Adverse Events during the Scleroderma Lung Study
Adverse Events during the Scleroderma Lung Study
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DOI：
10.1016/j.amjmed.2010.12.009
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发表时间：
2011-05-01
影响因子：
5.9
通讯作者：
Schraufnagel, Dean E.
中科院分区：
文献类型：
--
作者：
Furst, Daniel E.;Tseng, Chi-Hong;Schraufnagel, Dean E.
背景：硬皮病肺研究（SLS）是一项为期1年的随机对照试验，口服环磷酰胺治疗硬皮病相关肺泡炎。结论是口服环磷酰胺减缓了用力肺活量的下降（预期百分比），对呼吸困难、皮肤变化和系统性硬化症的一些生活质量指标有有益的影响。我们现在报告了环磷酰胺在治疗期间和治疗完成后一年的毒性的深入评估，在此期间调查人员仍然对治疗任务不知情。方法：口服环磷酰胺与安慰剂为期一年的双盲随机对照试验，随访1年。将不良事件（ae）制成表格，描述并使用描述性统计（例如，平均值和中位数）和t、Wilcoxon秩和、卡方或Fisher精确检验进行比较。结果：在第1年中，环磷酰胺（CYC）治疗相关的总体不良事件发生在环磷酰胺（CYC）治疗的患者中更频繁（CYC的总体不良事件= 154事件，而安慰剂= 60事件，P = 0.002），特别是轻度至中度白细胞减少症（CYC = 19例，而安慰剂= 0例；P = 0.002）
BACKGROUND: The Scleroderma Lung Study (SLS) was a 1-year, randomized, controlled trial of oral cyclophosphamide for scleroderma-related pulmonary alveolitis. It concluded that oral cyclophosphamide slowed the decline in the forced vital capacity (% predicted) and had a beneficial effect on dyspnea, skin changes, and several quality of life measures of systemic sclerosis. We now report an in-depth assessment of the toxicity of cyclophosphamide during the year of therapy and the year after therapy was completed, during which time the investigators were still masked to the treatment assignment.METHODS: One-year, double-blind, randomized controlled trial of oral cyclophosphamide versus placebo with 1-year masked follow-up. Adverse events (AEs) were tabulated, described, and compared using descriptive statistics (eg, mean and median) and t, Wilcoxon rank sum, chi-squared, or Fisher's exact tests as appropriate.RESULTS: During year 1, treatment-related overall AEs occurred more frequently in cyclophosphamide (CYC)-treated patients (overall AEs for CYC = 154 events vs placebo = 60 events; P = 0.002), and especially for mild to moderate leukopenia (CYC = 19 subjects vs placebo = 0 subjects; P