Eculizumab improves fatigue in refractory generalized myasthenia gravis

• Published in: Quality of life research Vol. 28; no. 8; pp. 2247 - 2255
• Main Authors: Andersen, Henning, Mantegazza, Renato, Wang, Jing Jing, O’Brien, Fanny, Patra, Kaushik, Howard, James F.
• Format: Journal Article
• Language: English
• Published: Cham Springer Science + Business Media 01.08.2019; Springer International Publishing; Springer Nature B.V
• Subjects: Activities of Daily Living; Antibodies, Monoclonal, Humanized - therapeutic use; BRIEF COMMUNICATION; Fatigue - drug therapy; Female; Humans; Male; Medicine; Medicine & Public Health; Middle Aged; Monoclonal antibodies; Myasthenia gravis; Myasthenia Gravis - drug therapy; NCT; NCT01997229; NCT02301624; Outcome Assessment, Health Care; Placebos - therapeutic use; Public Health; Quality of Life; Quality of Life Research; Receptors, Cholinergic - immunology; Sociology; Terminal complement inhibition; Myasthenia gravis; Eculizumab; Fatigue; Complement; Neuro-QOL Fatigue; Quality of life
• ISSN: 0962-9343; 1573-2649
• DOI: 10.1007/s11136-019-02148-2

Purpose To evaluate the effect of eculizumab on perceived fatigue in patients with anti-acetylcholine receptor antibody-positive, refractory, generalized myasthenia gravis (MG) using the Quality of Life in Neurological Disorders (Neuro-QOL) Fatigue subscale, and to evaluate correlations between improvements in Neuro-QOL Fatigue and other clinical endpoints. Methods Neuro-QOL Fatigue, MG Activities of Daily Living (MG-ADL), Quantitative MG (QMG), and the 15-item MG Quality of Life (MG-QOL15) scales were administered during the phase 3, randomized, placebo-controlled REGAIN study (eculizumab, n  = 62; placebo, n  = 63) and subsequent open-label extension (OLE). Data were analyzed using repeated-measures models. Correlations between changes in Neuro-QOL Fatigue and in MG-ADL, QMG, and MG-QOL15 scores were determined at REGAIN week 26. Results At REGAIN week 26, eculizumab-treated patients showed significantly greater improvements in Neuro-QOL Fatigue scores than placebo-treated patients (consistent with improvements in MG-ADL, QMG, and MG-QOL15 scores previously reported in REGAIN). Improvements with eculizumab were sustained through OLE week 52. Correlations between Neuro-QOL Fatigue and MG-QOL15, MG-ADL, and QMG scores were strong for eculizumab-treated patients at REGAIN week 26, and strong, moderate, and weak, respectively, for placebo-treated patients. Conclusions Compared with placebo, eculizumab was associated with improvements in perceived fatigue that strongly correlated with improvements in MG-specific outcome measures. Trial ID Registration : NCT01997229, NCT02301624.