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P0695 - Burden of disease in patients with neuromyelitis optica spectrum disorder: insights from the CIRCLES study cohort (ID 802)
In patients with neuromyelitis optica spectrum disorder (NMOSD), relapses may result in cumulative neurological damage and disability. Disease burden may include pain, sensory, cognitive and visual impairment, bowel/bladder dysfunction, weakness or paralysis, and necessitate caregiver support. Prior to 2019, no therapies had received regulatory approval for NMOSD. Thus, off-label immunosuppressive therapies (IST) were commonly used for maintenance therapy. CIRCLES is a prospective, longitudinal, cross-sectional study of disease epidemiology and treatment in North American patients with NMOSD.
This study analyzed real-world disease burden in North American patients with NMOSD enrolled in the CIRCLES study from 2013 to 2019.
Of 629 CIRCLES participants, 523 (83.1%) with anti-aquaporin-4 immunoglobulin G-positive (AQP4-IgG+) NMOSD were assessed for disease burden, including vision loss, paralysis, annual relapse rate (ARR) relative to mobility level, and steroid side effects.
Baseline assessment of disability indicated at least partial (136/523, 26.0%) or complete (55/523, 10.5%) dependence on caregiver support. Among patients on off-label maintenance IST and having ≥60 days of on-study follow-up (n = 469), 136 (29.0%) experienced a total of 209 on-study relapses. The unadjusted ARR (95% confidence interval [CI]) by mobility level was 0.17 (0.14–0.20), 0.21 (0.16–0.27), and 0.24 (0.16–0.34) for independent, partially dependent, and completely dependent patients, respectively. Side effects from steroids were assessed in 429 respondents, of whom, 35 (8.2%) had gastroesophageal reflux disease, 30 (6.9%) had depression/anxiety, and 28 (6.5%) had osteoporosis. Of 77 patients with on-study relapses and vision assessment, 24 (31.2%) had vision loss (unadjusted ARR, 0.18 [CI, 0.13–0.24]; P > 0.05 vs. no relapses). Furthermore, of 129 patients with on-study relapses and paralysis assessment, 68 (52.7%) had partial or complete paralysis (unadjusted ARR, 0.23 [CI, 0.19–0.27]; P = 0.03 vs. no relapses).
NMOSD imposes significant disease burden, including vision loss and paralysis resulting in dependence on caregiver support in over one-third of patients. Despite the use of off-label maintenance ISTs, a substantial proportion of patients with NMOSD continue to experience relapses, disability, and neurological damage. These findings underscore the need for safe, effective, and well-tolerated treatments for preventing relapses.