Eculizumab treatment leads to clinical improvements in most patients with generalized myasthenia gravis (MG), according to a new study published in Muscle and Nerve. Moreover, the treatment had a favorable safety profile even when combined with other immunosuppressants.

Because the safety of eculizumab in patients with generalized MG in clinical practice is limited and because the concomitant use of other medications may be different in the real world compared to clinical trials, a team of researchers from Brigham and Women’s Hospital, Harvard Medical School, and Massachusetts General Hospital set out to analyze the clinical and safety outcomes of patients who received eculizumab at their institutions.

The team first identified patients with acetylcholine receptor antibody positive (AChR+) generalized MG who received 1 or more doses of eculizumab and had at least 1 follow-up. There were 12 such patients in the database.

The researchers found that clinical improvements were seen as soon as 1 month after the initiation of treatment with eculizumab. More precisely, the Myasthenia Gravis Foundation of America Post Intervention Status improved in 80% of patients while Clinical Classification improved in 83% of patients at 1 month.

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The mean Myasthenia Gravis Activities of Daily Living scores decreased from 8.7 to 2.8 at 1 month. It remained at 3.5 or lower over 1.5 years. The mean dose of daily prednisone use also decreased (from 22.5 mg a day to 7.2 mg a day) at 1.5 years. 

Moreover, 5 of 7 patients who were receiving maintenance intravenous immune globulin or plasmapheresis treatment discontinued these treatments. No patients had meningococcal infections and any adverse events that occurred as a result of eculizumab treatment were mild.

Eculizumab sold under the brand name Soliris® is a late complement inhibitor for MG. It is approved by the US Food and Drug Administration to treat adult patients with the refractory disease who are AChR+.

Reference

Suh J, Clarke V, Amato AA, Guidon AC. Safety and outcomes of eculizumab for acetylcholine receptor-positive generalized myasthenia gravis in clinical practice. Muscle Nerve. Published online June 9, 2022. doi:10.1002/mus.27656