A randomized, double‐blind, placebo‐controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Issue 1 (30th April 2013)

• Record Type: Journal Article
• Authors: Howard, James F.; Barohn, Richard J.; Cutter, Gary R.; Freimer, Miriam; Juel, Vern C.; Mozaffar, Tahseen; Mellion, Michelle L.; Benatar, Michael G.; Farrugia, Maria Elena; Wang, Jing Jing; Malhotra, Suneil S.; Kissel, John T.
• Is Part Of: Muscle & nerve. Volume 48:Issue 1(2013:Jul.)
• Page Start: 76
• Page End: 84
• Publication Date: 2013-04-30
• Subjects: Neuromuscular diseases -- Periodicals; Muscles -- Periodicals; Nerves -- Periodicals; 616.74
• DOI: 10.1002/mus.23839
• Languages: English
• ISSNs: 0148-639X

Abstract

<abstract abstract-type="main"> <title>ABSTRACT</title> <p> <italic>Introduction</italic>: 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). <italic>Methods</italic>: This study was a randomized, double‐blind, placebo‐controlled, crossover trial involving 14 patients with severe, refractory generalized MG (gMG). <italic>Results</italic>: 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 (<italic>P</italic> = 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 (<italic>P</italic> &lt; 0.0001). Eculizumab was well tolerated. <italic>Conclusion</italic>: The data suggest that eculizumab may have a role in treating severe, refractory MG. Muscle Nerve, 2013</p> </abstract>