Rates of Myasthenic Crisis, Exacerbation and Healthcare Resource Utilization in Eculizumab-treated Patients with Generalized Myasthenia Gravis in a Global Registry
Rup Tandan1, Gary Cutter2, James Winkley3, Ericka Greene4, Ema Rodrigues5, Guido Sabatella5, Samir Macwan6
1Myasthenia Gravis Center, University of Vermont Medical Center, 2Department of Biostatistics, University of Alabama at Birmingham School of Public Health, 3Baptist Health Louisville, 4Stanley H Appel Department of Neurology, Houston Methodist Hospital, 5Alexion, AstraZeneca Rare Disease, 6Eisenhower Health
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.