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Efgartigimod as a promising add-on therapy for myasthenic crisis: a prospective case series.
Song, Jie; Wang, Haiyan; Huan, Xiao; Jiang, Qilong; Wu, Zongtai; Yan, Chong; Xi, Jianying; Zhao, Chongbo; Feng, Huiyu; Luo, Sushan.
Afiliación
  • Song J; Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, China.
  • Wang H; Department of Neurology, The First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.
  • Huan X; Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, China.
  • Jiang Q; Department of Neurology, The First Affiliated Hospital of Guangzhou University of Chinese Medicine, Guangzhou, China.
  • Wu Z; Faculty of Biology, University of Cambridge, Cambridge, United Kingdom.
  • Yan C; Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, China.
  • Xi J; Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, China.
  • Zhao C; Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, China.
  • Feng H; Department of Neurology, The First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.
  • Luo S; Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, China.
Front Immunol ; 15: 1418503, 2024.
Article en En | MEDLINE | ID: mdl-39136012
ABSTRACT

Introduction:

Efgartigimod is effective and well-tolerated in patients with anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (MG). However, the therapeutic potential and the safety profile of efgartigimod in myasthenic crisis (MC) remained largely unknown.

Methods:

This is an observational, prospective, multicenter, real-world study to follow 2 MC patients who initiated efgartigimod as a first-line rescue therapy and 8 cases who used it as an add-on therapy. Baseline demographic features and immunotherapies were collected, and the MG-activities of daily living (MG-ADL) scale was evaluated every week since efgartigimod treatment for 8 weeks. Additionally, serum IgG and anti-AChR antibody levels and the peripheral CD4+ T lymphocytes were measured before and after one cycle of treatment.

Results:

Ten patients with MC were enrolled in the study, including 9 anti-AChR antibody positive and 1 anti-muscle-specific kinase (MuSK) positive. All patients were successfully weaned from the ventilation after receiving efgartigimod treatment, with a length of 10.44 ± 4.30 days. After one cycle of infusions, the MG-ADL score reduced from 15.6 ± 4.4 at the baseline to 3.4 ± 2.2, while the corticosteroid dose was tapered from 55.0 ± 20.7 mg to 26.0 ± 14.1 mg. The proportions of regulatory T cells and naïve T cells (% in CD4+ T) significantly decreased post-efgartigimod treatment (5.48 ± 1.23 vs. 6.90 ± 1.80, P=0.0313, and 34.98 ± 6.47 vs. 43.68 ± 6.54, P=0.0313, respectively).

Conclusion:

These findings validated the rapid action of efgartigimod in facilitating the weaning process with a good safety profile in patients with MC.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Miastenia Gravis Límite: Adult / Aged / Female / Humans / Male / Middle aged Idioma: En Revista: Front Immunol Año: 2024 Tipo del documento: Article País de afiliación: China Pais de publicación: Suiza
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Miastenia Gravis Límite: Adult / Aged / Female / Humans / Male / Middle aged Idioma: En Revista: Front Immunol Año: 2024 Tipo del documento: Article País de afiliación: China Pais de publicación: Suiza