Most adults with NMOSD use biologic therapies: Registry study
SPHERES registry data show wide use of approved or off-label treatments
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A U.S.-based registry of adults with neuromyelitis optica spectrum disorder (NMOSD) shows that most patients are receiving biologic therapies, either approved or not approved for NMOSD, in real-world care, according to a study of its first 350 participants.
The registry, called SPHERES, has enrolled about half of its planned 800 participants and is designed to follow people with NMOSD over time to better understand how the disease affects them and how current treatments are used in the real world.
The study, “SPHERES: innovative registry addressing the new era of neuromyelitis optica spectrum disorders (NMOSD) therapy,” was published in Multiple Sclerosis and Related Disorders. It was funded by Corevitas, which launched the registry in the U.S. in 2021 in collaboration with the Guthy-Jackson Charitable Foundation.
NMOSD is a rare autoimmune disease in which self-reactive antibodies attack cells that support the nervous system, leading to inflammation and nerve damage, particularly in the spinal cord and optic nerves (which connect the eyes to the brain).
The disease is most commonly driven by antibodies targeting a protein called aquaporin-4 (AQP4), though some patients test negative for these antibodies.
Collecting high-quality data
Several biologic therapies have been approved in recent years to treat NMOSD, particularly in people with anti-AQP4 antibodies. Less is known about how the disease progresses and is treated in everyday clinical practice, especially among patients who test negative for known disease-causing antibodies.
“SPHERES was developed in response to these unmet needs to collect comprehensive and high-quality longitudinal data, aiming to study the natural history of NMOSD, as well as real-world therapy patterns, effectiveness, and safety,” the researchers wrote. Longitudinal data are collected from patients followed over time.
The registry gathers clinical, demographic, and lifestyle data, along with clinician- and patient-reported outcomes. Questionnaires are completed at enrollment and during regular clinic visits, usually about every six months, with additional data collected between visits.
Patients may join the registry if they are starting treatment at enrollment, have begun therapy within the previous year, or are untreated. The average age of the first 350 patients enrolled was 50.1, and most (84%) were women.
On average, patients had been living with NMOSD for nearly a decade, and 71% were using biologic therapies at the time they joined the registry.
About two-thirds of patients (66%) tested positive for and-AQP4 antibodies. Another 17% tested positive for antibodies against the MOG protein, which causes a related condition called MOG antibody-associated disease (MOGAD). Nine percent tested negative for both antibodies, while 8% had not been tested or had unknown results.
The time since diagnosis differed across antibody groups. Patients with anti-AQP4 antibodies had been diagnosed for an average of 6.9 years, while those with MOGAD had been diagnosed for 2.8 years. Diagnosis dated back further for double-negative patients (8.1 years) and for those who had not been tested or had unknown results (10.3 years).
Most patients had experienced at least one disease relapse before joining the registry. About one-third (35%) had experienced a single relapse, including their first episode, while 17% had experienced two relapses, and nearly half (48%) had experienced three or more.
The most common relapse symptoms were optic neuritis, or inflammation of the optic nerve (67%), and acute myelitis, or inflammation of the spinal cord (63%).
Among patients receiving biologic therapy, 113 were using a biologic approved for NMOSD, and 136 were using a biologic off label. Since all available NMOSD biologics are approved for people with anti-AQP4 antibodies, use of approved biologics was more common among these patients than among those with MOGAD (45% vs. 5%).
It was more common for patients with anti-AQP4 antibodies than for those with MOGAD to use biologics approved for anti-AQP4-positive NMOSD (45% vs. 5%), including Enspryng (satralizumab), Soliris (eculizumab), Ultomiris (ravulizumab), and Uplizna (inebilizumab).
Overall disability levels were in line with those reported in other real-world NMOSD studies, although the researchers noted a higher frequency of cognitive issues among patients enrolled in the registry.
“These insights may facilitate reduced disease burden and ultimately enhance patient care and quality of life,” the researchers wrote. They said ongoing follow-up will help them better understand how NMOSD progresses and how safe and effective current treatments are in everyday clinical care.
