Eculizumab improves fatigue in refractory generalized myasthenia gravis

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Andersen H., Mantegazza R., Wang J. J., O’Brien F., Patra K., Howard J. F., ...Daha Fazla

Quality of Life Research, cilt.28, sa.8, ss.2247-2254, 2019 (SCI-Expanded, SSCI, Scopus)

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 28 Sayı: 8
  • Basım Tarihi: 2019
  • Doi Numarası: 10.1007/s11136-019-02148-2
  • Dergi Adı: Quality of Life Research
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Social Sciences Citation Index (SSCI), Scopus
  • Sayfa Sayıları: ss.2247-2254
  • Anahtar Kelimeler: Complement, Eculizumab, Fatigue, Myasthenia gravis, Neuro-QOL Fatigue, Quality of life, Terminal complement inhibition
  • Açık Arşiv Koleksiyonu: AVESİS Açık Erişim Koleksiyonu
  • Ondokuz Mayıs Üniversitesi Adresli: Evet

Özet

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.