Department of Pediatrics, University of Padua, Padua, Italy.
J Am Acad Dermatol. 2012 Dec;67(6):1151-6. doi: 10.1016/j.jaad.2012.03.036. Epub 2012 May 30.
Recent studies report that methotrexate (MTX) is beneficial in the treatment of juvenile localized scleroderma (JLS) but little is known about its long-term effectiveness.
We assessed the therapeutic role of MTX in children with JLS who were followed up for a prolonged period.
A cohort of patients with JLS, previously enrolled in a double-blind, randomized controlled trial and treated with oral MTX (15 mg/m(2)/wk) and prednisone (1 mg/kg/d, maximum 50 mg) for the first 3 months, were prospectively followed up. Lesions were evaluated clinically, with infrared thermography, and by a computerized skin score. Response to treatment was defined as: (1) no new lesions; (2) skin score rate less than 1; and (3) decrease in lesion temperature by at least 10% compared with baseline. Clinical remission (CR) on medication was defined when response was maintained, on treatment, for at least 6 months, and complete CR when response was maintained, without treatment, for at least 6 months.
Of 65 patients treated with MTX, 48 (73.8%) were responders, 10 (15.4%) relapsed by 24 months since MTX start, and 7 (10.8%) were lost to follow-up. Among the responders, 35 (72.9%) maintained CR for a mean of 25 months and 13 (27.1%) were in CR on medication. Adverse effects seen in 28 patients (48.3%) were generally mild and never required treatment discontinuation.
The use of objective measures not widely available, such as infrared thermography and computerized skin score, makes it difficult to compare data from previous studies.
Long-term MTX therapy is beneficial and well tolerated for JLS.
最近的研究报告称，甲氨蝶呤（MTX）在治疗青少年局限性硬皮病（JLS）方面有益，但对其长期疗效知之甚少。
我们评估了在接受长期随访的 JLS 儿童中 MTX 的治疗作用。
一组 JLS 患者，先前参加过一项双盲、随机对照试验，在头 3 个月接受口服 MTX（15mg/m2/周）和泼尼松（1mg/kg/d，最大 50mg）治疗，前瞻性随访。通过临床、红外热成像和计算机皮肤评分评估病变。治疗反应定义为：（1）无新病变；（2）皮肤评分率小于 1；（3）与基线相比，病变温度下降至少 10%。药物治疗时的临床缓解（CR）定义为：在治疗期间，反应持续至少 6 个月；完全缓解（CR）定义为在没有治疗的情况下，反应持续至少 6 个月。
在接受 MTX 治疗的 65 例患者中，48 例（73.8%）为应答者，10 例（15.4%）在 MTX 开始后 24 个月复发，7 例（10.8%）失访。在应答者中，35 例（72.9%）平均持续 25 个月 CR，13 例（27.1%）在药物治疗时处于 CR。28 例患者（48.3%）出现不良反应，一般较轻，从未需要停药。
使用的客观措施（如红外热成像和计算机皮肤评分）不广泛，难以比较以前研究的数据。
长期 MTX 治疗对 JLS 有益且耐受良好。
