Efficacy of multi-cycle Efgartigimod in achieving minimal symptom expression in myasthenia gravis: A comparative multi-center study.

OBJECTIVES

Efgartigimod has been approved as an effective and safe treatment for myasthenia gravis (MG). However, real-world experience on multi-cycle efgartigimod treatment and its comparison with Standard of Care (SoC) remain limited. This study aimed to evaluate minimal symptom expression (MSE) as the treatment goal and compared the proportion and time to achieving it between two groups.

METHODS

Patients receiving multi-cycle efgartigimod and contemporaneous counterparts treated with SoC were included. The rate of MSE achievement were compared using Kaplan-Meier analysis and Cox regression. Subgroup analysis focused on efgartigimod group, observing involved muscles and oral medication for further insights.

RESULTS

A total of 76 and 124 MG patients were enrolled in the efgartigimod and SoC groups, respectively. Efgartigimod group demonstrated a higher rate (73.3 % vs. 22.6 %, p < 0.001) and shorter time [0.7 (0.5, 3.1) vs. 3.3 (3, 6.1), months, p < 0.001] to achieving MSE compared to SoC group. Kaplan-Meier analysis revealed efgartigimod group had a higher MSE probability with a median time of 2.27 (95 %CI, 0.70, 4.39) months. MG Patients had a 9.69 fold (95 %CI, 5.54, 16.92) greater chance of achieving MSE compared to SoC group, remaining significant at 9.44 fold (95 %CI, 5.36, 16.60) after adjusting for ADL scores. After treatment, respiratory and bulbar symptoms improved significantly, with average scores from 0.57 ± 0.87 to zero, and 2.62 ± 2.56 to 0.37 ± 0.96. Additionally, the daily dosage of corticosteroid dropped from 20(10, 25) mg to 10(10,20) mg, with only 7 (9.2 %) patients requiring over 20 mg/day.

CONCLUSION

Multi-cycle efgartigimod treatment achieves early MSE more effectively than SoC, serving as a fast-acting therapy for MG.