Soliris eases AChR+ refractory myasthenia gravis disease severity
Researchers in Italy conducted a real-world study with 14 patients
Soliris (eculizumab) effectively eased disease severity and reduced the need for immunosuppressive therapies in people with treatment-resistant generalized myasthenia gravis with anti-AChR antibodies, a real-world study in Italy shows.
The treatment also reduced the number of disease exacerbations that required rescue therapy, such as intravenous immunoglobulin (IVIG) and/or plasma exchange.
“[Soliris] rapidly improved (within the first month) all clinical outcome measures. Long-term efficacy was maintained, leading to remarkable immunosuppressant-sparing benefits,” the researchers wrote. “Its rapid effect onset also suggests its potential use in patients unresponsive to the rescue therapies in jeopardy of myasthenic crisis.”
The study, “Eculizumab in refractory myasthenia gravis: a real-world single-center experience,” was published in Neurological Sciences.
Myasthenia gravis (MG) is an autoimmune disease caused by autoantibodies that target proteins at the neuromuscular junction, the site where nerve and muscle cells communicate to coordinate voluntary movements. The antibodies most commonly target acetylcholine receptors (AChRs).
Treatment with immunosuppressive therapies is generally effective. However, 10% to 15% of patients are considered refractory, meaning they either fail to respond, are unable to tolerate it, or cannot reduce it.
Soliris, an antibody-based therapy that inhibits the activation of a part of the immune system called the complement cascade, is approved in Europe to treat patients with refractory generalized MG and anti-AChR antibodies.
Declines in disease severity with Soliris
Here, researchers in Italy retrospectively analyzed data from 14 patients (eight women, six men) with refractory AChR-positive gMG who were treated with Soliris between November 2022 and March 2024 for at least a year. The patients had a mean age of 53.6 and had been living with the disease for a mean of 11.3 years. Eight patients underwent a thymectomy, a surgical procedure to remove the thymus gland that may help decrease autoantibody production and ease MG symptoms, over two years before starting Soliris.
At the beginning of the study, all the patients were receiving several treatments, including corticosteroids and immunosuppressants. Also, in the year before starting Soliris, they had all received rescue treatment with IVIG and/or plasma exchange at least once to manage acute exacerbations, or episodes of sudden disease worsening.
During Soliris treatment, there was a progressive, clinically meaningful decline in the total score of the MG Activities of Daily Living (MG-ADL) scale, which assesses the impact of MG on day-to-day activities, and in the total score of the Quantitative MG (QMG) scale, which evaluates muscle weakness. MG-ADL and QMG scores fell significantly after one and three months of treatment, and remained consistently low thereafter for up to a year.
All the patients were also able to progressively reduce their steroid dose, with the daily dose of prednisone decreasing from a mean of 29.8 mg to 11.2 mg after a year. Three patients also stopped taking conventional immunosuppressants. No severe exacerbations were reported and no rescue therapies were required after starting Soliris.
Eight patients had adverse events that were possibly related to Soliris and all were considered mild. These included upper respiratory tract infection, headache, nausea, and herpes virus and bacterial skin infections.
“This real-world study confirmed safety and effectiveness of [Soliris] in 14 refractory [AChR-positive] MG patients during 12 months of treatment,” wrote the researchers, who said the benefits of Soliris should be evaluated in larger real-world studies with a “wider gMG refractory population.”
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