ADAPT NXT

Objective: This 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) d...

Descripción completa

Detalles Bibliográficos
Autores: Habib, A.A., Claeys, K.G., Bril, V., Hussain, Y., Gwathmey, K., Sahagian, G., Cortés-Vicente, Elena|||0000-0002-1428-1072, Brauer, E., Gelinas, D., Sumbul, A., Jimenez, R.H., Hristova, D., Masschaele, D., Mantegazza, R., Meisel, Andreas, Attarian, Sharam|||0000-0002-7211-4694
Tipo de recurso: artículo
Fecha de publicación:2025
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:322765
Acceso en línea:https://ddd.uab.cat/record/322765
https://dx.doi.org/urn:doi:10.1002/acn3.70051
Access Level:acceso abierto
Palabra clave:Efgartigimod
Generalized myasthenia gravis
Myasthenia gravis activities of daily living
Neonatal Fc receptor
Descripción
Sumario:Objective: This 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. Methods: Adult 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. Results: Sixty-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. Interpretation: Efgartigimod 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.