Research Article: Real-world experience with efgartigimod in generalized myasthenia gravis: a single-center retrospective study in China
Abstract:
This study aimed to evaluate the real-world efficacy, safety, and application patterns of efgartigimod in Chinese patients with generalized myasthenia gravis (gMG), with a focus on individualized treatment strategies.
We conducted a single-center, retrospective analysis of 81 gMG patients who received at least one standard cycle of efgartigimod (10 mg/kg weekly for 4 weeks). Disease severity was assessed using Myasthenia Gravis Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG), and Myasthenia Gravis Composite (MGC) scores at baseline, week 2, and week 4. Retreatment and regimen modifications were individualized, which included exploration of a reduced-frequency regimen (2 infusions every 2 weeks) in selected patients.
In the overall cohort, 65 (80.25%) and 63 (77.78%) patients achieved MG-ADL (reduction ?2) and QMG (reduction ?3) responses, respectively, after the first cycle. 65 (80.25%) patients received only one cycle; among them, 41 (63.07%) maintained disease control with concomitant immunosuppressive therapies without requiring additional efgartigimod. The 16 patients receiving multiple cycles (?2) had higher baseline disease severity (higher Myasthenia Gravis Foundation of America class, MG-ADL, and QMG scores) and showed progressive improvement with flexible retreatment. In a selected subgroup of 9 patients (based on age, robust response, and significant IgG reduction), a reduced-frequency regimen (2 infusions every 2 weeks) maintained efficacy while reducing the infusion burden. Adverse events were mostly mild, with no new safety signals.
Efgartigimod demonstrated rapid and broad efficacy in a real-world gMG cohort. An “induction-maintenance” strategy, using efgartigimod for acute control and conventional immunosuppressants for long-term management, proved effective in most patients. A reduced-frequency regimen may be feasible in selected cases, highlighting the value of individualized treatment approaches in optimizing outcomes and reducing treatment burden.
Introduction:
Myasthenia gravis (MG) is an autoimmune disorder affecting the neuromuscular junction, primarily mediated by autoantibodies targeting postsynaptic membrane proteins ( 1 ). These include antibodies against the acetylcholine receptor (AChR-Ab, the most common, detected in >85% of patients), muscle-specific tyrosine kinase (MuSK-Ab), or lipoprotein-related protein 4 (LRP4-Ab). A smaller proportion of patients are seronegative for these antibodies ( 2 ). Efgartigimod, a human immunoglobulin G1 (IgG1) Fc fragment,…
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