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Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its
Renato Mantegazza1, Gil I Wolfe1, Srikanth Muppidi1
1From the Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.), Milan, Italy; Department of Neurology (G.I.W.), Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, State University of New York; Department of Neurology and Neurological Sciences (S.M.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (H.W.), University of Münster, Germany; Alnylam Pharmaceuticals (K.P.F.), Cambridge, MA; Alexion Pharmaceuticals (F.L.O.), Boston, MA; Oxford PharmaGenesis (H.D.E.B.), UK; and Department of Neurology (J.F.H.), University of North Carolina, Chapel Hill. K.P.F. was formerly affiliated with Alexion Pharmaceuticals, Boston, MA.
Objective:
To evaluate whether eculizumab helps patients with anti-acetylcholine receptor-positive (AChR+) refractory generalized myasthenia gravis (gMG) achieve the Myasthenia Gravis Foundation of America (MGFA) post-intervention status of minimal manifestations (MM), we assessed patients' status throughout REGAIN (Safety and Efficacy of Eculizumab in AChR+ Refractory Generalized Myasthenia Gravis) and its open-label extension.
Methods:
Patients who completed the REGAIN randomized controlled trial and continued into the open-label extension were included in this tertiary endpoint analysis. Patients were assessed for the MGFA post-intervention status of improved, unchanged, worse, MM, and pharmacologic remission at defined time points during REGAIN and through week 130 of the open-label study.
Results:
A total of 117 patients completed REGAIN and continued into the open-label study (eculizumab/eculizumab: 56; placebo/eculizumab: 61). At week 26 of REGAIN, more eculizumab-treated patients than placebo-treated patients achieved a status of improved (60.7% vs 41.7%) or MM (25.0% vs 13.3%; common OR: 2.3; 95% CI: 1.1-4.5). After 130 weeks of eculizumab treatment, 88.0% of patients achieved improved status and 57.3% of patients achieved MM status. The safety profile of eculizumab was consistent with its known profile and no new safety signals were detected.
Conclusion:
Eculizumab led to rapid and sustained achievement of MM in patients with AChR+ refractory gMG. These findings support the use of eculizumab in this previously difficult-to-treat patient population.
Clinicaltrialsgov Identifier:
REGAIN, NCT01997229; REGAIN open-label extension, NCT02301624.
Classification Of Evidence:
This study provides Class II evidence that, after 26 weeks of eculizumab treatment, 25.0% of adults with AChR+ refractory gMG achieved MM, compared with 13.3% who received placebo.
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