Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension

OBJECTIVE: To evaluate whether eculizumab helps patients with anti-acetylcholine receptor-positive (AChR+) refractory generalized myasthenia gravis (gMG) achieve the Myasthenia Gravis Foundation of America (MGFA) post-intervention status of minimal manifestations (MM), we assessed patients' status throughout REGAIN (Safety and Efficacy of Eculizumab in AChR+ Refractory Generalized Myasthenia Gravis) and its open-label extension. METHODS: Patients who completed the REGAIN randomized controlled trial and continued into the open-label extension were included in this tertiary endpoint analysis. Patients were assessed for the MGFA post-intervention status of improved, unchanged, worse, MM, and pharmacologic remission at defined time points during REGAIN and through week 130 of the open-label study. RESULTS: A total of 117 patients completed REGAIN and continued into the open-label study (eculizumab/eculizumab: 56; placebo/eculizumab: 61). At week 26 of REGAIN, more eculizumab-treated patients than placebo-treated patients achieved a status of improved (60.7% vs 41.7%) or MM (25.0% vs 13.3%; common OR: 2.3; 95% CI: 1.1-4.5). After 130 weeks of eculizumab treatment, 88.0% of patients achieved improved status and 57.3% of patients achieved MM status. The safety profile of eculizumab was consistent with its known profile and no new safety signals were detected. CONCLUSION: Eculizumab led to rapid and sustained achievement of MM in patients with AChR+ refractory gMG. These findings support the use of eculizumab in this previously difficult-to-treat patient population. CLINICALTRIALSGOV IDENTIFIER: REGAIN, NCT01997229; REGAIN open-label extension, NCT02301624. CLASSIFICATION OF EVIDENCE: This study provides Class II evidence that, after 26 weeks of eculizumab treatment, 25.0% of adults with AChR+ refractory gMG achieved MM, compared with 13.3% who received placebo

Minimal Symptom Expression' in Patients With Acetylcholine Receptor Antibody-Positive Refractory Generalized Myasthenia Gravis Treated With Eculizumab

The efficacy and tolerability of eculizumab were assessed in REGAIN, a 26-week, phase 3, randomized, double-blind, placebo-controlled study in anti-acetylcholine receptor antibody-positive (AChR+) refractory generalized myasthenia gravis (gMG), and its open-label extension

Factors associated with quality of life of people with Myasthenia Gravis.

PURPOSE:As most of patients with Myasthenia Gravis have limitations in their physical functioning, many experience changes in psychological states and often have depression. The objective of the current study was to examine the roles of communication with medical professionals, patients' loneliness, and patients' depression, in relation to their effects on the patients' quality of life. METHODS:For 120 patients with MG of 18 years and older, demographic variables, along with communication with medical professionals, loneliness, depression, and quality of life were measured. RESULTS:As a result, people suffering from MG experienced lower quality of life when their career has changed due to the illness. At the same time, depression was a significant predictor of their quality of life, both in physical and mental domains. CONCLUSIONS:The implications for clinical settings and the suggestions for future research are discussed

Fatigue in patients with neuromyelitis optica spectrum disorder and its impact on quality of life

<div><p>Fatigue is a prevalent symptom and major burden in neuroimmunological diseases. In neuromyelitis optica spectrum disorder (NMOSD), a severe autoimmune central nervous system (CNS) inflammatory disease with autoantibodies reactive to aquaporin-4, there are few reports about fatigue and quality of life (QOL). We aimed to evaluate the severity of fatigue and its relationship with QOL in patients with NMOSD. We prospectively studied patients with NMOSD who were in remission and seropositive for anti-aquaporin-4 antibody, and they were divided into 2 groups based on the presence of fatigue assessed using the Functional Assessment of Chronic Illness Therapy-fatigue score. Sleep quality, depression, pain, and QOL were also evaluated. A total of 35 patients were enrolled (mean age, 46.5 ± 14.1 years; female: male = 29:6), and the median Expanded Disability Status Scale (EDSS) score was 2.0 (range, 0 to 8.0). The patients with fatigue (N = 25, 71.4%) had poorer sleep quality and more severe depression than those without fatigue (p = 0.009 and p = 0.001). Both the physical and mental QOL scores were lower in patients with fatigue than in those without fatigue (p = 0.033 and p = 0.004). Multiple linear regression analyses showed that the degree of fatigue with EDSS score and pain were independent predictors of physical aspects of QOL (B = 0.382, p = 0.001), whereas depression was the only predictor of the mental components of QOL (B = -0.845, p = <0.001). Fatigue is a common symptom and an important predictor of QOL in patients with NMOSD.</p></div

Fatigue, sleep, depression, pain, and health-related quality of life in patients with NMOSD between fatigue groups.

<p>Fatigue, sleep, depression, pain, and health-related quality of life in patients with NMOSD between fatigue groups.</p

The differences in sleep quality, depression, pain severity, and quality of life scores in patients with NMOSD based on the presence of fatigue.

<p>PSQI, BDI, and BPI scores were higher in patients with NMSOD and fatigue than in those without fatigue. Moreover, the physical and mental component scores of quality of life assessed by SF-36 were lower in patients with NMSOD and fatigue. PSQI, Pittsburgh Sleep Quality Index; BDI, Beck Depression Inventory; BPI, Brief Pain Inventory; SF-36, Short-Form 36 Health Survey.</p

Scatter plots of correlations among PSQI, BDI, the physical and mental component scores of SF-36, and FACIT-fatigue.

<p>There were negative correlations among PSQI score, BDI score, and FACIT-fatigue score. Furthermore, the physical and mental component scores of SF-36 were positively correlated with FACIT-fatigue score. PSQI, Pittsburgh Sleep Quality Index; BDI, Beck Depression Inventory; FACIT-fatigue, Functional Assessment of Chronic Illness Therapy-fatigue; SF-36, Short-Form 36 Health Survey.</p

Clinical characteristics of patients with NMOSD based on the presence of fatigue.

<p>Clinical characteristics of patients with NMOSD based on the presence of fatigue.</p

Multiple linear regression analysis of factors affecting quality of life in patients with NMOSD.

<p>Multiple linear regression analysis of factors affecting quality of life in patients with NMOSD.</p