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Quality of life, productivity loss, and mental health service utilization among parents of children with neuroinflammatory disorders: A cross-sectional study 神经炎性疾病患儿父母的生活质量、生产力损失和心理健康服务利用:一项横断面研究
IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2026-01-27 DOI: 10.1016/j.msard.2026.107032
Ramesh Lamsal , E. Ann Yeh , Eleanor Pullenayegum , Wendy J. Ungar

Background

Children with neuroinflammatory disorders (NDs) require caregiving, affecting parental health and employment. This study assessed parents’ productivity losses, mental health services use, health-related quality of life (HRQoL), and care-related QoL.

Methods

This cross-sectional study enrolled children with NDs and parents at a single center. Data on productivity losses and mental health services use for both parents were collected. Respondent parents’ HRQoL and care-related QoL were measured using Health Utilities Index (HUI3) and Care-related Quality of Life (CarerQol) instrument. HUI3 scores were compared to Canadian parental norms. Annual parental costs per two-parent household and societal costs were estimated.

Results

Forty-seven parent-child dyads participated. Mean ages were 43.76 (SD 6.31) years for respondent parents and 12.00 (SD 3.34) years for children. Work and/or usual activity disruptions were reported by 86 % (31/36) of respondent parents and 78 % (28/36) of partners. Twenty-two percent (8/36) of respondent parents and 8 % (3/36) of partners used mental health services due to caregiving stress. Median (IQR) annual household costs for parents and society were CAD 2,767.87 (1,414.78–7,894.43). Mann-Whitney U test revealed no statistical difference in HUI-3 scores between parents of children with NDs (n = 46, median:0.93) and Canadian population parental norms (n = 2,799, median:0.93), W = 426434, p = 0.13. Mean CarerQol-7D and VAS scores were 85.97 (SD11.94) and 7.68 (SD1.08), respectively.

Conclusion

Parents of children with NDs reported work and/or daily activity disruptions and household costs; their HRQoL was comparable to Canadian parental norms. Larger studies with comparison groups of parents of healthy children are needed to confirm these findings.
背景:患有神经炎性疾病(NDs)的儿童需要照顾,这影响了父母的健康和就业。本研究评估了父母的生产力损失、心理健康服务的使用、健康相关生活质量(HRQoL)和护理相关生活质量。方法本横断面研究在单一中心招募NDs患儿及其父母。收集了父母双方生产力损失和心理健康服务使用情况的数据。采用健康效用指数(Health Utilities Index, HUI3)和护理相关生活质量(careqol)量表测量被调查者父母的HRQoL和护理相关生活质量。将HUI3分数与加拿大父母标准进行比较。估算了双亲家庭的年度父母成本和社会成本。结果47对亲子对参与调查。受访者父母的平均年龄为43.76岁(SD 6.31),儿童的平均年龄为12.00岁(SD 3.34)。86%(31/36)的受访者父母和78%(28/36)的受访者伴侣报告了工作和/或日常活动中断。22%(8/36)的应答父母和8%(3/36)的伴侣因照顾压力而使用心理健康服务。父母和社会的年度家庭成本中位数(IQR)为2,767.87加元(1,414.78-7,894.43)。Mann-Whitney U检验显示,NDs患儿的父母(n = 46,中位数:0.93)与加拿大人口父母标准(n = 2799,中位数:0.93)之间的HUI-3得分无统计学差异,W = 426434, p = 0.13。平均CarerQol-7D和VAS评分分别为85.97 (SD11.94)和7.68 (SD1.08)。结论NDs患儿的父母报告了工作和/或日常活动中断和家庭成本;他们的HRQoL与加拿大父母的标准相当。需要对健康儿童的父母进行更大规模的研究来证实这些发现。
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引用次数: 0
Real-world evidence of clinical outcomes and adherence to ofatumumab in the UK and the impact of a patients support programme: A retrospective, non-interventional cohort study ofatumumab在英国临床结果和依从性的真实证据以及患者支持计划的影响:一项回顾性,非介入性队列研究
IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2026-01-27 DOI: 10.1016/j.msard.2026.107030
David Paling , David Cottrell , Ruth Hamlin , Colin O’Leary , Owen Pearson , Agne Straukiene , Pyry Kivi , Wendy Rice , Sarah Al-Azki , Nazanin Kondori

Background

Ofatumumab is the only anti-CD20 monoclonal antibody disease modifying therapy available for self- administration for adults with active relapsing remitting multiple sclerosis (RMS). This study aimed to investigate real-world adherence and clinical outcomes of ofatumumab-treated patients in the UK and looked at the impact of a patient support program called Kesimpta Connect (KC PSP) on these outcomes.

Methods

This retrospective observational cohort study examined adherence to ofatumumab in 155 RMS patients recruited from six UK MS centres. The primary objective of the study was to describe the medication possession ratio (MPR) adherence of ofatumumab treated patients over a 13-month period. The study also looked at clinical effectiveness measures, including change in annualized relapse rates (ARR). Safety and secondary-care use was also assessed in this study.

Results

103 patients had data available to be assessed for adherence and clinical effectiveness. 87 % (n=90) of patients had an MPR adherence of 0.80 or greater and higher proportion of non-KC PSP users reported adherence of greater than 0.80 than KC PSP users (98.4 % vs 69.2 %). The total ARR decreased during the follow-up period from 0.33 (95 % CI 0.23-0.40) prior to ofatumumab initiation to 0.06 (95 % CI 0.01-0.11) 12-months after initiation. In terms of safety, 38.1 % (n=59/155) patients reported experiencing an adverse event during the follow-up period; one was reported to be severe. However, no patients reported discontinuing ofatumumab during the study follow-up period.

Conclusion

This study provides evidence of good adherence for RMS patients using ofatumumab. Further, there is evidence of clinical effectiveness based on reduction in ARR post-ofatumumab initiation, as well as tolerability and a favourable safety profile. The results of this study support the continued use of ofatumumab in providing effective RMS disease management in the UK NHS.
背景:dofatumumab是唯一一种抗cd20单克隆抗体疾病修饰疗法,可用于成人活动性复发缓解型多发性硬化症(RMS)患者的自我给药。本研究旨在调查英国ofatumumab治疗患者的现实依从性和临床结果,并研究了称为Kesimpta Connect (KC PSP)的患者支持计划对这些结果的影响。方法:本回顾性观察队列研究调查了来自英国6个多发性硬化症中心的155名RMS患者对ofatumumab的依从性。该研究的主要目的是描述13个月期间ofatumumab治疗患者的药物占有比(MPR)依从性。该研究还考察了临床有效性指标,包括年化复发率(ARR)的变化。本研究还评估了安全性和二级保健使用情况。结果103例患者有可用于依从性和临床疗效评估的数据。87% (n=90)患者的MPR依从性为0.80或更高,非KC PSP使用者报告的MPR依从性大于0.80的比例高于KC PSP使用者(98.4% vs 69.2%)。在随访期间,总ARR从ofatumumab开始治疗前的0.33 (95% CI 0.23-0.40)下降到开始治疗后12个月的0.06 (95% CI 0.01-0.11)。在安全性方面,38.1% (n=59/155)的患者报告在随访期间出现不良事件;据报道,其中一人伤势严重。然而,在研究随访期间,没有患者报告停止使用阿图单抗。本研究为RMS患者使用ofatumumab提供了良好的依从性证据。此外,有证据表明临床有效性基于ofatumumab启动后ARR的降低,以及耐受性和良好的安全性。本研究结果支持继续使用ofatumumab在英国NHS提供有效的RMS疾病管理。
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引用次数: 0
Incidence of multiple sclerosis in UK general practice; Risk of infections and mortality 多发性硬化症在英国全科医生的发病率;感染和死亡风险
IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2026-01-27 DOI: 10.1016/j.msard.2026.107029
Ana Ruigomez , Parminderjit Kaur Jayia , Luis-Alberto Garcia-Rodriguez
Multiple sclerosis (MS) is a leading cause of severe neurological disability and is associated with substantial comorbidity. Infections represent a major complication, and patients with MS have an increased risk of mortality.
This population-based descriptive cohort study examined the natural history of MS in general practice, focusing on incidence, comorbidities, infection risk, and mortality. Patients with incident MS between 2000 and 2016 were identified from the Clinical Practice Research Datalink (CPRD) and compared with a matched cohort from the general population. Incidence rates, associated risk factors, and subsequent risks of infection and mortality were assessed.
The incidence rate of MS was 8.84 cases per 100,000 person-years (95% confidence interval: 8.63–9.06), with higher rates observed in females and individuals aged 30–49 years. Comorbidities and prior infections were significantly associated with an MS diagnosis. Patients with MS had an increased risk of developing infections, particularly central nervous system infections (hazard ratio: 3.43; 95% confidence interval: 1.90–6.19). Mortality rate among patients with MS was nearly twice that of the general population. Male sex, smoking, and being underweight were associated with an elevated risk of mortality, while respiratory diseases and infections were the most common causes of death.
These findings, derived from routine general practice data, provide valuable insights for clinicians managing patients with MS. They highlight the increased susceptibility of patients with MS to infections and their elevated mortality risk. Furthermore, they identify modifiable risk factors, such as smoking and comorbidities, that may be targeted to improve patient outcomes.
多发性硬化症(MS)是严重神经功能障碍的主要原因,并伴有大量合并症。感染是主要的并发症,多发性硬化症患者的死亡风险增加。这项以人群为基础的描述性队列研究在一般实践中检查了MS的自然史,重点关注发病率、合并症、感染风险和死亡率。从临床实践研究数据链(CPRD)中确定2000年至2016年间发生多发性硬化症的患者,并与来自普通人群的匹配队列进行比较。评估了发病率、相关危险因素以及随后的感染和死亡风险。MS的发病率为每10万人年8.84例(95%可信区间:8.63-9.06),女性和30-49岁人群的发病率较高。合并症和既往感染与MS诊断显著相关。MS患者发生感染的风险增加,特别是中枢神经系统感染(风险比:3.43;95%可信区间:1.90-6.19)。多发性硬化症患者的死亡率几乎是一般人群的两倍。男性、吸烟和体重过轻与死亡风险增加有关,而呼吸道疾病和感染是最常见的死亡原因。这些发现来源于常规的全科实践数据,为临床医生管理多发性硬化症患者提供了有价值的见解。他们强调了多发性硬化症患者对感染的易感性增加以及死亡风险增加。此外,他们确定了可改变的风险因素,如吸烟和合并症,这可能是改善患者预后的目标。
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引用次数: 0
Global, regional, and national burden of multiple sclerosis from 1990 to 2021 and projections to 2040: A comprehensive analysis from the global burden of disease study. 1990 - 2021年全球、区域和国家多发性硬化症负担及2040年预测:来自全球疾病负担研究的综合分析
IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2026-01-23 DOI: 10.1016/j.msard.2026.107027
Jing Tan, Daobin Han

Background: Multiple sclerosis (MS) is a leading cause of neurological disability in young adults, with a striking female predominance. Understanding of its temporal and geographic patterns is crucial for informing public health strategies.

Methods: Data from the Global Burden of Disease (GBD) 2021 study were used to assess the burden of MS from 1990 to 2021 at global, regional, and national levels. Estimates of incidence, deaths, and disability-adjusted life years (DALYs) were stratified by age, sex, and Socio-demographic Index (SDI). Temporal trends were evaluated using the estimated annual percentage change (EAPC). Bayesian Age-Period-Cohort (BAPC) modeling was employed to forecast the burden through 2040. Additionally, frontier analysis, decomposition analysis, inequality assessment, and estimation of the smoking-attributable burden were conducted.

Results: Between 1990 and 2021, the absolute numbers of MS incident cases, deaths, and DALYs increased globally due to population growth and aging. In contrast, age-standardized incidence rates remained relatively constant, while both age-standardized mortality and DALYs rates experienced modest declines. Females consistently bore a higher burden across all metrics and age groups. Frontier analysis revealed significant performance gaps, and BAPC projections suggested a continued gradual decline in global age-standardized mortality and DALY rates through 2040. Despite these encouraging trends, smoking remained a substantial modifiable risk factor.

Conclusion: MS continues to impose a significant global burden, with persistent regional and socioeconomic disparities. This highlights the necessity for targeted public health strategies, including early diagnosis, accessible treatment, functional rehabilitation, and preventive measures such as smoking cessation, to reduce disparities and optimize outcomes worldwide.

背景:多发性硬化症(MS)是年轻人神经功能障碍的主要原因,以女性为主。了解其时间和地理格局对于为公共卫生战略提供信息至关重要。方法:采用全球疾病负担(GBD) 2021研究的数据,在全球、地区和国家层面评估1990年至2021年MS的负担。发病率、死亡率和残疾调整生命年(DALYs)的估计按年龄、性别和社会人口指数(SDI)分层。使用估计的年百分比变化(EAPC)来评估时间趋势。采用贝叶斯年龄-时期-队列(BAPC)模型预测到2040年的负担。此外,还进行了前沿分析、分解分析、不平等评估和吸烟归因负担估算。结果:1990年至2021年间,由于人口增长和老龄化,全球多发性硬化症病例、死亡和DALYs的绝对数量增加。相比之下,年龄标准化发病率保持相对稳定,而年龄标准化死亡率和伤残调整生命年均略有下降。在所有指标和年龄组中,女性一直承受着更高的负担。前沿分析显示了显著的绩效差距,BAPC预测表明,到2040年,全球年龄标准化死亡率和DALY率将继续逐步下降。尽管有这些令人鼓舞的趋势,吸烟仍然是一个重大的可改变的危险因素。结论:多发性硬化症继续造成重大的全球负担,持续存在区域和社会经济差异。这突出表明有必要制定有针对性的公共卫生战略,包括早期诊断、可获得的治疗、功能康复和戒烟等预防措施,以缩小全球差距并优化结果。
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引用次数: 0
First demyelinating attack in children: A twelve year single center cohort. 儿童首次脱髓鞘发作:12年单中心队列。
IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2026-01-22 DOI: 10.1016/j.msard.2026.107026
Alessandro Santagostino Barbone, Thea Giacomini, Silvia Casabona, Elisa De Grandis, Lucrezia Sartore, Maria Grazia Calevo, Federica Maria Bozzano, Emanuela Maria Mobilia, Matilde Inglese, Maria Cellerino, Maria Stella Vari, Ramona Cordani, Giampaola Pesce, Elisabetta Amadori, Edoardo Canale, Pasquale Striano, Andrea Rossi, Martina Resaz, Silvia Buratti, Giacomo Brisca, Luana Benedetti, Diego Franciotta, Matteo Gastaldi, Lino Nobili, Maria Margherita Mancardi

Introduction: Acquired demyelinating syndromes (ADS) of the central nervous system in children present a diagnostic challenge due to overlapping presentations. Differentiating monophasic from potentially recurrent conditions, such as pediatric-onset multiple sclerosis (POMS), myelin-oligodendrocyte glycoprotein antibody-associated disease (MOGAD), and other seronegative ADS, is essential for treatment and prognosis. This study aimed to characterize the initial presentation of pediatric ADS and evaluate the evolution of diagnosis over time to better guide treatment.

Methods: A retrospective study of 59 children with ADS diagnosed at a tertiary pediatric center in Italy (2012-2024) was conducted. Initial classifications included MS, acute disseminated encephalomyelitis (ADEM), clinically isolated syndrome (CIS), optic neuritis (ON), neuromyelitis optica spectrum disorder (NMOSD), or "Indeterminate". Final diagnoses were categorized as MS, MOGAD, or "Other" demyelinating conditions. Demographic, clinical, MRI, CSF, and outcome were analyzed. Statistical comparisons among groups used Mann-Whitney U, Chi-square, or Fisher's exact tests (p < 0.05).

Results: At final diagnosis, older age at onset, absence of preceding infection and characteristic MRI findings (periventricular/callosal lesions, cerebellar involvement) were more frequent in MS group. Younger age, preceding infection, fever, irritability, and cortical involvement were associated with MOGAD. Nearly one-third of final MS cases (29%) were initially CIS or ON, while no ADEM cases converted to MS. "Other" ADS (non-MS/MOG-IgG antibody negative patients) showed more severe initial disability (p = 0.01). Transition to adult neurology was significantly higher in MS (p < 0.001).

Conclusion: these findings underscore the heterogeneity of pediatric ADS at onset and the value of accurate acute management and longitudinal follow-up to refine diagnosis and guide treatment.

儿童中枢神经系统的获得性脱髓鞘综合征(ADS)由于重叠表现而呈现诊断挑战。区分单相与潜在复发性疾病,如儿科起病多发性硬化症(POMS)、髓鞘-少突胶质细胞糖蛋白抗体相关疾病(MOGAD)和其他血清阴性ADS,对于治疗和预后至关重要。本研究旨在描述小儿ADS的初始表现,并评估诊断随时间的演变,以更好地指导治疗。方法:对意大利某三级儿科中心诊断的59例ADS患儿(2012-2024年)进行回顾性研究。最初的分类包括MS、急性播散性脑脊髓炎(ADEM)、临床孤立综合征(CIS)、视神经炎(ON)、视神经脊髓炎视谱障碍(NMOSD)或“不确定”。最终诊断为多发性硬化症、MOGAD或“其他”脱髓鞘疾病。对人口统计学、临床、MRI、CSF和结果进行分析。组间统计比较采用Mann-Whitney U、卡方检验或Fisher精确检验(p < 0.05)。结果:在最终诊断时,MS组发病年龄较大,既往无感染,MRI特征性表现(脑室周围/胼胝体病变,小脑受累)更为常见。年龄较小、既往感染、发热、易怒和皮层受累与MOGAD相关。近三分之一的最终多发性硬化症患者(29%)最初是CIS或ON,而没有ADEM患者转化为多发性硬化症。“其他”ADS(非ms /MOG-IgG抗体阴性患者)表现出更严重的初始残疾(p = 0.01)。多发性硬化症患者向成人神经内科过渡的比例显著高于其他患者(p < 0.001)。结论:这些发现强调了儿童ADS发病的异质性,以及准确的急性管理和纵向随访对改进诊断和指导治疗的价值。
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引用次数: 0
Multiple sclerosis and related disorders multiple sclerosis across Isfahan Province, Iran: Urban–rural disparities and no association with air quality measures 伊朗伊斯法罕省的多发性硬化症:城乡差异与空气质量措施无关。
IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2026-01-20 DOI: 10.1016/j.msard.2026.107025
Masoud Etemadifar , Shakila Bagheri , Ahmadreza Dehghani , Fatemeh Sabeti , Mohsen Raeisidehkordi , Mehri Salari , Kamran Rezaei

Background

Multiple sclerosis (MS) prevalence varies worldwide and appears to be rising in Iran. Environmental factors like air pollution have been hypothesized to contribute to MS risk. This study aimed to map MS prevalence across Isfahan Province and evaluate differences by sex and urban versus rural setting, as well as to assess any association between ambient air pollution and MS prevalence.

Methods

Data from 11,456 provincially registered MS patients in 28 counties in Isfahan, Iran, were examined in this cross-sectional ecological study. Official population estimates were used to calculate the county-level MS point prevalence per 100,000 population in 2023, stratified by sex. Ratios of females to males were calculated. Annual average PM₂.₅ levels for each county were obtained from state sources for the same period. MS prevalence was compared between urban and rural counties, and correlation/regression analyses were used to test associations with pollutant levels.

Results and conclusion

The prevalence of MS varied widely, ranging from 322.7 in Isfahan County to 15.4 per 100,000 in rural Jarghouyeh. Although there was no significant correlation between the sex disparity and overall prevalence, females were more affected in every county (F/M ratio: 2.0–9.0). Although PM₂.₅ levels in Isfahan were extremely high (annual mean=41 μg/m³, >8 times the WHO guideline of 5 μg/m³), we found no significant correlation between ambient pollutant concentrations and MS prevalence in the province.
背景:多发性硬化症(MS)的患病率在世界范围内有所不同,在伊朗似乎正在上升。像空气污染这样的环境因素被假设会导致多发性硬化症的风险。本研究旨在绘制伊斯法罕省多发性硬化症患病率地图,并评估性别、城市与农村环境的差异,以及评估环境空气污染与多发性硬化症患病率之间的关系。方法:对伊朗伊斯法罕28个县11456名省登记的多发性硬化症患者的数据进行了横断面生态学研究。使用官方人口估计数计算2023年每10万人中按性别分层的县级多发性硬化症点患病率。计算了男女比例。年平均PM 2。每个县的₅水平是同一时期从国家来源获得的。比较了城市和农村县之间的多发性硬化症患病率,并使用相关/回归分析来检验与污染物水平的关系。结果与结论:MS患病率差异很大,伊斯法罕县为322.7 / 10万,贾尔古耶农村为15.4 / 10万。性别差异与总体患病率无显著相关性,但各县女性患病率较高(F/M比值为2.0 ~ 9.0)。尽管₂下午。伊斯法罕的₅水平非常高(年平均值=41 μg/m³,是世卫组织5 μg/m³指南的80倍),我们发现该省环境污染物浓度与MS患病率之间没有显着相关性。
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引用次数: 0
Inter-assay variability in anti–JC virus testing for natalizumab: A Spanish cohort 纳塔利珠单抗抗jc病毒检测的测定间变异性:一项西班牙队列研究
IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2026-01-19 DOI: 10.1016/j.msard.2026.107023
Luis Fernández Espigares , Alicia Fernández Panadero , María Díaz Sánchez , José Luis Casado Chocán , Eduardo Durán Ferreras

Objective

Accurate anti–JC virus (JCV) antibody testing is essential for progressive multifocal leukoencephalopathy (PML) risk stratification in natalizumab-treated patients. This study assessed evaluated inter-assay concordance between ImmunoWELL™ JCV IgG and STRATIFYJCV™ DxSelect™.

Methods

We conducted a retrospective cross-sectional inter-assay concordance study including 48 patients with multiple sclerosis treated with natalizumab. Paired serum samples were analyzed using both assays. Binary serostatus and ordinal risk categories (negative, low, intermediate, high) according to antibody index value were compared.

Results

STRATIFYJCV™ identified 4.2% of patients as JCV positive versus 33.3% with ImmunoWELL™, an eightfold difference. Overall binary agreement was 66.7%, with PABAK 0.33 (95% CI 0.12–0.54) and significant directional discordance (p = 0.0005). Most discordant results reflected upward reclassification from negative to low or intermediate categories. Only one patient changed high-risk status. A single discordant negative result was identified.

Conclusions

ImmunoWELL™ and STRATIFYJCV™ show relevant inter-assay variability, predominantly affecting low antibody index values, while preserving substantial agreement at clinically decisive high-risk thresholds. These findings support cautious interpretation of ImmunoWELL™ results and highlight the need for further virological validation.
目的准确的抗jc病毒(JCV)抗体检测对纳他单抗治疗的进行性多灶性白质脑病(PML)患者进行风险分层至关重要。本研究评估了ImmunoWELL™JCV IgG和STRATIFYJCV™DxSelect™检测间的一致性。方法我们对48例接受natalizumab治疗的多发性硬化症患者进行了回顾性横断面分析间一致性研究。使用这两种方法分析配对血清样本。比较二值血清状态和按抗体指标值依次分为阴性、低、中、高危险等级。结果:stratifyjcv™鉴定出4.2%的患者为JCV阳性,而ImmunoWELL™鉴定出33.3%的患者为JCV阳性,差异为8倍。总体二元一致性为66.7%,PABAK为0.33 (95% CI 0.12-0.54),显著的方向不一致(p = 0.0005)。大多数不一致的结果反映了从负面到低或中等类别的向上重新分类。只有1例患者改变了高危状态。鉴定出一个不一致的阴性结果。结论:simmunowell™和STRATIFYJCV™显示出相关的检测间变异性,主要影响低抗体指数值,而在临床决定性的高危阈值上保持了实质性的一致性。这些发现支持对ImmunoWELL™结果的谨慎解释,并强调需要进一步的病毒学验证。
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引用次数: 0
Baseline severe disability as a predictor of treatment escalation to rituximab in AQP4-IgG–positive NMOSD patients: A retrospective cohort study 基线严重残疾作为aqp4 - igg阳性NMOSD患者升级到利妥昔单抗治疗的预测因素:一项回顾性队列研究
IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2026-01-18 DOI: 10.1016/j.msard.2026.107022
Chayangkoon Siripornmongkol, Metha Apiwattanakul, Saharat Aungsumart

Introduction

Azathioprine and mycophenolate mofetil remain widely used as first-line immunosuppressive therapies for neuromyelitis optica spectrum disorder (NMOSD) in resource-limited settings. However, a subset of patients experience ongoing disease activity or treatment intolerance requiring escalation to rituximab. Identification of clinical factors associated with treatment escalation may improve treatment strategies.

Objectives

To identify clinical factors associated with escalation from azathioprine and mycophenolate mofetil to rituximab in AQP4-IgG–positive NMOSD patients.

Methods

We conducted a retrospective cohort study of AQP4-IgG–positive NMOSD patients treated at the Neurological Institute of Thailand between 2019 and 2024. Patients initially treated with azathioprine or mycophenolate mofetil were followed until escalation to rituximab or last follow-up. Baseline demographic, clinical, and laboratory variables were analyzed using logistic regression to identify factors associated with treatment escalation.

Results

Among 173 patients, 35 (20.23%) required escalation to rituximab. In multivariable analysis, baseline severe disability (EDSS ≥6 prior to first-line therapy) was independently strongly associated with treatment escalation to rituximab (OR 18.19, 95% CI 2.22–148.95; p=0.007). Other demographic and laboratory variables were not independently associated.

Conclusion

Baseline severe disability was strongly associated with subsequent escalation to rituximab. Early identification of high-risk patients may support more individualized treatment strategies and inform future policy decisions in resource-limited healthcare systems.
在资源有限的情况下,硫唑嘌呤和霉酚酸酯仍被广泛用作治疗视神经脊髓炎谱系障碍(NMOSD)的一线免疫抑制疗法。然而,一部分患者经历持续的疾病活动或治疗不耐受,需要升级到利妥昔单抗。确定与治疗升级相关的临床因素可以改善治疗策略。目的探讨aqp4 - igg阳性NMOSD患者从硫唑嘌呤和霉酚酸酯向利妥昔单抗升级的相关临床因素。方法对2019年至2024年在泰国神经病学研究所接受治疗的aqp4 - igg阳性NMOSD患者进行回顾性队列研究。患者最初用硫唑嘌呤或霉酚酸酯治疗,直到升级到利妥昔单抗或最后一次随访。使用逻辑回归分析基线人口统计学、临床和实验室变量,以确定与治疗升级相关的因素。结果173例患者中,35例(20.23%)需要升级至利妥昔单抗。在多变量分析中,基线严重残疾(一线治疗前EDSS≥6)与治疗升级为利妥昔单抗独立强相关(OR 18.19, 95% CI 2.22-148.95; p=0.007)。其他人口统计学和实验室变量没有独立关联。结论:基线严重残疾与随后升级使用利妥昔单抗密切相关。在资源有限的卫生保健系统中,早期识别高风险患者可能支持更个性化的治疗策略,并为未来的政策决策提供信息。
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引用次数: 0
Low prevalence of inflammatory bowel disease among patients with seropositive neuromyelitis optica spectrum disorder in two large referral centers 在两个大型转诊中心,血清阳性视神经脊髓炎谱系障碍患者中炎性肠病的患病率较低
IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2026-01-17 DOI: 10.1016/j.msard.2026.107021
Carolyn Akers , Philippe A. Bilodeau , Mattia Wruble Clark , Taymour Hashemzadeh , Michael Levy , Shamik Bhattacharyya , Ilya Kister
Patients with Neuromyelitis Optica Spectrum Disorders (NMOSD) have a well-recognized predilection to autoimmune comorbidities, of which systemic lupus erythematosus (SLE), Sjögren's syndrome (SjS), and autoimmune thyroid diseases are the most common. The question of whether some autoimmune diseases, such as inflammatory bowel disease (IBD), may not be overrepresented (or may even be underrepresented) in NMOSD has not received much attention. Here, we retrospectively reviewed the electronic medical records of all patients diagnosed with NMOSD in our two large referral centers (NYU and MGB), as well as patients with two neuroinflammatory disorders as comparator groups (myelin oligodendrocyte glycoprotein antibody disease (MOGAD) and MS), and calculated the rates of IBD, SLE, SjS, autoimmune thyroid disorders, and myasthenia gravis (MG) in these groups. We found that 1 out of 347 NMOSD patients had a diagnosis of IBD (0.3%), and this patient was seronegative for anti-AQP4 autoantibodies, while 4/271 patients in the MS group (1.5%), 2 of whom were exposed to anti-CD20 therapy, had IBD, and 2/366 MOGAD patients (0.8%), neither of whom was exposed to anti-CD20 therapy, had IBD. In contrast, the rate of the other four autoimmune conditions was significantly higher in NMOSD (24%) than in MOGAD (8.5%, p < 0.0001) or MS (5.2%, p < 0.0001). Thus, IBD was not diagnosed in any of our 317 seropositive NMOSD patients, despite the fact that this group had a higher use of B-cell depletion than patients with MS and MOGAD. We discuss the various hypotheses that may explain this observation.
神经脊髓炎视谱障碍(NMOSD)患者普遍倾向于自身免疫性合并症,其中系统性红斑狼疮(SLE)、Sjögren综合征(SjS)和自身免疫性甲状腺疾病是最常见的。一些自身免疫性疾病,如炎症性肠病(IBD),在NMOSD中是否可能被过度代表(或甚至可能被低估)的问题尚未得到太多关注。在这里,我们回顾性地回顾了在我们的两个大型转诊中心(NYU和MGB)诊断为NMOSD的所有患者的电子病历,以及两种神经炎性疾病作为比较组(髓鞘少突胶质细胞糖蛋白抗体病(MOGAD)和MS)的患者,并计算了这些组中IBD、SLE、SjS、自身免疫性甲状腺疾病和重症肌无力(MG)的发生率。我们发现347例NMOSD患者中有1例诊断为IBD(0.3%),该患者抗aqp4自身抗体血清阴性,而MS组中有4/271(1.5%)患者(其中2例接受抗cd20治疗)患有IBD,而MOGAD组中有2/366(0.8%)患者(均未接受抗cd20治疗)患有IBD。相比之下,NMOSD中其他四种自身免疫性疾病的发生率(24%)明显高于MOGAD (8.5%, p < 0.0001)或MS (5.2%, p < 0.0001)。因此,在我们的317例血清阳性NMOSD患者中,没有诊断出IBD,尽管这组患者比MS和MOGAD患者使用更高的b细胞消耗。我们讨论了可能解释这一观察结果的各种假设。
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引用次数: 0
Clinical, imaging, and immunological features of GFAP astrocytopathy: a systematic review with regional comparison GFAP星形细胞病的临床、影像学和免疫学特征:系统回顾和区域比较
IF 2.9 3区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2026-01-17 DOI: 10.1016/j.msard.2026.107019
Ekdanai Uawithya , Piyachai Siriphiphatcharoen , Ben Benjapibal , Supawit Kittipadakul , Sasitorn Siritho

Objective

This systematic review aims to analyze the clinical, laboratory, and imaging characteristics of GFAP astrocytopathy, with attention to differences between Asian and non-Asian populations.

Methods

We searched PubMed, Embase, and Scopus from inception through June 2025 for case reports, case series, and observational studies involving adult patients with GFAP astrocytopathy. Fifteen studies, including 538 patients, were analyzed. Data on clinical presentations, MRI findings, and laboratory results were extracted and compared across regions.

Results

We identified a total of 2,272 studies, of which 15 were included in our final analysis, comprising 538 patients. Our analysis identified three main clinical phenotypes: meningoencephalitis (164/371, 44.2%), meningoencephalomyelitis (94/371, 25.3%), and myelitis (26/371, 7.0%). The most common clinical symptoms were headache (186/470, 39.6%), abnormal movements (160/473, 33.8%), fever (172/538, 32.0%), and psychiatric symptoms (111/471, 23.6%). MRI findings frequently showed lesions in the periventricular (85/434, 19.6%) and subcortical regions (80/432, 18.5%). Perivascular and meningeal enhancements represented the most common radiological findings. Laboratory findings revealed positive GFAP antibodies in either serum or CSF. Co-existence of other antibodies in CSF and serum was also observed, most notably anti-AQP4, anti-MOG, and anti-NMDAR.

Conclusion

GFAP astrocytopathy is characterized by a wide range of clinical and radiological manifestations, most commonly presenting as meningoencephalitis, meningoencephalomyelitis, or myelitis, with meningoencephalitis being the most prevalent clinical syndrome observed in this study. Commonly reported symptoms include headache, fever, abnormal movements, and psychiatric symptoms. The disease exhibits diverse neurological and imaging features. Regional differences suggest that genetics, diagnostic practices, and cultural factors may influence symptom profiles. These findings highlight the need for standardized diagnostic criteria and further research across diverse populations.
目的分析GFAP星形细胞病的临床、实验室和影像学特征,并分析亚洲和非亚洲人群的差异。方法我们检索PubMed、Embase和Scopus从成立到2025年6月的病例报告、病例系列和涉及成年GFAP星形细胞病患者的观察性研究。我们分析了15项研究,包括538名患者。临床表现、MRI表现和实验室结果的数据被提取出来并在不同地区进行比较。结果共纳入2272项研究,其中15项纳入最终分析,共纳入538例患者。我们的分析确定了三种主要的临床表型:脑膜脑炎(164/ 371,44.2%)、脑膜脑脊髓炎(94/ 371,25.3%)和脊髓炎(26/ 371,7.0%)。最常见的临床症状为头痛(186/470,39.6%)、运动异常(160/473,33.8%)、发热(172/538,32.0%)和精神症状(111/471,23.6%)。MRI常显示脑室周围(85/434,19.6%)和皮层下(80/432,18.5%)病变。血管周围和脑膜增强是最常见的影像学表现。实验室结果显示血清和脑脊液中GFAP抗体阳性。在CSF和血清中也观察到其他抗体共存,最显著的是抗aqp4、抗mog和抗nmdar。结论fap星形细胞病具有广泛的临床和影像学表现,最常见的表现为脑膜脑炎、脑膜脑脊髓炎或脊髓炎,其中脑膜脑炎是本研究中观察到的最常见的临床综合征。通常报告的症状包括头痛、发烧、异常运动和精神症状。本病表现出多种神经学和影像学特征。地区差异表明遗传、诊断方法和文化因素可能影响症状特征。这些发现强调了标准化诊断标准和在不同人群中进一步研究的必要性。
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引用次数: 0
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Multiple sclerosis and related disorders
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