Inhibition of complement in Guillain‐Barré syndrome: the ICA‐GBS study

Journal of the Peripheral Nervous System - Tập 22 Số 1 - Trang 4-12 - 2017
Amy Davidson1,2, Susan K. Halstead2, John Goodfellow1,2, Govind Chavada1, Arup K Mallik3, James Overell1, Michael P. Lunn4, Alex McConnachie5, P. van Doorn6, Hugh J. Willison1,2
1Department of Neurology, Institute of Neurological Sciences, Queen Elizabeth University Hospital, Glasgow, Scotland
2Institute of Infection, Immunity and Inflammation, College of Medical, Veterinary and Life Sciences, University of Glasgow, Glasgow, Scotland
3Department of Clinical Neurophysiology, Institute of Neurological Sciences, Queen Elizabeth University Hospital, Glasgow, Scotland
4Department of Neurology, National Hospital for Neurology and Neurosurgery, London, UK
5Robertson Centre for Biostatistics, University of Glasgow, Glasgow, Scotland
6Department of Neurology, Erasmus MC, Rotterdam, The Netherlands

Tóm tắt

AbstractThe 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.

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Journal of the Peripheral Nervous System

Tập 22 Số 1

4-12

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