Long-term safety and efficacy of eculizumab in generalized myasthenia gravis

Muppidi S., Utsugisawa K., Benatar M., Murai H., Barohn R. J., Illa I., ...Daha Fazla

Muscle and Nerve, cilt.60, sa.1, ss.14-24, 2019 (SCI-Expanded) identifier identifier

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 60 Sayı: 1
  • Basım Tarihi: 2019
  • Doi Numarası: 10.1002/mus.26447
  • Dergi Adı: Muscle and Nerve
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Sayfa Sayıları: ss.14-24
  • Anahtar Kelimeler: eculizumab, MG-ADL, MG-QOL15, MGC, myasthenia gravis, QMG
  • Hacettepe Üniversitesi Adresli: Evet

Özet

© 2019 The Authors. Muscle & Nerve published by Wiley Periodicals, Inc.Introduction: Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of an open-label extension of REGAIN, evaluating eculizumab's long-term safety and efficacy. Methods: Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients. Results: The safety profile of eculizumab was consistent with REGAIN; no cases of meningococcal infection were reported during the interim analysis period. Myasthenia gravis exacerbation rate was reduced by 75% from the year before REGAIN (P < 0.0001). Improvements with eculizumab in activities of daily living, muscle strength, functional ability, and quality of life in REGAIN were maintained through 3 years; 56% of patients achieved minimal manifestations or pharmacological remission. Patients who had received placebo during REGAIN experienced rapid and sustained improvements during open-label eculizumab (P < 0.0001). Discussion: These findings provide evidence for the long-term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019.