2026 American Academy of Neurology Abstract Website

Objective:

In this Systematic Review (SR) we aim to summarize the current available evidence on Eculizumab efficacy and safety in patients with NMOSD.

Background:

Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare autoimmune neuro-inflammatory disorder characterized by recurrent attacks of demyelination affects the optic nerve and spinal cord with often incomplete recovery. Numerous medications have emerged as a potential theraputic choices in NMOSD of these eculizumabe, a complement protein (C5) inhibitor has demonstrated efficacy in the clinical trials.

Design/Methods:

PubMed, ScienceDirect, and Cochrane Central Register of Controlled Trials were searched using a pre-defined search strategy from inception until June 2024 following the standard method of Cochrane Handbook and the PRISMA-statement guidelines. All studies studying Eculizumab in NMOSD patients with AQP4+IgG4 were included, case reports and case series got excluded.

Results:

Three Studies included in this SR with total of 225 participants of these one is a blinded randomized placebo-controlled trial, one is a pilot clinical trial and the third is post-marketing observational study. All the 3 studies demonstrated positive results in decreasing annual relapse rate. Included studies characteristic, outcomes and adverse events summarized in table-1 and table-2. Adverse events were reported in most of the participants (92% in eculizumab treatment group) of these the majority were non serious events; viral infections, headache, nausea and diarrhea were the most frequently encountered. And one reported mortality case in the randomized control trial.

Conclusions:

This systematic review shows that eculizumab is potentially effective in lowering relapse rate and improving levels of disability and quality of life in NMOSD Patients with AQP4+IgG4, Bigger and longer real-world studies are needed to establish the long-term efficacy and safety of eculizumab.