Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, China.
Department of Neurology, The First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.
Front Immunol. 2024 Jul 29;15:1418503. doi: 10.3389/fimmu.2024.1418503. eCollection 2024.
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.
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.
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, =0.0313, and 34.98 ± 6.47 vs. 43.68 ± 6.54, =0.0313, respectively).
These findings validated the rapid action of efgartigimod in facilitating the weaning process with a good safety profile in patients with MC.
依酚氯胺在乙酰胆碱受体 (AChR) 抗体阳性的全身性重症肌无力 (MG) 患者中有效且耐受性良好。然而，依酚氯胺在肌无力危象 (MC) 中的治疗潜力和安全性仍知之甚少。
这是一项观察性、前瞻性、多中心、真实世界的研究，共纳入 2 例接受依酚氯胺作为一线抢救治疗的 MC 患者和 8 例接受依酚氯胺作为附加治疗的患者。收集患者的基线人口统计学特征和免疫治疗情况，并在接受依酚氯胺治疗的 8 周内每周评估重症肌无力日常生活活动量表 (MG-ADL) 评分。此外，在一个治疗周期前后分别测量血清 IgG 和抗 AChR 抗体水平以及外周血 CD4 T 淋巴细胞。
共纳入 10 例 MC 患者，其中 9 例为抗 AChR 抗体阳性，1 例为抗肌肉特异性激酶 (MuSK) 阳性。所有患者在接受依酚氯胺治疗后均成功脱机，平均脱机时间为 10.44 ± 4.30 天。一个周期的输注后，MG-ADL 评分从基线时的 15.6 ± 4.4 分降至 3.4 ± 2.2 分，而皮质类固醇剂量从 55.0 ± 20.7 mg 减少至 26.0 ± 14.1 mg。依酚氯胺治疗后调节性 T 细胞和幼稚 T 细胞的比例（%在 CD4 T 细胞中）显著降低（5.48 ± 1.23 比 6.90 ± 1.80，=0.0313，34.98 ± 6.47 比 43.68 ± 6.54，=0.0313）。
这些发现验证了依酚氯胺在促进 MC 患者脱机过程中的快速作用，且具有良好的安全性。
