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Clinical Trial
. 2021 Dec;64(6):662-669.
doi: 10.1002/mus.27422. Epub 2021 Oct 14.

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

Zaeem A Siddiqi  1 , Richard J Nowak  2 , Tahseen Mozaffar  3 , Fanny O'Brien  4 , Marcus Yountz  4 , Francesco Patti  5 ; REGAIN Study Group
Collaborators, Affiliations
Clinical Trial

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

Zaeem A Siddiqi et al. Muscle Nerve. 2021 Dec.

Abstract

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.

Keywords: acetylcholine receptor; eculizumab; myasthenia gravis; refractory; rituximab.

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References

REFERENCES

    1. Lindstrom JM, Seybold ME, Lennon VA, Whittingham S, Duane DD. Antibody to acetylcholine receptor in myasthenia gravis. Prevalence, clinical correlates, and diagnostic value. Neurology. 1976;26(11):1054-1059.
    1. Mantegazza R, Pareyson D, Baggi F, et al. Anti AChR antibody: relevance to diagnosis and clinical aspects of myasthenia gravis. Ital J Neurol Sci. 1988;9(2):141-145.
    1. Oh SJ, Kim DE, Kuruoglu R, Bradley RJ, Dwyer D. Diagnostic sensitivity of the laboratory tests in myasthenia gravis. Muscle Nerve. 1992;15(6):720-724.
    1. Somnier FE. Clinical implementation of anti-acetylcholine receptor antibodies. J Neurol Neurosurg Psychiatry. 1993;56(5):496-504.
    1. Vincent A, McConville J, Farrugia ME, et al. Antibodies in myasthenia gravis and related disorders. Ann N Y Acad Sci. 2003;998:324-335.

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