Update on outcome assessment in myositis.
Update on outcome assessment in myositis.
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DOI：
10.1038/nrrheum.2018.33
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发表时间：
2018-05
期刊：
影响因子：
--
通讯作者：
Ruperto N
中科院分区：
文献类型：
--
作者：
Rider LG;Aggarwal R;Machado PM;Hogrel JY;Reed AM;Christopher-Stine L;Ruperto N
成人和青少年特发性炎症性肌病(IIM)是以肌肉无力和炎症为特征的全身性自身免疫性疾病。我们回顾了临床医生和研究人员可用来评估肌炎治疗结果的主要工具。国际肌炎网络开发了用于评估疾病活动性的经过验证的、标准化良好的测量方法，称为核心集合测量方法，用于临床试验。制定并验证了成人和青少年皮肌炎和成人多发性肌炎的综合反应标准，该标准使用疾病活动核心集措施的加权变化，成人和青少年的轻微改善、中度改善和重大改善的阈值不同。肌肉力量和功能的其他测量正在得到验证，以提高内容的有效性和对变化的敏感度。正在为成年肌炎患者开发一种与健康相关的生活质量测量方法，其中包括患者输入的内容。疾病状态标准，包括非活动性疾病和缓解的标准，正被用作次要试验终点。肌肉磁共振成像和免疫生物标记物有望区分疾病活动和损伤，并可能提供急需的客观结果。肌炎结果评估方面的这些进展，以及国际网络之间的合作，应该会促进肌炎患者新疗法的进一步发展。
The adult and juvenile idiopathic inflammatory myopathies (IIM) are systemic autoimmune diseases with the hallmark of muscle weakness and inflammation. We review the major tools available to clinicians and researchers to evaluate the outcome of myositis treatment. Validated, well-standardized measures to assess disease activity, known as core set measures, were developed by international myositis networks for use in clinical trials. Composite response criteria using weighted changes in the disease activity core set measures were developed and validated for adult and juvenile dermatomyositis and adult polymyositis, with different thresholds for minimal, moderate, and major improvement in adults and juveniles. Additional measures of muscle strength and function are being validated to improve content validity and sensitivity to change. A health-related quality-of-life measure with patient input in content is being developed for adult myositis patients. Disease state criteria, including criteria for inactive disease and remission, are being used as secondary trial endpoints. Muscle magnetic resonance imaging and immunologic biomarkers are promising to discriminate between disease activity and damage and may provide much-needed objective outcomes. These advances in the outcome assessment of myositis, along with collaborations among international networks, should facilitate further development of new therapies for myositis patients.
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