Eculizumab: a treatment option for myasthenia gravis?

Eculizumab is an expensive, humanised monoclonal antibody against the terminal complement protein C5. The drug was patented in 2007, and has since been used and approved for two rare diseases, paroxysmal nocturnal haemoglobulinuria and atypical haemolytic ureamic syndrome. Additionally, eculizumab has been used off-label, which constitutes up to a half of its use in some countries, 1 Ricklin D Barratt-Due A Mollnes TE Complement in clinical medicine: clinical trials, case reports and therapy monitoring. Mol Immunol. 2017; 89: 10-21 Crossref PubMed Scopus (60) Google Scholar for at least 25 different conditions. Refractory and generalised myasthenia gravis with anti-acetylcholine receptor antibodies is a rare disease that involves the complement system and for which eculizumab has a potential effect. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre studyThe change in the MG-ADL score was not statistically significant between eculizumab and placebo, as measured by the worst-rank analysis. Eculizumab was well tolerated. The use of a worst-rank analytical approach proved to be an important limitation of this study since the secondary and sensitivity analyses results were inconsistent with the primary endpoint result; further research into the role of complement is needed. Full-Text PDF CorrectionsGilhus N E. Eculizumab: a treatment option for myasthenia gravis? Lancet Neurol 2017; 16: 947–48—The original title of this comment was “Eculizumab: a treatment option for mysthenia gravis?”. This title has been corrected to “Eculizumab: a treatment option for myasthenia gravis?”. This correction has been made to the online version as of Dec 15, 2017. Full-Text PDF