Abstract Details

Title Rates of Myasthenic Crisis, Exacerbation and Healthcare Resource Utilization in Eculizumab-treated Patients with Generalized Myasthenia Gravis in a Global Registry

Topic Autoimmune Neurology

Presentation(s) S38 - Autoimmune Neurology: Peripheral Autoimmunity, Paraneoplastic Disease, Checkpoint Inhibitors, and Neurosarcoidosis (3:30 PM-3:42 PM)

Poster/Presentation
Number 001

Objective To assess the rates of myasthenic crisis, exacerbations and hospitalizations/emergency room (ER) visits among US patients with generalized myasthenia gravis (gMG) treated with eculizumab, a complement component C5 inhibitor, in clinical practice.

Background Generalized myasthenia gravis is a rare autoimmune disorder characterized by fatigable muscle weakness. Myasthenic crisis is a potentially life-threatening complication of gMG. Eculizumab has been shown to be effective and well-tolerated in treating patients with acetylcholine receptor antibody-positive gMG.

Design/Methods This analysis evaluated the frequency and type of hospitalizations for patients who were enrolled in an Alexion-sponsored global registry, including 12 months prior to and 12 months following initiation of eculizumab treatment (data cutoff: July 3, 2023). Demographics, hospitalizations/ER visits and intensive care unit (ICU) admissions were analyzed, irrespective of eculizumab treatment. In patients with known treatment dates, myasthenic crisis, exacerbations and hospitalizations rates per 100 person-years were calculated for before and during eculizumab treatment.

Results

Of 182 patients with completed hospitalization forms, 161 (89%) had complete eculizumab records. A higher proportion of patients who were vs. were not hospitalized and/or visited the ER were female (49% vs 43%), White (86% vs. 76%), and Hispanic (11% vs. 4%); a lower proportion were Black (4% vs. 11%).

Thirty-two (42%) hospitalized patients were admitted to an ICU, accounting for 45 total ICU admissions, of which 36 (80%) were MG-related. Following eculizumab initiation, decreases were observed for rates of myasthenic crisis (from 12 to 2 per 100 person-years), exacerbations (from 34 to 3), and hospitalizations (from 72 to 22). Rates of non-MG-related hospital visits decreased slightly (16 to 12). Patients without hospitalizations/ER visits increased from 100 (62.1%) to 121 (75.2%) following eculizumab initiation.

Conclusions Eculizumab is associated with decreased rates of myasthenic crisis, exacerbations and hospitalizations among patients with gMG in clinical practice.

Authors/Disclosures
Rup Tandan, MD, FRCP, FAAN (University of Vermont (UVM)/UVM Medical Center) PRESENTER	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.
Gary R. Cutter, PhD (University of Alabama At Birmingham)	Dr. Cutter has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for onsulting or Advisory Boards: Alexion, Antisense Therapeutics/Percheron, Avotres, Biogen, Clene Nanomedicine, Clinical Trial Solutions LLC, Endra Life Sciences, Cognito Therapeutics, Genzyme, Genentech, Immunic, Klein-Buendel Incorporated, Kyverna Therapeutics, Inc. , Linical, Merck/Serono, Noema, Neurogenesis, Perception Neurosciences, Protalix Biotherapeutics, Regeneron, Revelstone Consulting, Roche, SAB Biotherapeutics, Sapience Therapeutics, Scott&Scott LLP, Tenmile.. Dr. Cutter has received personal compensation in the range of $50,000-$99,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Data and Safety Monitoring Boards: Applied Therapeutics, AI therapeutics, AMO Pharma, Argenx, Astra-Zeneca, Avexis Pharmaceuticals, Bristol Meyers Squibb, CSL Behring, Cynata Therapeutics, DiamedicaTherapeutics, Horizon Pharmaceuticals, Immunic, Inhibrix, Karuna Therapeutics, Kezar Life Sciences, Medtronic, Merck, Meiji Seika Pharma, Mitsubishi Tanabe Pharma Holdings, Prothena Biosciences, Novartis, Pipeline Therapeutics (Contineum), Regeneron, Sanofi-Aventis, Teva Pharmaceuticals, United BioSource LLC, University of Texas Southwestern.. Dr. Cutter has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for JASN.
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.
Ericka P. Greene, MD, FAAN (Methodist Hospital)	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.
Ema Rodrigues, Other	Dr. Rodrigues has received personal compensation for serving as an employee of Alexion Pharmaceuticals. Dr. Rodrigues has stock in Alexion Pharmaceuticals.
Guido Sabatella (Alexion)	Guido Sabatella has received personal compensation for serving as an employee of Alexion.
Samir P. Macwan, MD	Dr. Macwan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion,Argenx,Grifols,Kabafusion, UCB. Dr. Macwan has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Abbvie,Alexion,Argenx,Grifols and UCB. The institution of Dr. Macwan has received research support from Alexion, Argenx,Immunovant, UCB.

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