Background:Guillain-Barré syndrome (GBS) is an acute autoimmune neuropathy with limited effective treatments. Efgartigimod, a neonatal Fc receptor (FcRn) antagonist, may offer a novel treatment by reducing pathogenic immunoglobulin G. Objectives:To evaluate the efficacy, safety, and immunological effects of efgartigimod in patients with GBS. Design:A retrospective, observational, monocentric study. Methods:The study included 36 patients with GBS who received efgartigimod treatment. Efficacy was evaluated using the GBS-Disability Scale (GBS-DS) and Medical Research Council (MRC) scores over 8 weeks. Additionally, exploratory lymphocyte subset analysis was conducted on 13 patients before and after treatment. Results:Patients were stratified into those receiving efgartigimod as first-line therapy (n = 17) and as escalation therapy after intravenous immunoglobulin or plasma exchange (n = 19). Both cohorts showed significant and sustained clinical improvement over 8 weeks (p < 0.05). More importantly, the first-line cohort demonstrated a more rapid initial response, with a significantly greater reduction in GBS-DS score (ΔGBS-DS: 2.0 vs 0.0 at week 1, p = 0.003) and increase in MRC score (ΔMRC: 9.0 vs 6.0, p = 0.016) compared to the escalation cohort. This led to a higher percentage of achieving a favorable outcome (GBS-DS ⩽2) and a shorter median time to this outcome (6 vs 25 days, p < 0.001) in the first-line cohort. Regression analysis within the entire cohort confirmed that greater baseline severity and need for mechanical ventilation were associated with a longer time to favorable outcome. Exploratory analysis suggested a decrease in the proportions of CD3+CD4+ and CD4+HLA-DR+ T cells following efgartigimod treatment. Only two mild adverse events were reported. Conclusion:Efgartigimod shows efficacy as both a rapid-onset first-line therapy and a rescue option for refractory GBS, with a favorable safety profile and potential immunomodulatory effects, supporting further prospective evaluation.

[This corrects the article DOI: 10.1016/j.lanwpc.2023.100846.].

Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) caused by pathogenic immunoglobulin G antibodies to myelin oligodendrocyte glycoprotein is a rare demyelinating disease of the central nerve system (CNS). The clinical phenotypes of MOGAD include acute disseminated encephalomyelitis, optic neuritis, and transverse myelitis. At present, the mechanism underlying the disease is unknown. Herein, we report two cases of MOGAD accompanied by Epstein-Barr virus (EBV) infection. Both presented inflammation response in the cerebrospinal fluid (CSF), demonstrating elevated level of cell accounts and protein. EBV genomic sequence was also detected in the CSF samples. The patients recovered substantially after 3 months following a combination treatment with methylprednisolone and immunosuppressive therapy with ofatumumab. These cases provide new insight into the production of MOG-IgG and the possible pathological mechanisms underlying MOGAD. The cases also confirm the association with EBV, a virus that infects human B cells and has been proposed to be a trigger for MOGAD. This is the first report on subcutaneous ofatumumab treatment for MOGAD triggered by EBV, suggesting that this is a potentially valuable therapeutic option.

The acute-remitting course (the time interval from remission to the next relapse) is a distinct signature of neuromyelitis optica spectrum disorder (NMOSD), which harms patients and perplexes physicians. However, clinically actionable biomarkers to anticipate NMOSD relapse timing are unavailable. Recently, it is found that Cuproptosis-related genes would have an impact on the acute-remitting course in patients with aquaporin-4 (AQP4)-immunoglobulin G antibody positive NMOSD. A machine learning-based algorithm is developed to construct a consensus Cuproptosis-related prognosis risk (CRPR) model, followed by training a proportional hazards model to divide patients with NMOSD into high/low relapse risk groups. The CRPR is related to the clinical symptoms of patients with NMOSD. Finally, a nomogram is created using CRPR to predict the possibility of relapse in new cases of NMOSD. This prediction model can assist physicians and help develop appropriate interventions to prevent disease relapse.

Based on its favourable benefit–risk profile in clinical trials, siponimod was approved in China in 2020 for relapsing forms of multiple sclerosis (RMS). We evaluated its real-world (RW) effectiveness and safety profile in patients with RMS in China. This was a retrospective chart review of adults (≥ 18 years) with RMS receiving their first siponimod prescription (index date) between 01 August 2020 and 01 August 2022 at two hospitals in China. Inclusion criteria were ≥ 3 months of siponimod treatment and ≥ 1 clinical visit after the index date. Annualised relapse rate (ARR), proportion of relapse-free patients, proportion of patients free of MRI activity, and incidence of treatment-emergent adverse events (TEAEs) were evaluated. Of 113 included patients (mean age, 37.2 years; 64.6

Ofatumumab is a fully human anti-CD20 monoclonal antibody that selectively and highly depletes B cells. However, limited data on ofatumumab treatment are available in patients with neuromyelitis optica spectrum disorders (NMOSD). In this study, we aimed to evaluate the efficacy and safety of subcutaneous ofatumumab in patients with NMOSD. We conducted a retrospective multicenter cohort study of patients with NMOSD who received ofatumumab treatment at 15 tertiary hospitals in China. The primary outcome was the annualized relapse rate (ARR). The secondary outcomes included disability measures (Expanded Disability Status Scale score, EDSS; the Aminoff–Logue Disability Scale, ALS), changes in aquaporin-4 IgG (AQP4-IgG) titers, and safety profiles during ofatumumab treatment. A total of 112 patients (88

IntroductionTemporal lobe epilepsy (TLE) is a significant clinical phenotype of anti-glutamic acid decarboxylase (GAD)-associated disease, which is characterized by disturbances in GABAergic inhibitory neurotransmission. Efgartigimod, a neonatal crystallizable fragment receptor antagonist, controls the trafficking and recycling of pathogenic anti-GAD immunoglobulin G, showing promise as a therapeutic target.MethodsWe present a case report involving the treatment of three patients affected by GAD-seropositive autoimmune encephalitis with efgartigimod. The patients’ overall disability was assessed using the modified Rankin scale.ResultsAfter 4 weeks of efgartigimod treatment, the patients demonstrated substantial improvements, including no dementia or behavioral abnormalities and well-controlled seizures.DiscussionOur findings suggest that efgartigimod is a potential candidate drug for the treatment of anti-GADassociated autoimmune encephalitis, particularly in patients presenting with TLE.

Background Aquaporin-4 immunoglobulin G antibody (AQP4-IgG) product by B cells is essential in Neuromyelitis optica (NMO). However, some patients with immunosuppressive drugs persistently high AQP4-IgG titers, possibly owing to Follicular helper T (Tfh) cells can assist B cells in antibody production. Roquin-1 has been linked to the regulation of immune balance and plays an important role in the peripheral homeostasis of T cells. However, whether Roquin-1 can target Tfh cells differentiation in NMO mechanism remain unclear. Hence, in this study, we aim to explore the relationship between Roquin-1 and clinical characteristic in NMO, and whether Roquin-1 can target AMPK to regulates the CXCR5 + PD-1 + Tfh differentiation aggravate NMO progression. Methods We enrolled 71 NMO patients in this study, Clinical characteristics, MRI lesion counts in the spinal cord or brain, Expanded Disability Status Scale (EDSS) scores, Roquin-1 expression levels, and the proportion of Tfh cells were recorded and analyzed in each group using cell flow assay and other studies. Then, to validate Roquin-1 ability, we knockout or overexpression the Roquin-1 along Tfh cell differentiation. Results In the acute phase, Roquin-1 mRNA expression was reduced, while in remission phase, it was increased compared to healthy controls. The proportion of CXCR5 + PD-1 + Tfh cells was higher in NMO patients than in controls, and there was a negative correlation between Tfh cells proportions and Roquin-1 expression. Roquin-1 expression was negatively correlated with the EDSS score and positive correlation between the percentage of Tfh cells and the EDSS score and MRI lesions. We found that Roquin-1 could affect Tfh cell function and ratio during Tfh cell differentiation, promoted the expression of glycolysis-related proteins by influencing the interaction between AMPK and mTOR, and improved the antibody secretion ability of B cells. Conclusions Our study elucidated the effect of Roquin-1 on Tfh cells in NMO and the corresponding protective mechanism in autoimmunity, explore the possible causes of immune imbalance in NMO mechanisms, thus aiming to provide novel insights into NMO pathogenesis.

Multiple sclerosis (MS) was defined as a rare disease in China due to its low prevalence. For a long time, interferon β was the only approved disease-modifying therapy (DMT). Since the first oral DMT was approved in 2018, DMT approval accelerated, and seven DMTs were approved within 5 years. With an increasing number of DMTs being prescribed in clinical practice, it is necessary to discuss the standardized MS treatment algorithms depending on the disease activity and DMT availability. In this review paper, more than 20 Chinese experts in MS have reviewed the therapeutic progress of MS in China and worldwide and discussed algorithms for treating relapsing MS (RMS) based on the available DMTs in China, providing insights for establishing the standardized RMS treatment algorithms in this country.

The T-helper 17 (Th17) cell and regulatory T cell (Treg) axis plays a crucial role in the development of multiple sclerosis (MS), which is regarded as an immune imbalance between pro-inflammatory cytokines and the maintenance of immune tolerance. Mesenchymal stem cell (MSC)-mediated therapies have received increasing attention in MS research. In MS and its animal model experimental autoimmune encephalomyelitis, MSC injection was shown to alter the differentiation of CD4+T cells. This alteration occurred by inducing anergy and reduction in the number of Th17 cells, stimulating the polarization of antigen-specific Treg to reverse the imbalance of the Th17/Treg axis, reducing the inflammatory cascade response and demyelination, and restoring an overall state of immune tolerance. In this review, we summarize the mechanisms by which MSCs regulate the balance between Th17 cells and Tregs, including extracellular vesicles, mitochondrial transfer, metabolic reprogramming, and autophagy. We aimed to identify new targets for MS treatment using cellular therapy by analyzing MSC-mediated Th17-to-Treg polarization.

目的 分析伴癫痫发作的髓鞘少突胶质细胞糖蛋白(MOG)抗体相关疾病的临床特征.方法 回顾性分析郑州大学第一附属医院2019年1月至2022年3月诊治的25例伴癫痫发作的MOG抗体相关疾病患者的临床表现、实验室检查结果、影像学检查结果、治疗及随访情况.结果 25例患者中男18例,女7例.起病年龄3～56岁.急性期主要症状包括癫痫(25例)、体温升高或头痛(16例)、恶心和呕吐(8例)、意识障碍(6例)、视物模糊(8例)、肢体麻木无力(2例)等.癫痫发作的主要类型为全面强直痉挛发作.腰穿结果示13例患者颅内压增高,21例患者脑脊液白细胞升高.头部核磁共振显示21例患者有异常病灶,主要表现为脑皮层及白质异常信号,其中仅累及皮质下10例.脊髓出现异常病灶9例,视神经核磁共振异常8例.脑电图异常13例,主要表现为局灶性慢波.25例患者均接受激素或免疫球蛋白治疗,18例患者接受抗癫痫药物治疗.随访6～41个月后,12例患者复发,2例遗留明显症状.结论 本研究中伴癫痫发作的MOG抗体相关疾病患者以儿童、男性多见.除癫痫外,头痛、体温升高、脑脊液白细胞增高等非特异性临床表现常见,皮质脑炎可能是其特征性病变,早期给予激素或联合免疫球蛋白治疗效果好.

To investigate acute ischemic stroke (AIS) patients with systemic lupus erythematosus (SLE) clinical and imaging features, and to explore the impact of SLE on the short-term prognosis of AIS patients. A nested case–control study was conducted in The First Affiliated Hospital of Zhengzhou University between October 1, 2019, and May 31, 2021. The case group consisted of 28 AIS patients diagnosed with SLE, and 112 AIS patients without SLE were selected by incidence density sampling as a control group. Among 140 patients, the mean age was (48.4 ± 15.6) years, and 130 (92.9