A case series of 3 patients with generalized myasthenia gravis (MG) demonstrated the long-term efficacy of convenient infusion intervals of ravulizumab (Ultomiris; AstraZeneca), an FDA-approved terminal compliment C5 inhibitor for adults who are antiacetylcholine receptor (AChR) antibody positive, in symptom reduction and stability. These results suggest ravulizumab shows promise as a chronic treatment for patients with this condition who were previously on concomitant immunomodulators.1
Among the 3 patients with MG treated with ravulizumab, findings showed that patients had extended their infusion intervals to 7 or 8 weeks, which were symptom-free. Results also revealed that ravulizumab stabilized symptoms and improved Myasthenia Gravis–Activities of Daily Living (MG-ADL) scores. Although patients had decreased exacerbations and showed no signs of significant adverse effects (AEs), authors recommended continuous monitoring while on the treatment.
Top Clinical Takeaways
- Ravulizumab, administered at 7-to-8–week intervals, demonstrated efficacy in stabilizing symptoms and reducing exacerbations in three generalized myasthenia gravis patients.
- The case series highlights significant improvement in MG-ADL scores and symptom reduction after switching to ravulizumab, suggesting its efficacy as a chronic treatment option.
- Continuous monitoring is recommended for patients on ravulizumab, even though the study reported no significant adverse effects, emphasizing the need for vigilance during long-term treatment.
The findings were presented as a poster at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 3-6, in Orlando, Florida, by senior author Raam Sambandam, MD, neurologist at Synapse Neurology, and colleagues. In this case series, investigators retrospectively reviewed the long-term impact of ravulizumab in patients with MG who received concomitant immunomodulatory treatments.
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In the first case, a 38-year-old woman with AChR antibody-positive generalized MG presented with ocular predominant, nonthymoma, with acute exacerbations of right eye ptosis. Diagnosed in 2016, the patient also displayed worsening limb weakness and shortness of breath. Previously, the patient had been treated with prednisone, mycophenolate, azathioprine, immunoglobulins, and efgartigimod (Vyvgart; Argenx). Following treatment with ravulizumab, the patient experienced symptom reduction, with an improvement observed on the MG-ADL score ranging from 8 to 5, and Morse Fall Scale ranging from 60 to 15. In addition, investigators reported no exacerbations observed at the 8-month follow-up after initiation of ravulizumab treatment with pyridostigmine extended-release dosage of 180 mg in May 2023.
In the second case, a 68-year-old man with long-standing generalized MG previously managed the condition with several treatments which included steroids, pyridostigmine, azathioprine, and immunoglobulin. Eculizumab (Soliris; Alexion) was introduced later because of persistent symptoms but the treatment resulted in AEs. When switched to ravulizumab in December 2022, alongside treatment of azathioprine 50 mg, findings showed stable clinical improvement in the patient. Then, at follow-up of 11 months, the patient achieved an MG-ADL score of 0 and resumed their normal activities.
