Therapeutic Advances in Neurological Disorders最新文献

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A rare adverse effect in inebilizumab therapy for neuromyelitis optica spectrum disorder: a case report. 伊维单抗治疗神经脊髓炎视谱系障碍的罕见不良反应:病例报告。
IF 4.7 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-07-26 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241258787
Xuefen Chen, Ziyan Shi, Rui Wang, Hongyu Zhou
{"title":"A rare adverse effect in inebilizumab therapy for neuromyelitis optica spectrum disorder: a case report.","authors":"Xuefen Chen, Ziyan Shi, Rui Wang, Hongyu Zhou","doi":"10.1177/17562864241258787","DOIUrl":"10.1177/17562864241258787","url":null,"abstract":"<p><p>Inebilizumab is one of the monoclonal antibodies approved as maintenance therapy for aquaporin-4 immunoglobulin G-seropositive neuromyelitis optica spectrum disorder (NMOSD). It is a humanized monoclonal antibody targeting cluster of differentiation 19 (CD19). Common adverse reactions include urinary tract infections, nasopharyngitis, arthralgia, infusion reactions, headaches and a decrease in immunoglobulin levels. Here, we present a case of an NMOSD patient who experienced transient hyperCKaemia after the use of inebilizumab. The adverse reactions of this very rare monoclonal antibody drug improved after discontinuation.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241258787"},"PeriodicalIF":4.7,"publicationDate":"2024-07-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11282551/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141789110","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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 safety and efficacy profile of eculizumab in myasthenic crisis: a prospective small case series. 依库珠单抗治疗肌无力危象的安全性和疗效:前瞻性小型病例系列研究。
IF 4.7 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-07-26 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241261602
Jie Song, Xiao Huan, Yuanyi Chen, Yeting Luo, Huahua Zhong, Yuan Wang, Lei Yang, Caihua Xi, Yu Yang, Jianying Xi, Jianming Zheng, Zongtai Wu, Chongbo Zhao, Sushan Luo
{"title":"The safety and efficacy profile of eculizumab in myasthenic crisis: a prospective small case series.","authors":"Jie Song, Xiao Huan, Yuanyi Chen, Yeting Luo, Huahua Zhong, Yuan Wang, Lei Yang, Caihua Xi, Yu Yang, Jianying Xi, Jianming Zheng, Zongtai Wu, Chongbo Zhao, Sushan Luo","doi":"10.1177/17562864241261602","DOIUrl":"10.1177/17562864241261602","url":null,"abstract":"<p><p>Eculizumab has improved recovery from ventilatory support in myasthenic crisis (MC) cases. However, the safety and efficacy profiles from prospective studies are still lacking. This study aimed to explore eculizumab's safety and efficacy in a prospective case series of patients with refractory MC. We followed a series of anti-acetylcholine receptor (AChR) antibody-positive myasthenia gravis (MG) patients who received eculizumab as an add-on therapy for 12 weeks during MC to facilitate the weaning process and reduced disease activity. Serum anti-AChR antibodies and peripheral immune molecules associated with the complement pathway were evaluated before and after eculizumab administration. Compared to the baseline Myasthenia Gravis Foundation of America (MGFA)-quantitative MG test (QMG) scores (22.25 ± 4.92) and MG-activities of daily living (MG-ADL; 18.25 ± 2.5) scores at crisis, improvements were observed from 4 weeks (14.5 ± 10.47 and 7.5 ± 7.59, respectively) through 12 weeks (7.5 ± 5.74 and 2.25 ± 3.86, respectively) post-treatment. Muscle strength consistently improved across ocular, bulbar, respiratory, and limb/gross domain groups. One patient died of cardiac failure at 16 weeks. Three cases remained in remission at 24 weeks, with a mean QMG score of 2.67 ± 2.89 and ADL score of 0.33 ± 0.58. No significant side effects were reported. Serum CH50 and soluble C5b-9 levels significantly declined, while there were no significant changes in serum anti-AChR antibody levels, C1q, C5a levels, or peripheral lymphocyte proportions. Eculizumab was well tolerated and showed efficacy in this case series. Large prospective cohort studies with extended follow-up periods are needed to further explore the safety and efficacy profile in real-world practice.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241261602"},"PeriodicalIF":4.7,"publicationDate":"2024-07-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11282533/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141789111","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Therapeutic strategies during cenobamate treatment initiation: Delphi panel recommendations. 开始使用仙诺巴马特治疗期间的治疗策略:德尔菲小组建议。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-06-14 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241256733
Bernhard J Steinhoff, Elinor Ben-Menachem, Pavel Klein, Jukka Peltola, Bettina Schmitz, Rhys H Thomas, Vicente Villanueva
{"title":"Therapeutic strategies during cenobamate treatment initiation: Delphi panel recommendations.","authors":"Bernhard J Steinhoff, Elinor Ben-Menachem, Pavel Klein, Jukka Peltola, Bettina Schmitz, Rhys H Thomas, Vicente Villanueva","doi":"10.1177/17562864241256733","DOIUrl":"10.1177/17562864241256733","url":null,"abstract":"<p><p>The goal of epilepsy treatment is seizure freedom, typically with antiseizure medication (ASM). If patients fail to attain seizure control despite two trials of appropriately chosen ASMs at adequate doses, they are classified as having drug-resistant epilepsy (DRE). Adverse events (AEs) commonly occur in people with DRE because they are typically on ⩾2 ASMs, increasing the potential for drug-drug interactions. Early emerging AEs may impact adherence, decrease quality of life, and delay achieving optimal treatment dosages. Cenobamate is an oral ASM with a long half-life which has proven to be highly effective in clinical trials. An international Delphi panel of expert epileptologists experienced in the clinical use of cenobamate and other ASMs was convened to develop consensus best practices for managing patients during and after cenobamate titration, with consideration for its known pharmacokinetic and pharmacodynamic interactions, to allow patients to reach the most appropriate cenobamate dose while limiting tolerability issues. The modified Delphi process included one open-ended questionnaire and one virtual face-to-face meeting. Participants agreed that cenobamate can be prescribed for most patients experiencing focal-onset seizures. Patients initiating cenobamate therapy should have access to healthcare professionals as needed and their treatment response should be evaluated at the 100-mg dose. Patients with intellectual disabilities may need additional support to navigate the titration period. Proactive down-titration or withdrawal of sodium channel blockers (SCBs) is recommended when concomitant ASM regimens include ⩾2 SCBs. When applicable, maintaining a concomitant clobazam dose at ~5-10 mg may be beneficial. Patients taking oral contraceptives, newer oral anticoagulants, or HIV antiretroviral medications should be monitored for potential interactions. Because clinical evidence informing treatment decisions is limited, guidance regarding dose adjustments of non-ASM drugs was not developed beyond specific recommendations presented in the Summary of Product Characteristics.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241256733"},"PeriodicalIF":5.9,"publicationDate":"2024-06-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11179501/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141331739","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Comparative effectiveness and safety of ozanimod versus other oral DMTs in relapsing-remitting multiple sclerosis: a synthesis of matching-adjusted indirect comparisons. 奥扎尼莫与其他口服 DMTs 在复发缓解型多发性硬化症中的有效性和安全性比较:匹配调整间接比较综述。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-06-07 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241237856
Damemarie Paul, Elyse Swallow, Oscar Patterson-Lomba, Tychell Branchcomb, Laetitia N'Dri, Andres Gomez-Lievano, Jingyi Liu, Akanksha Dua, Marisa McGinley
{"title":"Comparative effectiveness and safety of ozanimod <i>versus</i> other oral DMTs in relapsing-remitting multiple sclerosis: a synthesis of matching-adjusted indirect comparisons.","authors":"Damemarie Paul, Elyse Swallow, Oscar Patterson-Lomba, Tychell Branchcomb, Laetitia N'Dri, Andres Gomez-Lievano, Jingyi Liu, Akanksha Dua, Marisa McGinley","doi":"10.1177/17562864241237856","DOIUrl":"10.1177/17562864241237856","url":null,"abstract":"<p><strong>Background: </strong>Several oral disease-modifying therapies (DMTs) have been approved by the Food and Drug Administration for the treatment of relapsing-remitting multiple sclerosis (RRMS). In the absence of head-to-head randomized data, matching-adjusted indirect comparisons (MAICs) can evaluate the comparative effectiveness and safety of ozanimod <i>versus</i> other oral DMTs in RRMS.</p><p><strong>Objectives: </strong>To synthesize results from the published MAICs of ozanimod and other oral DMTs for 2-year outcomes in RRMS.</p><p><strong>Methods: </strong>Published MAICs involving ozanimod for the treatment of RRMS were identified. Extracted data elements included efficacy [annualized relapse rate (ARR), confirmed disability progression (CDP), and brain volume loss] and safety [adverse events (AEs), serious AEs (SAEs), AEs leading to discontinuation, and infection] outcomes.</p><p><strong>Results: </strong>The four MAIC studies identified compared ozanimod with fingolimod, teriflunomide, dimethyl fumarate (DMF), and ponesimod. All comparisons were adjusted for differences in age, sex, relapses within the previous year, Expanded Disability Status Scale score, and percentage of patients with prior DMTs. Outcomes at 2 years were analyzed based on comparisons that lacked a common comparator arm. Ozanimod was associated with significantly lower ARR <i>versus</i> teriflunomide [ARR ratio (95% CI) 0.73 (0.62, 0.84) and DMF 0.80 (0.67, 0.97)], with no significant difference <i>versus</i> fingolimod or ponesimod. The proportions of patients treated with ozanimod or fingolimod had similar 3- and 6-month CDP. Compared with teriflunomide and DMF, ozanimod was associated with a significantly lower risk of 3-month CDP; 6-month CDP was comparable. Ozanimod was associated with significantly lower rates of any AE and AEs leading to discontinuation compared with the other oral DMTs evaluated. Ozanimod also had significantly lower rates of SAEs <i>versus</i> teriflunomide and DMF and lower rates of reported infection outcomes <i>versus</i> fingolimod and ponesimod.</p><p><strong>Conclusion: </strong>Compared with the other oral DMTs evaluated in MAICs, ozanimod was associated with a favorable safety profile and improved or comparable efficacy outcomes.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241237856"},"PeriodicalIF":5.9,"publicationDate":"2024-06-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11162124/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141296738","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Comment on: Cytokines (IL1β, IL6, TNFα) and serum cortisol levels may not constitute reliable biomarkers to identify individuals with post-acute sequelae of COVID-19. 评论:细胞因子(IL1β、IL6、TNFα)和血清皮质醇水平可能不是识别 COVID-19 急性后遗症患者的可靠生物标志物。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-05-31 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241254268
Matthias Kohl
{"title":"Comment on: Cytokines (IL1β, IL6, TNFα) and serum cortisol levels may not constitute reliable biomarkers to identify individuals with post-acute sequelae of COVID-19.","authors":"Matthias Kohl","doi":"10.1177/17562864241254268","DOIUrl":"10.1177/17562864241254268","url":null,"abstract":"","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241254268"},"PeriodicalIF":5.9,"publicationDate":"2024-05-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11143864/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141200613","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Author response to Comment on: Cytokines (IL1β, IL6, TNFα) and serum cortisol levels may not constitute reliable biomarkers to identify individuals with post-acute sequelae of COVID-19. 作者对以下评论的回复:细胞因子(IL1β、IL6、TNFα)和血清皮质醇水平可能并不是识别 COVID-19 急性后遗症患者的可靠生物标志物。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-05-31 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241255819
Michael Fleischer, Fabian Szepanowski, Anne K Mausberg, Livia Asan, Ellen Uslar, Denise Zwanziger, Mark Stettner, Lothar Volbracht, Christoph Kleinschnitz
{"title":"Author response to Comment on: Cytokines (IL1β, IL6, TNFα) and serum cortisol levels may not constitute reliable biomarkers to identify individuals with post-acute sequelae of COVID-19.","authors":"Michael Fleischer, Fabian Szepanowski, Anne K Mausberg, Livia Asan, Ellen Uslar, Denise Zwanziger, Mark Stettner, Lothar Volbracht, Christoph Kleinschnitz","doi":"10.1177/17562864241255819","DOIUrl":"10.1177/17562864241255819","url":null,"abstract":"","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241255819"},"PeriodicalIF":5.9,"publicationDate":"2024-05-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11143844/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141200610","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Differential blood-based biomarkers of subcortical and deep brain small vessel disease. 皮层下和大脑深部小血管疾病的不同血液生物标志物。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-05-31 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241243274
Pablo Hervella, Maria Luz Alonso-Alonso, Ana Sampedro-Viana, Manuel Rodríguez-Yáñez, Iria López-Dequidt, José M Pumar, Alberto Ouro, Daniel Romaus-Sanjurjo, Francisco Campos, Tomás Sobrino, José Castillo, Yago Leira, Ramón Iglesias-Rey
{"title":"Differential blood-based biomarkers of subcortical and deep brain small vessel disease.","authors":"Pablo Hervella, Maria Luz Alonso-Alonso, Ana Sampedro-Viana, Manuel Rodríguez-Yáñez, Iria López-Dequidt, José M Pumar, Alberto Ouro, Daniel Romaus-Sanjurjo, Francisco Campos, Tomás Sobrino, José Castillo, Yago Leira, Ramón Iglesias-Rey","doi":"10.1177/17562864241243274","DOIUrl":"10.1177/17562864241243274","url":null,"abstract":"<p><strong>Background: </strong>Cerebral small vessel disease is the most common cause of lacunar strokes (LS). Understanding LS pathogenesis is vital for predicting disease severity, prognosis, and developing therapies.</p><p><strong>Objectives: </strong>To research molecular profiles that differentiate LS in deep brain structures from those in subcortical white matter.</p><p><strong>Design: </strong>Prospective case-control study involving 120 patients with imaging-confirmed LS and a 120 control group.</p><p><strong>Methods: </strong>We examined the relationship between Alzheimer's disease biomarkers [amyloid beta (Aβ<sub>1-40</sub>, Aβ<sub>1-42</sub>)], serum inflammatory marker (interleukin-6, IL-6), and endothelial dysfunction markers [soluble tumor necrosis factor-like weak inducer of apoptosis, and pentraxin-3 (sTWEAK, PTX3)] with respect to LS occurring in deep brain structures and subcortical white matter. In addition, we investigated links between LS, leukoaraiosis presence (white matter hyperintensities, WMHs), and functional outcomes at 3 months. Poor outcome was defined as a modified Rankin scale >2 at 3 months.</p><p><strong>Results: </strong>Significant differences were observed in levels of IL-6, PTX3, and sTWEAK between patients with deep lacunar infarcts and those with recent small subcortical infarcts (20.8 <i>versus</i> 15.6 pg/mL, <i>p</i> < 0.001; 7221.3 <i>versus</i> 4624.4 pg/mL, <i>p</i> < 0.0001; 2528.5 <i>versus</i> 1660.5 pg/mL, <i>p</i> = 0.001). Patients with poor outcomes at 3 months displayed notably higher concentrations of these biomarkers compared to those with good outcomes. By contrast, Aβ<sub>1-40</sub> and Aβ<sub>1-42</sub> were significantly lower in patients with deep LS (<i>p</i> < 0.0001). Aβ<sub>1-42</sub> levels were significantly higher in patients with LS in subcortical white matter who had poor outcomes. WMH severity only showed a significant association with deep LS and correlated with sTWEAK (<i>p</i> < 0.0001).</p><p><strong>Conclusion: </strong>The pathophysiological mechanisms of lacunar infarcts in deep brain structures seem different from those in the subcortical white matter. As a result, specific therapeutic and preventive strategies should be explored.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241243274"},"PeriodicalIF":5.9,"publicationDate":"2024-05-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11143814/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141200615","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Rescue treatment with add-on efgartigimod in a patient with impending myasthenic crisis: a case report. 用依加替莫德对一名即将发生肌无力危象的患者进行抢救治疗:病例报告。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-05-28 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241254895
Zhouao Zhang, Mingjin Yang, Tiancheng Luo, Xue Du, Zhouyi Wang, Xiaoyu Huang, Yong Zhang
{"title":"Rescue treatment with add-on efgartigimod in a patient with impending myasthenic crisis: a case report.","authors":"Zhouao Zhang, Mingjin Yang, Tiancheng Luo, Xue Du, Zhouyi Wang, Xiaoyu Huang, Yong Zhang","doi":"10.1177/17562864241254895","DOIUrl":"10.1177/17562864241254895","url":null,"abstract":"<p><p>Myasthenia gravis (MG) is an autoimmune disorder characterized by fluctuating muscle weakness. Severe patients may develop life-threatening respiratory failure and experience crisis. Plasma exchange or intravenous immunoglobulin (IVIg) is the first-line treatment option for myasthenia crisis, but some patients still poorly respond to them. Here, we first reported a generalized MG patient from China who was in a state of impending myasthenic crisis and did not respond effectively to IVIg but was successfully rescued by add-on efgartigimod. Especially, we also detected meaningful changes in T-cell and B-cell subsets after efgartigimod, promoting a potential role of efgartigimod in re-establishing immune homeostasis.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241254895"},"PeriodicalIF":5.9,"publicationDate":"2024-05-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11135074/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141176401","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Combined treatment with allogeneic Epstein-Barr- and human polyomavirus 1 specific T-cells in progressive multifocal leukoencephalopathy and EBV infection: a case report. 进行性多灶性白质脑病和 EB 病毒感染的异体巴氏和人类多瘤病毒 1 特异性 T 细胞联合治疗:病例报告。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-05-28 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241253917
Sandra Nay, Nora Möhn, Lea Grote-Levi, Agnes Bonifacius, Mieke L Saßmann, Kevin Karacondi, Sabine Tischer-Zimmermann, Henning Pöter, Nima Mahmoudi, Mike P Wattjes, Britta Maecker-Kolhoff, Günter Höglinger, Britta Eiz-Vesper, Thomas Skripuletz
{"title":"Combined treatment with allogeneic Epstein-Barr- and human polyomavirus 1 specific T-cells in progressive multifocal leukoencephalopathy and EBV infection: a case report.","authors":"Sandra Nay, Nora Möhn, Lea Grote-Levi, Agnes Bonifacius, Mieke L Saßmann, Kevin Karacondi, Sabine Tischer-Zimmermann, Henning Pöter, Nima Mahmoudi, Mike P Wattjes, Britta Maecker-Kolhoff, Günter Höglinger, Britta Eiz-Vesper, Thomas Skripuletz","doi":"10.1177/17562864241253917","DOIUrl":"10.1177/17562864241253917","url":null,"abstract":"<p><p>Opportunistic viral infections in individuals with severe immunodeficiency can lead to fatal conditions such as progressive multifocal leukoencephalopathy (PML), for which treatment options are limited. These infections pose significant risks, especially when co-infections with other viruses occur. We describe a combined therapy approach using directly isolated allogeneic Human Polyomavirus 1 (also known as BKV) and Epstein-Barr virus (EBV) specific cytotoxic T-cells for the treatment of PML in conjunction with identified EBV in the cerebrospinal fluid (CSF) of a male patient infected with human immunodeficiency virus (HIV). A 53-year-old HIV-positive male, recently diagnosed with PML, presented with rapidly worsening symptoms, including ataxia, tetraparesis, dysarthria, and dysphagia, leading to respiratory failure. The patient developed PML even after commencing highly active antiretroviral therapy (HAART) 3 months prior. Brain magnetic resonance imaging (MRI) revealed multifocal demyelination lesions involving the posterior fossa and right thalamus suggestive of PML. In addition to the detection of human polyomavirus 2 (also known as JCV), analysis of CSF showed positive results for EBV deoxyribonucleic acid (DNA). His neurological condition markedly deteriorated over the following 2 months. Based on MRI, there was no evidence of Immune Reconstitution Inflammatory Syndrome contributing to this decline. The patient did not have endogenous virus-specific T-cells. We initiated an allogeneic, partially human leukocyte antigen-matched transfer of EBV and utilizing the cross-reactivity between BKV and JCV-BKV specific T-cells. This intervention led to notable neurological improvement and partial resolution of the MRI lesions within 6 weeks. Our case of a patient with acquired immune deficiency syndrome demonstrates that PML and concurrent EBV co-infection can still occur despite undergoing HAART treatment. This innovative experimental therapy, involving a combination of virus-specific T-cells, was demonstrated to be an effective treatment option in this patient.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241253917"},"PeriodicalIF":5.9,"publicationDate":"2024-05-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11135084/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141176399","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
TAURUS-MS II: real-world use of teriflunomide in Germany and changes in treatment patterns over time. TAURUS-MS II:德国特立氟胺的实际使用情况和治疗模式的长期变化。
IF 5.9 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2024-05-27 eCollection Date: 2024-01-01 DOI: 10.1177/17562864241252722
Boris-Alexander Kallmann, Georg Zu Eulenburg, Jennifer S Kullmann, Mathias Mäurer
{"title":"TAURUS-MS II: real-world use of teriflunomide in Germany and changes in treatment patterns over time.","authors":"Boris-Alexander Kallmann, Georg Zu Eulenburg, Jennifer S Kullmann, Mathias Mäurer","doi":"10.1177/17562864241252722","DOIUrl":"10.1177/17562864241252722","url":null,"abstract":"<p><strong>Background: </strong>Teriflunomide is a once-daily oral disease-modifying therapy (DMT) for the treatment of relapsing-remitting multiple sclerosis (RRMS). Only limited information is available about its real-world use and changes over time.</p><p><strong>Objectives: </strong>To collect real-world data on teriflunomide use in clinical routine (and comparison to the previously conducted study TAURUS-MS).</p><p><strong>Design: </strong>National, open, non-interventional, prospective, multicenter study.</p><p><strong>Methods: </strong>TAURUS-MS II was conducted at 220 German sites between July 2017 and March 2022, including RRMS patients treated with teriflunomide. Data on patient demographics, MS history, previous treatment, therapy satisfaction, and safety were collected.</p><p><strong>Results: </strong>In total, 752 patients were included (65% female) with a mean age (±standard deviation) of 43 ± 11 years. Sixty-six percent had DMT before, and 46% had discontinued their last pretreatment ≤6 months prior to study entry. Among the latter, previous DMTs were interferon (21%), glatiramer acetate (11%), and dimethyl fumarate (9%), and reasons for discontinuation were adverse events (AEs; 55%) and insufficient efficacy (16%). Over 24 months, the mean treatment Satisfaction Questionnaire for Medication scores improved by 6 ± 29 points on effectiveness, 8 ± 20 on convenience, and 12 ± 25 on global satisfaction. The mean number of MS relapses decreased from 0.81 ± 0.81 in the 24 months prior to 0.27 ± 0.57 within 24 months after study entry. Non-serious AEs occurred in 423 patients (56%) and serious AEs in 49 patients (7%). Most reported AEs were alanine aminotransferase increase (11%), hypertension (8%), and alopecia (7%). Compared to TAURUS-MS, patients in TAURUS-MS II were younger, had a higher employment rate, and a higher share of treatment-naïve patients.</p><p><strong>Conclusion: </strong>Mean number of relapses was significantly reduced. Patient satisfaction was significantly improved compared to previous DMT. Tolerability was comparable to previous trials.</p><p><strong>Trial registration: </strong>Bundesinstitut für Arzneimittel und Medizinprodukte public database for non-interventional studies, number 7138.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"17 ","pages":"17562864241252722"},"PeriodicalIF":5.9,"publicationDate":"2024-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11131407/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141161804","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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