Generalized Myasthenia Gravis (gMG) is a rare chronic autoimmune disorder causing muscle weakness and fatigue, primarily due to autoantibodies that disrupt neuromuscular junction function. The most common antibodies target nicotinic acetylcholine receptors (AChR), with others such as anti-MuSK and anti-LRP4 being less prevalent. The conventional gMG treatments include acetylcholinesterase inhibitors, corticosteroids, immunosuppressant and, in case of myasthenic crisis, plasma exchange (PLEX) and intravenous immunoglobulins (IVIG). Treatment aims to achieve minimal manifestation status (MMS), but many patients face persistent symptoms or side effects. Corticosteroids, while effective, carry significant risks, especially for long-term use, such as, increased infection and cardiovascular risks, chronic conditions like hypertension, diabetes, and osteoporosis and quality of life impacts, including weight gain and mood changes. Elderly patients, who form the majority of the gMG population, are particularly vulnerable due to age-related comorbidities, which limit treatment options and prolong corticosteroid reliance. This contributes to increased mortality, disability, and dependency. Efgartigimod (EFG), a novel therapeutic targeting the neonatal Fc receptor (FcRn), accelerates degradation of pathogenic IgG antibodies, including anti-AChR. Clinical trials demonstrated its efficacy and safety in reducing antibody levels, improving muscle strength, and enhancing quality of life. Both intravenous (IV) and subcutaneous (SC) forms are effective and well tolerated. Approved in the United States and subsequently in Japan and Europe, EFG became available in France in 2023. The present multicenter observational study aims to evaluate the real-life impact of EFG in elderly gMG patients struggling with corticosteroid side effects or comorbidity exacerbations. The objectives of this study include the assessing EFG's ability to enable corticosteroid reduction and monitoring improvements in gMG symptoms, quality of life, comorbidities, and overall health. This approach highlights a shift towards targeted therapies that balance efficacy with reduced treatment-related burdens for vulnerable gMG populations.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
PREVENTION
Masking
NONE
Enrollment
50
Administration of efgartigimod, a neonatal Fc receptor (FcRn) antagonist, given either intravenously (IV) or subcutaneously (SC) to treat patients with generalized Anti-AChR Myasthenia Gravis.
CHU de Nice
Nice, Alpes-Maritimes, France
RECRUITINGevaluate at 12 months the change from baseline in the mean dose of corticosteroids in gMG patients switched to EFG
evaluate at 12 months the change from baseline in the mean dose of corticosteroids in gMG patients switched to EFG
Time frame: 12 mois
change in percentage of patients in low dose of corticosteroid (<20mg) at 12 months
change from baseline of the percentage of patient treated with \<20 mg of corticosteroids
Time frame: 12 months
change in percentage of patients in intermediate dose of corticosteroid (≥20 mg - ≤50 mg) at 12 months
change from baseline of the percentage of patient treated with ≥20 mg - ≤50 mg of corticosteroids
Time frame: 12 months
change in percentage of patients in high dose of corticosteroid (≥ 50mg) at 12 months
change from baseline of the percentage of patient treated with \>50 mg of corticosteroids
Time frame: 12 months
change in Glucocorticoid Toxicity Index (GTI) at 12 months
change from baseline in GTI
Time frame: 12 months
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