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病例报告:艾加莫德是治疗眼肌型重症肌无力的一种新的治疗选择:2例报告

Case report: Efgartigimod is a novel therapeutic option for ocular myasthenia gravis: a report of 2 cases.

作者信息

Ma Tianying, Zhu Ying, Zhu Ruixia

机构信息

Department of Neurology, The First Affiliated Hospital of China Medical University, Shenyang, China.

出版信息

Front Immunol. 2025 Jan 13;15:1497398. doi: 10.3389/fimmu.2024.1497398. eCollection 2024.

DOI:10.3389/fimmu.2024.1497398
PMID:39872535
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11769809/
Abstract

INTRODUCTION

Efgartigimod has been approved for the treatment of acetylcholine receptor antibodies-positive generalized myasthenia gravis (AChR-Ab+gMG), but its efficacy in patients with ocular myasthenia gravis (OMG) is not known.

CASE PRESENTATION

We describe 2 cases of patients with AChR-Ab+ OMG who showed unfavorable responses to corticosteroids and tacrolimus. Within 2 weeks of initiating efgartigimod, both patients showed rapid improvement and minimal symptom expression was achieved in weeks 3 to 4, which was maintained up to week 12.

CONCLUSION

The 2 cases described herein provide preliminary evidence for the effectiveness of efgartigimod for the treatment of OMG for patients who do not respond or are intolerant to conventional medications. Large-scale studies are needed to confirm these findings.

摘要

引言

艾加莫德已被批准用于治疗乙酰胆碱受体抗体阳性的全身型重症肌无力(AChR-Ab+gMG),但其对眼肌型重症肌无力(OMG)患者的疗效尚不清楚。

病例报告

我们描述了2例AChR-Ab+OMG患者,他们对皮质类固醇和他克莫司反应不佳。在开始使用艾加莫德的2周内,两名患者均显示出快速改善,在第3至4周症状表达降至最低,并维持到第12周。

结论

本文所述的2例病例为艾加莫德治疗对传统药物无反应或不耐受的OMG患者的有效性提供了初步证据。需要进行大规模研究来证实这些发现。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b91f/11769809/2998652ef921/fimmu-15-1497398-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b91f/11769809/e128c7619e50/fimmu-15-1497398-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b91f/11769809/2998652ef921/fimmu-15-1497398-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b91f/11769809/e128c7619e50/fimmu-15-1497398-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b91f/11769809/2998652ef921/fimmu-15-1497398-g002.jpg

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本文引用的文献

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Effect of Immunosuppression in Risk of Developing Generalized Symptoms in Ocular Myasthenia Gravis: A Retrospective Cohort Study.免疫抑制对眼肌型重症肌无力发生全身症状风险的影响:一项回顾性队列研究。
Neurology. 2024 Aug 27;103(4):e209722. doi: 10.1212/WNL.0000000000209722. Epub 2024 Jul 30.
2
Treating myasthenia gravis beyond the eye clinic.眼肌型重症肌无力的治疗不应局限于眼科。
Eye (Lond). 2024 Aug;38(12):2422-2436. doi: 10.1038/s41433-024-03133-x. Epub 2024 May 24.
3
Comparative effectiveness and safety of intravenous methylprednisolone and tacrolimus monotherapy in ocular myasthenia gravis with unsatisfactory prednisone responses: a retrospective study.
静脉注射甲泼尼龙和他克莫司单药治疗泼尼松反应不佳的眼肌型重症肌无力的疗效和安全性比较:一项回顾性研究。
Orphanet J Rare Dis. 2024 Jan 19;19(1):19. doi: 10.1186/s13023-024-03025-z.
4
Dynamic nomogram for predicting generalized conversion in adult-onset ocular myasthenia gravis.用于预测成人眼肌型重症肌无力广义转化的动态列线图。
Neurol Sci. 2023 Apr;44(4):1383-1391. doi: 10.1007/s10072-022-06519-5. Epub 2022 Dec 5.
5
Clinical profile and impact of comorbidities in patients with very-late-onset myasthenia gravis.非常晚发性重症肌无力患者的临床特征和合并症的影响。
Muscle Nerve. 2021 Oct;64(4):462-466. doi: 10.1002/mus.27369. Epub 2021 Jul 21.
6
Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial.依氟鸟氨酸苷单抗在全身性重症肌无力患者中的安全性、疗效和耐受性(ADAPT):一项多中心、随机、安慰剂对照、3 期临床试验。
Lancet Neurol. 2021 Jul;20(7):526-536. doi: 10.1016/S1474-4422(21)00159-9.
7
International consensus guidance for management of myasthenia gravis: Executive summary.重症肌无力管理的国际共识指南:执行摘要。
Neurology. 2016 Jul 26;87(4):419-25. doi: 10.1212/WNL.0000000000002790. Epub 2016 Jun 29.