A systematic review and meta-analysis of the efficacy and safety of eculizumab in AQP4-IgG-positive neuromyelitis optica spectrum disorder.

In 30 seconds

This systematic review and meta-analysis assessed eculizumab efficacy and safety in people with AQP4-IgG-positive neuromyelitis optica spectrum disorder, including phase III randomised controlled trials. Compared with placebo or conventional immunosuppressive therapy, eculizumab was associated with a substantially lower risk of adjudicated relapse (HR = 0.07; 95% CI: 0.03-0.18; p < 0.001) and a lower annualized relapse rate. More than 95% of treated patients remained relapse-free during follow-up. EDSS scores generally remained stable, and the review described the safety profile as acceptable, while noting limited evidence on long-term safety.

Key findings

• Eculizumab reduced adjudicated relapse risk versus placebo or conventional immunosuppressive therapy (HR = 0.07; 95% CI: 0.03-0.18; p < 0.001).
• Annualized relapse rate was lower with eculizumab (RR = 0.15; 95% CI: 0.08-0.29; p < 0.001).
• More than 95% of patients receiving eculizumab remained relapse-free during follow-up.
• EDSS scores generally remained stable; the available evidence indicated an acceptable safety profile.

Why it matters

Relapse prevention is a central treatment goal in AQP4-IgG-positive NMOSD, and the pooled findings indicate that eculizumab can markedly reduce relapse outcomes in the studied trials. The reported stability of disability scores is also clinically relevant, although it does not establish long-term preservation of function. Safety and durability remain important considerations because follow-up and trial evidence were limited.

What to keep in mind

The review notes limited numbers of trials, small sample sizes, heterogeneous follow-up periods, and limited data on long-term safety and cost-effectiveness.