Abstract Details

Title Real-World Eculizumab Effectiveness on US Patient Outcomes in Myasthenia Gravis (ELEVATE): a Retrospective Chart Review

Topic Autoimmune Neurology

Presentation(s) S41 - Autoimmune Neurology 3: Autoimmune Encephalitis, Paraneoplastic Neurologic Disorders, Antibody-Mediated Diseases, Immunology (5:18 PM-5:30 PM)

Poster/Presentation
Number 010

Background Eculizumab was approved in the US for adults with acetylcholine receptor autoantibody-positive generalized MG in October 2017; examination of its subsequent real-world impact is warranted.

Objective To describe the demographics, clinical characteristics and treatment outcomes of patients with myasthenia gravis (MG) treated with eculizumab in clinical practice in the US.

Design/Methods A retrospective chart review study was completed using data collected from patients aged ≥18 years with MG who initiated eculizumab between October 2017 and December 2019 in the US. Information was abstracted by study-site personnel from patients’ electronic medical records. Data on concomitant prednisone use and Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores were collected before and during eculizumab treatment. Results were analyzed using descriptive statistics.

Results Of the 100 eculizumab-treated patients enrolled in the study, 61% were female, 80% were White, and 16% were Black/African American. Mean (±SD) age was 48.4 (±21.2) years at MG diagnosis and 56.6 (±18.2) years at eculizumab initiation. The majority of patients (81%) received eculizumab for ≥12 months. Fifty-four patients were receiving prednisone at eculizumab initiation; 67% reduced their prednisone dosage during eculizumab treatment, and 11% discontinued prednisone (mean time to reduction and discontinuation: 190 and 180 days, respectively). The mean (±SD) MG-ADL score was 8.0 (±2.2) before eculizumab initiation (n=49) and 4.8 (±3.3) within 3 months after eculizumab initiation (n=51). Reduction in mean MG-ADL score was maintained in patients receiving eculizumab for ≥12 months (mean 3.5 [±3.3] at 12 months; n=51).

Conclusions

Results from this study suggest that treatment with eculizumab reduced patients’ treatment burden (shown by the decrease in concomitant prednisone use) and their disease burden (demonstrated by the improvement in MG-ADL score). These results support findings from the REGAIN trial and its open-label extension, and corroborate other real-world studies.

Authors/Disclosures
Ali A. Habib, MD (University of California, Irvine) PRESENTER	Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for argenx. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech/Roche. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NMD Pharma. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alpine Immune Sciences. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Arcellx. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Horizon/ Amgen. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NIH/NINDS. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunis Biomedical. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffman-La Roche. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for argenx. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. The institution of Dr. Habib has received research support from Alexion. The institution of Dr. Habib has received research support from Horizon/Amgen. The institution of Dr. Habib has received research support from Immunovant. The institution of Dr. Habib has received research support from UCB. The institution of Dr. Habib has received research support from argenx. The institution of Dr. Habib has received research support from CabalettaBio. The institution of Dr. Habib has received research support from Genentech. The institution of Dr. Habib has received research support from Regeneron.
Andrew Klink (Cardinal Health)	No disclosure on file
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.
Anju Parthan	Anju Parthan has received personal compensation for serving as an employee of Alexion. Anju Parthan has received personal compensation for serving as an employee of Alkermes. Anju Parthan has received stock or an ownership interest from Alexion.
Chloe S. Sader, PharmD (Alexion)	Chloe Sader has received personal compensation for serving as an employee of Alexion. Chloe Sader has received stock or an ownership interest from Alexion.
	No disclosure on file
	No disclosure on file
Richard J. Nowak, MD (Yale University School of Medicine)	Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argenx. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Momenta . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio . Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Viela Bio. Dr. Nowak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cour Pharma. The institution of Dr. Nowak has received research support from Ra Pharma. The institution of Dr. Nowak has received research support from Alexion . The institution of Dr. Nowak has received research support from Momenta . The institution of Dr. Nowak has received research support from Immunovant . Dr. Nowak has received research support from argenx. Dr. Nowak has received research support from Viela Bio . Dr. Nowak has a non-compensated relationship as a Member of the Board of Directors with Myasthenia Gravis Foundation of America (MGFA) that is relevant to AAN interests or activities.
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.