variants during the 2024 family meeting of the Dravet Syndrome Foundation Spain. Participants underwent standard 12-lead EKG, high-lead EKG for Brugada pattern detection, and a standing test to evaluate QT interval response. When available, echocardiogram data were collected. QTc and P wave dispersion were calculated. To establish a basis for comparison, cases were matched with age- and sex-matched epileptic patients without DS.</div></div><div><h3>Results</h3><div>No significant EKG abnormalities suggesting long QT syndrome or Brugada syndrome were detected. However, QT and P wave dispersion, previously reported as markers of autonomic dysfunction associated with arrhythmias and SUDEP risk, were elevated. Echocardiograms in 21 patients showed normal cardiac structure, even in those on fenfluramine.</div></div><div><h3>Conclusions</h3><div>Although no significant EKG or echocardiographic abnormalities were identified, elevated QTc and P wave dispersion, along with the elevated risk of SUDEP and past reports of arrhythmias, suggest the need for continued cardiac surveillance. Further studies are essential to explore the predictive value of QTc and P wave dispersion in assessing SUDEP and arrhythmia risk, and to identify other potential cardiac markers.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"164 ","pages":"Pages 53-57"},"PeriodicalIF":3.2,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143040918","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":"General Movements as Predictive Tool of Neurological Outcomes in Term-Born Infants With Hypoxic-Ischemic Encephalopathy at Ages Six and 12 Months","authors":"Rudresh Naik MD Pediatrics (Resident) , Lokesh Saini MD Pediatrics, DM Pediatric Neurology (Associate Professor) , Christa Einspieler PhD (Professor of Physiology) , Pradeep Kumar Gunasekaran MD Pediatrics (Resident) , Kanya Mukhopadhyay MD Pediatrics, DM Neonatology (Professor) , Prahbhjot Malhi PhD (Professor) , Arushi Gahlot Saini MD Pediatrics, DM Pediatric Neurology (Associate Professor) , Rajni Sharma PhD (Child Psychologist) , Naveen Sankhyan MD Pediatrics, DM Pediatric Neurology (Professor)","doi":"10.1016/j.pediatrneurol.2025.01.003","DOIUrl":"10.1016/j.pediatrneurol.2025.01.003","url":null,"abstract":"<div><h3>Background</h3><div>To explore the utility of general movements assessment as a predictive tool of the neurological outcome in term-born infants with hypoxic-ischemic encephalopathy (HIE) at ages six and 12 months.</div></div><div><h3>Methods</h3><div>This prospective observational study was conducted for 18 months (August 2018 to December 2019). Term-born newborns with HIE were included. General movements videos were recorded at 10 to 14 weeks (50 to 54 weeks postmenstrual age) and assessed. Further development was assessed with Developmental Assessment Scales for Indian Infants (DASII) and Ages and Stages Questionnaire, third edition, (ASQ-3) at ages six and 12 months.</div></div><div><h3>Results</h3><div>A total of 30 children were enrolled, 19 (63%) had normal fidgety movements (FMs) and 11 (37%) had absent FMs. Motor Optimality Score, Revised, (MOS-R) ranged from 6 to 8 in 11 infants with absent FMs. Among them, one had mild developmental delay (DD), two had moderate DD, and eight had severe DD on the DASII assessments (<em>P</em> < 0.001). Sensitivity between absent FMs and ASQ-3 developmental scales showed that the majority with absent FMs had severe DD. Absent FMs had 81.8% sensitivity and 89.4% specificity for detecting cerebral palsy (CP) (n = 11).</div></div><div><h3>Conclusions</h3><div>The assessment of the FMs has a very good predictive value for future neurodevelopmental outcomes. MOS-R can provide a fair estimate of the functional ability of infants who later develop CP, and better MOS-R scores correlate with better outcomes. Children with absent FMs had significantly lower median motor and mental developmental quotients as calculated by DASII at both ages six and 12 months.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"164 ","pages":"Pages 58-65"},"PeriodicalIF":3.2,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143040927","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}
Pediatric neurologyPub Date : 2025-01-07DOI: 10.1016/j.pediatrneurol.2025.01.001
Yuying Feng MM , Yannan Cheng MM , Xianjun Li PhD , Yao Ge MM , Congcong Liu MD , Miaomiao Wang MD , Xiaocheng Wei ME , Xiaoyu Wang MM , Qinli Sun PhD , Jie Zheng MM , Jian Yang PhD , Chao Jin PhD
{"title":"Preterm Neonates Exhibit a “Catch-Up” Pattern in Motor Development During the Neonatal Period: A Diffusion Tensor Imaging Study","authors":"Yuying Feng MM , Yannan Cheng MM , Xianjun Li PhD , Yao Ge MM , Congcong Liu MD , Miaomiao Wang MD , Xiaocheng Wei ME , Xiaoyu Wang MM , Qinli Sun PhD , Jie Zheng MM , Jian Yang PhD , Chao Jin PhD","doi":"10.1016/j.pediatrneurol.2025.01.001","DOIUrl":"10.1016/j.pediatrneurol.2025.01.001","url":null,"abstract":"<div><h3>Background</h3><div>Preterm infants are at high risk for subsequent neurodevelopmental disability. Early developmental characterization of brain and neurobehavioral function is critical for identifying high-risk infants. This study aimed to elucidate the early evolution of sensorimotor function in preterm neonates by exploring postnatal age-related changes in the brain white matter (WM) and neurobehavioral abilities.</div></div><div><h3>Methods</h3><div>One hundred eighteen neonates without abnormalities on magnetic resonance imaging were included. Diffusion tensor imaging-derived fractional anisotropy (FA) and neonatal neurobehavioral assessment were separately used to characterize the brain WM microstructure and neurobehavioral development levels. Scatterplots with linear fitting and Pearson correlation were used to investigate the relationships of FA and neurobehavioral scores (active tone and behavior scores) with postnatal age separately for preterm and term neonates. Here, the optical radiation (OR), auditory radiation, corticospinal tract (CST), posterior thalamic radiation (PTR), and thalamus-primary somatosensory cortex were selected as the regions of interest (ROIs).</div></div><div><h3>Results</h3><div>The preterm FAs in the ROIs were lower than term neonates (all Bonferroni-corrected <em>P</em> < 0.001). Preterm CST FA showed a significantly higher correlation with postnatal age (<em>P</em> = 0.042) than term (<em>r</em> = 0.29 vs 0.08), whereas significantly higher correlations were found in term OR (<em>P</em> = 0.018) and PTR (<em>P</em> = 0.002). Similarly, relatively high and low correlations between active tone (<em>r</em> = 0.48 vs 0.35; <em>P</em> = 0.049 for interactions with a postnatal age ≥14 days and preterm/term group status) and behavioral scores (<em>r</em> = 0.36 vs 0.52; <em>P</em> = 0.030 for interactions of postnatal age and preterm/term group status) were observed in preterm infants.</div></div><div><h3>Conclusions</h3><div>Although delayed, preterm neonates exhibit a “catch-up” pattern in motor development in the newborn stage.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"164 ","pages":"Pages 81-88"},"PeriodicalIF":3.2,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143066983","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}
Pediatric neurologyPub Date : 2025-01-03DOI: 10.1016/j.pediatrneurol.2024.12.016
Zizhang Cheng MM , Weijin Sun MD , Kaiqiang Ma MD , Xiongfei Wang MD , Junhong Pan MN , Haowei Ma MM , Xintao Peng MM , Guoming Luan MD , Yuguang Guan MD
{"title":"Exploring the Efficacy and Safety of Vagus Nerve Stimulation for the Treatment of Epilepsy in Patients With Sturge-Weber Syndrome: A Pilot Study","authors":"Zizhang Cheng MM , Weijin Sun MD , Kaiqiang Ma MD , Xiongfei Wang MD , Junhong Pan MN , Haowei Ma MM , Xintao Peng MM , Guoming Luan MD , Yuguang Guan MD","doi":"10.1016/j.pediatrneurol.2024.12.016","DOIUrl":"10.1016/j.pediatrneurol.2024.12.016","url":null,"abstract":"<div><h3>Background</h3><div>Sturge-Weber syndrome (SWS) is a rare congenital neurocutaneous disorder, often complicated by epilepsy. Approximately 50% of patients with SWS with epilepsy develop drug-resistant seizures, leaving limited treatment options. Vagus nerve stimulation (VNS) is a known therapy for refractory epilepsy, modulating neural activity to reduce seizures. This study examines the therapeutic outcomes, efficacy, and safety of VNS in five patients with SWS suffering from epilepsy.</div></div><div><h3>Methods</h3><div>A retrospective analysis of VNS treatment data from January 2021 to January 2022 in patients with SWS was conducted. Preoperative assessments included neuroimaging and video-electroencephalography monitoring. Cognitive function and quality of life were assessed using age-appropriate scales. VNS settings and seizure outcomes were recorded at different follow-up intervals. Seizure outcomes were classified using the modified Engel and McHugh classification. Cognitive function and quality of life were reassessed at two-year follow-up.</div></div><div><h3>Results</h3><div>Five patients, primarily pediatric, with seizure onset between age 0.5 and eight years, were included. After VNS therapy, all patients experienced a reduction in seizure frequency, with one patient becoming seizure free and three achieving a ≥50% reduction in seizures. Two children with cognitive impairments at baseline demonstrated cognitive improvements following treatment. All patients reported significant enhancements in quality of life. VNS was well tolerated, with no major adverse events reported.</div></div><div><h3>Conclusions</h3><div>VNS offers promising therapeutic benefits for epilepsy in patients with SWS, reducing seizure frequency, improving cognitive function in children, and enhancing quality of life with a favorable safety profile. Further research with larger sample sizes and control groups is warranted to validate efficacy and explore personalized treatment options.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"164 ","pages":"Pages 35-40"},"PeriodicalIF":3.2,"publicationDate":"2025-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143028949","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}
Pediatric neurologyPub Date : 2025-01-03DOI: 10.1016/j.pediatrneurol.2024.12.015
Benton Spirek BS , J. Nicholas Brenton MD
{"title":"Safety and Efficacy of Fingolimod and Ocrelizumab in Pediatric Patients With Multiple Sclerosis","authors":"Benton Spirek BS , J. Nicholas Brenton MD","doi":"10.1016/j.pediatrneurol.2024.12.015","DOIUrl":"10.1016/j.pediatrneurol.2024.12.015","url":null,"abstract":"<div><h3>Background</h3><div>Fingolimod and ocrelizumab are approved treatments for adults with multiple sclerosis (MS); however, only fingolimod is approved by the Food and Drug Administration for the treatment of pediatric MS. Currently, there are limited data for the safety and efficacy of ocrelizumab use in children.</div></div><div><h3>Methods</h3><div>This retrospective cohort study included patients with relapsing-remitting MS who started either ocrelizumab or fingolimod before age 18 years. Neuroimaging, electrocardiogram, laboratory evaluation, relapse history, and side effects were recorded at baseline and every six months.</div></div><div><h3>Results</h3><div>Thirty-six pediatric patients were included (fingolimod, n = 14; ocrelizumab, n = 22). Clinical relapses occurred in 14% of patients treated with fingolimod versus in none of the patients treated with ocrelizumab. Seventy-one percent of patients in the fingolimod group switched or discontinued therapy compared with 9% treated with ocrelizumab (<em>P</em> = 0.0001). From patients with greater than six months of treatment on the given therapy (fingolimod n = 10, ocrelizumab n = 17), 60% on fingolimod exhibited new/enlarged T2-hyperintense lesions on brain magnetic resonance imaging compared with 6% on ocrelizumab (<em>P</em> = 0.004). Of those treated with ocrelizumab, 10 of 22 (45%) had infusion reactions during their initial infusion. Reaction rates decreased to 20% with subsequent infusions.</div></div><div><h3>Conclusions</h3><div>Ocrelizumab is associated with fewer brain lesions, lower clinical relapse rates, and reduced discontinuation rates compared with fingolimod. Although both therapies have the potential for adverse effects, these are unlikely to prompt discontinuation of therapy in isolation. These findings highlight the benefits of ocrelizumab as a treatment option for children and youth living with MS.</div></div>","PeriodicalId":19956,"journal":{"name":"Pediatric neurology","volume":"164 ","pages":"Pages 89-96"},"PeriodicalIF":3.2,"publicationDate":"2025-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143066987","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}