| Title: |
Eculizumab improves fatigue in refractory generalized myasthenia gravis |
| Authors: |
Andersen, Henning; Mantegazza, Renato; Wang, Jing Jing; O’Brien, Fanny; Patra, Kaushik; Howard, James F.; Mazia, Claudio Gabriel; Wilken, Miguel; Barroso, Fabio; Saba, Juliet; Rugiero, Marcelo; Bettini, Mariela; Chaves, Marcelo; Vidal, Gonzalo; Garcia, Alejandra Dalila; De Bleecker, Jan; Van den Abeele, Guy; de Koning, Kathy; De Mey, Katrien; Mercelis, Rudy; Mahieu, Délphine; Wagemaekers, Linda; Van Damme, Philip; Depreitere, Annelies; Schotte, Caroline; Smetcoren, Charlotte; Stevens, Olivier; Van Daele, Sien; Vandenbussche, Nicolas; Vanhee, Annelies; Verjans, Sarah; Vynckier, Jan; D’Hondt, Ann; Tilkin, Petra; Alves de Siqueira Carvalho, Alzira; Dias Brockhausen, Igor; Feder, David; Ambrosio, Daniel; César, Pamela; Melo, Ana Paula; Martins Ribeiro, Renata; Rocha, Rosana; Bezerra Rosa, Bruno; Veiga, Thabata; da Silva, Luiz Augusto; Santos Engel, Murilo; Gonçalves Geraldo, Jordana; da Penha Ananias Morita, Maria; Nogueira Coelho, Erica; Paiva, Gabriel |
| Contributors: |
Alexion Pharmaceuticals |
| Source: |
Quality of Life Research ; volume 28, issue 8, page 2247-2254 ; ISSN 0962-9343 1573-2649 |
| Publisher Information: |
Springer Science and Business Media LLC |
| Publication Year: |
2019 |
| Description: |
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. |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| DOI: |
10.1007/s11136-019-02148-2 |
| DOI: |
10.1007/s11136-019-02148-2.pdf |
| DOI: |
10.1007/s11136-019-02148-2/fulltext.html |
| Availability: |
https://doi.org/10.1007/s11136-019-02148-2; https://link.springer.com/content/pdf/10.1007/s11136-019-02148-2.pdf; https://link.springer.com/article/10.1007/s11136-019-02148-2/fulltext.html |
| Rights: |
https://creativecommons.org/licenses/by/4.0 ; https://creativecommons.org/licenses/by/4.0 |
| Accession Number: |
edsbas.76DF6B19 |
| Database: |
BASE |