Neuromyelitis optica spectrum disorder is an autoimmune, inflammatory, and demyelinating disease of the central nervous system, primarily affecting the optic nerves, brainstem, and spinal cord. The discovery of AQP4-IgG established NMOSD as a distinct autoimmune entity, in contrast to multiple sclerosis, which is considered immune-mediated. The complement system is an essential mechanism in innate immunity and appears at the core of pathogenesis of NMOSD. Therapeutically, targeting the terminal complement cascade with C5 inhibitors, such as eculizumab and ravulizumab, is a promising strategy to prevent relapses and limit disability progression in AQP4-IgG–positive patients. This systematic review followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Included studies comprised randomized controlled trials (RCT) and observational studies evaluating C5 inhibitors in NMOSD. According to the PICOT framework: Participants: Adults with AQP4-IgG–positive NMOSD; Intervention: C5 inhibitors (eculizumab or ravulizumab); Comparison: Placebo; Outcomes: Efficacy (relapse risk, annualized relapse rate, and Expanded Disability Status Scale score changes); safety (adverse events, serious adverse events, and mortality); Timeframe: Studies published from 2019 to 2025. Evidence from RCT demonstrates a significantly reduced risk of relapses in patients receiving C5 inhibitors compared to placebo. Observational data from cohort studies further supports the clinical efficacy and safety of these agents in real-world settings.
Αντωνία Ν. Ζεγγίνη (Wed,) studied this question.