环磷酰胺与霉酚酸酯治疗小儿难治性肾病综合征47例回顾性分析

目的：比较环磷酰胺（ CTX）与霉酚酸酯（ MMF）治疗小儿难治性肾病综合征（ RNS）的临床疗效。方法收集2004年1月至2009年1月济南军区第一五九中心医院儿科收治的小儿RNS的临床资料。按照用药将患者分为CTX组和MMF组，比较两组患儿缓解率、尿蛋白转阴时间、肾功能变化、3年内复发和不良反应发生情况。结果研究最终纳入47例，CTX组21例，MMF组26例，两组性别、年龄、病程比较差异无统计学意义。治疗结束时CTX组和MMF组的完全缓解率分别为28.57%和42.31%，差异无统计学意义（χ2=0.949，P=0.330），且两组缓解情况分布差异无统计学意义（Z=-0.538，P=0.590）。两组肾功能指标均有好转（P均﹤0.05），但MMF组24 h尿蛋白、白蛋白、血肌酐水平优于CTX组（P均﹤0.05）。MMF组尿蛋白转阴时间为（23.9±12.36）d，CTX组为（73.67±40.33）d，差异有统计学意义（P=0.002）。治疗结束3年内，CTX组复发8例，MMF组6例，差异无统计学意义（χ2=1.253，P=0.263）。治疗过程中两组均发生不良反应，多为食欲不振、恶心、呕吐，未发生严重不良反应。结论小样本临床研究中，在小儿RNS的尿蛋白转阴时间、改善肾功能方面MMF优于CTX，但两者均有较好的缓解作用，且完全缓解率、复发率、不良反应发生情况差异无统计学意义。

A retrospective analysis of CTX or MMF in the treatment of children refractory nephrotic syndrome

Objective To compare the clinical effects of CTX and MMF on children with refractory nephrotic syndrome （RNS）. Methods The clinical data of children with RNS admitted in the 159th central hospital of Jinan military region from January 2004 to January 2009 were collected. According to different medications,the patients were divided into the CTX group and MMF group. The remission rate,urine protein clearance time,renal function changes,recurrence within 3 years and adverse reactions were compared between the two groups. Results The study ultimately included 47 cases,with 21 cases in CTX group and 26 cases in MMF group. There were no significant differences in the gender,age,disease duration between the two groups. The complete remission rate in the MMF group and CTX group at the end of treatment was 28. 57% and 42. 31% respectively, but the difference was not significant（χ2 =0. 949,P=0. 330）. And the overall remission of the two groups has no significant difference（Z= -0. 538,P=0. 590）. After treatment,the indicators of kidney function improved compared with those before treatment（P﹤0. 05）. But the levels of 24 h urine protein,Alb,Scr of the MMF group were superior to those of the CTX group （P﹤0. 05）. The urine protein clearance time was（23. 9 ＆#177; 12. 36）days in the MMF group,and（73. 67 ＆#177; 40. 33）days in the CTX group,the difference was significant（ P=0. 002）. During the 3 years after the treatment,8 patients in CTX group relapsed, and 6 patients in MMF group relapsed,the difference was not significant（χ2 = 1. 253,P=0. 263）. During the treatment,both groups showed adverse reactions,and the main adverse reactions were anorexia,nausea and vomiting,but there were no serious adverse reactions. Conclusion In the small sample clinical study,MMF is superior to CTX in the urine protein clearance time, improving the renal function of children with RNS,but both of MMF and CTX can alleviate the disease,and the complete remis-sion rates,recurrence rates and incidences of adv