Safety and Efficacy of a Neonatal Fc Receptor Antagonist in Patients With Anti-NMDAR Encephalitis

IF 5 1区医学 Q1 NEUROSCIENCES

CNS Neuroscience & Therapeutics Pub Date : 2025-08-11 DOI:10.1111/cns.70534

Zhihong Bian, Han Cai, Haotian Wu, Ping Liu, Yanfang Zuo, Fuhua Peng, Wei Qiu, Zhengqi Lu, Bingjun Zhang

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Abstract

Background

This retrospective study investigates the safety and effectiveness of efgartigimod in improving clinical outcomes for patients with anti-NMDAR encephalitis in a real-world setting.

Methods

We analyzed data from 26 patients diagnosed with anti-NMDAR encephalitis at the Third Affiliated Hospital of Sun Yat-sen University between October 2022 and June 2024. The patients were divided into two groups: 13 received efgartigimod treatment, while 13 did not. Clinical outcomes were assessed using the modified Rankin Scale (mRS), the Clinical Assessment Scale in Autoimmune Encephalitis (CASE), and clinical symptoms, along with an analysis of treatment-emergent adverse events (TEAEs), cerebrospinal fluid (CSF) antibody titers, and blood serum IgG levels.

Results

Efgartigimod treatment was associated with significant clinical improvement, as indicated by greater reductions in mRS and CASE scores at discharge and follow-up compared to the control group. At follow-up, 84.6% of patients in the efgartigimod group achieved an mRS score of ≤ 2, compared to 46.2% in the control group. Additionally, patients receiving Efgartigimod exhibited a notable reduction in CSF anti-NMDAR antibody titers and serum IgG levels. The most common TEAEs were mild to moderate infections, with no significant safety concerns identified.

Conclusion

In this exploratory study, efgartigimod demonstrated a favorable safety profile for patients with anti-NMDAR encephalitis and appeared to facilitate the recovery of clinical symptoms and neurological function. However, further prospective randomized studies with larger patient cohorts are necessary to confirm the safety and efficacy of efgartigimod.

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新生儿Fc受体拮抗剂在抗nmdar脑炎患者中的安全性和有效性

本回顾性研究探讨了在现实世界中，艾夫加替莫改善抗nmdar脑炎患者临床结局的安全性和有效性。方法分析2022年10月至2024年6月中山大学第三附属医院诊断为抗nmdar脑炎的26例患者的资料。患者分为两组：13例接受艾加替莫德治疗，13例未接受治疗。临床结果采用改进的Rankin量表（mRS）、自身免疫性脑炎临床评估量表（CASE）和临床症状进行评估，同时分析治疗后出现的不良事件（teae）、脑脊液（CSF）抗体滴度和血清IgG水平。结果：与对照组相比，Efgartigimod治疗与显著的临床改善相关，出院和随访时mRS和CASE评分下降幅度更大。随访时，efgartigimod组84.6%的患者mRS评分≤2，而对照组为46.2%。此外，接受Efgartigimod治疗的患者脑脊液抗nmdar抗体滴度和血清IgG水平显著降低。最常见的teae是轻度至中度感染，没有发现明显的安全问题。结论在这项探索性研究中，依加替莫德对抗nmdar脑炎患者具有良好的安全性，并可促进临床症状和神经功能的恢复。然而，进一步的前瞻性随机研究需要更大的患者队列来证实埃加替莫德的安全性和有效性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

CNS Neuroscience & Therapeutics 医学-神经科学

CiteScore

7.30

自引率

12.70%

发文量

240

审稿时长

2 months

期刊介绍： CNS Neuroscience & Therapeutics provides a medium for rapid publication of original clinical, experimental, and translational research papers, timely reviews and reports of novel findings of therapeutic relevance to the central nervous system, as well as papers related to clinical pharmacology, drug development and novel methodologies for drug evaluation. The journal focuses on neurological and psychiatric diseases such as stroke, Parkinson’s disease, Alzheimer’s disease, depression, schizophrenia, epilepsy, and drug abuse.