TY - JOUR
T1 - Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab
T2 - subgroup analysis of REGAIN and its extension study
AU - The REGAIN Study Group
AU - Siddiqi, Zaeem A.
AU - Nowak, Richard J.
AU - Mozaffar, Tahseen
AU - O'Brien, Fanny
AU - Yountz, Marcus
AU - Patti, Francesco
AU - Mazia, Claudio Gabriel
AU - Wilken, Miguel
AU - Wilken, Miguel
AU - Barroso, Fabio
AU - Saba, Juliet
AU - Rugiero, Marcelo
AU - Bettini, Mariela
AU - Chaves, Marcelo
AU - Vidal, Gonzalo
AU - Garcia, Alejandra Dalila
AU - De Bleecker, Jan
AU - Van den Abeele, Guy
AU - de Koning, Kathy
AU - De Mey, Katrien
AU - Mercelis, Rudy
AU - Mahieu, Délphine
AU - Wagemaekers, Linda
AU - Van Damme, Philip
AU - Depreitere, Annelies
AU - Schotte, Caroline
AU - Smetcoren, Charlotte
AU - Stevens, Olivier
AU - Van Daele, Sien
AU - Vandenbussche, Nicolas
AU - Vanhee, Annelies
AU - Verjans, Sarah
AU - Vynckier, Jan
AU - D'Hont, Ann
AU - Tilkin, Petra
AU - de Siqueira Carvalho, Alzira Alves
AU - Brockhausen, Igor Dias
AU - Feder, David
AU - Ambrosio, Daniel
AU - César, Pamela
AU - Melo, Ana Paula
AU - Ribeiro, Renata Martins
AU - Rocha, Rosana
AU - Rosa, Bruno Bezerra
AU - Veiga, Thabata
AU - da Silva, Luiz Augusto
AU - Engel, Murilo Santos
AU - Geraldo, Jordana Gonçalves
AU - da Penha Ananias Morita, Maria
AU - Bhavaraju-Sanka, Ratna
N1 - Publisher Copyright:
© 2021 Wiley Periodicals LLC.
PY - 2021/12
Y1 - 2021/12
N2 - 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.
AB - 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.
KW - acetylcholine receptor
KW - eculizumab
KW - myasthenia gravis
KW - refractory
KW - rituximab
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U2 - 10.1002/mus.27422
DO - 10.1002/mus.27422
M3 - Article
C2 - 34590717
AN - SCOPUS:85117104099
SN - 0148-639X
VL - 64
SP - 662
EP - 669
JO - Muscle and Nerve
JF - Muscle and Nerve
IS - 6
ER -