Reduction in oral glucocorticoid use after efgartigimod initiation in clinical practice among patients with generalized myasthenia gravis
- PMID: 40845416
- DOI: 10.1016/j.jns.2025.123652
Reduction in oral glucocorticoid use after efgartigimod initiation in clinical practice among patients with generalized myasthenia gravis
Abstract
This study evaluated changes in oral glucocorticoid (GC, prednisone equivalent) use among patients with generalized myasthenia gravis (gMG) previously using chronic GCs, who initiated and continued efgartigimod for ≥12 months, identified retrospectively from a US claims database. Myasthenia Gravis Activities of Daily Living (MG-ADL) scores were retrieved from a patient support program. The primary outcome was GC average daily dose (GC ADD) assessed during the 3 months before and 3, 6, 9, and 12 months after efgartigimod initiation. Overall, 266 patients were included (mean age: 59.8 years; 45 % female). Mean (95 % CI) GC ADD was significantly reduced at 3 (14.3 [12.6, 16.0] mg/day), 6 (13.4 [11.5, 15.4] mg/day), 9 (11.6 [10.0, 13.3] mg/day), and 12 (10.2 [8.8, 11.6] mg/day) months post-efgartigimod initiation compared to baseline (17.9 [15.7, 20.1] mg/day) (p < 0.001). At 12 months, 66 % (n = 176/266) reduced steroid dose. Among those, 68 % and 51 % achieved ≥50 % and ≥ 75 % ADD reduction, respectively. The proportion of patients using GC ADD of ≤5 mg/day increased from 13 % at baseline to 42 % at 12 months. Among 126 patients (47 %) who had MG-ADL scores available, there was a significant reduction from baseline in best follow-up mean (95 % CI) MG-ADL (from 7.9 [7.2, 8.5] to 3.1 [2.6, 3.6]), while mean GC ADD remained significantly reduced (p < 0.05). Thus, in patients with gMG using chronic GCs at baseline, GC use substantially reduced following efgartigimod initiation, while retaining improved MG-ADL scores, underscoring the potential steroid-sparing effect of efgartigimod in gMG treatment.
Keywords: Efgartigimod; Generalized myasthenia gravis; Glucocorticoid; Glucocorticoid tapering.
Copyright © 2024. Published by Elsevier B.V.
Conflict of interest statement
Declaration of competing interest NG has served as a paid consultant for argenx, UCB Pharma, Amgen, Janssen, and Alexion and has grant support from argenx. JS has consulted for argenx on glucocorticoid toxicity. TR received honoraria and/or research support from Alexion, argenx, Biogen, Merck, Novartis, Sanofi, UCB, J&J, and Roche. PN has received research support from NIH, PCORI, and Alexion/AstraZeneca Rare Disease; served as a member of advisory boards or provided paid consultations to Alexion/Astra Zeneca Rare Disease, Amgen, argenx, CVS, Cabaletta Bio, Dianthus, GSK, ImmuneAbs, Janssen, Merck-Serono, Novartis, and UCB; was on Data Monitoring Committee at Sanofi, argenx, and NMD Pharma; and received royalties from Springer Nature. GIW has served as member of advisory boards or provided paid consultations to Alexion, argenx, Cartesian, Janssen, and UCB; is on speaker bureaus for Alexion and UCB; and has received research support from Alexion, argenx, Immunovant, Roche, UCB, and the MG Foundation of America. AGS has served as a member of advisory boards or paid consultant for Alexion, Arcellx, argenx, Eidos, Lexicon, Lilly, Seismic, and UCB. CQ, DG, MJ, EB, and GP are employees of argenx. TBS, RRM, and MS are employees of ZS Associates (Evanston, IL, USA) and serve as paid consultants for argenx.
MeSH terms
Substances
LinkOut - more resources
Full Text Sources
Medical
Miscellaneous
