Format

Send to

Choose Destination
Muscle Nerve. 2019 Feb 14. doi: 10.1002/mus.26447. [Epub ahead of print]

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

Author information

1
Department of Neurology and Neurosciences, Stanford University School of Medicine, Stanford, California, USA.
2
Department of Neurology, Hanamaki General Hospital, Hanamaki, Japan.
3
Department of Neurology, University of Miami Miller School of Medicine, Miami, Florida, USA.
4
Department of Neurology, International University of Health and Welfare, Narita, Japan.
5
Department of Neurology, University of Kansas Medical Center, Kansas City, Kansas, USA.
6
Neurology Department, Hospital Sant Pau, Autonomous University of Barcelona, Barcelona, Spain.
7
Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Spain.
8
Queen Elizabeth Neuroscience Centre and Wellcome Trust Clinical Research Facility, University Hospital Birmingham, Birmingham, United Kingdom.
9
Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.
10
Department of Neurology, University of Virginia Health System, Charlottesville, Virginia, USA.
11
Department of Neurology, The Ohio State University, Columbus, Ohio, USA.
12
Department of Neurology, Yale School of Medicine, Yale University, New Haven, Connecticut, USA.
13
Department of Neurology, Aarhus University Hospital, Aarhus, Denmark.
14
Department of Neurology, Bellvitge University Hospital, Barcelona, Spain.
15
Neuromuscular Reference Centre, Ghent University Hospital, Ghent, Belgium.
16
Department of Neurology, University of South Florida, Tampa, Florida, USA.
17
Foundation of the Carlo Besta Neurological Institute, IRCSS, Milan, Italy.
18
Alexion Pharmaceuticals, Boston, Massachusetts, USA.
19
Department of Neurology, University of North Carolina, Chapel Hill, North Carolina, USA.

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

PMID:
30767274
DOI:
10.1002/mus.26447

Supplemental Content

Full text links

Icon for Wiley
Loading ...
Support Center