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

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' sta...

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Detalles Bibliográficos
Autores: Mantegazza, Renato|||0000-0002-9810-5737, Wolfe, Gil I.|||0000-0003-1471-9617, Muppidi, Srikanth|||0000-0002-2773-2506, Wiendl, Heinz|||0000-0003-4310-3432, Fujita, Kenji P.|||0000-0003-4320-7557, O'Brien, Fanny L.|||0000-0002-5840-4089, Booth, Heather D.E.|||0000-0003-2915-5578, Howard, James F.|||0000-0002-7136-8617, Illa, Isabel|||0000-0002-2186-2684, Cortés-Vicente, Elena|||0000-0002-1428-1072, Diaz-Manera, Jordi|||0000-0003-2941-7988, Querol, Luis|||0000-0002-4289-8264, Rojas-Garcia, Ricard|||0000-0003-1411-5573, Vidal-Fernández, Núria|||0009-0008-3749-6448
Tipo de recurso: artículo
Fecha de publicación:2021
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:272120
Acceso en línea:https://ddd.uab.cat/record/272120
https://dx.doi.org/urn:doi:10.1212/WNL.0000000000011207
Access Level:acceso abierto
Palabra clave:Adult
Antibodies, Monoclonal, Humanized
Complement Inactivating Agents
Female
Humans
Male
Middle Aged
Myasthenia Gravis
Treatment Outcome
Descripción
Sumario: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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