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Weiss, Michael D.; Freimer, Miriam; Leite, M. Isabel; Maniaol, Angelina; Utsugisawa, Kimiaki; Bloemers, Jos; Boroojerdi, Babak; Howard, Emily; Savic, Natasa; Howard, James F.

Improvement of fatigue in generalised myasthenia gravis with zilucoplan

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  • Medientyp: E-Artikel
  • Titel: Improvement of fatigue in generalised myasthenia gravis with zilucoplan
  • Beteiligte: Weiss, Michael D.; Freimer, Miriam; Leite, M. Isabel; Maniaol, Angelina; Utsugisawa, Kimiaki; Bloemers, Jos; Boroojerdi, Babak; Howard, Emily; Savic, Natasa; Howard, James F.
  • Erschienen: Springer Science and Business Media LLC, 2024
  • Erschienen in: Journal of Neurology
  • Sprache: Englisch
  • DOI: 10.1007/s00415-024-12209-3
  • ISSN: 1432-1459; 0340-5354
  • Entstehung:
  • Anmerkungen:
  • Beschreibung: <jats:title>Abstract</jats:title><jats:sec> <jats:title>Background</jats:title> <jats:p>Fatigue is a debilitating symptom of myasthenia gravis (MG). The impact of fatigue on MG can be assessed by Quality of Life in Neurological Disorders (Neuro-QoL) Short Form Fatigue scale. Transformation of raw Neuro-QoL fatigue scores to T-scores is a known approach for facilitating clinical interpretation of clinically meaningful and fatigue severity thresholds.</jats:p> </jats:sec><jats:sec> <jats:title>Methods</jats:title> <jats:p>In the Phase 3, double-blind, placebo-controlled RAISE study (NCT04115293), adults with acetylcholine receptor autoantibody-positive generalised MG (MG Foundation of America Disease Class II–IV) were randomised 1:1 to daily subcutaneous zilucoplan 0.3 mg/kg or placebo for 12 weeks. Patients completing RAISE could opt to receive zilucoplan 0.3 mg/kg in an ongoing, open-label extension study, RAISE-XT (NCT04225871). In this post-hoc analysis, we evaluated the long-term effect of zilucoplan on fatigue in RAISE patients who entered RAISE-XT. We report change in Neuro-QoL Short Form Fatigue T-scores and fatigue severity levels from RAISE baseline to Week 60.</jats:p> </jats:sec><jats:sec> <jats:title>Results</jats:title> <jats:p>Mean Neuro-QoL Short Form Fatigue T-scores improved from baseline to Week 12 in the zilucoplan group (<jats:italic>n</jats:italic> = 86) with a clinically meaningful difference versus placebo (<jats:italic>n</jats:italic> = 88; least squares mean difference: − 3.61 (nominal <jats:italic>p</jats:italic>-value = 0.0060]), and these improvements continued further to Week 60. At Week 12, more patients on zilucoplan (<jats:italic>n</jats:italic> = 34, 47.2%) experienced improvements in ≥ 1 fatigue severity level from baseline versus placebo (<jats:italic>n</jats:italic> = 23, 28.4%; <jats:italic>p</jats:italic> = 0.017). At Week 60, most (<jats:italic>n</jats:italic> = 55, 65.5%) patients had mild fatigue or none.</jats:p> </jats:sec><jats:sec> <jats:title>Conclusion</jats:title> <jats:p>Treatment with zilucoplan demonstrated statistical and clinically meaningful improvements in fatigue scores and severity versus placebo during RAISE, which were sustained to Week 60 in RAISE-XT.</jats:p> </jats:sec>