Improvement of fatigue in generalised myasthenia gravis with zilucoplan

Journal of Neurology, Год журнала: 2024, Номер 271(5), С. 2758 - 2767

Опубликована: Фев. 24, 2024

Abstract Background Fatigue is a debilitating symptom of myasthenia gravis (MG). The impact fatigue on MG can be assessed by Quality Life in Neurological Disorders (Neuro-QoL) Short Form scale. Transformation raw Neuro-QoL scores to T-scores known approach for facilitating clinical interpretation clinically meaningful and severity thresholds. Methods In the Phase 3, double-blind, placebo-controlled RAISE study (NCT04115293), adults with acetylcholine receptor autoantibody-positive generalised (MG Foundation America Disease Class II–IV) were randomised 1:1 daily subcutaneous zilucoplan 0.3 mg/kg or placebo 12 weeks. Patients completing could opt receive an ongoing, open-label extension study, RAISE-XT (NCT04225871). this post-hoc analysis, we evaluated long-term effect patients who entered RAISE-XT. We report change levels from baseline Week 60. Results Mean improved group ( n = 86) difference versus 88; least squares mean difference: − 3.61 (nominal p -value 0.0060]), these improvements continued further At 12, more 34, 47.2%) experienced ≥ 1 level 23, 28.4%; 0.017). 60, most 55, 65.5%) had mild none. Conclusion Treatment demonstrated statistical during RAISE, which sustained 60

Язык: Английский

Light Sensitivity in Myasthenia Gravis: Clinical Characteristics and Impact on Quality of Life

Muscle & Nerve, Год журнала: 2025, Номер unknown

Опубликована: Март 5, 2025

Light sensitivity is occasionally reported in myasthenia gravis patients, yet its prevalence, clinical characteristics, and impact on disease severity quality of life remain insufficiently explored. This study aimed to evaluate the frequency, correlation light with patients. Myasthenia patients consecutively admitted Fondazione Policlinico Universitario A. Gemelli Rome between March October 2023 were considered for study. A demographically matched group healthy controls was also included. Patients completed Visual Sensitivity Questionnaire-8 (VLSQ-8). The patient cohort additionally received Quantitative Gravis (QMG) score, Activities Daily Living (MG-ADL) scale, Quality-of-Life-15 (MG-QoL-15) questionnaires. total 92 75 participated. significantly more frequent among (36%, 33/92) than (4%, 3/75). exhibited higher QMG (p < 0.0001), MG-ADL a decreased MG-QoL-15 = 0.0003) scores those without sensitivity. Notably, positively correlated diminished life. These findings suggest that may be feature gravis, clinically relevant implications well-being. underscores value screening managing routine care. Proactive identification treatment strategies at help enhance overall

Язык: Английский

What Patients Don’t Say and Physicians Don’t Ask: A Needs Assessment in Myasthenia Gravis Integrating Patient and Healthcare Professional Perspectives

Neurology and Therapy, Год журнала: 2025, Номер unknown

Опубликована: Май 15, 2025

Язык: Английский