Post-hoc analyses from the ADAPT clinical study demonstrate aggregate sustained benefit of Efgartigimod in generalized myasthenia gravis.

Autor: Dewilde S; Services in Health Economics, Brussels, Belgium. Electronic address: sd@she-consulting.be., Griffiths A; Services in Health Economics, Brussels, Belgium., Qi CZ; argenx, Boston, MA, USA., Phillips G; argenx, Boston, MA, USA., Gelinas D; argenx, Boston, MA, USA., Brauer E; argenx, Boston, MA, USA., Mantegazza R; Fondazione IRCCS, Istituto Nazionale Neurologico Carlo Besta, Milan, Italy; Associazione Italiana Miastenia e Malattie Immunodegenerative, Milan, Italy., Howard JF Jr; The University of North Carolina School of Medicine, Department of Neurology, Chapel Hill, NC, USA.
Jazyk: angličtina
Zdroj: Journal of the neurological sciences [J Neurol Sci] 2024 Nov 15; Vol. 466, pp. 123264. Date of Electronic Publication: 2024 Oct 04.
DOI: 10.1016/j.jns.2024.123264
Abstrakt: Objectives: This post-hoc analysis evaluates the long-term efficacy of efgartigimod versus placebo in adult patients with generalized myasthenia gravis (gMG) with acetylcholine-receptor autoantibodies (AChR-Ab+), based on data from the ADAPT RCT and its open-label extension ADAPT+.
Methods: Changes from baseline in Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores were assessed by treatment group over the ADAPT (up to 20 weeks) and ADAPT+ time horizon (extended to 64 weeks for efgartigimod group patients). Response to treatment was defined as 5-point reduction in QMG or 3-point reduction in MG-ADL vs. baseline values.
Results: AChR-Ab+ patients treated with efgartigimod spent a substantially greater percentage of time in response in ADAPT based on at least a 5-point change in QMG compared to the placebo group (44 % versus 13 % respectively, p = 0.0034). Analyses based on a 3-point change in MG-ADL in ADAPT showed the percentage of time in response was nearly double for efgartigimod versus placebo (59 % versus 30 % respectively, p = 0.010). These trends were also maintained using different response definitions, as well as in patients with and without prior immune therapy exposure and by time from diagnosis (<7 years versus ≥7 years).
Conclusions: The clinical benefit of efgartigimod was sustained over repeat treatment cycles and maintained over the long term. Response to treatment was consistent regardless of response definition and was repeated in different patient subgroups. Overall, the results of this analysis indicate that efgartigimod is an effective therapeutic option, demonstrating a robust benefit among AChR-Ab+ patients with gMG.
Competing Interests: Declaration of competing interest CQ, GP, EB and DG are employees of argenx. SD and AG are employed by Services in Health Economics (SHE). SHE received fees from argenx in relation to this study. James F Howard Jr.: Research funding (paid to his institution) from Ad Scientiam, Alexion AstraZeneca Rare Disease, argenx, Cartesian Therapeutics, Centers for Disease Control and Prevention, MGFA, Muscular Dystrophy Association, NIH, PCORI, and UCB Pharma; honoraria/consulting fees from AcademicCME, Alexion, AstraZeneca Rare Disease, argenx, Biologix Pharma, CheckRare CME, F. Hoffmann-LaRoche Ltd., Horizon Therapeutics plc (now Amgen), Medscape CME, Merck EMB Serono, NMD Pharma, Novartis Pharma, PeerView CME, Physicians' Education Resource (PER) CME, PlatformQ CME, Regeneron Pharmaceuticals, Sanofi US, UCB Pharma, and Zai Labs; non-financial support from Alexion AstraZeneca Rare Disease, argenx, Cartesian Therapeutics, Toleranzia AB, UCB Pharma and Zai Labs.RM received funding for Travel, Meeting attendance or Advisory Board participation from Alexion, argenx, Biomarin, Catalyst, SANOFI, Regeneron and UCB.
(Copyright © 2024 The Authors. Published by Elsevier B.V. All rights reserved.)
Databáze: MEDLINE
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