Improvement in Myasthenia Gravis Activities of Daily Living Subdomain Scores in Patients Treated with Eculizumab: Results from a Generalized Myasthenia Gravis Registry Study
Vern Juel1, Andrew Gordon2, Ericka Greene3, Tahseen Mozaffar4, James Winkley5, Ema Rodrigues6, Guido Sabatella6, Rup Tandan7
1Neurology, Duke University Medical Center, 2Northwest Neurology, 3Neurology, Methodist Hospital, 4Neurology, University of California Irvine, 5Baptist Health Louisville, 6Alexion, AstraZeneca Rare Disease, 7Neurology, University of Vermont (UVM)/UVM Medical Center
Objective:
To assess changes in activities of daily living per muscle subdomain in patients with generalized myasthenia gravis (gMG) treated with the complement C5 inhibitor eculizumab in clinical practice.
Background:
Phase 3 clinical trial data with eculizumab showed improved symptoms of patients with anti-acetylcholine receptor antibody-positive (AChR Ab+) gMG, as assessed by the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale.
Design/Methods:
Patient demographics and disease characteristics are presented for the entire gMG registry population and the analysis subgroup of patients who had MG-ADL scores both before and after initiation of eculizumab. The analysis includes patients who discontinued treatment, with the data cut-off of October 4, 2022. The distribution and change in MG-ADL scores by subdomain (bulbar [0–9], ocular [0–6], respiratory [0–3], limbs [0–6]) before and after eculizumab initiation are reported.
Results:
Demographics and disease characteristics were similar between the overall registry population (N=162) and analysis subgroup (n=92). The mean (standard deviation) duration of treatment was 1.7 years (1.2). Following eculizumab treatment, MG-ADL scores improved across every subdomain, with mean percentage improvement of 58.2% (bulbar), 42.3% (ocular), 38.7% (respiratory), and 46.7% (limbs). 55 patients (60%) had a lower number of subdomains with any symptoms present. The proportions of patients with improvements in MG-ADL scores were: 65% (bulbar), 70% (ocular), 34% (respiratory) and 51% (limbs).
Conclusions:
These results from a gMG registry show that MG-ADL scores and the number of affected subdomains are reduced with eculizumab treatment in clinical practice. The findings substantiate the broad benefit of complement C5 inhibition for patients with gMG.