NMOSD therapy linked to fewer attacks, less disability in Chinese study
Estimated 1-year attack-free rate was 95.5% in adults with AQP4 antibodies
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Treatment with Uplizna (inebilizumab-cdon) was associated with fewer attacks and reduced disability in Chinese adults with neuromyelitis optica spectrum disorder (NMOSD) who have anti-aquaporin-4 (AQP4) antibodies, a real-world study found.
The estimated one-year attack-free rate was 95.5%.
According to the researchers, the findings “provided evidence on the real-world effectiveness and a manageable safety profile of [Uplizna] in a diverse population with [AQP4 antibody-positive] NMOSD.”
The study, “Inebilizumab in AQP4-Seropositive NMOSD: One-Year Follow-Up From a Multicenter, Real-World Study,” was published in Annals of Clinical and Translational Neurology.
How AQP4 antibodies drive NMOSD attacks
NMOSD is characterized by inflammation and damage to the spinal cord and the optic nerves, which carry signals between the eyes and the brain. Most patients have self-reactive antibodies that target a protein called aquaporin-4 (AQP4), thought to be key drivers of damaging immune activity.
Uplizna is an antibody-based therapy approved to treat adults with NMOSD who have anti-AQP4 antibodies. It is designed to reduce the number of B cells, the immune cells responsible for producing antibodies, including the self-reactive ones that contribute to NMOSD.
While the Phase 2/3 N-MOmentum trial (NCT02200770) established the treatment’s safety and efficacy, patients in clinical practice may differ from those enrolled in clinical trials, highlighting the need for real-world data. This may be particularly relevant for Chinese patients, as the N-MOmentum trial did not enroll participants from mainland China and only about 20% were of Asian descent.
To learn more, researchers conducted a study in 143 NMOSD patients at five tertiary hospitals in China. Participants had a mean age of 45 years, 85.3% were women, and they had been living with NMOSD for a median of 1.5 years before starting Uplizna.
Nearly all patients (96.5%) had received prior treatment, and around three-quarters had been on maintenance therapy, which included mycophenolate mofetil (22.4%), tacrolimus (16.8%), and rituximab (12.6%). Almost 20% of the patients had other autoimmune conditions, most commonly thyroid disease.
“By including a more heterogeneous population, this study provides real-world evidence for the effectiveness and safety of [Uplizna] in a broader NMOSD population,” the team wrote.
In a subset of 62 patients who had been on maintenance therapy and had available data, the most common reasons for starting Uplizna were relapse or inadequate disease control on previous therapy, patient preference, and side effects or tolerability issues.
Most patients remained attack-free after one year
As of Aug. 20, 2025, participants had received a median of four Uplizna infusions during a median follow-up of 12.4 months. Almost all patients (97.2%) completed the first two treatment infusions, 83.9% received a third dose, 57.3% a fourth dose, and 28.7% a fifth dose.
During follow-up, five patients experienced an NMOSD attack. The estimated 1-year attack rate was 4.5%, while the estimated 1-year attack-free rate was 95.5%.
Treatment with Uplizna was also associated with a lower annualized attack rate, which fell from 1.02 attacks per year before treatment to 0.03 after treatment. Among patients who previously received maintenance therapy, the median annualized attack rate decreased from 1.5 to zero.
Disability also decreased after starting Uplizna. The median Expanded Disability Status Scale score, a measure of neurological disability, decreased by 0.5 points after six months and one year. Only one patient (0.7%) experienced worsening disability after one year.
One year after starting Uplizna, only four patients (2.8%) had active lesions on MRI. Treatment was also associated with reductions in CD19-positive B-cell counts and levels of the immunoglobulins IgG, IgA, and IgM.
Overall, 32.9% of the patients experienced at least one treatment-emergent adverse event. The most common were urinary tract infections, high levels of lipids (fat-like molecules), anemia (low levels of red blood cells or of hemoglobin to carry oxygen to the body’s tissues), kidney dysfunction, fever, low blood protein levels, and an increased amount of white blood cells. One patient experienced a serious adverse event, pneumonia. No unexpected safety signals were reported.
“These results support the findings of the pivotal N-MOmentum trial and further establish [Uplizna] as a standard maintenance therapy for this patient population,” the researchers wrote.
A relatively short duration of follow-up and the lack of a control group were noted by the investigators as study limitations. The researchers also noted that attacks may have been underreported because follow-up was less standardized than in clinical trials, and that missing data limited conclusions from exploratory outcomes, including MRI and laboratory measures.
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