ADAPT NXT: Fixed Cycles or Every-Other-Week IV Efgartigimod in Generalized Myasthenia Gravis
Por:
Habib, AA, Claeys, KG, Bril, V, Hussain, Y, Gwathmey, K, Sahagian, G, Cortés-Vicente, E, Brauer, E, Gelinas, D, Sumbul, A, Jimenez, RH, Hristova, D, Masschaele, D, Mantegazza, R, Meisel, A, Attarian, S
Publicada:
1 jun 2025
Ahead of Print:
1 abr 2025
Resumen:
ObjectiveThis phase 3b, open-label, randomized ADAPT NXT study investigated the efficacy, safety, and tolerability of efgartigimod administered in either a fixed cycles dosing regimen (3 cycles of 4 once-weekly infusions, with 4 weeks between cycles) or a cycle followed by every-other-week (Q2W) dosing.MethodsAdult participants with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG) were randomized 3:1 to Q2W or fixed cycles dosing of efgartigimod (10 mg/kg intravenously) for 21 weeks. The primary endpoint was the mean change from baseline in total Myasthenia Gravis Activities of Daily Living (MG-ADL) score averaged across 21 weeks.ResultsSixty-nine participants were treated (fixed cycles, n = 17; Q2W, n = 52). Least squares (LS) mean (95% CI) of the change from baseline in MG-ADL total score from Weeks 1 to 21 was -5.1 (-6.5 to -3.8) in the fixed cycles arm and -4.6 (-5.4 to -3.8) in the Q2W arm. Clinical improvements were observed in MG-ADL total scores as early as Week 1 and were maintained throughout the study. Achievement of minimal symptom expression (MG-ADL: 0-1) from Weeks 1 to 21 occurred in 47.1% (n = 8/17) and 44.2% (n = 23/52) of participants in the fixed cycles and Q2W arms, respectively. Efgartigimod was well tolerated; COVID-19, headache, and upper respiratory tract infection were the most common treatment-emergent adverse events.InterpretationEfgartigimod administered as either fixed cycles or Q2W dosing results in rapid, robust, and sustained clinically meaningful improvement. These results build upon previous studies and provide additional efgartigimod dosing approaches to achieve and sustain clinical efficacy in patients with gMG.
Filiaciones:
Habib, AA:
Univ Calif Orange, Dept Neurol, Orange, CA 92868 USA
Claeys, KG:
Univ Hosp Leuven, Dept Neurol, Leuven, Belgium
Katholieke Univ Leuven, Lab Muscle Dis & Neuropathies, Leuven, Belgium
Bril, V:
Univ Hlth Network, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
Univ Toronto, Toronto, ON, Canada
Hussain, Y:
Austin Neuromuscular Ctr, Austin, TX USA
Gwathmey, K:
Virginia Commonwealth Univ, Dept Neurol, Richmond, VA USA
Sahagian, G:
Neurol Ctr Southern Calif, Carlsbad, CA USA
Cortés-Vicente, E:
Hosp Santa Creu i Sant Pau, Dept Neurol, Neuromuscular Dis Unit, Barcelona, Spain
Biomed Res Inst Sant Pau, Barcelona, Spain
CIBERER, Ctr Invest Biomed Red Enfermedades Raras, Valencia, Spain
Brauer, E:
argenx, Ghent, Belgium
Gelinas, D:
argenx, Ghent, Belgium
Sumbul, A:
argenx, Ghent, Belgium
Jimenez, RH:
argenx, Ghent, Belgium
Hristova, D:
argenx, Ghent, Belgium
Masschaele, D:
argenx, Ghent, Belgium
Mantegazza, R:
Fdn IRCCS Ist Neurol Carlo Besta, Milan, Italy
Meisel, A:
Charite Univ Med Berlin, Clin Res Ctr, Dept Neurol Expt Neurol & Neurosci, Berlin, Germany
Attarian, S:
Timone Hosp Univ, Reference Ctr Neuromuscular Disorders, Marseille, France
Timone Hosp Univ, ALS, Marseille, France
Green Accepted, Green Submitted, gold
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