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

Srikanth Muppidi; Kimiaki Utsugisawa; Michael Benatar; Hiroyuki Murai; Richard J Barohn; Isabel Illa; Saiju Jacob; John Vissing; Ted M Burns; John T Kissel; Richard J Nowak; Henning Andersen; Carlos Casasnovas; Jan L de Bleecker; Tuan H Vu; Renato Mantegazza; Fanny L O'Brien; Jing Jing Wang; Kenji P Fujita; James F Howard Jr; Regain Study Group

doi:10.1002/mus.26447

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

Journal article

Open access

Peer reviewed

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

Srikanth Muppidi, Kimiaki Utsugisawa, Michael Benatar, Hiroyuki Murai, Richard J Barohn, Isabel Illa, Saiju Jacob, John Vissing, Ted M Burns, John T Kissel, …

Muscle & nerve, Vol.60(1), pp.14-24

07/2019

DOI: 10.1002/mus.26447

PMCID: PMC6619057

PMID: 30767274

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Abstract

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.

Quality of Life

Meningococcal Infections - epidemiology

Meningococcal Vaccines - therapeutic use

Humans

Middle Aged

Male

Aspergillosis - epidemiology

Activities of Daily Living

Aspergillosis - etiology

Heart Diseases - epidemiology

Meningococcal Infections - etiology

Adult

Female

Heart Diseases - chemically induced

Injection Site Reaction - epidemiology

Muscle Strength

Angioedema - epidemiology

Antibodies, Monoclonal, Humanized - therapeutic use

Injection Site Reaction - etiology

Meningococcal Infections - prevention & control

Treatment Outcome

Disease Progression

Myasthenia Gravis - physiopathology

Complement Inactivating Agents - therapeutic use

Angioedema - chemically induced

Longitudinal Studies

Myasthenia Gravis - drug therapy

Details

Title: Subtitle: Long-term safety and efficacy of eculizumab in generalized myasthenia gravis
Creators: Srikanth Muppidi - Department of Neurology and Neurosciences, Stanford University School of Medicine, Stanford, California, USA
Kimiaki Utsugisawa - Department of Neurology, Hanamaki General Hospital, Hanamaki, Japan
Michael Benatar - Department of Neurology, University of Miami Miller School of Medicine, Miami, Florida, USA
Hiroyuki Murai - Department of Neurology, International University of Health and Welfare, Narita, Japan
Richard J Barohn - Department of Neurology, University of Kansas Medical Center, Kansas City, Kansas, USA
Isabel Illa - Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Spain
Saiju Jacob - Queen Elizabeth Neuroscience Centre and Wellcome Trust Clinical Research Facility, University Hospital Birmingham, Birmingham, United Kingdom
John Vissing - Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark
Ted M Burns - Department of Neurology, University of Virginia Health System, Charlottesville, Virginia, USA
John T Kissel - Department of Neurology, The Ohio State University, Columbus, Ohio, USA
Richard J Nowak - Department of Neurology, Yale School of Medicine, Yale University, New Haven, Connecticut, USA
Henning Andersen - Department of Neurology, Aarhus University Hospital, Aarhus, Denmark
Carlos Casasnovas - Department of Neurology, Bellvitge University Hospital, Barcelona, Spain
Jan L de Bleecker - Neuromuscular Reference Centre, Ghent University Hospital, Ghent, Belgium
Tuan H Vu - Department of Neurology, University of South Florida, Tampa, Florida, USA
Renato Mantegazza - Foundation of the Carlo Besta Neurological Institute, IRCSS, Milan, Italy
Fanny L O'Brien - Alexion Pharmaceuticals, Boston, Massachusetts, USA
Jing Jing Wang - Alexion Pharmaceuticals, Boston, Massachusetts, USA
Kenji P Fujita - Alexion Pharmaceuticals, Boston, Massachusetts, USA
James F Howard Jr - Department of Neurology, University of North Carolina, Chapel Hill, North Carolina, USA
Regain Study Group
Contributors: Ludwig Gutmann (Contributor) - University of Iowa, Neurology
Resource Type: Journal article
Publication Details: Muscle & nerve, Vol.60(1), pp.14-24
DOI: 10.1002/mus.26447
PMID: 30767274
PMCID: PMC6619057
NLM abbreviation: Muscle Nerve
ISSN: 0148-639X
eISSN: 1097-4598
Publisher: United States
Grant note: UL1 TR001863 / NCATS NIH HHS
Language: English
Date published: 07/2019
Academic Unit: Neurology
Record Identifier: 9984013108902771

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