Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension
Mantegazza, Renato (Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.) (Milà, Itàlia))
Wolfe, Gil I. (Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.) (Milà, Itàlia))
Muppidi, Srikanth (Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.) (Milà, Itàlia))
Wiendl, Heinz (Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.) (Milà, Itàlia))
Fujita, Kenji P. (Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.) (Milà, Itàlia))
O'Brien, Fanny L. (Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.) (Milà, Itàlia))
Booth, Heather D.E. (Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.) (Milà, Itàlia))
Howard, James F (Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.) (Milà, Itàlia))
Illa, Isabel (Institut d'Investigació Biomèdica Sant Pau)
Cortés-Vicente, Elena (Institut d'Investigació Biomèdica Sant Pau)
Diaz-Manera, Jordi (Institut d'Investigació Biomèdica Sant Pau)
Querol, Luis (Institut d'Investigació Biomèdica Sant Pau)
Rojas-Garcia, Ricard (Institut d'Investigació Biomèdica Sant Pau)
Vidal-Fernández, Núria (Institut d'Investigació Biomèdica Sant Pau)

Date:	2021
Abstract:	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.
Note:	Altres ajuts: Alexion Pharmaceuticals.
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Subject:	Adult ; Antibodies, Monoclonal, Humanized ; Complement Inactivating Agents ; Female ; Humans ; Male ; Middle Aged ; Myasthenia Gravis ; Treatment Outcome
Published in:	Neurology, Vol. 96 Núm. 4 (january 2021) , p. e610-e618, ISSN 1526-632X

DOI: 10.1212/WNL.0000000000011207
PMID: 33229455

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