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Abstract：
The ACR-EULAR Myositis Response Criteria (MRC) were developed as a composite measure using absolute percentage change in six core set measures (CSMs). We aimed to further validate the MRC by assessing the contribution of each CSM, frequency of strength vs extramuscular activity improvement, representation of patient-reported outcome measures (PROM), and frequency of CSM worsening.
Data from adult dermatomyositis/polymyositis patients in the rituximab (n = 147), etanercept (n = 14), and abatacept (n = 19) trials, and consensus patient profiles (n = 232) were evaluated. The Total Improvement Score (TIS), number of improving vs worsening CSMs, frequency of improvement with and without muscle-related CSMs, and contribution of PROM were evaluated by MRC category. Regression analysis was performed to assess contribution of each CSM to the MRC.
Of 412 adults with dermatomyositis/polymyositis, there were 37%, 24%, 25%, and 14% with no, minimal, moderate, and major MRC improvement, respectively. The number of improving CSMs and absolute percentage change in all CSMs increased by improvement category. In minimal-moderate improvement, only physician-reported disease activity contributed significantly more than expected by MRC. Of patients with at least minimal improvement, 95% had improvement in muscle-related measures and a majority (84%) had improvement in PROM. Patients with minimal improvement had worsening in a median of 1 CSM, and most patients with moderate-major improvement had no worsening CSMs. Physician assessment of change generally agreed with MRC improvement categories.
The ACR-EULAR MRC performs consistently across multiple studies, further supporting its use as an efficacy end point in future myositis therapeutic trials.
Data from adult dermatomyositis/polymyositis patients in the rituximab (n = 147), etanercept (n = 14), and abatacept (n = 19) trials, and consensus patient profiles (n = 232) were evaluated. The Total Improvement Score (TIS), number of improving vs worsening CSMs, frequency of improvement with and without muscle-related CSMs, and contribution of PROM were evaluated by MRC category. Regression analysis was performed to assess contribution of each CSM to the MRC.
Of 412 adults with dermatomyositis/polymyositis, there were 37%, 24%, 25%, and 14% with no, minimal, moderate, and major MRC improvement, respectively. The number of improving CSMs and absolute percentage change in all CSMs increased by improvement category. In minimal-moderate improvement, only physician-reported disease activity contributed significantly more than expected by MRC. Of patients with at least minimal improvement, 95% had improvement in muscle-related measures and a majority (84%) had improvement in PROM. Patients with minimal improvement had worsening in a median of 1 CSM, and most patients with moderate-major improvement had no worsening CSMs. Physician assessment of change generally agreed with MRC improvement categories.
The ACR-EULAR MRC performs consistently across multiple studies, further supporting its use as an efficacy end point in future myositis therapeutic trials.
摘要：
目的：ACR-EULAR肌炎反应标准（MRC）是使用六个核心指标（CSM）的绝对百分比变化作为复合指标。我们的目标是通过评估每个CSM的贡献来进一步验证MRC，强度与肌外活动改善的频率，患者报告结局指标(PROM)的代表性，以及CSM恶化的频率。
方法：来自利妥昔单抗（n=147）的成人皮肌炎/多发性肌炎患者的数据，依那西普（n=14），和abatacept(n=19)试验，和共识患者概况(n=232)进行了评估。总改善得分(TIS)，改善与恶化的CSM数量，有或没有肌肉相关CSM的改善频率，和PROM的贡献按MRC类别进行评估。进行回归分析以评估每个CSM对MRC的贡献。
结果：在412名患有皮肌炎/多发性肌炎的成年人中，有37%,24%,25%,14%没有，最小，中度,以及主要的MRC改进，分别。改进的CSM的数量和所有CSM中的绝对百分比变化随改进类别而增加。在最小适度的改善中，只有医生报告的疾病活动对MRC的贡献显著高于预期.至少有最小改善的患者，95％的肌肉相关措施有所改善，大多数（84％）的PROM有所改善。改善最小的患者在1个CSM的中位数中恶化，大多数中度-重度改善的患者没有CSM恶化。医师对变化的评估通常与MRC改善类别一致。
结论：ACR-EULARMRC在多项研究中表现一致，进一步支持将其用作未来肌炎治疗试验的疗效终点.
方法：来自利妥昔单抗（n=147）的成人皮肌炎/多发性肌炎患者的数据，依那西普（n=14），和abatacept(n=19)试验，和共识患者概况(n=232)进行了评估。总改善得分(TIS)，改善与恶化的CSM数量，有或没有肌肉相关CSM的改善频率，和PROM的贡献按MRC类别进行评估。进行回归分析以评估每个CSM对MRC的贡献。
结果：在412名患有皮肌炎/多发性肌炎的成年人中，有37%,24%,25%,14%没有，最小，中度,以及主要的MRC改进，分别。改进的CSM的数量和所有CSM中的绝对百分比变化随改进类别而增加。在最小适度的改善中，只有医生报告的疾病活动对MRC的贡献显著高于预期.至少有最小改善的患者，95％的肌肉相关措施有所改善，大多数（84％）的PROM有所改善。改善最小的患者在1个CSM的中位数中恶化，大多数中度-重度改善的患者没有CSM恶化。医师对变化的评估通常与MRC改善类别一致。
结论：ACR-EULARMRC在多项研究中表现一致，进一步支持将其用作未来肌炎治疗试验的疗效终点.
