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Pathogenesis studies on <jats:styled-content style=\"fixed-case\">GBS<\/jats:styled-content> have identified the terminal component of complement cascade as a key disease mediator and therapeutic target. We report the first use of terminal complement pathway inhibition with eculizumab in humans with <jats:styled-content style=\"fixed-case\">GBS<\/jats:styled-content>. In a randomised, double\u2010blind, placebo\u2010controlled trial, 28 subjects eligible on the basis of <jats:styled-content style=\"fixed-case\">GBS<\/jats:styled-content> disability grade of at least 3 were screened, of whom 8 (29%) were randomised. Five received eculizumab for 4 weeks, alongside standard <jats:styled-content style=\"fixed-case\">IVIg<\/jats:styled-content> treatment. The safety outcomes, monitored via adverse events capture, showed eculizumab to be well\u2010tolerated and safe when administered in conjunction with <jats:styled-content style=\"fixed-case\">IVIg<\/jats:styled-content>. Primary and secondary efficacy outcomes in the form of <jats:styled-content style=\"fixed-case\">GBS<\/jats:styled-content> disability scores (<jats:styled-content style=\"fixed-case\">GBS DS<\/jats:styled-content>), <jats:styled-content style=\"fixed-case\">MRC<\/jats:styled-content> sum scores, Rasch overall disability scores, and overall neuropathy limitation scores are reported descriptively. For the primary efficacy outcome at 4 weeks after recruitment, two of two placebo\u2010 and two of five eculizumab\u2010treated subjects had improved by one or more grades on the <jats:styled-content style=\"fixed-case\">GBS DS<\/jats:styled-content>. Although the small sample size precludes a statistically meaningful analysis, these pilot data indicate further studies on complement inhibition in <jats:styled-content style=\"fixed-case\">GBS<\/jats:styled-content> are warranted.<\/jats:p>","DOI":"10.1111\/jns.12194","type":"journal-article","created":{"date-parts":[[2016,11,1]],"date-time":"2016-11-01T15:47:00Z","timestamp":1478015220000},"page":"4-12","source":"Crossref","is-referenced-by-count":88,"title":["Inhibition of complement in Guillain\u2010Barr\u00e9 syndrome: the <scp>ICA\u2010GBS<\/scp> study"],"prefix":"10.1111","volume":"22","author":[{"given":"Amy I.","family":"Davidson","sequence":"first","affiliation":[{"name":"Institute of Infection, Immunity and Inflammation College of Medical, Veterinary and Life Sciences, University of Glasgow  Glasgow Scotland"},{"name":"Department of Neurology, Institute of Neurological Sciences Queen Elizabeth University Hospital  Glasgow 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Scotland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Arup","family":"Mallik","sequence":"additional","affiliation":[{"name":"Department of Clinical Neurophysiology, Institute of Neurological Sciences Queen Elizabeth University Hospital  Glasgow Scotland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"James","family":"Overell","sequence":"additional","affiliation":[{"name":"Department of Neurology, Institute of Neurological Sciences Queen Elizabeth University Hospital  Glasgow Scotland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael P.","family":"Lunn","sequence":"additional","affiliation":[{"name":"Department of Neurology National Hospital for Neurology and Neurosurgery  London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alex","family":"McConnachie","sequence":"additional","affiliation":[{"name":"Robertson Centre for Biostatistics University of Glasgow  Glasgow Scotland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pieter","family":"van Doorn","sequence":"additional","affiliation":[{"name":"Department of Neurology Erasmus MC  Rotterdam The Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hugh J.","family":"Willison","sequence":"additional","affiliation":[{"name":"Institute of Infection, Immunity and Inflammation College of Medical, Veterinary and Life Sciences, University of Glasgow  Glasgow Scotland"},{"name":"Department of Neurology, Institute of Neurological Sciences Queen Elizabeth University Hospital  Glasgow 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