Efgartigimod combined with steroids as a fast-acting therapy for myasthenic crisis: a case report

• Published in: BMC neurology Vol. 24; no. 1; pp. 292 - 7
• Main Authors: Ohara, Hiroya, Kikutsuji, Naoya, Iguchi, Naohiko, Kinoshita, Masako
• Format: Journal Article
• Language: English
• Published: England BioMed Central Ltd 22.08.2024; BioMed Central; BMC
• Subjects: Acetylcholine receptors; Acetylcholinesterase; Activities of daily living; Adrenal Cortex Hormones - administration & dosage; Adrenal Cortex Hormones - therapeutic use; Aged; Antibodies; Care and treatment; Case Report; Case reports; Cholinesterase inhibitors; Clinical trials; Corticosteroid; Corticosteroids; Deglutition disorders; Diplopia; Drug dosages; Drug Therapy, Combination; Dysphagia; Dyspnea; Efgartigimod; Enzyme inhibitors; Fc receptors; FcRn inhibitor; FDA approval; Female; Health aspects; Humans; Immunoglobulins; Immunosuppressive agents; Immunotherapy; Intravenous administration; Intubation; Myasthenia gravis; Myasthenia Gravis - drug therapy; Myasthenic crisis; Neonatal Fc receptor inhibitor; Neuromuscular junctions; Patients; Plasma exchange (Therapeutics); Plasmapheresis; Pyridostigmine; Respiration; Respiratory failure; Steroid hormones; Steroids; Tacrolimus; Japan; Myasthenic crisis; Corticosteroid; Efgartigimod; Myasthenia gravis; FcRn inhibitor; Neonatal Fc receptor inhibitor
• ISSN: 1471-2377; 1471-2377
• DOI: 10.1186/s12883-024-03804-y

Generalized myasthenia gravis (gMG) can be managed with acetylcholinesterase inhibitors (AChEis; e.g., pyridostigmine), corticosteroids, other immunosuppressive drugs (e.g., tacrolimus), and their combinations. Intravenous immunoglobulin (IVIg) or plasmapheresis (PLEX) may be administered if symptoms persist. PLEX and IVIg are also mainstays of treatment for myasthenic crisis. Recently, efgartigimod was approved in Japan for treating adults with gMG (irrespective of the antibody status) who do not have a sufficient response to corticosteroids and nonsteroidal immunosuppressive therapies. Efgartigimod is generally safe and well tolerated. However, since phase III trials of efgartigimod excluded those with myasthenic crisis, the efficacy of efgartigimod in treating myasthenic crisis is still unclear. Moreover, there are no reports that efgartigimod therapy can reduce the dose of corticosteroids needed to achieve a minimal manifestation status. We report the case of a 70-yeat-old woman with gMG who developed a myasthenic crisis. After she was diagnosed with gMG, the patient had been treated with oral corticosteroids and tacrolimus for 1 year. However, she refused to continue taking the medication, and two weeks later, she developed ptosis, dysphagia and dyspnea. The patient was intubated and treated with efgartigimod in combination with steroid therapy, and she recovered without PLEX or IVIg. Afterward, when she experienced worsening of fatigue and increased levels of anti-acetylcholine receptor antibodies, efgartigimod therapy was effective. The patient achieved minimal manifestation status even after the reduction of corticosteroids and showed improvements in the Myasthenia Gravis Activities of Daily Living scales after 4 cycles of efgartigimod infusion. Our case suggests that efgartigimod can be an alternative drug for achieving minimal manifestation status in patients with myasthenic crisis. Considering its strong efficacy and safety, efgartigimod could be expanded to use as bridging therapy in the acute and chronic phases of gMG.