Department of Medicine, Division of Rheumatology and Clinical Immunology, University of Pittsburgh, Pittsburgh, Pennsylvania, USA.
Northwell Health, Formerly North Shore-Long Island Jewish Medical Center, Great Neck, New York, USA.
Ann Rheum Dis. 2018 May;77(5):720-727. doi: 10.1136/annrheumdis-2017-212047. Epub 2017 Dec 13.
To evaluate the efficacy, safety, tolerability and steroid-sparing effect of repository corticotropin injection (RCI), in an open-label clinical trial, in refractory adult polymyositis (PM) and dermatomyositis (DM).
Adults with refractory PM and DM were enrolled by two centres. Inclusion criteria included refractory disease defined as failing glucocorticoid and/or ≥1 immunosuppressive agent, as well as active disease defined as significant muscle weakness and >2 additional abnormal core set measures (CSMs) or a cutaneous 10 cm Visual Analogue Scale score of ≥3 cm at least three other abnormal CSMs. All patients received RCI of 80 units subcutaneously twice weekly for 24 weeks. The primary end point for the trial was the International Myositis Assessment and Clinical Studies definition of improvement. Secondary end points included safety, tolerability, steroid-sparing as well as the 2016 American College of Rheumatology (ACR)/European League Against Rheumatism myositis response criteria (EULAR) RESULTS: Ten of the 11 enrolled subjects (6 DM, 4 PM) completed the study. Seven of 10 met the primary end point of efficacy at a median of 8 weeks. There was a significant decrease in prednisone dose from baseline to conclusion (18.5 (15.7) vs 2.3 (3.2); P<0.01). Most individual CSMs improved at week 24 compared with the baseline, with the muscle strength improving by >10% and the physician global by >40%. RCI was considered safe and tolerable. No patient developed significant weight gain or an increase of haemoglobin A1c or cushingoid features.
Treatment with RCI was effective in 70% of patients, safe and tolerable, and led to a steroid dose reduction in patients with adult myositis refractory to glucocorticoid and traditional immunosuppressive drugs.
NCT01906372; Results.
评估储存型促皮质素注射（RCI）在一项开放性临床试验中的疗效、安全性、耐受性和激素节约作用，用于治疗难治性成人多发性肌炎（PM）和皮肌炎（DM）。
由两个中心招募患有难治性 PM 和 DM 的成年人。纳入标准包括定义为糖皮质激素和/或≥1 种免疫抑制剂治疗失败的难治性疾病，以及定义为存在显著肌肉无力和≥2 项额外异常核心集测量指标（CSM）或至少 3 项其他异常 CSM 的皮肤 10cm 视觉模拟量表评分≥3cm 的活动性疾病。所有患者接受 RCI 80 单位皮下注射，每周 2 次，共 24 周。试验的主要终点是国际肌炎评估和临床研究的改善定义。次要终点包括安全性、耐受性、激素节约以及 2016 年美国风湿病学会（ACR）/欧洲抗风湿病联盟肌炎反应标准（EULAR）。
11 名入组患者中的 10 名（6 名 DM，4 名 PM）完成了研究。10 名患者中有 7 名在中位数为 8 周时达到了疗效的主要终点。与基线相比，泼尼松剂量在研究结束时显著下降（18.5（15.7）vs 2.3（3.2）；P<0.01）。与基线相比，24 周时大多数个体 CSM 均有所改善，肌肉力量提高>10%，医生总体评估提高>40%。RCI 被认为是安全且耐受的。没有患者出现显著体重增加或血红蛋白 A1c 或库欣特征增加。
RCI 治疗在 70%的患者中有效，安全且耐受，并导致糖皮质激素和传统免疫抑制剂治疗难治性成人肌炎患者的激素剂量减少。
NCT01906372；结果。
