Paediatric Rheumatology Unit, Department of Woman and Child Health, University of Padova, Padova, Italy.
Rheumatology (Oxford). 2021 Mar 2;60(3):1387-1391. doi: 10.1093/rheumatology/keaa392.
To investigate safety and efficacy of MMF in patients with severe or MTX-refractory juvenile localized scleroderma.
Consecutive juvenile localized scleroderma patients undergoing systemic treatment were included in a retrospective longitudinal study. Patients treated with MMF because they were refractory or intolerant to MTX (MMF-group) were compared with responders to MTX (MTX-group). Disease activity was assessed by Localized Scleroderma Cutaneous Assessment Tool and thermography. Disease course was established on the number of relapses and treatment changes. Relapse-free survival was examined by Kaplan-Meier analysis.
MMF and MTX groups included 22 and 47 patients, respectively. No significant difference in demographics, follow-up duration and treatment before diagnosis was observed between groups. The most represented clinical subtypes in the MMF-group were pansclerotic morphea and mixed subtype (P = 0.008 and P = 0.029, respectively), and linear scleroderma of the face in the MTX-group (P = 0.048). MMF was started because of MTX resistance (18 patients), relapse during MTX tapering/withdrawal (3 patients) and anaphylaxis to MTX (1 patient). After mean 9.4 years of follow-up, 90.9% of patients on MMF and 100% of those on MTX had inactive disease. No significant difference in relapse-free survival between the groups was found (P = 0.066, log-rank test), although MMF likely induced more persistent remission. MMF was well tolerated and combination of MMF and MTX did not increase its efficacy.
The present study adds strong evidence on the efficacy and tolerance of MMF in severe and/or MTX-refractory juvenile localized scleroderma. Further controlled studies are needed to prove its efficacy as first line treatment.
研究霉酚酸酯（MMF）治疗重症或 MTX 抵抗的青少年局限性硬皮病的安全性和疗效。
本研究纳入了接受系统性治疗的青少年局限性硬皮病患者进行回顾性纵向研究。因 MTX 抵抗或不耐受而接受 MMF 治疗的患者（MMF 组）与 MTX 治疗有效的患者（MTX 组）进行比较。采用局限性硬皮病皮肤评估工具和热成像来评估疾病活动度。根据复发次数和治疗变化来确定疾病过程。通过 Kaplan-Meier 分析来检查无复发生存率。
MMF 组和 MTX 组分别纳入 22 例和 47 例患者。两组在人口统计学、随访时间和诊断前治疗方面无显著差异。MMF 组最常见的临床亚型为泛发性硬斑病和混合型（P=0.008 和 P=0.029），而 MTX 组最常见的是面中部线状硬皮病（P=0.048）。MMF 开始应用于 MTX 抵抗（18 例）、MTX 减量/停药期间复发（3 例）和 MTX 过敏（1 例）。在平均 9.4 年的随访后，90.9%的 MMF 治疗患者和 100%的 MTX 治疗患者疾病处于无活动状态。两组之间无复发生存率无显著差异（P=0.066，对数秩检验），尽管 MMF 可能诱导更持久的缓解。MMF 耐受性良好，MMF 与 MTX 联合使用并未增加其疗效。
本研究为 MMF 治疗重症和/或 MTX 抵抗的青少年局限性硬皮病的疗效和耐受性提供了有力证据。需要进一步的对照研究来证明其作为一线治疗的疗效。
