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P.034 Minimal symptom expression following treatment with efgartigimod in patients with Generalized Myasthenia Gravis

Published online by Cambridge University Press:  05 June 2023

Z Siddiqi
Affiliation:
(Edmonton)*
JF Howard Jr
Affiliation:
(Chapel Hill)
V Bril
Affiliation:
(Toronto)
T Vu
Affiliation:
(Tampa)
C Karam
Affiliation:
(Philadelphia)
M Pasnoor
Affiliation:
(Kansas City)
S Muppidi
Affiliation:
(Palo Alto)
S Peric
Affiliation:
(Belgrade)
H Murai
Affiliation:
(Tokyo)
P Ulrichts
Affiliation:
(Ghent)
C T’joen
Affiliation:
(Ghent)
K Utsugisawa
Affiliation:
(Hanamaki)
J Verschuuren
Affiliation:
(Leiden)
R Mantegazza
Affiliation:
(Milan) ADAPT Investigator Study Group ()
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Abstract

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Background: Efgartigimod is a human IgG1 antibody Fc-fragment that reduces IgG levels through FcRn blockade. A key efficacy indicator in the treatment of IgG autoantibody-mediated generalized myasthenia gravis (gMG) is improvement in MG-ADL score. Methods: The ADAPT phase 3 trial evaluated safety and efficacy of efgartigimod in patients with gMG, including reaching and maintaining of minimal symptom expression (MSE; defined as an MG-ADL total score of 0 or 1). Results: 167 patients (AChR-Ab+, n=129; AChR-Ab-, n=38) were randomized to receive treatment cycles of 4 weekly infusions of efgartigimod or placebo. Significantly more AChR-Ab+ efgartigimod-treated patients achieved MSE during cycle 1 compared to placebo-treated patients (40.0% [n=26/65] vs 11.1% [n=7/63; P<0.0001]). In cycle 2, 31.4% (n=16/51) of AChR-Ab+ patients in the efgartigimod cohort achieved MSE compared to none in the placebo cohort. MG-ADL score improved by ≥6 points in 56.9% of AChR-Ab+ efgartigimod-treated patients compared to 20.6% of placebo-treated patients in cycle 1. Most patients achieved MSE by week 4 of a cycle, paralleling early reduction in IgG levels, and MSE duration ranged from 1 to ≥10 weeks. Adverse events were predominantly mild to moderate. Conclusions: Efgartigimod treatment resulted in more patients with AChR-Ab+ gMG achieving both MSE and clinically meaningful MG-ADL improvements.

Type
Abstracts
Copyright
© The Author(s), 2023. Published by Cambridge University Press on behalf of Canadian Neurological Sciences Federation