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Inhibition of complement in Guillain‐Barré syndrome: the ICA‐GBS study
Author(s) -
Davidson Amy I.,
Halstead Susan K.,
Goodfellow John A.,
Chavada Govind,
Mallik Arup,
Overell James,
Lunn Michael P.,
McConnachie Alex,
Doorn Pieter,
Willison Hugh J.
Publication year - 2017
Publication title -
journal of the peripheral nervous system
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1
H-Index - 67
eISSN - 1529-8027
pISSN - 1085-9489
DOI - 10.1111/jns.12194
Subject(s) - eculizumab , medicine , placebo , clinical trial , complement system , complement (music) , adverse effect , antibody , immunology , phenotype , pathology , alternative medicine , biochemistry , chemistry , complementation , gene
The outcome of Guillain‐Barré syndrome ( GBS ) remains unchanged since plasma exchange and intravenous immunoglobulin ( IVIg ) were introduced over 20 years ago. Pathogenesis studies on GBS 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 GBS . In a randomised, double‐blind, placebo‐controlled trial, 28 subjects eligible on the basis of GBS disability grade of at least 3 were screened, of whom 8 (29%) were randomised. Five received eculizumab for 4 weeks, alongside standard IVIg treatment. The safety outcomes, monitored via adverse events capture, showed eculizumab to be well‐tolerated and safe when administered in conjunction with IVIg . Primary and secondary efficacy outcomes in the form of GBS disability scores ( GBS DS ), MRC 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‐ and two of five eculizumab‐treated subjects had improved by one or more grades on the GBS DS . Although the small sample size precludes a statistically meaningful analysis, these pilot data indicate further studies on complement inhibition in GBS are warranted.