Methotrexate Treatment in Juvenile Localized Scleroderma A Randomized, Double-Blind, Placebo-Controlled Trial
Methotrexate Treatment in Juvenile Localized Scleroderma A Randomized, Double-Blind, Placebo-Controlled Trial
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DOI：
10.1002/art.30264
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发表时间：
2011-07-01
影响因子：
--
通讯作者：
Perilongo, Giorgio
中科院分区：
文献类型：
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作者：
Zulian, Francesco;Martini, Giorgia;Perilongo, Giorgio
目标。青少年局限性硬皮病是一种慢性进行性皮肤纤维性疾病，会导致永久性残疾和审美损害。本研究旨在评价甲氨蝶呤(MTX)治疗青少年局限性硬皮病的安全性和有效性。在这项双盲研究中，活动期青少年局限性硬皮病患者被随机(2：1)接受口服MTX(15 mg/m(2)，最多20 mg)或安慰剂每周一次，持续12个月或直到治疗失败。两组均在前3个月口服泼尼松(1 mg/kg/d，最大50 mg)。采用红外热像仪和皮肤评分率(SSR)评估计算机评分系统，对靶病变进行临床评估。对治疗的反应被定义为没有新的病变、SSR 1或病变温度升高。所有分析均对意向治疗人群进行。在筛选的85名患者中，70名(年龄6-17岁)被随机分配(46名接受MTX治疗，24名接受安慰剂治疗)。平均病程2.3年。在所有患者的初步反应后，接受甲氨蝶呤治疗的15名患者(32.6%)和安慰剂治疗的17名患者(70.8%)病情复发(P<；0.005)。接受MTX治疗的患者中有3例(6.5%)出现新的病变，而安慰剂治疗的患者中有4例(16.7%)。平均SSR在MTX组从1下降到0.79，在安慰剂组从1上升到1.1，平均靶点温度在MTX组下降了44.4%，而在安慰剂组下降了12.1%。MTX组26名患者(56.5%)和安慰剂组11名患者(45.8%)出现与治疗相关的轻微副作用。没有一种副作用严重到需要停止治疗。结果表明，甲氨蝶呤治疗青少年局限性硬皮病疗效确切，耐受性良好。
Objective. Juvenile localized scleroderma is a chronic progressive fibrotic disorder of the skin that causes permanent disability and aesthetic damage. This study was undertaken to assess the safety and efficacy of methotrexate (MTX) in the treatment of juvenile localized scleroderma.Methods. In this double-blind study, patients with active juvenile localized scleroderma were randomized (2:1) to receive oral MTX (15 mg/m(2), maximum 20 mg) or placebo once weekly, for 12 months or until treatment failure. Both groups received oral prednisone (1 mg/kg/day, maximum 50 mg) for the first 3 months. A target lesion was evaluated clinically, with infrared thermography and using a computerized scoring system with skin score rate (SSR) evaluation. Response to treatment was defined as the absence of new lesions, SSR 1, or increased lesion temperature. All analyses were done on the intent-to-treat population.Results. Of the 85 patients screened, 70 (ages 6-17 years) were randomized (46 to the MTX group, 24 to the placebo group). The mean disease duration was 2.3 years. After an initial response in all patients, disease relapsed in 15 MTX-treated patients (32.6%) and 17 placebo-treated patients (70.8%) (P < 0.005). New lesions appeared in 3 MTX-treated patients (6.5%) versus 4 placebo-treated patients (16.7%). The mean SSR decreased from 1 to 0.79 in the MTX group and increased from 1 to 1.1 in the placebo group, and the mean target lesion temperature decreased by 44.4% in the MTX group versus 12.1% in the placebo group. Twenty-six patients in the MTX group (56.5%) and 11 patients in the placebo group (45.8%) developed mild side effects related to treatment. None of the side effects were severe enough to necessitate treatment discontinuation.Conclusion. Our findings indicate that MTX is efficacious in the treatment of juvenile localized scleroderma and is well tolerated.