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



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.


Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients.


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).


These findings provide evidence for the long-term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019.





Regain Study Group, Humans, Meningococcal Infections, Aspergillosis, Myasthenia Gravis, Heart Diseases, Disease Progression, Meningococcal Vaccines, Treatment Outcome, Activities of Daily Living, Longitudinal Studies, Quality of Life, Adult, Middle Aged, Female, Male, Complement Inactivating Agents, Muscle Strength, Angioedema, Antibodies, Monoclonal, Humanized, Injection Site Reaction


Published Version (Please cite this version)


Publication Info

Muppidi, Srikanth, Kimiaki Utsugisawa, Michael Benatar, Hiroyuki Murai, Richard J Barohn, Isabel Illa, Saiju Jacob, John Vissing, et al. (2019). Long-term safety and efficacy of eculizumab in generalized myasthenia gravis. Muscle & nerve, 60(1). pp. 14–24. 10.1002/mus.26447 Retrieved from https://hdl.handle.net/10161/28445.

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