A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis

James F. Howard, Richard J. Barohn, Gary R. Cutter, Miriam Freimer, Vern C. Juel, Tahseen Mozaffar, Michelle L. Mellion, Michael G. Benatar, Maria Elena Farrugia, Jing Jing Wang, Suneil S. Malhotra, John T. Kissel

Research output: Contribution to journalArticlepeer-review

128 Scopus citations

Abstract

Introduction: Complement activation at the neuromuscular junction is a primary cause of acetylcholine receptor loss and failure of neuromuscular transmission in myasthenia gravis (MG). Eculizumab, a humanized monoclonal antibody, blocks the formation of terminal complement complex by specifically preventing the enzymatic cleavage of complement 5 (C5). Methods: This study was a randomized, double-blind, placebo-controlled, crossover trial involving 14 patients with severe, refractory generalized MG (gMG). Results: Six of 7 patients treated with eculizumab for 16 weeks (86%) achieved the primary endpoint of a 3-point reduction in the quantitative myasthenia gravis (QMG) score. Examining both treatment periods, the overall change in mean QMG total score was significantly different between eculizumab and placebo (P=0.0144). After assessing data obtained from all visits, the overall change in mean QMG total score from baseline was found to be significantly different between eculizumab and placebo (P<0.0001). Eculizumab was well tolerated. Conclusion: The data suggest that eculizumab may have a role in treating severe, refractory MG.

Original languageEnglish (US)
Pages (from-to)76-84
Number of pages9
JournalMuscle and Nerve
Volume48
Issue number1
DOIs
StatePublished - Jul 1 2013

Keywords

  • Complement
  • Eculizumab
  • Myasthenia gravis
  • Phase II
  • Pilot study

ASJC Scopus subject areas

  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)
  • Physiology

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