Neuromyelitis Optica Spectrum Disorders (NMOSD)
Conditions
Keywords
NMOSD, eculizumab, AQP4-ab, Neuromyelitis Optica Spectrum Disorders
Brief summary
This is an observational cohort study based on data from the hospital-based NMOSD registry (Chinese Medical Research Registration Number MR-31-22-008563; ChiCTR2000030651). Between October 2023 (when eculizumab was approved for NMOSD in China) and February 2025, 26 consecutive patients with AQP4-IgG-positive NMOSD received eculizumab, and 9 of them were included in this study.
Interventions
Eculizumab was administered intravenously at a dosage of 900 mg weekly for four consecutive weeks
Sponsors
Huashan Hospital
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
(1)Patients have been diagnosed with NMOSD and tested seropositive for AQP4 antibody; (2) Age ≥ 18 years; (3) Received eculizumab during an acute phase of NMOSD, defined as within 30 days of attack onset； (4) Adherence to an 8-week follow-up from eculizumab initiation.
Exclusion criteria
1. Patients with unresolved Neisseria meningitidis infection or severe infections that preclude the use of immunotherapy; 2. Patients with severe comorbidities (such as heart failure, respiratory failure, severe hepatic or renal dysfunction, etc.); 3. Patients with incomplete records of clinical symptoms and signs, as well as insufficient data on serum marker tests in their medical records.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The change in disability status measured by the MRC scale score | 0, 1, 2, 3, 4, 8 weeks | Measured by the MRC scale score for patients with LETM |
| The change in disability status | 0, 1, 2, 3, 4, 8 weeks | Measured by the best corrected visual acuity (BCVA) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The Change in Expanded Disability Status Scale (EDSS) scores | 0, 1, 2, 3, 4, 8 weeks | Measured by Expanded Disability Status Scale (EDSS) scores for the entire cohort. |
| The change in Visual Functional System Score (VFSS) | 0, 1, 2, 3, 4, 8 weeks | Measured by Visual Functional System Score (VFSS) for patients with ON. |
| The Change in Opticospinal Impairment Scale (OSIS) scores | 0, 1, 2, 3, 4, 8 weeks | Measured by Opticospinal Impairment Scale (OSIS) for the entire cohort. |
| Incidence of AEs and SAEs during eculizumab treatment | 1, 2, 3, 4, 8 weeks | dverse events were coded using MedDRA version 27.1. Adverse events were analyzed in the safety analysis population (all patients who received at least one dose of study treatment) in terms of percentage incidence and as rates by exposure time (number of events per 100 patient-years of exposure and the associated 95% CI) to adjust for any differences in duration of exposure. The severity of adverse events was measured by NCI CTCAE version 5.0. |
Other
| Measure | Time frame | Description |
|---|---|---|
| The changes of serological biomarkers (sGFAP and sNfL) | 0, 2, 4, 8 weeks | Serum GFAP and NfL concentrations were analyzed in duplicates using SIMOA. |
| The changes of OCT measurements (pRNFL and mGCIPL). | 0, 1, 2, 3, 4, 8 weeks | The peripapillary retinal nerve fiber layer (pRNFL) and macular ganglion cell-inner plexiform layer (mGCIPL) was analyzed by optical coherence tomography (OCT) measurements |
Countries
China
Outcome results
None listed