Rapamycin versus methotrexate in early diffuse systemic sclerosis: results from a randomized, single-blind pilot study.
Rapamycin versus methotrexate in early diffuse systemic sclerosis: results from a randomized, single-blind pilot study.
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DOI：
10.1002/art.24986
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发表时间：
2009-12
影响因子：
--
通讯作者：
Clements, Philip J.
中科院分区：
文献类型：
--
作者：
Su, Tien-I Karleen;Khanna, Dinesh;Furst, Daniel E.;Danovitch, Gabriel;Burger, Christina;Maranian, Paul;Clements, Philip J.
为了评估雷帕霉素（RAPA）治疗弥漫性系统性硬化症（SSc）的安全性和有效性，在一项为期48周的单盲研究中，18例≤5年的弥漫性系统性硬化症患者随机接受RAPA或甲氨蝶呤（MTX）治疗。比较两组患者临床及实验室指标的异常情况。通过比较基线和48周的评估来评估研究药物的潜在疗效，包括改进的罗德曼皮肤评分（mRSS）和健康评估问卷残疾指数。两组患者的基线特征相似（N = 9）。在RAPA组中，1例从未服用研究药物的患者被排除在分析之外。每组均有3例患者退出研究：2例与治疗相关（与RAPA相关的严重高甘油三酯血症；与MTX相关的全血细胞减少症），4例与ssc相关。高甘油三酯血症是与RAPA相关的最显著的副作用，但通常耐受性良好且可治疗。其他药物不良反应的发生率和严重程度在两组之间具有可比性。各组mRSS评分较基线均有显著改善（P < 0.05）。在RAPA组中，患者总体评估显示较基线有显著改善，而强制肺活量较基线下降。两组患者48周时的疾病活动度评分及其与基线相比的变化无显著差异。RAPA在一组选定的硬皮病患者中具有合理的安全性。需要更大规模的试验来评估其对早期弥漫性SSc的疗效。
To assess the safety and efficacy of rapamycin (RAPA) in the treatment of diffuse systemic sclerosis (SSc) Eighteen patients with ≤5 years of diffuse SSc were randomized to receive RAPA or methotrexate (MTX) in a single-blind, 48-week study. Abnormalities in clinical and laboratory parameters were compared between the two treatment groups. The potential efficacy of the study drugs was evaluated by comparing the baseline and 48-week assessments, including the modified Rodnan Skin score (mRSS) and the Health Assessment Questionnaire Disability Index. Baseline patient characteristics were similar in both groups (N = 9 in each). One patient who never took the study drug in the RAPA group was excluded from analysis. Three patients in each group withdrew from the study: two were treatment-related (severe hypertriglyceridemia associated with RAPA; pancytopenia associated with MTX) and four were SSc-related. Hypertriglyceridemia was the most notable side-effect associated with RAPA, but it was generally well-tolerated and treatable. The incidence and severity of other adverse drug reactions were comparable between two groups. Within each group, the mRSS score showed a significant improvement from baseline (P < 0.05). In the RAPA group, the patient global assessment showed a significant improvement from baseline while forced vital capacity declined from baseline. The disease activity scores at 48 weeks and their changes from baseline were not significantly different between two groups. RAPA has a reasonable safety profile in a select group of scleroderma patients. Larger trials are needed to assess its efficacy in early diffuse SSc.
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影响因子：
4
作者：
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通讯作者：
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影响因子：
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