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

<jats:title>Abstract</jats:title><jats:sec><jats:title>Introduction/Aims</jats:title><jats:p>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<jats:sup>+</jats:sup>) gMG previously treated with rituximab.</jats:p></jats:sec><jats:sec><jats:title>Methods</jats:title><jats:p>This <jats:italic>post hoc</jats:italic> 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.</jats:p></jats:sec><jats:sec><jats:title>Results</jats:title><jats:p>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.</jats:p></jats:sec><jats:sec><jats:title>Discussion</jats:title><jats:p>Eculizumab is an effective therapy for patients with refractory AChR<jats:sup>+</jats:sup> gMG, irrespective of whether they had received rituximab treatment previously.</jats:p></jats:sec>