Multiple sclerosis and related disorders最新文献

{"title":"A retrospective evaluation of patient characteristics and recommendations of a novel multidisciplinary clinic for persons with advanced disability from multiple sclerosis","authors":"Emma A. Bateman ,&nbsp;Teneille Gofton ,&nbsp;Courtney Casserly ,&nbsp;Luxshmi Nageswaran ,&nbsp;Sarah A. Morrow","doi":"10.1016/j.msard.2025.106287","DOIUrl":"10.1016/j.msard.2025.106287","url":null,"abstract":"<div><h3>Context</h3><div>Persons with advanced multiple sclerosis (MS) require care beyond the disease modifying treatments offered in conventional MS clinics to address their complex physical and psychosocial needs. In the novel MS Comprehensive and Palliative Care (MSCPC) Program, an MS neurologist, palliative care specialist, and physiatrist collaborate to identify these needs and improve symptom control.</div></div><div><h3>Objectives</h3><div>To characterize the medical, physical, and psychosocial concerns of persons with advanced disability from MS and describe the recommended interventions of the MSCPC Program.</div></div><div><h3>Methods</h3><div>Retrospective chart review of consecutive patients seen in the MSCPC Program from 2019 to 2022.</div></div><div><h3>Results</h3><div>54 patients were assessed over 74 clinic appointments. Patients’ mean age was 59.4 ± 10.8 years (range 37–81) and mean duration of MS was 24.8 ± 11.8 years (range 2–52); 79.7% of patients had secondary progressive MS with median and mode disease severity (EDSS) of 7.5 and 8.5, respectively (range 4–9.5). 70.3% lived at home with a caregiver; the primary caregiver was the spouse for 51.4% of cases. 85.1% of patients received publicly funded in-home assistance for activities of daily living. The most prevalent sequelae of MS were incontinence (89.9%), spasticity (82.6%), and pain (78.3%). ≥1 symptom was addressed at 95.7% of appointments, most often pain (63.8%), spasticity (60.9%), and bowel (59.4%); medication deprescribing was recommended at 29.0% of appointments. Caregiver burnout was identified at 56.5% of appointments.</div></div><div><h3>Conclusion</h3><div>This novel program identified high prevalence of symptoms and made recommendations to improve symptom control at &gt;95% of appointments, suggesting unmet symptom control needs in persons with advanced disability from MS.</div></div>","PeriodicalId":18958,"journal":{"name":"Multiple sclerosis and related disorders","volume":"94 ","pages":"Article 106287"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143047360","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Circulating insulin-like growth factor-1 and the risk of multiple sclerosis: a prospective cohort study","authors":"Xiao-Wei Pang ,&nbsp;Sheng Yang ,&nbsp;Lu-Yang Zhang ,&nbsp;Lian Chen ,&nbsp;Li-Fang Zhu ,&nbsp;Yun-Hui Chu ,&nbsp;Ming-Hao Dong ,&nbsp;Luo-Qi Zhou ,&nbsp;Jun Xiao ,&nbsp;Wei Wang ,&nbsp;Chuan Qin ,&nbsp;Dai-Shi Tian","doi":"10.1016/j.msard.2025.106281","DOIUrl":"10.1016/j.msard.2025.106281","url":null,"abstract":"<div><h3>Background</h3><div>Circulating insulin-like growth factor 1 (IGF-1) is positively associated with the risks of certain neurological disorders, including stroke, Alzheimer's disease, and Parkinson's disease. However, the association of IGF-1 with the risk of multiple sclerosis (MS) remains unclear.</div></div><div><h3>Methods</h3><div>A total of 348,324 participants at baseline were included from the UK biobank in this prospective study. The association of circulating IGF-1 level with MS was analyzed by Cox proportional hazard models. Further, subgroup analyses were conducted to investigate the variables influencing these associations.</div></div><div><h3>Results</h3><div>Among 348,324 individuals, lower circulating IGF-1 concentrations were associated with a reduced risk of MS (95 % CI, 0.5930–0.9700; <em>P</em> value = 0.02763). The association between lower IGF-1 levels and reduced risk of MS remains robust in older and female participants. Moreover, risk of MS appeared to be lower in IGF-1-low individuals who never smoked, currently drinking alcohol, with higher body mass index, and higher glucose concentrations.</div></div><div><h3>Conclusion</h3><div>Our findings indicate that a lower concentration of serum IGF-1 was associated with a reduced risk of MS. The results provide evidence that the circulating IGF-1 may play a significant role in the pathogenesis of MS.</div></div>","PeriodicalId":18958,"journal":{"name":"Multiple sclerosis and related disorders","volume":"94 ","pages":"Article 106281"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143066408","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":"Assessment of dysphagia and its associations in patients with secondary progressive multiple sclerosis","authors":"Majid Hamidi,&nbsp;Nasim Rezaeimanesh,&nbsp;Mohammad Ali Sahraian,&nbsp;Abdorreza Naser Moghadasi","doi":"10.1016/j.msard.2024.106254","DOIUrl":"10.1016/j.msard.2024.106254","url":null,"abstract":"<div><div>In this cross-sectional study in patients with secondary progressive multiple sclerosis receiving anti-CD20 monoclonal antibodies, we assessed complaint of dysphagia and evaluated their association with sex, age, disease duration, disability severity, and disease-modifying therapies (DMTs). The validated Persian version of Dysphagia in Multiple Sclerosis (DYMUS) questionnaire was used. A total of 66 patients were included. The median DYMUS score was 1.0 [interquartile range: 0.0-2.3] and 40 (60.6%) had positive DYMUS. Only DMT showed a significant association with DYMUS score, with patients receiving reituximab exhibiting higher scores (mean estimated difference: 0.96 (±0.39), <em>p</em> = 0.014). Grouping patients into positive and negative DYMUS scores, no significant associations were observed (<em>p</em> &gt; 0.05).</div></div>","PeriodicalId":18958,"journal":{"name":"Multiple sclerosis and related disorders","volume":"94 ","pages":"Article 106254"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142965978","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":"Cerebrospinal fluid biomarkers as predictors of multiple sclerosis severity","authors":"Miguel Tolentino ,&nbsp;Francesca Pace ,&nbsp;Dana C. Perantie ,&nbsp;Robert Mikesell ,&nbsp;Julia Huecker ,&nbsp;Salim Chahin ,&nbsp;Laura Ghezzi ,&nbsp;Laura Piccio ,&nbsp;Anne H. Cross","doi":"10.1016/j.msard.2025.106268","DOIUrl":"10.1016/j.msard.2025.106268","url":null,"abstract":"<div><h3>Background</h3><div>Prognostic biomarkers at multiple sclerosis (MS) onset to predict disease severity may help guide initial therapy selection for people with MS. Over 20 disease-modifying treatments (DMTs) of varying levels of risk and efficacy now exist. The ability to predict MS severity would help to identify those patients at higher risk where a highly effective, but potentially risky, therapy would be optimal. The goal of this project was to determine if cerebrospinal fluid (CSF) soluble markers obtained near time of diagnosis can predict disease severity in people with relapsing remitting MS (RRMS).</div></div><div><h3>Methods</h3><div>We identified 42 RRMS subjects with 4 or more years of clinical follow-up at our center, 8 subjects with other inflammatory neurological diseases (OIND), and 4 subjects with non-inflammatory neurological diseases (NIND) who had donated CSF samples collected for disease diagnosis. This study evaluated soluble CSF biomarkers chosen to reflect neuroinflammation (chemokine ligand 13 – CXCL13), microglia activity (soluble triggering receptor expressed on myeloid cells 2 – sTREM2), demyelination (myelin basic protein –MBP), axon injury and loss (neurofilament light, heavy, and intermediate chains – NFL, NFH, internexin-alpha – INT-α) and neuronal loss (parvalbumin – PVALB) to determine whether any of these CSF factors might predict future MS disease severity. The main outcome measure was MS Severity Score (MSSS), which takes into account disability accumulation (expanded disability status scale - EDSS) and duration of disease. EDSS at last clinical visit was a secondary outcome measure. Univariate and multivariable regression models were used for analysis. Spearman correlations were performed to evaluate correlation between laboratory and clinical variables.</div></div><div><h3>Results</h3><div>Forty-two RRMS patients with mean 9.4 years follow-up since lumbar puncture (LP) contributed data. Higher NFH, NFL, and sTREM2 each predicted worse MSSS using both univariate and multivariable regression models. Older age at the time of LP predicted worse MSSS both in the univariate and multivariable models. NFL correlated with NFH, and both were positively correlated with sTREM2 and CXCL13. In the combined OIND and NIND comparator group, NFH correlated with both NFL and CXCL13.</div></div><div><h3>Conclusion</h3><div>These data support that CSF sTREM2, NFH, and NFL are predictors of MSSS, a measure of MS disease aggressiveness. This study adds to a growing literature implicating microglial activity and axonal injury in MS progression, starting from early stages of the disease.</div></div>","PeriodicalId":18958,"journal":{"name":"Multiple sclerosis and related disorders","volume":"94 ","pages":"Article 106268"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143008735","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":"Comparative effectiveness, persistence, and adherence of dimethyl fumarate and fingolimod in patients with multiple sclerosis in Japan: A cohort study","authors":"Sho Shimazaki ,&nbsp;Toshiki Fukasawa ,&nbsp;Takayuki Kondo ,&nbsp;Masato Takeuchi ,&nbsp;Takayuki Okura ,&nbsp;Ryosuke Takahashi ,&nbsp;Koji Kawakami","doi":"10.1016/j.msard.2025.106306","DOIUrl":"10.1016/j.msard.2025.106306","url":null,"abstract":"<div><h3>Background</h3><div>Several oral disease-modifying drugs (DMDs) for multiple sclerosis (MS) are available but only two – dimethyl fumarate (DMF) and fingolimod (FTY) – are approved for relapsing-remitting MS in Japan. Although the efficacy of DMDs might be affected by differences in genetic and environmental factors, no study has compared the effectiveness, treatment persistence, and adherence of DMF and FTY in Asians. Here, we assessed relapse rates, persistence, and adherence of DMF and FTY in a Japanese real-world setting.</div></div><div><h3>Methods</h3><div>A cohort study was conducted using a large Japanese claims database. We included MS patients aged ≥18 years who initiated DMF or FTY between February 22, 2017 (the launch of DMF in Japan), and February 29, 2020. The primary effectiveness outcome was MS relapse, defined as treatment with intravenous methylprednisolone at a dosage of ≥250 mg/day for at least 3 consecutive days. Annualized relapse rates (ARRs) and ARR ratios for DMF and FTY groups were estimated using a Poisson regression model. Secondary outcomes included persistence and adherence. Persistence was evaluated using Kaplan–Meier survival analysis and a Cox proportional hazards model. Adherence was assessed by the proportion of days covered (PDC) at 30, 60, 90, 180, and 365 days. Propensity score overlap weighting was used to adjust for baseline covariates between groups.</div></div><div><h3>Results</h3><div>143 patients in the DMF group (mean age 41.45 years, 59 males, mean number of MS relapses at baseline: 0.54) and 36 in the FTY group (mean age 39.89 years, 18 males, mean number of MS relapses at baseline: 0.56) met the eligibility criteria. After overlap weighting, covariates were well balanced. The ARR was 0.05 (95 % confidence interval, 0.02–0.16) in the DMF group and 0.15 (0.05–0.45) in the FTY group, with a ratio of 0.34 (0.07–1.63). The 1-year persistence was 0.84 (0.74–0.95) for the DMF group and 0.53 (0.37–0.78) for the FTY group, resulting in a hazard ratio of 0.28 (0.11–0.70). Median PDC for the DMF group was 0.90 (interquartile range, 0.77–1.00) at 30 days, which improved over time as treatment duration increased to reach 0.98 (0.94–0.98) at 365 days. In contrast, median PDC for the FTY group remained constant at 0.98–1.00 across all time points.</div></div><div><h3>Conclusion</h3><div>The effectiveness, as measured by ARRs, was comparable between the DMF and FTY groups, although a small but clinically significant difference may not have been detected in this study. Persistence was higher in the DMF group than in the FTY group. Adherence was high for both drugs, but some patients treated with DMF had low adherence early in treatment.</div></div>","PeriodicalId":18958,"journal":{"name":"Multiple sclerosis and related disorders","volume":"94 ","pages":"Article 106306"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143075085","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":"Longitudinally extensive transverse myelitis: Impact on functional prognosis and mortality in a 10-year follow-up cohort","authors":"Paula Baleeiro Rodrigues Silva ,&nbsp;Samira Luisa Apóstolos Pereira ,&nbsp;Graziella Aguiar ,&nbsp;Sara Terrim ,&nbsp;Flávio Vieira Marques Filho ,&nbsp;Guilherme Diogo Silva ,&nbsp;Herval Neto ,&nbsp;Mateus Boaventura ,&nbsp;Tarso Adoni ,&nbsp;Douglas Kazutoshi Sato ,&nbsp;Alessandra Dellavance ,&nbsp;Paulo Eurípedes Marchiori ,&nbsp;Dagoberto Callegaro","doi":"10.1016/j.msard.2025.106279","DOIUrl":"10.1016/j.msard.2025.106279","url":null,"abstract":"<div><div>Background: Neuromyelitis optica spectrum disorder (NMOSD) with positivity for aquaporin-4 antibody (AQP4-IgG) represents one of the mains etiologies of longitudinally extensive transverse myelitis (LETM). Advancements in early diagnosis and treatment have led to a decline in NMOSD-related mortality. However, long-term prognostic data for patients experiencing their first episode of LETM remain scarce, especially in Brazil. This study aims to evaluate the final diagnosis and long-term prognosis of patients with first episode of LETM and investigate factors associated with worse prognosis. Methods: This is an observational retrospective study involving all consecutive patients diagnosed with longitudinally extensive myelopathy who were sequentially referred to the clinical neurology department of a brazilian tertiary hospital between January 2005 and December 2011. Only patients meeting the criteria for the first episode of idiopathic LETM were included. Data were retrieved from electronic medical records from October 2023 to January 2024. Results: 39 patients met the inclusion criteria. After a median follow-up of 12 years, 51% patients remained with isolated monophasic seronegative LETM, 28% were diagnosed with NMOSD AQP4-IgG positive, 7.7% with NMOSD AQP4-IgG negative, 5% with MOGAD, 5% experienced recurrent seronegative LETM, and only 1 (2.6%) developed multiple sclerosis. The mortality rate was 10% at last follow-up, with a median time to death of 3 years. Deceased patients had a higher age at onset of LETM (OR 1.09, 95% CI 1.01–1.21). Among survivors, 17% had an Expanded Disability Status Scale (EDSS) ≥7 at last follow-up. Predictors of severe sequelae included higher EDSS at nadir (OR 5.29; 95% CI 1.38–39), pain as an initial myelitis symptom (OR 11.1; 95% CI 1.51–230) and spinal shock during the first myelitis (p &lt; 0.001). Conclusion: In this cohort, after a median 12-year follow-up, half of the patients remained as isolated monophasic seronegative LETM, mortality reached 10% and 83% of survivors were ambulatory. Predictors of poor prognosis included older age, presence of pain as an initial symptom and higher initial severity.</div></div>","PeriodicalId":18958,"journal":{"name":"Multiple sclerosis and related disorders","volume":"94 ","pages":"Article 106279"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143075113","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":"Allopurinol use and risk of multiple sclerosis","authors":"Ida M. Heerfordt ,&nbsp;Elisabeth Framke ,&nbsp;Josefine Windfeld-Mathiasen ,&nbsp;Melinda Magyari ,&nbsp;Henrik Horwitz","doi":"10.1016/j.msard.2025.106299","DOIUrl":"10.1016/j.msard.2025.106299","url":null,"abstract":"<div><h3>Background</h3><div>Low uric acid levels have been observed in patients with multiple sclerosis (MS). As allopurinol is commonly used to lower uric acid in gout patients, this study examined allopurinol use in individuals with MS during the five years before diagnosis, compared to a matched control group.</div></div><div><h3>Methods</h3><div>This nationwide case-control study used Danish health registry data. 9,063 individuals diagnosed with MS between 2008 and 2023 were included on the day of MS diagnosis, while 90,630 controls, were matched from the general population. Controls were matched on age, gender, place of residence, and inclusion date. Allopurinol prescriptions were tracked annually for five years prior to the inclusion date. The primary outcome was the odds ratio of allopurinol use in MS patients compared to controls, analyzed using conditional logistic regression.</div></div><div><h3>Results</h3><div>In the five years prior to the MS diagnosis or matching date for controls, allopurinol use was consistently lower in MS cases. In total, 0.30% (<em>n</em> = 27) of MS cases had at least one allopurinol prescription, compared to 0.62% (<em>n</em> = 558) of controls, corresponding to an odds ratio of 0.48 (95% CI: 0.33–0.71).</div></div><div><h3>Conclusions</h3><div>Individuals diagnosed with MS had significantly lower allopurinol use in the five years preceding their diagnosis, suggesting an inverse relationship between uric acid levels and MS risk. Reduced uric acid levels may be associated with early MS development, possibly due to its antioxidant properties or metabolic changes. Alternatively, these findings suggest a possible protective effect of allopurinol against MS, warranting further investigation.</div></div>","PeriodicalId":18958,"journal":{"name":"Multiple sclerosis and related disorders","volume":"94 ","pages":"Article 106299"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143341465","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy and safety of disease-modifying therapies in pediatric-onset multiple sclerosis: A systematic review of clinical trials and observational studies","authors":"Katarzyna Śladowska ,&nbsp;Paweł Moćko ,&nbsp;Tomasz Brzostek ,&nbsp;Paweł Kawalec","doi":"10.1016/j.msard.2025.106263","DOIUrl":"10.1016/j.msard.2025.106263","url":null,"abstract":"<div><h3>Objective</h3><div>This study aimed to review the efficacy and safety profile of disease-modifying therapies (DMTs) in patients with relapsing pediatric-onset multiple sclerosis (POMS).</div></div><div><h3>Methods</h3><div>A systematic review was performed according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Published randomized controlled trials (RCTs), nonrandomized studies with a control group, large single-arm studies, and ongoing (unpublished) studies investigating the use of approved and unapproved DMTs in POMS were included. Eligible published studies were identified in MEDLINE (via PubMed), EMBASE, and the Cochrane Library, and unpublished studies were identified in a clinical trials registry (<span><span>www.clinicaltrials.gov</span><svg><path></path></svg></span>).</div></div><div><h3>Results</h3><div>A total of 13 published studies were included in the systematic review: 4 RCTs, 3 observational studies with a control group, and 6 large single-arm studies. The following DMTs for the treatment of POMS were evaluated in the included studies: interferon beta-1a, interferon beta-1b, teriflunomide, dimethyl fumarate, fingolimod, natalizumab, glatiramer acetate, and ocrelizumab. All DMTs were shown to be effective in reducing relapse rates, preventing disability progression, and reducing disease activity in MRI in patients with POMS. DMTs that are considered highly effective in adults with multiple sclerosis (natalizumab, fingolimod) were also shown to be more effective than interferon beta-1a in POMS. A total of 9 ongoing (unpublished) studies were identified, including 5 RCTs. The following drugs were evaluated: ozanimod, fingolimod, peginterferon beta-1a, ocrelizumab, ofatumumab, siponimod, alemtuzumab, and natalizumab.</div></div><div><h3>Conclusion</h3><div>The number of DMTs approved for the treatment of POMS is limited, and some of the available DMTs are used off-label. The available evidence from published studies of varying reliability supports the efficacy of DMTs in POMS. However, well-designed, long-term RCTs in the pediatric population are needed. The results of ongoing studies may fill the existing gap in clinical evidence, possibly leading to the approval of more highly effective DMTs for patients with POMS.</div></div>","PeriodicalId":18958,"journal":{"name":"Multiple sclerosis and related disorders","volume":"94 ","pages":"Article 106263"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142979209","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":"Recommendations on NMOSD treatment in Latin America: Utilizing consensus-based RAND/UCLA methodology","authors":"Ricardo Alonso ,&nbsp;Victor Rivera ,&nbsp;Edgar German Carnero Contentti ,&nbsp;Victor Fernando Hamuy Diaz de Bedoya ,&nbsp;Ethel Ciampi ,&nbsp;Berenice Silva ,&nbsp;Maria Zuluaga ,&nbsp;Jefferson Becker ,&nbsp;Juan Ignacio Rojas ,&nbsp;Fernando Gracia ,&nbsp;Luis Zarco ,&nbsp;Veronica Tkachuk ,&nbsp;Fernando Molt ,&nbsp;Ibis Soto ,&nbsp;Ramiro Fernandez Calderon ,&nbsp;Pablo López ,&nbsp;Liliana Patrucco ,&nbsp;Irene Treviño-Frenk ,&nbsp;Douglas Sato ,&nbsp;Deyanira Ramirez ,&nbsp;Lorna Galleguillos","doi":"10.1016/j.msard.2024.106244","DOIUrl":"10.1016/j.msard.2024.106244","url":null,"abstract":"<div><h3>Introduction</h3><div>Neuromyelitis optica spectrum disorder (NMOSD) is a serious condition affecting people worldwide, including Latin America (LATAM). Healthcare disparities and economic limitations make effective treatment access challenging. It is crucial to consider the best practice therapeutic decision-making, including emerging long-term preventive therapies, to ensure patients in LATAM and elsewhere can effectively manage their disease all over the world.</div></div><div><h3>Objectives/Aims</h3><div>To establish evidence-based guidelines for treatment approaches in NMOSD patients in LATAM. A consensus process was conducted to develop validated statements.</div></div><div><h3>Methods</h3><div>A group of NMOSD experts from LATAM utilized a 9-point Likert scale to vote on statements related to NMOSD management. The predetermined consensus threshold was set at a minimum expert agreement of 70 %. The RAND/UCLA methodology was employed to reach consensus recommendations.</div></div><div><h3>Results</h3><div>Nineteen experts completed the consensus process between March and April 2023. In round 1, no statement failed to reach the predetermined consensus, resulting in 31 agreed statements. The statements were divided between general recommendations (14/31) and clinical scenarios (17/31). The scenarios were based on treatment-naïve NMOSD AQP4 positive patients (4/17); treatment-naive seronegative NMOSD patients (4/17); treatment switching (5/17) and safety (4/17).</div></div><div><h3>Conclusions</h3><div>Consensus recommendations were developed on the most important areas of NMOSD treatment by a panel of experts in LATAM. These statements are a valuable tool to guide decision-making and improve patient outcomes, serving as the foundation for developing standardized practice guidelines in our region.</div></div>","PeriodicalId":18958,"journal":{"name":"Multiple sclerosis and related disorders","volume":"94 ","pages":"Article 106244"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143008027","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":"Effect of siponimod on retinal thickness, a marker of neurodegeneration, in participants with SPMS: Findings from the EXPAND OCT substudy","authors":"Patrick Vermersch ,&nbsp;Ralf Gold ,&nbsp;Amit Bar-Or ,&nbsp;Bruce A.C. Cree ,&nbsp;Robert J. Fox ,&nbsp;Gavin Giovannoni ,&nbsp;Friedemann Paul ,&nbsp;Sebastian Wolf ,&nbsp;Bingbing Li ,&nbsp;Marie-Catherine Mousseau ,&nbsp;Tina Maio-Twofoot ,&nbsp;Xiaofang Shi ,&nbsp;Ludwig Kappos","doi":"10.1016/j.msard.2025.106259","DOIUrl":"10.1016/j.msard.2025.106259","url":null,"abstract":"<div><h3>Background</h3><div>People with MS show abnormal thinning of the retinal layers, which is associated with clinical disability and brain atrophy, and is a potential surrogate marker of neurodegeneration and treatment effects.</div></div><div><h3>Objective</h3><div>To evaluate the utility of retinal thickness as a surrogate marker of neurodegeneration and treatment effect in participants with secondary progressive MS (SPMS) from the optical coherence tomography (OCT) substudy of the EXPAND Phase 3 clinical trial (siponimod versus placebo).</div></div><div><h3>Methods</h3><div>In the OCT substudy population (<em>n</em> = 159), treatment effects on change in the average thickness of the retinal layer, peripapillary retinal nerve fiber layer (pRNFL), and combined macular ganglion cell and inner plexiform layers (GCIPL) were analyzed by high-definition spectral domain OCT at months 3, 12, and 24.</div></div><div><h3>Results</h3><div>Thinning from baseline was observed across all retinal layers and time points in the placebo group. Siponimod significantly reduced GCIPL thinning versus placebo at month 24 (adjusted mean [SE] [µm]: −0.47 [0.81] vs. −4.29 [1.23]; <em>p</em> = 0.01), and overall retinal thinning at months 12 (+0.66 [0.54] vs. −1.86 [0.75]; <em>p</em> = 0.006) and 24 (−0.05 [0.59] vs. −2.30 [0.88]; <em>p</em> = 0.033). Although not significant, results for pRNFL consistently followed the same trends.</div></div><div><h3>Conclusion</h3><div>This exploratory substudy supports further investigation of OCT measurement of retinal atrophy as a non-invasive potential biomarker of treatment effects on neurodegeneration in SPMS.</div></div>","PeriodicalId":18958,"journal":{"name":"Multiple sclerosis and related disorders","volume":"94 ","pages":"Article 106259"},"PeriodicalIF":2.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143029202","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}