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

Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review

DOI

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  • Srikanth Muppidi
  • Kimiaki Utsugisawa
  • Michael Benatar
  • Hiroyuki Murai
  • Richard J Barohn
  • Isabel Illa
  • Saiju Jacob
  • John Vissing
  • Ted M Burns
  • John T Kissel
  • Richard J Nowak
  • Henning Andersen
  • Carlos Casasnovas
  • Jan L de Bleecker
  • Tuan H Vu
  • Renato Mantegazza
  • Fanny L O'Brien
  • Jing Jing Wang
  • Kenji P Fujita
  • James F Howard
  • REGAIN Study Group
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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.

OriginalsprogEngelsk
TidsskriftMuscle & Nerve
Vol/bind60
Udgave nummer1
Sider (fra-til)14-24
Antal sider11
ISSN0148-639X
DOI
StatusUdgivet - 1 jul. 2019

Bibliografisk note

© 2019 The Authors. Muscle & Nerve published by Wiley Periodicals, Inc.

ID: 58110164