Outcomes for Patients with Generalized Myasthenia Gravis Prescribed Ravulizumab or Efgartigimod Treatment: A Retrospective Medical Record Analysis
Christopher Scheiner1, Samir Macwan2, Nicholas Streicher3, Karen Yee4, Chloe Sader4, Michael Blackowicz4, Nana Numapau4, Danielle Gentile5, Jason Sharpe5, Prathamesh Pathak5, Michael Pulley6
1University of Tennessee Medical Center, 2Eisenhower Health Center, 3MedStar Georgetown University Hospital, UT Neurology, 4Alexion, AstraZeneca Rare Disease, 5Cardinal Health, 6University of Florida College of Medicine
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.
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