Division of Nephrology, Department of Pediatrics & Communicable Diseases, University of Michigan, Ann Arbor, Michigan, USA.
Kidney Int. 2011 Oct;80(8):868-78. doi: 10.1038/ki.2011.195. Epub 2011 Jul 6.
This NIH-funded multicenter randomized study of focal segmental glomerulosclerosis (FSGS) treatment compared the efficacy of a 12-month course of cyclosporine to a combination of oral pulse dexamethasone and mycophenolate mofetil in children and adults with steroid-resistant primary FSGS. Of the 192 patients enrolled, 138 were randomized to cyclosporine (72) or to mycophenolate/dexamethasone (66). The primary analysis compared the levels of an ordinal variable measuring remission during the first year. The odds ratio (0.59) for achieving at least a partial remission with mycophenolate/dexamethasone compared to cyclosporine was not significant. Partial or complete remission was achieved in 22 mycophenolate/dexamethasone- and 33 cyclosporine-treated patients at 12 months. The main secondary outcome, preservation of remission for 26 weeks following cessation of treatment, was not significantly different between these two therapies. During the entire 78 weeks of study, 8 patients treated with cyclosporine and 7 with mycophenolate/dexamethasone died or developed kidney failure. Thus, our study did not find a difference in rates of proteinuria remission following 12 months of cyclosporine compared to mycophenolate/dexamethasone in patients with steroid-resistant FSGS. However, the small sample size might have prevented detection of a moderate treatment effect.
这项由美国国立卫生研究院（NIH）资助的多中心、随机研究比较了局灶节段性肾小球硬化症（FSGS）治疗中，环孢素治疗 12 个月与口服脉冲地塞米松和霉酚酸酯联合治疗在儿童和成人激素抵抗性原发性 FSGS 患者中的疗效。在纳入的 192 例患者中，138 例随机分为环孢素（72 例）或霉酚酸酯/地塞米松（66 例）组。主要分析比较了第一年期间衡量缓解程度的有序变量水平。与环孢素相比，霉酚酸酯/地塞米松达到部分缓解的优势比（0.59）无显著差异。12 个月时，22 例霉酚酸酯/地塞米松组和 33 例环孢素组患者部分或完全缓解。这两种治疗方法在治疗停止后 26 周的缓解持续时间这一主要次要结局上没有显著差异。在整个 78 周的研究期间，8 例接受环孢素治疗的患者和 7 例接受霉酚酸酯/地塞米松治疗的患者死亡或发生肾衰竭。因此，我们的研究没有发现激素抵抗性 FSGS 患者接受 12 个月环孢素治疗后与霉酚酸酯/地塞米松治疗相比蛋白尿缓解率的差异。然而，小样本量可能导致无法检测到中度治疗效果。
