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

A new analysis method for accurate supercurrent distribution inside high-temperature superconducting bulk

It is difficult to accurately simulate supercurrent in high-temperature superconducting bulk even by adopting the finite-element method. The equivalent electrical conductivity of high-temperature superconducting bulk, which has a strong nonlinearity according to the E-J power law, is introduced for the supercurrent analysis. However, the strong nonlinearity results in bad convergency of the nonlinear equation solvers, i.e., the Newton-Raphson (NR) method. Consequently, the unsuitable and/or undulating supercurrent is observed. Therefore, in this paper, the accuracy of the supercurrent analysis in the high-temperature superconducting bulk is discussed and a new method is proposed for the accurate high-temperature superconducting bulk simulation. Then, the method combined a line search with the NR method is adopted as a nonlinear equation solver, and the improvement of convergency and computation time are investigate

Direct Dimesitylborylation of Benzofuran Derivatives by an Iridium-Catalyzed C-H Activation with Silyldimesitylborane

Direct dimesitylborylation of benzofuran derivatives by a C-H activation catalyzed by an iridium(I)/N-heterocyclic carbene (NHC) complex in the presence of Ph2MeSi-BMes(2) afforded the corresponding dimesitylborylation products in good to high yield with excellent regioselectivity. This method provides a straightforward route to donor-(pi-spacer)-acceptor systems with intriguing solvatochromic luminescence properties