Long-term safety and efficacy of eculizumab in generalized myasthenia gravis.
Document Type
Article
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.
Medical Subject Headings
Activities of Daily Living; Adult; Angioedema; Antibodies, Monoclonal, Humanized; Aspergillosis; Complement Inactivating Agents; Disease Progression; Female; Heart Diseases; Humans; Injection Site Reaction; Longitudinal Studies; Male; Meningococcal Infections; Meningococcal Vaccines; Middle Aged; Muscle Strength; Myasthenia Gravis; Quality of Life; Treatment Outcome
Publication Date
7-1-2019
Publication Title
Muscle & nerve
ISSN
1097-4598
Volume
60
Issue
1
First Page
14
Last Page
24
PubMed ID
30767274
Digital Object Identifier (DOI)
10.1002/mus.26447
Recommended Citation
Muppidi, Srikanth; Utsugisawa, Kimiaki; Benatar, Michael; Murai, Hiroyuki; Barohn, Richard J; Illa, Isabel; Jacob, Saiju; Vissing, John; Burns, Ted M; Kissel, John T; Nowak, Richard J; Andersen, Henning; Casasnovas, Carlos; de Bleecker, Jan L; Vu, Tuan H; Mantegazza, Renato; O'Brien, Fanny L; Wang, Jing Jing; Fujita, Kenji P; and Howard, James F, "Long-term safety and efficacy of eculizumab in generalized myasthenia gravis." (2019). Neurology. 1678.
https://scholar.barrowneuro.org/neurology/1678