A multicenter study of patients with juvenile myasthenia gravis (MG) found that efgartigimod is a safe and effective treatment option, according to findings published in Muscle & Nerve.
The study, which retrospectively followed patients for a median of 23 months, reported improvements across several measures of disease severity.
Efgartigimod is an antibody fragment that binds to and blocks the neonatal Fc receptor, promoting the breakdown of immunoglobulin G. Currently, the therapy is approved for adults with generalized MG who are anti-acetylcholine receptor (AChR) antibody positive.
The authors analyzed data from 17 children with MG, 14 of whom were female, across 12 medical centers in China. The majority of patients tested positive for the anti-AChR antibody and had moderate to severe symptoms. Participants received anywhere from one to four doses of efgartigimod over the study period.
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Participants benefited from efgartigimod, regardless of disease duration, resistance to previous therapies and type of MG at onset (generalized or ocular).
After one week, 70.6% of patients experienced a clinically meaningful improvement, defined as a decrease of at least two points in MG-related Activities of Daily Living (MG-ADL) scores. Of the 12 remaining participants at Week 4, 91.7% reported a clinically meaningful improvement.
In addition, at Week 4 66.7% achieved minimal symptom expression, defined as an MG-ADL score of zero or one. Quantitative Myasthenia Gravis (QMG) scores also declined consistently over the observation period, marked by improvements in all muscle groups.
From baseline to Week 4, serum immunoglobulin G levels decreased significantly. Levels of immunoglobulin M, immunoglobulin A, cholesterol or albumin levels did not change, though.
No patient experienced any treatment-related adverse events, including allergic reactions or infections. One individual developed COVID-19 and experienced a myasthenic crisis but recovered quickly.
“These preliminary findings support further investigation of efgartigimod as a therapeutic option; however, its effectiveness and safety require definitive validation in larger, well-controlled studies,” the researchers concluded.
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