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