The following is a summary of “Real-World experience with efgartigimod in patients with myasthenia gravis,” published in the March 2024 issue of Neurology by Fuchs et al.
Researchers conducted a retrospective study evaluating the effectiveness of efgartigimod, a human Fc-fragment blocking neonatal Fc receptor (FcRn) IgG1 antibody, in treating generalized (g) myasthenia gravis (MG).
They assessed the real-world clinical and safety impacts of efgartigimod in 22 patients diagnosed with gMG. The timing strategies for retreatment were also analyzed. In total, the participants underwent 59 cycles of efgartigimod treatment.
The results showed a median of 2 cycles (range 1–6). Improvement by ≥2 MG-ADL points after the first cycle was seen in 20 patients (86.3%). The median baseline MG-ADL score was 6.5 (range: 3–17), decreasing to 2.5 (range: 0–9) post-treatment (P<0.001). 18 patients consistently improved by ≥2 points per cycle. Treatment duration typically ranged from 4 to 12 weeks, showing two major clinical response patterns. Efgartigimod led to significant reductions in prednisone doses. Overall, it was safe, with minor adverse events; one fatality was attributed to severe respiratory failure. Efgartigimod demonstrated clinical efficacy, was a steroid-sparing agent, and was generally safe for gMG patients.
Investigators concluded that the most effective approach was personalized preventive treatment until patients with the condition reached a stable clinical state, followed by treatment discontinuation and periodic evaluations.
Source: link.springer.com/article/10.1007/s00415-024-12293-5