Department of Nephrology, Ruijin Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China.
Am J Nephrol. 2013;37(1):84-90. doi: 10.1159/000346256. Epub 2013 Jan 22.
The efficacy and safety of tacrolimus (TAC) and cyclophosphamide (CTX) were prospectively examined in steroid-dependent or steroid-resistant primary focal segmental glomerulosclerosis (FSGS).
Patients with biopsy-proven FSGS were enrolled and randomly divided into two groups: CTX and TAC. Patients treated with CTX (0.5-0.75 g/m(2)·month, i.v.) received prednisone at 0.8 mg/kg·day, while patients treated with TAC (0.1 mg/kg·day) received prednisone at 0.5 mg/kg·day. The plasma concentration of TAC was monitored and maintained at 5-10 ng/ml. After a 6-month treatment the patients were evaluated. Patients with complete remission (CR) and partial remission (PR) continued the treatment for 12 months with the dose tapered, whereas the patients with no response were excluded from the study and underwent an alternative treatment.
A total of 33 patients were recruited and 27 completed the 12-month follow-up. The TAC-treated patients (n = 15) showed a quick remission. The initial remission time averaged 1.23 ± 0.21 versus 2.21 ± 0.77 months in the CTX group (n = 18), but no significant difference was achieved (p > 0.05). At 6 months, the two groups showed a similar outcome. Ten patients from each group showed remission (7 CR and 3 PR). At 12 months, the CTX group had 9 CR and 3 PR while the TAC group had 6 CR and 5 PR. Remission rates in TAC tended to be higher than that in CTX, but there was no difference. CTX patients had a high prevalence of infections (50.0 vs. 13.3% in TAC, p < 0.05). In contrast, TAC-treated patients showed a high incidence of hyperglycemia (26.7 vs. 0.0% in CTX, p < 0.05).
These results suggest that CTX and TAC had a similar efficacy in steroid-dependent and steroid-resistant FSGS as manifested by reduced proteinuria, improved serum albumin level and renal function.
在激素依赖性或激素抵抗性局灶节段性肾小球硬化症（FSGS）患者中，前瞻性地评估了他克莫司（TAC）和环磷酰胺（CTX）的疗效和安全性。
入组经活检证实为 FSGS 的患者，并将其随机分为两组：CTX 组和 TAC 组。CTX 组患者（每月 0.5-0.75g/m²，静脉注射）接受泼尼松龙 0.8mg/kg·d，而 TAC 组患者（0.1mg/kg·d）接受泼尼松龙 0.5mg/kg·d。监测 TAC 的血药浓度并维持在 5-10ng/ml。治疗 6 个月后对患者进行评估。完全缓解（CR）和部分缓解（PR）的患者继续接受 12 个月的治疗，剂量逐渐减少，而无反应的患者则被排除在研究之外，并接受替代治疗。
共纳入 33 例患者，27 例完成 12 个月随访。TAC 治疗组（n=15）患者缓解迅速。初始缓解时间平均为 1.23±0.21 个月，而 CTX 组（n=18）为 2.21±0.77 个月，但差异无统计学意义（p>0.05）。6 个月时，两组的结果相似。每组各有 10 例患者缓解（7 例 CR 和 3 例 PR）。12 个月时，CTX 组 9 例 CR 和 3 例 PR，TAC 组 6 例 CR 和 5 例 PR。TAC 组的缓解率似乎高于 CTX 组，但差异无统计学意义。CTX 组感染发生率较高（50.0%比 TAC 组的 13.3%，p<0.05）。相比之下，TAC 治疗组患者发生高血糖的比例较高（26.7%比 CTX 组的 0.0%，p<0.05）。
这些结果表明，CTX 和 TAC 在激素依赖性和激素抵抗性 FSGS 中的疗效相似，表现为蛋白尿减少、血清白蛋白水平和肾功能改善。
