Eculizumab improves fatigue in refractory generalized myasthenia gravis.
Journal Article (Clinical Trial, Phase III;Journal Article)
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.
Full Text
Duke Authors
Cited Authors
- Andersen, H; Mantegazza, R; Wang, JJ; O'Brien, F; Patra, K; Howard, JF; REGAIN Study Group,
Published Date
- August 2019
Published In
Volume / Issue
- 28 / 8
Start / End Page
- 2247 - 2254
PubMed ID
- 30905021
Pubmed Central ID
- 30905021
Electronic International Standard Serial Number (EISSN)
- 1573-2649
Digital Object Identifier (DOI)
- 10.1007/s11136-019-02148-2
Language
- eng
Conference Location
- Netherlands