Efgartigimod improved health-related quality of life in generalized myasthenia gravis: results from a randomized, double-blind, placebo-controlled, phase 3 study (ADAPT)

• Published in: Journal of neurology 2023-04, Vol.270 (4), p.2096-2105
• Main Authors: Saccà, Francesco, Barnett, Carolina, Vu, Tuan, Peric, Stojan, Phillips, Glenn A., Zhao, Sihui, Qi, Cynthia Z., Gelinas, Deborah, Chiroli, Silvia, Verschuuren, Jan J. G. M.
• Format: Article
• Language: English
• Subjects: Acetylcholine receptors; Autoantibodies; Clinical trials; Double-blind studies; Fc receptors; Humans; Immunoglobulin G; Infant, Newborn; Medicine; Medicine & Public Health; Myasthenia; Myasthenia gravis; Myasthenia Gravis - diagnosis; Myasthenia Gravis - drug therapy; Neonates; Neurology; Neuromuscular junctions; Neuroradiology; Neurosciences; Original Communication; Patients; Quality of Life; Receptors, Cholinergic; Statistical analysis; Therapeutic applications; Treatment Outcome
• ISSN: 0340-5354; 1432-1459; 1432-1459
• DOI: 10.1007/s00415-022-11517-w

There are substantial disease and health-related quality-of-life (HRQoL) burdens for many patients with myasthenia gravis (MG), especially for those whose disease symptoms are not well controlled. HRQoL measures such as the Myasthenia Gravis Quality of Life 15-item revised (MG-QOL15r) and EuroQoL 5-Dimensions 5-Levels (EQ-5D-5L) are vital for evaluating the clinical benefit of therapeutic interventions in patients with MG, as they assess the burden of disease and the effectiveness of treatment, as perceived by patients. The phase 3 ADAPT study (NCT03669588) demonstrated that efgartigimod—a novel neonatal Fc receptor inhibitor—was well tolerated and that acetylcholine receptor antibody–positive (AChR-Ab+) participants who received efgartigimod had statistically significant improvements in MG-specific clinical scale scores. The ancillary data reported here, which cover an additional treatment cycle, show that these participants had similar significant improvements in HRQoL measures, the MG-QOL15r and EQ-5D-5L utility and visual analog scales, and that these improvements were maintained in the second treatment cycle. Positive effects on HRQoL were rapid, seen as early as the first week of treatment in both treatment cycles, and maintained for up to 4 weeks in the follow-up–only portion of treatment cycles. The pattern of improvements in HRQoL paralleled changes in immunoglobulin G level, and correlational analyses show that improvements were consistent across HRQoL measures and with clinical efficacy measures in the ADAPT study. The substantial and durable improvements in HRQoL end points in this study demonstrate the broader benefit of treatment with efgartigimod beyond relief of immediate signs and symptoms of gMG.