环磷酰胺与环孢霉素A治疗儿童激素耐药型肾病综合征临床疗效比较

Comparison of cyclophosphamide and cyclosporine in the treatment of steroid-resistant idiopathic nephrotic syndrome in children

目的：比较环磷酰胺（cyclophosphamide,CTX）与环孢霉素A（cyclosporine A,CsA）治疗儿童激素耐药型肾病综合征（steroid-resistant idiopathic nephrotic syndrome,SRNS）的临床疗效。方法：回顾性研究2001年1月-2006年12月我院收治并完整随访的SRNS37例,男女比例为5∶1,平均年龄5.0岁（4个月-12岁）。肾脏病理为微小病变（minimal change disease,MCD）28例,非微小病变9例,其中局灶节段性肾小球硬化（focal segmental glomerulosclerosis,FSGS）7例,系膜增生性肾小球肾炎（mesangial proliferative glomerulonephritis,MsPGN）2例。其中CTX治疗组30例（CTX冲击剂量500mg/m^2）,CsA治疗组21例[CsA初始剂量3-5mg/（kg·d）]。期间联合泼尼松龙口服并逐渐减量。对两组患儿随访12个月内疗效及副作用进行比较。结果：（1）随访12个月CTX治疗组、CsA治疗组总有效率分别为40.0%（12/30）和85.7%（18/21）。在MCD患儿中CsA治疗组有效率显著高于CTX治疗组（93.8%vs36.3%,P〈0.05）;而在FSGS患儿中CsA治疗组与CTX治疗组差异无统计学意义（75.0%vs50.0%,P〉0.05）。（2）无论在CTX治疗组或CsA治疗组,患儿的起病年龄、病理类型（MCD或FSGS）、临床分型（单纯型或肾炎型）均与疗效无显著相关（卡方检验,均P〉0.05）。（3）CTX治疗组合并肝功能损害、外周血白细胞减低、消化道副反应比例分别为10%,16.7%及33.3%,无患儿出现出血性膀胱炎。CsA治疗组出现多毛、齿龈增生、高血压比例分别为71.4%,23.8%及9.5%。2例出现神经系统副作用。2例患儿分别在1年和5年时进入终末期肾衰。结论：儿童激素耐药型肾病综合征的治疗中,激素联合免疫抑制剂CTX或CsA取得了较好的疗效。特别是在MCD患儿中,CsA组在随访12个月内的总有效率优于CTX组。

Objective To compare the therapeutic outcome of cyclophosphamide（CTX）and cyclosporine A（CsA）in the treatment of steroid-resistant idiopathic nephrotic syndrome（SRNS）in children.Methods Thirty-seven children with SRNS were analysed in a retrospective study from Jan 2001 to Dec.2006.There was initial renal histology of minimal change（MCD）in 28 children,focal segmental glomerulosclerosis（FSGS）in 7 and mesangial proliferative glomerulonephritis（MsPGN）in the other 2.These patients were divided into 2 groups：one group received the induction therapy consisting of intravenous CTX and prednisolon,and the another group were treated with CsA [initial dose 3-5mg/（kg·d）] and prednisolone.Results（1）Thirty children received CTX,while 21 received CsA,and the total efficacy was 40.0% and 85.7% respectively after the 12 month follow-up.Children with MCD receiving CsA had a better response than those treated with CTX（93.8% vs 36.3%,P〈0.05）.Children with FSGS receiving CsA did not show a significant difference compared with those treated with CTX（75.0% vs 50.0%,P〉0.05）.（2）There were no significant associations between age,pathology,clinic type and therapeutic outcome in the 2 groups（P〉0.05）.（3）The rate of liver functional impairment,leukocytopenia,vomiting and nausea was 10%,16.7% and 33.3%,respectively in children receiving CTX.The rate of hypertrichosis,gingival hyperplasia and hypertension was 71.4%,23.8% and 9.5% respectively in children receiving CsA.Two children had central adverse effect.Two patients with FSGS progressed into end-stage renal failure.Conclusion For children with MCD,CsA combining prednisolone could result in a higher remission rate than intravenous CTX combining prednisolone.