Therapeutic Advances in Neurological Disorders最新文献

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Safety and efficacy of oral anticoagulation in patients with intracranial hemorrhage and atrial fibrillation: a systematic review and meta-analysis of randomized controlled clinical trials. 口服抗凝治疗颅内出血和房颤患者的安全性和有效性:随机对照临床试验的系统回顾和荟萃分析
IF 4.1 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-12-21 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251406065
Nikolaos M Papageorgiou, Lina Palaiodimou, Aikaterini Theodorou, Eleni Bakola, Maria Chondrogianni, Georgia Papagiannopoulou, Apostolos Safouris, Eleni Anagnou, Panagiota-Eleni Tsalouchidou, Effrosyni Koutsouraki, Theodore Karapanayiotides, Efstathios Boviatsis, Christos Krogias, Sotirios Giannopoulos, Diana Aguiar de Sousa, Mira Katan, Thorsten Steiner, Georgios Tsivgoulis
{"title":"Safety and efficacy of oral anticoagulation in patients with intracranial hemorrhage and atrial fibrillation: a systematic review and meta-analysis of randomized controlled clinical trials.","authors":"Nikolaos M Papageorgiou, Lina Palaiodimou, Aikaterini Theodorou, Eleni Bakola, Maria Chondrogianni, Georgia Papagiannopoulou, Apostolos Safouris, Eleni Anagnou, Panagiota-Eleni Tsalouchidou, Effrosyni Koutsouraki, Theodore Karapanayiotides, Efstathios Boviatsis, Christos Krogias, Sotirios Giannopoulos, Diana Aguiar de Sousa, Mira Katan, Thorsten Steiner, Georgios Tsivgoulis","doi":"10.1177/17562864251406065","DOIUrl":"10.1177/17562864251406065","url":null,"abstract":"<p><strong>Background: </strong>Patients with atrial fibrillation (AF) who survive spontaneous intracerebral hemorrhage (ICH) face competing risks of thromboembolism and recurrent bleeding.</p><p><strong>Objectives: </strong>To evaluate the safety and efficacy of initiating oral anticoagulants versus avoiding anticoagulation in adults with AF after spontaneous ICH.</p><p><strong>Design: </strong>Systematic review and meta-analysis of randomized-controlled clinical trials (RCTs).</p><p><strong>Data sources and methods: </strong>We searched MEDLINE, Scopus, and ClinicalTrials.gov up to August 28, 2025, for eligible RCTs randomizing adults with AF and prior spontaneous ICH to start oral anticoagulation versus avoid anticoagulation. Efficacy outcomes included the occurrence of new ischemic stroke (primary) and ischemic major adverse cardiovascular events (MACE; secondary). Safety outcomes included recurrent ICH (primary), hemorrhagic-MACE, all-cause mortality at follow-up, and cardiovascular death (secondary). Risk ratios (RRs) with 95% confidence intervals (CIs) were pooled using random-effects meta-analysis.</p><p><strong>Results: </strong>Six RCTs were included, comprising 403 patients in the anticoagulation group and 395 in the avoid-anticoagulation group. Anticoagulants reduced the rates of new ischemic stroke (RR = 0.20; 95% CI: 0.06-0.72; <i>I</i> <sup>2</sup> = 60%; number needed to treat = 9) and ischemic-MACE (RR = 0.41; 95% CI: 0.23-0.75; <i>I</i> <sup>2</sup> = 32%). Anticoagulants were associated with higher rates of recurrent ICH (RR = 3.14; 95% CI: 1.41-7.01; <i>I</i> <sup>2</sup> = 0%; number needed to harm = 19) and hemorrhagic-MACE (RR = 2.35; 95% CI: 1.32-4.21; <i>I</i> <sup>2</sup> = 1%). All-cause mortality at 90 days (RR = 1.06; 95% CI: 0.69-1.64; <i>I</i> <sup>2</sup> = 28%) and cardiovascular death (RR = 0.98; 95% CI: 0.34-2.87; <i>I</i> <sup>2</sup> = 63%) did not differ between the two groups. Leave-one-out sensitivity analyses supported the overall direction of effects, with some attenuation when individual trials were omitted.</p><p><strong>Conclusion: </strong>In AF survivors of spontaneous ICH, restarting oral anticoagulation lowers ischemic events but raises risks of recurrent ICH and major bleeding, without a clear early mortality difference. Potential benefits may outweigh risks in selected patients within a multidisciplinary framework. Adequately powered RCTs are needed to refine agent choice, timing, and patient selection.</p><p><strong>Trial registration: </strong>PROSPERO CRD420251135299 (registered August 27, 2025).</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251406065"},"PeriodicalIF":4.1,"publicationDate":"2025-12-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12722659/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145828428","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
Long-term globus pallidus internus deep brain stimulation in a young patient with spinocerebellar ataxia type 3 initially presenting with levodopa-responsive parkinsonism: a 6-year follow-up case report and literature review. 1例以左旋多巴反应性帕金森病为首发的3型脊髓小脑共济失调年轻患者的长期内白球深部脑刺激:6年随访病例报告及文献综述
IF 4.1 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-12-16 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251396525
Jing Zhao, Shaochen Ma, Yunlei Gao, Jia Chen, Jiaqi Chen, Chong Shi, Peifu Wang, Jilai Li, Jichen Du, Zhirong Wan
{"title":"Long-term globus pallidus internus deep brain stimulation in a young patient with spinocerebellar ataxia type 3 initially presenting with levodopa-responsive parkinsonism: a 6-year follow-up case report and literature review.","authors":"Jing Zhao, Shaochen Ma, Yunlei Gao, Jia Chen, Jiaqi Chen, Chong Shi, Peifu Wang, Jilai Li, Jichen Du, Zhirong Wan","doi":"10.1177/17562864251396525","DOIUrl":"10.1177/17562864251396525","url":null,"abstract":"<p><p>Spinocerebellar ataxia type 3 (SCA3) is an inherited neurodegenerative disorder. Some of its clinical features resemble those of primary Parkinson's disease (PD), which can easily lead to misdiagnosis. There is currently no disease-modifying therapy available for SCA3, treatment is mainly symptomatic. Herein, we report a case of a young female patient with SCA3 who presented with Parkinsonian as the main manifestation and underwent globus pallidus internus (GPi) deep brain stimulation (DBS). This is a 36-year-old female patient. Her first symptoms occurred at the age of 28 in 2009, manifesting as gait abnormalities in the right lower limb. She was misdiagnosed with early-onset PD in 2011. Genetic testing showed abnormal numbers of CAG repeats (15/70) within the coding region of the ATXN3 genes. She was diagnosed with SCA3. The patient initially responded well to levodopa-based medication, but the treatment effects gradually attenuated over time, with the development of severe symptom fluctuations and dyskinesia in 2018. The patient underwent GPi-DBS surgery in the absence of cerebellar signs, cognitive, and mood disorders. Six-year postoperative follow-up results suggest that long-term GPi-DBS is effective for the control of dyskinesia, but the residual motor symptoms (parkinsonism and ataxia) had progressively worsened in the patient. Various targets have been reported to be selected for DBS treatment of SCA3, with substantial individual differences in treatment outcomes. This case emphasizes the importance of genetic testing for the diagnosis of SCA3 and provides a basis for personalized treatment of patients with SCA3.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251396525"},"PeriodicalIF":4.1,"publicationDate":"2025-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12712318/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145805493","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
Appropriate use of steroids for patients with generalized Myasthenia Gravis: an international Delphi study. 全身性重症肌无力患者适当使用类固醇:一项国际德尔菲研究。
IF 4.1 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-12-16 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251398375
Heinz Wiendl, Hiroyuki Murai, Pushpa Narayanaswami, Francesco Saccà, Nicholas J Silvestri, James F Howard
{"title":"Appropriate use of steroids for patients with generalized Myasthenia Gravis: an international Delphi study.","authors":"Heinz Wiendl, Hiroyuki Murai, Pushpa Narayanaswami, Francesco Saccà, Nicholas J Silvestri, James F Howard","doi":"10.1177/17562864251398375","DOIUrl":"10.1177/17562864251398375","url":null,"abstract":"<p><strong>Background: </strong>Oral corticosteroids (OCS) are commonly used in the management of autoimmune Myasthenia Gravis (MG), often in high doses for prolonged periods. Exposure to OCS is associated with significant and cumulative adverse effects. There is currently no universal consensus on the approach to OCS use in MG.</p><p><strong>Objectives: </strong>To reach a multinational consensus on the appropriate use of OCS in MG, including the role of treatment approaches to minimize dose and support tapering.</p><p><strong>Design: </strong>This modified Delphi study established consensus among a panel of 70 general neurologists/neuromuscular specialists from eight countries over two rounds of survey to establish best practice principles regarding the use of OCS in MG.</p><p><strong>Methods: </strong>The current literature on OCS use in MG was reviewed. Topics of interest were identified, and a modified Delphi consensus process was created. A steering committee of six experts in MG proposed statements for testing with the wider panel over two rounds of surveys. Consensus was reached if at least 75% agreed or strongly agreed on a 4-point Likert scale.</p><p><strong>Results: </strong>Consensus was achieved for all 37 final statements. These statements covered principles of OCS use, including a target for long-term use of ⩽5 mg/day; the role of biologic treatments in minimizing required OCS dose; the need for individualization of approach according to patient factors; and the need for detailed guidance regarding how and when to taper OCS dose.</p><p><strong>Conclusion: </strong>These findings reinforce a shift in the management of MG, advocating for the judicious and sparing use of OCS against the backdrop of expanding therapeutic options. As additional evidence-based data emerge, these recommendations should be updated.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251398375"},"PeriodicalIF":4.1,"publicationDate":"2025-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12709019/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145782860","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
Alterations of fecal short-chain fatty acids solely in the course of multiple sclerosis: rethinking the gut-brain axis in the early stages of MS. 多发性硬化症病程中粪便短链脂肪酸的改变:对多发性硬化症早期肠脑轴的重新思考
IF 4.1 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-12-12 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251396028
Jakob Stögbauer, Niklas Kämpfer, Anouck Becker-Dorison, Andreas Schwiertz, Sergiu Groppa, Marcus M Unger, Mathias Fousse
{"title":"Alterations of fecal short-chain fatty acids solely in the course of multiple sclerosis: rethinking the gut-brain axis in the early stages of MS.","authors":"Jakob Stögbauer, Niklas Kämpfer, Anouck Becker-Dorison, Andreas Schwiertz, Sergiu Groppa, Marcus M Unger, Mathias Fousse","doi":"10.1177/17562864251396028","DOIUrl":"10.1177/17562864251396028","url":null,"abstract":"<p><strong>Background: </strong>The role of gut microbiota in multiple sclerosis (MS) has become increasingly important, intestinal dysbiosis with reduced production of short-chain fatty acids (SCFA) being the prevailing paradigm. However, the direction of causality, that is, whether intestinal changes are cause or consequence of chronic central nervous system inflammation, remains to be elucidated. Previous studies have focused on long-term MS patients. Alteration in fecal SCFA concentrations in early MS, particularly during relapses, remains to be extensively studied.</p><p><strong>Objectives: </strong>To compare fecal SCFA concentrations in patients with a first diagnosis of MS with those in patients with long-term MS and in healthy controls (HCs).</p><p><strong>Design: </strong>Prospective cohort study.</p><p><strong>Methods: </strong>The prospective case-control study was conducted on relapsing-remitting MS (RRMS) patients at the time of first, acute relapse without ongoing immunotherapy (Early-RRMS). Clinical and demographic parameters, as well as fecal SCFA concentrations (measured by gas chromatography) were collected. The parameters were compared with those of matched RRMS patients under different, long-term immunotherapy (Late-RRMS) and HCs.</p><p><strong>Results: </strong>SCFA concentrations of propionate, butyrate, isobutyrate, valerate, and isovalerate were not significantly different between the early-RRMS cohort and HCs, but were lower in the late-RRMS cohort.</p><p><strong>Conclusion: </strong>The findings indicate that reduction in SCFA levels is exclusively observed in patients with RRMS during the further course of the disease and not at the onset. Decrease in SCFA concentration may be rather consequence or related to neurodegeneration than linked to the first demyelinating event. Further investigation related to disease trajectories of immunomodulatory or neuroprotective treatments are required.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251396028"},"PeriodicalIF":4.1,"publicationDate":"2025-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12701217/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145757615","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
Cellular and humoral vaccination response under immunotherapies-German consensus on vaccination strategies in neurological autoimmune diseases. 免疫疗法下的细胞和体液免疫应答——神经自身免疫性疾病疫苗接种策略的德国共识。
IF 4.1 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-12-12 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251396006
Muriel Schraad, Mathias Mäurer, Anke Salmen, Tobias Ruck, Timo Uphaus, Vinzenz Fleischer, Felix Luessi, Maria Protopapa, Falk Steffen, Nicholas Hanuscheck, Katrin Pape, Tobias Brummer, Josef Shin, Thomas Korn, Luisa Klotz, Jan D Lünemann, Marc Pawlitzki, Martin S Weber, Antonios Bayas, Brigitte Wildemann, Hans-Peter Hartung, Florian Then Bergh, Clemens Warnke, Uwe K Zettl, Achim Berthele, Aiden Haghikia, Ralf Linker, Hayrettin Tumani, Sven G Meuth, Bernhard Hemmer, Heinz Wiendl, Tania Kümpfel, Ralf Gold, Stefan Bittner, Frauke Zipp
{"title":"Cellular and humoral vaccination response under immunotherapies-German consensus on vaccination strategies in neurological autoimmune diseases.","authors":"Muriel Schraad, Mathias Mäurer, Anke Salmen, Tobias Ruck, Timo Uphaus, Vinzenz Fleischer, Felix Luessi, Maria Protopapa, Falk Steffen, Nicholas Hanuscheck, Katrin Pape, Tobias Brummer, Josef Shin, Thomas Korn, Luisa Klotz, Jan D Lünemann, Marc Pawlitzki, Martin S Weber, Antonios Bayas, Brigitte Wildemann, Hans-Peter Hartung, Florian Then Bergh, Clemens Warnke, Uwe K Zettl, Achim Berthele, Aiden Haghikia, Ralf Linker, Hayrettin Tumani, Sven G Meuth, Bernhard Hemmer, Heinz Wiendl, Tania Kümpfel, Ralf Gold, Stefan Bittner, Frauke Zipp","doi":"10.1177/17562864251396006","DOIUrl":"10.1177/17562864251396006","url":null,"abstract":"<p><strong>Background: </strong>With the development of highly effective disease-modifying treatments, vaccinations are becoming increasingly important in people with neurological autoimmune diseases. However, questions regarding the safety and efficacy of vaccinations under immunotherapy remain.</p><p><strong>Objective: </strong>To provide recommendations on types and timing of vaccinations for people with neuroimmunological diseases under different immunotherapies.</p><p><strong>Design: </strong>Our study presents a German evidence-based expert consensus on vaccination under immunotherapies in neurological autoimmune diseases.</p><p><strong>Methods: </strong>Based on literature research, a consortium of experts evaluated the quality of evidence, integrated clinical experience, and responded to a questionnaire determining an agreement (>75%) on statements concerning vaccination upon immune therapies in neuroimmunological diseases.</p><p><strong>Results: </strong>The specific humoral and cellular response to vaccination can be compromised under alemtuzumab, azathioprine, cladribine, cyclophosphamide, CD19/CD20 antibodies (inebilizumab, ocrelizumab, ofatumumab, rituximab, ublituximab), dimethyl fumarate/diroximel fumarate, FcRn inhibitors (efgartigimod, rozanolixizumab), complement C5 inhibitors (eculizumab, ravulizumab, zilucoplan), interleukin-6 receptor antibodies (tocilizumab, satralizumab), intravenous immunoglobulins, long-term steroid administration, methotrexate, mitoxantrone, mycophenolate mofetil, tacrolimus, teriflunomide, tumor necrosis factor-α blockers, and sphingosine-1-phosphate receptor modulators (fingolimod, ozanimod, ponesimod, siponimod), as well as after autologous stem cell transplantation. The lymphocyte count can have an influence here. Overall, it is generally advisable to complete vaccination before starting immunotherapy. However, in the case of an active inflammatory disease course with possible irreversible neurological deficits, a delay in therapy initiation until immunization has been completed cannot be justified. The application of live vaccines is contraindicated for most therapies and is only recommended after a strict risk-benefit assessment.</p><p><strong>Conclusion: </strong>Vaccinations are necessary for individuals on immunotherapy to reduce the risk of infections and the associated risk of worsening neurological autoimmune diseases. However, the humoral and cellular vaccination response may be impaired under immunotherapy necessitating close monitoring. Here, we provide applicable recommendations to optimize immunization for individuals receiving immunotherapy due to a neurological autoimmune disease.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251396006"},"PeriodicalIF":4.1,"publicationDate":"2025-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12701252/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145757648","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
CAR T-cell therapy for neurological disorders: scientific rationale and mechanistic insights. CAR - t细胞治疗神经系统疾病:科学原理和机制见解。
IF 4.1 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-12-10 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251396039
Dimitrios E Katsaros, Dimitrios Mougiakakos
{"title":"CAR T-cell therapy for neurological disorders: scientific rationale and mechanistic insights.","authors":"Dimitrios E Katsaros, Dimitrios Mougiakakos","doi":"10.1177/17562864251396039","DOIUrl":"10.1177/17562864251396039","url":null,"abstract":"<p><p>The development and successful clinical application of engineered T cells expressing synthetic chimeric antigen receptors (CARs) represents a milestone in cancer therapy. This approach optimizes physiological in vivo T-cell activation against specific target antigens expressed on defined cell subsets with the goal of their deep and sustained depletion. Significant progress has been made in redesigning CAR T-cell constructs to improve patient safety, therapeutic efficacy, and accessibility. Efforts have also focused on streamlining manufacturing to improve availability and reduce costs, two critical challenges to widespread adoption. Beyond hematologic malignancies, CAR T-cell therapies are now increasingly being repurposed to tackle B-cell-mediated autoimmune diseases (AIDs). This is primarily achieved through broad B-cell depletion, but more targeted approaches-such as the selective elimination of autoantibody-producing B-cell subpopulations-are also being explored. Important considerations in their implementation are identifying the most pertinent patient groups, tailoring their treatment up to the point of CAR-infusion, and following up on their unique toxicity-profile. In the context of neurological AIDs-including refractory myasthenia gravis, Lambert-Eaton syndrome, multiple sclerosis, and stiff-person syndrome-early clinical experience suggests promising efficacy and tolerability, leading to a growing number of registered clinical trials. In this review, we provide an overview of the mechanism and evolution of CAR T-cell therapy, highlighting why its application in AIDs, particularly in neurology, represents a highly promising therapeutic strategy.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251396039"},"PeriodicalIF":4.1,"publicationDate":"2025-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12696306/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145757580","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
Efficacy and safety of patisiran for the treatment of acquired amyloid polyneuropathy in domino liver transplant recipients. 帕西兰治疗多米诺骨牌肝移植受者获得性淀粉样多神经病变的疗效和安全性。
IF 4.1 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-12-10 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251396030
Velina Nedkova-Hristova, Laura Donadeu, Carmen Baliellas, José González-Costello, Laura Lladó, Emma González-Vilatarsana, Miosés Morales de la Prida, Valentina Vélez-Santamaría, Oriol Bestard, Carlos Casasnovas
{"title":"Efficacy and safety of patisiran for the treatment of acquired amyloid polyneuropathy in domino liver transplant recipients.","authors":"Velina Nedkova-Hristova, Laura Donadeu, Carmen Baliellas, José González-Costello, Laura Lladó, Emma González-Vilatarsana, Miosés Morales de la Prida, Valentina Vélez-Santamaría, Oriol Bestard, Carlos Casasnovas","doi":"10.1177/17562864251396030","DOIUrl":"10.1177/17562864251396030","url":null,"abstract":"<p><strong>Background: </strong>There is no standardized treatment for acquired amyloid polyneuropathy (AAP) in domino liver transplant (DLT) recipients.</p><p><strong>Objectives: </strong>Our objective is to analyze the efficacy and safety of patisiran for the treatment of AAP in DLT recipients.</p><p><strong>Design: </strong>We performed a postauthorization prospective longitudinal study of DLT recipients with AAP who received patisiran treatment for 22 months.</p><p><strong>Methods: </strong>The primary endpoint was change in the Neuropathy Impairment Scale (NIS) from baseline. Other assessments included neurophysiologic study, quantitative sensory testing, 10 m walking test, and quality of life and disability questionnaires. As safety parameters we analyzed evidence of graft rejection, immunosuppression levels, and renal and cardiac adverse effects.</p><p><strong>Results: </strong>Four patients were recruited. The mean NIS at baseline was 8.5 ± 2.08. All patients presented clinical improvement after 22 months of treatment, with a mean NIS of 4.75 ± 2.27 points. The mean change from baseline in the NIS was -3.75 ± 0.71 (95% CI: -0.47 to 7.97). The use of patisiran was not associated with cardiovascular or renal side effects. No patient presented relevant changes in immunosuppression levels or graft rejection.</p><p><strong>Conclusion: </strong>Our study suggests that patisiran may improve neurological manifestations in DLT recipients with AAP, producing no relevant adverse effects.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251396030"},"PeriodicalIF":4.1,"publicationDate":"2025-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12696296/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145757599","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
Lacosamide versus topiramate in episodic migraine: a randomized controlled double-blinded trial. 拉科沙胺与托吡酯治疗偏头痛:一项随机对照双盲试验。
IF 4.1 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-12-09 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251396529
Mohamed G Zeinhom, Mohamed Fouad Elsayed Khalil, Mohamed Almoataz, Tarek Youssif Omar Youssif, Ahmed Mohamed Ali Daabis, Hossam Mohamed Refat, Ahmed Ahmed Mohamed Kamal Ebied, Shady S Georgy, Ahmed Zaki Omar Akl, Mohamed Ismaiel, Salah Ibrahim Ahmed, Hesham Farouk Eissa, Asmaa Ibrahem Desouky Mostafa Ibrahem, Asmaa Mohammed Hassan, Mohamed Elshafei, Amir Ahmed Elsaeed Egila, Sherihan Rezk Ahmed
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引用次数: 0
The diagnosis comes before therapy, even with non-motor features. 诊断是在治疗之前进行的,即使是非运动特征。
IF 4.1 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-12-06 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251400476
Wolfgang H Jost, Peter A LeWitt
{"title":"The diagnosis comes before therapy, even with non-motor features.","authors":"Wolfgang H Jost, Peter A LeWitt","doi":"10.1177/17562864251400476","DOIUrl":"10.1177/17562864251400476","url":null,"abstract":"","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251400476"},"PeriodicalIF":4.1,"publicationDate":"2025-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12681568/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145709843","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
Brain volumetry and spinal cord imaging in patients with AQP4-IgG+ NMOSD-a systematic review and meta-analysis. AQP4-IgG+ nmoss患者的脑容量和脊髓成像-系统回顾和荟萃分析
IF 4.1 2区 医学
Therapeutic Advances in Neurological Disorders Pub Date : 2025-12-06 eCollection Date: 2025-01-01 DOI: 10.1177/17562864251394843
Josephine Heine, Claudia Chien
{"title":"Brain volumetry and spinal cord imaging in patients with AQP4-IgG+ NMOSD-a systematic review and meta-analysis.","authors":"Josephine Heine, Claudia Chien","doi":"10.1177/17562864251394843","DOIUrl":"10.1177/17562864251394843","url":null,"abstract":"<p><strong>Background: </strong>Magnetic resonance imaging (MRI) is often used to evaluate disease-related brain changes in patients with aquaporin-4-IgG seropositive neuromyelitis optica spectrum disorder (AQP4-IgG+ NMOSD).</p><p><strong>Objectives: </strong>To use a meta-analysis for assessment of quantitative volumetric brain and spinal cord changes in patients with AQP4-IgG+ NMOSD and healthy participants.</p><p><strong>Design: </strong>We analyzed volume estimates of the brain, gray matter, white matter, thalamus, T2/FLAIR-brain lesions, as well as mean upper cervical cord area (MUCCA). Inclusion criteria included patients with AQP4-IgG+ NMOSD, MRI-based segmentation data, and matched healthy participants. Data from NMOSD patients with mixed/unknown serostatus or significant comorbidities were excluded.</p><p><strong>Data sources: </strong>We searched MEDLINE through Pubmed for peer-reviewed articles published between 05/2006 (revised NMOSD diagnostic criteria) and 01/2025.</p><p><strong>Methods: </strong>Standardized mean differences and pooled effect sizes (Hedges' <i>g</i>) were determined with random-effects models, adjusting for duplicate reporting, outliers, and small study effects. Metaregressions were used to determine clinical associations.</p><p><strong>Results: </strong>Evidence of pooled data showed that whole brain volume (<i>g</i> = -0.61, 95% confidence interval (CI): -0.91 to -0.32, <i>p</i> < 0.001, <i>N</i> <sub>pat/con</sub> = 385/325, <i>k</i> = 11) and gray matter volume (<i>g</i> = -0.40, 95% CI: -0.72 to -0.09, <i>p</i> = 0.018, <i>N</i> <sub>pat/con</sub> = 259/267, <i>k</i> = 9) were significantly different between patients and healthy participants. Heterogeneity was moderate (τ² = 0.08 and τ² = 0.09, respectively). Moreover, we found a large effect for reduced MUCCA (<i>g</i> = -0.99, 95% CI: -1.59 to -0.39, <i>p</i> = 0.007, <i>N</i> <sub>pat/con</sub> = 189/162, <i>k</i> = 7) with moderate heterogeneity (τ² = 0.31). No conclusive evidence emerged for changes in thalamic or white matter volume. Bias analysis did not indicate that smaller studies affected effect sizes. A systematic review of voxel-based morphometry revealed that reduced gray matter volume was most likely in the bilateral thalamus (⩽69%) and occipital (44%), frontal (27%), and temporal cortices (27%).</p><p><strong>Conclusion: </strong>AQP4-IgG+ NMOSD patients have specific global and local central nervous system volume reductions, potentially induced by astrocytic damage and demyelination. Volumetric outcomes may therefore inform MRI-guided disease monitoring and endpoints in clinical studies.</p><p><strong>Trial registration: </strong>PROSPERO (CRD42024493121). This study follows the Preferred Reporting Items for Systematic Reviews and Meta-Analyses reporting guidelines.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"18 ","pages":"17562864251394843"},"PeriodicalIF":4.1,"publicationDate":"2025-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12681629/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145709779","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
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