Title : Eculizumab efficacy and safety in patients with neuromyelitis optica spectrum disorder: A systematic review
Abstract:
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 eculizumab, a complement protein (C5) inhibitor has demonstrated efficacy in the clinical trials. In this Systematic Review (SR) we aim to summarize the current available evidence on Eculizumab efficacy and safety in patients with NMOSD.
Methods: PubMed, ScienceDirect, and Cochrane Central Register of Controlled Trials were searched using a pre-defined search strategy from inception until April-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.
Conclusion: 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.
Audience Take Away Notes:
- Recently many medications have been tested for its efficacy in Neuromyelitis Optica syndrome, our abstract is summarizing the currently evidence which may aid child neurologists in updating them regards the latest evidence of its use.
- It will help neurologists in tailoring their choice of medications for patients with NMO.
- Yes, this research that other faculty could use to expand their research or teaching.
- Yes, our abstract summarizes the current evidence on eculizumab in Neuromyelitis Optica syndrome which would save practitioners time looking for all clinical trials evidence on eculizumab.
- Yes, our abstract clearly shows the need of bigger and longer randomized clinical trial to support the current evidence.
