Abstract Details

Title Myasthenia Gravis Activities of Daily Living (MG-ADL) Response to Eculizumab Treatment in Patients from the Generalized Myasthenia Gravis Registry

Topic Autoimmune Neurology

Presentation(s) P1 - Poster Session 1 (8:00 AM-9:00 AM)

Poster/Presentation
Number 6-020

Background

Eculizumab is effective in improving functional ability and muscle strength in patients with gMG, a rare autoimmune disease characterized by fatigable muscle weakness. To optimize treatment outcomes, it is important to understand the differences in characteristics between responders and non-responders.

Objective To quantify the proportion of patients with generalized myasthenia gravis (gMG) who responded to treatment with eculizumab, as assessed by the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale, and to investigate potential predictors of response in clinical practice.

Design/Methods

Patients were enrolled in a gMG registry that collects clinical practice data on the effectiveness of eculizumab in patients with gMG in the USA. The analysis population included patients with MG-ADL total scores both prior to eculizumab initiation, and at or after enrollment in the registry. Response to eculizumab was defined as an improvement of ≥ 3 points in MG-ADL total score; non-response was an improvement of < 3 points, no change or worsening in score. Predictors of response analyzed included patient demographics, disease characteristics and treatment duration. Analysis data cut-off was July 5, 2022.

Results

In the analysis population (n = 80), 87.5% of patients were White, 58.8% were male and mean (standard deviation; SD) age at diagnosis was 54.7 (19.9) years. At the time of analysis, 65% of patients met the definition of response to eculizumab treatment. There were no statistically significant differences in demographics between responders and non-responders. Responders had a significantly higher MG-ADL total score before treatment compared with non-responders (mean [SD] 9.1 [3.4] vs 6.4 [3.4]; p < 0.05). Duration of eculizumab treatment was similar between responders and non-responders (mean [SD] 1.8 [1.2] years vs 1.7 [0.9] years, respectively).

Conclusions Most patients analyzed responded to eculizumab treatment. Of the predictors analyzed, a higher MG-ADL total score before eculizumab was indicative of response.

Authors/Disclosures
Ericka P. Greene, MD, FAAN (Methodist Hospital) PRESENTER	Dr. Greene has received personal compensation for serving as an employee of Argenx. Dr. Greene has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion, Inc. Dr. Greene has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylx. Dr. Greene has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB . 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 $0-$499 for serving as an officer or member of the Board of Directors for ABPN.
Gary R. Cutter, PhD (University of Alabama At Birmingham)	Dr. Cutter has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biodelivery Sciences International, Biogen, Click Therapeutics, Genzyme, Genentech, GW Pharmaceuticals, Immunic, Klein-Buendel Incorporated, Medimmune/Viela Bio, Medday, Merck/Serono, Neurogenesis LTD, Novartis, Osmotica Pharmaceuticals, Perception Neurosciences, Recursion/Cerexis Pharmaceuticals, Regeneron, Reckover Pharmaceuticals, Roche, TG Therapeutics.. Dr. Cutter has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Applied Therapeutics, AI therapeutics, AMO Pharma, Astra-Zeneca, Avexis Pharmaceuticals, Biolinerx, Brainstorm Cell Therapeutics, Bristol Meyers Squibb/Celgene, CSL Behring, Galmed Pharmaceuticals, Green Valley Pharma, Horizon Pharmaceuticals, Immunic, Karuna Therapeutics, Mapi Pharmaceuticals LTD, Merck, Mitsubishi Tanabe Pharma Holdings, Opko Biologics,Prothena Biosciences, Novartis, Regeneron, Sanofi-Aventis, Reata Pharmaceuticals, NHLBI (Protocol Review Committee), University of Texas Southwestern, University of Pennsylvania, Visioneering Technologies, Inc.. Dr. Cutter has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for JASN.
Srikanth Muppidi, MD, FAAN	Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for argenx. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB/Ra Pharma. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Horizont Pharma. Dr. Muppidi has received publishing royalties from a publication relating to health care.
Vern C. Juel, MD, FAAN (Duke University Medical Center)	Dr. Juel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. Dr. Juel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Juel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Accordant Health Services. Dr. Juel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen. 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.
Ema Rodrigues, Other	Dr. Rodrigues has received personal compensation for serving as an employee of Alexion Pharmaceuticals. Dr. Rodrigues has stock in Alexion Pharmaceuticals.
Houari Korideck, MD (Sanofi)	Dr. Korideck has nothing to disclose.
James F. Howard, Jr., MD, FAAN (The University of North Carolina, Dept of Neurology, CB 7025)	Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for arenx . Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regeneron Pharmaceuticals. Dr. Howard 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 Pharmaceuticals. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ra Pharma (now UCB Biosciences). Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon Therapeutics. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi US. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Academic CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for PeerView CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Platform Q CME. Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Zai Laboratories. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Medscape CME. Dr. Howard has stock in General Electric dividends. Dr. Howard has stock in Johnson & Johnson dividends. Dr. Howard has stock in Pfizer dividends. An immediate family member of Dr. Howard has stock in GlaxoSmithKline dividends. An immediate family member of Dr. Howard has stock in GE Healthcare dividends. The institution of Dr. Howard has received research support from Alexion Pharmaceuticals. The institution of Dr. Howard has received research support from argenx . The institution of Dr. Howard has received research support from UCB Biosciences. The institution of Dr. Howard has received research support from NIH. The institution of Dr. Howard has received research support from Centers for Disease Control/Research Triangle Institute. The institution of Dr. Howard has received research support from Duke University (DCRI). The institution of Dr. Howard has received research support from Cartestian Therapeutics. Dr. Howard has a non-compensated relationship as a Scientific Advisiory Board member, Committee member with Myasthenia Gravis Foundation of America that is relevant to AAN interests or activities. Dr. Howard has a non-compensated relationship as a Committee member with American Assoc Neuromuscular and Electrodiagnostic Medicine that is relevant to AAN interests or activities.