Measuring Overall Severity of Myasthenia Gravis (MG): Evidence for the Added Value of the MG Symptoms PRO.
Measuring Overall Severity of Myasthenia Gravis (MG): Evidence for the Added Value of the MG Symptoms PRO.
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DOI：
10.1007/s40120-023-00464-x
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发表时间：
2023-10
影响因子：
3.7
通讯作者：
Habib, Ali A.
中科院分区：
文献类型：
--
作者：
Regnault, Antoine;Morel, Thomas;de la Loge, Christine;Mazerolle, Flora;Kaminski, Henry J.;Habib, Ali A.
重症肌无力（MG）严重程度的准确测量对于对MG患者进行适当的临床监测以及在临床试验中评估新疗法的益处是必要的。我们的目的是探索如何测量MG严重程度，并确定新开发的MG症状患者报告结局（PRO）工具如何补充现有的MG严重程度测量方法。
针对MG的核心症状：三个肌群（眼部、延髓部和呼吸肌）的肌无力、肌无力易疲劳性和身体疲劳，对定量MG（QMG）、MG综合评分（MGC）、MG日常生活活动能力（MG - ADL）和MG症状PRO的概念覆盖范围进行了仔细审查。对MG0002研究（一项在中重度全身性MG成人患者中进行的rozanolixizumab的2a期临床试验）进行事后分析，包括相关性和拉施模型分析。
定性评估强调只有MG症状PRO涵盖了身体疲劳。分析使用了541次评估（43名独特患者）的数据。MG - ADL、QMG、MGC和MG症状PRO肌无力易疲劳性评分之间的相关性在0.56到0.74之间，针对独立肌群的MG症状PRO评分之间的相关性在0.20到0.71之间。拉施模型分析估计出了一个有意义的MG严重程度连续体，包括来自四种工具的除眼部肌肉外的所有项目。QMG和MG症状PRO对MG严重程度连续体的覆盖范围最广。肌肉易疲劳性和身体疲劳在低严重程度时更具特征，而延髓肌无力则表明MG更严重。
MG的严重程度可以通过基于MG特异性结局指标的有意义的连续体来反映。只有眼部肌肉表现被证明可能反映了MG严重程度的一个不同方面。MG症状PRO具有模块化性质和全面的内容，为MG中广泛使用的结局指标提供了补充信息。
ClinicalTrials.gov标识符：NCT03052751
在线版本包含补充材料，可在10.1007/s40120 - 2023 - 00464 - x获取
重症肌无力（MG）是一种影响神经与肌肉之间通讯的慢性疾病。MG患者会经历肌无力，活动后加重，休息后改善。MG可影响身体不同肌群（例如眼周、四肢、面部或喉部的肌肉）。
我们表明MG的各种症状可用于总结疾病的总体严重程度：轻度和中度MG患者通常仅报告其四肢肌肉无力发作迅速以及轻度身体疲劳，而重度MG患者则报告更严重的疲劳，以及与面部和喉部肌肉无力相关的困难（导致吞咽或说话困难）和呼吸肌无力（导致呼吸困难）。这种MG表现的排序将有助于创建更准确的评估MG严重程度的方法，可用于评估新疗法或在临床中监测患者。
我们还提出眼周肌肉无力（导致眼睑下垂或复视）可能代表MG的一个独特方面，并且可能不像其他症状那样能提供大量信息来总结MG的严重程度。然而，这需要进一步研究，因为我们的研究未包括仅以眼部肌肉无力为症状的参与者。
我们还记录了MG症状患者报告结局问卷（一种由患者填写的新问卷）测量MG严重程度的能力。
在线版本包含补充材料，可在10.1007/s40120 - 2023 - 00464 - x获取
Accurate measurement of myasthenia gravis (MG) severity is required for appropriate clinical monitoring of patients with MG and assessment of the benefit of new treatments in clinical trials. Our objective was to explore how MG severity can be measured and to determine how the newly developed MG Symptoms Patient-Reported Outcome (PRO) instrument complements the available measures of MG severity. The conceptual coverage of the Quantitative MG (QMG), MG Composite (MGC), MG-Activities of Daily Living (MG-ADL), and MG Symptoms PRO was scrutinized against core symptoms of MG: muscle weakness in three muscle groups (ocular, bulbar, and respiratory), muscle weakness fatigability, and physical fatigue. Post hoc analyses of the MG0002 study, a Phase 2a clinical trial of rozanolixizumab in adults with moderate to severe generalized MG, included correlation and Rasch model analyses. The qualitative appraisal highlighted that only the MG Symptoms PRO captured physical fatigue. Data from 541 assessments (43 unique patients) were used for the analyses. Correlations ranged between 0.56 and 0.74 for the MG-ADL, QMG, MGC, and MG Symptoms PRO Muscle Weakness Fatigability score, and between 0.20 and 0.71 for the MG Symptoms PRO scores focusing on independent muscle groups. Analyses with the Rasch model estimated a meaningful continuum of severity of MG, including all items, except ocular muscles, from the four instruments. The QMG and MG Symptoms PRO had the broadest coverage of the MG severity continuum. Muscle fatigability and physical fatigue were more characteristic of low severity while bulbar weakness indicated more severe MG. The severity of MG can be reflected in a meaningful continuum underpinned by the MG-specific outcome measures. Only ocular muscle manifestations were shown to reflect a possibly different facet of MG severity. With its modular nature and comprehensive content, the MG Symptoms PRO provides complementary information to the outcome measures widely used in MG. ClinicalTrials.gov identifier: NCT03052751. The online version contains supplementary material available at 10.1007/s40120-023-00464-x. Myasthenia gravis (MG) is a chronic disease affecting the communication between nerves and muscles. People with MG experience muscle weakness that worsens after activity and improves after rest. MG can affect different groups of body muscles (e.g., around the eyes, in the limbs, and face or throat). We show that the various symptoms of MG can be used to summarize the overall severity of the disease: people with mild and moderate MG often report only the fast onset of weakness in their limb muscles and mild physical fatigue, while those with more severe MG report more severe fatigue and also difficulties associated with weakness in facial and throat muscles (leading to difficulty with swallowing or speaking) and in respiratory muscles (making breathing difficult). This ordering of MG manifestations will help create more accurate methods to assess the severity of MG that can be used to evaluate new treatments or to monitor patients in the clinic. We also suggest that weakness of muscles around the eyes (leading to eyelid drooping or double vision) may represent a unique aspect of MG, and may not provide as much information to summarize the severity of MG as other symptoms. However, this needs further investigation as our study did not include participants who had weakness in eye muscles as their only symptom. We also document the ability of the MG Symptoms Patient-Reported Outcome questionnaire, a new questionnaire completed by patients, to provide useful information for measuring the severity of MG. The online version contains supplementary material available at 10.1007/s40120-023-00464-x.
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影响因子：
2.4
作者：
Barnett C;Herbelin L;Dimachkie MM;Barohn RJ
通讯作者：
Barohn RJ
影响因子：
6
作者：
Alekseeva, T. M.;Gavrilov, Y. V.;Valko, Y.
通讯作者：
Valko, Y.
影响因子：
3.4
作者：
Sadjadi, Reza;Conaway, Mark;Burns, Ted M.
通讯作者：
Burns, Ted M.
影响因子：
4.5
作者：
Patrick, Donald L.;Burke, Laurie B.;Ring, Lena
通讯作者：
Ring, Lena
DOI：
10.1111/j.1749-6632.1998.tb11015.x
发表时间：
1998-01-01
期刊：
MYASTHENIA GRAVIS AND RELATED DISEASES
影响因子：
--
作者：
Barohn, RJ;McIntire, D;Bryan, WW
通讯作者：
Bryan, WW