Abstract Details

Title Change in Concomitant Therapies for Generalized Myasthenia Gravis in Patients Receiving Eculizumab: a Retrospective Analysis of Registry Data

Topic Autoimmune Neurology

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

Poster/Presentation
Number 6-002

Background Treatment for gMG includes a broad spectrum of therapies, including typical immunosuppressive therapies (ISTs) and eculizumab, a complement C5 inhibitor.

Objective To assess the use of concomitant therapies at and after eculizumab initiation in patients with generalized myasthenia gravis (gMG) enrolled in a registry.

Design/Methods

Patients enrolled in a US gMG registry that collects data on the use of eculizumab in clinical practice were included if they were treated with eculizumab for ≥ 1 year and had data on concomitant therapy use 12 months prior to eculizumab initiation. Use of azathioprine (AZA), mycophenolate mofetil (MMF), intravenous immunoglobulin (IVIg)/plasma exchange (PLEX), and oral corticosteroids at initiation of, and during eculizumab treatment were analyzed. The data cut-off was July 5, 2022.

Results

A total of 94 patients were included in the analysis. At eculizumab initiation, 25 (27%), 40 (43%), 25 (27%) and 3 (4%) of patients were receiving zero, one, two, or three of the concomitant therapies investigated, respectively. Nine (10%) patients were on AZA, 26 (28%) on MMF, 19 (20%) on IVIg/PLEX and 47 (50%) on oral corticosteroids. In 57 (61%) patients, the number of concomitant therapies did not change. In 26 (24%) patients, there was a reduction in the number of concomitant therapies. Thirteen (14%) patients needed more treatments after eculizumab initiation. Of the patients using each treatment at eculizumab initiation, 2/9 (22%) patients discontinued AZA, 8/26 (31%) MMF, 5/19 (26%) IVIg/PLEX and 11/47 (23%) oral corticosteroids.

Conclusions Approximately one quarter of patients with gMG treated with eculizumab were able to discontinue one or more concomitant therapy. These results from clinical practice in the US provide additional information about the ability to reduce concomitant therapies in patients with gMG on treatment with eculizumab.

Authors/Disclosures
Michael T. Pulley, MD, PhD (University of Florida Jacksonville) PRESENTER	Dr. Pulley has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion`. Dr. Pulley has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MT Pharma. Dr. Pulley has received personal compensation in the range of $500-$4,999 for serving as a Consultant for amylyx. Dr. Pulley has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amgen. Dr. Pulley has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Pulley has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Pulley has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for Multiple law firms.
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,Catalyst,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,Biohaven,Catalyst,Grifols,Supernus. The institution of Dr. Macwan has received research support from Alexion, Disimmune Disease Foundation .
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.
Tahseen Mozaffar, MD, FAAN (University of California Irvine)	Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi-Genzyme. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Modis Therapeutics. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB/ Ra Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Maze Therapeutics. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for National Institutes of Health. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Argenx. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for Neuromuscular Disease Foundation. The institution of Dr. Mozaffar has received research support from NIH. The institution of Dr. Mozaffar has received research support from Muscular Dystrophy Association. The institution of Dr. Mozaffar has received research support from Sanofi-Genzyme. The institution of Dr. Mozaffar has received research support from Argenx. The institution of Dr. Mozaffar has received research support from Amicus Therapeutics. The institution of Dr. Mozaffar has received research support from Spark Therapeutics. The institution of Dr. Mozaffar has received research support from Audentes Therapeutics. The institution of Dr. Mozaffar has received research support from Cartesian. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH.
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.
Brian Werneburg (Stoke Therapeutics)	Mr. Werneburg has received personal compensation for serving as an employee of Alexion.
Pushpa Narayanaswami, MD, MBBS, FAAN (Beth Israel Deaconess Medical Center)	Dr. Narayanaswami has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx USA. Dr. Narayanaswami 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. Narayanaswami 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 Muscle and Nerve, Wiley. Dr. Narayanaswami has stock in Moderna. Dr. Narayanaswami has stock in Dr. Reddys laboratories. Dr. Narayanaswami has stock in Pfizer. Dr. Narayanaswami has stock in Viatris. Dr. Narayanaswami has stock in Doximity. The institution of Dr. Narayanaswami has received research support from Alexion. Dr. Narayanaswami has a non-compensated relationship as a Member, Medical and Scientific Advisory Board with Myasthenia Gravis Foundation of America that is relevant to AAN interests or activities. Dr. Narayanaswami has a non-compensated relationship as a Member, Board of Directors with AANEM that is relevant to AAN interests or activities.