Efgartigimod improves non-AChR generalized Myasthenia Gravis: a real world experience.

IF 2.7 4区医学 Q2 CLINICAL NEUROLOGY

Neurological Sciences Pub Date : 2025-03-25 DOI:10.1007/s10072-025-08096-9

Carlo Antozzi, Rita Frangiamore, Elena Rinaldi, Fiammetta Vanoli, Francesca Andreetta, Eleonora Giacopuzzi Grigoli, Emilio Ciusani, Silvia Bonanno, Lorenzo Maggi, Renato Mantegazza

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引用次数: 0

Abstract

Introduction: The neonatal Fc receptor (FcRn) inhibitor Efgartigimod (EFG) has been approved for treatment of generalized Myasthenia Gravis (gMG) with anti-AChR antibodies. Information on the effect of EFG in non-AChR MG is limited. We investigated the efficacy of EFG in non-AChR gMG along a clinical follow-up of 2 years.

Methods: We treated 13 patients with gMG without anti-AChR antibodies: 5 MuSK+, 2 LRP4 + and 6 triple-negative (confirmed by live CBA). EFG was administered according to the GENERATIVE protocol (consisting of a Fixed period of 2 treatment cycles of 4 infusions at weekly intervals, followed by a Flexible period during which EFG was given in case of initial worsening) starting from November 2021. Outcomes were evaluated by means of the MG-ADL, QMG, MGC and MGQoL15r scales.

Results: The mean follow-up was 21 ± 5.3 months. Meaningful improvement was observed with the clinical scores adopted. The number of cycles/year was 3.92 ± 0.9. The interval between cycles was 10.1 ± 3.6 weeks. MG-ADL improvement of at least 5 points was recorded in 58% of cycles. 46% of patients required hospitalization during the two years before treament with EFG and 70% plasmaexchange/IVIG; during EFG none of the patients was hospitalized or required immunomodulation. No major side effects or infusion related reactions occurred.

Conclusion: EFG was effective in non-AChR gMG and modified significantly the course of the disease. Our experience strengthens the role of FcRn inhibition as a new therapeutic tool for MG without anti-AChR antibodies.

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Efgartigimod改善非achr广泛性重症肌无力：一个真实的世界经验。

新生儿Fc受体（FcRn）抑制剂Efgartigimod （EFG）已被批准用于抗achr抗体治疗广泛性重症肌无力（gMG）。关于EFG在非achr MG中的作用的信息有限。我们通过2年的临床随访研究了EFG在非achr gMG中的疗效。方法：对13例无抗achr抗体的gMG患者进行治疗，其中MuSK+ 5例，LRP4 + 2例，三阴性6例（经活体CBA证实）。从2021年11月开始，根据GENERATIVE方案给予EFG（包括固定周期的2个治疗周期，每周注射4次，然后是灵活周期，在初始恶化的情况下给予EFG）。通过MG-ADL、QMG、MGC和MGQoL15r量表评估结果。结果：平均随访21±5.3个月。采用临床评分后观察到有意义的改善。周期数/年为3.92±0.9。周期间隔为10.1±3.6周。在58%的周期中，MG-ADL至少改善了5个点。46%的患者在接受EFG和70%血浆置换/IVIG治疗前两年内需要住院；在EFG期间，没有患者住院或需要免疫调节。未发生重大副作用或输液相关反应。结论：EFG对非achr型gMG有效，并能明显改善病程。我们的经验加强了FcRn抑制作为无抗achr抗体的MG的新治疗工具的作用。

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

Neurological Sciences 医学-临床神经学

CiteScore

6.10

自引率

3.00%

发文量

743

审稿时长

4 months

期刊介绍： Neurological Sciences is intended to provide a medium for the communication of results and ideas in the field of neuroscience. The journal welcomes contributions in both the basic and clinical aspects of the neurosciences. The official language of the journal is English. Reports are published in the form of original articles, short communications, editorials, reviews and letters to the editor. Original articles present the results of experimental or clinical studies in the neurosciences, while short communications are succinct reports permitting the rapid publication of novel results. Original contributions may be submitted for the special sections History of Neurology, Health Care and Neurological Digressions - a forum for cultural topics related to the neurosciences. The journal also publishes correspondence book reviews, meeting reports and announcements.