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EFGARTIGIMOD TREATMENT IN A IN A IMMUNE CHECKPOINT INHIBITOR-ASSOCIATED MYASTHENIA GRAVIS: A CASE REPORT

European Statement

Clinical Pharmacy Services

Author(s)

E Paradela, M Rodríguez, A Romero, MD Alvarado

Why was it done?

To treat a 72-year-old patient admitted to the ICU who developed GMG as an adverse effect to his first cycle of pembrolizumab (an immune checkpoint inhibitor drug).

What was done?

This case report describes the use of efgartigimod, a high economic impact drug indicated in adult patients with generalized myasthenia gravis (GMG) who are antibody-positive against acetylcholine receptors (AChR), not controlled with pyridostigmine, corticosteroids and at least two conventional immunosuppressive therapies.

How was it done?

In June 2024, the patient was diagnosed with a melanoma recurrence and started an adjuvant treatment with pembrolizumab. 18 days after his first cycle, the patient went to the emergency room with characteristic GMG symptoms such as asthenia, myalgia, proximal limb weakness and ptosis in both eyes.

The treatment consisted of methylprednisolone (2 mg/kg), immunosuppressants (tacrolimus), high-dose pyridostigmine (90 mg/4 h), immunoglobulins (2 g/kg) and 7 plasmapheresis sessions. Tacrolimus was included as immunosuppressive therapy and cyclosporine was intended to be started, but both were discontinued due to the risk of worsening the bicytopenia. It was then decided to perform an antibody study in order to guide the treatment. The positive result of the anti-AChR antibodies and the lack of control of the pathology after conventional treatment, suggested starting a treatment with efgartigimod 800 mg/week for 4 weeks.

What has been achieved?

After four efgartigimod cycles, there was a progressive improvement in neurological weakness, with persistent oculomotor impairment and palpebral ptosis. Almost two months after the efgartigimod treatment, an excellent progressive evolution has been observed and discharge is expected in one week. Nowadays, the patient continues with high doses of pyridostigmine, prednisone in a descending regimen and follow-up by dysphagia and speech therapy units.

What next?

Myasthenia gravis is an immune-related adverse effect caused by checkpoint inhibitors, such as pembrolizumab, whose prevalence is growing with the increasing use of these drugs. In cases of persistent MG, efgartigimod is considered an effective option as an add-on treatment that provides symptom improvement in clinical practice.

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