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已发表论文
依库珠单抗治疗高活性乙酰胆碱受体阳性重症肌无力的有效性和安全性及其在不同亚型重症肌无力中的治疗反应
Authors Ma T, Liu Y, Zhu Y, Gao Y, Wang X, Liang Y, Wang B , Zhu R
Received 9 September 2025
Accepted for publication 25 December 2025
Published 8 January 2026 Volume 2026:15 559497
DOI https://doi.org/10.2147/ITT.S559497
Checked for plagiarism Yes
Review by Single anonymous peer review
Peer reviewer comments 2
Editor who approved publication: Professor Michael Shurin
Tianying Ma,1– 3,* Yanan Liu,4,* Ying Zhu,1– 3 Yan Gao,5 Xue Wang,6 Yong Liang,7 Benqiao Wang,1– 3 Ruixia Zhu1– 3
1Department of Neurology, The First Hospital, China Medical University, Shenyang, People’s Republic of China; 2Key Laboratory of Neurological Disease Big Data of Liaoning Province, Shenyang, People’s Republic of China; 3Shenyang Clinical Medical Research Center for Difficult and Serious Diseases of the Nervous System, Shenyang, People’s Republic of China; 4Department of Geriatrics, The second Hospital of Dalian Medical University, Dalian, People’s Republic of China; 5Department of Neurology, Shengjing Hospital of China Medical University, Shenyang, People’s Republic of China; 6Department of Neurology, Anshan Central Hospital, Anshan, People’s Republic of China; 7Department of Neurology, Tieling Central Hospital, Tieling, People’s Republic of China
*These authors contributed equally to this work
Correspondence: Ruixia Zhu, Email zrx_200626313@163.com
Background: Eculizumab had been approved for anti-acetylcholine receptor antibody-positive (AChR+) refractory myasthenia gravis (MG), but its effect on the broader group of highly active generalized myasthenia gravis (gMG) in clinical practice has not been fully uncovered.
Methods: We conduct a retrospective multi-center cohort study with prospectively collected data to explore the effectiveness and safety of eculizumab in highly active patients in clinical practice.
Results: Thirty-three patients from 4 research centers were included in our study treated with eculizumab. This study contains 1 early onset MG (EOMG), 13 late-onset MG (LOMG), and 19 thymoma-associated MG (TAMG) patients. At week 12, the mean Myasthenia Gravis Activities of Daily Living (MG-ADL) score decreased by 7.97 ± 3.96 points and Quantitative Myasthenia Gravis (QMG) score decreased by 12.88± 5.82 points,13 (40%) patients achieved minimal symptom expression (MSE) and 78.7% patients were ADL responders (the improvement of MG-ADL score ≥ 3 points and lasted for at least 4weeks). The improvement was sustained for 24 weeks. Overall, four patients had experienced myasthenic crisis (MC), and the time to wean from mechanical ventilation (MV) was 6.33± 3.21 days. In muscle subdomains analysis, QMG score decreased more obviously in the ocular group (66%) and bulbar group (49%) than in other muscle groups at week 4. Moreover, subgroup analysis showed that the TAMG and LOMG groups exhibited a similar degree of therapeutic effect in symptom control and ADL response. The mean prednisone dose was reduced by 12.84± 20.36 mg/day at week 24. Patients in our study showed well tolerance to eculizumab, and none of them experienced meningococcal infections.
Conclusion: Our study confirmed the safety and effectiveness of eculizumab in highly active gMG patients with reducing steroid dose. TAMG and MC may be suitable indications for treatment with eculizumab. All the muscle groups achieved improvements, especially for the extraocular muscles.
Keywords: eculizumab, highly active generalized myasthenia gravis, thymoma, safety, effectiveness
1Department of Neurology, The First Hospital, China Medical University, Shenyang, People’s Republic of China; 2Key Laboratory of Neurological Disease Big Data of Liaoning Province, Shenyang, People’s Republic of China; 3Shenyang Clinical Medical Research Center for Difficult and Serious Diseases of the Nervous System, Shenyang, People’s Republic of China; 4Department of Geriatrics, The second Hospital of Dalian Medical University, Dalian, People’s Republic of China; 5Department of Neurology, Shengjing Hospital of China Medical University, Shenyang, People’s Republic of China; 6Department of Neurology, Anshan Central Hospital, Anshan, People’s Republic of China; 7Department of Neurology, Tieling Central Hospital, Tieling, People’s Republic of China
*These authors contributed equally to this work
Correspondence: Ruixia Zhu, Email zrx_200626313@163.com
Background: Eculizumab had been approved for anti-acetylcholine receptor antibody-positive (AChR+) refractory myasthenia gravis (MG), but its effect on the broader group of highly active generalized myasthenia gravis (gMG) in clinical practice has not been fully uncovered.
Methods: We conduct a retrospective multi-center cohort study with prospectively collected data to explore the effectiveness and safety of eculizumab in highly active patients in clinical practice.
Results: Thirty-three patients from 4 research centers were included in our study treated with eculizumab. This study contains 1 early onset MG (EOMG), 13 late-onset MG (LOMG), and 19 thymoma-associated MG (TAMG) patients. At week 12, the mean Myasthenia Gravis Activities of Daily Living (MG-ADL) score decreased by 7.97 ± 3.96 points and Quantitative Myasthenia Gravis (QMG) score decreased by 12.88± 5.82 points,13 (40%) patients achieved minimal symptom expression (MSE) and 78.7% patients were ADL responders (the improvement of MG-ADL score ≥ 3 points and lasted for at least 4weeks). The improvement was sustained for 24 weeks. Overall, four patients had experienced myasthenic crisis (MC), and the time to wean from mechanical ventilation (MV) was 6.33± 3.21 days. In muscle subdomains analysis, QMG score decreased more obviously in the ocular group (66%) and bulbar group (49%) than in other muscle groups at week 4. Moreover, subgroup analysis showed that the TAMG and LOMG groups exhibited a similar degree of therapeutic effect in symptom control and ADL response. The mean prednisone dose was reduced by 12.84± 20.36 mg/day at week 24. Patients in our study showed well tolerance to eculizumab, and none of them experienced meningococcal infections.
Conclusion: Our study confirmed the safety and effectiveness of eculizumab in highly active gMG patients with reducing steroid dose. TAMG and MC may be suitable indications for treatment with eculizumab. All the muscle groups achieved improvements, especially for the extraocular muscles.
Keywords: eculizumab, highly active generalized myasthenia gravis, thymoma, safety, effectiveness