Long-term experience of mycophenolate mofetil for treatment of diffuse cutaneous systemic sclerosis
Long-term experience of mycophenolate mofetil for treatment of diffuse cutaneous systemic sclerosis
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DOI：
10.1136/ard.2010.142000
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发表时间：
2011-06-01
影响因子：
27.4
通讯作者：
Hummers, Laura K.
中科院分区：
文献类型：
--
作者：
Le, Elizabeth N.;Wigley, Fredrick M.;Hummers, Laura K.
背景免疫抑制治疗可能潜在地改变硬皮病的自然病程。目的分析霉酚酸酯(MMF)治疗活动性弥漫性皮肤硬皮病的经验。方法比较3个月、6个月、9个月和12个月的MMF队列中平均改良Rodnan皮肤评分(MRSS)的变化，并与历史对照组进行合并分析，这些临床试验包括重组人松弛素、D-青霉胺和口服牛I型胶原蛋白。6个月时MMF3.05+/-7.4vs松弛素组-4.83+/-6.99，P=0.059；12个月时显著降低(MMF7.59+/-10.1vs D-青霉胺-2.47+/-8.6，p<；0.001；胶原-3.4+/-7.12，p=0.002)。与基线相比，一般情况和肌肉严重程度评分以及生活质量指标也有所改善。肺功能保持稳定。结论MMF对弥漫性硬皮病患者的皮肤病有一定的疗效，但其作用尚需前瞻性研究。
Background Immunosuppressive therapy may potentially alter the natural disease course of scleroderma. There have been reports of using mycophenolate mofetil (MMF) for the treatment of scleroderma skin disease.Objective To analyse the experience of using MMF for the treatment of active diffuse cutaneous scleroderma.Methods The authors compared the change in mean modified Rodnan skin scores (mRSS) in an MMF cohort at baseline with scores at 3, 6, 9 and 12 months and with those of historical controls from a pooled analysis of three multicentre randomised clinical trials of recombinant human relaxin, D-penicillamine and oral bovine type I collagen.Results Improvement in mRSS after treatment with MMF compared with baseline was seen as early as 3 months and continued through the 12-month follow-up. The mRSS of the MMF cohort was not different from that of the historical controls at 6 months (MMF -3.05 +/- 7.4 vs relaxin -4.83 +/- 6.99, p=0.059), but was significantly lower at 12 months (MMF -7.59 +/- 10.1 vs D-penicillamine -2.47 +/- 8.6, p < 0.001; collagen -3.4 +/- 7.12, p=0.002). General and muscle severity scores and quality of life measures also improved compared with baseline. Pulmonary function remained stable.Conclusions MMF may benefit skin disease in patients with diffuse scleroderma, but prospective studies are required to determine its role.