Eculizumab improves fatigue in refractory generalized myasthenia gravis

The REGAIN Study Group

Research output: Contribution to journalArticleResearchpeer-review

1 Citation (Scopus)

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, 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.

Original languageEnglish
Pages (from-to)2247-2254
Number of pages8
JournalQuality of Life Research
Volume28
Issue number8
DOIs
StatePublished - 15 Aug 2019

Fingerprint

Myasthenia Gravis
Fatigue
Activities of Daily Living
Placebos
eculizumab
Quality of Life
Cholinergic Receptors
Nervous System Diseases
Outcome Assessment (Health Care)

Keywords

  • Complement
  • Eculizumab
  • Fatigue
  • Myasthenia gravis
  • Neuro-QOL Fatigue
  • Quality of life
  • Terminal complement inhibition

Cite this

The REGAIN Study Group. / Eculizumab improves fatigue in refractory generalized myasthenia gravis. In: Quality of Life Research. 2019 ; Vol. 28, No. 8. pp. 2247-2254.
@article{b23c15e380db429798ee25713c8dca70,
title = "Eculizumab improves fatigue in refractory generalized myasthenia gravis",
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, 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.",
keywords = "Complement, Eculizumab, Fatigue, Myasthenia gravis, Neuro-QOL Fatigue, Quality of life, Terminal complement inhibition",
author = "{The REGAIN Study Group} and Henning Andersen and Renato Mantegazza and Wang, {Jing Jing} and Fanny O’Brien and Kaushik Patra and Howard, {James F.} and Mazia, {Claudio Gabriel} and Miguel Wilken and Fabio Barroso and Juliet Saba and Marcelo Rugiero and Mariela Bettini and Marcelo Chaves and Gonzalo Vidal and Garcia, {Alejandra Dalila} and Jan De Bleecker and {Van den Abeele}, Guy and Kathy de Koning and Katrien De Mey and Rudy Mercelis and D{\'e}lphine Mahieu and Linda Wagemaekers and Philip Van Damme and Annelies Depreitere and Caroline Schotte and Charlotte Smetcoren and Olivier Stevens and Sien Van Daele and Nicolas Vandenbussche and Annelies Vanhee and Sarah Verjans and Jan Vynckier and Ann D’Hondt and Petra Tilkin and {Alves de Siqueira Carvalho}, Alzira and Igor Dias Brockhausen and David Feder and Daniel Ambrosio and Pamela C{\'e}sar and Melo, {Ana Paula} and Renata Martins Ribeiro and Rosana Rocha and Bruno Bezerra Rosa and Thabata Veiga and da Silva, {Luiz Augusto} and Murilo Santos Engel and Jordana Gon{\cc}alves Geraldo and {da Penha Ananias Morita}, Maria and Erica Nogueira Coelho and Yoon-Ho Hong",
year = "2019",
month = "8",
day = "15",
doi = "10.1007/s11136-019-02148-2",
language = "English",
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pages = "2247--2254",
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Eculizumab improves fatigue in refractory generalized myasthenia gravis. / The REGAIN Study Group.

In: Quality of Life Research, Vol. 28, No. 8, 15.08.2019, p. 2247-2254.

Research output: Contribution to journalArticleResearchpeer-review

TY - JOUR

T1 - Eculizumab improves fatigue in refractory generalized myasthenia gravis

AU - The REGAIN Study Group

AU - Andersen, Henning

AU - Mantegazza, Renato

AU - Wang, Jing Jing

AU - O’Brien, Fanny

AU - Patra, Kaushik

AU - Howard, James F.

AU - Mazia, Claudio Gabriel

AU - Wilken, Miguel

AU - Barroso, Fabio

AU - Saba, Juliet

AU - Rugiero, Marcelo

AU - Bettini, Mariela

AU - Chaves, Marcelo

AU - Vidal, Gonzalo

AU - Garcia, Alejandra Dalila

AU - De Bleecker, Jan

AU - Van den Abeele, Guy

AU - de Koning, Kathy

AU - De Mey, Katrien

AU - Mercelis, Rudy

AU - Mahieu, Délphine

AU - Wagemaekers, Linda

AU - Van Damme, Philip

AU - Depreitere, Annelies

AU - Schotte, Caroline

AU - Smetcoren, Charlotte

AU - Stevens, Olivier

AU - Van Daele, Sien

AU - Vandenbussche, Nicolas

AU - Vanhee, Annelies

AU - Verjans, Sarah

AU - Vynckier, Jan

AU - D’Hondt, Ann

AU - Tilkin, Petra

AU - Alves de Siqueira Carvalho, Alzira

AU - Dias Brockhausen, Igor

AU - Feder, David

AU - Ambrosio, Daniel

AU - César, Pamela

AU - Melo, Ana Paula

AU - Martins Ribeiro, Renata

AU - Rocha, Rosana

AU - Bezerra Rosa, Bruno

AU - Veiga, Thabata

AU - da Silva, Luiz Augusto

AU - Santos Engel, Murilo

AU - Gonçalves Geraldo, Jordana

AU - da Penha Ananias Morita, Maria

AU - Nogueira Coelho, Erica

AU - Hong, Yoon-Ho

PY - 2019/8/15

Y1 - 2019/8/15

N2 - 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.

AB - 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.

KW - Complement

KW - Eculizumab

KW - Fatigue

KW - Myasthenia gravis

KW - Neuro-QOL Fatigue

KW - Quality of life

KW - Terminal complement inhibition

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U2 - 10.1007/s11136-019-02148-2

DO - 10.1007/s11136-019-02148-2

M3 - Article

VL - 28

SP - 2247

EP - 2254

JO - Quality of Life Research

JF - Quality of Life Research

SN - 0962-9343

IS - 8

ER -