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

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.

• Authors: 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
• Journal: Muscle & nerve (Muscle Nerve) Vol. 60 Issue 1 Pg. 14-24 (07 2019) ISSN: 1097-4598 [Electronic] United States
• PMID: 30767274 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2019 The Authors. Muscle & Nerve published by Wiley Periodicals, Inc.

Chemical References

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

Topics

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