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Research Article: Real-world study of efgartigimod in AChR antibody-positive generalized myasthenia gravis: thymus status, multi-domain symptom improvement and steroid-sparing effect

Date Published: 2026-05-22

Abstract:
Efgartigimod has been approved for the treatment of gMG. However, real-world evidence regarding its efficacy across various functional domains in patients with differing thymic statuses, as well as its steroid-sparing effect when used in conjunction with conventional immunotherapy, remains limited. This single-center, real-world retrospective study included AChR antibody-positive patients with gMG who underwent at least two cycles of efgartigimod. The primary endpoints were the changes in MG-ADL and QMG scores at weeks 4 and 8. Secondary endpoints encompassed the MSE rate, IgG reduction, subgroup analyses stratified by thymic status and treatment regimens, and improvements in ocular, bulbar, limb, and trunk functional domains. Generalized estimating equations were employed for the analysis of longitudinal data. A total of 62 patients participated in the study, comprising 32 females and 30 males, with a mean age of 55 ± 14 years. Significant improvements in the MG-ADL (mean: 4.55 points) and QMG scores (mean: 7.52 points) were noted at week 4 (all P < 0.001), with these benefits sustained at week 8. After two treatment cycles, 77.4% (48/62) of patients experienced CMI. The MSE rate was significantly higher in the abnormal thymus group at week 4 (67.7% vs. 38.7%, P = 0.022), whereas no significant difference was observed between the two groups at weeks 8. Ocular symptoms, particularly ptosis, demonstrated the most substantial improvement, and significant enhancements were also recorded in bulbar and limb functions. Efficacy was comparable across the monotherapy, steroid combination, immunosuppressant combination, and triple therapy groups. Concomitant steroid treatment facilitated a maximum reduction in prednisone dosage of 78%. No adverse events were reported during the study. Efgartigimod rapidly, significantly, and sustainably alleviates symptoms in patients with AChR antibody-positive gMG, with its efficacy remaining unaffected by thymic status or concurrent immunotherapy. Patients with thymic abnormalities exhibit an earlier therapeutic response; however, long-term outcomes are comparable to those of patients with a normal thymus. The extent of improvement varies across functional domains, with the most pronounced benefits observed in ocular symptoms. Efgartigimod demonstrates independent therapeutic effects and facilitates substantial tapering of steroids when administered alongside glucocorticoids, underscoring its significant clinical value.

Introduction:
Efgartigimod has been approved for the treatment of gMG. However, real-world evidence regarding its efficacy across various functional domains in patients with differing thymic statuses, as well as its steroid-sparing effect when used in conjunction with conventional immunotherapy, remains limited.

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