Is eculizumab an effective treatment for Guillain-Barre syndrome (GBS)?

Updated: Apr 21, 2020
  • Author: Michael T Andary, MD, MS; Chief Editor: Milton J Klein, DO, MBA  more...
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Answer

Other possible treatments modulating the immune system include complement inhibitors such as eculizumab. This has been shown to be effective in animal models of Miller-Fisher syndrome [134, 135] and to be safe in humans. [136, 137, 138] The Japanese Eculizumab Trial for GBS (JET-GBS), a prospective, multicenter, placebo-controlled, double-blind, randomized phase 2 study, reported eculizumab to be safe in severe cases of GBS. The investigators also found that at 24 weeks, 91.6% of patients on eculizumab could walk independently and 74% could run, compared with 71.9% and 18%, respectively, for those on placebo. Nonetheless, the report noted that "[t]he primary outcome measure did not reach the predefined response rate." Moreover, the study, which was designed to evaluate safety, was considered underpowered with regard to showing efficacy. [137, 138]


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