2 research outputs found
Longâterm efficacy of eculizumab in refractory generalized myasthenia gravis: responder analyses
OBJECTIVE: Generalized myasthenia gravis (gMG) is an autoimmune disease that causes disabling weakness via damage to the neuromuscular junction. In most patients, the disease is mediated by autoantibodies to the acetylcholine receptor, which activate the complement cascade. Our objective was to analyze response profiles in adult patients with antiâacetylcholine receptor antibodyâpositive refractory gMG treated with eculizumabâa terminal complement inhibitorâin the REGAIN study or its openâlabel extension (OLE). METHODS: We retrospectively analyzed Myasthenia GravisâActivities of Daily Living (MGâADL) and Quantitative Myasthenia Gravis (QMG) scores recorded during REGAIN and its OLE. Early/late responses were defined as improvement in MGâADL score (â„3 points) or QMG score (â„5 points) at â€12 or >12 weeks, respectively, after eculizumab initiation. RESULTS: The analysis included 98 patients. By Week 12 and conclusion of the OLE, MGâADL response had been achieved at some point by 67.3% and 84.7% of patients, respectively, and QMG response by 56.1% and 71.4%, respectively. Response was observed over multiple consecutive assessments for most patients. At Week 130, the leastâsquares mean percentage changes (95% CI) from baseline in MGâADL score were â61.9% (â69.9%, â53.9%) and â47.5% (â59.0%, â36.0%) in early and late MGâADL responders, respectively; the leastâsquares mean percentage changes from baseline in QMG score were â40.8% (â48.3%, â33.4%) and â55.5% (â68.4%, â42.7%) in early and late QMG responders, respectively. INTERPRETATION: The findings suggest that, although most patients with refractory gMG will achieve clinical response by Week 12 of eculizumab treatment, first responses can be observed with longerâterm treatment