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

Muscle Nerve. 2019 Jul;60(1):14-24. doi: 10.1002/mus.26447. Epub 2019 Mar 8.

Abstract

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.

Keywords: MG-ADL; MG-QOL15; MGC; QMG; eculizumab; myasthenia gravis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

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

Substances

  • Antibodies, Monoclonal, Humanized
  • Complement Inactivating Agents
  • Meningococcal Vaccines
  • eculizumab