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Evaluation and treatment of refractory chronic inflammatory demyelinating polyradiculoneuropathy. 难治性慢性炎性脱髓鞘性多根神经病变的评价与治疗。
IF 2.4 Q3 CLINICAL NEUROLOGY Pub Date : 2025-11-13 eCollection Date: 2025-01-01 DOI: 10.1136/bmjno-2025-001318
Lynette Kiers, Belinda Cruse

Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is an inflammatory, immune-mediated neuropathy of the peripheral nerves and nerve roots. CIDP is unlikely to be a discrete disease entity, but rather a spectrum of related conditions, in which cell-mediated and humoral mechanisms act synergistically to cause damage to peripheral nerves. The 2021 guideline of the European Academy of Neurology/Peripheral Nerve Society on the diagnosis and treatment of CIDP has modified the CIDP spectrum to include typical CIDP and four well-defined CIDP variants. Patients with CIDP usually respond well to immunoglobulin therapy, steroids or plasmapheresis; however, 20-30% do not respond well, and approximately 15% remain refractory to all treatment modalities. Rituximab, mycophenolate mofetil and cyclophosphamide are of therapeutic benefit for some of these patients. Patients with some CIDP variants respond less well to immunotherapy, suggesting a difference in the pathogenic mechanisms underlying these variants. Potential novel treatments trialled in CIDP, targeting functionally relevant disease mechanisms, include neonatal Fc receptor blockers and complement inhibitors. These new treatment approaches are needed to optimise disease outcomes in refractory patients, and as an alternative for patients with suboptimal response requiring high doses or experiencing side effects from first-line therapies. Increasing the therapeutic options for patients with CIDP, particularly for refractory patients, highlights the need for more accurate diagnosis of typical CIDP and CIDP variants, objective evidence of treatment response and the need for reliable clinical biomarkers.

慢性炎症性脱髓鞘性多根神经病变(CIDP)是一种炎症性、免疫介导的周围神经和神经根神经病变。CIDP不太可能是一种独立的疾病实体,而是一系列相关疾病,其中细胞介导和体液机制协同作用导致周围神经损伤。欧洲神经病学学会/周围神经学会2021年关于CIDP诊断和治疗的指南修改了CIDP频谱,包括典型的CIDP和四种定义明确的CIDP变体。CIDP患者通常对免疫球蛋白治疗、类固醇或血浆置换反应良好;然而,20-30%的患者反应不佳,约15%的患者对所有治疗方式都难治。利妥昔单抗、霉酚酸酯和环磷酰胺对其中一些患者有治疗效果。一些CIDP变异的患者对免疫治疗的反应较差,这表明这些变异背后的致病机制存在差异。针对功能相关疾病机制的CIDP潜在新疗法包括新生儿Fc受体阻滞剂和补体抑制剂。需要这些新的治疗方法来优化难治性患者的疾病结局,并作为需要高剂量或经历一线治疗副作用的次优反应患者的替代方案。增加CIDP患者的治疗选择,特别是难治性患者,强调需要更准确地诊断典型CIDP和CIDP变异,治疗反应的客观证据以及可靠的临床生物标志物。
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引用次数: 0
Exploring the therapeutic potential of IL-17A inhibition in myasthenia gravis: a retrospective study on secukinumab. 探索IL-17A抑制在重症肌无力中的治疗潜力:对secukinumab的回顾性研究。
IF 2.4 Q3 CLINICAL NEUROLOGY Pub Date : 2025-11-11 eCollection Date: 2025-01-01 DOI: 10.1136/bmjno-2025-001249
Zhaoxu Zhang, Shuang Mei Zhang, Anrong Wang, Xiaodong Song, Pingbo Xu

Abstract:

Objective: Targeting interleukin-17A (IL-17A) represents a potential therapeutic strategy for myasthenia gravis (MG), but its clinical effects and immunomodulatory mechanisms remain incompletely defined. This study investigated the multidimensional efficacy and immune kinetic profile of secukinumab (SEC) in patients with MG.

Methods: We retrospectively enrolled acetylcholine receptor (AChR) antibody-positive (AChR-Ab+) generalised patients with MG (MG Foundation of America class IIa-IVb) treated at the Department of Neurology, Peking University People's Hospital between February 2023 and November 2024. All patients received SEC therapy (150 mg weekly for 4 weeks, followed by 150 mg every 4 weeks as maintenance for 24 weeks). Stable doses of conventional therapies (pyridostigmine, corticosteroids or immunosuppressive agents) were permitted. Longitudinal assessments were performed to evaluate changes in clinical scores (Quantitative MG (QMG) score, 15-item MG Quality of Life (MG-QOL15) scale, MG Activities of Daily Living (MG-ADL) scale), serum AChR-Ab titres, Th17/Tfh (T helper 17 cell/T follicular helper) cell frequencies and cytokine levels at multiple time points.

Results: A total of 29 patients with MG completed the study and were matched with 29 healthy controls. Following SEC treatment: (a) clinical outcomes: showed that by week 24, significant reductions from baseline were observed in QMG, MG-QOL15 and MG-ADL scores (reductions of 60.7%, 58.3% and 64.1%, respectively; all p<0.01). Significant clinical efficacy emerged by week 8 (25.0% reduction in QMG, p<0.01). (b) Correlations: the reduction in AChR-Ab titres strongly correlated with improvements in clinical scores (r=0.435-0.542, p<0.05). Furthermore, the decrease in Tfh cells showed a significant association with the decline in IL-6 levels (r=0.568-0.591, p<0.001).

Interpretation: In this study, SEC treatment was associated with rapid and sustained clinical improvement, as well as enhanced quality of life in patients with AChR-Ab+ MG. These benefits were correlated with modulation of the Th17/Tfh cell axis, downregulation of the IL-17A/IL-6 pathway and a reduction in autoantibody levels. These preliminary findings highlight the potential of IL-17A inhibition as a therapeutic strategy worthy of further investigation in MG.

摘要:目的:靶向白介素- 17a (IL-17A)是治疗重症肌无力(MG)的一种潜在的治疗策略,但其临床疗效和免疫调节机制尚不完全清楚。本研究探讨了secukinumab (SEC)在MG患者中的多维疗效和免疫动力学特征。方法:回顾性纳入2023年2月至2024年11月在北京大学人民医院神经内科治疗的乙酰胆碱受体(AChR)抗体阳性(AChR- ab +)全科MG(美国MG基金会IIa-IVb级)患者。所有患者均接受SEC治疗(每周150毫克,持续4周,随后每4周150毫克,维持24周)。允许使用稳定剂量的常规疗法(吡哆斯的明、皮质类固醇或免疫抑制剂)。纵向评估临床评分(定量MG (QMG)评分,15项MG生活质量(MG- qol15)量表,MG日常生活活动(MG- adl)量表),血清AChR-Ab滴度,Th17/Tfh (T辅助17细胞/T滤泡辅助)细胞频率和细胞因子水平在多个时间点的变化。结果:共有29名MG患者完成了研究,并与29名健康对照进行了匹配。(a)临床结果:显示到第24周,QMG、MG- qol15和MG- adl评分较基线显著降低(分别降低60.7%、58.3%和64.1%)。所有结果解释:在本研究中,SEC治疗与快速和持续的临床改善以及AChR-Ab+ MG患者的生活质量提高有关。这些益处与Th17/Tfh细胞轴的调节、IL-17A/IL-6通路的下调和自身抗体水平的降低有关。这些初步发现强调了IL-17A抑制作为一种值得进一步研究的治疗策略的潜力。
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引用次数: 0
Smartphone-based patient self-assessment of neuropathy grade: a validation study of the neuropathy tracker alignment with clinical tests. 基于智能手机的患者神经病变等级自我评估:神经病变追踪器与临床试验对齐的验证研究
IF 2.4 Q3 CLINICAL NEUROLOGY Pub Date : 2025-10-31 eCollection Date: 2025-01-01 DOI: 10.1136/bmjno-2025-001350
Astrid Emilie Kongsmark Madsen, Maria Joy Normann Haverberg, Jakob Eyvind Bardram, Martin Ballegaard

Background and aims: Repeated evaluation for grading of symptoms and signs in distal symmetrical peripheral neuropathy (DSPN) is a core aspect of patient care and inflicts a burden on both the patient and the healthcare system. We designed the smartphone-based Neuropathy Tracker to enable patients to document symptoms and perform a guided self-examination of signs of neuropathy by themselves. We investigate the alignment and classification strength of this grading with grading performed by professionals.

Methods: We included 21 patients with DSPN and 18 controls. In a test-retest paradigm, the patients used the Neuropathy Tracker, and they had examinations by clinical professionals to grade their neuropathy using the Utah Early Neuropathy Scale (UENS) and the Total Neuropathy Score - Clinical Version (TNSc). Further, they had quantitative examinations of Temperature Thresholds and Nerve Conduction Studies for amplitudes of Sural Sensory Nerve Action Potentials.

Results: In a Bland-Altman analysis of differences between repeated tests, we found similar 95% confidence limits between Neuropathy Tracker and UENS, while TNSc had fewer wide limits. The comparison between the Neuropathy Tracker and UENS showed no bias in test score differences. Classification with receiver operating curve analysis showed Neuropathy Tracker to be slightly inferior to UENS and TNSc to classify neuropathy, but still with excellent ability, with area under curve values of 0.903±0.053.

Conclusions: Patient-administered neuropathy grading using the Neuropathy Tracker could add to the clinical evaluation in DSPN without an in-person visit to the outpatient clinic.

背景和目的:远端对称性周围神经病变(DSPN)的症状和体征分级的反复评估是患者护理的一个核心方面,对患者和医疗保健系统都造成了负担。我们设计了基于智能手机的神经病变追踪器,使患者能够记录症状,并自行进行神经病变体征的指导自我检查。我们用专业人员进行的分级来调查这种分级的对齐和分类强度。方法:21例DSPN患者和18例对照组。在测试-再测试范式中,患者使用神经病变追踪器,他们接受临床专业人员的检查,使用犹他早期神经病变量表(UENS)和总神经病变评分-临床版(TNSc)对其神经病变进行评分。此外,他们还进行了温度阈值的定量检查和腓肠感觉神经动作电位幅度的神经传导研究。结果:在Bland-Altman重复试验之间的差异分析中,我们发现Neuropathy Tracker和UENS之间的95%置信限相似,而TNSc的范围较小。神经病变追踪器和UENS之间的比较没有显示出测试分数差异的偏倚。通过受试者工作曲线分析进行分类,Neuropathy Tracker对神经病变的分类能力略低于UENS和TNSc,但仍具有出色的能力,曲线下面积为0.903±0.053。结论:患者使用神经病变追踪器进行神经病变分级可以增加DSPN的临床评估,而无需亲自到门诊就诊。
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引用次数: 0
Pathophysiology of functional neurological disorder for the general neurologist. 一般神经科医生的功能性神经障碍病理生理学。
IF 2.4 Q3 CLINICAL NEUROLOGY Pub Date : 2025-10-31 eCollection Date: 2025-01-01 DOI: 10.1136/bmjno-2025-001309
Wendy Phillips

Functional neurological disorder (FND) is a common condition and outcome depends on good patient understanding. This article aims to summarise and simplify the prevailing model of FND, active inference, with key illustrative papers.

功能性神经障碍(FND)是一种常见的疾病,其预后取决于患者的良好理解。本文旨在总结和简化FND的主流模型,即主动推理,并提供关键的说明性论文。
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引用次数: 0
Macular OCT inner retinal changes reflect CNS involvement in m.3243A>G disease. 黄斑OCT视网膜内改变反映m.3243A >g病的中枢神经系统受累。
IF 2.4 Q3 CLINICAL NEUROLOGY Pub Date : 2025-10-20 eCollection Date: 2025-01-01 DOI: 10.1136/bmjno-2025-001232
Hatem Jouda, Andrew C Browning, Akhunzada M Aftab, Ikhlas Mahmoud, Saima Bibi, Robert McFarland, Sarah J Pickett, Helen Devine

Background: The m.3243A>G mitochondrial DNA variant is the most common cause of adult mitochondrial disease and is associated with a heterogeneous clinical phenotype. The retina and optic nerve are among the most metabolically active tissues, making them vulnerable to mitochondrial dysfunction. Optical coherence tomography (OCT) studies have demonstrated retinal nerve fibre layer (RNFL) thinning in mitochondrial and other neurodegenerative diseases. We investigated whether temporal RNFL thinning is associated with central nervous system (CNS) involvement in individuals with the m.3243A>G variant.

Methods: High-resolution OCT was used to assess peripapillary RNFL thickness and perform macular segmentation. Participants were categorised into normal RNFL (n=14) or temporal RNFL thinning (n=15) groups. Demographic data, mean-corrected m.3243A>G heteroplasmy, Newcastle Mitochondrial Disease Adult Scale (NMDAS) scaled scores and NMDAS neurological traits were compared.

Results: Temporal RNFL thinning was significantly associated with neurological features (Fisher's exact test, p=0.027). In multivariable analysis, RNFL thinning and age were independent predictors of neurological involvement. Macular OCT revealed concomitant thinning of the ganglion cell-inner plexiform (GC-IPL) complex in the RNFL thinning group, with preservation of outer retinal layers, supporting primary retinal ganglion cell vulnerability. No significant associations were found between RNFL thinning and m.3243A>G heteroplasmy or NMDAS scaled scores.

Conclusion: Temporal RNFL thinning, accompanied by GC-IPL loss, is associated with neurological involvement in m.3243A>G-related mitochondrial disease, supporting its potential as a non-invasive biomarker of CNS dysfunction. Longitudinal studies are needed to determine whether these retinal changes are progressive and predictive of neurological decline.

背景:m.3243A >g线粒体DNA变异是成人线粒体疾病的最常见原因,并与异质临床表型相关。视网膜和视神经是新陈代谢最活跃的组织,这使得它们很容易受到线粒体功能障碍的影响。光学相干断层扫描(OCT)研究表明,在线粒体和其他神经退行性疾病中,视网膜神经纤维层(RNFL)变薄。我们研究了m.3243A >g变异个体的颞叶RNFL变薄是否与中枢神经系统(CNS)受损伤有关。方法:采用高分辨率OCT评估乳头周围RNFL厚度并进行黄斑分割。参与者被分为正常RNFL组(n=14)和颞部RNFL变薄组(n=15)。比较人口统计学数据、经平均校正的m.3243A>G异质性、纽卡斯尔线粒体病成人量表(NMDAS)评分和NMDAS神经学特征。结果:颞部RNFL变薄与神经学特征显著相关(Fisher精确检验,p=0.027)。在多变量分析中,RNFL变薄和年龄是神经受累的独立预测因子。黄斑OCT显示,在RNFL减薄组中,神经节细胞-内丛状(GC-IPL)复合物同时变薄,视网膜外层保留,支持原发性视网膜神经节细胞易损性。RNFL变薄与m.3243A>G异质性或NMDAS评分之间无显著关联。结论:颞叶RNFL变薄,伴随GC-IPL丢失,与m.3243A> g相关线粒体疾病的神经系统受损伤有关,支持其作为中枢神经系统功能障碍的非侵入性生物标志物的潜力。需要纵向研究来确定这些视网膜变化是否是进行性的,并预测神经功能衰退。
{"title":"Macular OCT inner retinal changes reflect CNS involvement in m.3243A>G disease.","authors":"Hatem Jouda, Andrew C Browning, Akhunzada M Aftab, Ikhlas Mahmoud, Saima Bibi, Robert McFarland, Sarah J Pickett, Helen Devine","doi":"10.1136/bmjno-2025-001232","DOIUrl":"10.1136/bmjno-2025-001232","url":null,"abstract":"<p><strong>Background: </strong>The m.3243A>G mitochondrial DNA variant is the most common cause of adult mitochondrial disease and is associated with a heterogeneous clinical phenotype. The retina and optic nerve are among the most metabolically active tissues, making them vulnerable to mitochondrial dysfunction. Optical coherence tomography (OCT) studies have demonstrated retinal nerve fibre layer (RNFL) thinning in mitochondrial and other neurodegenerative diseases. We investigated whether temporal RNFL thinning is associated with central nervous system (CNS) involvement in individuals with the m.3243A>G variant.</p><p><strong>Methods: </strong>High-resolution OCT was used to assess peripapillary RNFL thickness and perform macular segmentation. Participants were categorised into normal RNFL (n=14) or temporal RNFL thinning (n=15) groups. Demographic data, mean-corrected m.3243A>G heteroplasmy, Newcastle Mitochondrial Disease Adult Scale (NMDAS) scaled scores and NMDAS neurological traits were compared.</p><p><strong>Results: </strong>Temporal RNFL thinning was significantly associated with neurological features (Fisher's exact test, p=0.027). In multivariable analysis, RNFL thinning and age were independent predictors of neurological involvement. Macular OCT revealed concomitant thinning of the ganglion cell-inner plexiform (GC-IPL) complex in the RNFL thinning group, with preservation of outer retinal layers, supporting primary retinal ganglion cell vulnerability. No significant associations were found between RNFL thinning and m.3243A>G heteroplasmy or NMDAS scaled scores.</p><p><strong>Conclusion: </strong>Temporal RNFL thinning, accompanied by GC-IPL loss, is associated with neurological involvement in m.3243A>G-related mitochondrial disease, supporting its potential as a non-invasive biomarker of CNS dysfunction. Longitudinal studies are needed to determine whether these retinal changes are progressive and predictive of neurological decline.</p>","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"7 2","pages":"e001232"},"PeriodicalIF":2.4,"publicationDate":"2025-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12542527/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145356698","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Dyadic Coping of NMOSD and MOGAD patients and their partners: a sociological and psychological examination of strategies (CoMMOnsense-Study). NMOSD和MOGAD患者及其伴侣的二元应对:策略的社会学和心理学检查(常识研究)。
IF 2.4 Q3 CLINICAL NEUROLOGY Pub Date : 2025-10-17 eCollection Date: 2025-01-01 DOI: 10.1136/bmjno-2025-001193
Anna Walz, Daria Tkachenko, Martin W Hümmert, Patrick Schindler, Judith Bellmann-Strobl, Friedemann Paul, Orhan Aktas, Marius Ringelstein, Katrin Giglhuber, Clarissa Zappe, Frank Leypoldt, Felix Lüssi, Ilya Ayzenberg, Carolin Schwake, Ulrike Wallwitz, Julian Reza Kretschmer, Franziska Bütow, Klemens Angstwurm, Leila Husseini, Vivien Häußler, Florian Then Bergh, Axel Haarmann, Dominik Lehrieder, Clemens Warnke, Tania Kümpfel, Daniel Engels, Tanja Zimmermann, Corinna Trebst

Background: Neuromyelitis optica spectrum disorders (NMOSD) and myelin oligodendrocyte glycoprotein antibody-associated diseases (MOGAD) impose psychological burdens on patients. Chronic illnesses create challenges for both patients and their partners, who also play a crucial role in managing disease-related stress. Despite its relevance, little is known about the role of dyadic coping (DC) in these conditions. This study investigates DC in NMOSD and MOGAD, aiming to provide clinical recommendations.

Methods: The CoMMOnsense-Study is a cross-sectional, prospective study of 59 NMOSD and 50 MOGAD patients and their respective partners, recruited from 15 centres of the German Neuromyelitis Optica Study Group registry. Participants completed self-report questionnaires on DC, depression, anxiety and quality of relationship. Correlation analyses were performed to compare findings based on antibody status. Subsequently, multivariate regression analyses were conducted to identify relevant predictors of DC.

Results: Patients with NMOSD and MOGAD demonstrated higher levels of depressive symptoms (NMOSD: p=0.007; MOGAD: p=0.023) and stress communication scores (NMOSD: p=0.022; MOGAD: p=0.013) than their partners. Negative coping was low across all subgroups (Stanine 1). Despite high DC and relationship quality, discrepancies were observed in the coping perceptions between partners.

Conclusions: Coping is highly shared within partnerships affected by NMOSD and MOGAD, while discrepancies in coping perceptions and protective buffering suggest the presence of unfavourable coping mechanisms. Reducing protective buffering and illness-related distortions shows potential areas for enhancing DC.

背景:视神经脊髓炎谱系障碍(NMOSD)和髓鞘少突胶质细胞糖蛋白抗体相关疾病(MOGAD)给患者带来心理负担。慢性疾病给患者和他们的伴侣都带来了挑战,他们在控制与疾病相关的压力方面也起着至关重要的作用。尽管其相关性,但对二元应对(DC)在这些条件下的作用知之甚少。本研究探讨DC在NMOSD和MOGAD中的作用,旨在为临床提供建议。方法:常识研究是一项横断面前瞻性研究,从德国视神经脊髓炎研究小组注册的15个中心招募59名NMOSD和50名MOGAD患者及其各自的伴侣。参与者完成了关于抑郁、抑郁、焦虑和关系质量的自我报告问卷。进行相关分析,比较基于抗体状态的结果。随后进行多变量回归分析,以确定DC的相关预测因素。结果:NMOSD和MOGAD患者的抑郁症状(NMOSD: p=0.007; MOGAD: p=0.023)和压力沟通评分(NMOSD: p=0.022; MOGAD: p=0.013)均高于其伴侣。所有亚组的消极应对均较低(图1)。尽管有较高的DC和关系质量,但在伴侣之间的应对感知上存在差异。结论:在受NMOSD和MOGAD影响的伙伴关系中,应对是高度共享的,而应对感知和保护性缓冲的差异表明存在不利的应对机制。减少保护性缓冲和与疾病相关的扭曲显示了增强DC的潜在领域。
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引用次数: 0
Organisational impact and patient management models for biomarker integration in multiple sclerosis care in Italy: the 0Tolerance project. 意大利多发性硬化症治疗中生物标志物整合的组织影响和患者管理模式:0Tolerance项目。
IF 2.4 Q3 CLINICAL NEUROLOGY Pub Date : 2025-10-17 eCollection Date: 2025-01-01 DOI: 10.1136/bmjno-2025-001269
Paolo Ragonese, Maria Chiara Buscarinu, Maria Cellerino, Elena Colombo, Jessica Frau, Maura Frigo, Marco Puthenparampil, Eleonora Rigoni, Valeria Zancan, Luca Pinto, Simone Parretti, Letizia Gazzaniga

Background: Despite advances in multiple sclerosis (MS) management, the need for more accurate biomarkers remains critical. Conventional MRI, while essential for diagnosis, prognosis and disease monitoring, has limitations in capturing the full complexity of disease progression. This paper aims to identify biomarkers likely to be available in clinical practice by 2028, define a prospective organisational follow-up model for patients with MS, explore organisational requirements and propose solutions to facilitate their implementation. These insights aim to inform and anticipate future discussions among policymakers regarding the adoption of prospective biomarkers into clinical practice.

Methods: A multimethod qualitative design was employed, including a systematic literature review of 82 studies, two modified Delphi consensus processes and semistructured interviews with nine neurologists and three healthcare programming experts, applying the Structural, Technological, Organisational and Professional (STOP) framework. The STOP framework was used to assess structural, technological, organisational and professional requirements and to explore solutions.

Results: The research identified a prospective organisational follow-up model that integrates the most probable prospective biomarkers into clinical practice. The prospective organisational follow-up model defined an optimal testing frequency of Serum Neurofilament Light Chain and Glial Fibrillar Acidic Protein every 6 months, as well as Cognitive Tests and Optical Coherence Tomography every 12 months. Combining biomarkers and aligning them with MRI was seen as beneficial. Despite the validation of the model through a modified Delphi consensus process based on organisational feasibility and economic sustainability, structural and organisational challenges need to be addressed to ensure smoother integration into clinical practice.

Conclusions: This article aims to define an organisational model for the integration of prospective biomarkers into clinical follow-up in MS. It also explores potential strategies to facilitate their transition from research settings to routine clinical practice. The proposed approach provides a framework with potential for replication across various care pathways.

背景:尽管多发性硬化症(MS)的治疗取得了进展,但对更准确的生物标志物的需求仍然至关重要。传统的MRI虽然对诊断、预后和疾病监测至关重要,但在捕捉疾病进展的全部复杂性方面存在局限性。本文旨在确定到2028年可能在临床实践中可用的生物标志物,定义MS患者的前瞻性组织随访模型,探索组织需求并提出促进其实施的解决方案。这些见解旨在告知和预测决策者之间关于将前瞻性生物标志物应用于临床实践的未来讨论。方法:采用多方法定性设计,采用结构、技术、组织和专业(STOP)框架,包括对82项研究的系统文献综述、2个改进的德尔菲共识过程和对9名神经科医生和3名卫生保健规划专家的半结构化访谈。STOP框架用于评估结构、技术、组织和专业要求,并探索解决方案。结果:该研究确定了一种前瞻性组织随访模型,该模型将最有可能的前瞻性生物标志物整合到临床实践中。前瞻性组织随访模型确定了每6个月一次的血清神经丝轻链和胶质纤维酸性蛋白的最佳检测频率,以及每12个月一次的认知测试和光学相干断层扫描。结合生物标志物并将其与MRI对齐被认为是有益的。尽管通过基于组织可行性和经济可持续性的改进德尔菲共识过程验证了该模型,但需要解决结构和组织方面的挑战,以确保更顺利地融入临床实践。结论:本文旨在定义一个将前瞻性生物标志物整合到ms临床随访中的组织模型,并探讨了促进其从研究环境过渡到常规临床实践的潜在策略。所提出的方法提供了一个框架,具有跨各种护理途径复制的潜力。
{"title":"Organisational impact and patient management models for biomarker integration in multiple sclerosis care in Italy: the 0Tolerance project.","authors":"Paolo Ragonese, Maria Chiara Buscarinu, Maria Cellerino, Elena Colombo, Jessica Frau, Maura Frigo, Marco Puthenparampil, Eleonora Rigoni, Valeria Zancan, Luca Pinto, Simone Parretti, Letizia Gazzaniga","doi":"10.1136/bmjno-2025-001269","DOIUrl":"10.1136/bmjno-2025-001269","url":null,"abstract":"<p><strong>Background: </strong>Despite advances in multiple sclerosis (MS) management, the need for more accurate biomarkers remains critical. Conventional MRI, while essential for diagnosis, prognosis and disease monitoring, has limitations in capturing the full complexity of disease progression. This paper aims to identify biomarkers likely to be available in clinical practice by 2028, define a prospective organisational follow-up model for patients with MS, explore organisational requirements and propose solutions to facilitate their implementation. These insights aim to inform and anticipate future discussions among policymakers regarding the adoption of prospective biomarkers into clinical practice.</p><p><strong>Methods: </strong>A multimethod qualitative design was employed, including a systematic literature review of 82 studies, two modified Delphi consensus processes and semistructured interviews with nine neurologists and three healthcare programming experts, applying the Structural, Technological, Organisational and Professional (STOP) framework. The STOP framework was used to assess structural, technological, organisational and professional requirements and to explore solutions.</p><p><strong>Results: </strong>The research identified a prospective organisational follow-up model that integrates the most probable prospective biomarkers into clinical practice. The prospective organisational follow-up model defined an optimal testing frequency of Serum Neurofilament Light Chain and Glial Fibrillar Acidic Protein every 6 months, as well as Cognitive Tests and Optical Coherence Tomography every 12 months. Combining biomarkers and aligning them with MRI was seen as beneficial. Despite the validation of the model through a modified Delphi consensus process based on organisational feasibility and economic sustainability, structural and organisational challenges need to be addressed to ensure smoother integration into clinical practice.</p><p><strong>Conclusions: </strong>This article aims to define an organisational model for the integration of prospective biomarkers into clinical follow-up in MS. It also explores potential strategies to facilitate their transition from research settings to routine clinical practice. The proposed approach provides a framework with potential for replication across various care pathways.</p>","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"7 2","pages":"e001269"},"PeriodicalIF":2.4,"publicationDate":"2025-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12542540/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145356679","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Relationship between personality and poststroke functional outcomes: a systematic review. 人格与脑卒中后功能结局的关系:一项系统综述。
IF 2.4 Q3 CLINICAL NEUROLOGY Pub Date : 2025-10-07 eCollection Date: 2025-01-01 DOI: 10.1136/bmjno-2025-001284
Desson Au-Yeung, Simon Matthews, Jonathan Hewitt, Benjamin Jelley

Background: Factors such as age and stroke severity are commonly used to predict poststroke functional outcomes and tailor stroke rehabilitation therapy. However, the role of personality in stroke rehabilitation and its influence on functional outcomes is unclear. This review aims to assess whether an association exists between personality and poststroke functional outcomes.

Methods: We searched Medline, AMED, APA PsychINFO, CENTRAL, CINAHL and Scopus for studies published between database inception and 22 October 2024. Studies were included if they recruited adults with stroke, used a validated method to assess personality and poststroke functional outcomes and were published in a peer-reviewed journal.

Results: Five studies were identified (n=424): four cohort and one cross-sectional. There were no major concerns regarding risk of bias. Methods of assessing personality and poststroke functional outcome both varied, with Eysenck's Personality Questionnaire and Barthel Index being the most frequently used. Extroversion, openness and lie-tendency were associated with improved poststroke functional outcomes, while type D personality was negatively associated with poststroke functional outcomes.

Conclusions: There is some evidence for an association between personality and poststroke functional outcomes, but this is limited by the small number of relevant studies and small sample sizes. Further studies are needed to investigate this potential relationship.

Prospero registration number: CRD42024592518.

背景:年龄和脑卒中严重程度等因素通常用于预测脑卒中后功能结局和定制脑卒中康复治疗。然而,人格在脑卒中康复中的作用及其对功能预后的影响尚不清楚。本综述旨在评估人格与脑卒中后功能预后之间是否存在关联。方法:检索Medline, AMED, APA PsychINFO, CENTRAL, CINAHL和Scopus,检索数据库建立至2024年10月22日之间发表的研究。如果研究招募了成年中风患者,使用一种有效的方法来评估性格和中风后的功能结果,并发表在同行评议的期刊上,那么这些研究就被纳入其中。结果:共纳入5项研究(n=424): 4项队列研究和1项横断面研究。没有关于偏倚风险的主要担忧。评估人格和脑卒中后功能结果的方法各不相同,最常用的是艾森克人格问卷和巴特尔指数。外向性、开放性和说谎倾向与卒中后功能预后的改善相关,而D型人格与卒中后功能预后呈负相关。结论:有一些证据表明人格与中风后功能结局之间存在关联,但这受到相关研究数量少和样本量小的限制。需要进一步的研究来调查这种潜在的关系。普洛斯彼罗注册号:CRD42024592518。
{"title":"Relationship between personality and poststroke functional outcomes: a systematic review.","authors":"Desson Au-Yeung, Simon Matthews, Jonathan Hewitt, Benjamin Jelley","doi":"10.1136/bmjno-2025-001284","DOIUrl":"10.1136/bmjno-2025-001284","url":null,"abstract":"<p><strong>Background: </strong>Factors such as age and stroke severity are commonly used to predict poststroke functional outcomes and tailor stroke rehabilitation therapy. However, the role of personality in stroke rehabilitation and its influence on functional outcomes is unclear. This review aims to assess whether an association exists between personality and poststroke functional outcomes.</p><p><strong>Methods: </strong>We searched Medline, AMED, APA PsychINFO, CENTRAL, CINAHL and Scopus for studies published between database inception and 22 October 2024. Studies were included if they recruited adults with stroke, used a validated method to assess personality and poststroke functional outcomes and were published in a peer-reviewed journal.</p><p><strong>Results: </strong>Five studies were identified (n=424): four cohort and one cross-sectional. There were no major concerns regarding risk of bias. Methods of assessing personality and poststroke functional outcome both varied, with Eysenck's Personality Questionnaire and Barthel Index being the most frequently used. Extroversion, openness and lie-tendency were associated with improved poststroke functional outcomes, while type D personality was negatively associated with poststroke functional outcomes.</p><p><strong>Conclusions: </strong>There is some evidence for an association between personality and poststroke functional outcomes, but this is limited by the small number of relevant studies and small sample sizes. Further studies are needed to investigate this potential relationship.</p><p><strong>Prospero registration number: </strong>CRD42024592518.</p>","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"7 2","pages":"e001284"},"PeriodicalIF":2.4,"publicationDate":"2025-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12506118/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145260246","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Rationale and design of 'StAtins in Frail oldEr patients with ischemic Stroke or Transient ischemic attack-the Randomized Controlled Trial' (SAFEST-RCT). “他汀类药物在老年体弱缺血性卒中或短暂性缺血性发作患者中的应用——随机对照试验”(safe - rct)的基本原理和设计。
IF 2.4 Q3 CLINICAL NEUROLOGY Pub Date : 2025-10-05 eCollection Date: 2025-01-01 DOI: 10.1136/bmjno-2025-001297
Susanna Rosa Prins, Birgit A Damoiseaux-Volman, Sarah E Vermeer, Patrick M M Bossuyt, Rik Van Eekelen, Judith E Bosmans, Eveline P Van Poelgeest, Fabrice M A C Martens, Marielle H Emmelot-Vonk, Esther Verstraete, Majon Muller, Eric P Moll Van Charante, Michiel Lindhout, Nathalie Van Der Velde, Renske M Van Den Berg-Vos

Introduction: Statin therapy is known to reduce subsequent cardiovascular events in patients who had an ischaemic stroke and transient ischaemic attack (TIA). However, its effectiveness and safety in frail older adults with a recent stroke or TIA are uncertain, leading to variations in clinical practice. 'StAtins in Frail oldEr patients with ischemic Stroke or Transient ischemic attack-the Randomized Controlled Trial' (SAFEST-RCT) aims to investigate the effectiveness of initiating versus not initiating statin therapy in this vulnerable population, to optimise secondary prevention strategies.

Methods and analysis: This multicentre, prospective, randomised, open-label study aims to enrol 612 frail adults ≥70 years with a recent acute ischaemic stroke or TIA across 22 Dutch hospitals. The study compares prescribing versus not prescribing statins in terms of health-related quality of life, major adverse cardiovascular event-free survival and societal costs over a 2-year follow-up period.

Ethics and dissemination: The SAFEST-RCT protocol was approved by the Ethics Committee of Amsterdam UMC. It complies with the Declaration of Helsinki and is classified as a healthcare evaluation. Recruitment began in March 2025. Results will be published in open access journals, presented at conferences, shared via the Dutch Brain Injury Association and integrated into national guidelines to support implementation in routine care.

Trial registration number: NCT06785727.

简介:已知他汀类药物治疗可减少缺血性卒中和短暂性缺血性发作(TIA)患者的后续心血管事件。然而,其在最近中风或TIA的体弱老年人中的有效性和安全性尚不确定,导致临床实践存在差异。“他汀类药物在老年体弱缺血性卒中或短暂性脑缺血发作患者中的应用——随机对照试验”(safe - rct)旨在研究在这一弱势人群中启动与不启动他汀类药物治疗的有效性,以优化二级预防策略。方法和分析:这项多中心、前瞻性、随机、开放标签的研究旨在招募来自荷兰22家医院的612名≥70岁、近期发生急性缺血性卒中或TIA的体弱成年人。该研究比较了处方和未处方他汀类药物在健康相关生活质量、主要不良心血管事件无生存和2年随访期间的社会成本。伦理与传播:阿姆斯特丹UMC伦理委员会批准了safety - rct方案。它符合《赫尔辛基宣言》,被归类为保健评估。招聘于2025年3月开始。研究结果将发表在开放获取期刊上,在会议上发表,通过荷兰脑损伤协会分享,并纳入国家指南,以支持在常规护理中实施。试验注册号:NCT06785727。
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引用次数: 0
Treatment outcomes in functional neurological disorder: a systematic review and meta-analysis exploring the influence of symptom chronicity. 功能性神经障碍的治疗结果:一项探讨症状慢性影响的系统综述和荟萃分析。
IF 2.4 Q3 CLINICAL NEUROLOGY Pub Date : 2025-10-05 eCollection Date: 2025-01-01 DOI: 10.1136/bmjno-2025-001150
Sarah T Thomas, Elizabeth T Thomas, Emily Schembri, Alexander C Lehn, David Dg Palmer

Background: Functional neurological disorder (FND) is a common cause of neurological disability with symptoms spanning motor, sensory and cognitive domains. While effective treatments exist, the impact of symptom chronicity on treatment outcomes is unclear. This systematic review and meta-analysis investigated whether longer symptom duration influences treatment outcomes across FND phenotypes: functional movement disorders, functional/dissociative seizures (FDS) and mixed presentations.

Methods: MEDLINE, Embase, Cochrane Central Register of Controlled Trials, PsycINFO and grey literature were systematically searched till 29 June 2024. Studies were included if they involved adult FND participants undergoing any intervention and evaluated symptom change, function and health-related quality of life (HrQoL). Studies were excluded with <10 participants, missing symptom duration data or irrelevant outcomes. Two reviewers independently extracted data and assessed risk of bias. Meta-analyses used random effects models, subgroup analyses and univariate meta-regression to examine associations with symptom chronicity.

Results: 63 studies met inclusion criteria; 27 studies (885 participants) were meta-analysed. Longer symptom duration modestly reduced improvements in motor symptoms (-3.24 points/year, scale: 0-100) and physical HrQoL (-1.2 points/year, scale: 0-100). Global improvements (mean Clinical Global Impression-Change 2.43, 95% CI: 2.28 to 2.59, scale: 1-7) and mental HrQoL gains (mean Short Form-Mental Component Summary +5.04 points, 95% CI: 1.67 to 8.41) were observed irrespective of chronicity. FDS frequency reduced after psychotherapy in eight of nine studies, even with prolonged symptoms.

Conclusions: Symptom chronicity modestly reduced motor and physical HrQoL improvements, but did not negate meaningful gains across a range of outcomes. Early diagnosis and treatment are critical for better outcomes, but remain beneficial in chronic stages.

背景:功能性神经障碍(FND)是一种常见的神经功能障碍,其症状跨越运动、感觉和认知领域。虽然存在有效的治疗方法,但症状的慢性性对治疗结果的影响尚不清楚。本系统综述和荟萃分析调查了更长症状持续时间是否会影响FND表型的治疗结果:功能性运动障碍、功能性/解离性癫痫(FDS)和混合表现。方法:系统检索MEDLINE、Embase、Cochrane Central Register of Controlled Trials、PsycINFO及灰色文献,检索截止至2024年6月29日。接受任何干预并评估症状改变、功能和健康相关生活质量(HrQoL)的成年FND参与者纳入研究。结果:63项研究符合纳入标准;27项研究(885名参与者)进行了荟萃分析。较长的症状持续时间适度降低了运动症状(-3.24分/年,评分范围:0-100)和身体HrQoL(-1.2分/年,评分范围:0-100)的改善。总体改善(平均临床总体印象-变化2.43,95% CI: 2.28至2.59,量表:1-7)和精神HrQoL的改善(平均短形式-精神成分总结+5.04分,95% CI: 1.67至8.41)与慢性无关。在9项研究中,有8项在心理治疗后FDS频率降低,即使症状延长。结论:慢性症状适度地降低了运动和身体HrQoL的改善,但并没有否定一系列结果的有意义的改善。早期诊断和治疗对于更好的结果至关重要,但在慢性阶段仍然有益。
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引用次数: 0
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BMJ Neurology Open
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