Long-term safety and efficacy of eculizumab in generalized myasthenia gravis

Muppidi S., Utsugisawa K., Benatar M., Murai H., Barohn R. J., Illa I., ...More

Muscle and Nerve, vol.60, no.1, pp.14-24, 2019 (SCI-Expanded) identifier identifier

  • Publication Type: Article / Article
  • Volume: 60 Issue: 1
  • Publication Date: 2019
  • Doi Number: 10.1002/mus.26447
  • Journal Name: Muscle and Nerve
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Page Numbers: pp.14-24
  • Keywords: eculizumab, MG-ADL, MG-QOL15, MGC, myasthenia gravis, QMG
  • Hacettepe University Affiliated: Yes

Abstract

© 2019 The Authors. Muscle & Nerve published by Wiley Periodicals, Inc.Introduction: Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of an open-label extension of REGAIN, evaluating eculizumab's long-term safety and efficacy. Methods: Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients. Results: The safety profile of eculizumab was consistent with REGAIN; no cases of meningococcal infection were reported during the interim analysis period. Myasthenia gravis exacerbation rate was reduced by 75% from the year before REGAIN (P < 0.0001). Improvements with eculizumab in activities of daily living, muscle strength, functional ability, and quality of life in REGAIN were maintained through 3 years; 56% of patients achieved minimal manifestations or pharmacological remission. Patients who had received placebo during REGAIN experienced rapid and sustained improvements during open-label eculizumab (P < 0.0001). Discussion: These findings provide evidence for the long-term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019.