Abstract Details

Title Outcomes for Patients with Generalized Myasthenia Gravis Prescribed Ravulizumab or Efgartigimod Treatment: A Retrospective Medical Record Analysis

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P1 - Poster Session 1 (11:45 AM-12:45 PM)

Poster/Presentation
Number 11-001

Objective Evaluate outcomes among patients with generalized myasthenia gravis (gMG) treated with ravulizumab or efgartigimod as first targeted immunotherapy.

Background Targeted therapies such as ravulizumab (terminal complement inhibitor) and efgartigimod (neonatal Fc receptor blocker) are approved to treat anti-acetylcholine receptor antibody-positive (AChR-Ab+) gMG. However, there is a lack of real-world data assessing clinical outcomes among patients treated with these therapies.

Design/Methods Physician-abstracted electronic medical records were included for adults with AChR-Ab+ gMG in Cardinal Health’s Neurology Provider Extended Network who initiated their first targeted immunotherapy on or after December 1, 2021. Outcomes included clinical characteristics, concomitant medication use, and Myasthenia Gravis Activities of Daily Living (MG-ADL) total scores up to 2 years preinitiation and after treatment initiation with additional efficacy measures (up to 17.7 months of follow-up).

Results Data were available for 152 patients (ravulizumab, n=45; efgartigimod, n=107). Mean±SD age at initiation was 61.5±13.6 years for the ravulizumab group and ‌57.0±16.6 for the efgartigimod group. Preinitiation, mean±SD MG-ADL total scores were 9.3±2.9 in the ravulizumab group and 8.7±3.8 in the efgartigimod group. Mean±SD MG-ADL total scores at 3 months post initiation were 4.7±3.1 and 5.6±3.4 with ravulizumab and efgartigimod, respectively, and at 6 months post initiation, total scores were 2.0±1.8 and 4.3±3.2, respectively. Among patients taking oral corticosteroids (OCS) at treatment initiation, 17/19 (89.5%) ravulizumab patients and 33/46 (71.7%) efgartigimod patients reduced their dose during treatment. No patients increased their OCS dose while on ravulizumab, and 3/46 (6.5%) efgartigimod patients increased OCS dose to >20 mg/day. Additional outcomes will be presented.

Conclusions Despite varying patient characteristics, results suggest both treatments improved patient outcomes and decreased OCS dosing. Patients who received ravulizumab trended toward greater improvements in MG-ADL total score than those who received efgartigimod.

Authors/Disclosures
Samir P. Macwan, MD PRESENTER	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.
Christopher A. Scheiner, MD, PhD (TCNI)	Dr. Scheiner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Scheiner 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 Pharmaceuticals . Dr. Scheiner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL Behring . Dr. Scheiner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx Pharmaceuticals. Dr. Scheiner 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. Scheiner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Scheiner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Jansssen. Dr. Scheiner has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for CSL Behring . Dr. Scheiner has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Alexion Pharmaceuticals . Dr. Scheiner has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Amgen. Dr. Scheiner has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Janssen. The institution of Dr. Scheiner has received research support from Alexion Pharmaceuticals .
Nicholas S. Streicher, MD	Dr. Streicher has nothing to disclose.
Karen Yee, PhD (Alexion)	Dr. Yee has received personal compensation for serving as an employee of Alexion. Dr. Yee has stock in Alexion. Dr. Yee has stock in Takeda.
Chloe S. Sader, PharmD (Alexion)	Chloe Sader has received personal compensation for serving as an employee of Alexion. Chloe Sader has received stock or an ownership interest from Alexion.
Michael Blackowicz, PhD (Alexion)	Dr. Blackowicz has received personal compensation for serving as an employee of Alexion Pharmaceuticals. Dr. Blackowicz has stock in Alexion Pharmaceuticals.
Nana Numapau (Alexion)	Nana Numapau has received personal compensation for serving as an employee of Alexion Pharmaceutical.
Danielle Gentile	Dr. Gentile has received personal compensation for serving as an employee of Cardinal Health.
Jason Sharpe, PhD, PT	Dr. Sharpe has received personal compensation for serving as an employee of Department of Veterans Affairs. Dr. Sharpe has received personal compensation for serving as an employee of Cardinal Health. Dr. Sharpe has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant with University of North Carolina.
Prathamesh Pathak, MS, BPharm	Mr. Pathak has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Cardinal Health.
Michael T. Pulley, MD, PhD, FAAN (University of Florida Jacksonville)	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 $500-$4,999 for serving as a Consultant for csl/behring. Dr. Pulley has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta. Dr. Pulley has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for Multiple law firms.

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