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

Publication ,  Journal Article
Howard, JF; Barohn, RJ; Cutter, GR; Freimer, M; Juel, VC; Mozaffar, T; Mellion, ML; Benatar, MG; Farrugia, ME; Wang, JJ; Malhotra, SS ...
Published in: Muscle Nerve
July 2013

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.

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Published In

Muscle Nerve

DOI

EISSN

1097-4598

Publication Date

July 2013

Volume

48

Issue

1

Start / End Page

76 / 84

Location

United States

Related Subject Headings

  • Pilot Projects
  • Neurology & Neurosurgery
  • Myasthenia Gravis
  • Middle Aged
  • Male
  • Humans
  • Female
  • Double-Blind Method
  • Cross-Over Studies
  • Antibodies, Monoclonal, Humanized
 

Citation

APA
Chicago
ICMJE
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Howard, J. F., Barohn, R. J., Cutter, G. R., Freimer, M., Juel, V. C., Mozaffar, T., … MG Study Group, . (2013). A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Muscle Nerve, 48(1), 76–84. https://doi.org/10.1002/mus.23839
Howard, James F., Richard J. Barohn, Gary R. Cutter, Miriam Freimer, Vern C. Juel, Tahseen Mozaffar, Michelle L. Mellion, et al. “A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis.Muscle Nerve 48, no. 1 (July 2013): 76–84. https://doi.org/10.1002/mus.23839.
Howard JF, Barohn RJ, Cutter GR, Freimer M, Juel VC, Mozaffar T, et al. A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Muscle Nerve. 2013 Jul;48(1):76–84.
Howard, James F., et al. “A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis.Muscle Nerve, vol. 48, no. 1, July 2013, pp. 76–84. Pubmed, doi:10.1002/mus.23839.
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. A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Muscle Nerve. 2013 Jul;48(1):76–84.
Journal cover image

Published In

Muscle Nerve

DOI

EISSN

1097-4598

Publication Date

July 2013

Volume

48

Issue

1

Start / End Page

76 / 84

Location

United States

Related Subject Headings

  • Pilot Projects
  • Neurology & Neurosurgery
  • Myasthenia Gravis
  • Middle Aged
  • Male
  • Humans
  • Female
  • Double-Blind Method
  • Cross-Over Studies
  • Antibodies, Monoclonal, Humanized