Mycophenolate mofetil: a possible therapeutic agent for children with juvenile dermatomyositis.

Mycophenolate mofetil: a possible therapeutic agent for children with juvenile dermatomyositis.
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DOI:
10.1002/acr.20269
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发表时间:
2010-10
影响因子:
4.7
通讯作者:
Pachman, Lauren M.
Pachman, Lauren M.
中科院分区:
医学2区
文献类型:
--
作者:
Rouster-Stevens, Kelly A.;Morgan, Gabrielle A.;Wang, Deli;Pachman, Lauren M.

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确定霉酚酸酯(MMF)是否能减少幼年型皮肌炎(JDM)患儿的皮肤和肌肉疾病活动,允许减少皮质类固醇剂量。对50例JDM儿童的回顾性数据审查确定了以下内容:38例(76%)女孩,39例(78%)白色,10例(20%)西班牙裔,1例(2%)黑人,平均年龄12.2 ± 5.0岁,接受了12个月的MMF治疗。获得MMF剂量和频率、感染类型、白色血细胞计数(WBC)、皮质类固醇剂量和经验证的疾病活动性评分(DAS)(皮肤[DAS-S]、肌肉[DAS-M]的分项评分)。开始MMF治疗后12个月,平均DAS-S从5.24 ± 0.29降至3.72 ± 0.29(p=0.001),DAS-M从2.44 ± 0.39降至1.17 ± 0.28(p=0.002)。泼尼松剂量从0.39 mg/kg/天± 0.06 mg降至0.23 mg/kg/天± 0.02 mg(p=0.0001),恢复线性生长(p=0.008)。接受MMF治疗的12个月内,WBC/淋巴细胞计数保持不变。在26名JDM儿童中评估感染率,并在开始使用MMF前随访12个月,并与随后使用MMF的12个月进行比较。治疗前和MMF前6个月之间无显著差异(p=0.44),但感染率在7-12个月内下降(p=0.001)。霉酚酸酯似乎是值得考虑作为一个额外的治疗方式,治疗儿童JDM。这些数据表明,使用霉酚酸酯可以减少皮肤和肌肉疾病的活动,并节省类固醇。霉酚酸酯似乎耐受性良好,但应监测患者的感染情况。
To determine if mycophenolate mofetil (MMF) diminished skin and muscle disease activity in children with juvenile dermatomyositis (JDM) permitting decrease in corticosteroid dosage. Retrospective data review for 50 JDM children identified the following: 38 (76%) girls, 39 (78%) white, 10 (20%) Hispanic, 1 (2%) black, mean age 12.2 ± 5.0 years who had been given MMF for 12 months. MMF dose and frequency, type of infection, white blood cell count (WBC), corticosteroid dose and validated disease activity score (DAS), (sub scores for skin [DAS-S], muscle [DAS-M]), were obtained. Twelve months after start of MMF, mean DAS-S decreased from 5.24 ± 0.29 to 3.72 ± 0.29 (p=0.001) and DAS-M dropped from 2.44 ± 0.39 to 1.17 ± 0.28 (p=0.002). Prednisone dose decreased from 0.39 mg/kg/day ± 0.06 mg to 0.23mg/kg/day ± 0.02 mg (p=0.0001), with resumption of linear growth (p=0.008). The WBC/lymphocyte count was unchanged over 12 months on MMF. Infection rate was assessed in a subset of 26 children with JDM followed for 12 months before start of MMF and compared with ensuing 12 months on MMF. There was no significant difference between pretreatment and first 6 months of MMF (p=0.44), but infection rate dropped in months 7–12 (p=0.001). MMF appears to be worthy of consideration as an additional therapeutic modality for treatment of children with JDM. These data suggest that use of MMF decreases skin and muscle disease activity and is steroid sparing. MMF appears to be well tolerated, but patients should be monitored for infection.
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发表时间: 2008-02-01
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DOI: 10.1097/00005792-196607000-00001
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