Neurology and Therapy最新文献

筛选
英文 中文
Phase 1 Trials of Gatralimab, a Next-Generation Humanized Anti-CD52 Monoclonal Antibody, in Participants with Progressive Multiple Sclerosis. 新一代人源化抗 CD52 单克隆抗体 Gatralimab 在进展性多发性硬化症患者中的 1 期试验。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-09-09 DOI: 10.1007/s40120-024-00659-w
Fredrik N Albach, Christian Geier, Christian Keicher, Maximilian G Posch, Stephan J Schreiber, Gerald Grütz, Levent Akyüz, Xiaodong Luo, Annaig Le-Halpere, Philippe Truffinet, Frank Wagner
{"title":"Phase 1 Trials of Gatralimab, a Next-Generation Humanized Anti-CD52 Monoclonal Antibody, in Participants with Progressive Multiple Sclerosis.","authors":"Fredrik N Albach, Christian Geier, Christian Keicher, Maximilian G Posch, Stephan J Schreiber, Gerald Grütz, Levent Akyüz, Xiaodong Luo, Annaig Le-Halpere, Philippe Truffinet, Frank Wagner","doi":"10.1007/s40120-024-00659-w","DOIUrl":"10.1007/s40120-024-00659-w","url":null,"abstract":"<p><strong>Introduction: </strong>Lymphocyte depletion via anti-CD52 monoclonal antibody (mAb) therapy is an effective treatment strategy for relapsing-remitting multiple sclerosis (MS) but is associated with infusion/injection-associated reactions (IARs) and autoimmune-related adverse events (AEs). Gatralimab is a next-generation humanized anti-CD52 mAb.</p><p><strong>Methods: </strong>Two first-in-human trials were conducted in participants with progressive MS to assess the pharmacodynamics, pharmacokinetics, and safety of gatralimab administered via subcutaneous (SC) and intravenous (IV) routes, and to determine the effect of different comedication regimes on IARs to SC gatralimab. A Phase 1 trial (NCT02282826) included double-blind, placebo-controlled sequential ascending single IV (1, 3.5, and 12 mg) and SC (12, 36, and 60 mg) dose groups. A Phase 1b trial (NCT02977533) involved five groups who received SC gatralimab (36, 48, or 60 mg) and different comedications. A long-term safety (LTS) study (NCT02313285) examined safety and pharmacodynamics over 4 years.</p><p><strong>Results: </strong>Gatralimab produced depletion of lymphocytes (dose-dependently) and CD4+ regulatory T cells, with partial repopulation to normal values by approximately 12 months. Peak serum gatralimab concentrations followed dose-proportionality and were delayed by 6.0-7.5 days following SC administration. Treatment-emergent AEs, including IARs, were reported for most participants but were generally of mild or moderate severity, and treatment-emergent serious AEs were mostly MS-related. Methylprednisolone and antihistamine comedications were associated with reduced incidence of fevers and skin and subcutaneous tissue AEs, respectively. During the LTS study, one participant (3.0%) experienced an autoimmune-related AE (Basedow's disease), and subsequently died from pulmonary sepsis deemed unrelated to gatralimab by the investigator.</p><p><strong>Conclusions: </strong>These data show that gatralimab achieves the desired pharmacodynamic effect of lymphocyte depletion followed by repopulation, and has an acceptable safety profile, including low risk of non-MS autoimmunity. Although gatralimab is no longer in development for MS, insights from these trials may inform the development of comedication regimes of future anti-CD52 mAbs and subcutaneous formulations of other lymphocyte-depleting mAbs.</p><p><strong>Trial registration: </strong>NCT02282826, NCT02977533, NCT02313285.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1607-1625"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541066/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142292223","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Navigating the Landscape of Plasma Biomarkers in Alzheimer's Disease: Focus on Past, Present, and Future Clinical Applications. 阿尔茨海默病血浆生物标志物的导航:聚焦过去、现在和未来的临床应用。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-09-07 DOI: 10.1007/s40120-024-00658-x
Sarrah E Ankeny, Julia R Bacci, Boris Decourt, Marwan N Sabbagh, Michelle M Mielke
{"title":"Navigating the Landscape of Plasma Biomarkers in Alzheimer's Disease: Focus on Past, Present, and Future Clinical Applications.","authors":"Sarrah E Ankeny, Julia R Bacci, Boris Decourt, Marwan N Sabbagh, Michelle M Mielke","doi":"10.1007/s40120-024-00658-x","DOIUrl":"10.1007/s40120-024-00658-x","url":null,"abstract":"<p><p>As the prevalence of Alzheimer's disease (AD) and its impact on healthcare systems increase, developing tools for accurate diagnosis and monitoring of disease progression is a priority. Recent technological advancements have allowed for the development of blood-based biomarkers (BBMs) to aid in the diagnosis of AD, but many questions remain regarding the clinical implementation of these BBMs. This review outlines the historical timeline of AD BBM development. It highlights key breakthroughs that have transformed the perspective of AD BBMs from theoretically ideal but unattainable markers, to clinically valid and reliable BBMs with potential for implementation in healthcare settings. Technological advancements like single-molecule detection and mass spectrometry methods have significantly improved assay sensitivity and accuracy. High-throughput, fully automated platforms have potential for clinical use. Despite these advancements, however, significant work is needed before AD BBMs can be implemented in widespread clinical practice. Cutpoints must be established, the influence of chronic conditions and medications on BBM levels must be better understood, and guidelines must be created for healthcare providers related to interpreting and communicating information obtained from AD BBMs. Additionally, the development of BBMs for synaptic dysfunction, inflammation, and cerebrovascular disease may provide better precision medicine approaches to treating AD and related dementia. Future research and collaboration between scientists and physicians are essential to addressing these challenges and further advancing AD BBMs, with the goal of integration in clinical practice.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1541-1557"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541985/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142146067","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Development of a General Composite Scale (GENCOMS) for Progressive Neurodegenerative Diseases and Implications for the Assessment of Disease-Modifying Therapies. 针对渐进性神经退行性疾病的通用综合量表(GENCOMS)的开发及其对疾病调整疗法评估的影响。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-09-17 DOI: 10.1007/s40120-024-00661-2
Samuel P Dickson, Craig H Mallinckrodt, Basia Rogula, Lauren C Powell, Michele H Potashman, Vladimir Coric, Gilbert J L'Italien, Suzanne B Hendrix
{"title":"Development of a General Composite Scale (GENCOMS) for Progressive Neurodegenerative Diseases and Implications for the Assessment of Disease-Modifying Therapies.","authors":"Samuel P Dickson, Craig H Mallinckrodt, Basia Rogula, Lauren C Powell, Michele H Potashman, Vladimir Coric, Gilbert J L'Italien, Suzanne B Hendrix","doi":"10.1007/s40120-024-00661-2","DOIUrl":"10.1007/s40120-024-00661-2","url":null,"abstract":"<p><strong>Introduction: </strong>The reliable assessment of treatment outcomes for disease-modifying therapies (DMT) in neurodegenerative disease is challenging. The objective of this paper is to describe a generalized framework for developing composite scales that can be applied in diverse, degenerative conditions, termed \"GENCOMS.\" Composite scales optimize the sensitivity for detecting clinically meaningful effects that slow disease progression.</p><p><strong>Methods: </strong>The GENCOMS method relies on robust natural history data and/or placebo arm data from DMT trials. Validated scales that are core to the disease process have been identified, and item level data obtained to standardize the response outcomes from 0 (best possible score) to 1 (worst possible score). A partial least squares regression analysis was conducted with temporal change as the dependent variable and change scores in standardized items as the explanatory variables. The derived model coefficients constitute a weighted sum of items that most effectively measure disease progression.</p><p><strong>Results: </strong>The resultant composite scale was optimized to detect disease progression and can be examined in a range of slow or fast progressing populations. The scale can be used in studies with comparable patient populations as an endpoint optimized to measure disease progression and therefore ideally suited to assess treatment effects in DMTs.</p><p><strong>Conclusion: </strong>The methodology presented here provides a generalizable framework for developing composite scales in the assessment of neurodegenerative disease progression and evaluation of DMT effects. By objectively selecting and weighting items from previously validated measures based solely on their sensitivity to disease progression, this methodology allows for the creation of a more responsive measurement of clinical decline. This heightened sensitivity to clinical decline can be utilized to detect modest yet meaningful treatment effects in the early stages of neurogenerative diseases, when it is optimal to begin a DMT.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1627-1639"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541966/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142292221","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
What Is Combination Treatment in Migraine? Moving Toward a Uniform Definition of a Familiar Principle. 什么是偏头痛的综合治疗?为一个熟悉的原则制定统一的定义。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-10-15 DOI: 10.1007/s40120-024-00669-8
Richard B Lipton, Jessica Ailani, Andrew M Blumenfeld
{"title":"What Is Combination Treatment in Migraine? Moving Toward a Uniform Definition of a Familiar Principle.","authors":"Richard B Lipton, Jessica Ailani, Andrew M Blumenfeld","doi":"10.1007/s40120-024-00669-8","DOIUrl":"10.1007/s40120-024-00669-8","url":null,"abstract":"","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1535-1540"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541989/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142470989","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Patient and Physician Perspectives of Treatment Burden in Multiple Sclerosis. 多发性硬化症患者和医生对治疗负担的看法。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-09-04 DOI: 10.1007/s40120-024-00654-1
Barry A Singer, Dawn Morgan, Julie A Stamm, Anita A Williams
{"title":"Patient and Physician Perspectives of Treatment Burden in Multiple Sclerosis.","authors":"Barry A Singer, Dawn Morgan, Julie A Stamm, Anita A Williams","doi":"10.1007/s40120-024-00654-1","DOIUrl":"10.1007/s40120-024-00654-1","url":null,"abstract":"<p><p>The number of disease-modifying therapies (DMTs) approved for the treatment of multiple sclerosis (MS) has greatly increased in recent decades, leading to higher treatment complexity. DMTs can differ in mode and frequency of administration, benefit-risk profile, and associated costs. Patients with MS contend not only with the burden of their chronic disease but also with the treatment burden of their MS therapy. Adhering to dosing schedules and infusion appointments can be difficult for busy, working-age patients or those with limited access to transportation. Patients and healthcare professionals (HCPs) may have differing priorities, concerns, and preferences when selecting treatment, potentially affecting treatment satisfaction and, importantly, adherence. Additionally, patients face direct and indirect costs related to treatment. These factors can all contribute to a high treatment burden on patients, impacting their quality of life and potentially leading to worse patient outcomes. HCPs, patients, and caregivers must work together to alleviate treatment burden through effective communication, shared decision-making, appreciating each other's perspectives, and additional HCP support. Consideration of treatment burden into clinical guidelines is also warranted. In this review, we examine key factors impacting treatment burden for patients with MS, with a focus on the patient perspective as provided by our patient authors, and provide strategies to minimize treatment burden.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1507-1525"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541994/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142126276","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Discrete-Choice Experiment Assessing the Patient Preferences and Real-World Experiences of Patients with Migraine in Japan. 一项离散选择实验,评估日本偏头痛患者的偏好和真实世界的经历。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-09-30 DOI: 10.1007/s40120-024-00663-0
Takao Takeshima, Akira Yuasa, Yukie Michelle Lloyd, Louis Patrick Watanabe, Kazumasa Kamei, Akiho Terasawa, Josh Coulter, Lucy Abraham, Brett Hauber, Masahiro Iijima
{"title":"A Discrete-Choice Experiment Assessing the Patient Preferences and Real-World Experiences of Patients with Migraine in Japan.","authors":"Takao Takeshima, Akira Yuasa, Yukie Michelle Lloyd, Louis Patrick Watanabe, Kazumasa Kamei, Akiho Terasawa, Josh Coulter, Lucy Abraham, Brett Hauber, Masahiro Iijima","doi":"10.1007/s40120-024-00663-0","DOIUrl":"10.1007/s40120-024-00663-0","url":null,"abstract":"<p><strong>Introduction: </strong>Migraine is a debilitating headache disorder with a high prevalence in Japan that imposes significant societal burden. Although the Japanese Clinical Practice Guideline for Headache Disorders 2021 recommends both acute and preventive migraine treatments, the usage of preventive treatments is still limited. Therefore, it is crucial to understand the treatment preferences of patients with migraine pertaining to both acute and preventive treatments.</p><p><strong>Methods: </strong>A mixed-methods study including a discrete choice experiment (DCE) was conducted with Japanese patients with migraine (10 for the qualitative interviews, and 400 for the DCE) who were recruited from the Rakuten Insight panel. The DCE presented hypothetical treatment options including oral acute, oral preventive, and injectable preventive medications. Six attributes (method of delivery, reduction of pain, impact of headaches on daily routines, dosage adjustability, and temporary and persistent side effects) each with three levels were included in the survey. A hierarchical Bayesian model was used to estimate relative attribute importance scores (RAI) for all attributes.</p><p><strong>Results: </strong>For the 400 participants in the DCE, the most common age bracket was 40-49 years old, and the majority were female (66.75%). RAI estimates indicated that \"method of delivery\" was the most important attribute for patients (RAI 51.92, SD = 10.20), followed by \"reduction of pain when experiencing a headache\" (RAI 17.00, SD = 7.74). Oral preventive treatments were preferred over injectable preventive treatments. The qualitative interviews showed that patients prefer oral medications to injectable treatments, and a lack of awareness regarding preventive treatments.</p><p><strong>Conclusion: </strong>This study found that the \"method of delivery\" was the most important driver of treatment preferences of patients with migraine in Japan, with oral acute medications being preferred. Oral preventive treatments were found to be preferred over injectable treatments. These results may indicate the need for increased education regarding preventive treatments, as well as the need for further development of these treatments.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1661-1683"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541993/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142350926","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Real-World Persistence with Ocrelizumab in Multiple Sclerosis: a Systematic Review. 多发性硬化症患者使用奥克立珠单抗的真实世界持久性:系统综述。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-10-19 DOI: 10.1007/s40120-024-00667-w
John L Petrie, Charlie A Smith, Donna Fountain, Gerardo Machnicki
{"title":"Real-World Persistence with Ocrelizumab in Multiple Sclerosis: a Systematic Review.","authors":"John L Petrie, Charlie A Smith, Donna Fountain, Gerardo Machnicki","doi":"10.1007/s40120-024-00667-w","DOIUrl":"10.1007/s40120-024-00667-w","url":null,"abstract":"<p><p>In clinical trials, the percentage of patients discontinuing treatment with ocrelizumab due to adverse events was low. However, real-world populations are often more diverse than randomized controlled trials (RCTs), therefore it is important to assess discontinuation rates in real-world studies. This systematic literature review (SLR) was conducted to identify real-world discontinuation and persistence data for ocrelizumab in studies of patients with relapsing remitting multiple sclerosis (RRMS) and primary progressive multiple sclerosis (PPMS). Searches were conducted in MEDLINE and Embase to identify relevant real-world studies that met pre-determined Population, Intervention, Comparison, Outcomes, and Study (PICOS) criteria. Only articles published in English were included, but the study country was not restricted. A total of 30 studies were included, with the majority reporting real-world persistence data that appear to be similar to or better than in the pivotal clinical trials, with only 1 study reporting higher discontinuation rates due to adverse events compared with the clinical trials. Other studies identified reported that the risk of discontinuation was higher for other disease-modifying therapies (DMTs) compared with ocrelizumab, and adherence was also higher for ocrelizumab versus other DMTs. These findings have clinical relevance, as other studies have reported improved clinical outcomes and lower care costs for patients that are persistent or adherent to other DMTs.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1597-1605"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541982/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142470987","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Association Between Constipation and Positron Emission Tomography and Blood-Based Biomarkers in Older Cognitively Unimpaired Adults with Higher Amyloid-β Burden. 淀粉样蛋白-β负荷较高的认知功能未受损的老年人便秘与正电子发射断层扫描和血液生物标志物之间的关系
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-10-22 DOI: 10.1007/s40120-024-00666-x
Heling Chu, Chuyi Huang, Fang Xie, Qihao Guo
{"title":"The Association Between Constipation and Positron Emission Tomography and Blood-Based Biomarkers in Older Cognitively Unimpaired Adults with Higher Amyloid-β Burden.","authors":"Heling Chu, Chuyi Huang, Fang Xie, Qihao Guo","doi":"10.1007/s40120-024-00666-x","DOIUrl":"10.1007/s40120-024-00666-x","url":null,"abstract":"<p><strong>Introduction: </strong>Constipation may be linked to cognitive decline and a higher risk of Alzheimer's disease (AD). We aimed to investigate the association between constipation and positron emission tomography (PET) and blood-based AD biomarkers in older cognitively unimpaired (CU) adults with higher Aβ burden.</p><p><strong>Methods: </strong>Constipation was diagnosed according to Rome IV criteria and the severity of constipation was evaluated by using a validated self-reported questionnaire. The participants underwent the examination of plasma AD biomarkers and <sup>18</sup>F-florbetapir PET and <sup>18</sup>F-MK6240 PET scans; the latter was only performed in the validation cohort. Correlation and multiple linear regression analyses were used to investigate the association between constipation and AD biomarkers.</p><p><strong>Results: </strong>Two cohorts were included in our study. A total of 404 older participants with 126 of whom Aβ-PET positive were enrolled in the development cohort. Multiple linear regression analysis showed constipation was associated with plasma t-Tau, p-Tau-181, and neurofilament light chain (NfL) in participants with Aβ-PET (+). Meanwhile, no/mild constipation was associated with lower Aβ-PET standard uptake value ratio. The association between constipation and plasma biomarkers was different in the subgroups stratified by age, sex and APOE ε4 genotype. The above associations were further validated in the validation cohort containing 36 Aβ-PET (+) participants. Importantly, no/mild constipation was associated with less Tau burden evaluated by <sup>18</sup>F-MK6240 PET Braak stages.</p><p><strong>Conclusion: </strong>Our data indicate that no/mild constipation may be associated with lower plasma t-Tau, p-Tau-181, and NfL as well as less Aβ and Tau burden in older CU adults with Aβ deposition. Improving constipation and being away from defecation disorders may help reduce the risk of AD development.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1701-1715"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541973/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142470988","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
COVID-19 Vaccination Response in Patients with Multiple Sclerosis Treated with Ofatumumab in the United States: A Medical Record Review. 美国接受奥法妥木单抗治疗的多发性硬化症患者对 COVID-19 疫苗接种的反应:病历回顾。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-10-23 DOI: 10.1007/s40120-024-00671-0
Rahul H Dave, Heidi Crayton, Augusto Miravalle, Ming-Hui Tai, Kerri Wyse, Katherine Houghton, Abby Hitchens, Regina Berkovich
{"title":"COVID-19 Vaccination Response in Patients with Multiple Sclerosis Treated with Ofatumumab in the United States: A Medical Record Review.","authors":"Rahul H Dave, Heidi Crayton, Augusto Miravalle, Ming-Hui Tai, Kerri Wyse, Katherine Houghton, Abby Hitchens, Regina Berkovich","doi":"10.1007/s40120-024-00671-0","DOIUrl":"10.1007/s40120-024-00671-0","url":null,"abstract":"<p><strong>Introduction: </strong>Real-world data are required to provide a greater understanding of the impact of ofatumumab on the ability to mount an effective immune response following the receipt of approved COVID-19 vaccinations. This retrospective real-world analysis aimed to describe the humoral immune response to COVID-19 vaccination during ofatumumab treatment in patients with multiple sclerosis (MS).</p><p><strong>Methods: </strong>Data from patients with MS treated with ofatumumab who were fully vaccinated against COVID-19 infection were abstracted from medical charts at four clinical sites in the USA. Patient characteristics and humoral response were summarized descriptively. Differences in humoral response were documented on the basis of vaccination status during ofatumumab treatment (i.e., after full vaccination and after booster vaccination) and prior disease-modifying treatment (DMT) exposure (i.e., DMT naïve, prior anti-CD20/sphingosine 1-phosphate [S1P] therapy, prior non-anti-CD20/S1P therapy). The sample size precluded formal statistical analysis.</p><p><strong>Results: </strong>Thirty-eight patients were included. The mean (standard deviation) duration of ofatumumab treatment upon data collection was 20.4 (4.6) months (treatment ongoing for 35 [92%] patients). Definitive humoral response after full vaccination was documented for 34 patients, of whom 20 (60%) were seropositive. Definitive humoral response after booster vaccination was documented among five patients, of whom three (60%) were seropositive. Among patients who were DMT naïve prior to ofatumumab (n = 15), 73% were seropositive; among patients exposed to prior anti-CD20/S1P therapy (n = 14), 33% were seropositive; and among patients exposed to prior non-anti-CD20/S1P therapy (n = 9), 56% were seropositive. Patients naïve to DMT had been living with an MS diagnosis for a shorter duration than those experienced with DMTs.</p><p><strong>Conclusion: </strong>Patients with MS receiving ongoing treatment with ofatumumab can mount a positive humoral response to a COVID-19 vaccination. Prior treatment with anti-CD20 or S1P DMTs may be a risk factor for lower humoral response.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1737-1745"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541983/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142504886","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Symptoms of Hereditary Transthyretin Amyloidosis: The Patient and Physician Perspective. 遗传性转甲状腺素淀粉样变性的症状:患者和医生的视角。
IF 3.9 3区 医学
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-09-06 DOI: 10.1007/s40120-024-00657-y
Michael Lane, Michael Polydefkis
{"title":"Symptoms of Hereditary Transthyretin Amyloidosis: The Patient and Physician Perspective.","authors":"Michael Lane, Michael Polydefkis","doi":"10.1007/s40120-024-00657-y","DOIUrl":"10.1007/s40120-024-00657-y","url":null,"abstract":"<p><p>This article has been co-authored by a patient living with hereditary transthyretin (ATTRv) amyloidosis and a neurologist. This rare, progressive disease is associated with impairment of multiple organ systems, including the nerves, heart, and the gastrointestinal tract, forcing patients to live with and adapt to a range of debilitating symptoms. Here, the patient and physician discuss how the symptoms of ATTRv amyloidosis profoundly impact day to day life, the difficulties with identifying the disease, and how this effects the diagnosis experience. In recent years, significant advancements have been made in the treatment and management of ATTRv amyloidosis. However, the authors highlight the urgency of increasing awareness of the disease among the wider medical community, as well as in patients who notice the symptoms, to ensure that earlier diagnosis and appropriate treatment are achieved.</p>","PeriodicalId":19216,"journal":{"name":"Neurology and Therapy","volume":" ","pages":"1527-1533"},"PeriodicalIF":3.9,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11541984/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142140655","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
相关产品
×
本文献相关产品
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信