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

Journal Article (Journal Article;Multicenter Study)

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.

Full Text

Duke Authors

Cited Authors

  • Howard, JF; Barohn, RJ; Cutter, GR; Freimer, M; Juel, VC; Mozaffar, T; Mellion, ML; Benatar, MG; Farrugia, ME; Wang, JJ; Malhotra, SS; Kissel, JT; MG Study Group,

Published Date

  • July 2013

Published In

Volume / Issue

  • 48 / 1

Start / End Page

  • 76 - 84

PubMed ID

  • 23512355

Electronic International Standard Serial Number (EISSN)

  • 1097-4598

Digital Object Identifier (DOI)

  • 10.1002/mus.23839


  • eng

Conference Location

  • United States