We found a match
Your institution may have rights to this item. Sign in to continue.
- Title
Improvement of fatigue in generalised myasthenia gravis with zilucoplan.
- Authors
Weiss, Michael D.; Freimer, Miriam; Leite, M. Isabel; Maniaol, Angelina; Utsugisawa, Kimiaki; Bloemers, Jos; Boroojerdi, Babak; Howard, Emily; Savic, Natasa; Howard Jr, James F.
- Abstract
Background: 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. Methods: 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. Results: Mean Neuro-QoL Short Form Fatigue T-scores improved from baseline to Week 12 in the zilucoplan group (n = 86) with a clinically meaningful difference versus placebo (n = 88; least squares mean difference: − 3.61 (nominal p-value = 0.0060]), and these improvements continued further to Week 60. At Week 12, more patients on zilucoplan (n = 34, 47.2%) experienced improvements in ≥ 1 fatigue severity level from baseline versus placebo (n = 23, 28.4%; p = 0.017). At Week 60, most (n = 55, 65.5%) patients had mild fatigue or none. Conclusion: 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.
- Subjects
MYASTHENIA gravis; FATIGUE (Physiology); CHOLINERGIC receptors; NEUROLOGICAL disorders; LEAST squares
- Publication
Journal of Neurology, 2024, Vol 271, Issue 5, p2758
- ISSN
0340-5354
- Publication type
Article
- DOI
10.1007/s00415-024-12209-3