Clinical trials treating focal segmental glomerulosclerosis should measure patient quality of life.
Clinical trials treating focal segmental glomerulosclerosis should measure patient quality of life.
复制标题
DOI：
10.1038/ki.2010.485
复制
发表时间：
2011-03
影响因子：
19.6
通讯作者：
中科院分区：
文献类型：
--
作者：
类固醇抵抗性原发性局灶节段性肾小球硬化症 (FSGS) 患者的最佳治疗仍存在争议。本报告描述了参加 FSGS 临床试验的患者的初始研究设计、基线特征和生活质量，这是一项针对这种肾小球病的大型多中心随机研究，比较了 12 个月的环孢素治疗方案与吗替麦考酚酯和口服地塞米松的组合。年龄在 2-40 岁之间、估计肾小球滤过率 >40 ml/min 每 1.73 m2、第一次晨尿蛋白与肌酐比值超过 1、并且对皮质类固醇有抵抗力的患者符合资格。主要结局是随机分组后 52 周内蛋白尿完全或部分缓解。总共筛选了 192 名患者，其中 138 名被随机分配接受治疗。种族分布为 53 名黑人、78 名白人和 7 名其他种族。通过自我报告或家长代理报告，138 名患者中有 26 名被确定为西班牙裔。基线肾小球滤过率为112.4（76.5，180.0）ml/min/1.73 m2，尿蛋白为4.0（2.1，5.3）g/g。总体而言，FSGS 患者的生活质量低于健康对照，与终末期肾病患者相似。因此，FSGS 对生活质量的影响是显着的，所有试验都应包括该测量。
Optimal therapy of patients with steroid-resistant primary focal segmental glomerulosclerosis (FSGS) remains controversial. This report describes the initial study design, baseline characteristics, and quality of life of patients enrolled in the FSGS Clinical Trial, a large multicenter randomized study of this glomerulopathy comparing a 12-month regimen of cyclosporine to the combination of mycophenolate mofetil and oral dexamethasone. Patients with age ranging 2–40 years, with an estimated glomerular filtration rate >40 ml/min per 1.73 m2, a first morning urine protein-to-creatinine ratio over one, and resistant to corticosteroids were eligible. The primary outcome was complete or partial remission of proteinuria over 52 weeks after randomization. In all, 192 patients were screened, of whom 138 were randomized for treatment. Ethnic distributions were 53 black, 78 white, and 7 other. By self- or parent-proxy reporting, 26 of the 138 patients were identified as Hispanic. The baseline glomerular filtration rate was 112.4 (76.5, 180.0) ml/min per 1.73 m2, and urine protein was 4.0 (2.1, 5.3) g/g. Overall, the quality of life of the patients with FSGS was lower than healthy controls and similar to that of patients with end-stage renal disease. Thus, the impact of FSGS on quality of life is significant and this measurement should be included in all trials.