Eculizumab improves fatigue in refractory generalized myasthenia gravis.
| dc.contributor.author | Andersen, Henning | |
| dc.contributor.author | Mantegazza, Renato | |
| dc.contributor.author | Wang, Jing Jing | |
| dc.contributor.author | O'Brien, Fanny | |
| dc.contributor.author | Patra, Kaushik | |
| dc.contributor.author | Howard, James F | |
| dc.contributor.author | REGAIN Study Group | |
| dc.date.accessioned | 2023-07-19T20:49:15Z | |
| dc.date.available | 2023-07-19T20:49:15Z | |
| dc.date.issued | 2019-08 | |
| dc.date.updated | 2023-07-19T20:49:13Z | |
| dc.description.abstract | 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, nā=ā62; placebo, nā=ā63) 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. | |
| dc.identifier | 10.1007/s11136-019-02148-2 | |
| dc.identifier.issn | 0962-9343 | |
| dc.identifier.issn | 1573-2649 | |
| dc.identifier.uri | ||
| dc.language | eng | |
| dc.publisher | Springer Science and Business Media LLC | |
| dc.relation.ispartof | Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation | |
| dc.relation.isversionof | 10.1007/s11136-019-02148-2 | |
| dc.subject | REGAIN Study Group | |
| dc.subject | Humans | |
| dc.subject | Myasthenia Gravis | |
| dc.subject | Fatigue | |
| dc.subject | Receptors, Cholinergic | |
| dc.subject | Placebos | |
| dc.subject | Activities of Daily Living | |
| dc.subject | Quality of Life | |
| dc.subject | Middle Aged | |
| dc.subject | Female | |
| dc.subject | Male | |
| dc.subject | Antibodies, Monoclonal, Humanized | |
| dc.subject | Outcome Assessment, Health Care | |
| dc.title | Eculizumab improves fatigue in refractory generalized myasthenia gravis. | |
| dc.type | Journal article | |
| pubs.begin-page | 2247 | |
| pubs.end-page | 2254 | |
| pubs.issue | 8 | |
| pubs.organisational-group | Duke | |
| pubs.organisational-group | School of Medicine | |
| pubs.organisational-group | Clinical Science Departments | |
| pubs.organisational-group | Neurology | |
| pubs.organisational-group | Neurology, Neuromuscular Disease | |
| pubs.publication-status | Published | |
| pubs.volume | 28 |
Files
Original bundle
- Name:
- Eculizumab improves fatigue in refractory generalized myasthenia gravis.pdf
- Size:
- 802.04 KB
- Format:
- Adobe Portable Document Format