A randomized, pilot trial of etanercept in dermatomyositis.
A randomized, pilot trial of etanercept in dermatomyositis.
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皮肌炎中依那耐酸的随机试验试验。
DOI：
10.1002/ana.22477
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发表时间：
2011-09
影响因子：
11.2
通讯作者：
Downing, Sharon
中科院分区：
文献类型：
--
作者：
Amato, Anthony A.;Tawil, Rabi;Kissel, John;Barohn, Richard;McDermott, Michael P.;Pandya, Shree;King, Wendy;Smirnow, Alexis;Annis, Christine;Roe, Kristen;Tawil, Rabi;McDermott, Michael P.;Janciuras, Joanne;Dilek, Nuran;Martens, William B.;Eastwood, Eileen;Amato, Anthony;Cochrane, Thomas;Donlan, Merideth;Chused, Samantha;Roe, Kristen;Barohn, Richard;Dimachkie, Mazen;Aires, Daniel J.;Latinis, Kevin M.;Herbelin, Laura;Michaels, Hiwot;Cupler, Edward;Deodhar, Atul;Simpson, Eric;Burusnukul, Prinyarat;Edgar, Eric;Serdar, Andrea;Brennan, Thomas;Gance, Kathryn;Kissel, John;Freimer, Miriam L.;Hackshaw, Kevin V.;Lawson, Victoria;King, Wendy M.;Bartlett, Amy;Wolfe, Gil;Nations, Sharon;McLin, Rhonda;Gorham, Nina;Briemberg, Hannah;Chapman, Kristine M.;Dutz, Jan P.;Wilson, Judy;Varelas, Franca;Wagner, Kathryn;Stine, Lisa Christopher;Anhalt, Grant James;Meyerle, Jon H.;Swain, Jennifer O.;Brock-Simmons, Regina;Weiss, Michael;Distad, B. Jane;Lin, John;Haug, Joanna A.;Downing, Sharon
本初步研究的目的是评估（1）依那西普治疗皮肌炎（DM）的安全性和耐受性，（2）强制泼尼松减量的可行性和安全性，以及（3）结局指标，包括国际肌炎评估临床研究组（IMACS）推荐的指标。我们在DM受试者中进行了一项随机、双盲、安慰剂对照的依那西普（每周50 mg SQ）试验，持续52周。受试者在研究的最初24周内按照标准化时间表逐渐减少泼尼松的耐受性。主要结果包括不良事件、从随机化到治疗失败（无法按计划停用泼尼松）的时间以及第24周后的平均泼尼松剂量。16例受试者随机分组，11例接受依那西普治疗，5例接受安慰剂治疗。治疗组间不良事件发生率无显著差异，但5例依那西普治疗组和1例安慰剂治疗组受试者发生皮疹恶化。接受安慰剂的所有5例受试者均为治疗失败（至治疗失败的中位时间为148天）。相比之下，依那西普组5/11例受试者成功停用泼尼松;该组至治疗失败的中位时间为358天（p = 0.0002）。第24周后，安慰剂组和依那西普组的平均泼尼松剂量中位数分别为29.2 mg/d和1.2 mg/d（p = 0.02）。IMACS和其他结果的措施表现出良好的重测信度（组内相关系数0.79 - 0.99）。治疗对功能结局无显著影响。在我们的研究中没有观察到重大的安全性问题和类固醇节约效应，这表明有必要进一步研究依那西普作为糖尿病的治疗。
The aims of this pilot study were to assess (1) the safety and tolerability of etanercept in dermatomyositis (DM), (2) the feasibility and safety of a forced prednisone taper, and (3) outcome measures, including those recommended by the International Myositis Assessment Clinical Study Group (IMACS). We conducted a randomized, double-blind, placebo-controlled trial of etanercept (50 mg SQ weekly) for 52 weeks in DM subjects. Subjects were tapered off prednisone in a standardized schedule as tolerated over the initial 24 weeks of the study. Principal outcomes included adverse events, time from randomization to treatment failure (inability to wean off prednisone on schedule), and average prednisone dosage after Week 24. Sixteen subjects were randomized, 11 to etanercept and 5 to placebo. There were no significant differences in adverse event rates between the treatment groups, although 5 etanercept-treated and 1 placebo-treated subjects developed worsening rash. All 5 subjects receiving placebo were treatment failures (median time to treatment failure 148 days). In contrast, 5/11 subjects in the etanercept arm were successfully weaned off prednisone; the median time to treatment failure in this group was 358 days (p = 0.0002). The median of the average prednisone dosage after Week 24 was 29.2 mg/day in the placebo group and 1.2 mg/day in the etanercept group (p = 0.02). IMACS and other outcome measures demonstrated excellent test-retest reliability (intraclass correlation coefficients 0.79 - 0.99). There was no significant treatment effect on functional outcome. The observation of no major safety concerns and a steroid-sparing effect in our study suggest that further investigation of etanercept as a treatment for DM is warranted.
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