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

Abstract

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.

Efgartigimod in generalized myasthenia gravis: A real‐life experience at a national reference center

“…EFG represents a well‐tolerated therapeutic option that changed considerably the course of the disease in our patients, as it prevented the most severe forms of the disease, hospitalization, and respiratory insufficiency. The present data also expand the observation of improved quality of life in MG as reported in patients participating in the ADAPT study [28]. A longer follow‐up will be of particular interest, as it will provide further information regarding the actual impact on ongoing treatments, particularly corticosteroids, and on the overall quality of life of our patients.…”

“…We had the opportunity to study the efficacy of EFG outside The present data also expand the observation of improved quality of life in MG as reported in patients participating in the ADAPT study [28]. A longer follow-up will be of particular interest, as it will provide further information regarding the actual impact on ongoing treatments, particularly corticosteroids, and on the overall quality of life of our patients.…”

What Is in the Neuromuscular Junction Literature?

“…HRQOL correlated with immunoglobulin G and clinical measures. 8 This report provides additional evidence of the type of rapid and durable benefit seen with efgartigimod in AChR Ab+ generalized MG.…”

“…More recently, a report by Sacca et al on improved health-related (HR) quality of life (QOL) in patients enrolled in the ADAPT trial followed. 8 These patients received a second treatment cycle. Measures of HRQOL were the MG-QOL15r and EuroQoL 5-dimensions 5-Levels as well as visual analog scales.…”

Immune-Mediated Neuropathies: Pathophysiology and Management

“…IVIG is considered to act by providing anti-idiotype antibodies, by blocking active sites of Fc receptors on macrophages, inhibition of the complement cascade, modulation of T cell regulatory processes and saturation of neonatal Fc receptor [ 191 , 192 , 193 , 194 ]. Several promising trials involving FcRn inhibitors are ongoing and show promise by reducing autoantibodies especially in myasthenia gravis [ 192 , 195 , 196 ]. In contrast, corticosteroids have not been proven to be beneficial in the treatment of GBS [ 197 , 198 ].…”