From the Duke University School of Medicine (J.T.G.), Durham, NC; argenx US (J.T.G.), Boston, MA; University of Miami School of Medicine (M.B., V.G.), FL; Biohaven Pharmaceuticals (V.G.), New Haven, CT; University of California, Irvine (A.A.H.); The University of North Carolina School of Medicine (J.F.H.), Chapel Hill; Division of Neurology (C.B.-T.), Department of Medicine, University of Toronto, Ontario, Canada; Yale University School of Medicine (R.J.N.), New Haven, CT; Columbia University (I.L.), New York, NY; Medical University of South Carolina (K.R.), Charleston; Kansas University Medical Center (M.M.D.), Kansas City; School of Public Health (G.R.C.), University of Alabama at Birmingham; and George Washington University School of Medicine & Health Sciences (H.J.K.), DC.
Neurology. 2023 Sep 5;101(10):442-451. doi: 10.1212/WNL.0000000000207278. Epub 2023 Apr 19.
An increasing number of clinical trials are enrolling patients with myasthenia gravis (MG). A lack of standardization in the performance of outcome measures leads to confusion among site research teams and is a source of variability in clinical trial data. MGNet, the NIH-supported Rare Disease Clinical Research Network for MG, views standardization of MG outcome measures as a critical need. To address this issue, a group of experts summarized key outcome measures used in MG clinical trials and a symposium was convened to address issues contributing to outcome measure variability. Consensus recommendations resulted in changes to outcome measure instructions and, in some cases, modifications to specific instruments. Recommended changes were posted for public commentary before finalization. Changes to the MG-Activities of Daily Living, MG-Quality of Life-15r, and MG-Impairment Index were limited to adding details to the administration instructions. Recommendations for proper positioning of participants and how to score items that could not be performed because of non-MG reasons were provided for the MG Composite. The Quantitative MG (QMG) score required the most attention, and changes were made both to the instructions and the performance of certain items resulting in the QMG-Revised. The Postintervention Status was believed to have a limited role in clinical trials, except for the concept of minimal manifestation status. As a next step, training materials and revised source documents, which will be freely available to study teams, will be created and posted on the MGNet website. Further studies are needed to validate changes made to the QMG-Revised.
越来越多的临床试验正在招募重症肌无力（MG）患者。由于在实施结果测量方面缺乏标准化，导致研究地点的研究团队感到困惑，这也是临床试验数据存在差异的一个原因。MGNet 是 NIH 支持的 MG 罕见病临床研究网络，它认为 MG 结果测量的标准化是一项关键需求。为了解决这个问题，一组专家总结了 MG 临床试验中使用的关键结果测量指标，并召开了一次专题研讨会来解决导致结果测量指标变化的问题。达成共识的建议导致对结果测量指标说明进行了修改，在某些情况下，还对特定仪器进行了修改。建议的修改在最终确定之前已发布供公众发表意见。MG 日常生活活动量表、MG 生活质量量表-15r 和 MG 损伤指数的变化仅限于在管理说明中添加细节。针对 MG 综合量表，还提出了有关正确定位参与者以及如何对因非 MG 原因而无法进行的项目进行评分的建议。定量 MG（QMG）评分需要最多的关注，因此对说明和某些项目的执行进行了修改，从而形成了修订后的 QMG 评分。干预后状态除了最小表现状态的概念外，在临床试验中作用有限。下一步，将为研究团队创建和发布培训材料以及修订后的原始文件，这些材料和文件将免费提供。需要进一步的研究来验证对修订后的 QMG 所做的更改。
