RT期刊文章SR电子T1比较量化的重症肌无力(评分)和重症肌无力活动的日常生活(MG-ADL)分数MGTX随机试验(p2.2 - 102)摩根富林明神经病学神经学乔FD Lippincott Williams &威尔金斯SP p2.2补充92 - 102签证官是15 A1 Tarrant麦克弗森A1 Immaculada波斯历八月A1佩特拉杜达A1白木Farzaneh-Far A1吉尔·沃尔夫A首页1亨利·卡明斯基A1加里刀年2019 UL //www.ez-admanager.com/content/92/15_supplement/p2.2 - 102. -文摘AB目的:定性、定量比较的表现评分和MG-ADL措施在MGTX审判。背景:评分和MG-ADL是两个最常见的措施重症肌无力(MG)疾病严重程度,但黄金标准尚未建立。MGTX试验收集纵向MG-ADL和评分办法患者随机分配到胸腺切除术+强的松(TPP)或强的松(PA)。设计/方法:MG-ADL评分和疾病措施分析了118例治疗组和随访时间至少有一个后续的访问使用广义线性模型和重复措施(glm)和自相关AR(1)协方差。模型比较来确定措施同样捕获组不同疾病状态和时间趋势。分布的假设也评估模型。之间的相关性的措施检查123例观察。评分和MG-ADL意味着之间的多元关系,标准差,观察到的相关性进行图形和使用多元方差分析。评分结果:假设常态出现足够,但是MG-ADL更好符合泊松分布。治疗组和时间都显著(p < 0.05)两种模型。AR(1)参数是同样高的评分(0.8448)和MG-ADL (0.7225)。整体视觉评分模型更适合病人特别是TPP。The baseline correlation between the measures was 0.65 (TPP) and 0.53 (PA). The increasing multivariate relationship describing the observed correlation between the two measures and their means (HLT p<0.0001) and standard deviations (HLT p<0.0001) at each time point suggests the measures perform more similarly when the disease is less well controlled.Conclusions: QMG and MG-ADL are clearly correlated and appear to perform similarly in measuring MG severity. The model curves and AR(1) coefficients are similar reflecting treatment modifications across study visits. Normality assumptions apply in modeling QMG, thereby facilitating statistical analyses and enabling a wider range of model assessments.Disclosure: Dr. McPherson has received research support from Ra Pharmaceuticals, Alexion Pharmaceuticals, Myasthenia Gravis Foundation of America. Dr. Aban has nothing to disclose. Dr. Duda has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Ra Pharmaceuticals. Dr. Duda holds stock and/or stock options in Ra Pharmaceuticals. Dr. Farzaneh-Far has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Ra Pharmaceuticals. Dr. Farzaneh-Far holds stock and/or stock options in Ra Pharmaceuticals. Dr. Wolfe has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Grifols, Shire, and Alexion. Dr. Wolfe has received research support from ArgenX, Ra, and Immunovant. Dr. Kaminski has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Ra Pharmaceuticals and GT Biopharma. Dr. Cutter has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with AMO Pharmaceuticals, Biolinerx, Horizon Pharmaceuticals, Hisun Pharmaceuticals, Merck, Merck/Pfizer, Opko Biologics, Neurim, Novartis, Ophazyme, Sanofi-Aventis, Reata Pharmaceuticals, Receptos/Celgene, Teva pharmaceuticals, NHLBI, NICHD, Atara Biotherapeutics, Axon, Biogen, Argenix, Brainstorm Cell Therapeutics, Charleston Labs Inc, Click Therapeutics, Genzyme, Genentech, GW Pharma, Klein-Buendel Incorporated, Medimmune, Medday, Novartis, Roche, Scifluor, Somahlution, Teva pharmaceuticals, TG Therapeutics, UT Houston. Dr. Cutter has received personal compensation in an editorial capacity for Statistical editor for the Journal of the American Society of Nephrology. Dr. Cutter has received research support from Via MGFA.