Abstract Details

Title Treatment Fluctuations in Patients with Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis on Maintenance Ravulizumab Treatment

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P10 - Poster Session 10 (11:45 AM-12:45 PM)

Poster/Presentation
Number 11-001

Objective To describe a “wearing off” phenomenon between standard maintenance infusions of ravulizumab for a subset of patients with acetylcholine receptor antibody positive generalized myasthenia (AChR gMG).

Background

Ravulizumab is a monoclonal antibody complement C5 inhibitor approved in April 2022 for the treatment of AChR gMG. CHAMPION MG, the pivotal phase III study, showed sustained improvement at 60 weeks with ravulizumab and demonstrated that scheduled dosing every 8 weeks results in rapid initial improvement followed by sustained symptom control. Complete terminal complement inhibition was sustained throughout 26 weeks of treatment in CHAMPION MG and treatment-related fluctuations were not described. However, some patients in clinical practice have reported a “wearing off” of benefit between treatment cycles.

Design/Methods This multicenter case series will identify through record review patients with AChR gMG who have received ravulizumab and report symptom fluctuation despite receiving a loading infusion schedule followed by at least 3 maintenance infusions as standard of care therapy with efficacious response. Patient demographics, symptom characteristics and treatment-specific data will be extracted and analyzed with descriptive statistics. Data regarding age at treatment initiation, disease duration and severity, concomitant and prior therapies, and treatment outcomes will be presented.

Results In our clinical practice, 46 patients have undergone treatment with ravulizumab, of whom 16 (35%) report a wearing off before their next scheduled infusion. Detailed longitudinal data will be presented.

Conclusions We will present our early clinical experience with ravulizumab in patients with AChR gMG, and provide preliminary characterization of the treatment-related fluctuations observed. We aim to identify any patient specific factors among those that experience this drift in efficacy. This information will provide important insights into this wearing off and the role of ravulizumab in the treatment of AChR gMG.

Authors/Disclosures
Dave S. Ho, MD (Cedars Sinai) PRESENTER	Dr. Ho has stock in Moderna.
Katherine M. Clifford, MD	Dr. Clifford has nothing to disclose.
Samantha Blum, DO	Dr. Blum has nothing to disclose.
Miriam L. Freimer, MD, FAAN (The Ohio State University)	Dr. Freimer has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx. Dr. Freimer 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. Freimer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for J and J. Dr. Freimer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. The institution of Dr. Freimer has received research support from Alnylum. The institution of Dr. Freimer has received research support from UCB. The institution of Dr. Freimer has received research support from NIH. The institution of Dr. Freimer has received research support from Janssen. Dr. Freimer has received research support from Avidity. Dr. Freimer has received research support from Fulcrum. The institution of Dr. Freimer has received research support from Dept of defense. The institution of an immediate family member of Dr. Freimer has received research support from Abcurro. Dr. Freimer has received personal compensation in the range of $10,000-$49,999 for serving as a presentations/teaching with UCB.
Neelam Goyal, MD, FAAN (Stanford University)	Dr. Goyal has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Goyal has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Goyal has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Janssen. Dr. Goyal has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. The institution of Dr. Goyal has received research support from Argenx.
Tahseen Mozaffar, MD, FAAN (University of California Irvine)	Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 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 $10,000-$49,999 for serving as a Consultant for Amicus. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Grifols. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Astellas Gene Therapy. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Bayer. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 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 Applied Therapeutics. 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. 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 Astellas Gene Therpay. The institution of Dr. Mozaffar has received research support from Cartesian. The institution of Dr. Mozaffar has received research support from Cabaletta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH.
Pushpa Narayanaswami, MD, MBBS, FAAN (Beth Israel Deaconess Medical Center)	Dr. Narayanaswami has received personal compensation in the range of $50,000-$99,999 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 CVS. 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 $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Narayanaswami has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for NMD pharma. 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 EMD-Serono (Merck usa). Dr. Narayanaswami has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Immuneabs. 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 Viridian. 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 Cabaletta-Bio. 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 Immunovant. 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 Amgen. 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 Dianthus. 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 Cartesian. 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 Vor Bio. 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 received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Neurology. Dr. Narayanaswami has or had stock in Dr. Reddys laboratories.Dr. Narayanaswami has or had stock in Doximity. The institution of Dr. Narayanaswami has received research support from Alexion. The institution of Dr. Narayanaswami has received research support from NIH. The institution of Dr. Narayanaswami has received research support from Argenx. The institution of Dr. Narayanaswami has received research support from Cabaletta Bio. Dr. Narayanaswami has received publishing royalties from a publication relating to health care. Dr. Narayanaswami has a non-compensated relationship as a Member, Finance Committee with AANEM that is relevant to AAN interests or activities.
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 $5,000-$9,999 for serving as a Consultant for UCB/Ra Pharma. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Horizont Pharma. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for J & J pharma. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dianthus Pharma. Dr. Muppidi has received publishing royalties from a publication relating to health care.
Anahit C. Mehrabyan, MD (UNC)	Dr. Mehrabyan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UVB. Dr. Mehrabyan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenex.
Amanda C. Guidon, MD, MPH (Massachusetts General Hospital)	An immediate family member of Dr. Guidon has received personal compensation for serving as an employee of GE Healthcare. Dr. Guidon has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Guidon has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Guidon has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regeneron. Dr. Guidon has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen. Dr. Guidon has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for AAN - Continuum. The institution of Dr. Guidon has received research support from NINDS. Dr. Guidon has received publishing royalties from a publication relating to health care. Dr. Guidon has a non-compensated relationship as a Committee Member with Myasthenia Gravis Foundation of America that is relevant to AAN interests or activities. Dr. Guidon has a non-compensated relationship as a Executive Committee Member with Neuromuscular Study Group that is relevant to AAN interests or activities.
Michael K. Hehir II, MD (Larner College of Medicine at the University of Vermont)	Dr. Hehir has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Hehir has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Hehir has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB Pharma. Dr. Hehir has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Janssen. Dr. Hehir has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Hehir has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Continuum- ��ɫ��. Dr. Hehir has received personal compensation in the range of $0-$499 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for MedLink Neurology. The institution of Dr. Hehir has received research support from Myasthenia Gravis Foundation of America. The institution of Dr. Hehir has received research support from University of Vermont Medical Center.
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 $500-$4,999 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 $5,000-$9,999 for serving as a Consultant for NMD Pharma. Dr. Habib has received personal compensation in the range of $5,000-$9,999 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 $5,000-$9,999 for serving as a Consultant for Kyverna. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Jansen/Johnson & Johnson. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EMDSerono/Merck. 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 $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. 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. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Jansen/Joohnson & Johnson. 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 Regeneron. The institution of Dr. Habib has received research support from Arcellx. The institution of Dr. Habib has received research support from Novartis. The institution of Dr. Habib has received research support from EMDSerono/Merck. The institution of Dr. Habib has received research support from Cour Pharmaceuticals.

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