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

for the Regain Study Group

Research output: Contribution to journalArticle

4 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)14-24
Number of pages11
JournalMuscle and Nerve
Volume60
Issue number1
DOIs
StatePublished - Jul 1 2019

Fingerprint

Myasthenia Gravis
Safety
Meningococcal Infections
Muscle Strength
Activities of Daily Living
eculizumab
Placebos
Quality of Life
Demography
Pharmacology
Muscles
Antibodies

Keywords

  • eculizumab
  • MG-ADL
  • MG-QOL15
  • MGC
  • myasthenia gravis
  • QMG

ASJC Scopus subject areas

  • Physiology
  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)

Cite this

Long-term safety and efficacy of eculizumab in generalized myasthenia gravis. / for the Regain Study Group.

In: Muscle and Nerve, Vol. 60, No. 1, 01.07.2019, p. 14-24.

Research output: Contribution to journalArticle

for the Regain Study Group. / Long-term safety and efficacy of eculizumab in generalized myasthenia gravis. In: Muscle and Nerve. 2019 ; Vol. 60, No. 1. pp. 14-24.
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AU - Vissing, John

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AU - Schotte, Caroline

AU - Smetcoren, Charlotte

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