在即将发生的肌无力危象中,皮下施伐替莫第一线治疗:1例报告。

IF 4.7 2区 医学 Q1 CLINICAL NEUROLOGY Therapeutic Advances in Neurological Disorders Pub Date : 2024-12-23 eCollection Date: 2024-01-01 DOI:10.1177/17562864241307687
Isabelle Kwiedor, Martina Menacher, Monika Ellßel, Markus Naumann, Antonios Bayas
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引用次数: 0

摘要

在乙酰胆碱受体(AChR)抗体阳性的广泛性重症肌无力(gMG)中,新生儿fc受体(FcRn)抑制拓宽了治疗范围。重症肌无力危象(MC),预示着即将来临的重症肌无力危象(iMC),是一种需要快速发作和持续疗效治疗的危重疾病。目前用于iMC的治疗方法,包括静脉注射免疫球蛋白和血浆交换/免疫吸附,具有局限性,如延迟起效和潜在的副作用。到目前为止,关于使用FcRn抑制剂治疗即将发生或明显的MC (mMC)的数据有限。在这里,我们报告了一例AChR抗体阳性的gMG伴iMC,皮下给药FcRn抑制剂efgartigimod导致快速临床缓解。在给药24小时内,患者呼吸和球肌功能明显改善,防止进展为MC和需要机械通气。这种快速反应伴随着4周内AChR抗体水平显著降低89.8%。该病例支持了efgartigimod作为治疗iMC的快速有效治疗选择的潜力,为现有治疗提供了一种替代方案。
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First line treatment with subcutaneous efgartigimod in impending myasthenic crisis: a case report.

In acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG), neonatal Fc-receptor (FcRn) inhibition has broadened the therapeutic spectrum. Myasthenic crisis (MC), heralded by an impending myasthenic crisis (iMC), is a critical condition requiring treatments with rapid onset and sustained efficacy. Currently treatments used for iMC, including intravenous immunoglobulins and plasma exchange/immunoadsorption, have limitations, such as delayed onset of action and potential side effects. So far, there is limited data on the use of FcRn inhibitors in the management of impending or manifest MC (mMC). Here, we present a case of AChR antibody-positive gMG with iMC, where subcutaneous administration of the FcRn inhibitor efgartigimod resulted in rapid clinical remission. Within 24 h of administration, the patient exhibited significant improvement in respiratory and bulbar muscle function, preventing progression to manifest MC and the need for mechanical ventilation. This rapid response was accompanied by a marked reduction in AChR antibody level by 89.8% within 4 weeks. This case supports the potential of efgartigimod as a fast-acting and effective treatment option for managing iMC, offering an alternative to existing therapies.

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来源期刊
CiteScore
8.30
自引率
1.70%
发文量
62
审稿时长
15 weeks
期刊介绍: Therapeutic Advances in Neurological Disorders is a peer-reviewed, open access journal delivering the highest quality articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of neurology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in neurology, providing a forum in print and online for publishing the highest quality articles in this area.
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