Neurology® Neuroimmunology & Neuroinflammation最新文献

{"title":"Early Adversity and Socioeconomic Factors in Pediatric Multiple Sclerosis: A Case-Control Study.","authors":"Sarah K G Jensen, Susana Camposano, Anne Berens, Michael Waltz, Lauren B Krupp, Leigh Charvet, Anita L Belman, Gregory S Aaen, Leslie A Benson, Meghan Candee, Theron C Casper, Tanuja Chitnis, Jennifer Graves, Yolanda S Wheeler, Ilana Kahn, Timothy E Lotze, Soe S Mar, Mary Rensel, Moses Rodriguez, John W Rose, Jennifer P Rubin, Jan-Mendelt Tillema, Amy T Waldman, Bianca Weinstock-Guttman, Lisa F Barcellos, Emmanuelle Waubant, Mark P Gorman","doi":"10.1212/NXI.0000000000200282","DOIUrl":"10.1212/NXI.0000000000200282","url":null,"abstract":"<p><strong>Background and objectives: </strong>Psychosocial adversity and stress, known to predispose adults to neurodegenerative and inflammatory immune disorders, are widespread among children who experience socioeconomic disadvantage, and the associated neurotoxicity and proinflammatory profile may predispose these children to multiple sclerosis (MS). We sought to determine associations of socioeconomic disadvantage and psychosocial adversity with odds of pediatric-onset MS (POMS), age at POMS onset, and POMS disease activity.</p><p><strong>Methods: </strong>This case-control study used data collected across 17 sites in the United States by the Environmental and Genetic Risk Factors for Pediatric Multiple Sclerosis Study. Cases (n = 381) were youth aged 3-21 years diagnosed with POMS or a clinically isolated demyelinating syndrome indicating high risk of MS. Frequency-matched controls (n = 611) aged 3-21 years were recruited from the same institutions. Prenatal and postnatal adversity and postnatal socioeconomic factors were assessed using retrospective questionnaires and zip code data. The primary outcome was MS diagnosis. Secondary outcomes were age at onset, relapse rate, and Expanded Disability Status Scale (EDSS). Predictors were maternal education, maternal prenatal stress events, child separation from caregivers during infancy and childhood, parental death during childhood, and childhood neighborhood disadvantage.</p><p><strong>Results: </strong>MS cases (64% female, mean age 15.4 years, SD 2.8) were demographically similar to controls (60% female, mean age 14.9 years, SD 3.9). Cases were less likely to have a mother with a bachelor's degree or higher (OR 0.42, 95% CI 0.22-0.80, <i>p</i> = 0.009) and were more likely to experience childhood neighborhood disadvantage (OR 1.04 for each additional point on the neighborhood socioeconomic disadvantage score, 95% CI 1.00-1.07; <i>p</i> = 0.025). There were no associations of the socioeconomic variables with age at onset, relapse rate, or EDSS, or of prenatal or postnatal adverse events with risk of POMS, age at onset, relapse rate, or EDSS.</p><p><strong>Discussion: </strong>Low socioeconomic status at the neighborhood level may increase the risk of POMS while high parental education may be protective against POMS. Although we did not find associations of other evaluated prenatal or postnatal adversities with POMS, future research should explore such associations further by assessing a broader range of stressful childhood experiences.</p>","PeriodicalId":19472,"journal":{"name":"Neurology® Neuroimmunology & Neuroinflammation","volume":"11 5","pages":"e200282"},"PeriodicalIF":7.8,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11379435/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141988495","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Different Patterns of Autoantibody Secretion by Peripheral Blood Mononuclear Cells in Autoimmune Nodopathies.","authors":"Sophia Rohrbacher, Sabine Seefried, Beate Hartmannsberger, Rosa Annabelle, Luise Appeltshauser, Friederike A Arlt, Dirk Brämer, Christian Dresel, Johannes Dorst, Zeynep Elmas, Christiana Franke, Christian Geis, Tobias Högen, Sabine Krause, Martin Marziniak, Mathias Mäurer, Harald Prüss, Florian Schoeberl, Bertold Schrank, Claudia Steen, Helena Teichtinger, Andrea Thieme, Lena Wessely, Alma Zernecke, Claudia Sommer, Kathrin Doppler","doi":"10.1212/NXI.0000000000200295","DOIUrl":"10.1212/NXI.0000000000200295","url":null,"abstract":"<p><strong>Background and objectives: </strong>Autoimmune nodopathies with antibodies against the paranodal proteins show a distinct phenotype of a severe sensorimotor neuropathy. In some patients, complete remission can be achieved after treatment with rituximab whereas others show a chronic course. For optimal planning of treatment, predicting the course of disease and therapeutic response is crucial.</p><p><strong>Methods: </strong>We stimulated peripheral blood mononuclear cells in vitro to find out whether secretion of specific autoantibodies may be a predictor of the course of disease and response to rituximab.</p><p><strong>Results: </strong>Three patterns could be identified: In most patients with anti-Neurofascin-155-, anti-Contactin-1-, and anti-Caspr1-IgG4 autoantibodies, in vitro production of autoantibodies was detected, indicating autoantigen-specific memory B cells and short-lived plasma cells/plasmablasts as the major source of autoantibodies. These patients generally showed a good response to rituximab. In a subgroup of patients with anti-Neurofascin-155-IgG4 autoantibodies and insufficient response to rituximab, no in vitro autoantibody production was found despite high serum titers, indicating autoantibody secretion by long-lived plasma cells outside the peripheral blood. In the patients with anti-pan-Neurofascin autoantibodies-all with a monophasic course of disease-no in vitro autoantibody production could be measured, suggesting a lack of autoantigen-specific memory B cells. In some of them, autoantibody production by unstimulated cells was detectable, presumably corresponding to high amounts of autoantigen-specific plasmablasts-well in line with a severe but monophasic course of disease.</p><p><strong>Discussion: </strong>Our data suggest that different B-cell responses may occur in autoimmune nodopathies and may serve as markers of courses of disease and response to rituximab.</p>","PeriodicalId":19472,"journal":{"name":"Neurology® Neuroimmunology & Neuroinflammation","volume":"11 5","pages":"e200295"},"PeriodicalIF":7.8,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11379437/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142036510","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Antibodies Against ZSCAN1 in Pediatric and Adult Patients With Non-Paraneoplastic ROHHAD Syndrome.","authors":"Ana Beatriz Serafim, Gemma Olivé-Cirera, Ángel Ortega-González, Michael C Kruer, Debra Weese-Mayer, Casey M Rand, Carmen Fons, Joaquín Alejandro Fernández-Ramos, Maria Clemente, Mateus Mistieri Simabukuro, Emilia Katiane Embiruçu, Salvador Ibáñez-Micó, Josep O Dalmau, Francesc Graus, Thais Armangué, Lidia Sabater","doi":"10.1212/NXI.0000000000200276","DOIUrl":"10.1212/NXI.0000000000200276","url":null,"abstract":"<p><strong>Objectives: </strong>To report the association of zinc finger and SCAN domain containing 1 antibodies (ZSCAN1-abs) with rapid-onset obesity, hypothalamic dysfunction, hypoventilation, and autonomic dysregulation (ROHHAD) syndrome in patients without tumor.</p><p><strong>Methods: </strong>Patients with symptoms compatible with ROHHAD syndrome but without an associated tumor were selected from our database. Serum and CSF samples were examined for the presence of ZSCAN1-abs by an in-house cell-based assay. In addition, samples from 149 patients with several inflammatory and noninflammatory disorders and 50 healthy participants served as controls.</p><p><strong>Results: </strong>Thirteen patients with ROHHAD syndrome were identified. Of these, we had paired serum/CSF samples from 6 patients and only serum from the other 7. Five of 6 patients (83.3%) with paired serum/CSF (4 children, 1 adult) had ZSCAN-abs only in CSF and 1 had antibodies in serum and CSF. ZSCAN1-abs were not detected in the remaining 7 patients with ROHHAD with only serum available or in any of the 199 control samples.</p><p><strong>Discussion: </strong>Patients with ROHHAD syndrome should be investigated for the presence of ZSCAN1-abs in CSF. The antibodies do not necessarily predict the presence of a tumor. The detection of ZSCAN1-abs in an adult patient suggests that this condition also occurs beyond the pediatric age.</p>","PeriodicalId":19472,"journal":{"name":"Neurology® Neuroimmunology & Neuroinflammation","volume":"11 5","pages":"e200276"},"PeriodicalIF":7.8,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11204383/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141451059","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Presentation and Outcome in S1P-RM and Natalizumab-Associated Progressive Multifocal Leukoencephalopathy: A Multicenter Cohort Study.","authors":"Julie C Blant, Nicola N De Rossi, Ralf Gold, Aude Maurousset, Markus Kraemer, Lucía Romero-Pinel, Tatsuro Misu, Jean-Christophe Ouallet, Maud Pallix Guyot, Simonetta Gerevini, Christos Bakirtzis, Raquel Piñar Morales, Benjamin Vlad, Panajotis Karypidis, Xavier Moisset, Tobias J Derfuss, Ilijas Jelcic, Guillaume Martin-Blondel, Ilya Ayzenberg, Corey McGraw, David A Laplaud, Renaud A Du Pasquier, Raphael Bernard-Valnet","doi":"10.1212/NXI.0000000000200281","DOIUrl":"10.1212/NXI.0000000000200281","url":null,"abstract":"<p><strong>Background and objectives: </strong>Progressive multifocal leukoencephalopathy (PML) is a severe neurologic disease resulting from JC virus reactivation in immunocompromised patients. Certain multiple sclerosis (MS) disease-modifying therapies (DMTs) are associated with PML risk, such as natalizumab and, more rarely, sphingosine-1-phosphate receptor modulators (S1P-RMs). Although natalizumab-associated PML is well documented, information on S1P-RM-associated PML is limited. The aim of this study is to compare clinical presentations and outcomes between the 2 groups.</p><p><strong>Methods: </strong>A retrospective multicenter cohort study included patients with PML from 2009 to 2022 treated with S1P-RMs or natalizumab. Data on clinical and radiologic presentation, outcomes, immune reconstitution inflammatory syndrome (IRIS), survival, disability (using the modified Ranking scale-mRS), and MS relapses post-PML were analyzed.</p><p><strong>Results: </strong>Of 88 patients, 84 were analyzed (20 S1P-RM, 64 natalizumab). S1P-RM-associated PML was diagnosed in older patients (median age 52 vs 44 years, <i>p</i> < 0.001) and after longer treatment duration (median 63.9 vs 40 months, <i>p</i> < 0.001). Similarly, S1P-RM patients were more prone to show symptoms at diagnosis (100 vs 80.6%, <i>p</i> = 0.035), had more disseminated lesions (80% vs 34.9%, <i>p</i> = 0.002), and had higher gadolinium enhancement (65% vs 39.1%, <i>p</i> = 0.042). Natalizumab patients had a higher IRIS development rate (OR: 8.3 [1.92-33.3]). Overall, the outcome (mRS) at 12 months was similar in the 2 groups (OR: 0.81 [0.32-2.0]). Yet, post-treatment MS activity was higher in S1P-RM cases (OR: 5.7 [1.4-22.2]).</p><p><strong>Discussion: </strong>S1P-RM-associated PML shows reduced IRIS risk but higher post-treatment MS activity. Clinicians should tailor post-PML treatment based on pre-PML medication.</p>","PeriodicalId":19472,"journal":{"name":"Neurology® Neuroimmunology & Neuroinflammation","volume":"11 5","pages":"e200281"},"PeriodicalIF":7.8,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11256981/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141590943","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A 73-Year-Old Woman With Confusion, Visual Field Disturbances, and Edematous White Matter Lesions: From the National Multiple Sclerosis Society Case Conference Proceedings.","authors":"Zachery Rohm, Myla D Goldman, Claire Riley, Scott S Zamvil, Siddharama Pawate","doi":"10.1212/NXI.0000000000200300","DOIUrl":"10.1212/NXI.0000000000200300","url":null,"abstract":"<p><p>We describe the case of a 73-year-old woman presenting with headaches, confusion, and vision disturbances. Brain MRI showed a large T2-hyperintense lesion in the right temporo-occipital region with vasogenic edema and leptomeningeal enhancement. A leptomeningeal biopsy was performed, which led to a definitive diagnosis.</p>","PeriodicalId":19472,"journal":{"name":"Neurology® Neuroimmunology & Neuroinflammation","volume":"11 5","pages":"e200300"},"PeriodicalIF":7.8,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11379432/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141982916","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Association Between Alcohol Consumption and Disability Accumulation in Multiple Sclerosis.","authors":"Jing Wu, Tomas Olsson, Jan A Hillert, Lars Alfredsson, Anna Karin Hedström","doi":"10.1212/NXI.0000000000200289","DOIUrl":"10.1212/NXI.0000000000200289","url":null,"abstract":"<p><strong>Background and objectives: </strong>Previous studies have indicated that alcohol consumption is associated with multiple sclerosis (MS) disease progression. We aimed to study the influence of alcohol consumption habits on disease progression and health-related quality of life in MS.</p><p><strong>Methods: </strong>We categorized patients from 2 population-based case-control studies by alcohol consumption habits at diagnosis and followed them up to 15 years after diagnosis through the Swedish MS registry regarding changes in the Expanded Disability Status Scale (EDSS) and Multiple Sclerosis Impact Scale 29 (MSIS-29). We used Cox regression models with 95% confidence intervals (CIs) using 24-week confirmed disability worsening, EDSS 3, EDSS 4, and physical and psychological worsening from the patient's perspective as end points.</p><p><strong>Results: </strong>Our study comprised 9,051 patients with MS, with a mean age of 37.5 years at baseline/diagnosis. Compared with nondrinking, low and moderate alcohol consumption was associated with reduced risk of EDSS-related unfavorable outcomes (hazard ratios between 0.81 and 0.90) and with reduced risk of physical worsening. The inverse association was confined to relapsing-remitting MS and was more pronounced among women. High alcohol consumption did not significantly affect disease progression. The inverse relationship between low-moderate alcohol consumption and disability progression became stronger when we only included those who had not changed their alcohol consumption during follow-up (hazard ratios between 0.63 and 0.71). There were no differences in measures of disability at baseline between drinkers who continued drinking alcohol after diagnosis and those who later discontinued. Our findings speak against bias due to reverse causation.</p><p><strong>Discussion: </strong>Low and moderate alcohol consumption was associated with more favorable outcomes in relapsing-remitting MS, compared with nondrinking, while there was no significant influence of high alcohol consumption on disease outcomes.</p>","PeriodicalId":19472,"journal":{"name":"Neurology® Neuroimmunology & Neuroinflammation","volume":"11 5","pages":"e200289"},"PeriodicalIF":7.8,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11379438/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141875480","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Absence of Pathogenic Mutations and Strong Association With HLA-DRB1*11:01 in Statin-Naïve Early-Onset Anti-HMGCR Necrotizing Myopathy.","authors":"Laura Llansó, Alba Segarra-Casas, Cristina Domínguez-González, Edoardo Malfatti, Solange Kapetanovic, Benjamín Rodríguez-Santiago, Oscar de la Calle, Rosa Blanco, Amelia Dobrescu, Andrés Nascimento-Osorio, Andrés Paipa, Aurelio Hernandez-Lain, Cristina Jou, Anaís Mariscal, Laura González-Mera, Ana Arteche, Cinta Lleixà, Marta Caballero-Ávila, Álvaro Carbayo, Ana Vesperinas, Luis Querol, Eduard Gallardo, Montse Olivé","doi":"10.1212/NXI.0000000000200285","DOIUrl":"10.1212/NXI.0000000000200285","url":null,"abstract":"<p><strong>Background and objectives: </strong>Immune-mediated necrotizing myopathy (IMNM) caused by antibodies against 3-hydroxy-3-methylglutaryl coenzyme A reductase (HMGCR) is an inflammatory myopathy that has been epidemiologically correlated with previous statin exposure. We characterized in detail a series of 11 young statin-naïve patients experiencing a chronic disease course mimicking a limb-girdle muscular dystrophy. With the hypothesis that HMGCR upregulation may increase immunogenicity and trigger the production of autoantibodies, our aim was to expand pathophysiologic knowledge of this distinct phenotype.</p><p><strong>Methods: </strong>Clinical and epidemiologic data, autoantibody titers, creatine kinase (CK) levels, response to treatment, muscle imaging, and muscle biopsies were assessed. HMGCR expression in patients' muscle was assessed by incubating sections of affected patients with purified anti-HMGCR+ serum. Whole-exome sequencing (WES) with a special focus on cholesterol biosynthesis-related genes and high-resolution human leukocyte antigen (HLA) typing were performed.</p><p><strong>Results: </strong>Patients, aged 3-25 years and mostly female (90.9%), presented with subacute proximal weakness progressing over many years and high CK levels (>1,000 U/L). Diagnostic delay ranged from 3 to 27 years. WES did not reveal any pathogenic variants. HLA-DRB1*11:01 carrier frequency was 60%, a significantly higher proportion than in the control population. No upregulation or mislocalization of the enzyme in statin-exposed or statin-naïve anti-HMGCR+ patients was observed, compared with controls.</p><p><strong>Discussion: </strong>WES of a cohort of patients with dystrophy-like anti-HMGCR IMNM did not reveal any common rare variants of any gene, including cholesterol biosynthesis-related genes. HLA analysis showed a strong association with HLA-DRB1*11:01, previously mostly described in statin-exposed adult patients; consequently, a common immunogenic predisposition should be suspected, irrespective of statin exposure. Moreover, we were unable to conclusively demonstrate muscle upregulation/mislocalization of HMGCR in IMNM, whether or not driven by statins.</p>","PeriodicalId":19472,"journal":{"name":"Neurology® Neuroimmunology & Neuroinflammation","volume":"11 5","pages":"e200285"},"PeriodicalIF":7.8,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11309561/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141897935","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"CD19-Directed CAR T-Cells in a Patient With Refractory MOGAD: Clinical and Immunologic Follow-Up for 1 Year.","authors":"Jose Maria Cabrera-Maqueda, Maria Sepulveda, Raquel Ruiz García, Guillermo Muñoz-Sánchez, Nuria Martínez-Cibrian, Valentín Ortíz-Maldonado, Daniel Lorca-Arce, Mar Guasp, Sara Llufriu, Eugenia Martinez-Hernandez, Thais Armangue, Elianet G Fonseca, María Teresa Alba-Isasi, Julio Delgado, Josep Dalmau, Manel Juan, Albert Saiz, Yolanda Blanco","doi":"10.1212/NXI.0000000000200292","DOIUrl":"10.1212/NXI.0000000000200292","url":null,"abstract":"<p><strong>Objectives: </strong>In MOG antibody-associated disease (MOGAD), relapse prevention and the treatment approach to refractory symptoms are unknown. We report a patient with refractory MOGAD treated with CD19-directed CAR T-cells.</p><p><strong>Methods: </strong>CD19-directed CAR T-cells (ARI-0001) were produced in-house by lentiviral transduction of autologous fresh leukapheresis and infused after a conventional lymphodepleting regimen.</p><p><strong>Results: </strong>A 18-year-old man developed 2 episodes of myelitis associated with serum MOG-IgG, which were followed by 6 episodes of left optic neuritis (ON) and sustained the presence of MOG-IgG over 6 years despite multiple immunotherapies. After the sixth episode of ON, accompanied by severe residual visual deficits, CAR T-cell treatment was provided without complications. Follow-up of cell counts showed complete depletion of CD19⁺ B cells at day +7; reconstituted B cells at day +141 showing a naïve B-cell phenotype, and low or absent memory B cells and plasmablasts for 1 year. MOG-IgG titers have remained undetectable since CAR T-cell infusion. The patient had an early episode of left ON at day +29, when MOG-IgG was already negative, and since then he has remained free of relapses without immunotherapy for 1 year.</p><p><strong>Discussion: </strong>This clinical case shows that CD19-directed CAR T-cell therapy is well-tolerated and is a potential treatment for patients with refractory MOGAD.</p><p><strong>Classification of evidence: </strong>This provides Class IV evidence. It is a single observational study without controls.</p>","PeriodicalId":19472,"journal":{"name":"Neurology® Neuroimmunology & Neuroinflammation","volume":"11 5","pages":"e200292"},"PeriodicalIF":7.8,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11309560/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141897936","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Should We Measure CSF Complement Levels in Patients With MOGAD?","authors":"Carson E Moseley, Scott S Zamvil","doi":"10.1212/NXI.0000000000200302","DOIUrl":"10.1212/NXI.0000000000200302","url":null,"abstract":"","PeriodicalId":19472,"journal":{"name":"Neurology® Neuroimmunology & Neuroinflammation","volume":"11 5","pages":"e200302"},"PeriodicalIF":7.8,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11379433/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141971546","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"High NEDA and No PIRA in Natalizumab-Treated Patients With Pediatric-Onset Multiple Sclerosis.","authors":"Marco Puthenparampil, Marta Gaggiola, Marta Ponzano, Giovanni Zanotelli, Alessandro Miscioscia, Margherita Nosadini, Alessandro Di Paola, Stefano Sartori, Paola Perini, Francesca Rinaldi, Francesca Bovis, Paolo Gallo","doi":"10.1212/NXI.0000000000200303","DOIUrl":"10.1212/NXI.0000000000200303","url":null,"abstract":"<p><strong>Background and objectives: </strong>Although pediatric-onset multiple sclerosis (POMS) is characterized by a more rapid accumulation of CNS inflammation than adult-onset MS (AOMS), the therapeutic algorithms applied in POMS are usually based on AOMS therapeutic outcomes. To define a high-efficacy treatment (HET)-based strategy to treat POMS, we designed an observational retrospective study aimed at evaluating the efficacy and safety of natalizumab (NTZ) in naïve POMS and AOMS.</p><p><strong>Methods: </strong>Starting from 160 patients, we applied a 2:1 (adult:pediatric) matching on propensity scores and obtained 32 patients with NTZ-treated POMS and 64 with AOMS, estimated from a multivariable logistic regression model. All patients were clinically and radiologically followed up every 6 months for a mean period of 46.0 ± 26.9 months.</p><p><strong>Results: </strong>Following re-baseline at month 6, no difference (log-rank test: <i>p</i> = 0.924) in new and enlarging T2 white matter lesions, postcontrast T1 lesions, and relapse rate were observed between POMS and AOMS throughout the study. Progression independent of relapse activity (PIRA) was never observed in POMS, while 9 of 64 patients with AOMS (12.5%) had PIRA events during the follow-up (40.0 ± 25.9 months; log-rank <i>p</i> value 0.0156). JCV seroconversion rate during NTZ infusion did not differ between POMS and AOMS (log-rank test <i>p</i> = 0.3231). Finally, no serious adverse event was observed in both POMS and AOMS.</p><p><strong>Discussion: </strong>The favorable outcomes observed on clinical, especially in PIRA, and radiologic parameters strongly support the use of NTZ as a first-choice HET in POMS.</p>","PeriodicalId":19472,"journal":{"name":"Neurology® Neuroimmunology & Neuroinflammation","volume":"11 5","pages":"e200303"},"PeriodicalIF":7.8,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11379434/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141982917","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}