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

Authors :: Muppidi S
Utsugisawa K
Benatar M
Murai H
Barohn RJ
Illa I
Jacob S
Vissing J
Burns TM
Kissel JT
Nowak RJ
Andersen H
Casasnovas C
de Bleecker JL
Vu TH
Mantegazza R
O'Brien FL
Wang JJ
Fujita KP
Howard JF Jr
Source :: Muscle & nerve [Muscle Nerve] 2019 Jul; Vol. 60 (1), pp. 14-24. Date of Electronic Publication: 2019 Mar 08.
Publication Year :: 2019
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.<br />Methods: Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients.<br />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).<br />Discussion: These findings provide evidence for the long-term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019.<br /> (© 2019 The Authors. Muscle & Nerve published by Wiley Periodicals, Inc.)