Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension
Por:
Mantegazza, R, Wolfe, GI, Muppidi, S, Wiendl, H, Fujita, KP, O'Brien, FL, Booth, HDE, Howard, JF, Illa I., Cortés-Vicente E., Díaz-Manera J., Querol L.A., Rojas-García R., Vidal-Fernandez N., REGAIN Study Group
Publicada:
26 ene 2021
Resumen:
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.
Filiaciones:
Mantegazza, R:
Fdn IRCCS Ist Neurol Carlo Besta, Milan, Italy
Wolfe, GI:
Univ Buffalo State Univ New York, Jacobs Sch Med & Biomed Sci, Dept Neurol, Buffalo, NY USA
Muppidi, S:
Stanford Univ, Sch Med, Dept Neurol & Neurol Sci, Palo Alto, CA 94304 USA
Wiendl, H:
Univ Munster, Dept Neurol, Munster, Germany
Fujita, KP:
Alnylam Pharmaceut, Cambridge, MA USA
Alex Pharmaceut, Boston, MA USA
O'Brien, FL:
Alex Pharmaceut, Boston, MA USA
Booth, HDE:
Oxford PharmaGenesis, Oxford, England
Howard, JF:
Univ N Carolina, Dept Neurol, Chapel Hill, NC 27515 USA
Illa I.:
Institut d’Investigació Biomèdica Sant Pau (IIB SANT PAU), Sant Quintí 77-79, 08041 Barcelona, Spain
Cortés-Vicente E.:
Institut d’Investigació Biomèdica Sant Pau (IIB SANT PAU), Sant Quintí 77-79, 08041 Barcelona, Spain
Díaz-Manera J.:
Institut d’Investigació Biomèdica Sant Pau (IIB SANT PAU), Sant Quintí 77-79, 08041 Barcelona, Spain
Querol L.A.:
Institut d’Investigació Biomèdica Sant Pau (IIB SANT PAU), Sant Quintí 77-79, 08041 Barcelona, Spain
Rojas-García R.:
Institut d’Investigació Biomèdica Sant Pau (IIB SANT PAU), Sant Quintí 77-79, 08041 Barcelona, Spain
Vidal-Fernandez N.:
Institut d’Investigació Biomèdica Sant Pau (IIB SANT PAU), Sant Quintí 77-79, 08041 Barcelona, Spain
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