This study aims to evaluate the efficacy and safety of different monoclonal antibody in the acute phase of neuromyelitis optica spectrum disorder (MAAP-NMO). It will also examine immune-related biomarkers and their relationship with treatment response to provide evidence for optimizing acute-phase therapeutic strategies.
Neuromyelitis optica spectrum disorder (NMOSD) is an inflammatory demyelinating disease of the central nervous system characterized primarily by humoral immune dysfunction. The acute phase is highly disabling; therefore, improving the effectiveness of acute-phase interventions represents a critical challenge in the clinical management of NMOSD. Conventional acute treatments such as intravenous methylprednisolone (IVMP), plasma exchange (PE), and intravenous immunoglobulin (IVIg) provide only limited rates of remission. The advent of novel biologics has expanded therapeutic options for NMOSD, but consensus regarding the optimal treatment approach during the acute phase has not yet been established. This project is a multicenter, prospective, real-world observational study. A total of 35-45 patients with acute-phase NMOSD from 12 centers across China will be enrolled and followed systematically for at least 6 months according to a standardized protocol. The study will evaluate the real-world efficacy and safety of different monoclonal antibodies, primarily focusing on efgartigimod and eculizumab, in the treatment of acute-phase NMOSD. It will further assess their impact on symptom and neurological disability improvement, as well as their effects on immunological parameters, biomarkers, and imaging outcomes, in order to explore the optimal acute-phase immunotherapy strategy in NMOSD.
Study Type
OBSERVATIONAL
Enrollment
40
Change in Expanded Disability Status Scale (EDSS) score
Change in Expanded Disability Status Scale (EDSS) score from baseline to 1 months after treatment (EDSS: Minimum Score 1, Maximum score 10, higher scores mean a worse outcome).
Time frame: 1 months
Change in Expanded Disability Status Scale (EDSS) score
Change in Expanded Disability Status Scale (EDSS) score from baseline at 3 and 6 months (EDSS: Minimum Score 1, Maximum score 10, higher scores mean a worse outcome).
Time frame: 3 and 6 months
Percentage of Participants with Improvement
Improvement is defined as: 1. ≥1.0-point decrease from baseline if baseline EDSS ≤5.5; 2. ≥0.5-point decrease from baseline if baseline EDSS \>5.5.
Time frame: 1, 3 and 6 months
Change in Low-Contrast Visual Acuity (LCVA)
Low-Contrast Visual Acuity (LCVA), using a standardized low-contrast ETDRS visual acuity chart, the number of letters that the patient can identify at a sitting position and a distance of 4 meters / 1 meter is measured to reflect the recovery of visual function.
Time frame: 1, 3 and 6 months
Percentage of Participants without relapse
Relapse is defined as new neurological symptoms or significant worsening of existing symptoms lasting ≥24 hours during the follow-up period, excluding causes such as infection or fever, and confirmed by imaging or clinical evaluation.
Time frame: 1, 3 and 6 months
MRI changes after immunotherapy
MRI changes including improvement of acute-phase pre-existing T2 hyperintense lesions and enhancing lesions (volume reduction, signal attenuation, or decreased enhancement), and the number of new and/or enlarged T2 hyperintense lesions or new enhancing lesions.
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Time frame: 1 and 6 months
Change in serum AQP4-IgG titer
Change in serum AQP4-IgG titer before treatment and at 1, 3, and 6 months after immunotherapy, measured by cell-based assay.
Time frame: 1, 3 and 6 months
Change in serum GFAP level
Change in serum GFAP level before treatment and at 1, 3, and 6 months after immunotherapy, measured by Simoa immunoassay.
Time frame: 1, 3, and 6 months
Change in serum NfL level
Change in serum NfL level before treatment and at 1, 3, and 6 months after immunotherapy, measured by Simoa immunoassay.
Time frame: 1, 3, and 6 months
Changes in serum IL-6, IL-17, TNF-α, IFN-γ, and IL-10 levels
Changes in serum IL-6, IL-17, TNF-α, IFN-γ, and IL-10 levels before treatment and at 1, 3, and 6 months after immunotherapy, measured by multiplex bead-based immunoassay. Units: pg/mL.
Time frame: 1, 3 and 6 months
Changes in T/B cell subsets
Changes in T/B cell subsets (immune function monitoring) before treatment and at 1, 3, and 6 months after immunotherapy.
Time frame: 1, 3 and 6 months
Changes in serum lactate, pyruvate, and glucose levels
Energy metabolism will be assessed by quantifying serum lactate, pyruvate, and glucose concentrations at baseline, 1, 3, and 6 months using liquid chromatography-tandem mass spectrometry (LC-MS/MS). Units: mmol/L.
Time frame: 1, 3 and 6 months
Changes in serum lipid metabolites
Lipid metabolism will be evaluated by measuring serum cholesterol at baseline, 1, 3 and 6 months using LC-MS/MS. Units: µmol/L.
Time frame: 1, 3 and 6 months
Incidence of adverse events (AEs) and serious adverse events (SAEs)
AEs are any unfavorable medical occurrences during the study period, regardless of causality. SAEs include events resulting in death, life-threatening conditions, permanent or significant disability or loss of function, hospitalization or prolongation of hospitalization, congenital anomaly, or birth defect. Record the time of onset, type, severity, and outcome. Drug-related adverse effects of biologics (e.g., efgartigimod, inebilizumab) will be specifically monitored, including hypogammaglobulinemia (assessed at baseline, 1, 3, and 6 months) and occurrence of meningococcal infection.
Time frame: 1, 3 and 6 months