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Abstract Details

Improvement in Myasthenia Gravis Activities of Daily Living Subdomain Scores in Patients Treated with Eculizumab: Results from a Generalized Myasthenia Gravis Registry Study
Neuromuscular and Clinical Neurophysiology (EMG)
P4 - Poster Session 4 (11:45 AM-12:45 PM)
11-008
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.
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.
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.
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).
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.
Authors/Disclosures
Guido Sabatella (Alexion)
PRESENTER 		Guido Sabatella has received personal compensation for serving as an employee of Alexion.
Vern C. Juel, MD, FAAN (Duke University Medical Center) Dr. Juel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Juel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. The institution of Dr. Juel has received research support from argenx. The institution of Dr. Juel has received research support from Alexion. The institution of Dr. Juel has received research support from Janssen. The institution of Dr. Juel has received research support from NIH Rare Diseases Network.
Andrew J. Gordon, MD (Northwest Neurology) Dr. Gordon has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Catalyst. Dr. Gordon has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Gordon has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avion . Dr. Gordon has received personal compensation in the range of $0-$499 for serving as a Consultant for Vertex. Dr. Gordon has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Gordon has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Lundbeck. Dr. Gordon has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Abbvie. Dr. Gordon has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Cassiday.
Ericka P. Greene, MD, FAAN (Methodist Hospital) Dr. Greene has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ionis . Dr. Greene has received personal compensation in the range of $10,000-$49,999 for serving as an officer or member of the Board of Directors for ABPN. Dr. Greene has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Abraham & Watkins Texas Law Firm. The institution of Dr. Greene has received research support from National Science Foundaton .
Tahseen Mozaffar, MD, FAAN (University of California Irvine) Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi-Genzyme. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Amicus. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Grifols. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Astellas Gene Therapy. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Bayer. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Applied Therapeutics. The institution of Dr. Mozaffar has received research support from NIH. The institution of Dr. Mozaffar has received research support from Muscular Dystrophy Association. The institution of Dr. Mozaffar has received research support from Sanofi. The institution of Dr. Mozaffar has received research support from Argenx. The institution of Dr. Mozaffar has received research support from Amicus Therapeutics. The institution of Dr. Mozaffar has received research support from Astellas Gene Therpay. The institution of Dr. Mozaffar has received research support from Cartesian. The institution of Dr. Mozaffar has received research support from Cabaletta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH.
James M. Winkley, MD Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Winkley has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Sanofi. Dr. Winkley has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Teva. Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. The institution of Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for J&J. Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for amgen . Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for TG therapeutics. An immediate family member of Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for BMS. Dr. Winkley has received personal compensation in the range of $100,000-$499,999 for serving on a Speakers Bureau for Teva. Dr. Winkley has received personal compensation in the range of $100,000-$499,999 for serving on a Speakers Bureau for Alexion. Dr. Winkley has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for BMS. Dr. Winkley has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for amgen . Dr. Winkley has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for UCB . Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for J&J. Dr. Winkley has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for TG therapeutics. The institution of Dr. Winkley has received research support from Noveratis. The institution of Dr. Winkley has received research support from Merck. The institution of Dr. Winkley has received research support from Sanofi. The institution of Dr. Winkley has received research support from Biogen. The institution of Dr. Winkley has received research support from Alexion.
Ema Rodrigues, Other Dr. Rodrigues has received personal compensation for serving as an employee of Alexion Pharmaceuticals. Dr. Rodrigues has stock in Alexion Pharmaceuticals.
Rup Tandan, MD, FRCP, FAAN (University of Vermont (UVM)/UVM Medical Center) Dr. Tandan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Tandan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Apellis. Dr. Tandan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Amylyx. The institution of Dr. Tandan has received research support from Alexion. The institution of Dr. Tandan has received research support from Apellis. The institution of Dr. Tandan has received research support from Cytokinetics.