Short course of approved drug effective for treatment-resistant NMOSD
Study: Patients saw meaningful gains in mobility or vision within three months
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A short-course of Soliris (eculizumab) treatment during relapses may help people with neuromyelitis optica spectrum disorder (NMOSD) recover faster when standard therapies fail, a small study in China suggests.
Patients who saw little or no improvement after corticosteroids, with or without plasma exchange, experienced meaningful gains in mobility or vision within three months after receiving up to four weekly doses of Soliris, without serious treatment-related side effects.
“Our study provides preliminary evidence that limited-dose [Soliris] (1–4 weekly infusions) as an add-on therapy can accelerate neurological recovery during NMOSD attacks,” researchers wrote.
The study, “A novel limited-dose C5 inhibitor add-on as rescue therapy for refractory NMOSD attacks: a practical alternative to conventional multi-dose regimens,” was published in BMC Neurology.
Soliris approved for adults with NMOSD and anti-AQP4 antibodies
NMOSD is characterized by inflammation and damage to the spinal cord and optic nerves, which carry visual signals between the eyes and the brain. Most cases are caused by self-reactive antibodies that target a protein called aquaporin-4 protein (AQP4).
The disease is typically marked by relapses (periods of new or worsening symptoms), which can lead to vision problems, muscle weakness, or paralysis, making rapid treatment crucial to prevent permanent disability.
High-dose corticosteroids, which are potent anti-inflammatory medications, and plasma exchange (PLEX) — a procedure to help remove harmful antibodies from the blood — are commonly used as rescue therapies for relapses. However, some patients continue to experience severe symptoms despite these therapies.
Soliris is an approved therapy for adults with NMOSD and anti-AQP4 antibodies. It is administered via infusions into the bloodstream, once weekly for the first five times (loading doses) and every other week for subsequent infusions (maintenance doses). The first four loading doses are given at a dose of 900 mg, and the fifth loading dose, as well as subsequent maintenance doses, are given at a dose of 1,200 mg.
However, there is limited evidence of the therapeutic potential of Soliris to manage relapses.
All patients achieved good response after 3 months
To explore whether a limited-dose, add-on regimen of Soliris could be an effective and economically feasible rescue strategy in NMOSD, a team of researchers retrospectively analyzed data from eight female patients, with a median age of 57 years.
All were admitted to a single Chinese hospital between 2024 and 2025 due to an NMOSD attack that did not respond to standard rescue therapy. Six women were positive for anti-AQP4 antibodies, five had optic neuritis, or inflammation of the optic nerves, and three had spinal cord inflammation.
In terms of conventional rescue therapy, all received into-the-vein methylprednisolone, a corticosteroid, for five days, and four also underwent PLEX sessions. Overall, six women presented a poor response to these treatments, while the remaining two had an insufficient response.
All women subsequently received one to four weekly doses of Soliris (900 mg). One month after treatment, 42.9% had achieved a good response (full recovery or marked improvements), with this rate increasing to 100% after three months. This meant that the two women without anti-AQP4 antibodies also responded favorably, even though the therapy is not approved for this subgroup of patients.
Among women with spinal cord inflammation, disability scores measured by the Expanded Disability Status Scale dropped from 8.5, a level indicating they were bedridden most of the day, to 3.5 after three months, consistent with being able to walk independently.
[Soliris] facilitated rapid, clinically meaningful neurological and visual recovery, even in patients [resistant] to conventional first-line therapies.
Those with optic neuritis also experienced marked improvements in vision, with average vision scores approaching normal levels by three months.
These results demonstrated that Soliris “facilitated rapid, clinically meaningful neurological and visual recovery, even in patients [resistant] to conventional first-line therapies,” the researchers wrote.
After limited-dose Soliris treatment, women transitioned to long-term treatments, including Uplizna (inebilizumab), Enspryng (satralizumab), mycophenolate mofetil, and oral corticosteroids. No allergic or infusion-related reactions, infections, or treatment-related problems affecting blood cells, liver, or kidney function were reported.
The researchers cautioned that the study was small and retrospective in nature, but said the findings suggest limited-dose Soliris could offer a practical rescue treatment for severe NMOSD relapses that fail to respond to conventional therapies.
Overall, these findings underscore a “practical and effective therapeutic option, offering valuable real-world evidence to guide management, and laying a foundation for future … studies [following patients over time] aimed at optimizing treatment strategies and improving patient outcomes,” the team concluded.
