Myasthenie.com

Muscle Nerve. 2021 Sep 30. doi: 10.1002/mus.27422. Online ahead of print.

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 previously received rituximab (placebo, 7; eculizumab, 7). In the previous-rituximab group, 57% had used ≥4 other immunosuppressants vs 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 [standard error of the mean] -4.4 [1.0], n = 9, vs -4.6 [0.3], n = 67; difference, 0.2 [95% confidence interval: -1.88, 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 previously received rituximab treatment.

PMID:34590717 | DOI:10.1002/mus.27422


Source: https://pubmed.ncbi.nlm.nih.gov/3459071 ... 3&v=2.15.0