Efficacy and safety of eculizumab in Guillain-Barré syndrome: a meta-analysis of randomized controlled trials

Abstract

Background: Guillain-Barré syndrome (GBS) is an acute, immune-mediated disorder of the peripheral nervous system, often leading to significant morbidity and, in severe cases, mortality. Despite the availability of treatments such as intravenous immunoglobulin and plasma exchange, a substantial proportion of patients experience incomplete recovery. Eculizumab, a humanized monoclonal antibody targeting complement protein C5, has emerged as a potential therapeutic option aimed at mitigating complement-mediated nerve damage. Objective: This meta-analysis aims to evaluate the efficacy and safety of eculizumab in the treatment of Guillain-Barré syndrome (GBS). Methods: PubMed, EMBASE, and Cochrane Central databases were searched for randomized controlled trials (RCTs) comparing eculizumab with placebo for treating GBS. Statistical analysis was performed in R, using risk ratios (RR) with a 95% confidence interval (CI). Random-effects model was applied to all endpoints. Results: Three RCTs with 99 patients were included, of whom 65.7% received eculizumab. The pooled analysis did not find significant variations between eculizumab and placebo regarding an improvement of 1≥ GBS score (RR 0.90; 95%CI 0.54-1.49, P>0.05). There was no statistical difference in Rasch-built Overall Disability Scale score (R-ODS) between patients receiving the monoclonal antibody and placebo (RR 0.99; 95%CI 0.63-1.55, P>0.05). The proportion of patients with Hughes FG score ≤2 was similar among groups (RR 1.06; 95%CI 0.64-1.77, P>0.05). There was not an association between a clinically relevant improvement in Overall Neuropathy Limitations Scale (ONLS) score and the treatment with eculizumab when compared to placebo (RR 0.93; 95%CI 0.63-1.31, P>0.05). The number of patients requiring ventilation support was similar between eculizumab and placebo groups (RR 1.06; 95%CI 0.12-9.60, P>0.05). Conclusion: This meta-analysis suggests that eculizumab may not offer a promising therapeutic option for patients with Guillain-Barré syndrome. Even though pathogenesis studies on GBS have identified the terminal component of complement cascade as a key disease mediator and therapeutic target, individual studies demonstrating potential benefits in reducing disease severity and improving recovery rates are limited, with significant heterogeneity observed across studies in terms of efficacy outcomes and safety profiles.