Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study

Zaeem A Siddiqi; Richard J Nowak; Tahseen Mozaffar; Fanny O'Brien; Marcus Yountz; Francesco Patti; REGAIN Study Group

doi:10.1002/mus.27422

$Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study$

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Open access

Peer reviewed

Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study

Zaeem A Siddiqi, Richard J Nowak, Tahseen Mozaffar, Fanny O'Brien, Marcus Yountz, Francesco Patti and REGAIN Study Group

Muscle & nerve, Vol.64(6), pp.662-669

12/2021

DOI: 10.1002/mus.27422

PMID: 34590717

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Abstract

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. 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. 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. Eculizumab is an effective therapy for patients with refractory AChR gMG, irrespective of whether they had received rituximab treatment previously.

Activities of Daily Living

Antibodies, Monoclonal, Humanized - adverse effects

Humans

Myasthenia Gravis - drug therapy

Rituximab - therapeutic use

Details

Title: Subtitle: Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study
Creators: Zaeem A Siddiqi - University of Alberta
Richard J Nowak - Department of Neurology, Yale University School of Medicine, Yale University, New Haven, Connecticut, USA
Tahseen Mozaffar - University of California, Irvine
Fanny O'Brien - Alexion Pharmaceuticals
Marcus Yountz - Alexion Pharmaceuticals
Francesco Patti - University of Catania
REGAIN Study Group
Contributors: Christopher S Nance (Contributor) - University of Iowa, Neurology
Ludwig Gutmann (Contributor) - University of Iowa, Neurology
Andrea Swenson (Contributor) - University of Iowa, Neurology
Resource Type: Journal article
Publication Details: Muscle & nerve, Vol.64(6), pp.662-669
DOI: 10.1002/mus.27422
PMID: 34590717
NLM abbreviation: Muscle Nerve
ISSN: 0148-639X
eISSN: 1097-4598
Grant note: DOI: 10.13039/100006396, name: Alexion Pharmaceuticals
Language: English
Date published: 12/2021
Academic Unit: Neurology
Record Identifier: 9984302211302771

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