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 study

Howard J. F., Utsugisawa K., Benatar M., Murai H., Barohn R. J., Illa I., ...More

LANCET NEUROLOGY, vol.16, no.12, pp.976-986, 2017 (SCI-Expanded) identifier identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 16 Issue: 12
  • Publication Date: 2017
  • Doi Number: 10.1016/s1474-4422(17)30369-1
  • Journal Name: LANCET NEUROLOGY
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Page Numbers: pp.976-986
  • Karadeniz Technical University Affiliated: Yes

Abstract

Background Complement is likely to have a role in refractory generalised myasthenia gravis, but no approved therapies specifically target this system. Results from a phase 2 study suggested that eculizumab, a terminal complement inhibitor, produced clinically meaningful improvements in patients with anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis. We further assessed the efficacy and safety of eculizumab in this patient population in a phase 3 trial.