Soliris reduces relapse rates in AQP4-positive NMOSD patients: Study
Therapy also shown to stabilize disability progression
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Soliris (eculizumab) is linked to a reduced relapse rate and stabilization of disability progression in people with neuromyelitis optica spectrum disorder (NMOSD) who test positive for anti-AQP4 antibodies, according to a real-world study in Turkey.
“These results support [Soliris] as a valuable therapy for [AQP4-related] NMOSD, while stressing the need for careful safety monitoring and personalized treatment decisions,” researchers wrote.
The study, “Real-world effectiveness and safety of eculizumab in AQP4-IgG-positive neuromyelitis optica spectrum disorder,” was published in the Journal of Neurology.
NMOSD usually follows a relapsing pattern
NMOSD is an autoimmune disease marked by damaging inflammation of the spinal cord and the optic nerve, which relays signals between the eye and the brain. Most cases are associated with the presence of self-reactive antibodies against the AQP4 protein, which is highly present on the surface of nerve-supporting cells.
The disease usually follows a relapsing pattern, meaning episodes of worsening symptoms (relapses) are interspersed with periods when symptoms ease.
Soliris is approved for the treatment of adults with NMOSD who are positive for anti-AQP4 antibodies. It works to reduce the risk of relapses by suppressing activation of the complement pathway, a part of the immune system known to contribute to NMOSD. It is administered by an into-the-vein infusion once every two weeks, following five initial weekly doses.
However, “despite promising results from clinical trials, real-world evidence on the effectiveness and safety of [Soliris] remains limited,” the researchers wrote.
Mean relapse rate decreased after 2 years of treatment
To learn more, a team of researchers in Turkey retrospectively analyzed data from 46 NMOSD patients with anti-AQP4 antibodies who were treated with Soliris across 26 centers in five countries.
Most participants were from Turkey (91.3%) and were women (91.3%). They had a mean age at disease onset of 36.8 years. They were living with the disease for 9.4 years, and started Soliris treatment between 2019 and 2025.
The most common initial symptoms included optic neuritis (82.6%) — signs of optic nerve inflammation — and transverse myelitis (63%), or signs of spinal cord inflammation.
In more than half of patients (56.5%), Soliris was the third-line therapy after two other immunosuppressive medications; in 21.7%, it was second-line, and in 19.6%, it was fourth-line.
Soliris treatment lasted a mean of 27.2 months, or a little over two years, and in 28.2% of patients, it was combined with other immunosuppressants (corticosteroids or azathioprine).
Before starting Soliris, participants experienced a mean of 6.2 relapses, 2.4 in the two years before and 1.4 in the previous year. After two years of treatment, the mean relapse rate decreased to 0.3. The proportion of patients free from relapses significantly increased from 6.5% in the two years before and 8.7% in the prior year, to 80.4% during treatment with Soliris.
The annualized release rate of the estimated number of relapses per year was 1.1 in the two years prior to Soliris initiation, 1.4 in the year prior, and 0.1 while on Soliris.
Half of patients had reduction in disease-related disability after 6 months
After six months of treatment, half of the patients experienced a reduction in disease-related disability, while 21 participants remained stable and two worsened, as assessed by the Expanded Disability Status Scale (EDSS). After two years, 13 patients had lower disability, while in seven it stabilized, and in two it worsened.
Also, the mean EDSS score decreased from 4.2 at the time of Soliris initiation to 3.7 after six months of treatment, 3.4 after 1.5 years, and 3.6 after two years.
“These findings collectively indicate a beneficial effect of [Soliris] on disability outcomes, particularly during the first year of treatment, with subsequent stabilization or sustained response in most patients,” the researchers wrote.
Our findings strengthen the evidence for complement [suppression] as an effective treatment option while highlighting key clinical considerations for patient selection and monitoring.
The presence of area postrema syndrome, or uncontrollable nausea, vomiting, or hiccups, before starting on Soliris was significantly associated with a lessening of disability after one and 1.5 years of Soliris treatment.
In contrast, a relapse affecting the spinal cord before Soliris treatment was significantly more common in patients who did not experience a reduction in disability with Soliris, “suggesting a possible association with poorer treatment response,” the researchers wrote.
The therapy was well tolerated, with seven patients (18.9%) experiencing treatment-related adverse events and four discontinuing the treatment.
“Our findings strengthen the evidence for complement [suppression] as an effective treatment option while highlighting key clinical considerations for patient selection and monitoring,” the researchers wrote. “Special attention should be given to spinal attack history as a potential negative predictor … and [area postrema syndrome] as a possible positive predictor of EDSS improvement.”
