Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study
Por:
Siddiqi, ZA, Nowak, RJ, Mozaffar, T, O'Brien, F, Yountz, M, Patti, F, Illa I., Cortés-Vicente E., Díaz-Manera J., Querol L.A., Rojas-García R., Vidal-Fernandez N., Nye, Joan L.
Publicada:
1 dic 2021
Ahead of Print:
1 oct 2021
Resumen:
Introduction/Aims Individuals with refractory generalized myasthenia gravis (gMG) who have a history of rituximab use and experience persistent symptoms represent a population with unmet treatment needs. The aim of this analysis was to evaluate the efficacy and safety of eculizumab in patients with refractory anti-acetylcholine receptor antibody-positive (AChR(+)) gMG previously treated with rituximab. Methods This post hoc subgroup analysis of the phase 3 REGAIN study (NCT01997229) and its open-label extension (OLE; NCT02301624) compared baseline characteristics, safety, and response to eculizumab in participants who had previously received rituximab with those who had not. Rituximab use was not permitted within the 6 months before screening or during REGAIN/OLE. Results Of 125 REGAIN participants, 14 had received rituximab previously (7 received placebo and 7 received eculizumab). In the previous-rituximab group, 57% had used at least four other immunosuppressants compared with 16% in the no-previous-rituximab group. Myasthenia Gravis Activities of Daily Living total scores from eculizumab baseline to week 130 of eculizumab treatment improved in both the previous-rituximab and no-previous-rituximab groups (least-squares mean -4.4, standard error of the mean [SEM] 1.0 [n = 9] and least-squares mean -4.6, SEM 0.3 [n = 67], respectively; difference = 0.2, 95% confidence interval -1.88 to 2.22). In addition, in both groups, most patients who were treated with eculizumab for 130 weeks achieved a Myasthenia Gravis Foundation of America post-intervention status of minimal manifestations (66.7% and 65.0%, respectively). The eculizumab safety profile was similar between groups and consistent with its established profile. Discussion Eculizumab is an effective therapy for patients with refractory AChR(+) gMG, irrespective of whether they had received rituximab treatment previously.
Filiaciones:
Siddiqi, ZA:
Univ Alberta, Div Neurol, Dept Med, 7-112 Clin Sci Bldg,11350-83 Ave, Edmonton, AB T6G 2G3, Canada
Nowak, RJ:
Yale Univ, Sch Med, Dept Neurol, New Haven, CT 06510 USA
Mozaffar, T:
Univ Calif Irvine, Dept Neurol, Orange, CA 92668 USA
O'Brien, F:
Alex Pharmaceut Inc, Boston, MA USA
Yountz, M:
Alex Pharmaceut Inc, Boston, MA USA
Patti, F:
Univ Catania, Dept Med & Surg Sci & Adv Technol GF Ingrassia, Catania, Italy
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
Nye, Joan L.:
Yale School of Medicine, New Haven, United States
hybrid, Green Published
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