Efgartigimod as Fast-Acting Rescue Therapy in Very Late-Onset Myasthenia Gravis

Area of Science:

• Neurology
• Immunology
• Pharmacology

Background:

• Myasthenic crisis (MC) is a life-threatening exacerbation of myasthenia gravis (MG).
• Conventional treatments like IVIG and corticosteroids are standard but may be insufficient in severe cases.
• Pathogenic IgG antibodies play a crucial role in the neuromuscular junction dysfunction seen in MG.

Purpose of the Study:

• To report the successful use of efgartigimod as a rescue therapy for a severe myasthenic crisis.
• To evaluate the efficacy and safety of efgartigimod in a patient refractory to standard treatments.
• To highlight efgartigimod's potential in managing refractory myasthenic crisis, including in elderly patients.

Main Methods:

• Case report of a 77-year-old male patient with anti-acetylcholine receptor (AChR) positive generalized MG.
• Administration of intravenous efgartigimod as rescue therapy during a severe myasthenic crisis.
• Assessment of clinical response to efgartigimod following inadequate response to IVIG and high-dose corticosteroids.

Main Results:

• The patient experienced rapid and sustained clinical improvement after receiving intravenous efgartigimod.
• Efgartigimod demonstrated efficacy in a patient with severe MC unresponsive to conventional therapies.
• The treatment was well-tolerated, suggesting a favorable safety profile in this context.

Conclusions:

• Efgartigimod is a promising novel therapeutic option for managing myasthenic crisis, particularly in cases resistant to standard care.
• This case underscores the potential of targeting neonatal Fc receptor (FcRn) with efgartigimod for rapid symptom control in severe MG exacerbations.
• Efgartigimod may be a valuable addition to the treatment armamentarium for myasthenic crisis, even in older patients with comorbidities.