This is a multi-centre, retrospective-prospective, single-arm, non-interventional (observational) cohort study with secondary data collection within real-world settings of participants with AQP4-IgG positive NMOSD.
This is a multicenter, retrospective-prospective, single-arm observational cohort study using secondary data collection from routine care medical records. The primary objective is to describe baseline demographic and clinical characteristics, diagnostic algorithms, and treatment approaches. Secondary objectives are to describe Expanded Disability Status Scale (EDSS) levels and dynamics, collect the rate, duration, and reasons for hospitalizations, and evaluate physician-reported relapse profiles. Approximately 100 adults will be enrolled consecutively across about 10 specialized sites. The study will sequentially include only those patients who have signed the informed consent form (ICF). Eligible patients will be enrolled consecutively at each site to minimize selection bias. Each participant will be followed for 36 months from informed consent (T0), with data collection every 6 months (T1-T6). The baseline period is defined as the time from NMOSD diagnosis until inclusion, with retrospective data abstraction; subsequent data are collected prospectively at routine visits. All data are entered into an electronic case report form (eCRF) from paper/electronic medical records. No study-specific interventions are performed; treatment is determined by usual care.
Study Type
OBSERVATIONAL
Enrollment
100
Research Site
Novosibirsk, Russia
RECRUITINGBaseline demographics and clinical characteristics
Summary of participant demographics and clinical history at inclusion: age at inclusion and at NMOSD diagnosis, sex, ethnicity, BMI at inclusion, disease duration, clinical symptoms at inclusion, comorbidities.
Time frame: At inclusion
Pre-inclusion relapse history
Proportion of patients with ≥1 and \>1 physician-reported relapses and severe relapses (EDSS increase ≥2.0 points from baseline per event); annualized relapse rate (ARR) prior to inclusion.
Time frame: From NMOSD diagnosis to inclusion (retrospective baseline)
Pre-inclusion NMOSD-related hospitalizations
Number of patients with NMOSD-related hospitalizations from the time of NMOSD diagnosis; median cumulative duration (days) of NMOSD-related hospitalizations prior to inclusion.
Time frame: From NMOSD diagnosis to inclusion (retrospective baseline)
Time from first symptoms to NMOSD diagnosis
Median time (months) from patient-reported first NMOSD symptoms to confirmed NMOSD diagnosis according to 2015 IPND criteria.
Time frame: From first NMOSD symptoms to date of diagnosis (retrospective baseline)
Prior misdiagnoses profile
Proportion of patients with any prior misdiagnosis and by type (e.g., MS, MOGAD, CNS infections, SLE only, Sjögren's only, Behçet's, neurosarcoidosis, CNS vascular disease, toxic/metabolic, neoplasms/paraneoplastic, congenital CNS, other).
Time frame: From first NMOSD symptoms to confirmed NMOSD diagnosis (retrospective baseline)
AQP4-IgG testing method
Number and proportion of patients tested by cell-based assay versus ELISA for AQP4-IgG serostatus determination.
Time frame: At time of NMOSD diagnostic workup (retrospective baseline)
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MRI brain T2-hyperintense lesion count change
Mean change from baseline in the number of T2-hyperintense brain lesions; presence of T1 contrast-enhancing lesions recorded as categorical variables.
Time frame: Baseline (≤12 months pre-inclusion) and Months 6, 12, 18, 24, 30, and 36 post-inclusion
Optic nerve MRI findings
Presence of contiguous lesions, bilateral neuritis, chiasmal extension, and optic nerve atrophy recorded as categorical variables per timepoint.
Time frame: Baseline (≤12 months pre-inclusion) and Months 6, 12, 18, 24, 30, and 36 post-inclusion
Spinal cord MRI lesion metrics
T2 lesion count; presence of longitudinally extensive lesions (≥3 segments), transverse lesions, and spinal cord atrophy extending ≥3 segments recorded as categorical variables per timepoint.
Time frame: Baseline (≤12 months pre-inclusion) and Months 6, 12, 18, 24, 30, and 36 post-inclusion
Relapse prevention therapy patterns
Number of patients receiving relapse prevention therapy by regimen: immunosuppressive drugs monotherapy; biologic monotherapy; biologic plus immunosuppressive combination; mean daily corticosteroid dose if low-dose steroids used (prednisolone-equivalent).
Time frame: From NMOSD diagnosis to inclusion and Months 6, 12, 18, 24, 30, and 36 post-inclusion
Acute relapse treatment modalities
Number of patients receiving high-dose corticosteroids, plasma exchange, or immunoadsorption for acute relapses; summarized per period.
Time frame: From NMOSD diagnosis to inclusion and Months 6, 12, 18, 24, 30, and 36 post-inclusion
Concomitant medications
Number of patients by class/type of concomitant medications for comorbid conditions recorded from diagnosis to inclusion and prospectively.
Time frame: From NMOSD diagnosis to inclusion and Months 6, 12, 18, 24, 30, and 36 post-inclusion
EDSS mean at each time point
Expanded Disability Status Scale (EDSS) mean score across participants at each scheduled assessment; EDSS assessed per routine clinical practice.
Time frame: Baseline (T0) and Months 6 (T1), 12 (T2), 18 (T3), 24 (T4), 30 (T5), 36 (T6)
EDSS mean change from baseline
Mean change in EDSS from baseline (T0) to each follow-up time point; change calculated as EDSS at time point minus baseline EDSS.
Time frame: Months 6 (T1), 12 (T2), 18 (T3), 24 (T4), 30 (T5), 36 (T6)
All-cause hospitalizations
Number of patients with ≥1 hospitalization from any cause during follow-up; counts summarized overall and by hospitalization cause categories.
Time frame: From inclusion (T0) through Month 36 (T6)
Median cumulative duration of hospitalizations
Median cumulative number of days spent hospitalized per patient during follow-up; calculated across all hospitalizations per participant.
Time frame: From inclusion (T0) through Month 36 (T6)
NMOSD-related hospitalizations
Number of patients with ≥1 hospitalization due to NMOSD during follow-up; NMOSD-related as documented in medical records.
Time frame: From inclusion (T0) through Month 36 (T6)
Median cumulative duration of NMOSD-related hospitalizations
Median cumulative number of days spent hospitalized per patient for NMOSD-related causes during follow-up.
Time frame: From inclusion (T0) through Month 36 (T6)
Patients with physician-reported relapse(s)
Number of patients with ≥1 and \>1 physician-reported NMOSD relapse during follow-up; relapses defined per protocol and documented by clinician assessment.
Time frame: From inclusion (T0) through Month 36 (T6)
Patients with severe relapse(s)
Number of patients with ≥1 and \>1 severe NMOSD relapse during follow-up; severe relapse defined as EDSS increase ≥2.0 points from baseline (for myelitis) or major OSIS exacerbation, per protocol.
Time frame: From inclusion (T0) through Month 36 (T6)
Annualized relapse rate (ARR)
ARR calculated as total number of physician-reported relapses divided by person-years observed during follow-up for each patient, summarized at the cohort level.
Time frame: From inclusion (T0) through Month 36 (T6)
Median time to first physician-reported relapse
Time from inclusion (T0) to first physician-reported NMOSD relapse; analyzed using Kaplan-Meier methods with censoring at Month 36 or withdrawal.
Time frame: From inclusion (T0) to first relapse event, up to Month 36 (T6)