Yana Said, Dimitrios C Ladakis, Julia M Lefelar, Jenny M Khazen, Jennifer Gould, Kathryn C Fitzgerald, Elias S Sotirchos
{"title":"Quality of life is impaired in myelin oligodendrocyte glycoprotein antibody associated disease.","authors":"Yana Said, Dimitrios C Ladakis, Julia M Lefelar, Jenny M Khazen, Jennifer Gould, Kathryn C Fitzgerald, Elias S Sotirchos","doi":"10.1177/20552173241274605","DOIUrl":"10.1177/20552173241274605","url":null,"abstract":"<p><strong>Background: </strong>There is a paucity of studies examining quality of life (QoL) in people with myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD).</p><p><strong>Methods: </strong>A cross-sectional, online, self-administered survey was distributed. Data elements included demographic and clinical characteristics, and QoL in Neurological Disorders (Neuro-QoL) short form questionnaires. Neuro-QoL domain scores were compared to reference populations, yielding standardized T-scores. Symptom severity was categorized as mild, moderate, or severe, using standard Neuro-QoL cut points.</p><p><strong>Results: </strong>A total of 259 participants completed the survey. Neuro-QoL domain impairment was present in a significant proportion of respondents (anxiety: 58.1%, depression: 30.7%, stigma 29.8%, cognition: 58.5%, social function: 57.7%). T-scores were significantly worse than the reference population for anxiety (p<0.001), stigma (p=0.005), cognitive function (p<0.001) and social interactions (p<0.001). There was no clear association between QoL domains and demographics, disease-modifying therapy class, or type of clinical presentation. A relapsing vs monophasic disease course was associated with worse anxiety, stigma, cognition, and social interactions (p<0.05).</p><p><strong>Conclusion: </strong>People with MOGAD may exhibit impairment in multiple domains of QoL. Practicing clinicians should be aware of this burden in MOGAD. Further research is needed to better understand factors associated with QoL impairment in MOGAD.</p>","PeriodicalId":18961,"journal":{"name":"Multiple Sclerosis Journal - Experimental, Translational and Clinical","volume":"10 3","pages":"20552173241274605"},"PeriodicalIF":2.5,"publicationDate":"2024-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11342330/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142054142","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Linda Nguyen, Sumit Singh, Fabricio S Feltrin, Lauren M Tardo, Rebekah L Clarke, Cynthia X Wang, Benjamin M Greenberg
{"title":"The positive predictive value of MOG-IgG testing based on the 2023 diagnostic criteria for MOGAD.","authors":"Linda Nguyen, Sumit Singh, Fabricio S Feltrin, Lauren M Tardo, Rebekah L Clarke, Cynthia X Wang, Benjamin M Greenberg","doi":"10.1177/20552173241274610","DOIUrl":"10.1177/20552173241274610","url":null,"abstract":"<p><strong>Background: </strong>Myelin oligodendrocyte glycoprotein antibody associated disease (MOGAD) is a relatively new disease entity in the field of demyelinating disorders. Its first diagnostic criteria have recently been published.</p><p><strong>Objectives: </strong>We evaluated the positive predictive value (PPV) for MOG-IgG testing and report the clinical and radiologic features with respect to the recently published criteria.</p><p><strong>Methods: </strong>A retrospective study was conducted at three centers in Dallas, Texas. Patients with positive MOG-IgG testing on cell-based assays at any time were included. Positive cases were reviewed by at least two neuroimmunologists for fulfillment of the criteria.</p><p><strong>Results: </strong>We included 235 patients. The PPV of seropositivity at any time was 78.3% overall, 52.6% for low titer, and 90.1% for high titer. Children had a higher PPV than adults (93.9% versus 67.2%). Positive predictive value was 6.3% in those without a core clinical demyelinating attack. Children more often have the typical imaging features of MOGAD in optic neuritis than adults.</p><p><strong>Conclusions: </strong>We report a PPV of 78.3% for MOG-IgG testing using the 2023 MOGAD diagnostic criteria. Children had higher PPV and frequency of supporting imaging features. Careful consideration is necessary when assigning patients with no core demyelinating event and low titers a MOGAD diagnosis.</p>","PeriodicalId":18961,"journal":{"name":"Multiple Sclerosis Journal - Experimental, Translational and Clinical","volume":"10 3","pages":"20552173241274610"},"PeriodicalIF":2.5,"publicationDate":"2024-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11325327/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141988392","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Adel Hassanein Gad, Nirmeen Kishk, Nevin M Shalaby, Eman Salah Heikal, Amr Mohamed Fouad, Nahla Merghany, Hend Abdelghany
{"title":"Pregnancy characteristics in Egyptian female patients with NMOSD.","authors":"Adel Hassanein Gad, Nirmeen Kishk, Nevin M Shalaby, Eman Salah Heikal, Amr Mohamed Fouad, Nahla Merghany, Hend Abdelghany","doi":"10.1177/20552173241271878","DOIUrl":"10.1177/20552173241271878","url":null,"abstract":"<p><strong>Background: </strong>Neuromyelitis optica spectrum disorder (NMOSD) primarily affects women of childbearing age.</p><p><strong>Objectives: </strong>Studying the potential relationship between NMOSD and pregnancy characteristics and outcomes.</p><p><strong>Subjects and methods: </strong>This is a retrospective cohort study that was conducted on 66 married female patients diagnosed with NMOSD. All patients underwent a thorough review of their demographic and clinical history through their medical records and personal interviews. Additionally, a complete neurological examination was performed, along with the expanded disability status scale (EDSS) and a pregnancy registry questionnaire.</p><p><strong>Results: </strong>After comparing married patients before and after disease onset, there was a significant increase in the number of abortions and the percentage of cesarean sections, as well as a decrease in the percentage of breastfeeding after disease onset. The <i>p</i> values were .02, <.001, and <.001, respectively, with odds ratios of 2.03, 5.13, and 6.17. Additionally, there was a rise in the occurrence of postpartum relapses, which accounted for 66% of all relapses after the disease onset. Most of these relapses (88.7%) occurred within the first 3 months postpartum.</p><p><strong>Conclusion: </strong>Presence of NMOSD increased the percentage of miscarriage, delivery by cesarean section, and decreased the chance of breastfeeding. In addition, pregnancy increases NMOSD relapse and subsequent disability.</p>","PeriodicalId":18961,"journal":{"name":"Multiple Sclerosis Journal - Experimental, Translational and Clinical","volume":"10 3","pages":"20552173241271878"},"PeriodicalIF":2.5,"publicationDate":"2024-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11320409/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141976197","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Disseminated histoplasmosis mimicking post-vaccination side effects in an immunocompromised person with multiple sclerosis.","authors":"Ahmad A Toubasi, Steven Allon, Francesca Bagnato","doi":"10.1177/20552173241271790","DOIUrl":"10.1177/20552173241271790","url":null,"abstract":"<p><p>We describe the case of a gentleman with relapsing-remitting multiple sclerosis and chronic lymphocytopenia secondary to treatment with fingolimod who presented with disseminated histoplasmosis after receiving the third dose of the Moderna coronavirus disease 2019 (mRNA-1273) vaccine. Following the vaccination the patient noted fatigue which worsened over time along with gradual weight loss. A few months later he noted low-grade fever and finally shortness of breath. A diagnosis of disseminated histoplasmosis was performed based on urine, blood, and imaging data. He responded well to prolonged antifungal treatment. Fingolimod was discontinued and replaced with glatiramer acetate. He has been clinically stable until the time of this report, 33 months following symptom onset.</p>","PeriodicalId":18961,"journal":{"name":"Multiple Sclerosis Journal - Experimental, Translational and Clinical","volume":"10 3","pages":"20552173241271790"},"PeriodicalIF":2.5,"publicationDate":"2024-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11307347/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141907088","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Sergio Sauri-Suarez, Sandra Quiñones, Manuel De la Maza-Flores, Arturo Marin-Contreras, Gil Playas-Pérez, Brenda Bertado-Cortes, Francisco Frias-Marquez, Gilberto Zuñiga-García, Francisco Rodriguez-Leal, Carlos Blaisdell-Vidal, Enrique Gomez-Figueroa
{"title":"Early clinical effect of cladribine in patients with highly active multiple sclerosis in Mexico.","authors":"Sergio Sauri-Suarez, Sandra Quiñones, Manuel De la Maza-Flores, Arturo Marin-Contreras, Gil Playas-Pérez, Brenda Bertado-Cortes, Francisco Frias-Marquez, Gilberto Zuñiga-García, Francisco Rodriguez-Leal, Carlos Blaisdell-Vidal, Enrique Gomez-Figueroa","doi":"10.1177/20552173241260156","DOIUrl":"10.1177/20552173241260156","url":null,"abstract":"<p><strong>Background: </strong>Cladribine shows efficacy in multiple sclerosis (MS), but Latin American (LATAM) real-world data is limited, despite potential sociodemographic variations.</p><p><strong>Objective: </strong>Investigate baseline characteristics and clinical response in highly active MS patients in Mexico, identifying predictors of early treatment response.</p><p><strong>Method: </strong>A multicenter cohort study analyzed retrospective data from individuals with \"highly active\" MS in the Cladribine Patient Support Program across 11 Mexican clinics. Criteria included one-year prior treatment with another disease-modifying treatment and recent relapse with specific MRI findings. Primary outcomes focused on achieving NEDA-3 status after 12 months.</p><p><strong>Results: </strong>In the follow-up, 67.5% maintained NEDA-3 status. Baseline EDSS scores decreased significantly from 1.50 to 1.00 (<i>p</i> = 0.011), with no confirmed disability worsening. No significant differences were observed between NEDA-3 achievers and non-achievers in demographic and clinical variables. No severe adverse events were reported.</p><p><strong>Conclusion: </strong>Cladribine showed early and effective control of active MS in Mexican patients, demonstrating a secure profile with minimal adverse events. This study provides valuable real-world evidence in the LATAM context.</p>","PeriodicalId":18961,"journal":{"name":"Multiple Sclerosis Journal - Experimental, Translational and Clinical","volume":"10 3","pages":"20552173241260156"},"PeriodicalIF":2.5,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11292702/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141875371","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Julia M Mandler, Johanna Härtl, Isabell Cordts, Marc Sturm, Dennis M Hedderich, Cemsel Bafligil, Enayatullah Baki, Benedikt Becker, Gerrit Machetanz, Tobias B Haack, Achim Berthele, Bernhard Hemmer, Marcus Deschauer
{"title":"Uncovering genetic mimics in multiple sclerosis: A single-center clinical exome sequencing study.","authors":"Julia M Mandler, Johanna Härtl, Isabell Cordts, Marc Sturm, Dennis M Hedderich, Cemsel Bafligil, Enayatullah Baki, Benedikt Becker, Gerrit Machetanz, Tobias B Haack, Achim Berthele, Bernhard Hemmer, Marcus Deschauer","doi":"10.1177/20552173241263491","DOIUrl":"10.1177/20552173241263491","url":null,"abstract":"<p><strong>Background: </strong>Multiple sclerosis (MS) shares clinical/radiological features with several monogenic diseases that can mimic MS.</p><p><strong>Objective: </strong>We aimed to determine if exome sequencing can identify monogenic diseases in patients diagnosed with MS according to the McDonald criteria thus uncovering them as being misdiagnosed.</p><p><strong>Methods: </strong>We performed whole exome sequencing in a cohort of 278 patients with MS, clinically or radiologically isolated syndrome without cerebrospinal fluid-specific oligoclonal bands (CSF-OCBs) (n = 228), a positive family history of MS (n = 44), or both (n = 6), thereby focusing on individuals potentially more likely to have underlying monogenic conditions mimicking MS. We prioritized 495 genes associated with monogenic diseases sharing features with MS.</p><p><strong>Results: </strong>A disease-causing variant in <i>NOTCH3</i> was identified in one patient without CSF-OCBs, no spinal lesions, with non-response to immunotherapy, and a family history of dementia, thereby converting the diagnosis to cerebral autosomal-dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL). Moreover, 18 patients (6.5% of total) carried variants of unclear significance.</p><p><strong>Conclusion: </strong>Monogenic diseases being misdiagnosed as MS seem rare in patients diagnosed with MS according to the McDonald criteria, even in CSF-OCB negative cases. The detected pathogenic <i>NOTCH3</i> variant emphasizes CADASIL as a rare differential diagnosis and highlights the relevance of genetic testing in selected MS cases with atypical presentations.</p>","PeriodicalId":18961,"journal":{"name":"Multiple Sclerosis Journal - Experimental, Translational and Clinical","volume":"10 3","pages":"20552173241263491"},"PeriodicalIF":2.5,"publicationDate":"2024-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11273569/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141788706","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Lara Marleen Fricke, Kathrin Krüger, Corinna Trebst, Anna Levke Brütt, Elise-Marie Dilger, Kerstin Eichstädt, Peter Flachenecker, Anja Grau, Melissa Hemmerling, Dyon Hoekstra, Kristina Schaubert, Alexander Stahmann, Jona Theodor Stahmeyer, Annett Thiele, Uwe Klaus Zettl, Fedor Heidenreich, Christian Krauth
{"title":"Subgroup analyses and patterns of multiple sclerosis health service utilisation: A cluster analysis.","authors":"Lara Marleen Fricke, Kathrin Krüger, Corinna Trebst, Anna Levke Brütt, Elise-Marie Dilger, Kerstin Eichstädt, Peter Flachenecker, Anja Grau, Melissa Hemmerling, Dyon Hoekstra, Kristina Schaubert, Alexander Stahmann, Jona Theodor Stahmeyer, Annett Thiele, Uwe Klaus Zettl, Fedor Heidenreich, Christian Krauth","doi":"10.1177/20552173241260151","DOIUrl":"10.1177/20552173241260151","url":null,"abstract":"<p><strong>Background: </strong>Previous investigations of multiple sclerosis (MS)-related healthcare have focused on utilisation of specific individual health services (e.g. hospital care, office-based neurologists) by people with MS (PwMS). Meanwhile, little is known about possible patterns of utilisation across health services and their potential differences across patient characteristics.</p><p><strong>Objective: </strong>To comprehensively analyse and identify patterns of MS-related health service utilisation and detect patient characteristics explaining such patterns.</p><p><strong>Methods: </strong>In 2021, we invited all PwMS insured by the largest insurance company in Lower Saxony, Germany, to take part in an online survey. We merged respondents' survey and health insurance claims data. We analysed MS-related health service utilisation and defined individual characteristics for subgroup analyses based on Andersen's Behavioural Model. We executed non-parametric missing value imputation and conducted hierarchical clustering to find patterns in health service utilisation.</p><p><strong>Results: </strong>Of 6928 PwMS, 1935 responded to our survey and 1803 were included in the cluster analysis. We identified four distinct health service utilisation clusters: (1) regular users (n = 1130), (2) assistive care users (n = 443), (3) low users (n = 195) and (4) special services users (n = 35). Clusters differ by patient characteristics (e.g. age, impairment).</p><p><strong>Conclusion: </strong>Our findings highlight the complexity of MS-related health service utilisation and provide relevant stakeholders with information allowing them to tailor healthcare planning according to utilisation patterns.</p>","PeriodicalId":18961,"journal":{"name":"Multiple Sclerosis Journal - Experimental, Translational and Clinical","volume":"10 2","pages":"20552173241260151"},"PeriodicalIF":2.5,"publicationDate":"2024-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11191614/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141443085","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Lubna Almouzain, Fiona L Hamilton, Declan Chard, Fiona Stevenson
{"title":"Qualitative user experience evaluation of the MS trust's online treatment decision aid tool's accommodation of planning pregnancy.","authors":"Lubna Almouzain, Fiona L Hamilton, Declan Chard, Fiona Stevenson","doi":"10.1177/20552173241262181","DOIUrl":"10.1177/20552173241262181","url":null,"abstract":"<p><strong>Background: </strong>Decision-making about treatment when planning a pregnancy (family planning) is complex for women living with multiple sclerosis (MS). Decision tools can help this process, in 2016 MS Trust launched their online digital treatment decision tool to support people with MS.</p><p><strong>Objectives: </strong>To evaluate user-experience of this tool by exploring women's opinions about its content, interface, and usefulness in the context of family planning; and to synthesize recommendations to improve the tool.</p><p><strong>Methods: </strong>Thirty participants qualitatively evaluated the tool using Think Aloud methodology. Sessions were conducted online using Microsoft Teams and were video recorded. Transcription was automated and data were thematically analyzed.</p><p><strong>Results: </strong>Women's first impression was that the tool presented a lot of information at once, which was difficult to take in, and they found it difficult to navigate. Although the tool was helpful in allowing them to compare treatment options, the filters were confusing, and the information related to pregnancy sometimes contradicted advice from their healthcare practitioners. They suggested rewording the pregnancy recommendations and filters, updating some content, and making some changes to the interface to meet users' cognitive needs.</p><p><strong>Conclusion: </strong>The MS Trust treatment decision tool is excellent in helping women with treatment choices at initial diagnosis. However, it is not currently as useful when considering family plans. Recommendations were conveyed to MS Trust where some are now applied to the new live version and the rest are to be considered for future updating projects.</p>","PeriodicalId":18961,"journal":{"name":"Multiple Sclerosis Journal - Experimental, Translational and Clinical","volume":"10 2","pages":"20552173241262181"},"PeriodicalIF":2.8,"publicationDate":"2024-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11171435/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141317840","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Abnormal eye movements increase as motor disabilities and cognitive impairments become more evident in Multiple Sclerosis: A novel eye-tracking study.","authors":"Fernández Gerardo, Eizaguirre Bárbara, Gonzalez Cecilia, Marinangeli Aldana, Ciufia Natalia, Bacigalupe Lucia, Berenice Silva, Cohen Leila, Pita Cecilia, Garcea Orlando, Casas Magdalena, Lazaro Luciana, Pardo Gabriel, Alonso Ricardo","doi":"10.1177/20552173241255008","DOIUrl":"10.1177/20552173241255008","url":null,"abstract":"<p><strong>Background: </strong>Eye movements can reflect brain alterations and inform on the presence of motor disabilities and cognitive impairments in people with multiple sclerosis (pwMS).</p><p><strong>Objective: </strong>The aim of the study was to determine the correlation between motor and cognitive measurements and eye movement parameters when performing the <i>n-back</i> task (NBKT).</p><p><strong>Methods: </strong>This was a cross-sectional study carried out at Ramos Mejía Hospital, a center specialized in demyelinating diseases in Buenos Aires, Argentina. The study population consisted of 66 patients with relapsing-remitting multiple sclerosis (RRMS) and 5 patients with secondary progressive multiple sclerosis (SPMS). pwMS performed the n-back test while using a device head mounted display (HMD) with eyetracking capabilities in order to capture eye movement. Clinical motor and cognitive measures were assessed with Expanded Disability Status Scale (EDSS), Nine Hole Peg Test (NHPT), Timed 25-Foot Walk (T25FW), and Symbol Digit Modalities Test (SDMT).</p><p><strong>Results: </strong>pwMS showed strong and statistically significant correlations between gaze duration; number of fixations, saccade amplitude and motor disabilities and cognitive impairments as measured by EDSS, NHPT, T25FW, and SDMT.</p><p><strong>Conclusion: </strong>This study found significant correlations between eye movement behavior and motor and cognitive disability in pwMS. These findings suggest that eye movements have the potential to be used as a surrogate biomarker in MS progression.</p>","PeriodicalId":18961,"journal":{"name":"Multiple Sclerosis Journal - Experimental, Translational and Clinical","volume":"10 2","pages":"20552173241255008"},"PeriodicalIF":2.8,"publicationDate":"2024-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11138185/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141179380","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Michael T G Hayes, Robert J Adam, Pamela A McCombe, Michael Walsh, Stefan Blum
{"title":"Long-term efficacy and safety of rituximab in the treatment of neuromyelitis Optica Spectrum disorder.","authors":"Michael T G Hayes, Robert J Adam, Pamela A McCombe, Michael Walsh, Stefan Blum","doi":"10.1177/20552173241257876","DOIUrl":"10.1177/20552173241257876","url":null,"abstract":"<p><strong>Background: </strong>Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing, autoimmune, inflammatory astrocytopathy. Rituximab for B-cell suppression is a common treatment for NMOSD; however, large-scale randomised controlled trials are lacking.</p><p><strong>Objective: </strong>Evaluate long-term efficacy and safety of rituximab for NMOSD.</p><p><strong>Methods: </strong>Retrospective observational study of patients with NMOSD treated with rituximab. Annualised relapse rates (ARRs) before and during rituximab treatment were evaluated; Modified Rankin Scores (mRS) were measured as a marker of disability.</p><p><strong>Results: </strong>In total, 37 patients were included: 27 aquaporin-4-IgG-seropositive and 10 seronegative NMOSD. The predominant rituximab dosing regimen was an initial 1000 mg, split over two 500 mg infusions, two weeks apart, followed by single 500 mg doses. Over a median follow-up of 54 months, ARR for the whole cohort was 0.136 (95% CI 0.088-0.201), significantly lower than the pretreatment ARR of 0.366 (95% CI 0.271-0.483, <i>p</i> < 0.001). There was a significant reduction in ARR for the seropositive subgroup, but not seronegative. Significant improvement in mRS was seen post-treatment. Infections were reported in 32% of patients during follow-up; most were mild.</p><p><strong>Conclusion: </strong>Rituximab, at doses lower than traditionally used, may be an efficacious therapy for NMOSD, with a favourable safety profile.</p>","PeriodicalId":18961,"journal":{"name":"Multiple Sclerosis Journal - Experimental, Translational and Clinical","volume":"10 2","pages":"20552173241257876"},"PeriodicalIF":2.8,"publicationDate":"2024-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11131406/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141162109","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}