Abstract Details

Title Effectiveness of Eculizumab Treatment by Time from Diagnosis in Patients with Generalized Myasthenia Gravis: A Retrospective Electronic Medical Record Analysis

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) S15 - Autoimmune Neuromuscular Diseases: New Observations and Therapeutic Approaches (2:00 PM-2:12 PM)

Poster/Presentation
Number 006

Background Complement component 5 inhibitors eculizumab and ravulizumab are approved treatments for acetylcholine receptor antibody positive generalized myasthenia gravis (gMG). In the pivotal CHAMPION MG trial, improvements in functioning were numerically greater in patients receiving ravulizumab ≤2 vs >2 years from diagnosis (assessed by Myasthenia Gravis Activities of Daily Living; MG-ADL).

Objective To assess MG-ADL total score changes from baseline to week 26 in patients initiated on eculizumab before or after 2 years of gMG diagnosis in clinical practice.

Design/Methods Physician-reported electronic medical records of adults with gMG who initiated eculizumab between October 23, 2017 and December 31, 2019 in 14 US sites were included. Patients initiating eculizumab ≤2 years or >2 years after diagnosis were defined as early or late initiators, respectively. Median MG-ADL scores and percentage changes from baseline are reported due to non-normal data distribution; age at eculizumab initiation was a covariate in the mixed model repeated measure analysis.

Results Fourteen early and 41 late initiators were identified. Respectively, mean (standard deviation) ages at eculizumab initiation were 66.5 (9.5) and 54.4 (17.7) years, and mean baseline MG-ADL total scores were 8.64 (1.74) and 6.78 (3.84). MG-ADL scores and percentage changes for all patients improved at week 26 compared to baseline; however, improvements were significantly greater for early compared with late initiators (6.5 vs 3.0, p<0.05; 78% vs 50%, p<0.05). A sensitivity analysis using cutoff of ≤5 or >5 years after diagnosis found a confirming trend for greater symptom improvement in earlier initiators, but this did not meet statistical significance compared with late initiators (6.0 vs 2.0, p=0.07; 71% vs 50%, p=0.57).

Conclusions In addition to previous findings from CHAMPION MG, this retrospective data analysis from US clinical practice provides further evidence that eculizumab or ravulizumab treatment within 2 years of gMG diagnosis may offer a greater clinical benefit compared with later initiation.

Authors/Disclosures
Srikanth Muppidi, MD, FAAN PRESENTER	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.
Justin Lee	No disclosure on file
Ali A. Habib, MD (University of California, Irvine)	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.
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.
Andrew Klink (Cardinal Health)	No disclosure on file
Adrian Kielhorn	Adrian Kielhorn has received personal compensation for serving as an employee of Alexion. Adrian Kielhorn has stock in Alexion.
Guido Sabatella (Alexion)	Guido Sabatella has received personal compensation for serving as an employee of Alexion.
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.