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

REGAIN Study Group

Research output: Contribution to journalArticlepeer-review

56 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)14-24
Number of pages11
JournalMuscle & Nerve
Issue number1
Publication statusPublished - Jul 2019

Bibliographical note

© 2019 The Authors. Muscle & Nerve published by Wiley Periodicals, Inc.


  • Activities of Daily Living
  • Adult
  • Angioedema/chemically induced
  • Antibodies, Monoclonal, Humanized/therapeutic use
  • Aspergillosis/epidemiology
  • Complement Inactivating Agents/therapeutic use
  • Disease Progression
  • Female
  • Heart Diseases/chemically induced
  • Humans
  • Injection Site Reaction/epidemiology
  • Longitudinal Studies
  • Male
  • Meningococcal Infections/epidemiology
  • Meningococcal Vaccines/therapeutic use
  • Middle Aged
  • Muscle Strength
  • Myasthenia Gravis/drug therapy
  • Quality of Life
  • Treatment Outcome


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