Deng Yufei, Luo Haocheng, Zhang Chaoyue, Yang Li, Wang Shuangshuang, Zhang Xuxiang, Yan Xianni, Yang Xiaojun, Jiang Qilong
Department of Neuromuscular Diseases, The First Affiliated Hospital of Guangzhou University of Chinese Medicine, Guangzhou, China.
Front Immunol. 2025 Jul 31;16:1596283. doi: 10.3389/fimmu.2025.1596283. eCollection 2025.
Highly active myasthenia gravis refers to a subset of refractory patients who exhibit recurrent exacerbations and crises. Eculizumab, a complement C5 inhibitor, has shown its efficacy and safety for patients with anti-acetylcholine receptor antibody-positive(AchR +)refractory generalized myasthenia gravis(gMG) in the REGAIN trial. However, the efficacy and safety of eculizumab in treating MG patients with severe infections have not yet been supported by clinical evidence. This is a case series reporting four patients with highly active myasthenia gravis complicated by severe infections. Changes in Myasthenia Gravis-Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores were recorded before and after 12 injections of eculizumab to assess efficacy. Pathogen characteristics of infections were summarized using bacterial culture and next-generation sequencing (NGS) results, presented as a heatmap to illustrate pathogen species and abundance. Inflammatory markers, including Procalcitonin (PCT), C-Reactive Protein (CRP), neutrophil count, and total lymphocyte count, were monitored to evaluate the safety. Treatment regimens were retrospectively analyzed to further assess clinical outcomes and safety. The baseline ADL data for the four patients was 22 ± 2.31 (Mean ± SD), and the baseline QMG data was 30.5 ± 8.23. After 12 injections of eculizumab treatment, the scores decreased to ADL 4.75 ± 3.3 and QMG 14 ± 3.37. During the treatment, no apparent worsening of infections related to Eculizumab was noted. Three patients successfully had their tracheostomy tubes removed, and none of the four patients experienced further myasthenic crises. Eculizumab demonstrated clinical improvement in this series, and the treatment was well-tolerated. This case series addresses the need for data on complement inhibitors in highly active myasthenia gravis patients with severe infections, provides clinical reference support for the expanded application of eculizumab.
重症肌无力高度活跃型是指难治性患者的一个亚组，这些患者会反复出现病情加重和危象。依库珠单抗是一种补体C5抑制剂，在REGAIN试验中已显示出对乙酰胆碱受体抗体阳性（AchR+）难治性全身型重症肌无力（gMG）患者的疗效和安全性。然而，依库珠单抗治疗合并严重感染的重症肌无力患者的疗效和安全性尚未得到临床证据的支持。这是一篇病例系列报告，报道了4例合并严重感染的重症肌无力高度活跃型患者。记录了依库珠单抗12次注射前后重症肌无力日常生活活动（MG-ADL）和重症肌无力定量（QMG）评分的变化，以评估疗效。利用细菌培养和二代测序（NGS）结果总结感染的病原体特征，并以热图形式呈现，以说明病原体种类和丰度。监测包括降钙素原（PCT）、C反应蛋白（CRP）、中性粒细胞计数和总淋巴细胞计数在内的炎症标志物，以评估安全性。对治疗方案进行回顾性分析，以进一步评估临床结局和安全性。4例患者的基线ADL数据为22±2.31（均值±标准差），基线QMG数据为30.5±8.23。依库珠单抗治疗12次后，评分降至ADL 4.75±3.3和QMG 14±3.37。治疗期间，未观察到与依库珠单抗相关的感染明显恶化。3例患者成功拔除气管造口管，4例患者均未再次发生重症肌无力危象。依库珠单抗在本系列中显示出临床改善，且治疗耐受性良好。该病例系列满足了严重感染的重症肌无力高度活跃型患者对补体抑制剂数据的需求，为依库珠单抗的扩大应用提供了临床参考支持。
