Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension.

Autor:	Mantegazza R; From 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., Wolfe GI; From 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., Muppidi S; From 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., Wiendl H; From 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., Fujita KP; From 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., O'Brien FL; From 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., Booth HDE; From 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., Howard JF Jr; From 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. howardj@neurology.unc.edu.
Jazyk:	angličtina
Zdroj:	Neurology [Neurology] 2021 Jan 26; Vol. 96 (4), pp. e610-e618. Date of Electronic Publication: 2020 Nov 23.
DOI:	10.1212/WNL.0000000000011207
Abstrakt:	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. (Copyright © 2020 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology.)
Databáze:	MEDLINE
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