AB029. Eculizumab as a new option for perioperative treatment in thymoma-associated myasthenia gravis: a prospective case series
Original Research

AB029. Eculizumab as a new option for perioperative treatment in thymoma-associated myasthenia gravis: a prospective case series

Hui Wu1, Xianglin Chu2, Huahua Zhong1, Liewen Pang2, Lei Jin1, Wenji Jia3, Ran Chen1, Zongtai Wu4, Ji Xiong5, Chongbo Zhao1, Jie Song1, Sushan Luo1

1Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, China; 2Department of Cardiothoracic Surgery, Huashan Hospital, Fudan University, Shanghai, China; 3Department of Neurology, No. 2 Affiliated Hospital, Kunming Medical University, Kunming, China; 4Faculty of Public Health and Primary Care, University of Cambridge, Cambridge, UK; 5Department of Pathology, Huashan Hospital, Fudan University, Shanghai, China

Correspondence to: Sushan Luo, MD, PhD. Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Wulumuqi Road, Shanghai, 200040, China. Email: luosushan@fudan.edu.cn.

Background: Eculizumab in patients with thymoma-associated myasthenia gravis (TAMG) for perioperative treatment have not been evaluated. The aim of this study is to explore the perioperative efficacy and safety of eculizumab in patients with TAMG.

Methods: This is a single-centre observational prospective study. Patients were stratified into two groups with conventional immunotherapies, including corticosteroid, with (n=10) or without eculizumab as an add-on (n=15). The clinical outcomes, surgical outcomes, MG-activities of daily living (ADL), Myasthenia Gravis Foundation of America-quantitative MG (QMG), Manual Muscle Test (MMT), were compared between groups. Immune profiling and adverse events were also assessed pre-thymectomy and post-thymectomy. The intercostal muscle was collected and stained for C5b-8/9 to evaluate the membrane attack complex (MAC) deposition.

Results: Both groups exhibited significant postoperative reductions in QMG, MMT and ADL scores compared to preoperative baseline levels. Eculizumab group presented earlier improvement for ΔQMG (median 8.5 vs. −1.0, P<0.001), ΔMMT (6.0 vs. −3.0, P=0.001), and ΔADL (5.0 vs. −3.5, P=0.001) at 1 week. The relative superiority in clinical scores was consistent through week 2. For eculizumab group, the percentages of CD3+CD4+T helper cells significantly declined from 41.17%±7.98% to 30.39%±8.12% (P=0.003), CD3+T lymphocytes significantly declined from 74.21%±8.91% to 67.82%±11.39% (P=0.045). In contrast, there were no significant changes in CD3+CD8+T and CD19+B lymphocytes. The retrospective analysis revealed an increase in the expression of C5b-8/9 in patients with TAMG, as compared with healthy controls (P=0.04).

Conclusions: This small case series highlights the use of eculizumab in TAMG as a rapid symptom-control treatment during the perioperative period. Future prospective cohort studies with a large sample size are expected to validate these findings, particularly for those TAMG with moderate to severe myasthenia before thymectomy.

Keywords: Thymoma; perioperative; eculizumab

Acknowledgments

We thank the patients for their participation in this study.

Footnote

Funding: This study is supported by financial grants from the National Natural Science Foundation of China (No. 82471426), the National Key Research and Development Plan (Nos. 2022YFC3501305, 2022YFC3501303), and Shanghai Hospital Development Center Program (No. SHDC2023CRD007).

Conflicts of Interest: All authors have completed the ICMJE uniform disclosure form (available at https://med.amegroups.com/article/view/10.21037/med-25-ab029/coif). C.Z. and S.L. received grants from National Key Research and Development Plan, The National Natural Science Foundation of China. The other authors have no conflicts of interest to declare.

Ethical Statement: The authors are accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved. The study was conducted in accordance with the Declaration of Helsinki and its subsequent amendments. The study was approved by the institutional Ethics Committee of Huashan Hospital (2020–999) and registered on clinical trial databases (ChiCTR2000037676, NCT04535843) and informed consent was obtained from all individual participants.

Open Access Statement: This is an Open Access article distributed in accordance with the Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International License (CC BY-NC-ND 4.0), which permits the non-commercial replication and distribution of the article with the strict proviso that no changes or edits are made and the original work is properly cited (including links to both the formal publication through the relevant DOI and the license). See: https://creativecommons.org/licenses/by-nc-nd/4.0/.

doi: 10.21037/med-25-ab029
Cite this abstract as: Wu H, Chu X, Zhong H, Pang L, Jin L, Jia W, Chen R, Wu Z, Xiong J, Zhao C, Song J, Luo S. AB029. Eculizumab as a new option for perioperative treatment in thymoma-associated myasthenia gravis: a prospective case series. Mediastinum 2025;9:AB029.

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