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

Prevalence of lymphoedema more than five years after breast cancer treatment

AbstractAimA lack of consistency in the definition of breast cancer related lymphoedema (BCRL) and of uniform measurement criteria contribute to the wide prevalence range found in current literature. This report aims to describe the long-term prevalence of BCRL and secondly, to compare the long-term prevalence of BCRL when assessed by two objective measures and one subjective measure.MethodsThe upper-limbs of 145 post-surgical breast cancer patients were evaluated for the presence of lymphoedema using the water displacement method. Two circumference methods and patient perceived swelling were applied secondarily for comparison. Limb measurements were performed once, more than five years after surgery.ResultsThe long-term prevalence of BCRL using water displacement was 8%. Prevalence varied when the sum of arm circumference (SOAC), the arm circumference and the self-report methods were used: 16, 31 and 17% [P < 0.001], respectively. Of the women identified with BCRL using the water displacement technique, 82% were detected with the SOAC method, 82% with the arm circumference method and 91% by self-report. Using water displacement as the gold standard the methods with the highest specificities were the SOAC (90%) and self-report method (89%), arm circumference resulted in a low specificity of 73%.ConclusionThe prevalence of BCRL more than five years after surgical treatment differs depending on the measuring method used. Our data underlines the necessity for consensus on the diagnostic criteria for BCRL

Biomechanical Properties of the Skin in Patients with Breast Cancer-Related Lymphedema Compared to Healthy Individuals

BACKGROUND: Biomechanical skin changes in breast cancer-related lymphedema (BRCL) have barely been described and objectively tested. This study aims to compare the skin of upper limb lymphedema with skin of the healthy contralateral arm, in order to demonstrate changes of elasticity, viscoelasticity, and level of hydration of the skin in BCRL. The secondary aim is to investigate the correlation between biomechanical skin changes and measurements that are currently used in clinical practice, such as volume measurement and lymph-ICF score. METHODS AND RESULTS: Eighteen patients with BCRL and 18 healthy individuals were included in the study. A Cutometer(R) was used for measurements for skin elasticity and viscoelasticity on both arms of each subject. A Corneometer(R) was used for measurements of skin hydration. Measurements of both test groups were compared. In BCRL patients, there was a significant difference (p=<0.028) between the elasticity of the skin of the lymphedema arm compared to the healthy contralateral arm. There were no significant differences for level of skin hydration or viscoelasticity in lymphedema patients between the measurements on the skin of the lymphedematous and healthy arm. In healthy individuals, there were no significant differences for all measurements between skin of both arms. Spearman's correlation was significant (p=<0.01) for difference in volume and difference in elasticity in BCRL patients. CONCLUSION: This study shows an impaired elasticity for the skin of the lower arm in patients with lymphedema compared to the contralateral healthy arm. Promising evidence is suggested for the use of the Cutometer device in the diagnostic evaluation of BCRL