Niklas Huntemann, University Hospital of Düsseldorf, discusses real-world experience with therapies targeting either the complement system (C5) or neonatal Fc receptor (FcRn) in patients with myasthenia gravis (MG).
MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. The condition results from a defect in the transmission of nerve impulses to muscles, which is due to the presence of antibodies against acetylcholine. The exact reason this occurs is not known. Weakness tends to increase during periods of activity and improve after periods of rest. Common symptoms include weakness of the muscles that control:
- Eye and eyelid
- Facial expressions
- Chewing
- Talking
- Swallowing
Immunosuppression is the standard care treatment for MG, however such therapies are not fully effective and do have a plethora of adverse events. Recent advancements are giving hope to patients who require more targeted therapies or refractory to standard immunotherapies. These include complement C5 inhibition which targets the complement system, and FcRn antagonism, which targets IgG antibody recycling. Studies are currently underway to discover which of these therapies is more effective.
Study
The study led by Mr. Huntemann aims to observe real-world experience with C5 complement inhibition and FcRn modulation in patients with MG. The analyses focused on complement inhibitors eculizumab and rozanolixizumab, and FcRn antagonist, efgartigimod. Clinical outcomes and safety were observed in 21 patients (C5 inhibitors) and 15 patients (FcRn antagonists), respectively. The primary endpoint was reduction in MG Activities of Daily Living scores. Additionally, changes in pyridostigmine and prednisolone doses and improvement to Quantitative Myasthenia Gravis and MG Quality of Life 15 scores were assessed.
It was observed that both C5 inhibition and FcRn antagonism significantly improved symptoms and enhanced patients’ quality of life. However, one therapy did not significantly perform better than the other and some patients expressed insufficient treatment response. These findings highlight the need for further research and additional therapeutic options for patients with MG.
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To learn more about MG and other rare musculoskeletal disorders, visit https://checkrare.com/diseases/musculoskeletal-diseases/
